Schedule of Events
Wednesday | Thursday | Friday | Saturday
Most up-to-date session information and room locations can be found in the conference app. Please search 'ASPHO Events' in your app store or visit http://app.core-apps.com/aspho2018.
Wednesday, May 2
7:30 am – 11:30 am
Preconference Workshop | (PC01) Essentials of Effective Mentoring
Moderators: A. Lindsay Frazier, MD ScM; Jennifer C. Kesselheim, MD MEd; Christine Pfund, PhD
Objectives:
- Identify the core competencies of mentorship.
- Implement new strategies and skills within each competency.
Mentorship is not an innate ability but a skill to be cultivated and honed. Mentoring skills are a requisite of our day-to-day work in academics, yet we do not often discuss the key elements of being an effective mentor. This 4-hour preconference workshop will break mentorship down into its key competencies: aligning expectations, maintaining effective communication, assessing understanding, addressing equity and inclusion, fostering independence, and promoting professional development. Through interactive, case-based discussion, participants will acquire practical, concrete skills and tools to take back to their academic practices.
Advanced registration is required. The registration fee is $125.
Recommended by the Professional Development Committee.
No relevant financial relationships and no discussion of off-label drug use: Jennifer Kesselheim; Christine Pfund
Relevant financial relationships to disclose and no discussion of off-label drug use: A. Lindsay Frazier-Decibel Therapeutics: Advisory board member, stock
7:45 am–8 pm
Pediatric Blood and Marrow Transplant Consortium (PBMTC) Meeting
OMNI WILLIAM PENN HOTEL
1–2:30 pm
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 lectureship will be presented by Stephen P. Hunger, MD.
Improving Survival for Children and Young Adults with Acute Lymphoblastic Leukemia
Acute lymphoblastic leukemia (ALL) was a fatal disease until the early to mid-1960s. Today more than 85% of children diagnosed with ALL will be cured. This remarkable change in prognosis is due in large part to multi-institutional clinical trials conduced by groups in North America, Western Europe and other high-income countries and most of these gains have been achieved using cytotoxic chemotherapy agents that have been widely available for decades. A historical perspective on how cure rates have improved, with a particular focus on recent advances and remaining challenges will be discussed.
Objectives:
- Explain the incidence of childhood ALL and the improvements in survival that have occurred over the past 50 years.
- Describe the risk factors that predict outcome in childhood ALL.
- Discuss how augmented post-induction therapy has improved survival for ALL.
Click here for more information about this year's recipient.
Relevant financial relationships to disclose; discussion of off-label drug use: Stephen Hunger-Novartis: Consultant, honoraria; Amgen: Stock; Jazz Pharmaceutical, Erytech: Advisory board member, honoraria
Plenary Paper Session
Moderators: Denise M. Adams, MD; Donald L. Yee, MD MS
Objectives:
- Discuss new approaches related to diagnosis and treatment in pediatric hematology/oncology.
- Examine current research outcomes from a variety of the latest in basic and clinical research.
The 2018 Conference Planning Committee has chosen the abstracts listed below as among the best submissions.
(2001) Excellent Event-free (EFS) and Overall Survival (OS) for Children with Down Syndrome (DS) with Standard Risk B-acute lymphoblastic Leukemia (SR B-ALL): Results of Children's Oncology Group (COG) AALL0331
Kelly Maloney, MD
(2002) Induced Pluripotent Stem Cell Model of 7Q Deletion in Bone Marrow Failure Identifies a Novel Therapeutic Strategy
Melisa Ruiz-Gutierrez, MD PhD
No relevant financial relationships and no discussion of off-label drug use: Melisa Ruiz-Gutierrez
Relevant financial relationships to disclose and no discussion of off-label drug use: Kelly Maloney-Johnson and Johnson: Stock holder, stock
Paper Co-Authors Disclosures
3:00–4:45 pm
St. Baldrick’s Foundation Robert J. Arceci Innovation Award Announcement
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.
(A1) Plenary Session—Health Care in the Current Political Climate: Stormy Days Ahead?
Moderators: Lindsay Mize, MSN RN CPNP; Daniel S. Wechsler, MD PhD
Objectives:
- Discuss a 30,000 foot view of current issues surrounding healthcare reform.
- Provide an update on current initiatives and how they impact pediatric hematology-oncology.
- Discuss the 304B program in the context of the current healthcare environment and then discuss how this will effect the pediatric hemophilia population.
Since the 2016 presidential election, our country has entered into an uncertain and tumultuous chapter in healthcare and health policy. Discussions about replacing the Affordable Care Act are ongoing, and it is unclear what the future will hold. The governmental changes in healthcare affect the care that we deliver to our patients and their families. Speakers in this session will address current efforts to reinvent healthcare, and the implications for pediatric hematology/oncology.
Healthcare Reform in 2018: Where Do We Stand?
David K. Jones, PhD
Advocacy and Congressional Initiatives that Impact Pediatric Oncology
Danielle Leach, MPH
Current Status and Future of the 304B Drug Pricing Program for Hemophilia Treatment Centers
Joseph Pugliese
Recommended by the Advocacy Committee
No relevant financial relationships and no discussion of off-label drug use: Lindsay Mize; Daniel S. Wechsler; David K. Jones; Danielle Leach; Joseph Pugliese
5–6:30 pm
(A2) Pediatric Ependymoma: The Science Driving Patient Care
Moderator: Andrew W. Walter, MD MS
Objectives:
- Describe the evolution of the classification of ependymomas into high risk and low risk groups based on anatomic/histologic classification schemes vs molecular classification.
- Identify the critical role that neurosurgery plays in the cure of children with ependymoma.
- Describe the potential role of vaccine/immunobiology therapy in the treatment of ependymoma.
Recent progress in the study of ependymoma brain tumors in children has revolutionized the way we think about, classify and even possibly treat ependymomas. Much in the same way that science has turned upside down the way we think about medulloblastoma, the study of ependymoma should allow us to treat children with ependymoma more effectively and with potentially less toxicity than ever before. Advances in basic science, immunobiology and neurosurgery are all contributing to the potential for continued improvement in outcomes.
Treatment of Ependymoma: History and Future
Nicholas Foreman, MD
The Molecular Classification of Pediatric Ependymoma
David Ellison, MD PhD MA
New Paradigms in the Management of Ependymoma
Vijay Ramaswamy, MD PhD
The Role of Surgery, Molecular Characterization, and Immunotherapy in the Treatment of Ependymomas
Ian F. Pollack, MD FAANS FAAP FACS
No relevant financial relationships and no discussion of off-label drug use: Andrew W. Walter; Nicholas Foreman; David Ellison; Vijay Ramaswamy
No relevant financial relationships to disclose; discussion of off-label drug use: Ian F. Pollack
(A3) Iron: Let's Get Pumped
Moderators: Shelley Crary, MD MS
Objectives:
- Apply knowledge regarding the indications and various dosing strategies of intravenous and oral iron formulations.
- Discuss advances in the diagnosis and management of iron refractory states such as anemia of inflammation and IRIDA.
- Examine current management strategies for iron overload in chronically or excessively transfused patients.
Hematologists are frequently consulted for anemia that is refractory to therapy. New strategies utilizing IV iron and oral iron challenge the status quo of iron deficiency management. Knowledge of advances in diagnosis and management of iron refractory conditions will improve the management of this common condition. Knowledge of new treatment strategies for iron overload is important to optimize care for chronically or excessively transfused patients.
New Treatment Strategies for Iron Deficiency
Jacquelyn M. Powers, MD
Iron Refractory States
Matthew M. Heeney, MD
Update on Management of Iron Overload
Thomas D. Coates, MD
No relevant financial relationships and no discussion of off-label drug use: Shelley Crary
No relevant financial relationships to disclose; discussion of off-label drug use: Jacquelyn M. Powers
Relevant financial relationships to disclose and no discussion of off-label drug use: Matthew M. Heeney-Intrinsic Life Sciences: Principal investigator, research grant to institution; Thomas D. Coates-Apo Pharma, Celgene: Consultant, honoraria
(A4) Extracellular Vesicle Trafficking—Biology and Translational Impact in Hematology and Cancer
Moderator: Peter Kurre, MD
Objectives:
- Describe vesicle biogenesis.
- Discuss the role of EVs in blood, coagulation and cancer biology.
- Discuss the potential for EV as liquid biopsies.
