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  • 2019 Session | Hematological Manifestations of Immune Deficiencies and Dysregulatory Disorders: When should you look harder for underlying causes of Autoimmune Cytopenias?
  • 2019 Session | Oncofertility Programs: More Than Just Sperm Banking
  • 2019 Session | The (Financial) Cost of a "Cure"
  • 2019 Session | (2001) Potential Utility of MRD to Identify Relapse in pALL Patients Treated with Tisagenlecleucel
  • 2019 Session | (2002) Lentiglobin Gene Therapy in Transfusion-Dependent Β-Thalassemia Patients with Non-β0/β0 Genotypes
  • 2019 Session | (2003) Mechanisms of Resistance to the Type II JAK2 Inhibitor CHZ868 in B-Cell Acute Lymphoblastic Leukemia
  • 2019 Session | (2004) Discovering Noncoding Genetic Elements that Regulate Globin Synthesis
  • 2019 Session | (2005) Emapalumab in Pediatric Patients with Primary Hemophagocytic Lymphohistiocytosis: Safety & Efficacy
  • 2019 Session | (2006) Modeling IKZF1 Lesions in B-cell Acute Lymphoblastic Leukemia Reveals Potential Therapeutic Targets
  • 2019 Session | (2007) Prognostic Determinants in Childhood T-cell Acute Lymphoblastic leukemia: Results of DFCI 05001
  • 2019 Session | (2008) Gap Junction Interference increases Apoptosis in ALL cells and augments effects of Antimetabolites
  • 2019 Session | (2009) Hydroxyurea Lowers Tricuspid Regurgitant Jet Velocity in Children with Sickle Cell Anemia
  • 2019 Session | (2010) Novel Next-Generation Sequence based Assay for Non-Invasive Prenatal Testing of Sickle Cell Disease
  • 2019 Session | (2011) Gabapentin for Pain in Sickle Cell Disease: Results of a Randomized Phase II Clinical Trial
  • 2019 Session | (2012) Quality of Life is the most important Indication for Second-Line ITP Treatment in Children
  • 2019 Session | (2013) BCL2-Inhibitor Response in Neuroblastoma: Biomarkers and Therapy Resistance
  • 2019 Session | (2014) The Clinical Application of Molecular Testing in Pediatric Solid Tumors: An Institutional Experience
  • 2019 Session | (2015) Enhancing Tumor Directed T Cells with an Interleukin-7 Signal Modulator
  • 2019 Session | (2016) Epigenome Screening Identifies Transcriptional Elongation as Therapeutic Vulnerability in DIPG
  • 2019 Session | (2017) Levofloxacin Prophylaxis
  • 2019 Session | (2018) Renal Tumors: Future Roadmap
  • 2019 Session | (2019) Soft Tissue Sarcoma
  • 2019 Session | (2020) Realizing Effectiveness across Continents with Hydroxyurea (REACH): A Prospective Multi-National Trial of Hydroxyurea for Sickle Cell Anemia in Sub-Saharan Africa
  • 2019 Session | (2021) Leucine for the Treatment of Transfusion Dependence in Patients with Diamond Blackfan Anemia
  • 2019 Session | (2022) A Phase 1/2 Trial of Investigational Spk-8011 in Hemophilia a Demonstrates Durable Expression and Prevention of Bleeds
  • 2019 Session | (2023) Peripherally Inserted Central Catheters (PICCs), the Leading Cause of Central Venous Catheter Associated Thrombosis in Children
  • 2019 Session | The Role of Survivorship Research in Advancing Childhood Cancer Care and Quality of Survival
  • 2019 Session | (2024) Septic Shock (SS) Incidence in National ALL & AML Cohorts using Administrative & Clinical Trial Data
  • 2019 Session | (2025) Nelarabine Abrogates Relapse Rates in CNS-3 T-ALL: A Report from Children’s Oncology Group AALL0434
  • 2019 Session | (2026) Current Cancer Survivorship Practices: A Report from the Children’s Oncology Group (COG)
  • 2019 Session | (2027) Intra-Arterial Chemotherapy for Retinoblastoma: A 7-year Single Institution Experience
  • 2019 Session | Advancing Discovery in Hematology: Bench to Bedside? No. Bedside to Bench to Bedside...
  • 2019 Session | (2028) The Shwachman Diamond Syndrome Registry: Hematologic Complications
  • 2019 Session | (2029) Characteristics and Outcomes of Unprovoked Venous Thromboembolism in the Pediatric Population
  • 2019 Session | (2030) Third Party VST are an effective Therapy for the Treatment of BK-Virus Reactivation after Transplant
  • 2019 Session | (2031) Inhibition of Nemo-Like Kinase improves Erythropoiesis in Models of Diamond Blackfan Anemia
  • 2019 Annual Meeting Session Recordings
  • CI SIG Webinar
  • October 2021 eNews

The 2019 ASPHO Conference

May 1-4, 2019
New Orleans, LA
Ernest N. Morial Convention Center

Schedule of Events

All conference sessions are listed by day and time. Register online or contact Member Services at 847-375-4716 or This email address is being protected from spambots. You need JavaScript enabled to view it. .

The ASPHO 2019 Conference App will have real-time updates on all conference programs. To download the app, visit your app store and search "ASPHO Events." For BlackBerry, Windows, and other web browser-enabled devices, go to https://www.core-apps.com/dl/aspho2019 to be directed to the proper download version.

Wednesday | Thursday | Friday | Saturday

 

Wednesday, May 1

11:30 am – 12:30 pm

Clinical Conundrums

New this year – ASPHO is providing the opportunity to interact with a recognized expert on a challenging diagnostic and management issue. Each session is scheduled for 1 hour and will include a 15-20 minute presentation by the expert on their general approach to the clinical problem followed by small group discussion of additional cases shared by the attendees.

Advanced registration is required. Seating is limited to 20. The registration fee is $25.

(CC1) Refractory Immune Cytopenias
Jenny M. Despotovic, DO

(CC2) Relapsed Hodgkin Lymphoma
Paul D. Harker-Murray, MD PhD

No relevant financial relationships and no discussion of off-label use of a drug: Paul D. Harker-Murray
Relevant financial relationships; discussion of off-label drug use: Jenny M. Despotovic-Novartis: Research grant to my institution, PI; Amgen: Research grant to my institution, site investigator

11:30 am–12:45 pm

Non-CME Corporate Forum Luncheon Symposium: Role of IFNγ in Pathogenesis of Primary HLH: Preclinical Data and Translation to Clinical Practice

Learn more

Non-CME Corporate Forum Luncheon Symposium: Expert Perspectives: A Closer Look at a Treatment Option for Pediatric Immune Thrombocytopenia

Learn more

1:00–2:15 pm

Plenary Paper Sessions

The 2019 Conference Planning Committee will select abstracts considered the best submissions for the Plenary Paper Sessions.

Moderators: Donald L. Yee, MD MS; Valerie I. Brown, MD PhD

Objectives:

  1. Discuss new approaches related to diagnosis and treatment in pediatric hematology/oncology.
  2. Examine current research outcomes from a variety of the latest in basic and clinical research.

(2001) Potential Utility of MRD to Identify Relapse in pALL Patients Treated with Tisagenlecleucel
Michael A. Pulsipher, MD

(2002) Lentiglobin Gene Therapy in Transfusion-Dependent Β-Thalassemia Patients with Non-β0/β0 Genotypes
Timothy Olson, MD PhD

Relevant financial relationships to disclose and no discussion of off-label drug use: Michael A. Pulsipher - Novartis: Consulting, Study Steering Committee, Speaker, Honorarium; Adaptive: Consultant and Study support, Honorarium; Timothy Olson - Novartis: Advisory board member, hourly compensation for limited advisory board participation; Merck: Consultant, hourly compensation for 1 day SIE participation

Young Investigator Award Presentations

The ASPHO Young Investigator Award was established to formally recognize excellence in pediatric hematology/oncology research and to promote basic and clinical investigation into the fields of hematology and oncology by fellows and faculty members who are less than 4 years post-fellowship.

Moderators: Donald L. Yee, MD MS; Valerie I. Brown, MD PhD

(2003) Mechanisms of Resistance to the Type II JAK2 Inhibitor CHZ868 in B-Cell Acute Lymphoblastic Leukemia
Loretta Li, MD

Objectives: 

  1. Define mechanisms of acquired resistance to the type II JAK2 inhibitor CHZ868.

No relevant relationships and no discussion of off-label drug use: Loretta Li

(2004) Discovering Noncoding Genetic Elements that Regulate Globin Synthesis
Akshay Sharma, MBBS

Objectives:

  1. Discuss the transcription factors that regulate fetal hemoglobin expression.
  2. Generate a high-throughput approach for identifying previously unknown genomic regulatory elements.

No relevant relationships and no discussion of off-label drug use: Akshay Sharma 

Click here for more information about this year's award recipients.

The Early Career Travel Stipend recipients will also be announced during this time.

Epigenome Screening Identifies Transcriptional Elongation as Therapeutic Vulnerability in DIPG
Nathan Dahl, MD, University of Colorado School of Medicine, Denver, CO

BCL2-Inhibitor Response in Neuroblastoma: Biomarkers and Therapy Resistance
Claudia Zapata, MD, Children’s Hospital of Philadelphia, Philadelphia, PA

Third Party VST are an Effective Therapy for the Treatment of BK-Virus Reactivation after Transplant
Jeremy Rubinstein, MD PhD, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH

Circulating Hybrid Cells in Pediatric Patients with Glioma
Matthew Dietz, DO Med, Oregon Health & Science University, Portland, OR

Acute Kidney Injury after CD19-Targeted CAR T cell Therapy for Acute Lymphoblastic Leukemia
Regina Myers, MD, Children’s Hospital of Philadelphia, Philadelphia, PA

2:30–4:00 pm

(A1) Splenectomy - The Great Debate

Moderators: Fabienne L. Gray, MD; Jennifer Rothman, MD; Theodosia A. Kalfa, MD PhD

Objectives:

  1. Examine the current indications for splenectomy for ITP and CHA.
  2. Recognize the risks of splenectomy and assess how to weigh these risks with potential benefits of splenectomy for ITP and CHA versus alternative treatments.
  3. Analyze the current evidence for partial splenectomy in CHA.

