Conference Schedule
Tuesday, April 2
6:30 am – 8:30 pm
Pediatric Transplantation and Cellular Therapy Consortium (PTCTC) Meeting
Wednesday, April 3
7:30 am – 11:30 am
Pre-Conference Session - Pediatric Vascular Anomalies: From Molecular Diagnostics to Clinical Change
Sponsored by Novartis
The ASPHO Vascular Anomalies SIG presents an educational primer for providers and trainees on clinical, radiographic, and molecular updates in pediatric vascular anomalies. The primer will cover board review material and go further to equip pediatric hematologists/oncologists with knowledge on how to recognize common vascular anomalies based on their clinical features and presentation, as well as the availability of advanced diagnostic techniques and multi-modality treatment approaches including medical therapies.
Learning Objectives:
- Review current approaches to diagnosis and management of vascular anomalies, discuss the differential diagnosis using a multi-disciplinary and multi-modal treatment approach
- Review conventional and advanced imaging modalities, review interventional radiology techniques utilized for the treatment of vascular anomalies
- Review current understanding of molecular pathways and molecular testing in the pathophysiology and diagnosis of vascular anomalies
Moderator:
- Classifying Vascular Anomalies
- Taizo Nakano, MD
Speakers:
- Dynamic Vascular Tumors
- Sally J. Cohen-Cutler, MD MS
- Dynamic Vascular Malformations
- Kiersten Ricci, MD
- Advances in Molecular Diagnostics
- Jimmy Bennet, MD
- Targeted Pharmacotherapy for Vascular Anomalies
- Whitney Eng, MD MPH
- Comprehensive Care and Quality of Life
- Bryan Sisk, MD MS
- IR/Surgical/Derm
- Matthew Hawkins, MD
- Coagulopathies of Vascular Anomalies
- Joana Mack, MD
10:30-11:30 am
Vascular Anomalies SIG Meeting
The Vascular Anomalies SIG will meet at the conclusion of the Pre-Conference Primer.
11:30 am – 12:30 pm
Satellite Symposium: Precision & Progress Against NF1: Solutions for Better Outcomes With MEKi & Multimodal Care for NF1 PNs and Other Tumors
Satellite Symposium: Mastering Asparaginase Regimens in the Care of Pediatric and AYA Patients With Acute Lymphoblastic Leukemia
12:45 pm – 2:05 pm
Welcome, Plenary Papers, and Young Investigator Award Presentations
Welcome
President, Caroline Hastings, MD, welcomes all attendees to the 2024 ASPHO Conference.
Plenary Papers
The Plenary Papers were selected by the 2024 Conference Planning Committee as the most highly rated abstract submissions.
Moderators: Karen E. Effinger, MD MS; Jessica Heath, MD
Zaina Inam
IMPACT OF PRE-HSCT RBC ALLOIMMUNIZATION ON POST-HSCT OUTCOMES IN PEDIATRIC PATIENTS WITH SCD (Plenary Paper)
C. Michel Zwaan
PIVOTAL PHASE 2 RESULTS OF AUGMENT-101 FOR REVUMENIB IN KMT2Ar ACUTE LEUKEMIA: PEDIATRIC EXPERIENCE (Plenary Paper)
Young Investigator Award Presentations
The ASPHO Young Investigator Award was established to formally recognize excellence in research in pediatric hematology/oncology and to promote basic and clinical investigation into the fields of hematology/oncology by fellows and faculty members who are less than 4 years post-fellowship.
Moderators: Karen E. Effinger, MD MS; Jessica Heath, MD
Lindsay Schwartz, MD MS - University of Chicago
LONGITUDINAL OVARIAN RESERVE IN ADOLESCENTS WITH LYMPHOMA: CHILDREN’S ONCOLOGY GROUP STUDY ALTE 11C1
Rachel Offenbacher, MD
TARGETING THE UREA CYCLE TO PREVENT AND TREAT METASTATIC OSTEOSARCOMA
Early Career Travel Stipend Award Announcements
- Franklin Chien, MD MS - Emory University School of Medicine/Children's Healthcare of Atlanta
CIRCULATING TUMOR CELLS IN MEDULLOBLASTOMA: A POTENTIAL NOVEL PERIPHERAL BIOMARKERS FOR CNS DISEASE
Presenting an oral abstract on Friday, April 5 at 8 am during the "Oncology Paper Session with Luminary Investigator". - Dipti Kamath, MD - UCSF Benioff Children's Hospital
MICROFLUIDIC INSIGHT INTO GENE-EDITED RBCs FOR SICKLE CELL DISEASE
Presenting a poster on Thursday evening in the Exhibit Hall - poster #103 - Julie Ma, MD - Children's Hospital Los Angeles
TARGETING MICROENVIRONMENT PATHWAYS TO IMPROVE CAR-T CELL EFFICACY IN B-ALL
Presenting an oral abstract on Friday at 8 am during the "Oncology Paper Session with Luminary Investigator". - Jaime Josue Baldeon Mendoza, MD FAAP - Cleveland Clinic Children's
THE UNCONVENTIONAL MYOSIN FAMILY PROTEIN MYO18A IS A NOVEL REGULATOR OF B CELL DEVELOPMENT
Presenting a poster on Friday in the Exhibit Hall - poster #702 - Katelyn Purvis, MD - St. Jude Children's Research Hospital
OUTCOMES IN PATIENTS WITH ETV6: RUNX1 OR HYPERDIPLOID B-ALL IN THE ST. JUDE TOTAL 15 AND 16 STUDIES
Presenting a poster on Friday in the Exhibit Hall - poster #710
Advanced Practice Provider Travel Stipend Award Annoucement
- Bonnie Yates, MS CPNP-AC - National Institutes of Health (NIH)
CD22 CART FOR CHILDREN AND YOUNG ADULTS WITH R/R B-CELL ALL: RESULTS FROM A 10-YEAR-EXPERIENCE
Presenting an oral abstract on Friday at 8 am during the "Oncology Paper Session with Luminary Investigator".
2:15 pm – 6:15 pm
NEW! Division Directors Academy
Sponsored by Pfizer, Inc.
Join fellow Division Directors for a deep dive into relevant and timely topics, gain new insights to address challenges in your role, and enjoy engaging with colleagues during this newly designed event. A social hour will follow the event and is included in the fee.
Advanced registration and fee are required.