Cell-cell crosstalk is central to the regulation of tissues, sustaining physiologic function and often contributing to disease deregulation. Recent studies indicate the existence of lipid bilayer extracellular vesicles (EVs) that traffic protein and RNA between cells and tissues. The selective enrichment of cargo in EV, trafficking in circulation and targeted uptake provide a compelling new signaling paradigm and biomarker platform. Work over the past decade has begun to shed light on key aspects of vesicle biogenesis and intercellular trafficking. With the discovery of their role in hematology and cancer biology recent work has focused on clinical translation.
The Role of EV in Hematopoiesis (Provisional)
Peter Kurre, MD
The Role of EV in Coagulation (Provisional)
Nigel Mackman, PhD
Natural Killer Cell EVs for Immunotherapy of Neuroblastoma and ALL
Robert C. Seeger, MD MS
No relevant financial relationships and no discussion of off-label drug use: Peter Kurre; Nigel Mackman; Robert Seeger
6:30–7:15 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 president Amy Billett, MD will make short remarks and ASPHO’s Board members will be in attendance to greet attendees.
7–8:30 pm
Division Directors’ Dinner Meeting (DDM)
Advanced registration is required. The registration fee is $100.
7:30–9 pm
Non-CME Corporate Forum Dinner Symposium
Thursday, May 3
6:15–7:45 am
Non-CME Corporate Forum Breakfast Symposium
6:30–7:30 am
5K Fun Run/Walk
ASPHO is holding its fifth annual 5K Fun Run/Walk event. Advanced registration is recommended. Registration fee is $25, after May 2, fee is $35.
7–8 am
Small Program Special Interest Group
Leadership: Sarah Vaiselbluh, MD; Andrea Watson, MD MS, Jamie Dargart, MD
Small programs face unique challenges, including the establishment or maintenance of COG membership. Join us for a Q&A session with Dr. Linda Stork from the COG Membership Committee as we explore ways to improve COG clinical trial access for all children, regardless of geographical location or size of treating institution.
No registration required.
Hemoglobinopathy Special Interest Group
Leadership: Howard Grodman, MD; Monica Hulbert, MD
Speaker: Dana LeBlanc, MD MS
The Hemoglobinopathy SIG meeting will focus on new treatments for patients with sickle cell disease, including L-glutamine which gained FDA approval in 2017; pain management, including strategies for acute and chronic pain treatment that members can implement in their practices; and community outreach opportunities.
No registration required.
Palliative Care Special Interest Group
Leadership: Justin N. Baker, MD; Julienne Bracket, MD; Emma Jones, MD; Lawrence Wolfe, MD
Speakers: Justin N. Baker, MD; Julienne Bracket, MD; Emma Jones, MD; Christina Ulrich, MD MPH; Lawrence Wolfe, MD
This year’s Palliative Care SIG meeting will once again include our hematology/oncology version of the “State of the Science.” Time also will be allotted to discussion clinical problems, educational issues, and the growth of the field. Finally, we will discuss ways to make the SIG active between meetings.
No registration required.
8–9:30 am
(B5) Relapsed, Refractory, Metastatic Bone Sarcomas: New Insights on Pathogenesis, Prognosis and Treatment
Moderator: Jessica L. Heath, MD
Objectives:
- Provide a thorough understanding of the molecular biology of metastasis.
- Explain the current and potential future role of liquid biopsy in clinical practice.
- Comprehend diagnostic and treatment options for advanced bony sarcomas beyond current up-front therapy.
Standard chemotherapeutic options for relapsed, refractory and metastatic bone sarcomas are sub-optimal, and outcomes in these patients are poor. Major advances are being made in understanding the molecular mechanisms that drive sarcoma pathogenesis and these scientific discoveries are leading to the development of exciting new therapies. This session will provide an overview of the molecular biology of sarcoma metastasis and an update of the use of correlative biomarkers and genomics to predict disease response. Finally, novel targeted treatment strategies in development based on these scientific advances will be discussed.
The Tumor Microenvironment and Key Molecular Pathways in Metastasis of Bone Sarcoma
Kelly M. Bailey, MD PhD
Turn On, Tune In, Drop Out! LSD1 in Ewing Sarcoma
Stephen L. Lessnick, MD
Liquid Biopsies in the Evaluation of Pediatric Bone Sarcomas
Brian Crompton, MD
No CME will be provided for this session.
(B6) Alpha Thalassemia—A Growing Public Health Disease with Changing Landscape of In-Utero and Post-Natal Therapy
Moderator: Elliott P. Vichinsky, MD
Objectives:
- Summarize the global epidemiologic of alpha thalassemia including its health and economic burden and discuss approaches to prevention, screening, counseling, and control.
- Define molecular and phenotypic expression of alpha thalassemia syndromes including their long term prognosis and describe changes in prenatal diagnosis, intrauterine therapy, the clinical rational for newborn screening and overall post-natal management.
- Explain the status of in utero stem cell therapy and its progress in alpha thalassemia major.
Five percent of the global population has alpha thalassemia. Population migration has increased the birth rate of thalassemia in North America. Hemoglobin H disease, once considered mild, is now recognized to have severe phenotypes that benefit from early diagnosis and treatment. Hydrops fetalis cases are increasing. Advances in fetal therapy with transfusion and in utero stem cell transplantation are possible, resulting in new options and ethical challenges for the family and providers.
A Summary of the Global Epidemiologic Knowledge of Alpha Thalassemia Including Health and Economic Burden and Approaches to Prevention Control and Management
Frédéric B. Piel, PhD
Review of Hemoglobin H and Alpha Thalassemia Major Disorders
Ashutosh Lal, MD
Advances in Management of Alpha Thalassemia Major, including Preclinical and Phase I Studies involving In Utero Stem Cell Therapy for Alpha Thalassemia Major
Elliott P. Vichinsky, MD
Mark Walters, MD
No relevant financial relationships and no discussion of off-label drug use: Elliott P. Vichinsky; Frédéric B. Piel
Relevant financial relationships to disclose and no discussion of off-label drug use: Ashutosh Lal-Novartis Pharmaceuticals, Celgene Corporation: Principal investigator, research grant to institution; La Jolla Pharmaceuticals: Consultant, honoraria; Mark Walters - ViaCord Processing Lab, AllCells, Inc.: Medical Director, research grant; Sangamo Biotherapeutics: Consultant, research grant
(B7) Decision Making in Benign Hematology: Living in a World Without Randomized Trials
Moderator: Cindy E. Neunert, MD MSCS
Objectives:
- Discuss areas of decision-making in benign hematologic conditions, specifically sickle cell disease, ITP, and neutropenia.
- Describe factors that should be considered when discussing treatment options and understand a shared-decision making approach.
While standardized treatment protocols create uniform practice it might be that each patient has unique factors that need to be considered in determining the best management strategy. As we expand our treatment options we often have several therapeutic modalities to offer a patient, however there are rarely randomized trials to guide our decision-making. Applying patient-related outcomes, understanding disease modifiers, and appreciate the patient perspective can start to shape individualized treatment plans. Many hematologists may be unaware of research in this area and how to approach patients with hematologic conditions when there is an absence of evidence-based guidelines.
Shared Decision Making in Sickle Cell Disease
Jane S. Hankins, MD MS
Applying Patient-Related Outcomes in Pediatric ITP
Rachael F. Grace, MD
Treatment Approaches to Rare Neutropenic Conditions
David C. Dale, MD
No relevant financial relationships to disclose; discussion of off-label drug use: Jane S. Hankins; Rachael F. Grace
Relevant financial relationships to disclose; discussion of off-label drug use: Cindy E. Neunert-Genzyme: Consultant, honoraria; David C. Dale-Amgen: Research support, research grant to institution; Amgen: Consultant, honoraria; Sanofi Aventis, X4 Pharmaceuticals, Merck: Principal investigator, research grant to institution
10–11:30 am
Northwestern Mutual Award for Excellence in Childhood Cancer Survivorship Announcement
This year, ASPHO will present the inaugural Northwestern Mutual Award for Excellence in Childhood Cancer Survivorship, funded by the Northwestern Mutual Foundation. In its inaugural year, the award will recognize a pioneer in childhood cancer survivorship – someone who has given shape and credibility to the field through landmark contributions.
This year's award will be presented to Les Robison, MPH PhD, St. Jude Children’s Research Hospital.
