The 2020 ASPHO Conference is cancelled due to the public safety impact of the COVID-19 crisis. Learn more.
The ASPHO Conference Schedule
Our Conference Planning and Education Committees are assessing opportunities to deliver some content originally meant to be presented at the 2020 Conference in the future. In the meantime, explore the 2020 ASPHO Conference schedule to learn about the programming our Conference Planning and Education Committee worked tirelessly to build and to get a taste of the content you may see at the 2021 ASPHO Conference, April 21-24 in Portland, OR.
Schedule at a Glance
Check out an abbreviated schedule with sessions and times so you can plan your conference experience.
Wednesday, May 6
7:30–11 am
Pre-Conference Session – Vascular Anomalies: A Primer*
8 am–8 pm
Pediatric Transplantation and Cellular Therapy Consortium (PTCTC, formerly PBMTC) Meeting*
11–11:30 am
Vascular Anomalies Special Interest Group
11:15 am–12:15 pm
Non-CME Corporate Luncheon Symposium
12:30–1:50 pm
12:30–1:50 pm
Welcome, Plenary Papers, and Award Presentations
Plenary Paper and Young Investigator Award Presentations
Early Career and Advanced Practice Professional Travel Award Announcements
2–3:30 pm
Concurrent Sessions
4–5 pm
Concurrent Sessions
5:15–6:15 pm
Special Interest Group (SIG) Workshops – Integrative PHO, Palliative Care, and Physician Scientist SIGs
Genius Bar
Clinical Conundrums*
6:15–7 pm
New Member, First Time and International Attendee Reception
7–9 pm
Division Directors Dinner Meeting*
7–8:30 pm
Non-CME Corporate Dinner Symposium
Thursday, May 7
6:30–8 am
Non-CME Corporate Breakfast Symposium
6:30–7:30 am
5K Fun Run/Walk*
7–8 am
Special Interest Group (SIG) Workshops – Advanced Practice Provider, Quality, Clinical Immunology, Diversity, and Hemoglobinopathies SIGs
8:15–9:15 am
Paper Sessions
9:45–11:15 am
Concurrent Sessions
11:30 am–12:20 pm
Business Meeting
Clinician Educator Award and 5k Fun Run/Walk Winner will be announced.
12:30–1:45 pm
Early Career Roundtable Luncheon*
12:30–1:30 pm
Special Interest Group (SIG) Workshops – Adolescent and Young Adult, Global PHO, and Well Being SIGs
Clinical Conundrums*
12:30–1:45 pm
Non-CME Corporate Luncheon Symposium
2–3:30 pm
Concurrent Sessions
3:45–4:30 pm
St. Baldrick's Foundation Robert J. Arceci Innovation Award Announcement and Presentation
4:45–6 pm
Presidential Symposium
6–7:30 pm
Opening Reception with Exhibits and Posters
Sponsored in part by Cook Children’s Health Care System
6–7 pm
Odd-numbered author-attended poster session
Meet the SIGs
7–7:30 pm
Even-numbered poster tour
7:30–9 pm
Fellowship Program Directors Dinner Meeting
7:30–9 pm
Non-CME Corporate Dinner Symposium
Friday, May 8
6:30–8 am
Non-CME Corporate Breakfast Symposium
7:30–8:30 am
Continental Breakfast with Exhibits and Posters
8:30–9:30 am
Northwestern Mutual Award for Excellence in Childhood Cancer Survivorship Announcement
Frank A. Oski Memorial Lectureship Award Presentation
9:45–10:45 am
Concurrent Sessions
10:45–11:45 am
Break with Exhibits and Posters
11–11:30 am
Odd-numbered poster tour
Genius Bar
11:45 am–12:30 pm
George R. Buchanan Lectureship Award Presentation
12:30–2 pm
Lunch with Exhibits and Posters
12:30–1:30 pm
Even-numbered author-attended poster session
12:30–1:05 pm
Speed Mentoring Session I**
12:30–1:15 pm
ASH-ASPHO Choosing Wisely® List Q&A
1:10–1:45 pm
Speed Mentoring Session II**
1:20–1:50 pm
Expert Perspective: A Closer Look at a New Treatment Option for Sickle Cell Disease
12:45–1:45 pm
Non-CME Luncheon Symposium
2:15–3:45 pm
Concurrent Sessions
4–5:30 pm
Concurrent Sessions
5:45–6:45 pm
Distinguished Career Award Presentation and Reception
6:30–8 pm
Non-CME Corporate Dinner Symposium
Saturday, May 9
7–8 am
Special Interest Group (SIG) Workshops – Small Program SIG
7:30–8 am
Diversity SIG Business Meeting
Hemoglobinopathy SIG Business Meeting
8:15–9:15 am
COG and ASH Paper Sessions
9:30–11 am
Concurrent Sessions
11:15 am–12:45 pm
Luminary Presenters with Paper Sessions
12:45 pm
Conference Concludes
*Advance registration and fee required.
**Advance registration required.
Full Schedule
All conference programs are listed by day and time. Register online or contact ASPHO Member Services, 847-375-4716 or This email address is being protected from spambots. You need JavaScript enabled to view it. .
Wednesday | Thursday | Friday | Saturday
Wednesday, May 6
7:30–11 am
Pre-Conference Session—Vascular Anomalies: A Primer
Pediatric hematologists and oncologists (PHOs) are increasingly consulted on patients with vascular anomalies (VA). Yet, there is little VA training for the majority of fellows and practicing PHOs. New causative mutations have recently been identified in VAs and targeted therapies are becoming central in the care of this population. Many treatments, currently in use or under study, are immunosuppressive or oncologic agents. With more medicinal management, PHOs will have a greater role in the care patients with VA. This VA Primer will address these needs and provide an educational forum to familiarize attendees in this growing field.
Objectives:
- Identify basic and complex vascular anomalies and the appropriate methods useful in their diagnosis.
- Recognize and recommend treatments for vascular anomalies, including medications and procedural methods and choose appropriate supportive care.
- Virtually experience a Vascular Anomalist's day-to-day patient case load upon which they can build their own focused understanding of this discipline.
Moderator: Kristen M. Snyder, MD
Presenters:
- Ionela Iacobas, MD
- Denise Metry, MD
- Shelley Crary, MD, MS
- Sudhen Desai, MD
- Adrienne M. Hammill, MD PhD
No relevant financial relationships; discussion of off-label drug use: Kristen M. Snyder, Ionela Iacobas, Denise Metry, Sudhen Desai, Adrienne M. Hammill
Relevant financial relationships; no discussion of off-label drug use: Shelley Crary—Genentech: Advisory Board Member, Honoraria
Advanced registration is required. Seating is limited to 150. The registration fee is $50.
8 am–8 pm
Pediatric Transplantation and Cellular Therapy Consortium (PTCTC) (formerly PBMTC) Meeting
11–11:30 am
Vascular Anomalies Special Interest Group
Leadership: Francine Blei, MD; Ionela Iacobas, MD
Focus on multi-institutional collaboration within Vascular Anomalies
During our meeting, we will present updates from our 3 committees: Education, Practice and Research regarding multiple projects (either completed or on-going). We will discuss about new/updated educational resources, transition to adult care, multi-institutional research projects, collaboration with the patient advocacy groups, etc. Everybody is welcome!
11:15 am–12:15 pm
Non-CME Corporate Luncheon Symposium (Hematology Topic)
12:30–1:50 pm
Plenary Paper Session
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.
Moderators: Valerie I. Brown, MD PhD; Karen E. Effinger, MD
(2001) REDUCED FREQUENCY OF MAINTENANCE PULSES IN STANDARD-RISK B ACUTE LYMPHOBLASTIC LEUKEMIA AALL0932
Anne Angiolillo, MD
(2002) INCREASED EXPRESSION OF CXCR4 LEADS TO ENHANCED CELL MIGRATION IN CALM-AF10 DRIVEN LEUKEMIA
Jessica L. Heath, MD
No relevant financial relationships and no discussion of off-label drug use: Anne Angiolillo, Jessica Heath
12:30–1:50 pm
Young Investigator Award Presentations
The ASPHO Young Investigator Award recognizes excellence in pediatric hematology/oncology research and promotes basic and clinical investigation in the fields of hematology and oncology by fellows and faculty members who are less than 4 years post-fellowship. Award recipients will be announced in February.