Moderators:
- Rishi Lulla, MD MS
- Linda McAllister-Lucas, MD PhD
Speakers:
- Recruitment and Retention
- Valerie Opipari, MD (University of Michigan Medical School; Mott Children’s Hospital)
- Mentorship Workshop
- Giana H Davidson, MD, MPH, FACS (University of Washington School of Medicine)
2:15–3:15 pm
Concurrent Sessions
(A1) Mixed Phenotype Acute Leukemia (MPAL): An Update on Classification, Biology and Treatment Strategies
Mixed phenotype acute leukemia (MPAL) is a rare subtype of acute leukemia, accounting for 2-5% of all cases, with blasts having both lymphoid and myeloid features. Advancements in understanding MPAL biology and tailoring specific therapy for this rare leukemia have remained challenging due to: (a) the changing and relatively subjective diagnostic criteria for MPAL, (b) the wide phenotypic and genomic diversity among MPAL cases and (c) the historical exclusion of MPAL cases from clinical trials. Here, we will learn about the updated diagnostic criteria, review the latest biological findings, and discuss the current treatment strategies for this rare disease.
Learning Objectives:
- Review the changes in diagnostic criteria for MPAL over the past several decades and learn about the most updated classification system described by the World Health Organization (WHO) and International Consensus Classification (ICC)
- Describe our current understanding of the biology of MPAL by reviewing the most updated MPAL literature in terms of genetics, transcriptomics, and single cell analysis
- Discuss the current treatment strategies used to treat pediatric MPAL and examine potential new strategies to treat this rare malignancy
Speakers:
- Defining the genomic and molecular landscape of pediatric MPAL
- Sunil S. Raikar, MD
- Sunil S. Raikar, MD
- Flow cytometric analysis of MPAL: Update of the WHO and ICC diagnostic criteria
- Alexandra Kovach, MD
- Alexandra Kovach, MD
- Current updates in treatment strategies for pediatric MPAL
- Thomas Alexander, MD MPH
(A2) Psychology in Pediatric Hematology and Oncology
Experts will discuss the psychosocial challenges faced by pediatric patients and their families, highlighting evidence-based interventions and support programs aimed at promoting emotional well-being, coping strategies, and resilience for pediatric patients and their families during treatment and survivorship. They will explore the neurocognitive impact of CNS impacting disease and treatments and describe emerging empirical support for interventions designed to mitigate (or even prevent) neurocognitive late effects. They will focus on the unique psychosocial needs of AYA patients and survivors and discuss tailored support programs to improve functioning and quality of life during and after treatment.
Learning Objectives:
- Understand the psychosocial challenges faced by pediatric patients and their families, and explore evidence-based interventions to enhance emotional well-being and resilience during treatment and survivorship
- Gain insights into the neurocognitive impact of CNS impacting disease and treatments and evidence-based interventions that support cognitive functioning in this patient population
- Recognize and address unique psychosocial needs of Adolescent and Young Adult (AYA) cancer patients and explore tailored interventions to enhance their overall quality of life during and after cancer treatment
Moderator:
- Adam Esbenshade, MD MSCI
Speakers:
- Dina Hankin, PhD
- Leanne Embry, PhD
- Heather Conklin, PhD
(A3) Hematology Consults in Transgender Medicine
About 10% of high school students identify as gender non-congruent, and many of those are transgender leading to an increased use of medications with potentially serious hematologic adverse effects (thrombosis, erythrocytosis, etc.) Hematologists are being asked to provide consultation to the transgender community regarding critical management questions. In addition, new nomenclature regarding the diagnosis of hemophilia carriers which is gender neutral has been published. Moreover, there are clinics for adolescents which often use gender-specific terms (e.g., Young Women’s Clinic) whose names should be reconsidered. It is imperative that using appropriate terms/names for diagnoses/clinics be appreciated by all pediatric hematologists.
Learning Objectives:
- Gain knowledge on the basics of transgender medicine including appropriate terminology and the medical aspects of therapy
- Describe the hematologic manifestations of medications utilized in transgender medicine and how to mitigate the risks of such treatments
- Learn the new gender-neutral hemophilia nomenclature and to discuss the proper terminology for specialty clinics caring for adolescents
Moderator:
- Guy Young, MD
Speakers:
- Ximena Lopez, MD
- Angela C. Weyand, MD
- Robert Sidonio, Jr., MD MSc
3:15– 3:45 pm
Networking Break
Resident/Medical Student Meetup
3:45–5:15 pm
Concurrent Sessions
(A4) ASPHO/PTCTC Joint Session - The EBV Puzzle: Dissecting PTLD, HLH, and IEI Connections
Epstein-Barr virus (EBV) associated lymphoproliferation and hyperinflammation occur in various contexts, often presenting unique challenges to Hematology oncology and BMT physicians. This joint session aims to delve into the complexities of EBV-driven lymphoma, post-transplant lymphoproliferative disorder (PTLD), and hemophagocytic lymphohistiocytosis (HLH) in relation to inborn errors of immuinty (IEI) allogeneic bone marrow transplantation (BMT), solid organ transplants, and systemic EBV-positive T-cell lymphomas of childhood. By dissecting the connections between EBV, PTLD, HLH, and IEI, this session seeks to unmask the spectrum of EBV-associated lymphoproliferation and hyperinflammation complications. Many of these complications need active collaboration between Hematology, oncology, BMT, and immunology. We aim to stimulate discussion and collaboration among clinicians, researchers, and other stakeholders through a multidisciplinary approach to address the challenges and opportunities in managing these complex and often life-threatening conditions.
Learning Objectives:
- Discuss the challenges in identifying these rare IEI presenting with EBV, lymphoma and the implications for treatment and management
- Examine the prevalence and risk factors and treatment of PTLD following both stem cell and solid transplantation
- Investigate the complex manifestations of EBV-driven hyperinflammation and lymphoproliferation, particularly when EBV infects T and NK cells
Moderator:
- Hemalatha G. Rangarajan, MD
Speakers:
- EBV-driven lymphoma as a presenting manifestation of underlying primary immune deficiency
- Shanmuganathan Chandrakasan, MD
- EBV-associated HLH, chronic active EBV infection (CAEBV), and systemic EBV-positive T-cell lymphoma
- Nader El-Mallawany, MD
- EBV Lymphoproliferative Disease in Allogeneic Hematopoietic Stem Cell and Solid Organ Transplant
- Susan Prockop, MD
(A5) LCH 2024: Enlightenment, Chaos and Opportunities
This session will review the paradigm-changing advances in mechanistic insights of LCH over the past decade. We will discuss the current front-line standard of care chemotherapy, rooted in the pre-molecular era, with reliably suboptimal outcomes. With updated conceptual framework of LCH being caused by activating MAPK pathway somatic mutations in myeloid precursor cells, we will explore opportunities to improve outcomes for patients with refined approaches to diagnosis, front-line therapy, salvage therapy and treatment for LCH-associated neurodegeneration. We will review clinical trials underway, and finally, will discuss management strategies while we await results from clinical trials testing transformational therapeutic strategies.
Learning Objectives:
- Understand mechanistic frameworks for LCH pathogenesis
- Review rationale for current diagnostic and therapeutic approaches
- Discuss opportunities to optimize improvement in outcomes through clinical trials
Moderator:
- Nitya Gulati
Speakers:
- LCH: History, modern mechanistic insights and therapeutic opportunities
- Carl Allen, MD PhD
- Is this LCH?