Click here for more information about this year's recipient.
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 Julie R. Park, MD.
Improvements in Neuroblastoma Therapy: The Fruits of Collaboration
Neuroblastoma is a heterogeneous malignancy with prognosis ranging from near uniform survival to high risk for fatality. Our understanding of genetic predispositions and tumor biology have not translated into actionable therapies. Randomized clinical trials for high risk neuroblastoma have defined a multi-modal intensive standard therapy including multi-agent conventional chemotherapy, myeloablative chemotherapy and autologous stem cell transplant, radiotherapy and immunotherapy. Despite a doubling of survival from high risk neuroblastoma, better, less toxic therapies are required.
Objectives:
- Review clinical trial results and their impact on high risk neuroblastoma standard therapy.
- Describe revisions to the international response criteria.
- Evaluate novel therapy options for high risk neuroblastoma.
Click here for more information about this year's recipient.
Relevant financial relationships to disclose; discussion of off-label drug use: Julie R. Park-Bristol Myer Squibb: Advisory board member, honoraria
Young Investigator Award Presentations
Moderators: Denise M. Adams, MD; Donald L. Yee, MD MS
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.
(2003) Car T Cells Targeting B7-H3, A Pan-Cancer Antigen, Demonstrate Potent Preclinical Activity against Pediatric Solid Tumors and Brain Tumors
Robbie G. Majzner, MD
Objectives:
- Review the expression of B7-H3 on pediatric tumor tissues and its possible role as an immunotherapeutic target.
- Evaluate novel B7-H3 CAR T cells for the use in pediatric solid tumors and CNS malignancies.
(2004) Natural Killer Cells Targeting the Suppressive Immune Microenvironment of Pediatric Solid Tumors Rescue the Impaired Activity of Car T Cells
Robin Parihar, MD PhD
Objectives:
- Present clinical evidence that the tumor microenvironment contains highly suppressive cells that are increased in patients who fail immune-based therapies.
- Describe an approach to target the suppressive tumor microenvironment by using natural killer (NK) cells that rescue microenvironment-mediated inhibition.
Click here for more information about this year's recipients.
No relevant financial relationships and no discussion of off-label drug use: Robbie G. Majzner; Robin Parihar
11:45 am–12:35 pm
Business Meeting
12:45–2 pm
Early-Career Roundtable Luncheon
Moderator: Mona D. Shah, MD
Objectives:
- Discuss pathways to successfully achieve career development goals.
- Review the experiences of experts in the field.
- 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 $25.
Recommended by the Professional Development Committee.
Basic Science/Translational Research
Douglas K. Graham, MD PhD; Daniel S. Wechsler, MD PhD; Kathleen M. Sakamoto, MD PhD; Betty Pace, MD
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; Guy Young, MD
Clinical Research—Oncology
Linda C. Stork, MD; William G. Woods, MD; Mark P. Atlas, MD; Patrick A. Brown, MD
Foreign Medical Graduates
Sinisa Dovat, MD PhD; Pinki Prasad, MD MPH
Medical Students and Residents
Kimberly Whelan, MD, MSPH; Thomas B. Russell, MD; Maria C. Velez, MD; Emily R. Meier, MD MSHS
Industry
Patrick Zweidler-McKay, MD PhD
No relevant financial relationships and no discussion of off-label drug use: Mona D. Shah; Daniel S. Wechsler; Betty Pace; Timothy P. Garrington; Caroline A. Hastings; Jennifer C. Kesselheim; Jeffrey M. Lipton; George R. Buchanan; Linda C. Stork; William G. Woods; Mark P. Atlas; Patrick A. Brown; Sinisa Dovat; Pinki Prasad; Kimberly Whelan; Thomas B. Russell; Emily R. Meier; Patrick Zweidler-McKay
Relevant financial relationships to disclose and no discussion of off-label drug use: Douglas Graham-Meryx: Board of directors, no compensation received; founder, stock; Kathleen Sakamoto-Acerta: Collaboration, research grant to institution; Amgen: Consultant, honoraria; Maria C. Velez-Bayer: Advisory board member, honoraria; Novo Nordisk, Octapharma: Principal investigator, research grant to institution; Novo Nordisk, CSL Behring: Speaker bureau, honoraria
Relevant financial relationships to disclose; discussion of off-label drug use: Guy Young-Genentech, Novo Nordisk: Advisory board member, honoraria; Alnylam: Consultant, honoraria
12:45–1:45 pm
Non-CME Corporate Forum Lunch Symposium
12:45–1:45 pm
Advanced Practice Providers Special Interest Group
Leadership: Lesley Arland, PA; Nicholette Breier-Adkins, MSN RN CPNP; Kate A. Mazur, MSN RN CPNP
The APP SIG is dedicated to providing a forum for the exchange of ideas and information among APPs and for promotion of their role in the field of pediatric hematology and oncology. At this year’s meeting we will continue to encourage our colleagues from around the country to share their experiences, challenges, and achievements unique to our profession as well as explore educational opportunities for the coming year.
No registration required.
Clinical Immunology Special Interest Group
Leadership: David Buchbinder, MD MS; Shanmuganathan Chandrakasan, MD
The Clinical Immunology SIG meeting will focus on developing ways to incorporate clinical immunology into daily practice. We will focus on education related to the diagnosis and management of immunologic disorders relevant to pediatric hematologists and oncologists. We will also lay the foundation for the development of future collaborative research.
Provided by CHOC Children’s Hospital-UC Irvine through a grant from Leadiant Biosciences.
No registration required.
Global Pediatric Hematology Oncology Special Interest Group
Leadership: Uma Athale, MD; Patrick McGann, MD MS; Andrea Orsey, MD
Speakers include Lawrence Faulkner, MD, who will address “Implementing Accessible Allogeneic Transplant in Low- and Middle-Income Countries,” and Scott Howard, MD, who will speak on “Using Data Systems to Save Lives for Children with Cancer in Low- and Middle-Income Countries.”
No registration required.
2–3:30 pm
(B8) Management of Extracranial Germ Cell Tumors in Adolescent and Young Adult Population
Moderator: Rajkumar Venkatramani, MD MRCPCH MS
Objectives:
- Discuss the approach to a AYA patient with extracranial germ cell tumor.
- Review the latest staging, risk stratification and management schema for extracranial germ cell tumors in adolescent and young adults.
- Review the role of surgery and autologous stem cell transplantation for extracranial germ cell tumors in adolescent and young adults.
Testicular and ovarian germ cell tumors account for 15% of all cancers that occur in patients age 15–19 years. When confronted with an adolescent with extracranial germ cell tumor, pediatric oncologists face the dilemma of which staging system, risk stratification and treatment protocol to use. AYA with extracranial germ cell tumor do consistently worse than children or older adults. This workshop will discuss the current standard of care for these patients. Audience will hear from three leaders in the field. This will be followed by case based discussions that will reinforce the knowledge gained during the talks.
Overview of the MaGIC Consortium: Objectives and Accomplishments
A. Lindsay Frazier, MD ScM
Management of Ovarian and Testicular Germ Cell Tumor in AYA Population
Deborah Billmire, MD
Biology of Germ Cell Tumors
James F. Amatruda, MD PhD
No relevant financial relationships and no discussion of off-label drug use: Rajkumar Venkatramani; Deborah Billmire; James F. Amatruda
Relevant financial relationships to disclose and no discussion of off-label drug use: A. Lindsay Frazier-Decibel Therapeutics: Advisory board member, stock
(B9) Challenging Hematologic Complications Post–Stem Cell Transplantation and Beyond
Moderators: Meret Henry, MD MS; Valerie I. Brown, MD PhD; Dean A. Lee, MD PhD
Objectives:
- Describe the clinical and laboratory manifestations and treatment strategies for TA-TMA/HUS.
- Discuss the approach to autoimmune hemolytic anemia in the transplant setting and otherwise.
- Summarize the new biologic discoveries and novel treatment options for autoimmune thrombocytopenia.
TMA/HUS and autoimmune cytopenias can be seen in previously healthy children and are also rare complications after stem cell transplant. They are often challenging conditions to treat in either setting. Transplant practitioners and hematologists may be unfamiliar with emerging understanding of the pathophysiology, diagnostic criteria and treatment strategies for TMA/HUS and autoimmune cytopenias. They receive little training on this topic. TMA/HUS and autoimmune cytopenias are rare complications after stem cell transplant, but may be life-threatening and difficult both to recognize and manage. Similarly, they may be refractory to frontline therapy, and require additional treatment strategies in non-transplant patients. Data has emerged in recent years regarding the symptoms of TMA/HUS as well as its management. New drugs have also been developed for the treatment of autoimmune cytopenias. It is important for pediatric transplant physicians and hematologists to be aware of these advancements.