Moderators: Valerie I. Brown, MD PhD; Karen E. Effinger, MD
(2003) LEUKEMIA PREDISPOSITION OR CLONAL HEMATOPOIESIS IN SHWACHMAN DIAMOND SYNDROME: DIFFERENT PATHWAYS
Alyssa Kennedy, MD PhD
Objectives:
- Demonstrate that clonal hematopoiesis is common in patients with SDS
- Review the two different genetic pathways in clonal hematopoiesis: leukemogenic and compensatory for the underlying ribosome defect
(2004) MECOM DYSREGULATION VIA ENHANCER HIJACKING IN PEDIATRIC THERAPY-RELATED MYELOID NEOPLASMS
Jason R. Schwartz, MD PhD
Objectives:
- Define the somatic and germline genomic profiles of pediatric tMN
- Demonstrate high MECOM expression secondary to structural variations and enhancer hijacking
No relevant relationships and no discussion of off-label drug use: Alyssa Kennedy, Jason R. Schwartz
Early Career and Advanced Practice Professional Travel Stipend Award Announcements
2020 Early Career Travel Stipend Award Recipients
Harry Lesmana, MD FAAP FACMG (Paper #2024)
Discovery of A Novel Ral Pathway Disorder (“Ralopathy”) with Early Onset Myeloproliferative Disease
Lisa Maurer, MD PhD (Poster #517)
Disruption of DNA Damage Repair Enhances Expression of Immune Checkpoint Proteins in Ewing Sarcoma
Ryan Summers, MD (Poster #710)
Next-Generation Sequencing Reveals Increased Prevalence of PICALM-MLLT10 Fusions in T-ALL and T-LLy
Mallery Olsen, MD (Poster #301)
Canine NK Cell Expansion and Novel Xenograft Model for Adoptive Immunotherapy of Osteosarcoma
Anthony Sabulski, MD (Poster #308 and #309)
Circulating Endothelial Cells Identify Vascular Injury in Pediatric Stem Cell Transplant Recipients and Bone Health Outcomes in a Diverse Cohort of Pediatric Hematopoietic Stem Cell Transplant Recipients
2020 Advanced Practice Professional Travel Stipend Award Recipient
Sara Graciaa, CPNP (Paper #2030)
Long Term Neurological Outcomes in Pediatric Thrombotic Thrombocytopenic Purpura
2–3:30 pm
Concurrent Sessions
(A1) Understanding Challenges in Pediatric Thrombosis
Quality evidence for optimal treatment of venous thromboembolism (VTE) in children is lacking. Practitioners are often faced with questions of whether it is appropriate to treat a thrombosis, such as an asymptomatic VTE, a superficial thrombophlebitis, or in the setting of bleeding risk. Another common problem is catheter associated VTE, and effective prevention strategies are needed. Recent studies have addressed these important clinical questions about VTE management in children. Lastly, DOACs are FDA approved for use in adults, but published data on safety and efficacy in pediatrics are needed to guide their use in this population.
Objectives:
- Understand the natural history of asymptomatic VTEs.
- Apply the available evidence for using DOACs to treat VTE in pediatric patients.
- Describe strategies to prevent catheter-related VTE.
Moderator: Shelley Crary, MD MS
Presentations:
- Do You Need to Treat All VTEs?
Clifford Takemoto, MD - How to Use DOACs for Children?
Jennifer Davila, MD - Can Catheter-Related Clots Be Prevented?
E. Vince Faustino, MD MHS
No relevant financial relationships; discussion of off-label drug use: E. Vince Faustino
Relevant financial relationships; no discussion of off-label drug use: Shelley Crary - Genentech: Advisory Board Member, Honoraria; Clifford Takemoto - Genetech: Advisory Board Member, Honoraria; Novarts: DSMB (data and safety monitoring board) member, Honoraria
Relevant financial relationships; discussion of off-label drug use: Jennifer Davila - Genentech: Advisory Board Member, Honoraria; Octapharma: Consultant, Travel Grant; Spire Learning: Speaker Bureau, Honoraria
(A2) Practical Wellness: Taking Care of Your Mind & Body to Battle Burnout
Physician and provider burnout is a recognized problem in all fields of medicine, including in pediatric hematology-oncology. There is increasing literature on the incidence of burnout in our field; however, there is a paucity of data on how to combat burnout and build physician wellness, job satisfaction, and resiliency. There is a need for practical information on how to incorporate wellness techniques into your daily life as a healthcare provider, and how to build a wellness program within our current medical culture and hospital systems. This session will include an experiential component, including meditation and simple sitting and standing yoga poses that can be done at home or at work. This will not be a full yoga class, and the stretching exercises are designed to be accessible to all participants.
Objectives:
- Acquire techniques in meditation and mindfulness and identify practical ways that these techniques can be woven into one's personal and professional life.
- Develop simple yoga skills that can be readily practiced at work and home, to improve physical and mental wellness.
- Learn how to build a wellness program at one's home institution, making it accessible to providers and staff.
Moderator: Jessica L. Heath, MD
Presenters:
- Karen Moody, MD
- Joy Elmore, Registered Yoga Teacher
- Jason Freedman, MD MSC
No relevant financial relationships and no discussion of off-label drug use: Jessica Heath, Karen Moody, Jason Freedman
(A3) Rhabdomyosarcoma: Where Do We Stand and Where Are We Going?
Rhabdomyosarcoma is a rare soft tissue sarcoma with only 350 cases diagnosed each year in the United States. As with all rare diseases, it can be challenging to stay informed of biologic advances and changes in treatment recommendations. This session is designed to update pediatric oncologists on recent results of US and European cooperative group trials and how they may affect treatment recommendations, identification of cancer predisposition syndromes in the context of rhabdomyosarcoma, and developmental therapeutics under investigation for rhabdomyosarcoma.
Objectives:
- Formulate standard of care treatment plans for rhabdomyosarcoma patients by comparing recent cooperative group trial results.
- Recognize which rhabdomyosarcoma patients may have underlying cancer predisposition syndromes and who should be referred for genetic counseling.
- Identify developmental therapies that may be efficacious in patients with relapsed or refractory rhabdomyosarcoma.
Moderator: Wendy Allen-Rhoades, MD
Presentations:
- Clinical Updates for Rhabdomyosarcoma Treatment
Douglas S. Hawkins, MD - Developmental Therapies for Rhabdomyosarcoma
Leo Mascarenhas, MD - Cancer Predisposition and Rhabdomyosarcoma: What You Need to Know and Who You Need to Test
Douglas R. Stewart, MD
No relevant financial relationships; discussion of off-label drug use: Wendy Allen-Rhoades
Relevant financial relationships; no discussion of off-label drug use: Douglas Stewart - Genome Medical, Inc: Consultant, Honoraria
Relevant financial relationships; discussion of off-label drug use: Douglas S. Hawkins - Bayer, LOXO Oncology: Advisory Board Member, Reimbursement for travel; Amgen, Seattle Genetics, Eisai, Merck Sharpe & Dohme, Incyte, Jazz Pharmaceuticals, Eli Lilly, E R Squibb & Sons: Principal Investigator, Research Grant to my Institution; Leo Mascarenhas - Bayer: Consultant, Honoraria; AstraZeneca: Principal Investigator, Research Grant to my Institution; Lilly Oncology: Consultant, Travel reimbursement to present abstract
4–5 pm
Concurrent Sessions
(A4) Multidisciplinary Management of Cerebral Sinus Venous Thrombosis: What's New and What Have We Forgotten?
Neonatal and pediatric cerebral sinus venous thrombosis (CSVT) is frequently associated with significant morbidity and mortality, including neurocognitive dysfunction, brain herniation, and death. Delay in diagnosis or treatment and failure to monitor for elevated intracranial pressure likely contribute to poor outcomes. Optimizing CSVT care requires a coordinated effort between disciplines, but what needs to be done, who does what, and how do we find common ground in the face of management uncertainty? After reviewing the epidemiology, pathophysiology and clinical presentations, this seminar will emphasize a multidisciplinary approach to CSVT, including more accepted (anticoagulation) and emerging (endovascular therapy) therapeutic options.
Objectives:
- Understand the epidemiology, common presentations and pathophysiology of neonatal and pediatric CSVT.
- Review the evidence for anticoagulation in CSVT and describe the potential role for endovascular therapy in CSVT.
- Facilitate the critical interplay between the Neurologist, Ophthalmologist and Hematologist in detecting and managing early and late sequelae of CSVT.
Moderator: Lori Luchtman-Jones, MD
Presenters:
- Char Witmer, MD MSCE
- Alexander Boucher, MD
- John M. Taylor, MD
No relevant financial relationships; discussion of off-label drug use: Char Witmer, Alexander Boucher, John M. Taylor
No relevant financial relationships and no discussion of off-label drug use: Lori Luchtman-Jones
(A5) "Why is My Clinic so SLOW?" Stop Complaining and Do Something About it NOW!
Inefficient clinic patient flow often results from challenges with scheduling and impaired processes. It is a major cause of physician/nursing stress, resulting in wasted resources and poor patient satisfaction. Its downstream effects may cause delays in initiation of treatments (outpatient/inpatient), with unsafe timings and potentially extended stays adding to parental hardship. Despite unique differences in individual practices, there are several useful tools that can improve clinic flow and efficiency. This session demonstrates examples from ASPHO member institutions combining process improvements with current and expanded EMR tools, which can be implemented at other practices.
Objectives:
- Discuss the tools available in EMR systems for measuring clinic flow, and how a multi-disciplinary analysis of this data allows for sustainable improvement.
- Identify how process improvement can be implemented on an ongoing basis in clinics to make improved clinic efficiency a sustainable goal.
- Recognize how Real-Time Location System (RTLS) can be implemented and used in the outpatient clinic.
Moderator: Amir Mian, MD MBA
Presentations:
- Using Real Time Locating System (RTLS) in Adult and Pediatric Oncology Clinics to Improve Clinic Flow
Amy L. Billett, MD - Implementing and Maintaining Process Improvement in the Outpatient PHO Clinic
Hung Tran, MD - How to Use the EMR Tools You Already Have to Measure and Improve Clinic Flow
Rachel True, BSc
No relevant financial relationships and no discussion of off-label drug use: Amir Mian, Amy L. Billett, Hung Tran, Rachel True
(A6) Iron Overload in Pediatric Oncology and BMT Patients – Screening, Management and Long-Term Toxicity
Transfusional iron overload may cause extensive organ toxicity and remains a leading cause of death among patients with transfusion-dependent anemias. Principal organs at risk include heart, liver, pancreas and pituitary gland. Iron overload can also put patients at risk for opportunistic infections. Relatively little is known about the long-term clinical consequences of transfusional iron overload in childhood cancer survivors and patients who undergo hematopoietic stem cell transplantation for non-malignant and malignant disorders. Following this session, participants will be knowledgeable about iron overload screening and management options in childhood cancer survivors and patients undergoing blood and marrow transplantation.