- Jennifer Picarsic, MD
- Stun or Kill? Clinical challenges and opportunities to improve lives of patients with LCH
- Michelle Hermiston, MD PhD
(A6) Thinking Clearly about Cognition in Sickle Cell Disease
Sickle Cell disease was once considered a disease of childhood. Through advances in clinical care pediatric hematology practitioners can expect their patients to live well into adulthood. It has been described that academic achievement of children with sickle cell disease is below that of their peers. The focus for practitioners must now shift from mere survival and prevention of serious physical complications to optimization of educational and occupational opportunities in childhood and adulthood. In order to achieve these goals, patients must have access to neurocognitive assessment and neuropsychiatric services as appropriate. Published guidelines recommend neurocognitive screening for patients with sickle cell disease however this practice is not universal. Beyond that, practitioners must know when to seek out neuropsychiatric services for their patients as access to such services can be a challenge. Pediatric hematology providers need to learn the most recent evidence, available tools, and best practices to optimize neurocognitive outcomes for their patients.
Learning Objectives:
- Describe the role of cognitive assessment in sickle cell disease and list barriers and facilitators to implementation of cognitive assessment
- Discuss what is known and unknown about the role hydroxyurea may play in improving cognition in patients with sickle cell disease
- Describe how neurocognitive testing and interventions can be beneficial to adolescents with sickle cell disease as they transition to adult care.
Moderator:
- Alex George, MD PhD
- Patrick McGann, MD MS
Speakers:
- Neurocognitive Assessment in Sickle Cell Disease
- Andrew Heitzer, PhD ABPP-CN
- Andrew Heitzer, PhD ABPP-CN
- Optimizing Neurocognition Through Medical Therapy
- Kristine Karkoska, MD
- Neurocognition and Transition
- Allison King, MD MPH PhD
5:15–6:00 pm
Welcome Reception for New Members, First-Time Attendees, and International Attendees
Join fellow new members, first-time attendees, and international attendees during this opportunity to connect and network. Learn about the Society’s special interest groups at the SIG Meet & Greet which will take place at the reception.
Thursday, April 4
7-8am
SIG Meetings
- Advanced Practice Provider
- Hospitalist Medicine
- Well-Being
(CC1) Clinical Conundrum – Treatment Considerations in Cancer Predisposition Syndromes
Join this session and interact with a recognized expert on a challenging diagnostic and management issue. The session is scheduled for 1 hour and will include a 15-20 minute presentation by Dr. Joanna Weinstein on the general approach to treatment of cancer in patients with a cancer predisposition syndrome followed by small group discussion of additional cases shared by the attendees.
Come prepared to ask questions and share your cases.
Advanced registration and fee are required. Seating is limited to 30. The registration fee is $25.
(CC2) Clinical Conundrum – Severe Aplastic Anemia
Join this session and interact with a recognized expert on a challenging diagnostic and management issue. The session is scheduled for 1 hour and will include a 15-20 minute presentation by Dr. Melissa Rose on the general approach to severe aplastic anemia followed by small group discussion of additional cases shared by the attendees.
Come prepared to ask questions and share your cases.
Advanced registration is required. Seating is limited to 30. The registration fee is $25.
7:30-8:15 am
Networking with Continental Breakfast
Sponsored by Nemours Children's Health
8:15–9:15 am
Keynote Presentation by Dr. Glaucomflecken
Sponsored by Seattle Children's Hospital
Dr. Will Flanary is an ophthalmologist and comedian who moonlights in his free time as “Dr. Glaucomflecken,” a social media personality who creates medical-themed comedy shorts to an audience of over 4 million across social media. His humor has been shaped by the challenges of medical training and by his experiences as a patient.
Dr. Flanary is a 2-time testicular cancer survivor as well as a survivor of cardiac arrest, saved by his intrepid wife and her timely CPR. Initially used as a creative outlet to cope with these health challenges, Dr. Flanary’s comedy has evolved over time to incorporate biting satire of the US health care system, academic publishing, and interpersonal conflicts pervasive in the medical system. He is a frequent keynote speaker who encourages medical audiences to embrace humor in everyday life.
9:30–11 am
Concurrent Sessions
(B7) Must Act Fast! Clinical Emergencies in Sickle Cell Disease
There are a number of true emergencies in sickle cell disease which require the on-call hematologist to quickly recognize that a situation has the potential to become rapidly fatal. With expedited management and mobilization of additional resources, patients can be appropriately treated. It has been well described that acute fat embolism syndrome, delayed hemolytic transfusion reaction, and acute pulmonary embolism are life threating complications for patients with sickle cell disease. These conditions can be mistaken for the more common an acute painful crisis and acute chest syndrome. Thus, these life-threatening complications are under-recognized and under-reported putting patients at risk for increased morbidity and mortality. It is critical that pediatric hematology providers are aware of these conditions in pediatric patients and are ready to act fast.
Learning Objectives:
- Discuss three emergencies in sickle cell disease in which providers must act intervene quickly to prevent morbidity and mortality
- List clinical features, diagnostic tools, and management strategies for the 3 emergencies in sickle cell disease covered in this session
Moderator:
- Caitlin Neri, MD
Speakers:
- Acute Fat embolism Syndrome
- Radhika Gangaraju, MD MSPH
- Delayed Hemolytic Transfusion Reaction
- Satheesh Chonat, MD
- Acute Pulmonary Embolism in Sickle Cell Disease
- Clay Cohen, MD
(B8) Cellular Immunotherapy for Pediatric Brain Tumors
Brain tumors are the leading cause of cancer deaths in children, and relapsed patients are rarely able to achieve a cure. Cellular immunotherapy regimens, including CAR T cell therapies, have emerged as promising new treatment options and have begun early-phase clinical trials in children. These novel treatments vary in their molecular targets, design of gene therapy constructs or manner of ex vivo leukocytes stimulation, lympholytic preparative regimens, timing of interventions, etc. This session will provide the audience with updates and interim results for active clinical studies, followed by an interactive panel discussion.
Learning Objectives:
- Update knowledge regarding availability, safety, and potential benefit of cellular immunotherapy clinical trials for patients with childhood CNS tumors
- Engage experts in the cellular immunotherapy field in a discussion of the finer points of designing such therapies and the clinical trials that provide access to them
Moderator:
- Theodore S. Johnson, MD PhD
Speakers:
- HER2 CAR T cells (NCT02442297, NCT04903080)
- Shoba A. Navai, MD
- IL13Rα2 CAR T cells (NCT04510051)
- Leo Wang, MD PhD
- From single to multi-antigen targeting CAR T cell trials (NCT04185038, NCT03500991, NCT05768880)
- Nicholas A. Vitanza, MD
- Autologous tumor antigen associated T cells (NCT03652545)
- Eugene Hwang, MD
(B9) Language Justice in Pediatric Hematology/Oncology: Challenges and Practical Tips in Providing High Quality, Equitable Clinical Care, and Increasing Research Participation
Over 25 million individuals in the US speak languages other than English (LOE). Despite federal regulations mandating meaningful access for healthcare, patients who speak LOE receive unequal access to clinical care and research, consequently experiencing lower patient satisfaction, lower medication adherence, lower research participation, and greater preventable morbidity and mortality. These disparities are particularly impactful in pediatric hematology/oncology, where complex diagnoses and treatment regimens, and lengthy clinical trial consents are commonplace. This session will provide background about language disparities in research and clinical practice and provide practical strategies for improving language equity in pediatric hematology/oncology.