Transplant Associated Thrombotic Microangiopathy
Sonata Jodele, MD
Autoimmune Hemolytic Anemia
Jenny M. Despotovic, DO
Autoimmune Thrombocytopenia
Cindy E. Neunert, MD MSCS
No relevant financial relationships and no discussion of off-label drug use: Meret Henry; Valerie I. Brown
Relevant financial relationships to disclose; discussion of off-label drug use: Dean A. Lee-Miltenyi Biotec: Speakers bureau, honoraria; Sonata Jodele-Patent: Inventor, licensing fee related to patent; Jenny M. Despotovic-Sanofi: Advisory board member, honoraria; Cindy E. Neunert-Genzyme: Consultant, honoraria
(B10) Advanced Symptom Management and End-of-Life Care: Multidisciplinary Primary Palliative Care for Pediatric Hematologists/Oncologists
Moderators: Justin N. Baker, MD
Speakers: Scott H. Maurer, MD; Melissa S. Mark, MD; Kathy Perko, MS PNP
Objectives:
- Develop skills for high quality symptom assessment.
- Review up to date evidence for advance symptom management including pain, nausea, and dyspnea.
- Discuss best practices around end-of-life care in pediatric oncology.
Access to specialized pediatric palliative care teams is variable across institutions. Pediatric oncologists are often at the front lines of managing complex symptoms that result from malignancies and treatment. This active and engaging session will use the cutting-edge EPEC-pediatric curriculum led by master facilitators to build skills and confidence in managing symptoms for children with cancer, including but not limited to end-of-life (EOL) care.
No relevant financial relationships and no discussion of off-label drug use: Scott H. Maurer; Melissa S. Mark; Kathy Perko
No relevant financial relationships to disclose; discussion of off-label drug use: Justin N. Baker
3:45–4:45 pm
Paper Session - Solid Tumor
Moderators: Cindy L. Schwartz, MD MPH; Valerie I. Brown, MD PhD
This session will consist of four 15-minute presentations of scientific research from selected solid tumor related abstracts.
Objectives:
- Discuss new approaches related to diagnosis and treatment in pediatric hematology/oncology.
- Examine current research outcomes from a variety of the latest in basic and clinical research.
(2005) Mutational Signatures and Immunophenotype in Recurrent Medulloblastoma: An Indication for Novel Therapeutic Approach
Aditi Bagchi, MD
(2006) Ex Vivo Expanded Multi-Antigen specific Lymphocytes for the Treatment of Solid Tumors
Amy Houghtelin, MD
(2007) Phase 1 Study of the EZH2 Inhibitor, Tazemetostat, in Children with Relapsed or Refractory INI1-Negative Tumors including Rhabdoid Tumors, Epithelioid Sarcoma, Chordoma, and Synovial Sarcoma
Susan Chi, MD
(2008) Changes in Antiemetic Prescribing Practices for Children Receiving Highly Emetogenic Chemotherapy
Marc Rosenman, MD
No relevant financial relationships and no discussion of off-label drug use: Aditi Bagchi, Amy Houghtelin
No relevant financial relationships; discussion of off-label drug use: Marc Rosenman
Relevant financial relationships and no discussion of off-label drug use: Susan Chi-Epizyme: Consultant, travel support
Paper Co-Authors Disclosures
3:45–4:45 pm
Paper Session - Hematology
Moderators: Shelley Crary, MD MS; Cindy E. Neunert, MD MSCS
This session will consist of four 15-minute presentations of scientific research from selected hematology related abstracts.
Objectives:
- Discuss new approaches related to diagnosis and treatment in pediatric hematology/oncology.
- Examine current research outcomes from a variety of the latest in basic and clinical research.
(2009) Neuroprotective Effects of Hydroxyurea in Sickle Cell Anemia: Maintenance of Normal Cerebral Oxygenation
Charles T. Quinn, MD MS
(2010) Outcomes of Unrelated Donor Peripheral Stem Cell Transplantation for Patients with Non-malignant Hematologic Disorders using Two Partial T Cell Depletion Strategies
Joseph H. Oved, MD MA
(2011) Caregiver perceptions of Iron Deficiency Anemia and Iron Replacement Therapies in Young Children with Nutritional Iron Deficiency Anemia
Jacquelyn M. Powers, MD
(2012) Clinical Effect of SC411 (Altemia TM) on Children with Sickle Cell Disease in the Scot Trial: A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Dose-Finding Multi-Center Study
Ahmed Daak, MD MSc PhD
No CME will be provided for this session.
No relevant financial relationships and no discussion of off-label drug use: Charles T. Quinn, Joseph Oved
No relevant financial relationships; discussion of off-label drug use: Jacquelyn Powers
Paper Co-Authors Disclosures
5–6:30pm
(B11) Vascular Anomalies: The Great Mimickers
Moderator: Irina Boyanova Pateva, MD
Objectives:
- Describe the optimal diagnostic strategies when evaluating vascular lesions in infants, children and adolescents.
- Establish which patients warrant imaging, how best to image these lesions, and to describe the differences in imaging characteristics of benign and malignant tumors.
- Identify clinical and radiologic features of vascular lesions suspicious for potential malignancy.
Vascular malformations and tumors are rare conditions. Frequently there is an overlap between the imaging and clinical characteristics of benign and malignant lesions, and accurate diagnosis may be difficult to discern. We review several real-life cases which initially posed diagnostic dilemmas. Subsequently, with the use of multiple diagnostic modalities and multidisciplinary expertise, the Great Mimickers’ diagnoses were revealed. In most cases, the clinical history and key aspects of the physical examination were key to tipping the balance towards suspicion for malignancy. As a resource for pediatric hematologists/oncologists we will provide an algorithm to facilitate the most appropriate approach for such patients.
Visible Vascular Anomalies
Francine Blei, MD
Vascular Lesions of the Visceral Organs and Extremities
Taizo Nakano, MD
When Vascular Anomalies Occur in Syndromes
Irina Boyanova Pateva, MD
No relevant financial relationships and no discussion of off-label drug use: Irina Pateva; Taizo Nakano
Relevant financial relationships to disclose and no discussion of off-label drug use: Francine Blei-Pierre Fabre: Speaker, educational grant .
(B12) Addressing Sexual Health in Adolescent and Young Adult Patients: What You Should Know as a Pediatric Hematology/Oncology Care Provider
Moderator: Natasha Frederick, MD MPH MS
Objectives:
- Describe the importance of sexual health communication in adolescent and young adult cancer patients and survivors.
- Develop strategies for discussing sexual health with AYA cancer patients and survivors.
- Identify specific resources and referrals to support AYAs reporting problems with sexual function.
Adolescent and young adult (AYA) cancer patients and survivors consistently indicate the need for improved provider-patient communication on sexual health topics, including dating, safe sex practices during treatment, contraception, body image, sexuality, fertility, and psychosexual adjustment. Unfortunately, providers often underestimate the relevance of sexual health issues among AYAs and rarely include sexual health in routine conversations through treatment and survivorship. Here we will share data on the sexual healthcare practices and information needs of AYAs, review frameworks for how providers may communicate with AYAs about sexual health issues, and identify appropriate resources and referrals for AYAs reporting sexual dysfunction.
Painting the Picture: Why We Need to Discuss Sexual Health with AYA Hematology and Oncology Patients
Abby R. Rosenberg, MD MS MA
The Conversation: How to Discuss Sexual and Reproductive Health with Adolescent and Young Adult Patients
Natasha Frederick, MD MPH MS
Sexual Dysfunction in AYA Patients: Problems Encountered and What to Do
Sharon Bober, PhD
No relevant financial relationships and no discussion of off-label drug use: Natasha Frederick; Abby R. Rosenberg; Sharon Bober
(B13) Navigating Intravascular Bottlenecks: A Critical Mini-Review of Pediatric Venous Thromboembolism in the Setting of an Anatomical Thrombophilia
Moderator: Riten Kumar, MD MSC
Objectives:
- Describe the prevalence, pathophysiology and clinical presentation of VTE in children with anatomical thrombophilias, namely May-Thurner syndrome, Paget-Schroetter syndrome and congenital IVC anomalies.