For children with chronic or refractory immune thrombocytopenia (ITP) or congenital hemolytic anemias (CHA) of severe phenotype, splenectomy may prove invaluable in improving clinical outcomes. The decision to pursue splenectomy must be balanced with the risks associated with the procedure. In ITP, debate exists regarding splenectomy vs. use of newer second-line agents. In CHA, opinions vary regarding timing and degree of splenectomy (partial vs. total). Partial splenectomy is thought to preserve a degree of splenic function, thereby theoretically reducing the risk of long-term complications associated with total splenectomy. However, long-term data on outcomes of partial splenectomy is limited.

No relevant financial relationships and no discussion of off-label drug use: Fabienne L. Gray

Relevant financial relationships; no discussion of off-label drug use: Jennifer Rothman - Novartis/Pfizer/Agios Pharmaceuticals: Principal investigator, research grant to my Institution; Novartis/Pfizer: Advisory board member, honoraria; Theodosia L. Kalfa: Agios Pharmaceuticals, Inc.- Institution is site for clinical trial, research grant to my institution; FORMA Therapeutics, Inc.- Independent contractor, research grant to my institution

(A2) DICER1: Updates on Biology and Clinical Management

Moderator: Kris Ann Schultz, MD

Objectives:

  1. Describe the spectrum of DICER1-related tumors including recently described phenotypes.
  2. Discuss the role of DICER1 testing for individuals with various benign and malignant tumors.
  3. Discuss screening strategies and associated challenges of surveillance for individuals with DICER1 mutations.

An update on phenotypes associated with DICER1 syndrome as well as recent screening and management recommendations for individuals with DICER1 mutations will be interactively discussed during this session. Case presentations highlighting potential benefits and challenges of surveillance will be also be presented. Finally, emerging biologic information from ongoing research studies and tumor models will be discussed.

*DICER1-related tumors include PPB, SLCT, cystic nephroma, Wilms tumor, renal sarcoma, eRMS, pineoblastoma and thyroid cancer.

DICER1: Genetics, Pathophysiology and Testing
D. Ashley Hill, MD

Surveillance for DICER1-related Conditions
Junne Kamihara, MD PhD

No relevant financial relationships and no discussion of off-label drug use: Kris Ann Schultz, Junne Kamihara

Relevant financial relationships; no discussion of off-label drug use: D. Ashley Hill - ResourcePath LLC: Founder, ownership interest; Driver Inc: Independent contractor, logistic support of Beijing Children's Hospital PPB study

No CME will be offered for this session.

(A3) Burnout in Pediatric Hematology/Oncology: Moving from Discussion to Action

Moderators: Jonathan Fish, MD; Adit Tal, MD; Brittney Whitford, MD; Patricia Tritt, RN MA; Margaret Rea, PhD; Adam Levy, MD

Objectives:

  1. Recognize that burnout is prevalent, has an impact on patient care, physician wellness and health system functioning.
  2. Identify proactive forms of burnout prevention (such as strengthening resilience) and reactive forms of burnout prevention (such as critical incident stress management).
  3. Recognize that the stressors contributing to burnout among faculty and educators are different than those for trainees.
  4. Identify strategies to sustain physician health, work/life balance, and prevent burnout that can be brought back to each participant’s home institution.

Burnout among physicians is epidemic, and pediatric hematology/oncology has not been spared. While there is growing awareness of the impact of burnout on medicine, no standard approach has emerged to address its prevention, alleviation and management. Strategies remain highly variable between institutions and individuals.

In this workshop Jonathan Fish, MD, will present a very brief overview of burnout, after which participants will break into 2 groups where strategies to combat burnout will be discussed and developed. One group will focus on proactive and reactive strategies to burnout alleviation, and will be led by Margaret Rhea, PhD, an expert in resilience development and proactive approaches to burnout prevention, and Patricia Tritt, RN MA, an expert in addressing stress that arises following critical incidents. The second group will focus on addressing the unique factors that contribute to burnout from the educator and trainee perspectives, and will be led by Adam Levy, MD, chair of graduate medical education for Montefiore Medical Center, as well as Adit Tal, MD, and Brittney Whitford, MD, fellows in pediatric hematology/oncology. Half way through the workshop, participants will switch groups. Finally, all participants in the workshop will be invited to form a community of practice by continuing the conversation through different media platforms.

In advance of the conference, check out the following resources to help you prepare for the session:
Handout 1
Handout 2
Handout 3
Well-Being ASPHO SIG Petition

Recommended by the Professional Development Committee

No relevant financial relationships and no discussion of off-label drug use: Jonathan Fish; Adit Tal; Patricia Tritt; Margaret Rea; Adam Levy; Brittney Whitford

4:00–4:30 pm

Networking Break

 

4:30–6:00 pm

(A4) Diagnostic and Management Conundrums Surrounding Women with Heavy Menstrual Bleeding and Underlying Bleeding Disorders

Moderator: Ayesha Zia, MD

Objectives:

  1. Differentiate normal from heavy menstrual bleeding (HMB), classify abnormal menstrual patterns according to recently proposed terminologies, and recognize predictors of bleeding disorders.
  2. Identify the type and timing of laboratory evaluation required to optimally diagnose bleeding disorders in adolescents presenting with HMB.
  3. Recognize the genetic ramifications and management considerations for adolescents with decreased von Willebrand levels or hemophilia carriership.

Heavy menstrual bleeding (HMB) at menarche is attributed to anovulation. Intuitively, a proportion of adolescents should have an underlying bleeding disorder (BD), but the age of women identified with BD is 35 years, implying that the diagnosis is made late. Management is symptomatic, not attempting to identify the underlying etiology. This workshop, relevant for small and medium sized programs without dedicated Young Women’s Hematology Clinics, will discuss diagnostic conundrums surrounding HMB and management of BDs.

Heavy Menstrual Bleeding in Adolescents: To Test or Not to Test
Ayesha Zia, MD

When is Heavy Menstrual Bleeding in Adolescents Really Heavy?
Sarah H. O'Brien, MD MSC

Low VWF and Hemophilia Carriership: Medical or Genetic Diagnosis?
Robert F. Sidonio, MD MSc

No relevant financial relationships and no discussion of off-label drug use: Ayesha Zia; Sarah H. O’Brien

Relevant financial relationships to disclose; no discussion of off-label drug use: Robert F. Sidonio - Shire/Novo Nordisk/Uniqure/Biomarin/CSL Behring/Genentech: Advisory board member, honoraria; Shire/Bioverativ/Grifols/Kedrion/Genentech: Principal investigator, research grant to my institution

(A5) All That Glitters is Not Gold – How We Differentiate Primary HLH from Fake News

Moderators: Ashish Kumar, MD PhD; Arun Gurunathan, MD; Seth Rotz; Philip Roehrs, MD

Objectives:

  1. Describe the limitations of the HLH-2004 diagnostic criteria.
  2. Identify mimickers, and atypical clinical presentations of HLH.
  3. Discuss improved diagnostic approaches to primary HLH.

Hemophagocytic lymphohistiocytosis (HLH) is characterized by dysregulated immune activation. Primary HLH involves hereditary defects in cytotoxic lymphocytes while secondary HLH is triggered by extrinsic factors. The HLH-2004 criteria are widely used for diagnosis, yet their specificity for HLH, and ability to differentiate primary from secondary disease is unclear. This distinction is critical when a malignancy is the trigger of immune activation. Failure to recognize and treat these conditions can lead to misdirected treatment and fatal outcomes. Such errors can be reduced with increased awareness, appropriate investigations, and use of an alternative diagnostic approach using flow cytometry-based assays.

No relevant financial relationships and no discussion of off-label drug use: Arun Gurunathan; Philip Roehrs

No relevant financial relationships; discussion of off-label drug use: Seth Rotz

Relevant financial relationship and no discussion of off-label drug use: Ashish Kumar-Novimmune: Consultant, honoraria

(A6) Models for International Pediatric Oncology Outreach

Moderators: Judith F. Margolin, MD; David G. Poplack, MD; Scott Macfarlane, MD; Carlos Rodriguez-Galindo, MD; Joseph Lubega, MD; Julia M. Challinor, PhD RN

Objectives:

  1. Identify and discuss the vast difference in diagnosis and cure rates between HIC and LMIC settings.
  2. Describe the impediments to making accurate diagnoses and providing comprehensive care in LMIC settings.
  3. Describe the successful models for performing International Outreach.

While the cure rates for Pediatric Cancer in High Income Countries (HICs) are now > 80%, over 80% of the children with cancer live in Low and Middle Income countries (LMICs), where many are never accurately diagnosed or fully treated. When these children are properly diagnosed, the difficulties of providing appropriate treatment and supportive care result in cure rates that are far less than half of what would be expected in HIC.

No relevant financial relationships and no discussion of off-label drug use: Judith F. Margolin; Scott Macfarlane; David G. Poplack; Carlos Rodriguez-Galindo; Julia M. Challinor; Joseph Lubego

6:00 – 6:45 pm

New Member and International and First-time Attendee Reception

Join fellow new members, first-time attendees, and international attendees during this opportunity to connect and network at the start of the ASPHO Conference. ASPHO Board President Patrick Leavey, MD, will make short remarks and ASPHO’s Board members will be in attendance to greet attendees.

 

7:00 – 9:00 pm

Division Directors Dinner Meeting (DDM)

Join fellow Division Directors for an opportunity to network and connect around topics important to your work.

Advanced registration is required. The registration fee is $100.

Thursday, May 2

6:30–8:15 am

Non-CME Corporate Breakfast Symposium: Applications of NGS & PGx testing in advancing precision medicine in pediatric oncology

Learn more

6:30–7:30 am

5K Fun Run/Walk

ASPHO is holding its sixth annual 5K Fun Run/Walk event.

Advanced registration is recommended. Registration fee is $25, after May 1, fee is $35.

7:00–8:00 am

Adolescent and Young Adult Hematology/Oncology Special Interest Group

Leadership: Stefanie Thomas, MD MS; Jacquelyn Baskin, MD MSc
Speakers: Karen Albritton, MD; Jacquelyn Baskin, MD MSc; Alison Grimes, MD; Amy Sobota, MD MPH; Stefanie Thomas, MD MS 

Understanding and Overcoming Barriers to Hematology/Oncology AYA Program Development
Dr. Karen Albritton, medical director of the Fort Worth AYA Oncology Coalition, will discuss lessons learned through the development of a multi-institutional AYA program. Additionally, we will report results of a landscape survey addressing current hematology/oncology AYA care and workshop attendees will participate in defining next-steps for the SIG.