Objectives:
- Describe iron metabolism and regulation and the role of non-transferrin bound iron in potential complications in intensively treated pediatric oncology and BMT patients.
- Recognize the potential long-term consequences of iron overload.
- Demonstrate different laboratory and imaging surveillance modalities that can be used in surveillance of iron overload during and after treatment in pediatric cancer and BMT survivors.
Presentations: - Introduction
Catherine Aftandilian, MD - Review of Iron Metabolism and Regulation; Role of Non-transferrin Bound Iron in Infection and Organ Toxicity
Elizabeta Nemeth, PhD - Iron Overload Among Childhood Cancer Survivors; Potential Long-Term Organ Toxicities and Clinical Impact of Iron Overload
David R. Freyer, DO MS - Surveillance and Management of Iron Overload
Nahal R. Lalefar, MD
No relevant financial relationships and no discussion of off-label drug use: Catherine Aftandilian, David R. Freyer, Nahal R. Lalefar
Relevant financial relationships; no discussion of off-label drug use: Elizabeta Nemeth - Intrinsic LifeSciences, Silarus Therapeutics: Stock Holder, Ownership Interest; Ionis, Protagonist: Consultant, Honoraria
5:15–6:15 pm
Clinical Conundrums
Join one of these sessions and interact with a recognized expert on a challenging diagnostic and management issue. Each session is scheduled for 1 hour and will include a 15-20 minute presentation by the expert on their general approach to the clinical problem followed by small group discussion of additional cases shared by the attendees.
Advanced registration is required. Seating is limited to 30. The registration fee is $25.
(CC1) Management of Relapsed/Refractory T-cell ALL
David T. Teachey, MD
(CC2) Thrombophilia Testing
Courtney D. Thornburg, MD MS
No relevant financial relationships and no discussion of off-label drug use: Courtney D. Thornburg
Relevant financial relationships; discussion of off-label drug use: David T. Teachey - Novartis: Researcher, Research Grant to my Institution; Amgen, La Roche: Advisory Board Member, Honoraria; Janssen: Advisory Board Member, Research Grant to my Institution; Humanigen: Advisory Board Member, No Compensation Received
ASH-ASPHO Choosing Wisely® List Presentation
Choosing Wisely® is an initiative that seeks to advance a national dialogue on avoiding unnecessary medical tests, treatments, and procedures. ASPHO has collaborated with the American Society of Hematology (ASH) to develop a five-item pediatric hematology-focused Choosing Wisely list. This presentation will highlight these impactful, evidence-based item recommendations.
Moderator: Sarah O’Brien, MD MSc
Presenters:
- Cassandra D. Josephson, MD
- Jacquelyn M. Powers, MD, MS
No relevant financial relationships and no discussion of off-label drug use: Sarah O’Brien
Relevant financial relationships; discussion of off-label drug use: Jacquelyn M. Powers - American Regent: Site PI for industry-sponsored trial, research grant to my institution
Physician Scientist Special Interest Group
Leadership: Kathleen M. Sakamoto, MD PhD; Sinisa Dovat, MD PhD
Introduction to Grant Writing and Mentoring Physician Scientists
Speakers: Kathleen M. Sakamoto, MD PhD, Sinisa Dovat, MD PhD
Integrative Pediatric Hematology/Oncology Special Interest Group
Leadership: Holly Spraker-Perlman, MD MS; Tracey Jubelirer, MD
Palliative Care Special Interest Group
Leadership: Julienne Brackett, MD; Katharine Brock, MD; Lawrence Wolfe, MD
Genius Bar
Bring your questions and any cases you’d like to discuss on Iron Overload. The speakers from the “Iron Overload in Pediatric Oncology and BMT Patients – Screening, Management and Long-Term Toxicity” session will help answer questions and guide the discussion.
6:15–7 pm
New Member, First Time and International Attendee Reception
Join new members, first-time attendees, and international attendees during this opportunity to connect and network at the start of the ASPHO Conference. ASPHO's Board of Trustee and Membership Committee members will be in attendance to greet and chat attendees.
7–9 pm
Division Directors Dinner Meeting
Join fellow Division Directors for an opportunity to network and connect around topics important to your work.
Advanced registration is required. The registration fee is $100.
7–8:30 pm
CME Corporate Dinner Symposium
Targeting the RAS/MAPK Pathway in Neurofibromatosis Type 1 and Plexiform Neurofibromas: Improving Patient Outcomes With an Innovative Therapeutic Approach
Thursday, May 7
6:30–7:30 am
5K Fun Run/Walk
ASPHO is holding its seventh annual 5k Fun Run/Walk event.
Advanced registration is recommended. Registration fee is $25. After May 5, fee is $35.
6:30–8:00 am
CME Corporate Breakfast Symposium
Shifting the Paradigm in the Management of Hemophilia A: Transitioning to a Young Adult
7–8 am
Diversity and Hemoglobinopathy Joint SIG Workshop
The Hemoglobinopathy and Diversity SIGs will have a joint featured speaker to highlight their shared interests. Lydia H. Pecker, MD Assistant Professor of Hematology at Johns Hopkins University School of Medicine. Dr. Pecker will discuss her work with young adults with sickle cell disease, especially their reproductive health. Her presentation will also connect issues of diversity in clinical research and challenges of securing research funding.
Advanced Practice Provider Special Interest Group
Leadership: Nicholette Breier-Adkins, MSN, RN, CPNP; Sarah Hall, MHS, PA-C
The Advanced Practice Provider Special Interest Group provides a forum for the exchange of ideas and information among advanced practice providers (APPs) and for promotion of their role in the field of pediatric hematology, oncology, and transplant. At this year’s meeting, we will be featuring the research of two APPs and will introduce the recipient of the APP Travel Award. We will also discuss updates and status of the new ASPHO APP mentorship program as well as plans for SIG activities in the coming year.
Clinical Immunology Special Interest Group
Leadership: David Buchbinder, MD; Shanmuganathan Chandrakasan, MD
Primary Immunodeficiency and Malignancy – Who and How to Test
The ASPHO Clinical Immunology Special Interest Group Workshop will provide a case-based discussion focusing on malignancy and primary immunodeficiency disorders. Three cases will be presented including the following: 1) a case demonstrating malignancy as a presenting feature of a primary immunodeficiency disorder, 2) a case in which a patient with a known primary immunodeficiency disorder develops a malignancy, and 3) a case demonstrating the relationship between autoimmunity which predisposes to malignancies in the context of a primary immunodeficiency disorder. A brief review of those factors that should raise the suspicion of an underlying primary immunodeficiency disorder in the context of providing care to pediatric hematology / oncology patients will be provided. Finally, a brief review of the diagnostic approach to patients with malignancy in association with a suspected primary immunodeficiency will be provided.
Speakers: Nicola Wright, MD, Blachy Davila-Saldana, MD, Kim Nichols, MD, and Sharat Chandra, MD
Quality Special Interest Group
Leadership: Meghan Drayton Jackson, DO, MBOE, CPPS; Christine Moore Smith, MD
Navigating the American Board of Pediatrics (ABP) Maintenance of certification (MOC) Part 4 requirements for QI project approval
Speakers: Laura Couch, ABP Continuing Certification Quality and Education Representative; Courtney Lyle MD
Panel: Laura Couch, Courtney Lyle MD, Natalie Bezler MD, Meghan Drayton Jackson DO
8:15–9:15 am
Concurrent Paper Sessions
This session will consist of four 15-minute presentations of research from selected basic and translational science related abstracts.
Moderators: TBD
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) ENHANCEMENT OF ANTI-TUMOR IMMUNITY BY T CELLS ENGINEERED TO RESTORE HLA EXPRESSION IN NEUROBLASTOMA
Fiorella Iglesias, MD
(2006) HIF EXPRESSION IN ANTIGEN-SPECIFIC AND CAR-T CELLS IMPROVES ANTITUMOR RESPONSE TO SOLID TUMORS
Colette Lauhan, MD
(2007) OBESITY IMPAIRS T-CELL FUNCTION WHICH MAY IMPACT THE EFFICACY OF CAR T-CELLS IN PEDIATRIC LEUKEMIA
Anthony Ross, MD
(2008) TARGETING ADENOSINE IN THE MARROW MICROENVIRONMENT AFFECTS STROMAL AND LEUKEMIA CELL SURVIVAL
Craig A. Mullen, MD PhD
No relevant financial relationships and no discussion of off-label drug use: Fiorella Iglesias, Colette Lauhan, Anthony Ross, Craig A. Mullen
Clinical Trials and Outcomes (Hematology)
This session will consist of four 15-minute presentations of research from selected hematology clinical trials and outcomes related abstracts.