- Assess the quality of existing evidence to make recommendations about the initial management and duration of therapy for VTE in these cohorts, focusing on the role of thrombolysis.
- Review long-term outcomes including post-thrombotic syndrome in children with anatomic thrombophilia and VTE.
Anatomical conditions like May-Thurner syndrome, Paget-Schroetter syndrome and congenital Inferior Vena Cava (IVC) anomalies (absence, interruption and/or hypoplasia) are associated with venous thrombo-embolism (VTE). Given the rarity of these conditions, making evidence-based recommendations on appropriate diagnostic and therapeutic strategies can often be challenging. Additionally, the relevance of upfront thrombolytic therapy in these conditions is unclear. This case-based, interactive session will equip participants with up-to-date, evidence-based knowledge on the clinical-presentation, diagnosis and management of VTE in children with anatomical thrombophilia, using a focused mini-review approach.
Diagnosis and Management of Paget-Schroetter Syndrome
Riten Kumar, MD MSC
Diagnosis and Management of May-Thurner Syndrome
Leonardo R. Brandao, MD MSC
Diagnosis and Management of Congenital IVC Anomalies (e.g., Absence, Interruption, and Hypoplasia)
Cristina Tarango, MD
Update on Advances in Thrombolytic Therapy
Patrick Warren, MD
No relevant financial relationships and no discussion of off-label drug use: Riten Kumar, Patrick Warren
No relevant financial relationships to disclose; discussion of off-label drug use: Leonardo R. Brandao
Relevant financial relationships to disclose; discussion of off-label drug use: Cristina Tarango-Bayer: Consultant, honoraria
6:30–7:30 pm
Author-Attended Poster Session A: Odd-numbered Posters
6:30–7:30 pm
Meet the SIGs
Learn about ASPHO’s Special Interest Groups at this informal Meet & Greet. Learn which specialized community matches your interests and can offer resources and insights to support your work.
6:30–8 pm
Opening Reception with Exhibits and Posters
7-7:15 pm
Awardee Spotlight @ ASPHO Booth
Featuring Julie Park, MD, Frank A. Oski Memorial Lectureship recipient. Join an informal conversation with Dr. Park as she shares insights from her career.
7:30–9 pm
Fellowship Program Directors’ Dinner Meeting (PDD)
Advanced registration is required. The registration fee is $85.
7:30–9:00 pm
Non-CME Corporate Forum Dinner Symposium
Westin
Learn more
Friday, May 4
6-7:30 AM
Non-CME Corporate Forum Breakfast Symposium
7–8 am
Continental Breakfast with Exhibits and Posters
7 am–2:45 pm
Exhibits and Posters in Exhibit Hall
7:30–8 am
Even-Numbered Poster Tour
ASPHO will provide two poster tours during exhibit hours. The Conference Planning Committee will select five highly ranked posters and provide 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 plan to meet at the Exhibit Hall entrance and look for a member of the Conference Planning Committee 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:
(#204) MDS AND AML IN SHWACHMAN DIAMOND SYNDROME: CLINICAL FEATURES AND OUTCOMES
Kasiani Myers, MD
(#338) COMPARATIVE EFFECTIVENESS OF A PATIENT DECISION AID FOR THERAPEUTIC OPTIONS FOR SICKLE CELL DISEASE
Lakshmanan Krishnamurti, MD
(#342) HYDROXYUREA USE IN INFANTS WITH SICKLE CELL DISEASE: THE EARLIER THE BETTER
Stephen Nelson, MD
(#526) CHANGING BLOOD CULTURE STRATEGY: A QUALITY IMPROVEMENT PROJECT TO ELIMINATE THE PRACTICE OF AUTOMATIC DAILY BLOOD CULTURES IN HOSPITALIZED PEDIATRIC PATIENTS WITH FEVER AND NEUTROPENIA
Joanna Grossman Newton, MD MSc
(#718) THE UNFOLDED PROTEIN RESPONSE AS A PREDICTOR OF CHEMOSENSITIVITY IN PEDIATRIC ACUTE MYELOID LEUKEMIA
Saman Hashmi
8–9 am
Paper Session - Children's Oncology Group (COG)
Moderators: Karen E. Effinger, MD MS; Christopher Porter, MD
This session will consist of four-15 minute presentations of high impact results, promising studies or other initiatives recently presented at COG or other national/international meetings.
Objectives:
- Discuss new approaches related to diagnosis and treatment in pediatric hematology/oncology.
- Examine current research outcomes from a variety of the latest in basic and clinical research.
(2013) COG Developmental Therapeutics: Moving New Therapies Forward
Brenda J. Weigel, MD FAAP
(2014) Targeted Therapies in NHL
Birte Wistinghausen, MD
(2015) Survivorship: Going from Observation to Intervention
Eric Chow, MD MPH
(2016) Novel Therapeutics, Endpoints, and Outcomes in Hodgkin Lymphoma
Sharon Castellino, MD FAAP MSc
No relevant financial relationships to disclose; discussion of off-label drug use: Brenda J. Weigel, Birte Wistinghausen, Eric Chow, Sharon Castellino
Paper Co-Authors Disclosures
8–9 am
Paper Session - Hematology Science
Moderators: Jessica L. Heath, MD; Rochelle Maxwell, MD
This session will consist of four-15 minute presentations of hematology science - two from the 2018 Hemostasis and Thrombosis Research Society's meeting and two from Hematology Science as presented at the 2017 ASH Annual Meeting.
Objectives:
- Discuss new approaches related to diagnosis and treatment in pediatric hematology/oncology.
- Examine current research outcomes from a variety of the latest in basic and clinical research.
(2017) Prenatal Transplantation of Placental Cells Engineered to Express FVIII Leads to Corrective Plasma Levels of FVIII after Birth
Graça D. Almeida-Porada, MD PhD
(2018) Understanding the Use of Primary Thromboprophylaxis in Hospitalized Pediatric Patients
Lauren Amos, MD
(2019) Storage Pool Deficiency: Atypical Labs and Diagnostic Trends
Nate Nessle, DO
(2020) Customized Induced Pluripotent Stem Cell-Derived Red Cell Reagents
Stella Chou, MD
No relevant financial relationships to disclose and no discussion of off-label drug use: Graça D. Almeida-Porada, Lauren Amos, Nate Nessle, Stella Chou
Paper Co-Authors Disclosures
9:15–10:45 am
(C14) Can Late Effects be Prevented in Pediatric Oncology?
Moderators: Karen E. Effinger, MD MS; Cindy L. Schwartz, MD MPH
Objectives:
- Identify medications that can be used to prevent toxic late effects in pediatric oncology patients.
- Describe the benefit of exercise and early physical therapy in prevention of late effects in pediatric oncology patients.
- Describe methods to help prevent neurocognitive decline in at risk pediatric oncology patients.
Pediatric oncologic condition survivors are at increased risk for developing significant late effects of their treatment leading to morbidity and mortality. Recent research has explored methods to decrease the risk of late effects through adaptations to therapy made during treatment. These adaptations include medications to prevent toxic side effects, early use of physical therapy/exercise, and neurocognitive training. Many oncologists are unaware of the new research in this field that may impact their patients.
Medications That Can Prevent Long-Term Toxicity
Eric Chow, MD MPH
Exercise and Early Physical Therapy to Prevent Late Effects
Nina S. Kadan-Lottick, MD MSPH
Prevention of Neurocognitive Decline in at-Risk Pediatric Oncology Patients
Kristina Hardy, PhD
No relevant financial relationships and no discussion of off-label drug use: Karen E. Effinger; Cindy L. Schwartz; Kristina Hardy
No relevant financial relationships to disclose; discussion of off-label drug use: Eric Chow
Relevant financial relationships to disclose and no discussion of off-label drug use: Nina S. Kadan-Lottick-Medtronic: Consultant, honoraria
(C15) Clash of the Titans: Debates on Best Management of Patients with Bleeding Disorders
Moderator: Donald L. Yee, MD MS
Debaters: Manuel Carcao, FAAP FRCPC MD; Ellis J. Neufeld, MD PhD
Objectives:
- Choose among available factor concentrates to use as first-line therapy for previously untreated patients and those undergoing immune tolerance induction.