Diversity Special Interest Group

Leadership: Kerice Pinkney, MD; Pinki Prasad, MD MPH
Speaker: Dr. Bonnie C. Desselle, MD

Diversity in Medicine

Physician Scientist Special Interest Group

Leadership: Kathleen M. Sakamoto MD PhD; Sinisa Dovat, MD PhD 
Speakers: Kathleen Sakamoto, MD PhD, Ernest Frugé, PhD, and Sinisa Dovat, MD PhD

Strategies for Research Funding
Kathleen Sakamoto, MD, PhD, Ernest Frugé, PhD, and Sinisa Dovat, MD PhD, will present the survey results of the physician scientist SIG members and Jeffrey Lipton, MD PhD, will discuss various strategies for obtaining research funding, a priority issue identified by the SIG members.

Vascular Anomalies Special Interest Group

Leadership: Francine Blei, MD; Ionela Iacobas, MD 

Like it or Not, Now you Have to Learn about Vascular Anomalies: It's in the Boards!
This meeting will provide updates from Research, Practice and Education Committees and review of selected difficult cases. We will also review opportunities to become involved in this exciting community, including ongoing and upcoming research. We welcome trainees and fellowship program representatives, as vascular tumors and malformations are now included in the 2018 ABP Content Outline for the Pediatric Hematology-Oncology Boards.

8:15 – 9:15 am

(B7) Navigating Genetic Testing in the Pediatric Hematology Setting

Moderators: Stefanie Dugan, MS CGC; Katie Bergstrom, MS CGC

Objectives:

  1. Identify the optimal testing strategy in real-life clinical scenarios and recognize advantages and limitations of genetic testing in clinical pediatric hematology.
  2. Appreciate implications of complex genomic tests to patients and families.
  3. Assess genetic test results and understand variant classification.

Addressing frequent and often complex genetics questions such as recognition of hereditary disorders, determination of familial recurrence risks, and implication of genetic test results can pose a challenge to clinical pediatric hematology providers, especially in small or medium-sized programs without the support of a genetic counselor. In the era of genomic medicine, with increased availability and clinical utility of genetic testing, providers need to be equipped with tools and resources to successfully navigate these issues with their patients in the pediatric hematology clinic. In this session certified genetic counselors with expertise in clinical pediatric and laboratory hematology genetics will share cases (hemophilia, thrombophilia, hereditary thrombocytopenia, and bone marrow failure) to help audience members optimize testing strategy, educate patients on the complexity and nuances of testing, and integrate even inconclusive results into patient care.

No relevant financial relationships; no discussion of off-label drug use: Katie Bergstrom

Relevant financial relationships to disclose; no discussion of off-label drug use: Stefanie Dugan – BloodCenter of Wisconsin: Employee, salary

(B8) Radiation Therapy for Pediatric Cancer Patients: Updates and New Modalities

Moderators: Andrew W. Walter, MD; Tom Merchant, DO PhD

Objectives: 

  1. Explain the indications and differences in radiation therapy delivery methods and how to best utilize these technologies for patients.
  2. Describe the use of a radioisotope to treat large or unresectable liver cancers.

Radiation therapy plays a critical role in the treatment and cure of many childhood cancers. Pediatric oncologists may not be fully aware of the pros/cons of the different delivery methods including protons, photons, radiopharmaceuticals and specialized techniques including gamma knife, radiosurgery and stereotactic body radiotherapy. This session will examine these issues for the pediatric oncology audience.

The Puzzle of Proton Therapy: Opportunities and Obligations in Pediatrics
Christine Hill-Kayser, MD

Pediatric Radiosurgery
Erin Murphy, MD

Transarterial Radioembolization with Yttrium-90 of Unresectable Pediatric Primary Liver Malignancy
Allison Aguado, MD

No relevant financial relationships and no discussion of off-label drug use: Andrew W. Walter; Tom Merchant; Christine Hill-Kayser; Erin Murphy

No relevant financial relationships to disclose and no discussion of off-label drug use: Allison Aguado

(B9) The Microbiome: What Is It and Why Do We Care?

Moderators: Valerie I. Brown, MD PhD; Michael Silverman, MD PhD; Matthew Kelly, MD MPH

Objectives: 

  1. Discuss how exposure to chemotherapy and antibiotics can alter a host’s microbiome.
  2. Recognize that the microbiota composition of pre-HCT is distinct from healthy volunteers and this difference may impact the risk for infections and GVHD post-HCT.
  3. Explain how the modulation of microbiome may affect patients’ outcomes.

The microbiome plays critical roles within the context of the development of the immune system. It can alter how a patient will respond to chemotherapy or immunotherapy, and can impact post-transplant morbidity and mortality; all through its ability to educate and modulate the host immune system. The clinician need to be aware of the impact of these interactions to make informed decisions regarding the use of probiotics, antibiotics, and immunosuppressant medications.

No relevant financial relationships and no discussion of off-label drug use: Valerie I. Brown; Michael Silverman; Matthew Kelly

9:15–9:45 am

Networking Break

Genius Bar

Join us in the foyer on the 3rd floor and bring your cases on Radiation Therapy. Speakers from the “Radiation Therapy for Pediatric Cancer Patients: Updates and New Modalities” session will be there to help answer questions and guide the discussion.

 

9:45 - 11:15 am

(B10) Brain Attack: Therapeutic Advances in Neonatal and Childhood Stroke

Objectives:

  1. Recognize the common presenting signs of stroke based on age, risk factors and predisposing disease states (sickle cell disease, cardiac disease, nephrosis, autoimmune syndromes…etc.), and plan the diagnostic evaluation of a neonatal and pediatric stroke patient.
  2. Identify the indications for anticoagulation and antiplatelet therapy, and recognize the emerging role of and controversies surrounding hyperacute therapies including thrombolysis, thrombectomy and clot retrieval in pediatric arterial ischemic stroke.
  3. Describe the importance of a multidisciplinary approach for the early recognition, diagnosis and management of pediatric stroke patients.

The past decade has marked an increase in incidence and awareness of childhood and neonatal stroke. Albeit rare, pediatric stroke associates with significant morbidity and mortality, and a compelling need exists for early recognition, diagnosis and treatment. Using an interactive review of actual cases, this workshop will address the clinical presentations, differential diagnoses and existing challenges/delays in diagnosis of pediatric stroke in general and in high-risk patient populations, including sickle cell anemia, cardiac disease, nephrosis and autoimmune syndromes. The panelists will also provide a comprehensive review of existing therapies and emerging data on hyperacute interventions such as thrombolysis and thrombectomy.

Pediatric Stroke: A Neurologist's Perspective
Jessica L. Carpenter, MD

Pediatric Stroke: A Hematologist's Perspective

Nihal Bakeer, MD

New Insights into Sickle Cell Disease and Strokes
Monica L. Hulbert, MD

Stroke in Special Pediatric Populations
Yaser Diab, MBBS

No relevant financial relationships and no discussion of off-label drug use: Jessica L. Carpenter

No relevant financial relationships; discussion of off-label drug use: Nihal Bakeer, Yaser Diab

Relevant financial relationships to disclose; discussion of off-label drug use: Monica L. Hulbert - Pfizer, Inc: Employee, salary (family); Global Blood Therapeutics: Study investigator, research grant to my institution

(B11) Advances in Pediatric AML

Moderators: Nobuko Hijiya, MD; E. Anders Kolb, MD; Soheil Meshinchi, MD PhD; Sarah K. Tasian, MD

Objectives:

  1. Discuss the recent clinical trial findings and plan for future trials (and incorporation of new agents).
  2. Describe the biologic advances in pediatric AML, including results of the TARGET project.
  3. Summarize the development of immunotherapies for AML, including DART and CART.

The outcomes for pediatric AML remain relatively poor compared to other hematologic malignancies, with EFS of about 50%. To this end, much research is ongoing including the recent TARGET project, as well as introduction of targeted therapies into treatment regimens, and the development of immunotherapy. These advancements both inform prognosis and therapeutic decisions, and lead to improved patient outcomes. Immunotherapy strategies have not yet been widely adapted for AML, but are likely to be in clinical trials soon, and important for practitioners to be aware of.

No relevant financial relationships and no discussion of off-label drug use: E. Anders Kolb; Soheil Meshinchi; Sarah K. Tasian

Relevant financial relationships to disclose; no discussion of off-label drug use: Nobuko Hijiya - Novartis: Data monitoring committee, honoraria

(B12) Improving Medication Adherence for Adolescents and Young Adults: Is Technology the Answer?

Moderator: Amy Sobota, MD MPH

Objectives:

  1. Describe facilitators of and barriers to medication adherence that are specific in AYA patients.
  2. Compare existing technology based interventions (mobile health applications and websites) for use in clinical practice.
  3. Apply motivational interviewing techniques to improve medication adherence among adolescent and young adult patients regardless of diagnosis.

Lower rates of medication adherence among adolescents and young adults (AYA) contribute to less favorable outcomes compared to younger hematology/oncology patients. Improving adherence requires tools and techniques tailored to the unique needs of this population. Both technology and motivational interviewing have shown promise in engaging AYA, but are not widely used. This workshop will allow participants to test existing mobile health applications and learn evidence based interviewing techniques they can apply to their practice.

Hydroxyurea Adherence in Sickle Cell Disease
Jane S. Hankins, MD MS

Teens with Haemophilia
Vicky R. Breakey, MD

AYA with Cancer
Jennifer Stinson, PhD RN

No relevant financial relationships; no discussion of off-label drug use: Amy Sobota; Vicky R. Breakey; Jennifer Stinson

Relevant financial relationships to disclose; no discussion of off-label drug use: Jane S. Hankins - Bluebird Bio/NCQA: Consultant, honoraria; Global Blood Therapeutics/Novartis: Principal investigator, Research grant to my institution

11:30 am–12:20 pm

Business Meeting

ASPHO’s annual business meeting allows attendees to learn more about the Society’s progress toward meeting the goals of its strategic plan, as well as learn about the Society’s financial health. Additionally, outgoing Board members and committee chairs are recognized, a report is presented from the Pediatric Blood & Cancer Journal editor, and the 5K Fun Run winners are recognized. 

12:30–1:45 pm

Early Career Roundtable Luncheon

Moderator: Thomas Russell, MD

Objectives:

  1. Discuss pathways to successfully achieve career development goals.
  2. Review the experiences of experts in the field.
  3. Provide trainees and early career members an opportunity to network with leaders in the field of pediatric hematology/oncology.