Moderators: TBD
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) FOLLOW-UP OF HYDROXYUREA (HU) IN INFANTS WITH SICKLE CELL ANEMIA(SCA):FINDINGS FROM BABY HUG STUDIES
Zora R. Rogers, MD
(2010) TCD VELOCITIES CONVERSION RATE BASED ON INCREASING HB CONCENTRATION: ANALYSIS FROM THE SCCRIP COHORT
Jeremie H. Estepp, MD
(2011) HOSPITALIZATION FOR IRON DEFICIENCY ANEMIA IN YOUNG CHILDREN: A MULTICENTER ANALYSIS
Julia Segal, MD
(2012) THE SHWACHMAN-DIAMOND SYNDROME REGISTRY: 10 YEAR UPDATE
Kasiani C. Myers, MD
No relevant financial relationships and no discussion of off-label drug use: Julia Segal, Kasiani C. Myers
No relevant financial relationships; discussion of off-label drug use: Zora R. Rogers
Relevant financial relationships and no discussion of off-label drug use: Jeremie H. Estepp - Global Blood Therapeutics, Daiichi Sankyo: Advisory Board Member, Honoaria; Global Blood Therapeutics, Pfizer, Novartis, Forma Therapeutics: Principal Investigator, Research Grant to Institution
Clinical Trials and Outcomes (Oncology)
This session will consist of four 15-minute presentations of research from selected oncology clinical trials and outcomes related abstracts.
Moderators: TBD
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) RETROSPECTIVE REVIEW OF NEUROLOGICAL MANIFESTATIONS IN 189 PATIENTS DIAGNOSED WITH CLOVES SYNDROME
Ian Erkkila
(2014) ATTRIBUTES OF HIGH QUALITY END-OF-LIFE CARE FOR CHILDREN WITH CANCER: A STAKEHOLDER-DRIVEN STUDY
Prasanna Ananth
(2015) SELUMETINIB IN CHILDREN WITH CLINICALLY ASYMPTOMATIC INOPERABLE NF1 RELATED PLEXIFORM NEUROFIBROMAS
Brittany Glassberg, BS
(2016) LAROTRECTINIB EFFICACY AND SAFETY IN PEDIATRIC PATIENTS WITH TRK FUSION CANCER
Catherine M. Albert, MD
No relevant financial relationships and no discussion of off-label drug use: Ian Erkkila, Prasanna Ananth, Brittany Glassberg, Catherine Albert
9:45–11:15 am
(B7) ASPHO/PTCTC Joint Session: Cellular Therapy for Sickle Cell Disease
Sickle cell disease (SCD) continues to cause morbidity and mortality in affected patients. Until now, the only curative option has been hematopoietic stem cell transplant (HSCT). Alternative donor HSCT continues to be evaluated as a potential option for more patients. Additionally, there are developments in the use of gene therapy and gene editing strategies as curative modalities. The attitudes and beliefs of patients and family members in the SCD community with regards to these emerging therapies are not well understood. Awareness of new developments and patient/family perspectives are both important for practitioners to understand as we discuss emerging treatment options.
Objectives:
- Discuss the use of HSCT for sickle cell disease, including alternative donor HSCT.
- Summarize the recent developments in gene editing techniques and gene therapy for sickle cell disease and discuss their clinical implications.
- Review the emerging literature in the space of patient, family, and community engagement in curative therapies for sickle cell disease.
Moderator: Christine N. Duncan, MD MMSC
Presentations:
- Gene Editing for Sickle Cell Disease
Daniel Bauer, MD PhD
Lori Crosby, PhD
Monica Bhatia, MD
No relevant financial relationships and no discussion of off-label drug use: Lori Crosby
No relevant financial relationships; discussion of off-label drug use: Monica Bhatia
Relevant financial relationships; no discussion of off-label drug use: Christine N. Duncan - AztraZeneca, SpringWorks: Consultant, No Compensation Received; Daniel Bauer - Pfizer, Merck: Consultant, Honoraria
(B8) Risk-adapted Therapy for Medulloblastoma: The Integration of Molecular Discoveries into Clinical Care to Reduce Late Effects of Treatment
Over the past decade, we have gained a clearer understanding of the molecular pathogenesis and epigenetic heterogeneity of medulloblastoma. Because of the breadth and frequency of recent scientific discoveries, many pediatric oncologists are unsure how to integrate these molecular advances into clinical care. This session will review the integrated molecular and clinical risk stratification of medulloblastoma and highlight carefully chosen groups of patients in which therapy de-escalation should be clinically investigated in order to decrease long-term treatment-induced morbidities. This session will improve participants' understanding of recent biologic advances in medulloblastoma and highlight their clinical implications.
- Define risk groups of medulloblastoma based on molecular, epidemiologic, and clinical outcomes data.
- Identify risk groups of childhood medulloblastoma that are candidates for less intensive therapy in order to reduce late effects without impacting survival outcomes.
- Formulate strategies to decrease treatment-related neurocognitive deficits in infants and young children with medulloblastoma while improving survival outcomes.
Moderator: Holly Lindsay, MD MS
Presentations:
- Biologic Advances in Medulloblastoma: Toward a Molecularly-Based Risk Stratification in Medulloblastoma
Vijay Ramaswamy, MD PhD - De-Escalation of Therapy in Childhood Medulloblastoma: Historical Experience and Future Directions
Jeff Michalski, MD - De-escalation of Therapy in Infant Medulloblastoma: Identifying the Appropriate Patients
Sarah Leary, MD MS
No relevant financial relationships and no discussion of off-label drug use: Holly Lindsay, Vijay Ramaswamy, Jeff Michalski
No relevant financial relationships; discussion of off-label drug use: Sarah Leary
(B9) Cardio-oncology: Beyond Anthracyclines and Echocardiograms
The purpose of this session is to address gaps in our understanding of cardiovascular complications associated with cancer therapy in pediatric patients. First, we will emphasize that the risk for these outcomes is not limited to anthracycline exposure. Second, we will discuss the rationale for a multidisciplinary team approach to address the cardiovascular complications due to cancer therapy, and present the concept of a preventive cardio-oncology clinic. Lastly, we will discuss how to combine different imaging techniques with blood biomarkers to identify heart disease in patients undergoing cancer treatment and in survivors of childhood cancer.
Objectives:
- Describe the current understanding of cardiovascular toxicities associated with anticancer drugs, including targeted agents and checkpoint inhibitors, and radiation therapy.
- Recognize the importance of a multidisciplinary approach to prevent, detect and diagnose the cardiovascular complications associated with cancer treatment.
- Discuss the potential role of biomarkers and different imaging modalities in the evaluation of cardiovascular outcomes of cancer treatment.
Presentations:
- Introduction: What is Cardio-oncology?
Nino C. Rainusso, MD - Prevention Strategies in Children with Cancer
Saro Armenian, MD - Evaluation of Cardiotoxicity in Cancer Patients
William Border, MBChB MPH - Multidisciplinary Approach to Prevent and Treat Cardiovascular Complications in Pediatric Cancer Patients
Robert W. Loar
No relevant financial relationships and no discussion of off-label drug use: Saro Armenian, William Border, Robert W. Loar
No relevant financial relationships; discussion of off-label drug use: Nino C. Rainusso
11:30 am–12:20 pm
Business Meeting
ASPHO's annual business meeting allows attendees to learn more about the Society's progress toward meeting the goals of its strategic plan, as well as learn about the Society's financial health. Additionally, outgoing Board members and committee chairs are recognized, a report is presented from the Pediatric Blood & Cancer Journal editor, and the Clinician Educator Awardee(s) and 5K Fun Run winners are recognized.
All are welcome and encouraged to attend!
12:30–1:45 pm
Early Career Roundtable Luncheon
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.
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.
Clinical Research – Oncology
Mark Atlas, MD
Linda C. Stork, MD
Clinical Research – Hematology
George R. Buchanan, MD
Jeffrey M. Lipton, MD PhD
Basic Science/Translational Research
Kathleen M. Sakamoto, MD PhD
Daniel S. Wechsler, MD PhD
Clinician Educator
Caroline A. Hastings, MD
Pinki Prasad, MD MPH
YoungNa Lee-Kim, MD
Medical Students/Residents
Maria C. Velez, MD
Thomas Russell, MD
Kimberly Whelan, MD MSPH
Pharmaceutical Industry
TBD
Cell & Gene Therapies
TBD
Advanced registration is required. The registration fee is $30.
Recommended by the Professional Development Committee.
No relevant financial relationships and no discussion of off-label drug use: Mark Atlas, George R. Buchanan, Caroline A. Hastings, YoungNa Lee-Kim, Jeffrey M. Lipton, Pinki Prasad, Kimberly Whelan, Kathleen Sakamoto, Linda C. Stork
Relevant financial relationships; no discussion of off-label drug use: Maria C. Velez - NovoNordisk: Principal Investigator, Research Grant to my Institution; Kendrion, CSL Behring: Advisory Board Member, Honoraria; Daniel S. Wechsler - American Board of Pediatrics: Advisory Board Member, Honoraria; Hyundai Hope on Wheels Medical Advisory Board: Advisory Board Member, Other Financial Benefit, Pediatric Blood & Cancer: Associate Editor, Honoraria
12:30–1:30 pm
Clinical Conundrums
Join one of these sessions and interact with a recognized expert on a challenging diagnostic and management issue. Each session is scheduled for 1 hour and will include a 15-20 minute presentation by the expert on their general approach to the clinical problem followed by small group discussion of additional cases shared by the attendees.
Advanced registration is required. Seating is limited to 30. The registration fee is $25.