- Debate the utility of imaging studies to routinely follow joint health in patients with hemophilia.
- Articulate sound evidence-based recommendations regarding participation in sports activities for patients with bleeding disorders.
Despite a steady stream of new products and clinical trial results in recent years, best practices in a number of domains for patients with bleeding disorders remain indeterminate and controversial. Providers must understand the rationale and currently available data underlying distinct and often polarized management approaches to make informed medical decisions about therapies, diagnostic tools and activity recommendations. Such decision making must include consideration of critical factors such as patient convenience, safety, overall health, and quality of life. This spirited debate session will feature opposing expert speakers assigned dogmatic positions on controversial topics and include time for rebuttals and counter-rebuttals.
No relevant financial relationships and no discussion of off-label drug use: Manuel Carcao
No relevant financial relationships to disclose; discussion of off-label drug use: Donald L. Yee
Relevant financial relationships to disclose; discussion of off-label drug use: Ellis J. Neufeld-Genentech, Octapharma: Consultant, honoraria; Novo Nordisk: Advisory board member, honoraria
(C16) Providing Effective Feedback: The Currency of Conflict Resolution
Moderators: Vandy Black, MD MSC; Michael Ferguson, MD MS
Speaker: Jo Shapiro, MD
Objectives:
- Identify barriers to resolving conflicts among team members and providing effective feedback.
- Develop skills for using frame-based feedback as an effective form of evaluation and conflict resolution.
- Apply the skills of frame-based feedback to relationship building on all levels.
Pediatric hematologists/oncologists are often provided opportunities to direct multidisciplinary clinical and academic teams. Thus, it is imperative that we are prepared to address conflicts between team members, which may arise from a lack of clear expectations. This session, sponsored by the Professional Development Committee, will focus on understanding the barriers to resolving conflict among team members, and developing skills for using frame-based feedback as an effective tool to conflict resolution. Attendees will work together during an interactive workshop applying the skills of frame-based feedback to foster relationship building on all levels.
No relevant financial relationships and no discussion of off-label drug use: Vandy Black; Michael Ferguson
Relevant financial relationships to disclose and no discussion of off-label drug use: Jo Shapiro-Safe and Reliable Health Care, LLC: Independent contractor, fee
10:45–11:45 am
Break with Exhibits and Posters
10:45–11:15 am
Odd-Numbered Poster Tour
ASPHO will provide two poster tours during exhibit hours. The Conference Planning Committee will select five highly ranked posters and provide 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 plan to meet at the Exhibit Hall entrance and look for a member of the Conference Planning Committee 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:
(#207) INTERROGATING THE HUMAN NEUTROPHIL GRANULOCYTE IN MONOGENIC DISEASE BY NEXT GENERATION PROTEOMICS
Sebastian Hesse
(#403) CHARACTERIZING ADOLESCENTS WITH HEAVY MENSTRUAL BLEEDING AND GENERALIZED JOINT HYPERMOBILITY
Nicole Kendel, MD
(#605) INCIDENCE RATES AND TRENDS OF PEDIATRIC CANCER — UNITED STATES, 2001–2014
David Siegel
(#725) CONTINUED IMPROVEMENTS IN OVERALL SURVIVAL (OS) IN CHILDREN WITH NEWLY DIAGNOSED ACUTE LYMPHOBLASTIC LEUKEMIA (ALL): A CHILDREN’S ONCOLOGY GROUP (COG) REPORT
Elizabeth Raetz, MD
(#809) NONINVASIVE MOLECULAR PROFILING BY CIRCULATING CELL-FREE DNA IN PEDIATRIC SOLID TUMORS
Prachi Kothari
11–11:35 am
Speed Mentoring-Session I
Exhibit Hall
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 members registering for these sessions 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 in Pittsburgh that addresses the focus of the speed mentoring session's topic of interest. Mentees should come prepared with two to three 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 the two Speed Mentoring sessions.
11:15–11:45 am
11:45 am–1:15 pm
(C17) Recent Advances and New Approaches to Rare Hematological Malignancies
Moderator: Nobuko Hijiya, MD
Objectives:
- Discuss clinical practice and issues in pediatric CML.
- Identify common endocrine effects of TKI drugs used in pediatric CML patients, and devise plans to screen at risk patients.
- Gain proficiency in the workup and diagnostic criteria of pediatric MDS and differentiation from other bone marrow failure disorders and understand genetic predisposition that can lead to pediatric MDS.
- Describe the clinical presentation, biology, treatment and outcome of TAM in Down syndrome newborns.
Although chronic myeloid leukemia (CML), myelodysplastic syndrome (MDS) and transient abnormal myelopoiesis (TAM) of Down’s syndrome are rare diseases, they are still encountered routinely by pediatric oncologists. The treatment landscape and emerging management strategies of these diseases have changed significantly in recent years but standard care is not well established. Recent advances in biology and in clinical care will be presented in this symposium.
Pediatric CML: A Unique Disease That Requires a Different Approach
Nobuko Hijiya, MD
Endocrine effects of TKI's in Pediatric CML Patients
Briana Patterson, MD MSCR
Clinical Features and Molecular Genetics of Pediatric MDS
Inga Hofmann, MD PhD
Transient Abnormal Myelopoiesis: The “Disappearing” Leukemia of Down’s Syndrome
Johann Hitzler, MD
No relevant financial relationships and no discussion of off-label drug use: Briana Patterson; Inga Hofmann; Johann Hitzler
Relevant financial relationships to disclose and no discussion of off-label drug use: Nobuko Hijiya-Novartis: Data monitoring committee, honoraria
(C18) Primary and Secondary Stroke Prevention in Sickle Cell Disease: The Debate
Moderators: Rochelle Maxwell, MD; Clifford Takemoto, MD
Objectives:
- Discuss the pros and cons of HU versus transfusions for primary stroke prevention.
- Discuss the pros and cons of BMT versus transfusions for secondary stroke prevention.
Patients with sickle cell disease are at increased risk for stroke. Chronic PRBC transfusions are an effective therapy for both secondary stroke prevention and primary stroke prevention with an abnormal TCD. Bone marrow transplantation is potentially curative and is also therapeutic option for sickle cell patients with stroke. In addition, the recently published TWiTCH trial demonstrated that hydroxyurea can be substituted for chronic transfusions for primary stroke prevention. Therapies such as BMT or HU may be appropriate for selected patients therefore it is important that pediatric hematologists are aware of the appropriate indications for each.
Debate #1: HU versus Chronic PRBC Transfusion for Primary Stroke Prevention (Pro HU)
Russell E. Ware, MD PhD
Debate #1: HU versus Chronic PRBC Transfusion for Primary Stroke Prevention (Pro Transfusion)
Deepa Manwani, MD
Debate #2: Chronic PRBC Transfusion versus BMT for Secondary Stroke Prevention (Pro BMT)
Michael R. DeBaun, MD MPH
Debate #2: Chronic PRBC Transfusion versus BMT for Secondary Stroke Prevention (Pro Transfusion)
Elliott P. Vichinsky, MD
No relevant financial relationships and no discussion of off-label drug use: Rochelle Maxwell; Michael R. DeBaun; Elliott P. Vichinsky
No relevant financial relationships to disclose; discussion of off-label drug use: Deepa Manwani
Relevant financial relationships to disclose; discussion of off-label drug use: Russell E. Ware-Global Blood Therapeutics: Advisory board member, honoraria; Agios: Consultant, honoraria; Clifford Takemoto-Novo Nordisk, Alnylam: Principal investigator, research grant to institution; Novartis: DSMB, fee
(C19) Combating an Unmet Need: Psychosocial Health
Moderator: Kate A. Mazur, MSN RN CPNP
Objectives:
- Develop an understanding for the need of early and effective psychosocial intervention and care by a review of the current literature.
- Discuss strategies for risk assessment and models of referral for psychosocial services.
- Review most commonly used pharmacological and non-pharmacological treatment strategies.
Children with cancer and their caregivers experience a significant amount of psychological distress throughout the disease trajectory. Access to psychosocial services is needed to decrease this distress and improve overall quality of life. Depression, anxiety, behavioral problems, and other psycho-somatic symptoms are frequently overlooked or suboptimally treated due to a primary focus on anti-cancer therapy. It is imperative to develop improved delivery of multi-disciplinary psychosocial care. This session aims to provide the skills necessary to adequately screen 'at risk' patients and families, and offers a basic understanding of interventions to effectively care for this patient population.