Attendees will have the opportunity to participate in discussion groups led by experts in defined areas of interest. The purpose of this luncheon is to provide early-career professionals a forum to discuss issues one on one with leaders in the field, ask questions related to career development in a small-group setting, and receive positive reinforcement regarding career goals specific to their interests. One to two discussion leaders will be placed at each luncheon table, with the option for participants to switch tables for further discussion on a wide range of topics.

Advanced registration is required. The registration fee is $30.

Recommended by the Professional Development Committee.

Basic Science/Translational Research
Daniel S. Wechsler, MD PhD; Kathleen M. Sakamoto, MD PhD

Clinician Educator
Timothy P. Garrington, MD; Caroline A. Hastings, MD; Jennifer C. Kesselheim, MD MEd

Clinical Research—Hematology
Jeffrey M. Lipton, MD PhD; George R. Buchanan, MD 

Clinical Research—Oncology
William G. Woods, MD; Mark Atlas, MD; Pinki Prasad, MD MPH

Medical Students and Residents
Thomas Russell, MD; Kimberly Whelan, MD MSPH; Maria C. Velez, MD

Cell and Gene Therapy
Erica Esrick, MD

Pharmaceutical Industry
Kerri Nottage, MD

Advanced registration is required. The registration fee is $30.

Recommended by the Professional Development Committee.

No relevant financial relationships; no discussion of off-label drug use: George Buchanan; Thomas Russell; Kathleen M. Sakamoto; Daniel S. Wechsler; Timothy P. Garrington; Caroline A. Hastings; Jennifer C. Kesselheim; Jeffery M. Lipton; William G. Woods; Mark Atlas; Pinki Prasad; Kimberly Whelan; Erica Esrick

Relevant financial relationships and no discussion of off-label drug use: Maria Velez - Novo Nordisk: Principal investigator, research grant to my institution; Novo Nordisk: Speaker bureau, honorarium

Relevant financial relationships; discussion of off-label drug use: Kerri Nottage - Janssen: stock holder, stock options; Janssen: employment, salary

12:30–1:30 pm

Non-CME Corporate Forum Luncheon Symposium: A Novel Approach to Prophylaxis for Hemophilia A Patients

Learn more

Non-CME Corporate Forum Luncheon Symposium: A New Treatment for TRK Fusion Cancer

Learn more

12:30–1:30 pm

Palliative Care Special Interest Group

Leadership: Lawrence Wolfe, MD; Julienne Brackett, MD

This session will include a "State of the Science" review of recent key palliative care articles. We will discuss clinical problems, educational issues and growth of the field. We will also discuss how to keep the SIG active between meetings and increase its usefulness to members.

Quality Improvement and Patient Safety Special Interest Group

Leadership: Meghan Drayton Jackson, DO MBOE CPPS; Christine Moore Smith, MD

This session will begin with a lecture on “The History and Current State of Quality Improvement within Pediatric Hematology/Oncology” followed by a moderated panel discussing multiple themes (career development, mentorship, resources/QI tools, funding). Interactive modalities will be used to find out more about the audience’s goals for the SIG.

Speaker: Jeffrey Hord, MD
Panel Moderator: Christine Moore Smith, MD
Panel Members: Jeffrey Hord MD; Christopher E. Dandoy, MD MSc; Joanna Newton, MD MSc; Tiffany Lin, MD; Meghan Drayton Jackson, DO MBOE CPPS

Small Program Special Interest Group

Leadership: Andrea M. Watson, MD, MS; Jamie Dargart, MD

Small Program, Big Patients: Caring for Adolescents and Young Adults at Smaller Centers
Small programs are uniquely positioned to care for adolescents and young adults in collaboration with our adult colleagues. However, this population brings different needs and challenges than our younger patients. A panel of AYA experts in hematology and oncology will join us to discuss the care of AYA patients at smaller centers.

Speakers:
Jacquelyn Baskin, MD MSc, Children’s Hospital Los Angeles
Allison Grimes, MD, University of Texas Health Science Center at San Antonio
Aniket Saha, MD MS, Children’s Hospital of Greenville Health System
Amy Sobota, MD MPH, Boston Medical Center
Stefanie Thomas, MD, Children’s Hospital Los Angeles

2:00–3:00 pm

Northwestern Mutual Award for Excellence in Childhood Cancer Survivorship Announcement

ASPHO will present the Northwestern Mutual Award for Excellence in Childhood Cancer Survivorship, funded by the Northwestern Mutual Foundation. The award will recognize an individual who has translated research findings into intervention-based approaches and/or has made outstanding contributions to the clinical care of survivors of childhood cancer.

Funded by: Northwestern Mutual Foundation

Frank A. Oski Memorial Lectureship Award Presentation

The annual Frank A. Oski Memorial Lectureship Award gives us an opportunity to remember and honor Dr. Oski and his many contributions to the field of pediatric hematology/oncology. It is awarded to an outstanding clinical or laboratory investigator in pediatric hematology/oncology whose cutting-edge research has significantly impacted the field. This year's lectureship will be presented by Wilbur A. Lam, MD PhD, Aflac Cancer and Blood Disorders Center/Emory University.

Development and Clinical Translation of Engineered Microsystems for Hematologic Applications

Objectives

  1. Recognize the research-enabling capabilities and diagnostic potential of microsystems-based technologies for hematologic applications
  2. Explain how the biophysical properties of blood cells and their microenvironment affect hematologic processes and the pathophysiology of various blood disorder

Wilbur A. Lam, MD, PhD is a physician-scientist-engineer whose basic research interests involve developing micro/nanotechnologies to investigate the biophysics of hematologic processes at the micro- to nanoscales. Accordingly, the Lam laboratory takes a multidisciplinary approach spanning medicine, cellular biology, physics, and engineering to develop research enabling tools such as microfluidic and microchip-based systems to answer questions in hematology that are technologically infeasible with current assays. Examples include “microvasculature-on-a-chip” systems that function as in vitro models of sickle cell disease or hemostasis/thrombosis. The Lam laboratory is also dedicated to clinically translating the technologies they invent, including home-based anemia tests and several smartphone-based diagnostic platforms as well as novel drug delivery systems for blood diseases. Overall, the Lam laboratory has developed a “basement-to-bench-to-bedside” approach in which the invention, development, and clinical translation of micro/nanotechnologies takes place under one scientific “roof” with the ultimate goal of improving the lives of children and adolescents with hematologic disorders.

Relevant financial relationships and no discussion of off-label drug use: Wilbur A. Lam - Sanguina, LLC: Founder, stock

Click here for more information about this year's recipient.

3:15–4:15 pm

Concurrent Paper Sessions 

Leukemia
Moderators: Nobuko Hijiya, MD; Meret Henry, MD MS

Objectives:

  1. Discuss new approaches related to diagnosis and treatment in pediatric hematology/oncology.
  2. Examine current research outcomes from a variety of the latest in basic and clinical research.

(2005) Emapalumab in Pediatric Patients with Primary Hemophagocytic Lymphohistiocytosis: Safety & Efficacy
Carl Allen, MD PhD

(2006) Modeling IKZF1 Lesions in B-cell Acute Lymphoblastic Leukemia Reveals Potential Therapeutic Targets
Rohit Gupta, MD

(2007) Prognostic Determinants in Childhood T-cell Acute Lymphoblastic leukemia: Results of DFCI 05001
Melissa Burns, MD

(2008) Gap Junction Interference increases Apoptosis in ALL cells and augments effects of Antimetabolites
Craig Mullen, MD PhD

No relevant financial relationships and no discussion of off-label drug use: Carl Allen; Rohit Gupta; Melissa Burns; Craig Mullen

Hematology
Moderators: Shelley Crary, MD; Caitlin Neri, MD MPH

Objectives:

  1. Discuss new approaches related to diagnosis and treatment in pediatric hematology/oncology.
  2. Examine current research outcomes from a variety of the latest in basic and clinical research.

(2009) Hydroxyurea Lowers Tricuspid Regurgitant Jet Velocity in Children with Sickle Cell Anemia
Parul Rai, MD

(2010) Novel Next-Generation Sequence based Assay for Non-Invasive Prenatal Testing of Sickle Cell Disease
Nelda Itzep, MD

(2011) Gabapentin for Pain in Sickle Cell Disease: Results of a Randomized Phase II Clinical Trial
Latika Puri, MD

(2012) Quality of Life is the most important Indication for Second-Line ITP Treatment in Children
Kristin Shimano, MD

No relevant financial relationships and no discussion of off-label drug use: Parul Rai; Nelda Itzep; Latika Puri

Relevant financial relationships; discussion of off-label drug use: Kristin Shimano – Novartis/Pfizer: Principal investigator, research grant to my institution

Solid Tumor
Moderators: Jessica Heath, MD; Karen E. Effinger, MD MS

Objectives:

  1. Discuss new approaches related to diagnosis and treatment in pediatric hematology/oncology.
  2. Examine current research outcomes from a variety of the latest in basic and clinical research.

(2013) BCL2-Inhibitor Response in Neuroblastoma: Biomarkers and Therapy Resistance
Claudia Zapata, MD

(2014) The Clinical Application of Molecular Testing in Pediatric Solid Tumors: An Institutional Experience
Laura Wiltsie, DO

(2015) Enhancing Tumor Directed T Cells with an Interleukin-7 Signal Modulator
Bilal Omer, MD

(2016) Epigenome Screening Identifies Transcriptional Elongation as Therapeutic Vulnerability in DIPG
Nathan Dahl, MD

No relevant financial relationships and no discussion of off-label drug use: Claudia Zapata; Laura Wiltsie; Bilal Omer

No relevant financial relationships; discussion of off-label drug use: Nathan Dahl

4:30–5:30 pm

(B13) Metabolism in Pediatric Hematology/Oncology - Presidential Symposium

Moderator: Patrick Leavey, MD

Objectives: 

  1. Describe the importance of metabolism to cancer and opportunity to consider metabolic pathways as potentially targetable.
  2. Explain that mitochondria is more than responsible for creating ATP and understand its role in cellular/leukemia fate.
  3. Discuss the work being performed in treating patients with pyruvate kinase deficiency.