(CC3) CAR-T Cell Therapy versus HSCT for Treatment of R/R ALL
Michael A. Pulsipher, MD
(CC4) Neurologic Complications in Sickle Cell Disease: Recognition. Prevention, and Management
Michael R. DeBaun, MD MPH
Relevant financial relationships; no discussion of off-label drug use: Michael A. Pulsipher - Novartis: Steering Committee, Honoraria; Bellicum: Speaker, Honoraria; Miltenyi, Adaptive: Study Support; Novartis: Advisory Board Member, Honoraria; Michael R. DeBaun - Global Blood Therapeutics: Advisory Board Member, Honoraria
Well Being Special Interest Group
Leadership: Jonathan Fish, MD; Adit Tal, MD
Setting Direction for our Well Being
Burnout is now well-recognized as an occupational hazard in medicine, and pediatric hematology / oncology is not immune. Despite increasing awareness of the problem, strategies to maintain well-being and prevent burnout have remained highly localized, non-uniform and underreported. The mission of the Well Being SIG is to develop and maintain a community of practice surrounding well-being in pediatric hematology / oncology, in part by providing a forum in which to share strategies and ideas. This inaugural workshop of the ASPHO Well Being SIG will include an introduction to the SIG and its leadership, a discussion of the results of a survey of the SIG members to set the direction of the SIG, and presentation of several well-being strategies currently implemented in divisions of pediatric hematology / oncology.
Adolescent and Young Adult Special Interest Group
Leadership: Stefanie Thomas, MD; Jacquelyn Baskin, MD
Global Pediatric Hematology Oncology Special Interest Group
Leadership: Patrick McGann, MD MS; Joseph Lubega, MD; Andrea Orsey, MD
12:30–1:45 pm
Non-CME Corporate Luncheon Symposium (Hematology Topic)
Gene Therapy: New Horizons for b-Hemoglobinopathies
2–3:30 pm
Concurrent Sessions
(B10) Updates on Thrombotic Microangiopathy Syndrome
Thrombotic microangiopathy (TMA) is a pathologic lesion that leads to abnormalities in the vessel wall leading to microvascular thrombosis. The pathophysiology of TMA syndromes, including acquired and hereditary thrombotic thrombocytopenic purpura (TTP), complement-mediated TMA and hemolytic uremic syndrome (HUS), varies. The diagnosis and treatment of TMA, whether primary or secondary, has evolved over the last several years. Pediatric Hematologists and Oncologists need to better understand these TMA syndromes in terms of their pathologies, newer treatments, the impact on renal function and implications in the transplant setting.
Objectives:
- Identify transplant associated TMA (TA-TMA) and its treatment implications.
- Identify clinical features and new treatments for hereditary and acquired TTP.
- Recognize the renal impact of TMA.
Moderators: Sonata Jodele, MD; Benjamin Laskin, MD
Understanding Thrombotic Thrombocytopenic Purpura: Hereditary and Acquired
James N. George, MD
No relevant financial relationships; discussion of off-label drug use: James N. George, Benjamin Laskin
Relevant financial relationships; discussion of off-label drug use: Sonata Jodele - Pending patents, no compensation received
(B11) FDA Pediatric Hematology and Oncology: Updates and Opportunities
FDA plays a crucial role the development, approval, and safe use of drugs and biologic agents in pediatric patients with cancer and blood diseases. Pediatric hematologists-oncologists from the FDA will provide an update on recent new drug approvals in pediatrics, and tips for every-day, practical use of FDA resources including locating important pediatric information in hematology and oncology product labels. The session will include a review of new legislation promoting pediatric drug development. The FDA will also provide tips to streamline the process of obtaining compassionate use access to investigational treatments. This session will utilize scenario-based discussions, and answer participant questions regarding the regulatory review process.
Objectives:
- Understand FDA regulations and role in the development of safe and effective therapies for pediatric cancers and ways to engage with FDA to further the goals of the pediatric oncology community.
- Gain awareness of recent FDA drug approvals in pediatrics and data supporting approval.
- Locate and review important pediatric dosing and toxicity information in the drug and biologic product label.
- Recognize the role of expanded access to investigational treatments in the treatment of pediatric patients unable to enroll in clinical trials and the procedures for obtaining expanded access.
- Recognize the role of legislation in pediatric drug development for pediatric patients with cancer.
Presentations:
- Overview of FDA Mission and Session Objectives
Lori Ehrlich, MD, PhD - What's Involved in Review and Overview of Recent FDA Approvals in Pediatrics
Najat Bouchkouj, MD - Important Resources for Clinicians in the Drug Label
Sonia Singh, MD - Update on New Legislation (FDARA) and Regulations Promoting Pediatric Oncology Drug Development
Diana Bradford, MD
No relevant financial relationships and no discussion of off-label drug use: Lori Ehrlich, Najat Bouchkouj, Sonia Singh
No relevant financial relationships; discussion of off-label drug use: Diana Bradford
(B12) Approach to the Patient with Recurrent Sarcoma
Decision making for recurrent sarcomas is becoming increasingly complex with expanding options for both systemic and local treatment. Personalized medicine approaches further complicate therapy prioritization, and recent palliative care advances may offer symptom control apart from cancer-directed therapy. Oncologists often lack confidence in their knowledge and competency to counsel families about treatment options, and patients may not receive cancer-directed and palliative therapies appropriate for their prognosis and goals of care. This session will utilize a case-based format to enhance attendees' knowledge about the range of available treatment options and how to prioritize these to support goal-directed care.
Objectives:
- Know what issues merit consideration in planning therapy for recurrent sarcoma patients.
- Understand the potential value of tumor genomic profiling in recurrent sarcomas and how to integrate this information into treatment planning.
- Understand available palliative therapy options for recurrent sarcomas.
Presentations:
- Case Presentation and Commentary
Sheri L. Spunt, MD MBA - Case Presentation and Commentary
Cindy L. Schwartz, MD MPH - Personalized Genomics
Avanthi Shah, MD - Palliative Care
Katharine Brock, MD MS - Radiation Therapy
Stephanie Terezakis, MD
No relevant financial relationships and no discussion of off-label drug use: Sheri L. Spunt, Cindy L. Schwartz; Katharine Brock
No relevant financial relationships; discussion of off-label drug use: Avanthi Shah
Relevant financial relationships; no discussion of off-label drug use: Stephanie Terezakis - ASELL, Elekta Industries: Principal investigator, research grant to my institution
3:45–4:30 pm
St. Baldrick's Foundation Robert J. Arceci Innovation Award Announcement and Presentation
The St. Baldrick’s Foundation Robert J. Arceci Innovation Award was established in loving memory of Robert J. Arceci, MD PhD. Two awards are presented per year, one to a U.S. or Canadian researcher to be presented at the ASPHO Conference, 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.
Alex Kentsis, MD PhD, the 2018 award recipient, will be presenting the outcomes of his research.
How do young people develop cancer? Most have not lived long enough to accumulate mutations due to aging or environmental exposure, and most do not have any apparent cancer predisposition from inheritance of cancer-causing gene mutations. Recently, my laboratory has discovered an unanticipated cause of childhood solid tumors, resulting from the aberrant activity of the PGBD5 DNA transposase that can promote rearrangements of human genes. Related mechanisms are responsible for chromosomal translocations and deletions of tumor suppressor and oncogenes induced by the RAG1/2 DNA recombinase in lymphoid leukemias and lymphomas, and unexpectedly in a large proportion of childhood myeloid leukemias. We reason that PGBD5, RAG1/2 and other domesticated DNA transposases are normally controlled to promote development of healthy cells in early life, but become dysregulated in cells that give rise to various tumors in children and young adults. Here, I will present recent evidence for this hypothesis, and outline future studies needed to understand the causes of childhood cancers and rational strategies for their improved treatment.
Objectives:
- Review causes of somatic mutations in developing tissues
- Define domesticated DNA transposases and their evolutionary origins and physiologic functions
- Outline current and future research to translate these insights into improved therapies for patients.
No relevant financial relationships and no discussion of off-label drug use: Alex Kentsis, MD PhD
4:45–6 pm
Presidential Symposium: Artificial Intelligence (AI) in Pediatric Hematology Oncology – Not Just a Neural Net!
Artificial intelligence (AI), within the discipline of which machine learning (ML) is an essential technique, delivers the potential to fundamentally affect healthcare. Digital image analysis using ML tools discerns malignant from benign lung nodules, adds to risk classification of glioma and accurately classifies clinical images of melanoma, while the field of natural language processing is beginning to analyze text-based data within the electronic medical record. Pediatric Hematologist/Oncologists who will with certainty experience the impact of AI in their patient care, will learn about this AI/Medicine intersection.
Objectives:
- Discuss the fundamental concepts of artificial Intelligence and machine learning
- Identify the opportunities for application of artificial intelligence tools in the discipline of pediatric hematology/oncology
Moderator: Patrick Leavey, MD
Presentations:
- AI 101 – What Is It?