Learning Objectives:
- Describe how language access impacts patient safety, quality, and equity
- Identify strategies to promote language equity during informed consent for pediatric hematology/oncology research
- Discuss practical strategies to improve clinical care and increase research participation for families who speak languages other than English in pediatric hematology/oncology and stem cell transplantation
Moderator:
- Jenny Ruiz, MD MSCE
Speakers:
- Language Equity and Patient Safety in Pediatrics
- Alisa Khan, MD
- “COMPRENDO”: A culturally and linguistically- appropriate intervention to improve informed consent
- Paula Aristizabal, MD MS
- Practical Strategies to Improve Clinical Care
- Joanna Robles, MD
- Practical Strategies to Increase Research Participation
- Luisanna M. Sanchez, MD
11:10–12 pm
ASPHO 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, efforts in advocating for its members and our patients, and the Society’s financial health. Additionally, outgoing Board members and committee chairs are recognized and a report is presented from the Pediatric Blood & Cancer Journal editor.
12:15–1:30 pm
Satellite Symposium: ALL IN: Staying the Course with ALL Using Therapeutic Drug Monitoring
12:15–1:15 pm
SIG Meetings
- Hemoglobinopathies
- Physician Scientist
- Quality Improvement and Patient Safety
- Small Program
12:15–1:30 pm
Early Career Roundtable Luncheon
Sponsored by St. Jude Children's Research Hospital
This session provides early-career professionals (new attendings, trainees and medical students/residents) a forum to discuss specific issues with leaders in the field, ask questions related to career development, and receive positive reinforcement regarding career goals specific to their interests, all in a small group setting.
Advanced registration is required.
1:45–2:45 pm
Paper Sessions
Leukemia/Lymphoma
- Shaikha Alqahtani
UNDERSTANDING TRADITIONAL AND E-CIGARETTE SMOKING METABOLITES EFFECT ON ACUTE MYELOID LEUKEMIA - Waitman Aumann
SIX1 KNOCKDOWN AND SMALL MOLECULE INHIBITION IMPAIR PROLIFERATION OF T-ALL AND AML - Joseph Schramm
DETERMINING BIOLOGIC RISKS THAT DRIVE HIGH-RISK OUTCOMES IN B-ALL OF HISPANIC/LATINO CHILDREN - Alix Seif
AML CARE AT HOME: EVIDENCE-BASED GUIDELINES FOR OUTPATIENT ACUTE MYELOID LEUKEMIA (AML) MANAGEMENT
Solid Tumor
- Nawal Merjaneh
RCM1 DOWNREGULATES ATP2B4 EXPRESSION AND ENHANCES VENETOCLAX-MEDIATED APOPTOSIS IN RHABDOMYOSARCOMA - Mitchell Cairo
COMBINATORIAL MACROPHAGE INDUCED INNATE IMMUNOTHERAPY AGAINST EWING SARCOMA - Hizuru Amano
URINARY CYSTATHIONINE IS A BIOMARKER FOR NEUROBLASTOMA - Emily Larimer
FEASIBILITY AND USABILITY OF ELECTRONIC ROADMAP APPLICATION
Sickle Cell Disease
- Ofelia Alvarez
SUCCESSFUL LABORATORY-BASED AND POINT-OF-CARE NEWBORN SCREENING FOR HEMOGLOBINOPATHIES IN HAITI - Akhila Vadivelan
PRELVALANCE AND SIGNIFICANCE OF PITUITARY SIDEROSIS IN TRASNFUSION DEPENDENT SICKLE CELL DISEASE - Erica Mamauag
PAIN COMMUNICATION IN PEDIATRIC SCD: DATA OF NOVEL PAIN ASSESSMENT TOOL AND PARENT/PROVIDER REPORTS - Yaw Ansong-Ansongton
MITOCHONDRIA DECREASES LIFE SPAN OF RBC AND CONTRIBUTES TO COLD INDUCED PAIN IN MOUSE SICKLE MODEL
3–4 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 Prakash Satwani, MBBS MD from Columbia University Medical Center and NewYork-Presbyterian Hospital.
4–4:30 pm
Networking Break
4:30–6 pm
Concurrent Sessions
(B10) By the Pricking of My Thumbs: New Insights and Challenges in Neoplastic Complications of Inherited Bone Marrow Failure Syndromes
Although an association between inherited bone marrow failure syndromes (IBMFS) and cancer predisposition has been well established, emerging data from registry and collaborative research studies have shown an unexpectedly high incidence of previously unrecognized cancer types along with unique and disease-specific mechanisms of transformation in commonly recognized malignancies. These findings have significant implications on surveillance methods and schedules, while also providing potential strategies for therapeutic intervention prior to transformation or targeted treatment of established cancers. In this session, we will review new findings regarding the incidence and distinctive mechanisms of myeloid malignancies, solid tumors, and myelodysplastic syndromes in pediatric IBMFS.
Learning Objectives:
- Describe the risk, spectrum, and biology of hematologic and solid tumor neoplasms in IBMFS
- Identify the rationale and modalities for cancer screening in IBMFS populations including both traditional and molecularly based methods
- Recognize that pediatric-onset myelodysplastic syndrome may be the presenting feature of an underlying BMF disorder
- Understand the role for registry-based and collaborative research efforts to develop evidence-based guidelines for cancer screening in IBMFS
Speakers:
- Moderator and Introduction to Pediatric MDS and Novel IBMFS
- Jason Farrar, MD
- Leukemia Surveillance in Shwachman Diamond Syndrome
- Akiko Shimamura, MD PhD
- Non-Hematologic Cancers in Diamond Blackfan Anemia and Fanconi Anemia
- Adrianna Vlachos, MD
- Pediatric MDS: SAMD9/9L syndromes and Beyond
- Marcin Wlodarski, MD PhD
- Transplant for hematologic malignancy in IBMFs
- Jane Koo, MD
(B11) Demystifying Artificial Intelligence: What Does It Mean?