Cellular metabolism has an unquestionable role in phenotypic diversity and maintenance of tissue function. We will present three talks that will elucidate the importance of metabolism in cell function, the opportunity to explore the potential for vulnerabilities in metabolism in cancer, the role of mitochondria in leukemia cell fate and the exciting work being done to clinically target metabolic deficiencies in pediatric hematological disease.

Metabolic Reprogramming in Human Tumors in Vivo
Ralph deBerardinis, MD PhD

Mitochondria Control Physiology and Disease: Beyond ATP
Navdeep S. Chandel, PhD

Clinical Research Progress in Pyruvate Kinase Deficiency
Rachael F. Grace, MD MMSc

No relevant financial relationships; no discussion of off-label drug use: Patrick Leavey, Navdeep S. Chandel

Relevant financial relationships to disclose; no discussion of off-label drug use: Ralph deBerardinis - Agios: Advisory board member, stock options

Relevant financial relationships to disclose; discussion of off-label drug use: Rachael F. Grace - Agios Pharmaceuticals: Advisory board member, honoraria; Agios Pharmaceuticals: Research funding, research grant to my institution

5:30–7:00pm

Opening Reception with Exhibits and Posters

 

5:30–6:30 pm

Author-Attended Poster Session A: Odd-numbered Posters

 

5:30–6:30 pm

Meet the SIGs

 

5:40 - 5:55 pm

Product Demonstration: KARE (Knowledge and Assessment REsources)

Learn about ASPHO's new line of educational products. 

5:45–6:15 pm

Even-Numbered Poster Tour 

ASPHO will provide two poster tours during exhibit hours. The Conference Planning Committee selected five highly ranked posters that will be included in a 30-minute tour for all interested attendees. Authors of these selected posters will provide a brief overview of their research and answer attendees’ questions. Advanced registration is not required. Attendees should meet in the back of Exhibit Hall and look for a staff member holding a Poster Tour sign. Don’t miss the opportunity to receive a quick overview of top-rated abstracts!

Meet in the back of the Exhibit Hall.

The following posters will be featured during this tour:

(#046) TRANSCRIPTIONAL AND POST-TRANSCRIPTIONAL BIOMARKERS FOR MINIMAL RESIDUAL DISEASE IN PEDIATRIC AML
Liron J. Grossmann, MD

(#208) PLASMA CELL-FREE DNA FOR NONINVASIVE MOLECULAR DIAGNOSTICS IN WILMS TUMOR PATIENTS
Prachi Kothari

(#228) TUMOR MARKER SURVEILLANCE FOR EARLY DETECTION OF RELAPSE IN PEDIATRIC EXTRACRANIAL SOLID TUMORS
Rachana Shah, MD MS

(#230) CLINICAL CHARACTERIZATION OF LONG-TERM OUTCOMES IN PEDIATRIC EPITHELIOID HEMANGIOENDOTHELIOMA
Eily Cournoyer, MSc

(#824) COAGULOPATHY AND RELATED COMPLICATIONS FOLLOWING SCLEROTHERAPY OF CONGENITAL VENOUS MALFORMATIONS
Rachel Swerdlin

6:30 - 6:45 pm

Awardee Spotlight @ ASPHO Booth with 2019 ASPHO Frank A. Oski Award recipient Wilbur Lam, MD PhD

 

7:15 - 8:45 pm

Fellowship Program Directors Dinner Meeting (PDD)

Join fellow Fellowship Program Directors for an opportunity to network and connect around topics important to your work.

Advanced registration is required. The registration fee is $85.

 

Friday, May 3

6:30–8:15 am

Continental Breakfast with Exhibits and Posters

 

Non-CME Breakfast Symposium: Sickle Cell Disease: The Impact of Chronic Anemia and Hemolysis and Emerging Therapeutic Options

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7:00 am–1:30 pm

Exhibits and Posters in Exhibit Hall

 

8:15–9:00 am

George R. Buchanan Lectureship Award Presentation

The annual George R. Buchanan Lectureship is presented in honor of Dr. Buchanan and his many contributions to ASPHO and the field of pediatric hematology/oncology. It is awarded to an internationally recognized expert in pediatric hematology/oncology. This year's lecture will be presented by Kenneth McClain, MD PhD from Texas Children's Hospital.

An Oncogene-Driven Orphan Disease: A Short History of Langerhans Cell Histiocytosis

Objectives

  1. Provide the listeners with an overview of how the biology of Langerhans cell histiocytosis has evolved over the past 40 years.
  2. Illustrate a model for studying rare diseases through a designated center of excellence.

Translational research has several barriers which may inhibit success: sufficient numbers of patients to adequately study a disease, technological innovations to uncover the pathophysiology, funding to support the research, and motivated investigators. Contrasts in the trajectory of progress for acute lymphoblastic leukemia (ALL) vs Langerhans cell histiocytosis (LCH) could not be more striking. The organization of cooperative groups to treat ALL led to pooling of thousands of patients and collection of critical specimens for research. Cell surface marker and cytogenetic data quickly led to risk stratification and improved outcomes with treatment modifications. Many impressive molecular discoveries over the past few years have profoundly advanced our understanding of ALL biology. LCH research suffered from a lack of organized tissue acquisition in the international trials and paucity of patients at any one center anywhere in the world. There are no cytogenetic abnormalities in LCH cells. Evaluations of LCH tissue were challenged by lack of technical innovations to separate the pathologic cells from others in biopsy tissue and isolate sufficient amounts of DNA and RNA from purified cells to do molecular research until 2006. Since then advances in our understanding of LCH biology have been impressive, but much is yet to be learned. This lecture will provide an overview about early efforts led to intriguing, but incomplete results and how it was possible to acquire sufficient numbers of patients at a single center, establish critical collaborations, and begin to bring LCH research into the 21st century.

No relevant financial relationship; discussion of off-label drug use: Kenneth L. McClain

9:15–10:15 am

(C14) Too Much of a Good Thing – Myeloproliferation in Children

Moderator: Nicole Kucine, MD MS

Objectives:

  1. Describe the causes of thrombocytosis in children and describe the workup and management.
  2. Recognize the primary and secondary causes of polycythemia in children and review treatment strategies.
  3. Discuss the causes, work-up, and care of children with eosinophilia. 

There is a lack of knowledge surrounding myeloproliferative conditions in children, likely because they are rare in the pediatric population. Anyone who has prepared to see a patient with eosinophilia, or with Budd-Chiari syndrome and a platelet count of 800,000, recognizes how little information is available regarding these disorders in children. This session will give providers the tools they need to feel confident when working-up and treating a child with thrombocytosis, polycythemia, or eosinophilia.

Polycythemia - Diagnostic Work-Up and Management
Nicole Kucine, MD MS

Thrombocytosis - When it's More Than Reactive
Michele P. Lambert, MD MSTR

Approach to the Child with Eosinophilia
Amy Klion, MD

No relevant financial relationships; discussion of off-label drug use: Nicole Kucine, Amy Klion

Relevant financial relationships to disclose; discussion of off-label drug use: Michele Lambert- Novartis/Educational Concepts in Medicine/Shionogi/Sysmex: Consultant, honoraria;
Bayer: Advisory board member, honoraria; Astra Zeneca: Principal investigator, research grant to my institution; CSL Behring/Rigel: Advisory board member, no compensation received

(C15) Multiple Bony Lytic Lesions, but Not Langerhans Cell Histiocytosis (LCH) – Now What?

Moderator: Ionela Iacobas, MD

Objectives:

  1. Formulate the differential diagnosis of multiple bony lytic lesions (solid tumor metastases, histiocytosis (LCH), Gorham-Stout disease or another complex lymphatic anomaly, epithelioid hemangioma) and define required radiological imaging to assess disease extent.
  2. Create a reflex step-wise immunostaining algorithm including the differential diagnosis entities, to maximize use of the biopsied specimen.
  3. Outline the natural history of progression if left untreated, specific to each possible diagnosis. Summarize the management of multifocal bony lytic lesions (disease targeted therapy and “bone protection” agents – bisphosphonates and denosumab).

Multifocal bony lytic lesions as a presentation sign in a pediatric patient opens a wide-range of differential diagnoses: histiocytosis, bone metastases from an unknown source, other primary malignancies (like leukemia or lymphoma), or vascular anomalies affecting the bones. Reaching the correct diagnosis through the least number of invasive or costly procedures is a very difficult task. Once the diagnosis is identified, the therapy should target the etiology but also specifically address the bone lesions to prevent pathological fractures and long-term morbidity.

Four Clinical Cases of Multifocal Bony Disease - What Next?
Ionela Iacobas, MD

Radiologic Differential Diagnosis of Multifocal Bone Lytic Lesions
Arnold C. Merrow, MD

What Clinical Information Does the Pathologist Need to Get to the Right Diagnosis?
Jonathan L. Metts MD

Targeted Medical Therapy for Multifocal Bone Lytic Lesions Depending on Etiology
Michael Briones, DO

No relevant financial relationships and no discussion of off-label drug use: Arnold C. Merrow

No relevant financial relationships; discussion of off-label drug use: Ionela Iacobas, Michael Briones

Relevant financial relationships to disclose; no discussion of off-label drug use: Jonathan L. Metts - Celgene Corporation: Principal investigator, research grant to my institution

(C16) Savior Siblings: Medical, Ethical, and Psychosocial Considerations in using Preimplantation Genetic Diagnosis (PGD) to Ensure a Matched Sibling Donor for Hematopoietic Stem Cell Transplantation (HSCT)

Moderator: Jonathan Marron, MD MPH

Objectives:

  1. Describe the potential role of a “savior sibling” for pediatric patients for whom hematopoietic stem cell transplantation (HSCT) is a therapeutic option.
  2. Describe the ethical and psychosocial challenges encountered when considering utilization of pre-implantation genetic diagnosis (PGD) to create a savior sibling.
  3. Explore various scenarios in which a savior sibling might be more or less acceptable; and consider how such scenarios might be approached in one’s own clinical practice.

With pre-implantation genetic diagnosis (PGD), families can “create” an HLA-matched donor for a child requiring transplantation. Most families know little about this controversial option but express great interest in learning more. Session attendees will become familiar with the potential role for–and numerous challenges inherent in–using PGD to create so-called “savior siblings.” They will get to interactively examine their own perspectives on this topic and will be armed with tools for discussing this option with families.