Background and Support of Need for Effective Psychosocial Interventions
Lori Wiener, PhD
Warning Signs and Interventions: A Psychologist’s Perspective
Meghan Marsac, PhD; Rachel Thienprayoon, MD
Sibling Grief: An Area Often Ignored
Mary Jane Gandour, PhD; Molly Gandour, MFA Candidate
No CME will be provided for this session.
1:15–2:15 pm
Author-Attended Poster Session B: Even-numbered Posters
1:30–1:45 pm
Awardee Spotlight @ ASPHO Booth
Featuring Stephen Hunger, MD, George R. Buchanan Lectureship Award recipient. Join an informal conversation with Dr. Hunger as he shares insights from his career.
1:15–2:45 pm
Lunch with Exhibits and Posters
1:45–2:20 pm
Speed Mentoring-Session II
Exhibit Hall
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 members registering for these sessions 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 in Pittsburgh that addresses the focus of the speed mentoring session's topic of interest. Mentees should come prepared with two to three 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 the two Speed Mentoring sessions.
1:45–2:30 pm
2:45–4:15 pm
(C20) Renal Tumors Beyond Wilms
Moderator: James I. Geller, MD
Objectives:
- Describe up-to-date diagnostic and treatment approaches for rare renal tumors: Rhabdoid, clear cell carcinoma of the kidney, and renal cell carcinoma.
- Discuss strategies related to drug development and trial design for rare diseases and opportunities for national and international collaboration.
Pediatric oncologists at most centers do not have the volume to develop expertise in rare renal tumors and keep up-to-date on current protocols, clinical trials, and new therapeutic horizons. Leading experts will give pediatric oncologists the best knowledge to appropriately counsel patients for rare diagnoses they may only see every 1–2 years. This presentation will expand their knowledge base and develop an opportunity to network with world experts in this field.
Renal Rhabdoid Tumors
James I. Geller, MD
Renal Cell Carcinoma
Nicholas G. Cost, MD
Clear Cell Carcinoma of the Kidney
Marry van den Heuvel-Eibrink, MD PhD
This session is supported in part by the International Society of Paediatric Oncology.
No relevant financial relationships and no discussion of off-label drug use: James Geller; Nicholas Cost; Marry van den Heuvel-Eibrink
(C21) Pediatric and Neonatal Transfusion Medicine
Moderator: Ziad Solh, MD
Objectives:
- Review red blood cell alloimmunization in transfusion and pregnancy scenarios, recipient variables impacting alloimmunization, and strategies for prevention.
- Discuss the impact of red blood cell storage time on outcomes in critically ill children.
- Discuss current controversies in necrotizing enterocolitis (NEC) and its potential associations with red cell transfusion and anemia in premature neonates, and to explain potential mechanisms of anemia leading to NEC.
Blood transfusion practice in pediatric and neonatal patients is identified to be in urgent need of evidence development. Data from adult studies are available but cannot be extrapolated to pediatric populations. Novel pediatric data are shedding light on best transfusion practices recommended for the blood supplier and the pediatric practitioner.
The audience will learn about novel concepts in transfusion safety from transfusion medicine experts. Topics will include red cell alloimmunization, storage age of blood, and predisposing factors to neonatal morbidities. The audience will have an opportunity to interact with the speakers, ask questions, and debate concepts.
Red Blood Cell Alloimmunization in At-Risk Children
Jeanne Hendrickson, MD
The Impact of Red Blood Cell Storage Time on Critically Ill Children
Marisa Tucci, MD
New Insights into Anemia of Prematurity, Transfusions, and Neonatal Morbidities
Cassandra Josephson, MD
No relevant financial relationships and no discussion of off-label drug use: Ziah Solh; Jeanne Hendrickson; Marisa Tucci
Relevant financial relationships to disclose and no discussion of off-label drug use: Cassandra Josephson-Immucor, Biomet Zimmer, Octapharma: Consultant, honoraria
(C22) Mobile Applications in Clinical Practice
Moderator: Christopher Porter, MD
Objectives:
- Discuss a mobile application to track pain and mood developed for the Adolescent and Young Adult population that may facilitate engagement in health promoting behaviors.
- Discuss the development of a mobile application to detect anemia.
- Discuss the use of wearables to improve clinical care in patients with pain.
Mobile technology and social networks are pervasive in our lives, but are not routinely used by providers to promote health. Given that we are in the 21st century and the majority of our patients have and use mobile technology, it is important as clinicians to stay current and up-to-date on mobile applications that can help and improve the care that we deliver. Mobile technology offers the opportunity to engage patients in unprecedented ways. Pediatric hematologists/oncologists can benefit from employing existing technologies.
Mobile Applications in Clinical Practice
Christopher Porter, MD; Lori E. Crosby, PsyD; Wilbur Lam, MD PhD; Nirmish Shah, MD
No relevant financial relationships and no discussion of off-label drug use: Christopher Porter; Lori Crosby
Relevant financial relationships to disclose and no discussion of off-label drug use: Wilbur Lam-Sanguina LLC: Board of directors, equity; Nirmish Shah-Novartis, Alexion: Speaker bureau, honoraria
4:30–6 pm
(C23) Presidential Symposium—Partnering with Patients and Families to Improve Research and Clinical Decision-making
Moderators: Amy L. Billett, MD; Katie Greenzang, MD EdM
Objectives:
- Describe the value of engaging patients/families in research from project conception through implementation.
- Identify the key elements of Shared Decision Making and its application in pediatrics.
The goal of this presidential symposium is to enable PHO providers to improve both research and clinical outcomes by fostering partnerships with patients and families in conducting patient-centered research, and by engaging in shared decision making for clinical care. Patient and family involvement in the design of clinical trials can further research through identification of novel research questions, effective research strategies, and impactful outcomes. Similarly, elucidating and incorporating family values in medical decision making can lead to meaningful improvements in outcomes and care.
Speakers:
Ethan Basch, MD; Eric D. Kodish, MD
No relevant financial relationships and no discussion of off-label drug use: Amy L. Billett; Katie Greenzang; Ethan Basch
Relevant financial relationships to disclose and no discussion of off-label drug use: Eric D. Kodish-Solid Bio: Consultant, honoraria
6–7 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 Michael Link, MD, this year’s winner, and is followed by a reception to congratulate him on the award.
Click here for more information about this year's recipient.
7–8 pm
Battle of the Superstars
Back by popular demand, Guy Young will host a competitive game show. Join your friends and colleagues for good laughs as two teams compete for winning bragging rights.
Saturday, May 5
7–8 am
Diversity Special Interest Group
Leadership: Jacqueline Casillas, MD MSHS; Emily R. Meier, MD MSHS; Kerice Pinkney, MD
Organizational Capacity Building: Using Diversity as a Driver of Excellence
During the meeting, we will identify diversity-related gaps and challenges in academic medicine from which we can improve our pediatric hematology/oncology professional Society’s organizational ability to use diversity as a driver of excellence. Followed by an attendee-driven exercise to discuss diversity-related gaps/challenges that span the academic spectrum from basic science to clinical educator.
No registration required.
Vascular Anomalies Special Interest Group
Leadership: Francine Blei, MD; Cameron Trenor, MD
The meeting will provide updates from the Research, Practice, and Education Committees about ongoing studies and brainstorm for new projects. Interesting cases will be presented.
No registration required.
Physician Scientists Special Interest Group
Leadership: Sinisa Dovat, MD; Kathleen M. Sakamoto, MD PhD
Assessing the Needs of Physician Scientists and Developing Solutions
Kathleen Sakamoto, M.D., Ph.D., Ernest Frugé, Ph.D., and Sinisa Dovat, M.D. will discuss the background, survey results, and goals of the SIG. Panel members Alejandro Sweet-Cordero, M.D., Smita Bhatia, M.D., M.P.H., Steve Hunger, M.D., and Betty Pace, M.D. will discuss issues in basic/translational oncology, population science, clinical research, and translational hematology.
No registration required.
6:45–8:15 am
Continental Breakfast
8–9:30am
(D24) Review of Rare Pediatric Head and Neck Tumors for the General Oncologist
Moderator: Winston W. Huh, MD
Objectives:
- Describe the epidemiology and clinical presentation of the presented rare head and neck tumors in pediatric patients.