Emerging artificial intelligence (AI) technologies, including machine learning and large language models (e.g., ChatGPT) are widely applied outside of healthcare. Applications in medicine–including pediatric hematology/oncology–have lagged, in part due to reliance on unstructured data, limited interoperability of technology systems and privacy regulations. While AI has potential benefits for pediatric hematology/oncology, it also poses potential pitfalls. In this interactive workshop, AI technologies will be demystified and explained at a conceptual level. Attendees will understand strengths and limitations of various AI technologies and explore how they can be applied to problems in pediatric hematology/oncology.
Learning Objectives:
- Understand basics of artificial intelligence, machine learning and large language models, such as ChatGPT
- Identify strengths and limitations of various artificial intelligence technologies
- Explore use of artificial intelligence to solve contemporary problems in pediatric hematology/oncology and in medicine
Moderator:
- Kirk Wyatt, MD MAS
Speakers:
- Wayne H. Liang, MD MS
- Gerard Hills, MD
(B12) Current State of High-Risk Neuroblastoma: Research, Clinical Care, and Survivorship
Treatment for high-risk (HR) neuroblastoma has changed throughout the years with huge strides made in improving survival through the last several decades. The FDA approval of GD2-targeted antibodies, dinutuximab and naxitamab, has paved the way for more precise treatments, but we continue to work to understand their full value. In a subset of HR neuroblastoma patients with ALK alterations, novel inhibitors such lorlatinib are changing treatment as we still work to understand their biology and the potential to develop resistance. With all these additional therapies, it has become exceedingly important to also study the long-term health impact on survivors.
Learning Objectives:
- Review GD2-related antibody therapies and use of these antibodies in upfront therapy for patients with high-risk neuroblastoma
- Review the role of ALK inhibitors in neuroblastoma and the utility of liquid biopsy for monitoring of resistance
- Discuss the late effects encountered in survivors of HR neuroblastoma therapy
Moderators:
- Michael Ferguson, MD MS
- Colin Moore, MD
Speakers:
- Sara Federico, MD
- Kelly Goldsmith, MD
- Eric J. Chow, MD MPH
6–7:30 pm
Grand Reception with Exhibits and Odd-Numbered Posters
Sponsored by UCSF Benioff Children's Hospital
Education Theater: Advancing the Treatment of PIK3CA-Related Overgrowth Spectrum Disorders with VIJOICE
7–8:30 pm
Fellowship Program Directors Dinner
Supported by Pfizer, Inc.
Advanced registration is required.
Satellite Symposium: Pediatric Low Grade Glioma (pLGG): Disease Overview & Evolving Paradigm
Friday, April 5
6:30 – 7:45 am
Satellite Symposium: Plasminogen Deficiency Type 1 (PLGD-1): A Pediatric Rare Disease
7 – 8 am
Continental Breakfast with Exhibits and Posters
8–9:15 am
Oncology Paper Session with Luminary Investigator
Luminary Speaker: Maryam Fouladi, MD
Paper Presenters:
- Frank Chien, MD
CIRCULATING TUMOR CELLS IN MEDULLOBLASTOMA: A POTENTIAL NOVEL PERIPHERAL BIOMARKERS FOR CNS DISEASE (Early Career Travel Stipend Award Recipient) - Jay Shah, MBBS MD
TARGETING C-MET: IMPROVING EFFICACY OF CAR T-CELLS FOR TREATMENT OF AGGRESSIVE SOLID & BRAIN TUMORS - Julie Ma, MD
TARGETING MICROENVIRONMENT PATHWAYS TO IMPROVE CAR-T CELL EFFICACY IN B-ALL (Early Career Travel Stipend Award Recipient) - Bonnie Yates, MS CPNP-AC
CD22 CART FOR CHILDREN AND YOUNG ADULTS WITH R/R B-CELL ALL: RESULTS FROM A 10-YEAR-EXPERIENCE
Hematology Paper Session with Luminary Investigator
Luminary Speaker: Jorge A. Di Paola, MD
Paper Presenters:
- Sally Azer, MD
EVALUATING THE BIOPHYSICS OF PLATELET ADHESION AND GEOMETRY-SENSING IN MILD BLEEDING DISORDERS - Annegret Holm, MD
TARGETING A NOVEL SOX18-MEVALONATE PATHWAY AXIS TO TREAT INFANTILE HEMANGIOMA - Mithra Lakshmi Narasimhan, MD
OUTCOMES OF HSCT USING REDUCED INTENSITY CONDITIONING IN PEDIATRIC SEVERE APLASTIC ANEMIA - Rachael Grace, MD MMSc
THE CHARACTERISTICS OF PEDIATRIC PATIENTS WITH PYRUVATE KINASE DEFICIENCY AND IRON OVERLOAD
9:15–10:15 am
Networking Break with Exhibits and Posters
9:15–10:15 am
Headshot Station
9:30–10 am
Education Theater: Cellular and genetic therapies for hemoglobinopathies- a transplanter’s perspective
10:15–11:15 am
Concurrent Sessions
(C13) Improving the Health of Survivors of Childhood Cancer
Supported by Northwestern Mutual
With contemporary therapy, survival rates for childhood cancer have improved to >85%. However, these survivors face lifelong risk for chronic health conditions. Researchers are exploring ways to improve the health of these survivors to ensure they are not only surviving but also thriving after completion of therapy. Recipients of ASPHO's Northwestern Mutual Award for Excellence in Childhood Cancer Survivorship will present their groundbreaking research to address the risks of chronic conditions faced by survivors and ways to address barriers faced by survivors in order to improve health and quality of life.
Learning Objectives:
- Characterize factors associated with endocrine late effects in survivors of childhood cancer and strategies to improve outcomes
- Identify barriers to survivor care and approaches to improve access and promote equity in care delivery
- Describe late effects risk prediction models for survivors of childhood cancer and methods to ameliorate risk
Moderator
- Karen Effinger, MD MS
Speakers:
- Endocrine Late Effects in Survivors of Childhood Cancer
- Goli Mostoufi-Moab, MD MSCE
- Addressing Access to Care and Disparities in Survivors of Childhood Cancer
- M. Monica Gramatges, MD PhD
- Prediction and Prevention of Late Effects in Survivors of Childhood Cancer
- Smita Bhatia, MD MPH
(C14) Novel Advanced Molecular Infectious Diseases Diagnostics
Infectious diseases remain the most important non-cancer cause of death and disability in children undergoing therapy for malignancy or receiving hematopoietic stem-cell transplantation. Identifying the causative pathogen to target treatment remains elusive because of low sensitivity of easily obtainable cultures and potential harms of invasive procedures. Novel metagenomic sequencing and other molecular tests from plasma, respiratory specimens, or cerebrospinal fluid are revolutionizing management of infectious diseases; clinicians need to use them optimally. In this workshop, we will discuss opportunities, costs and pitfalls of these new techniques, and new research directions that will answer some of the persistent burning questions.