Introduction
Jonathan M. Marron, MD MPH

Matched Sibling Donor Transplantation and the Potential Role for Savior Siblings
Leslie Lehmann, MD

Savior Siblings: Psychosocial Issues and Challenges
Wendy Pelletier, MSW RSW

Ethical Considerations When Creating a Savior Sibling
Jonathan M. Marron, MD MPH

No relevant financial relationships and no discussion of off-label drug use: Jonathan M. Marron; Leslie Lehmann; Wendy Pelletier

10:15–11:15 am

Break in Exhibit Hall

10:15 am–10:45 am

Genius Bar 

Join us in the Exhibit Hall and bring your cases on Bony Lytic Lesions. Speakers from the “Multiple Bony Lytic Lesions, but Not Langerhans Cell Histiocytosis (LCH) – Now What?” session will be there to help answer questions and guide the discussion.

 

10:30–11:05 am

Speed Mentoring-Session I

Speed mentoring is an opportunity to network with leaders in the field through a series of short, focused conversations about specific questions. It is a concept that focuses on quick-hit information, allowing for time-efficient networking. Fellows and early career attendees registering for this session will have the opportunity to meet one-on-one with up to three mentors for 10 minutes each. Mentees will be required to submit a one-page document prior to arriving onsite that addresses a specific topic of interest. Mentees should come prepared with two concrete questions to discuss for their chosen subject. Mentors will review the mentees’ documents in advance for meaningful insight and efficient engagement.

Each person will be assigned to one of two sessions (either 10:30 am or 12:30 pm).

Advanced registration is required.

10:30–11:00 am

Education Theater Presentation: Strategies to minimize iron overload and alloimmunization in Sickle Cell Patients

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Odd-Numbered Poster Tour 

ASPHO will provide two poster tours during exhibit hours. The Conference Planning Committee selected five highly ranked posters that will be included in a 30-minute tour for all interested attendees. Authors of these selected posters will provide a brief overview of their research and answer attendees’ questions. Advanced registration is not required. Attendees should meet in the back of Exhibit Hall and look for a staff member holding a Poster Tour sign. Don’t miss the opportunity to receive a quick overview of top-rated abstracts!

Meet in the back of the Exhibit Hall.

The following posters will be featured during this tour:

(#003) IMPACT OF MINIMAL RESIDUAL DISEASE AT END CONSOLIDATION IN STANDARD RISK B-LYMPHOBLASTIC LEUKEMIA
Rachel Rau, MD

(#119) GENOMIC CHARACTERIZATION OF A PEDIATRIC COHORT WITH NON-MALIGNANT LYMPHOPROLIFERATIVE DISORDERS
Nitya Gulati

(#439) NEXT-GENERATION SEQUENCING AS A NONINVASIVE TEST TO IDENTIFY PATHOGENS IN PEDIATRIC PATIENTS
Jenna Rossoff

(#501) TCRαβ/CD19 DEPLETED HAPLO-HSCT + ZOLEDRONATE FOR PEDIATRIC MALIGNANCIES: PRELIMINARY FINDINGS
Nicholas Pytel, DO MS

(#755) EPIDEMIOLOGY AND OUTCOME OF ACUTE KIDNEY INJURY IN CHILDREN WITH SICKLE CELL DISEASE
Meghan McCormick, MD

 

11:15 am–12:00 pm

2019 St. Baldrick’s Foundation Robert J. Arceci Innovation Award Announcement and Past Recipient Presentation

The St. Baldrick’s Foundation Robert J. Arceci Innovation Award was established in loving memory of Robert J. Arceci, MD PhD. Two awards are presented per year, one to a U.S. or Canadian researcher to be presented at the ASPHO meeting, and one international award to be presented at the SIOP meeting. Each award is $250,000 per year for three years, to support truly innovative childhood cancer research. This year’s North American winner will be announced during this session.

Charles G. Mullighan, MBBS MSc MD, the 2016 award recipient, will be presenting the outcomes of his research.

Recent Insights into High Risk Leukemia
Recent years have witnessed remarkable advances in our understanding in the genetic basis of acute lymphoblastic leukemia, and rapid translation of many of these findings to new diagnostic and therapeutic approaches. The St. Baldrick’s Foundation Robert J. Arceci Innovation Award enabled the awardee to expand and develop new avenues of investigation, including the genomic analysis of poorly understood subtypes including mixed phenotype acute leukemia and acute erythroleukemia. These studies have yielded not only fundamental insights into the genetic basis of these diseases, but have resulted in a revised classification of leukemia, identification of new therapeutic targets, the establishment of new experimental approaches to understand the mechanistic basis of leukemogenesis. The award has also facilitated landmark studies of the genomics of clonal evolution in ALL, and in parallel, mechanistic studies that have utilized classical in vivo experimental modeling and functional genomic screens to examine the role of mutations of individual genes, and pathways, in driving treatment resistance. Highlights from these studies will be presented.

Relevant financial relationships to disclose and no discussion of off-label drug use: Charles G. Mullighan-Amgen: Speaker bureau, honoraria; Loxo Oncology: Principal investigator, research grant to my institution; Pfizer: Speakers bureau, honoraria; principal investigator, research grant to my institution

12:00–1:30 pm

Lunch with Exhibits and Posters

 

12:00–1:00 pm

Author-Attended Poster Session B: Even-numbered Posters

 

12:30–1:05 pm

Speed Mentoring-Session 2

Speed mentoring is an opportunity to network with leaders in the field through a series of short, focused conversations about specific questions. It is a concept that focuses on quick-hit information, allowing for time-efficient networking. Fellows and early career attendees registering for this session will have the opportunity to meet one-on-one with up to three mentors for 10 minutes each. Mentees will be required to submit a one-page document prior to arriving onsite that addresses a specific topic of interest. Mentees should come prepared with two concrete questions to discuss for their chosen subject. Mentors will review the mentees’ documents in advance for meaningful insight and efficient engagement.

Each person will be assigned to one of two sessions (either 10:30 am or 12:30 pm).

Advance registration is required.

 

12:30–12:45 pm

Awardee Spotlight @ ASPHO Booth with 2019 George R. Buchanan Award recipient Kenneth McClain, MD PhD

 

12:45 –1:15 pm

Education Theater Presentation: Targeting IFNγ in Primary HLH: Changing the Treatment Paradigm

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1:15pm

Passport Prize Drawing

1:30–3:00 pm

(C17) New Paradigms for Severe Aplastic Anemia: Genetic Screening, Eltrombopag, and Up Front Unrelated Donor BMT

Moderator: Michael A. Pulsipher

Objectives:

  1. Describe how to put genetic screening for inherited bone marrow failure syndromes such as severe aplastic anemia into practice.
  2. Discuss current outcomes with IST in North America and what we know about the short and long-term effectiveness of Eltrombopag in combination with IST as up front therapy in children with SAA.
  3. Describe the effectiveness of up front unrelated donor BMT and use of alternative donors such as haploidentical-related donors in SAA patients who fail IST.

The approach to initial therapy for severe aplastic anemia has traditionally been transplantation (BMT) for those with fully matched sibling donors and immunosuppressive therapy (IST) for all others. With recent improvements in outcomes of unrelated donor transplantation, some European and Asian groups are advocating up front unrelated donor transplantation for those with fully matched donors. At the same time, the addition of eltrombopag to IST has been shown to improve complete remission rates and is now FDA-approved as first line therapy, but data in children is limited and durability of response is poorly understood. Thus, first-line therapy for upfront SAA seems to be shifting and the very best approach is unknown. Finally, with the emergence of genetic screens for SAA, clinicians are also unsure how to fit these results into treatment paradigms.

Overview of the Science of Severe Aplastic Anemia and Genetic Screening
Akiko Shimamura, MD PhD

Results of North American Pediatric Aplastic Anemia Consortium (NAPAAC) Outcomes Study and Use of Eltrombopag in Children
Taizo A. Nakano, MD

Overview of MRD BMT for Up Front SAA and Haplo and URD BMT for IST Failure
Michael A. Pulsipher, MD

No relevant financial relationships and no discussion of off-label drug use: Taizo A. Nakano
Relevant financial relationships to disclose; discussion of off-label drug use: Michael A. Pulsipher - Novartis: Consulting, Study Steering Committee, Speaker, Honorarium; Adaptive: Consultant and Study support, Honorarium; Akiko Shimamura - Novartis: Local investigator for a Novartis-sponsored trial, Novartis funds trial

(C18) Not Too Young for That Cancer: Adolescent and Young Adult Carcinomas

Moderator: Karen E. Effinger, MD MS

Objectives:

  1. Summarize unique biological features and current treatment recommendations for breast carcinoma in AYAs and possible modifications in treatment of younger patients.
  2. Summarize unique biological features and current treatment recommendations for colorectal carcinoma in AYAs and possible modifications in treatment of younger patients.
  3. Recognize obstacles to the appropriate treatment of AYAs with cancer and determine methods to improve collaboration between adult and pediatric programs.

Cancer incidence in adolescents and young adults (AYAs) aged 15-39 years is over 4 times greater than in younger children. Care for these patients is complex. AYAs may present with carcinomas more commonly seen in older patients. Often pediatric oncologists are not as familiar with treatment of these tumors and adult facilities do not have the psychosocial resources to care for younger AYAs. Additionally, unique biological features in AYA carcinomas may impact treatment; yet, clinical trial enrollment is lower in AYAs compared with other populations. Collaboration is needed between pediatric and adult specialists to improve the care of AYAs.

Molecular Mechanisms of Carcinogenesis in AYA patients and Integrating of Adult and Pediatric Services to Treat AYA Carcinomas
Louis Rapkin, MD

Breast Cancer in AYA Patients
Carey Anders, MD

Colorectal Cancer in AYA Patients
Michael La Quaglia, MD FACS FAAP FRCS

No relevant financial relationships; no discussion of off-label drug use: Karen E. Effinger; Louis Rapkin; Michael La Quaglia

Relevant financial relationships to disclose; discussion of off-label drug use: Carey Anders - Novartis/Merrimack/Lilly/Nektar/Cascadian/Seattle Genetics/Genentech: Advisory Board Member, No Compensation Received; Novartis/Merrimack/Puma/Lilly/Merck/Cascadian/Nektar/Tesaro/G1 Therapeutics: Principal Investigator, Research Grant to my Institution; Merck: Consultant, Honoraria

(C19) Transforming the Treatment of Chronic Pain

Moderator: Caitlin Neri, MD MPH

Objectives:

  1. Review current research into novel mechanisms to address chronic pain.
  2. Describe evidence for psychological therapies for chronic pain and provide a framework to translate this into clinical practice.
  3. Discuss current evidence around pharmacology, safety, and legality of medical marijuana.