Anthony Chang, MD MBA MPH MS - Data Driven AI
Adam Resnick, PhD - Case-Based Study—AI at Work
Ovidiu Daescu, PhD
No relevant financial relationships and no discussion of off-label drug use: Patrick Leavey, Anthony Chang, Adam Resnick, Ovidiu Daescu
6–7:30 pm
Opening Reception with Exhibits and Posters
Sponsored in part by Cook Children’s Health Care System
6–7 pm
Author-Attended Poster Session: Odd-numbered Posters
Meet the SIGs
6:15–6:45 pm
Non-CME Education Theater
Targeting IFNy In Primary Hemophagocytic Lymphohistiocytosis (HLH)
7–7:30 pm
Even-numbered Poster Tour
ASPHO will provide two poster tours during exhibit hours. The Conference Planning Committee will select five highly ranked posters that will be included in a 30-minute tour for all interested attendees. Authors of these posters will provide a brief overview of their research and answer attendees' questions. Advanced registration is not required, and the tour is open to all attendees. Don't miss the opportunity to receive a quick overview of top-rated abstracts!
7:30–9 pm
Fellowship Program Directors Dinner Meeting
Join fellow Fellowship Program Directors for an opportunity to network and connect around topics important to your work.
Advanced registration is required. The registration fee is $85.
7:30–9 pm
Non-CME Corporate Dinner Symposium
Friday, May 8
6:30–8 am
Non-CME Corporate Breakfast Symposium
7:30–8:30 am
Continental Breakfast with Exhibits and Posters
8:30–9:30 am
Northwestern Mutual Award for Excellence in Childhood Cancer Survivorship Announcement
ASPHO will present the Northwestern Mutual Award for Excellence in Childhood Cancer Survivorship, funded by the Northwestern Mutual Foundation. The award will recognize an individual who has translated research findings into intervention-based approaches and/or has made outstanding contributions to the clinical care of survivors of childhood cancer.
Funded by: Northwestern Mutual Foundation
Frank A. Oski Memorial Lectureship Award Presentation
The annual Frank A. Oski Memorial Lectureship Award gives us an opportunity to remember and honor Dr. Oski and his many contributions to the field of pediatric hematology/oncology. It is awarded to an outstanding clinical or laboratory investigator in pediatric hematology/oncology whose cutting-edge research has significantly impacted the field. This year's lectureship will be presented by Akiko Shimamura, MD PhD, Dana-Farber/Boston Children's Cancer and Blood Disorders Center.
Bone Marrow Failure and MDS: Lessons From Rare Diseases
Objectives:
- Summarize Precision Medicine approaches for pediatric bone marrow failure and MDS.
- Describe clinical implications of recent advances in the molecular pathogenesis of bone marrow failure and MDS.
Relevant financial relationships; discussion of off-label drug use: Akiko Shimamura - Novartis: Local investigator for Novartis-sponsored trial, no financial benefit received.
9:45–10:45 am
Concurrent Sessions
(C13) Emicizumab in Pediatric Patients with Hemophilia A: Decisions You Will Be Expected to Make in the Clinic
Emicizumab, a bispecific monoclonal antibody, restores the function of activated FVIII by bridging activated FIX and FX. Subcutaneous Emicizumab was recently approved for prophylaxis in patients with hemophilia A (both with and without inhibitors). However, in order to maximize its therapeutic potential, clinical experience of using Emicizumab needs to be widened, specifically in situations like combination with other hemostatic agents, laboratory-monitoring, management of breakthrough bleeds and surgery. This need is particularly pronounced in non-inhibitor patients. This case-based, interactive session will summarize the current knowledge on Emicizumab, and equip participants with up-to-date, evidence-based information on the appropriate use of this agent.
Objectives:
- Review the mechanism of action of Emicizumab and discuss principal findings from the recently published HAVEN clinical trials.
- Review the role of Emicizumab in previously untreated patients (PUPs) with hemophilia A, newly diagnosed inhibitor patients and tolerized inhibitor patients, specifically contrasting it to current standard of care.
- Review laboratory monitoring of patients on Emicizumab, discuss treatment options for breakthrough bleeds and perioperative hemostatic strategies for major and minor surgical procedures.
Presentations
- Mechanism of Action of Emicizumab and Principal Findings from the HAVEN Clinical Trials
Riten Kumar, MD MSc - Management of a Newly Diagnosed Inhibitor Patient in the Emicizumab Era
Guy Young, MD - Emicizumab versus FVIII in Previously Untreated Hemophilia A Patients: Pros and Cons
Robert Sidonio, MD, MSc. - My Patient on Emicizumab is Bleeding/Needs Surgery – What Should I Do?
Manuel Carcao, MD MSc FAAP FRCPC
Relevant financial relationships; no discussion of off-label drug use: Riten Kumar - Bayer, Genentech: Advisory Board Member, Honoraria; Guy Young - Genentech/Roche, Grifols, Sanofi/Genizyme, Takeda, UniQure, Freeline, CSL Behring: Consultant, Honoraria; Robert Sidonio - Genentech: Principal Investigator, Research Grant to my Institution; Manuel Carcao - Bioverativ/Sonofi, Novo Nordisk: Principal Investigator, Research Grant to my Institution; Bioverativ/Sonofi: Advisory Board Member, Honoraria; Bayer, Novo Nordisk, Octapharma, Takeda, Grifols: Speaker Bureau, Honoraria; Roche: Consultant, Honoraria; LFB: Consultant, No Compensation Received
(C14) Post-Transplant Lymphoproliferative Disorder: Utilization of IPTA EBV/PTLD Consensus Evidence-Based Practice Guidelines
In March of 2019, the International Pediatric Transplant Association (IPTA) sponsored a consensus conference assembling experts from across the globe to develop Evidence-Based Practice Guidelines for Post-Transplant Lymphoproliferative Disorder (PTLD) in Children. Internationally recognized authorities from transplant medicine, infectious disease, pathology and oncology developed guidelines for the prevention, diagnosis, evaluation and management of pediatric patients with PTLD. This session will assemble attendees from this conference, and cases will be presented that highlight the key elements from the generated guidelines. The audience will be engaged to render their opinions and perspectives to provide a lively forum of discussion.
Objectives:
- Educate our community on the Evidence Based Practice Guidelines generated from the IPTA EBV/PTLD Consensus conference, to fill in gaps in knowledge and to disseminate the consensus guidelines from the internationally recognized authorities in the field.
- Assist clinicians in translating the consensus guidelines to make them relevant to their own clinical practice, as there have been few standardized practice guidelines published in the past.
Presentations:
- Overview; Definitions and Diagnosis
Robert J. Hayashi, MD - Prevention
Thomas Gross, MD PhD - Management
Catherine Bollard, MD MBChB - Viral Load
Michael Green, MD MPH
No relevant financial relationships and no discussion of off-label drug use: Robert J. Hayashi
No relevant financial relationships; discussion of off-label drug use: Thomas Gross, Michael Green
Relevant financial relationships; discussion of off-label drug use: Catherine Bollard—Caballetta Bio: Board of Directors, Stock Options; Mana Therapeutics: Founder, Stock Options; Torque and Neximmune: Stock Holder, Stock; Cellectis: Advisory Board Member, Honoraria
(C15) Financial Toxicity – A Critical Gap in Care for Survivors of Childhood Cancer
Cancer survivors face unique lifelong challenges including therapy related toxicity, higher burden of chronic diseases and quality-of-life issues. For survivors, financial toxicity (from cancer treatment) remains largely ignored despite its adverse impact: ~50% experience moderate-severe lifelong financial hardship that correlates with poor quality-of-life, 2.6 times higher-risk of filing for bankruptcy, 2.1 times increased suicidal ideation and overall high-risk for early mortality. Clinicians are unaware of financial toxicity and, without standardized screening tools, are unable to direct resources for timely intervention. With increasing out-of-pocket expenses, the prevalence and degree of financial toxicity is expected to worsen, thereby, adversely affecting long-term patient outcomes.
Objectives:
- Describe prevalence and adverse effects of financial toxicity on long-term outcomes for cancer-survivors.
- Highlight physical and psycho-social challenges that could add to undue burdens and potentially exacerbate financial toxicity.
- Develop preventative strategies for timely intervention and management of financial toxicity for childhood cancer patients.
Presentations:
- Introduction and Overview of Cancer Patient's Financial Toxicity and Potential Gaps in Knowledge
Amir Mian, MD MBA - Financial Toxicity: An Under-appreciated Late Effect of Childhood Cancer
Melissa M. Hudson, MD - A Public Health Approach to Financial Toxicity: From Primary to Tertiary Prevention
Fumiko Chino, MD - Financial Toxicity: It is Not One Size Fits All
Linda Rivard, RN
No relevant financial relationships and no discussion of off-label drug use: Amir Mian, Melissa M. Hudson, Fumiko Chino, Linda Rivard
10:45–11:45 am
Break with Exhibits and Posters
11–11:30 am
Odd-Numbered Poster Tour
ASPHO will provide two poster tours during exhibit hours. The Conference Planning Committee will select five highly ranked posters that will be included in a 30-minute tour for all interested attendees. Authors of these posters will provide a brief overview of their research and answer attendees' questions. Advanced registration is not required, and the tour is open to all attendees. Don't miss the opportunity to receive a quick overview of top-rated abstracts!
Genius Bar
Bring your questions and any cases you’d like to discuss on PTLD. The speakers from the “Post-Transplant Lymphoproliferative Disorder: Utilization of IPTA EBV/PTLD Consensus Evidence-Based Practice Guidelines” session will help answer questions and guide the discussion.
Non-CME Education Theater
11:45 am–12:30 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 Donald Mahoney, MD, from Texas Children's Hospital.