Learning Objectives:
- Discover newly available molecular techniques for diagnosis of life-threatening infections in blood, respiratory tract and central nervous system in children with cancer or receiving hematopoietic stem cell therapies
- Evaluate and compare the sensitivity and specificity of available molecular techniques for diagnosis and prediction of life-threatening infections in children with cancer or receiving hematopoietic stem cell therapies
- Weigh benefits and costs of newly available molecular diagnostic techniques for diagnosis of life-threatening infections in children with cancer or receiving hematopoietic stem cell therapies
Moderator:
- Joshua Wolf, FRACP MBBS PhD
Speakers:
- Use of novel molecular techniques for diagnosis of respiratory infection in children with cancer or receiving hematopoietic stem cell therapies
- Pratik Patel, MD
- Use of novel molecular techniques for diagnosis of bloodstream infection in children with cancer or receiving hematopoietic stem cell therapies
- Catherine Aftandilian, MD
- Use of novel molecular techniques for diagnosis of central nervous system infection in children with cancer or receiving hematopoietic stem cell therapies
- Christopher E. Dandoy, MD MS
(C15) Advances in the Diagnosis and Management of Thrombotic Microangiopathy in Children and Adolescents
This session will focus on the latest developments in the diagnosis and management of pediatric thrombotic microangiopathy (TMA), which is characterized by microangiopathic hemolytic anemia, thrombocytopenia, and end organ damage. The syndromes under the umbrella of TMA are diverse and marked by severe endothelial damage. TMAs require rapid assessment, diagnosis, and initiation of appropriate treatment to avoid permanent organ damage, high morbidity and mortality. The complement system plays an integral role in maintaining homeostasis between the endothelium, coagulation, and immune system and loss of this homeostasis can lead to complement mediated-TMA (CM-TMA). While atypical hemolytic uremic syndrome (aHUS) is the archetype of complement dysregulation, CM-TMA can also occur secondary to complement amplifying disorders, such as infection, drugs, cancer, sickle cell disease, and transplantation. The diagnosis of TMA can be challenging, and delayed diagnosis can result in significant morbidity and mortality. Experts in the field will present current research and evidence-based strategies to address this challenging group of disorders. Additionally, this workshop will discuss creation of registries for Pediatric TTP and CM-TMA.
Learning Objectives:
- Understand the pathophysiology and risk factors associated with CM-TMA and TTP
- Provide valuable insights into recently published updates on diagnostic approaches to CM-TMA and TTP
- Evaluate the efficacy and safety of emerging treatment modalities in children and adolescents with CM-TMA and TTP
Moderator:
- Satheesh Chonat, MD
Speakers:
- Advances in the diagnosis and management of CM-TMA
- Shruti Chaturvedi, MD
- TTP in children: consideration in the management and long-term outcomes
- Sara Graciaa, CPNP
11:30 am–12:30 pm
Frank A. Oski Memorial Lectureship Award Presentation and Northwestern Mutual Award for Excellence in Childhood Cancer Survivorship Announcement
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 Nirali N. Shah, MD MHSc from the National Institutes of Health (NIH).
Northwestern Mutual Award for Excellence in Childhood Cancer Survivorship Announcement
ASPHO and Northwestern Mutual will present the Award for Excellence in Childhood Cancer Survivorship. The award honors a mid-career (8-15 years post-fellowship) childhood cancer survivorship investigator who has demonstrated his or her commitment to this segment of the field through a growing portfolio of research and project funding, while accelerating in his or her output and gaining prominence.
This year's award will be presented to Katie A. Greenzang, MD EdM from Boston Children's Hospital.
Funded by: Northwestern Mutual Foundation
12:30–2:30 pm
Lunch with Exhibits and Even-Numbered Posters
12:45–2:15 pm
Headshot Station
12:45–2 pm
Satellite Symposium
12:45–2:05 pm
Speed Mentoring
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 be matched with three separate mentors for the session and have the opportunity to meet one-on-one with them for 10 minutes each. Mentees should come prepared with focused questions to discuss with their mentors. Each person will be assigned to one of two sessions (either 12:45-1:20 or 1:30-2:05pm).
Advanced registration is required.
12:45–1:15 pm
Education Theater: A look beyond chronic neutropenia- diagnosis of WHIM syndrome
1:30–2:05 pm
Education Theater: Treating NF1 Plexiform Neurofibromas: Case Studies
2:05–2:15 pm
Passport Prize Drawings at ASPHO Booth
2:30–3:45 pm
Presidential Symposium: Trust, Communication, Advocacy, and Barriers to Ethnical Care of Patients with Ethnically Based Diseases – What We Learn from Caring for Patients with Sickle Cell Disease
Using sickle cell disease (SCD) as a prototype for ethnically based disease, the interdisciplinary panel will present a historical perspective on the interface between medical science with a focus on clinician communication and patient/family understanding. Recent innovations and perceptions of this patient community will be presented. A family representative will offer a unique perspective of experiencing the disease and its influence in addressing global health disparities. A physician-bioethicist will discuss how the concepts of hope and cure influence families’ interpretation of scientific advances and physician communication. We will hear a perspective on development and globalization of curative therapies in this disease community and understand the barriers and challenges to access these therapies.
Learning Objectives:
- Identify barriers and perceptions patients with sickle cell disease and their families experience in understanding novel and curative therapies
- Understand the challenge of living with and caring for those with sickle cell disease, as well as the need for specialists and advocates to ensure expert compassionate care
- Recognize how patient-physician communication influence the concepts of hope and cure on medical-decision making in sickle cell disease
- Examine the development and globalization of curative therapies in Sickle Cell disease and the impact on access to care and health equity
Moderator:
- Caroline A. Hastings, MD
Speakers:
- Historical Overview of the Development of Diagnostic and Novel Therapeutics in Sickle Cell Disease
- Elliott Vichinsky, MD
- Welcoming, Raising, Loving, and Losing Kwame - and Why That's a Global Issue
- Janet Frempong, Family representative
- Hope, Hype, and Cure: Four-Letter Words and Innovative Therapies for Sickle Cell Anemia - Listening to and Learning from Patients
- Yoram Unguru, MD MS MA HEC-C
- Democratizing Education for Sickle Cell Disease Gene Therapy - Building a Deliberative Community Engaged Model
- Vence Bonham, Jr., JD
3:55–4:55 pm
Concurrent Sessions
(C16) Continuing to Fine Tune Our Precision: An Update on Pediatric Precision Oncology
Although we have seen improvement in the overall outcome of children and adolescents with cancer over the past few decades, challenges remain in improving the outcomes of all children with cancers particularly those with high risk and relapsed/refractory diseases. Precision medicine holds the promise to accelerate the outcomes of these populations and has quickly started to change the diagnostic and therapeutic landscape of pediatric oncology. In this session, we will review the implementation of precision medicine in pediatric oncology and the unique challenges being faced with these efforts.