- Discuss updated treatment trends for the presented rare head and neck tumors in pediatric patients.
- Describe potential treatment-related effects for these patients.
The head and neck region is the primary site for approximately 10% of all pediatric malignant tumors, yet the differential diagnosis is quite broad. There are several types of malignant head and neck tumors that are quite rare in the pediatric population, thus, many pediatric oncologists may lack experience in recognizing and treating these tumors. This session will provide an update so that the general oncologist will become familiar with the clinical presentation, epidemiology and treatment of these diagnoses.
Update on Salivary Gland Tumors in Children
Winston W. Huh, MD
Update on Nasopharyngeal Carcinoma in Children
Carlos Rodriguez-Galindo, MD
Update on Esthesioneuroblastoma in Children
Matthew Ladra, MD MPH
Case Presentations
Brianna Murphy, MD
No relevant financial relationships and no discussion of off-label drug use: Winston W. Huh; Carlos Rodriguez-Galindo; Brianna Murphy
Relevant financial relationships to disclose and no discussion of off-label drug use: Matthew Ladra-Proknow: Consultant, honoraria
(D25) Emerging Therapies in Hemophilia
Moderator: Shelley Crary, MD MS
Objectives:
- Examine the real-world implementation strategies for extended half-life factor products including pharmacokinetic approaches.
- Describe the current experience and future role of gene therapy for Hemophilia A and B.
- Discuss emerging non-factor therapies for patients with hemophilia, including those with inhibitors.
The management of hemophilia is experiencing rapid progress. Pediatric hematologists may have difficulty staying abreast of therapies and know when and how to implement them into clinical practice. Newer extended half-life factor products are available, but understanding when and how to use these products may be challenging. Gene therapy and non-factor therapies are all on the horizon. An update of new and emerging therapies will aid in the understanding of how to implement them into clinical practice, including which population would benefit and how to monitor these new products.
Practical Approach to Utilizing Extended Half-Life Products in Hemophilia
Jennifer Maahs, RN-BC MSN PNP
Update on Gene Therapy for Hemophilia A and B
Ulrike M. Reiss, MD
Emerging Nonfactor Therapies for Hemophilia
Guy Young, MD
No relevant financial relationships and no discussion of off-label drug use: Shelley Crary; Ulrike M. Reiss
Relevant financial relationships to disclose and no discussion of off-label drug use: Jennifer Maahs-Shire, Genentec, CSL: Advisory board member, honoraria; Bioverativ, Novo Nordisk: Speaker bureau, honoraria
Relevant financial relationships to disclose; discussion of off-label drug use: Guy Young-Genentech, Novo Nordisk: Advisory board member, honoraria; Alnylam: Consultant, honoraria
(D26) How Should You Treat That Bug? A Debate About Antimicrobial Management in Fever and Neutropenia
Moderators: Karen E. Effinger, MD MS; Valerie I. Brown, MD PhD
Objectives:
- Categorize fever/neutropenia (FN) patients as low-risk or high-risk at presentation.
- Determine options for ongoing management of FN including when to broaden/narrow coverage or stop empiric antibiotics.
- Describe the recommendations surrounding prophylactic anti-fungals.
Fever and neutropenia is a common complication in pediatric oncology that may lead to significant morbidity and mortality; however, antimicrobial management is not standardized across institutions. Infectious disease physicians and oncologists often differ in their approach to antimicrobial management. Few oncologists know the data behind the practices at their institution. This session seeks to inform members of our community about updated guidelines for the management of fever and neutropenia in pediatric oncology.
Oncology Perspective
Lillian Sung, MD PhD; Adam Esbenshade, MD
Infectious Disease Perspective
Brian T. Fisher, DO MSCE; Theoklis Zaoutis, MD
No relevant financial relationships and no discussion of off-label drug use: Karen E. Effinger; Valerie I. Brown; Lillian Sung, Theoklis Zaoutis
No relevant financial relationships to disclose; discussion of off-label drug use: Adam Esbenshade
Relevant financial relationships to disclose; discussion of off-label drug use: Brian T. Fisher: Pfizer/Merck: Principal Investigator, research grant to my institution
9:45-11:15 am
Paper Session with Luminary Investigator - Leukemia
Moderators: Linda McAllister-Lucas, MD PhD; Nobuko Hijiya, MD
Dr. Stephen Hunger will present "Development of Precision Medicine Therapies for Pediatric Acute Lymphoblastic Leukemia" followed by four 15-minute presentations of scientific research from selected leukemia related abstracts.
Objectives:
- Discuss new approaches related to diagnosis and treatment in pediatric hematology/oncology.
- Examine current research outcomes from a variety of the latest in basic and clinical research.
(2021) The Six1 Homeobox Gene is a Novel CRM1-Dependent Target in Calm-AF10 Leukemias
Waitman Aumann, MD
(2022) Transcriptional Co-Regulation by Ikaros and RUNX1 in Myeloid Leukemia
Jessica L. Heath, MD
(2023) Novel Biologic for the Treatment of PH-like B-Cell Acute Lymphoblastic Leukemia with over Expression of CRLF2
Kimberly Payne, PhD
(2024) Universal Pretreatment for Asparaginase-Based Therapy should be Standard of Care
David J. Young, FAAP MD PhD
No relevant financial relationships to disclose; no discussion of off-label drug use: Waitman Aumann, David J. Young
No relevant financial relationships to disclose; discussion of off-label drug use: Jessica L. Heath
Relevant financial relationships to disclose; no discussion of off-label drug use: Kimberly Payne-Elf Zone, Inc.: Stock holder, stock
Relevant financial relationships to disclose; discussion of off-label drug use: Stephen Hunger-Novartis: Consultant, honoraria; Amgen: Stock; Jazz Pharmaceuticals, Erytech: Advisory board member, honoraria
Paper Co-Authors Disclosures
9:45-11:15 am
Paper Session with Luminary Investigator - Hematology
Moderators: Clifford Takemoto, MD; Dana Matthews, MD
Dr. Diane Nugent will present "Finding 'True North': Research and Clinical care pathways in Pediatric Hematology Oncology Past and Present" followed by four 15-minute presentations of scientific research from selected hematology related abstracts.
Objectives:
- Discuss new approaches related to diagnosis and treatment in pediatric hematology/oncology.
- Examine current research outcomes from a variety of the latest in basic and clinical research.
(2025) Mechanisms Linking Fibrin (ogen) Structure/Function to Tumor Metastasis
Allison S. Remiker, MD
(2026) Safety and Patient-Reported Efficacy of Sirolimus in 120 Patients with Vascular Anomalies: A Report from the Lymphatic Anomalies Registry
Cameron C. Trenor, MD
(2027) Peripheral Arterial Thrombosis in Hospitalized Children: Characteristics and Short-Term Outcomes of Extended Anticoagulation
Julie Weisman, MD
(2028) Platelet Ontogeny: A Closer Look at Platelet Immune Function
Akua Asante, MD
No relevant financial relationships and no discussion of off-label drug use: Diane Nugent, Allison S. Remiker, Julie Weisman, Akua Asante
Relevant financial relationships to disclose; discussion of off-label drug use: Cameron C. Trenor-Novartis: Employee, salary; Boehringer-Ingelheim/Pfizer/Daichii-Sankyo: Principal Investigator, self, research grant to institution
Paper Co-Authors Disclosures
11:30-1:30 pm
(MOCS) Maintenance of Certification (MOC) Learning Session
Moderator: Glen Lew, MD
Speakers: Eric Bouffet, MD; Eric Chow, MD MPH; Lakshmanan Krishnamurti, MD; Leslie Lehmann, MD; Ellis J. Neufeld, MD PhD; Sarah H. O’Brien, MD MS; Akiko Shimamura, MD PhD; Guy Young, 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 2018 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 drug use: Glen Lew; Lakshmanan Krishnamurti; Leslie Lehmann; Sarah H. O’Brien; Akiko Shimamura
No relevant financial relationships to disclose; discussion of off-label drug use: Eric Chow
Relevant financial relationships to disclose; discussion of off-label drug use: Eric Bouffet-Roche, BMS: Principal Investigator, research grant to institution; Guy Young-Genentech, Novo Nordisk: Advisory board member, honoraria; Alnylam: Consultant, honoraria; Ellis J. Neufeld-Genentech, Octapharma: Consultant, honoraria; Novo Nordisk: Advisory board member, honoraria