An Adventurer's Journey: Forty-Three Years as a Pediatric Hematologist Oncologist
This presentation will provide reflections on a forty-three year adventure of discovery in pediatric hematology at Texas Children’s Cancer and Hematology Center. Clinical research endeavors in the fields of ALL, CNS tumors, bone marrow transplantation, and finally the transition to a broad array of challenging clinical problems within pediatric hematology will be briefly highlighted. These transitional stages were marked by challenges, uncertainties, some disappointments, as well as rewards and immense satisfaction. Career transitions within the institution were ultimately driven by real-time clinical needs assessment and by a drive to advocate for the best medical care for patients with hematological and/or oncological conditions, especially for patient groups that did not rise to a high interest level within our field of practice. This presentation will focus on the journey from protégé to mentor to the most satisfying role of co-mentor and will provide examples of how these mentorship experiences translated into major academic opportunities for fellows and junior faculty.
Objectives:
- Recognize examples of clinical experiences and mentor interactions that can lead to academic opportunities for fellows and faculty.
- Demonstrate the importance of interpersonal relationships in career development.
- Recognize clinical opportunities for co-mentorship.
Relevant financial relationships; no discussion of off-label drug use: Donald Mahoney - Wolters Kluwer: Section Editor - Pediatric Hematology, Honoraria
12:30–2 pm
Lunch with Exhibits and Posters
12:30–1:30 pm
Author-Attended Poster Session: Even-numbered Posters
12:30–1:05 pm
Speed Mentoring Session I
Speed mentoring is an opportunity to network with leaders in the field through a series of short, focused conversations about specific questions. It is a concept that focuses on quick-hit information, allowing for time-efficient networking. Fellows and early career attendees registering for this session will have the opportunity to meet one-on-one with up to three mentors for 10 minutes each. Mentees will be required to submit a one-page document prior to arriving onsite that addresses a specific topic of interest. Mentees should come prepared with two concrete questions to discuss for their chosen subject. Mentors will review the mentees' documents in advance for meaningful insight and efficient engagement.
Each person will be assigned to one of two sessions (either 12:30 pm or 1:10 pm).
Advanced registration is required.
12:40–1:10 pm
Non-CME Education Theater
VITRAKVI®: A First-in-Class TRK Inhibitor For TRK Fusion Cancer Across Solid Tumors1-3
12:40–1:15 pm
ASH-ASPHO Choosing Wisely® List Q&A
Stop by to ask any questions regarding the ASH-ASPHO pediatric hematology-focused Choosing Wisely® list, including those on list development, item evidence or ideas for implementation, in an informal setting.
12:45–1:45 pm
Non-CME Luncheon Symposium (Hematology Topic)
1:10–1:45 pm
Speed Mentoring Session II
Speed mentoring is an opportunity to network with leaders in the field through a series of short, focused conversations about specific questions. It is a concept that focuses on quick-hit information, allowing for time-efficient networking. Fellows and early career attendees registering for this session will have the opportunity to meet one-on-one with up to three mentors for 10 minutes each. Mentees will be required to submit a one-page document prior to arriving onsite that addresses a specific topic of interest. Mentees should come prepared with two concrete questions to discuss for their chosen subject. Mentors will review the mentees' documents in advance for meaningful insight and efficient engagement.
Each person will be assigned to one of two sessions (either 12:30 pm or 1:10 pm).
Advanced registration is required.
1:20–1:50 pm
Non-CME Education Theater
Expert Perspective: A Closer Look at a New Treatment Option for Sickle Cell Disease
2:15–3:45 pm
Concurrent Sessions
(C16) Challenges in Pediatric Transfusion Medicine
Practitioners in pediatric hematology/oncology are often faced with decisions about how and when to transfuse blood products. Certain populations such as neonates and patients with sickle cell disease who require transfusions are particularly challenging. There is increasing interest by health care providers to reduce unnecessary blood transfusions, and providers may encounter patients who refuse blood. Blood-sparing approaches for perioperative management and will be reviewed. A discussion of evidence-based transfusion practices will benefit trainees and experienced clinicians alike.
Objectives:
- Understand indications for transfusion of blood products in the neonate.
- Acquire practical strategies to reduce exposure to transfusions.
- Utilize tools for managing adverse transfusion-related reactions.
Moderator: Clifford Takemoto, MD
Presenters:
- Rowena Punzalan, MD
- Stacey Valentine, MD MPH
- Ross Fasano, MD
No relevant financial relationships and no discussion of off-label drug use: Stacey Valentine
No relevant financial relationships; discussion of off-label drug use: Rowena Punzalan
Relevant financial relationships; no discussion of off-label drug use: Clifford Takemoto - Genetech: Advisory Board Member, Honoraria; Novarts: DSMB (data and safety monitoring board) member, Honoraria; Ross Fasano - Novartis: Advisory Board Member, Honoaria; Terumo BCT: Educational Speaker, Honoraria
(C17) Immunology and Immunotherapy: A Review for the Pediatric Hematologist/Oncologist
With the increasing use of immunotherapeutic approaches, pediatric hematologists and oncologists are faced with the use of treatments that require understanding of clinical immunology. Clinicians often lack basic knowledge of immunology. The primary aim is to understand the immunological underpinnings of immunotherapeutic approaches which are at the forefront of pediatric hematology/oncology care, and illustrate how these underpinnings, with respect to toxicities, may overlap with concepts of (primary) immunodeficiency and immune dysregulation.
Objectives:
- Describe the salient aspects of adaptive immunity that underlie the function and toxicities of immunotherapy (e.g., development and function of T cells, antigen processing, presentation, recognition, effector functions, senescence, and regulatory aspects of the adaptive immune response).
- Illustrate the previously described aspects of adaptive immunity using examples of common immunotherapeutic approaches such as adoptive cell therapy (e.g., Chimeric Antigen Receptor [CAR] T cell therapy) as well as immune checkpoint inhibition (e.g., CTLA-4 blockade, PD-1/PD-L1 blockade).
- Illustrate the previously described aspects of adaptive immunity using examples of toxicities and potential treatment strategies following common immunotherapeutic approaches such as adoptive cell therapy (e.g., CAR T cell therapy) as well as immune checkpoint inhibition (e.g., CTLA-4 blockade, PD-1/PD-L1 blockade).
Moderators: Kim Nichols, MD; Blachy J. Dávila Saldaña, MD
Presenters:
- Jacob Bleesing, MD
- Shanmuganathan Chandrakasan, MD
No relevant financial relationships and no discussion of off-label drug use: Blachy J. Dávila Saldaña, Jacob Bleesing, Shanmuganathan Chandrakasan
Relevant financial relationships; no discussion of off-label drug use: Kim Nichols - Incyte: Principal Investigator, Research Grant to my Institution
(C18) Advances in Genomics of Pediatric Solid Tumors
As the genomic tools for diagnosis and sub-classification of tumors rapidly evolve, they are also becoming more critical to clinical practice. However, the wide variety of genomics testing platforms and the data they provide can be challenging for the practicing oncologist.
Pediatric oncologists should be knowledgeable regarding the different types of genomic testing modalities available and their clinical implications, and they need be able to apply them in clinical practice.
Objectives:
- Describe evolving technologies, specify the most appropriate testing and interpret the data from genomic analyses.
- Distinguish subgroups of pediatric high-grade glioma based on genomic and epigenetic features.
- Be familiar with emerging data on the genomics of solid tumors.
Moderator: TBD
Presenters:
- Somak Roy, MD
- Carl Koschmann, MD PhD
- Sharon Diskin, PhD
No relevant financial relationships and no discussion of off-label drug use: Carl Koschmann, Sharon Diskin
Relevant financial relationships; no discussion of off-label drug use: Somak Roy - Roche Sequencing Solution: Advisory Board Member, Honoraria
4–5:30 pm
Concurrent Sessions
(C19) New Insights into Pediatric Lymphoma
Current efforts in pediatric lymphoma aim to improve outcomes for those with high risk or relapsed disease and limit long-term sequelae for those with highly curable disease. Many new targeted and cellular therapies have been developed in recent years for both Hodgkin and non-Hodgkin lymphomas. However, practitioners may not be familiar with emerging data, and, specifically, how to incorporate these agents into the current treatment landscape.
This session will discuss new developments in therapeutic options for pediatric lymphoma as well as developments in understanding the biology of these malignancies, which informs risk classification and directs therapeutic discoveries.
Objectives:
- Discuss the use of EBV-targeted cellular therapy in pediatric lymphoma.
- Summarize new and targeted therapies for pediatric Hodgkin lymphoma.
- Describe new findings in the biology of pediatric lymphoma and potential implications for therapy.
Presentations:
- Developing T Cell Therapies for Pediatric Lymphomas: Beyond CARs
Catherine Bollard, MD MBChB - Strategies to Incorporate Targeted Agents for Pediatric Patients with Lymphoma
Peter Cole, MD - The Pathogenic and Prognostic Importance of the Tumor Microenvironment in Hodgkin Lymphoma
Christian Steidl, MD
No relevant financial relationships and no discussion of off-label drug use: Peter Cole
Relevant financial relationships; no discussion of off-label drug use: Christian Steidl - Bayer: Expert Testimony, Honoraria; Seattle Genetics, Roche, Curis Inc: Consultant, Honoraria; Trillium, Bristol-Myers Squibb: Principal Investigator, Research Grant to my Institution
Relevant financial relationships; discussion of off-label drug use: Catherine Bollard - Caballetta Bio: Board of Directors, Stock Options; Mana Therapeutics: Founder, Stock Options; Torque and Neximmune: Stock Holder, Stock; Cellectis: Advisory Board Member, Honoraria
(C20) Surveillance to Detect Relapse in Pediatric Solid Tumors: Is Less More?