Learning Objectives:
- Review the inaugural WHO classification of pediatric cancers highlighting the transition of traditional histologic based approach to more integrated approach utilizing molecular profiling
- Discuss utility of liquid biopsy for diagnosis and disease monitoring in pediatric oncology
- Describe the integration of normal/tumor match approach in tumor profiling in discovering germline predisposition and its actionability
Moderators:
- Harry A. Lesmana, MD
- Michael Ferguson, MD MS
Speakers:
- Jessica Davis, MD
- Brian D. Crompton, MD
- Sun Young Kim, MD PhD
(C17) Alleviating Anxiety and Pain in the Pediatric Oncology Population through Sedation or Alternative Interventions
Although cure rates for childhood cancer have improved over the past 5 decades, it is accomplished with often painful and anxiety producing procedures. The most common childhood cancer, ALL treatment protocols include LPs which are done using sedation at most pediatric oncology centers. In addition to in the challenges of scheduling and logistics for patients and families, it is now recognized that there may be long term consequences of these sedation methods including risk of neurocognitive impairment. This session will review the current practices in sedation at different pediatric oncology centers with attention to risks and benefits and alternative approaches. Discussion of alternative approaches will include consideration of unsedated lumbar punctures with simulation and education and incorporation of virtual reality for these children.
Learning Objectives:
- Current Sedation Practices
- Potential long-term effects of sedation especially neurocognitive impairment
- Alternative distraction approaches to relieve pain and anxiety
Moderators:
- Krystal Bottom, MD
- Katie McGuinn, MD
Speakers:
- Lisa Jacola, PhD
- David Dickens, MD
- Karen Arane, MD
- Sarah Alexander, MD
(C18) Quality Improvement (QI): Delivering High Quality Care in Pediatric Hematology/Oncology
Learn from experts who have successfully completed PHO relevant QI work such as implementing guidelines for chemotherapy induced nausea and vomiting, improving flow in a multidisciplinary hemostasis clinic, establishing a revaccination clinic post HSCT, and designing a clinical pathway to standardize approach to tumor lysis. Speakers will highlight tools used to make these efforts successful and how integrating QI into your practice can be approachable, practical, and achievable. Basic overview of QI methodology will also be highlighted to help those without a strong QI background lay groundwork for future projects.
Learning Objectives:
- Learn from successful implementation of QI in both inpatient and outpatient oncology and hematology settings to improve patient care
- Highlight the QI tools used to assess a process to improve it
- Understand a basic framework for implementing a QI project in the PHO setting
Moderator:
- Mahvish Rahim, MD
Speakers:
- Meghan Drayton Jackson, DO MBOE
- Jason Freedman, MD MSCE
- Christine M. Smith, MD
5–6 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 their career has had a major impact on the subspecialty through some combination of research, education, patient care, and advocacy. This session celebrates David A. Williams, MD, this year’s winner, and is followed by a reception to congratulate him on the award.
5:30–6:30 pm
SIG Meetings
- Clinical Immunology
- Diversity, Equity, and Inclusion
- Informatics, Innovation & Entrepreneurship (IIE)
- Palliative Care
7:00 pm
Flatstick Pub: A night of fun! (hosted by Seattle Children’s Hospital)
Special Invitation for Conference Attendees
Join Seattle Children’s Hospital for a Friday evening reception at South Lake Union Flatstick Pub at 7:00 pm. Advance ticket purchase is required; space is limited. Register here.
Saturday, April 6
7–8 am
(CC3) Clinical Conundrum - Recurrent AML
Join this session and interact with a recognized expert on a challenging diagnostic and management issue. The session is scheduled for 1 hour and will include a 15-20 minute presentation by Dr. Todd Cooper on the general approach to recurrent AML followed by small group discussion of additional cases shared by the attendees.
Come prepared to ask questions and share your cases.
Advanced registration and fee are required. Seating is limited to 30. The registration fee is $25.
(CC4) Clinical Conundrum - Novel Diagnostics for Immune Deficiencies
Join this session and interact with a recognized expert on a challenging diagnostic and management issue. The session is scheduled for 1 hour and will include a 15-20 minute presentation by Dr. Sharat Chandra on the general approach to novel diagnostics for immune deficiencies followed by small group discussion of additional cases shared by the attendees.
Come prepared to ask questions and share your cases.
Advanced registration and fee are required. Seating is limited to 30. The registration fee is $25.
7–8 am
SIG Meetings
- Adolescent & Young Adult (AYA)
- Cancer Predisposition
- Global PHO
- Integrative PHO
7:30–8:15 am
Networking with Continental Breakfast
8:15–9:45 am
Concurrent Sessions
(D19) Thalassemia in Transition
Worldwide, thalassemia is a common disorder but, in the U.S., it is relatively rare. Recently cases have been rising. Many patients are cared for by providers who have had little or no training or experience in thalassemia diagnosis and management. Additionally, we have seen a significant shift in the demographics with more patients entering care as either international adoptees or refugees from under-resourced countries. These patients have unique complications requiring evaluation and care. We will review the diagnostic work-up, the standards of care for transfusion, chelation, BMT, gene therapy and other emerging therapies and how to integrate them into practice.
Learning Objectives:
- Improve the understanding of the changing prevalence and demographics of thalassemia and increase the understanding of the diagnostic workup
- Familiarize providers with the standards of care for management
- Introduce the new available therapies and those in trials for thalassemia
Speakers:
- Changes in Prevalence and Demographics in North America
- Jeanne Boudreaux, MD
- Standards of Care
- Maa-Ohui Quarmyne, MBCHB MS
- Novel Therapies for Alpha and Beta Thalassemia
- Sujit Sheth, MD
- Newborn Screening and Laboratory Diagnosis of Thalassemia
- Ashutosh Lal, MD
(D20) Lymphoid Malignancies Associated with Inborn Errors of Immunity: Considerations for Diagnosis and Treatment
Children with inborn errors of immunity (IEI), including DNA repair deficiencies, are at an increased risk for malignancy. Lymphoid neoplasm is among the most common malignancies in these patients. Lymphoproliferation and lymphoma might even be the presenting manifestation of an underlying IEI. In many cases, distinguishing between benign lymphoproliferation and progression to malignant transformation may be challenging. Furthermore, these patients require careful consideration of treatment approaches due to the increased risk of infections and the toxicity experienced with cytotoxic chemotherapy and stem cell transplantation.