Within the current climate of the opioid crisis, there is increased concern for the use of opioids in children and adults. Current guidelines call for use of non-pharmacologic pain approaches and non-opioid pharmacologic interventions for the treatment of chronic pain. Patients and families are increasingly interested in cannabinoids, although potential benefit must be assessed with consideration of concerns related to safety and legality.

Mechanism-based Actionable Targets to Prevent and Treat Pain in Sickle Cell Disease
Kalpna Gupta, PhD

Psychological Treatment for Pediatric Chronic Pain: Translating Evidence-based Science into Widespread Practice
Rachael Coakley, PhD

It’s High Time to Talk about Medical Marijuana
William Zempsky, MD MPH

No relevant financial relationships and no discussion of off-label drug use: Rachael Coakley

No relevant financial relationships; discussion of off-label drug use: Caitlin Neri

Relevant financial relationships to disclose; discussion of off-label drug use: William Zempsky - Pfizer/Endo Pharmaceuticals: Consultant, Honoraria; GSK: Advisory Board Member, Honoraria; Vapogenix: Advisory Board Member, Stock Options

Relevant financial relationships to disclose; no discussion of off-label drug use: Kalpna Gupta - Tau tona Group/Novartis: Advisory Board Member, Honoraria

3:00–3:30 pm

Break

 

3:30–4:30 pm

Concurrent Paper Session

Paper Session - Children's Oncology Group (COG)

This session will consist of three-15 minute presentations of high impact results, promising studies or other initiatives recently presented at COG or other national/international meetings.

Objectives:

  1. Discuss new approaches related to diagnosis and treatment in pediatric hematology/oncology.
  2. Examine current research outcomes from a variety of the latest in basic and clinical research.

Moderators: Christopher Porter, MD; Cindy Schwartz, MD

(2017) Levofloxacin Prophylaxis
Sarah Alexander, MD

(2018) Renal Tumors: Future Roadmap
Rajkumar Venkatramani, MD MRCPCH MS

(2019) Soft Tissue Sarcoma
Douglas Hawkins, MD

No relevant financial relationships and no discussion of off-label drug use: Rajkumar Venkatramani

No relevant financial relationships; discussion of off-label drug use: Sarah Alexander

Relevant financial relationships; discussion of off-label drug use: Douglas Hawkins – LOXO Oncology/Bayer/Bristol Myers Squibb/Celgene: Advisory board member, travel reimbursement

Paper Session - Hematology Science as presented at the 2018 ASH Annual Meeting

This session will consist of four 15 minute presentations of hematology science - from Hematology Science as presented at the 2018 ASH Annual Meeting.

Objectives:

  1. Discuss new approaches related to diagnosis and treatment in pediatric hematology/oncology.
  2. Examine current research outcomes from a variety of the latest in basic and clinical research.

Moderators: Dana Matthews, MD; Shelley Crary, MD

(2020) Realizing Effectiveness across Continents with Hydroxyurea (REACH): A Prospective Multi-National Trial of Hydroxyurea for Sickle Cell Anemia in Sub-Saharan Africa
Patrick McGann, MD

(2021) Leucine for the Treatment of Transfusion Dependence in Patients with Diamond Blackfan Anemia
Adrianna Vlachos, MD

(2022) A Phase 1/2 Trial of Investigational Spk-8011 in Hemophilia a Demonstrates Durable Expression and Prevention of Bleeds
Spencer Sullivan, MD

(2023) Peripherally Inserted Central Catheters (PICCs), the Leading Cause of Central Venous Catheter Associated Thrombosis in Children
Julie Jaffray, MD

No relevant financial relationships and no discussion of off-label drug use: Julie Jaffray; Patrick McGann

No relevant financial relationships; discussion of off-label drug use: Adrianna Vlachos

Relevant financial relationships and no discussion of off-label drug use: Spencer Sullivan - Octapharma; Consultant, honoraria

4:45–6:15 pm

(C20) Putting an End to End Organ Damage in Sickle Cell Disease

Moderator: Courtney D. Thornburg, MD MS

Objectives:

  1. Describe the molecular pathogenesis, novel diagnostic imaging techniques, and treatment of sickle cell retinopathy.
  2. Review the molecular pathogenesis of bony changes in SCD, including avascular necrosis. Treatments including Vitamin D supplementation will also be covered.
  3. Discuss updates on pathogenesis, diagnosis, and management of sickle nephropathy.

Though the survival for pediatric patients with sickle cell disease (SCD) has substantially improved in the recent years, the mortality has not decreased for adults with SCD. Adults with SCD have excess morbidity and mortality from end organ damage which typically starts in early childhood. Pediatric hematologists must understand the pathogenesis, screening methods and treatment options for these complications so that they can help to prevent excess morbidity and mortality later in life for patients with SCD.

Introduction
Ifeyinwa Osunkwo, MD MPH

Sickle Cell Retinopathy

Adrienne Scott, MD

Sickle Cell Nephropathy
Ram Kalpatthi, MD

Promoting Bone Health in Patients with SCD
Ifeyinwa Osunkwo, MD MPH

No relevant financial relationships; discussion of off-label drug use: Courtney D. Thornburg; Ifeyinwa Osunkwo; Ram Kalpatthi

Relevant financial relationships to disclose; no discussion of off-label drug use: Adrienne Scott - Allergan, Inc.: Consultant, Honoraria

(C21) Defining Pediatric MDS: A Morphologic or Molecular Diagnosis?

Moderator: Taizo A. Nakano, MD

Objectives: 

  1. Describe the evolving morphologic definition of pediatric MDS and how cellular dysplasia impacts diagnosis, prognosis and therapeutic decision making.
  2. Describe the evolving molecular definition of pediatric MDS and review both primary and secondary molecular predisposition to pediatric MDS.
  3. Review the practical application of the WHO 2016 revised definition of MDS and debate the indications of pharmacotherapy and transplant.

How influential are the morphologic versus the molecular features in the diagnosis and prognosis of pediatric MDS? Comprehensive guidelines to accurately define and prognosticate pediatric MDS remain elusive despite ongoing efforts to revise the WHO classification. We turn to hematopathologists to define cellular dysplasia and, concurrently, have identified an increased number of somatic driver mutations found to impact prognosis. Renewed efforts to organize a unified approach to pediatric MDS will require multidisciplinary cooperation.

Secondary MDS: A Case-Based Exercise to Diagnose and Treat Pediatric MDS
Taizo A. Nakano, MD

In Defense of a Molecular Definition of Pediatric MDS
Timothy Olson, MD PhD

In Defense of a Morphologic Definition of Pediatric MDS
Mark Fleming, MD DPhil

No relevant financial relationships and no discussion of off-label drug use: Taizo A. Nakano; Mark Fleming

Relevant financial relationships to disclose; discussion of off-label drug use: Timothy Olson - Novartis: Advisory board member, hourly compensation for limited advisory board participation; Merck: Consultant, hourly compensation for 1 day SIE participation

(C22) The Power of One: Present Challenges in Treating Rare Diseases

Moderator: Clifford Takemoto, MD

Objectives: 

  1. Discuss collaborative approaches to clinical management of rare diseases.
  2. Recognize the unique barriers to performing clinical and basic research for rare diseases.
  3. Know the current approaches to overcome challenges of trial design, regulatory issues and funding for rare diseases.

Pediatric hematologists and oncologists often care for children with diseases that are rare in the general population. Yet these conditions can be life-threatening and present tremendous challenges because of a paucity of clinical expertise and lack of evidence-based guidelines for management. Collaborative groups of clinicians and researchers with a common focus in a rare diseases can provide a valuable expertise and resources for patients and primary providers. Advancing knowledge and improving care for rare diseases is hampered by challenges in trial design and limited research funding. Advocacy and collaboration are critical to overcome these barriers.

The Story
Clifford Takemoto, MD

The Family Perspective
Kimberley E. Steele, MD PhD; Gregory P. Prokopowicz, MD

The Physician Perspective: Pros and Cons of Precision Medicine
Julia L. Glade Bender, MD

The NIH Perspective: Overcoming Roadblocks in Rare Disease Research
Philip John (P.J.) Brooks, PhD

No relevant financial relationships and no discussion of off-label drug use: Kimberly E. Steele; Gregory P. Prokopowicz; Philip John Brooks

Relevant financial relationships; discussion of off-label drug use: Clifford Takemoto: Novonordisk/Alnylm: Principal Investigator, Research Grant to my Institution; Genentech: Advisory Board Member, Honoraria; Julia L. Glade Bender - Eisai/Amgen/Merck/Incyte/Bristol Meyers Squibb/Celegene/Lilly/Novartis/Bayer/Astra Zeneca: Principal Investigator, Research Grant to my Institution

6:30–7:30 pm

Distinguished Career Award Presentation and Reception

The Distinguished Career Award is presented annually by ASPHO to a senior physician or other professional who during his or her career has had a major impact on the subspecialty through some combination of research, education, patient care, and advocacy. This session celebrates Elliott Vichinsky, MD, from UCSF Benioff Children's Hospital Oakland, and is followed by a reception to congratulate him on the award.  

Saturday, May 4

6:45–8:15 am

Continental Breakfast

 

7:00–8:00 am

Advanced Practice Provider Special Interest Group

Leadership: Lesley Arland, PA; Nicholette Breier-Adkins, MSN, RN, CPNP

The Advanced Practice Provider Special Interest Group is dedicated to providing a forum for the exchange of ideas and information among advanced practice providers (APPs) and for promotion of their role in the field of pediatric hematology and oncology. At this year’s meeting, we will be highlighting a poster submitted by an APP as well as continuing discussions of issues pertaining to APPs in hematology, oncology and transplant.

Clinical Immunology Special Interest Group

Leadership: David Buchbinder, MD MS; Shanmuganathan Chandrakasan, MD

The Clinical Immunology SIG workshop will focus on broadening our understanding and application of clinical immunology within pediatric hematology/oncology practice. This will include education related to the diagnosis and management of immunologic disorders pertinent to hematologists/oncologists. We will continue to develop a foundation for the development of future research initiatives.