Learning Objectives:
- Understand the gamut of IEI that present with lymphoproliferation and lymphoma
- Recognize the pathological spectrum of benign lymphoproliferation and lymphoma in the setting of IEI, and the pathological challenges in differentiating benign from malignant lymphoproliferation
- Appreciate the unique challenges of managing lymphoma and lymphoproliferation in the setting of IEI. Understand the role of virus-specific T cells and checkpoint inhibitors in the management of lymphoma in the context of IEI
Moderator:
- Harry A. Lesmana, MD
Speakers:
- Inborn errors of immunity predisposing to nonneoplastic or neoplastic lymphoproliferation
- Shanmuganatha Chandrakasan, MD
- The pathological spectrum of nonneoplastic or neoplastic lymphoproliferation in patients with IEI
- Jinjun Cheng, MD
- Unique challenges in the management of lymphoma in the setting of IEI
- Jeremy Rubinstein, MD PhD
(D21) Career Development Opportunities for Young Investigators in Pediatric Hematology/Oncology: Perspectives and Opportunities from Four (Inter)National Organizations
This session supports the advancement of multi-disciplinary early and mid-career young investigators working in pediatric hematology/oncology. Learners will hear from four speakers representing national and international pediatric hematology-oncology organizations (ASPHO, ASH, COG, SIOP). Attendees will gain knowledge about the roles and scope of the respective societies as well as specific topics such as: how to engage in global research, how to seek effective mentorship, the importance of advocacy and EDI (equity, diversity, inclusion) principles, and an additional professional development topic to be confirmed.
Learning Objectives:
- Develop an understanding of the mentorship, career development, and research opportunities available through membership and participation with ASH, ASPHO, SIOP, and COG.
- Network with peers and experienced members from the highlighted organizations
Moderator:
- Louise Guolla, MD
Speakers:
- How to seek effective mentorship
- Adam Esbenshade, MD MSCI
- How young investigators can engage in global research
- Julia Challinor
- Topic TBD
- Pinki Prasad, MD MPH
- Topic TBD
- Lachelle Weeks, MD PhD
10–11 am
Concurrent Paper Sessions - COG and ASH
- Best of Children's Oncology Group (COG)
- Erin Rudzinski, MD (Molecular Characterization Initiative)
- David Teachey, MD (New Insights into the Biology and Management of T-ALL)
- M. Monica Gramatges, MD PhD (Outcomes and Survivorship)
- Best of Hematology Science as presented at the 2023 ASH Meeting
- Kristin Maher, MD PhD (Emicizumab Prophylaxis in Infants with Severe Hemophilia A without Factor VIII Inhibitors: Results from the Primary Analysis of the HAVEN7 Study)
- Masanori Yoshida, MD PhD (Branching Trajectories and Diversification of Clonal Escape in Aplastic Anemia Revealed by Single-Cell Genomics)
- Brianna Murphy, DO (A Collaborative Model for Improving Patient Engagement and Access for Hematopoietic Stem Cell Transplant and Clinical Gene Therapy Trials for Patients with Sickle Cell Disease in the Upper Midwest United States)
- Vilmarie Rodriguez, MD (Apixaban is Effective and Safe in Reducing Venous Thromboembolism (VTE) in Obese Patients with Newly Diagnosed Pediatric Acute Lymphoblastic Leukemia/Lymphoblastic Lymphoma (ALL/LL) a Sub-Study Analysis of the Prevapix-ALL/Children’s Oncology Group ACCL1333 Trial)
11:15–12:30 pm
Concurrent Sessions
(D22) Pediatric Neuro-vascular Anomalies: Advances in Diagnosis and Treatment Beyond Stroke
Neurovascular anomalies account for the vast majority of non-traumatic hemorrhagic strokes in children. These lesions can occur sporadically or may be associated with a systemic syndrome, requiring expertise from a hematologist/oncologist. This session aims to bring together experts from various fields to discuss the current state of knowledge regarding the diagnosis and treatment of these anomalies and syndromes, as well as to explore new approaches and strategies for improving patient outcomes. Topics will be case based and include presentations of unresectable AVMs, sickle cell associated moya moya, and more. Discussions will include review of genetics, radiological findings, pathogenesis, and medical management of these complex clinical situations.
Learning Objectives:
- Highlight the role of genetics in the development of neuro-vascular anomalies and implications for management
- Discuss novel treatments for neuro-vascular anomalies
- Discuss the role of the multidisciplinary team in management of neurovascular malformations and syndromes
Moderator:
- Taizo Nakano, MD
Speakers:
- Ionela Iacobas, MD
- John Taylor, MD
- Mesha Martinez, MD
(D23) Retinoblastoma: Bridging the Bench to Bedside Gap
Retinoblastoma is the most common primary intraocular malignancy of childhood and accounts for 10-15% of cancers in the first year of life. Unlike other cancers, retinoblastoma cannot be directly biopsied due to iatrogenic risk of tumor seeding. Recent scientific advancements show that blood, CSF, and aqueous humor can be used to as a surrogate for tumor biopsy to evaluate for circulating tumor DNA (ctDNA) and cell-free DNA (cfDNA). These results are proving to be important as diagnostic and prognostic biomarkers for patients with retinoblastoma. We will examine and compare these methods and their future applications using a case-based approach.
Learning Objectives:
- Identify the limitations in the traditional diagnosis and disease monitoring strategies for patients with retinoblastoma
- Examine and compare rapidly emerging technologies for liquid biopsy in patients with retinoblastoma
- Discuss future clinical applications of these liquid biopsy techniques for retinoblastoma
Moderator:
- Murali M. Chintagumpala, MD
Speakers:
- Gaps in Retinoblastoma
- Emma Cantor, MD
- Blood and CSF ctDNA Liquid Biopsy, including case-based examples
- Sameer Farouk Sait, MBBS
- Aqueous Humor and Liquid Biopsy in Retinoblastoma
- Andrew Stacey, MD
(D24) Weathering the Storm: Ethical Considerations and Practical Challenges of Critical Drug Shortages in Pediatric Hematology/Oncology
Nationally, at the time of this writing, severe shortages of drugs critical to the treatment of childhood cancers and blood disorders have reached an all-time high. Though this has been most notable of late in chemotherapy, it has been a major issue across many different critical medications. Consensus guidelines and an allocation framework provide helpful direction specific to pediatric hematology/oncology drug shortages.1,2 Yet, significant practical challenges continually confront pharmacists, pediatric hematologists/oncologists, and institutions in the wake of these shortages. In this workshop, we identify common barriers, highlight ethical challenges, and suggest practical strategies that participants may apply to approach future drug shortages. (1. Decamp M et al, Pediatrics 2014; 2. Unguru Y et al, J Natl Cancer Inst 2016)
Learning Objectives:
- Identify barriers pediatric hematology/oncology teams face in approaching critical drug shortages on a local, regional, and national level
- Consider real-world cases of critical drug shortages and how to make challenging decisions at individual pediatric centers
Speakers:
- Introduction and Case Discussion
- Jonathan M. Marron, MD MPH HEC-C
- Ethical considerations and approaches to allocation
- Yoram Unguru, MD, MS, MA, HEC-C
- The Pharmacist’s Role in Critical Drug Shortages
- Delia Carias, PHARMD
- “On The Ground” in Critical Drug Shortages
- Brittany (Lee) Greene, MD MA
12:30 pm
Conference Concludes
Visit the registration page to learn more about #ASPHO2024!