Speakers:
David Buchbinder, MD MS
Shanmuganathan Chandrakasan, MD
Luigi Notarangelo, MD

Global Pediatric Hematology/Oncology Special Interest Group

Leadership: Andrea Orsey, MD; Patrick McGann, MD MS

Hemoglobinopathy Special Interest Group

Leadership: Howard Grodman, MD; Monica Hulbert, MD

The Hemoglobinopathy SIG’s goal is to promote collaboration among hematologists caring for children and young adults with hemoglobinopathies including sickle cell disease.
Discussion topics:

  • Mobile Health Interventions, Hydroxyurea Adherence, and Quality of Life in Youth with Sickle Cell Disease
  • Ascertaining Sickle Cell Trait Knowledge
  • Hemoglobinopathy SIG leadership election results

Featured Speaker:
Sherif Badawy, MD MS MBBCh
Assistant Professor of Pediatrics
Ann & Robert H. Lurie Children’s Hospital of Chicago
Northwestern University Feinberg School of Medicine

7:00–8:00 am

Clinical Conundrums

New this year – ASPHO is providing the opportunity to interact with a recognized expert on a challenging diagnostic and management issue. Each session is scheduled for 1 hour and will include a 15-20 minute presentation by the expert on their general approach to the clinical problem followed by small group discussion of additional cases shared by the attendees.

Advanced registration is required. Seating is limited to 20. The registration fee is $25.

(CC3) Thrombotic Storm
Marilyn Manco-Johnson, MD

(CC4) – Relapsed Sarcomas
Sheri Spunt, MD

No relevant financial relationships and no discussion of off-label use of a drug: Marilyn Manco-Johnson

No relevant financial relationships; discussion of off-label drug use: Sheri Spunt

8:15-9:45 am

(D23) Hematological Manifestations of Immune Deficiencies and Dysregulatory Disorders: When should you look harder for underlying causes of Autoimmune Cytopenias?

Moderator: Troy R. Torgerson, MD PhD

Objectives: 

  1. Describe the hematological manifestations of Primary Immune Deficiencies (PID) and Primary Immune Regulatory Disorders (PIRD).
  2. Explain how best to work up and treat these patients.
  3. Review potential definitive treatment options for these disorders with transplantation or gene therapies.

New sequencing technologies have shown underlying disorders in many patients with autoimmune cytopenias. Practitioners are unsure which immune deficiency/dysregulatory disorders are associated with hematological manifestations and how to diagnose and treat them. Understanding whether patients have underlying genetic conditions prompting hematological issues is key to proper diagnosis and therapy.

Pathogenesis and Hematologic Manifestations of PID/PIRD
Troy R. Torgerson, MD PhD

Medical Management of Cytopenias in patients with PID/PIRD
Kristin Shimano, MD

Curative Approaches to Patients with PID/PIRD
Sung-Yun Pai, MD

No relevant financial relationships; discussion of off-label drug use: Kristin Shimano; Sung-Yun Pai

Relevant financial relationships to disclose; discussion of off-label drug use: Troy R. Torgerson: Shire: Advisory board member, honoraria; CSL Behring/Octapharma/UBC pharma: Consultant, honoraria

(D24) Oncofertility Programs: More Than Just Sperm Banking 

Moderator: Wendy Allen-Rhoades, MD

Objectives:

  1. Recognize the need for oncofertility care for pediatric oncology patients.
  2. Identify barriers to providing oncofertility care.
  3. Develop strategies to overcome the barriers to oncofertility care.

Oncofertility is the field of care for oncology patients provided by cancer and fertility healthcare providers. Oncofertility is complex both medically and pyschosocially, which underscores the need for an innovative and multidisciplinary approach. These complexities create barriers that hinder the timely development of oncofertility programs. This workshop will discuss strategies used in established and emerging programs and the logistics of pursuing fertility preservation options. Case-based examples will highlight the role of fertility patient navigators.

Development of an Established Oncofertility Program
Karen C. Burns, MD MS

Navigating Fertility Preservation Procedures
Krista Childress, MD

Outsourcing Oncofertility Services
Nicholas D. Yeager, MD

Case Based Examples Utilizing Fertility Patient Navigators
Olivia J. Frias, MSN RN III CNL

No relevant financial relationships; no discussion of off-label drug use: Wendy Allen-Rhoades; Karen C. Burns; Krista Childress; Nicholas D. Yeager; Olivia J. Frias

(D25) The (Financial) Cost of a "Cure"

Moderator: Valerie I. Brown, MD, PhD

Objectives:

  1. Interpret the financial implications of strategies such as gene editing/therapy for nonmalignant hematologic disorders such as sickle cell disease and thalassemia.
  2. Implement strategies to assess use and improve accessibility of novel, but costly, commercially available cellular therapies, such as CAR-T cells.
  3. Discuss the ethical considerations and dilemmas regarding access to and payment of these costly therapies.

There have been exciting new developments using gene manipulation to cure pediatric hematologic and oncologic diseases, such as gene editing/therapy for hemoglobinopathies and CAR-T cell therapy for relapsed/refractory ALL. As these therapies become commercially available, it is critical that providers be aware of their cost, how to navigate access to these therapies and recognize how the cost impacts accessibility on an individual as well as global basis. Ethical dilemmas regarding access of these therapies to all patients needs to be addressed.

Overview of the Financial Costs of Innovative and Potentially Curable Therapies
Valerie I. Brown, MD PhD

The Cost of Customized Cellular Therapy: Can We Afford It?
Joseph Alvarnas, MD

Novel Gene Editing/Therapy Strategies for the Treatment of Hemoglobinopathies: The Cost Beyond Clinical Trials
Mark Trusheim, MSc

No relevant financial relationships and no discussion of off-label drug use: Valerie I. Brown; Joseph Alvarnas; Mark Trusheim

 

10:00-11:30 am

Paper Session with Luminary Investigator - Oncology/Outcomes Research/Survivorship

Melissa M. Hudson, MD will present The Role of Survivorship Research in Advancing Childhood Cancer Care and Quality of Survival followed by four 15-minute presentations of scientific research from selected oncology related abstracts.

Moderators: Jessica Heath, MD; Denise Adams, MD

Objectives:

  1. Discuss new approaches related to diagnosis and treatment in pediatric hematology/oncology.
  2. Examine current research outcomes from a variety of the latest in basic and clinical research.

(2024) Septic Shock (SS) Incidence in National ALL & AML Cohorts using Administrative & Clinical Trial Data
Viviane Cahen, BS

(2025) Nelarabine Abrogates Relapse Rates in CNS-3 T-ALL: A Report from Children’s Oncology Group AALL0434
Nathan Gossai, MD

(2026) Current Cancer Survivorship Practices: A Report from the Children’s Oncology Group (COG)
Karen E. Effinger, MD MS

(2027) Intra-Arterial Chemotherapy for Retinoblastoma: A 7-year Single Institution Experience
Ali Sanati-Mehrizy, MD FAAP

No relevant financial relationships and no discussion of off-label drug use: Viviane Cahen; Karen E. Effinger; Ali Sanati-Mehrizy

No relevant financial relationships; discussion of off-label drug use: Nathan Gossai

No relevant financial relationships and no discussion of off-label use of a drug: Melissa M. Hudson

Paper Session with Luminary Investigator - Hematology

Blanche Alter, MD MPH, will present Advancing Discovery in Hematology: Bench to Bedside? No. Bedside to Bench to Bedside.... followed by four 15-minute presentations of scientific research from selected hematology related abstracts.

Moderators: Cliff Takemoto, MD; Akiko Shimamura, MD PhD

Objectives:

  1. Discuss new approaches related to diagnosis and treatment in pediatric hematology/oncology.
  2. Examine current research outcomes from a variety of the latest in basic and clinical research.

(2028) The Shwachman Diamond Syndrome Registry: Hematologic Complications
Kasiani Myers, MD

(2029) Characteristics and Outcomes of Unprovoked Venous Thromboembolism in the Pediatric Population
Liny John, MD

(2030) Third Party VST are an effective Therapy for the Treatment of BK-Virus Reactivation after Transplant
Jeremy Rubinstein, MD PhD

(2031) Inhibition of Nemo-Like Kinase improves Erythropoiesis in Models of Diamond Blackfan Anemia
Kaoru Takasaki, MD

No relevant financial relationships and no discussion of off-label drug use: Liny John; Jeremy Rubinstein; Kaoru Takasaki; Blanche Alter

Relevant financial relationships and no discussion of off-label drug use: Kasiani Myers –Novartis/Bellicum: Advisory board member, honoraria 

11:45-1:45 pm

(MOCS) Maintenance of Certification (MOC) Learning Session

Moderator: Erika Friehling, MD 
Speakers: Leo Mascarenhas, MD MS; Glen Lew, MD; Robert Sidonio, MD MSc; Brenda Weigel, MD FAAP; Jorge DiPaola, MD; Jeffrey M.Lipton, MD PhD; Naomi Winick, MD

The Maintenance of Certification (MOC) learning session will feature an American Board of Pediatrics (ABP) Lifelong Learning and Self-Assessment Module. It is intended for those who are enrolled in the MOC process and would like to earn MOC activity points, but also for anyone wishing for a broad review of recent literature relevant to pediatric hematology/oncology. The session will be conducted in an interactive group setting led by authors or experts in their fields. The session will cover multiple-choice questions in the 2019 Pediatric Hematology/Oncology Self-Assessment. Please bring your laptop to enter the answers to each question during the session. At the completion of this session, those enrolled in the ABP MOC program will have been able to submit their answers to the online module for scoring to receive 10 MOC Part 2 points and 6 AMA PRA Category 1 Credits™.

Advanced registration is required. The registration fee is $50.

No relevant financial relationships and no discussion of off-label use of a drug: Erika Friehling; Glen Lew; Jorge DiPaola; Jeffrey M. Lipton; Naomi Winick

No relevant financial relationships; discussion of off-label drug use: Brenda Weigel

Relevant financial relationships; discussion of off-label drug use: Leo Mascarenhas - Bayer: Speaker bureau, honoraria; Lilly Oncology: Advisory board member, funds to institution; Lilly Oncology/Astra Zeneca: Principal investigator, research grant to my institution

Relevant financial relationships and no discussion of off-label use of a drug: Robert Sidonio-Shire/Novo Nordisk/Uniqure/Biomarin/CSL Behring/Genentech: Advisory Board Member, Honoraria; Shire/Bioverativ/Grifols/Kedrion/Genentech: Principal Investigator, Research Grant to my Institution

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