Scheduled surveillance imaging is standard practice for pediatric solid tumors, with the goal of detecting relapse before clinical signs and symptoms develop. With the widespread availability of cross-sectional CT and MRI scans, as well as nuclear imaging studies such as PET, bone, and MIBG scans, the practice of surveillance imaging has shifted to frequent utilization of these modalities. However, it is unclear whether cross-sectional and nuclear imaging leads to improved survival compared to surveillance with x-rays, ultrasounds, or physical exams. This workshop will explore emerging evidence from recent studies in neuroblastoma, Wilms tumor, and bone and soft tissue sarcomas.
Objectives:
- Understand the evidence supporting surveillance imaging in neuroblastoma, Wilms tumor, and bone and soft tissue sarcomas.
- Describe the risks and costs associated with different modalities of surveillance imaging.
- Discuss new developments that may transform the practice of surveillance for relapse.
Presentations:
- Surveillance Imaging for Pediatric Solid Tumors: Is Less More?
Jeffrey S. Dome, MD PhD - Surveillance Imaging for Wilms Tumor and Neuroblastoma
Elizabeth A. Mullen, MD - Surveillance Imaging for Sarcomas
Damon Reed, MD - Surveillance Imaging: The Radiology Perspective
Sue Kaste, DO
No relevant financial relationships and no discussion of off-label drug use: Jeffrey S. Dome, Elizabeth A. Mullen, Sue Kaste
Relevant financial relationships; discussion of off-label drug use: Damon Reed - Janssen, LOXO, Epizyme, Shire: Advisory Board Member, Honoraria
(C21) Predicting Infectious Outcomes and Early Detection Methods in Immunocompromised Patients
Management of febrile pediatric cancer patients is varied and often does not include using quantitative data for decision making. There are now validated risk prediction models for pediatric cancer presenting with both non-neutropenic and neutropenic fever that can aid decision making. There are also new molecular techniques that detect bloodstream DNA fragments that may diagnose infection early. Different practice styles can result in different patient outcomes. Over-treating with antibiotics in patients can select for resistant organisms that are difficult to control; conversely, under-treating puts patients at risk for complications. Familiarity with these tools will help oncologists make improved evidence-based treatment decisions.
Objectives:
- Discuss the creation and implementation of risk prediction models for pediatric cancer patients presenting with both non-neutropenic and neutropenic fever and describe how they are useful in clinical practice.
- Describe the current evidence and potential clinical use of DNA molecular techniques to detect infections prior being isolated in culture.
Presentations:
- Predicting Blood Stream Infections in Pediatric Cancer Patients Presenting with Non-neutropenic Fever to Limit Unnecessary Antibiotic Use
Adam J. Esbenshade, MD MSCI - Predicting Blood Stream Infections/Complications in Pediatric Cancer Patients Presenting with Severe Febrile Neutropenia
Robert Phillips, MBBS - Using DNA Molecular Techniques to Detect Infections Prior to a Positive Blood Culture in Pediatric Cancer Patients
Brian T. Fisher, DO MSCE
No relevant financial relationships and no discussion of off-label drug use: Robert Phillips
No relevant financial relationships; discussion of off-label drug use: Adam J. Esbenshade
Relevant financial relationships; discussion of off-label drug use: Brian Fisher - Pfizer, Merck: Principal Investigator, Research Grant to my Institution; Astellas: Advisory Board Member, DSMB
5:45–6:45 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 year's recipient is Garrett Brodeur, MD, from Children's Hospital of Philadelphia.
6:30–8 pm
Non-CME Corporate Dinner Symposium
Saturday, May 9
7–8 am
Small Program Special Interest Group
Leadership: Jamie Dargart, MD; Courtney Lyle, MD; Andrea Watson, MD.
Providing Palliative Care: Program Optimization at Smaller Centers
At many smaller centers, the pediatric oncology team faces challenges secondary to limited pediatric palliative care resources. Justin Baker, MD will discuss strategies to optimize palliative care at your center. A discussion panel will follow, highlighting practice techniques and resources implemented at several small programs.
Speakers: Justin Baker, MD St Jude Children’s Research Hospital, Katie Harmoney, DO University of Missouri Women and Children’s Hospital, Lisa Hartman, MD El Paso Children’s Hospital, Ginna Priola, MD Mission Health Asheville, NC, Ross Perko, MD Essentia Health Duluth, MN, and Nicole Giamanco, MD Tripler Army Medical Center
7:30–8 am
Diversity SIG Business Meeting
Leadership: Pinki Prasad, MD MPH; Daniel Ortiz, MD; Kerice Pinkney, MD
Hemoglobinopathy SIG Business Meeting
Leadership: Monica Hulbert, MD; Kerry Morrone, MD; Howard Grodman, MD
8:15–9:15 am
Concurrent Paper Sessions
Children's Oncology Group
This session will consist of three-20-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.
Antifungals in AML
Brian Fisher, DO MSCE
Ewing Sarcoma
Damon Reed, MD
Overview of GCT, Risk Stratification, and Current Trials
Farzana Pashankar, MD
No relevant financial relationships and no discussion of off-label drug use: Farzana Pashankar
Relevant financial relationships; no discussion of off-label drug use: Damon Reed - Janssen, LOXO, Epizyme, Shire: Advisory Board Member, Honoraria
Relevant financial relationships; discussion of off-label drug use: Brian Fisher - Pfizer, Merck: Principal investigator, Research grant to my institution, Astellas: Advisory Board Member, DSMB
Hematology Science as presented at the 2019 ASH Annual Meeting
This session will consist of four 15-minute presentations of hematology science - from Hematology Science as presented at the 2019 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.
Single Cell Transcriptome Analysis Reveals Disease-Defining T Cell Subsets in the Tumor Microenvironment of Classic Hodgkin Lymphoma
Tomohiro Aoki, MD PhD
Monitoring the “Lifetime” of a Thrombus over Long Timescales By Leveraging a Novel Microvasculature-on-Chip Thrombosis Resolution Assay
Yongzhi Qiu, PhD
Gene Editing ELANE in Human Hematopoietic Stem and Progenitor Cells Reveals Disease Mechanisms and Therapeutic Strategies for Severe Congenital Neutropenia
Daniel E. Bauer, MD PhD
A Randomized Phase 3 Trial of Blinatumomab vs. Chemotherapy As Post-Reinduction Therapy in High and Intermediate Risk (HR/IR) First Relapse of B-Acute Lymphoblastic Leukemia (B-ALL) in Children and Adolescents/Young Adults (AYAs) Demonstrates Superior Efficacy and Tolerability of Blinatumomab: A Report from Children’s Oncology Group Study AALL1331
Michael A. Pulsipher, MD
No relevant financial relationships and no discussion of off-label drug use: Tomohiro Aoki; Yongzhi Qiu
Relevant financial relationships; no discussion of off-label drug use: Daniel Bauer - Pfizer, Merck: Consultant, Honoraria; Michael A. Pulsipher - Novartis: Steering Committee, Honoraria; Bellicum: Speaker, Honoraria; Miltenyi, Adaptive: Study Support; Novartis: Advisory Board Member, Honoraria
9:30–11 am
Concurrent Sessions
(D22) Pediatric Oncology Supportive Care: Beyond Nausea and Vomiting
Development of new drug classes in pediatric oncology have led to a broader range of side effects being experienced by patients than with traditional chemotherapy. Other therapies such as radiation, have well-described toxicities. However, novel interventions are being developed to mitigate these toxicities. There is also a growing body of evidence detailing the role of modifiable risk factors such as body weight and nutrition on cancer treatment outcomes. It is critical for practicing clinicians to be aware of these unique areas of supportive care in order to appropriately manage, educate and empower patients and their families.
Objectives:
- Identify skin toxicities associated with immunotherapy treatment and discuss their management.
- Describe the common treatment related toxicities associated with radiotherapy and proton beam therapy and emerging agents that may assist in alleviating these toxicities.
- Recognize the role of nutrition and body weight on treatment outcomes for patients with cancer.
Moderator: Allison Grimes, MD
Presentations:
- Nutrition, Integrative Medicine and Symptom Management in Pediatric Oncology
Elena J. Ladas, PhD RD - Skin Deep into Treatment-Related Toxicities
Mario Lacouture, MD - Recognition, Mitigation and Prevention of Neurologic and Neurocognitive Late Effects
Nicole Ullrich, MD PhD
No relevant financial relationships and no discussion of off-label drug use: Allison Grimes, Elena J. Ladas
No relevant financial relationships; discussion of off-label drug use: Nicole Ullrich
Relevant financial relationships; discussion of off-label drug use: Mario Lacouture - Legacy Healthcare Services, Adgero Bio Pharmaceuticals, Amryt Pharmaceuticals, Celldex Therapeutics, Debiopharm, Galderma Research and Development, Johnson and Johnson, Novocure Inc., Lindi, Merck Sharp and Dohme Corporation, Helsinn Healthcare SA, Janssen Research & Development, LLC, Menlo Therapeutics, Novartis Pharmaceuticals Corporation, F. Hoffmann-La Roche AG, Abbvie, Inc., Boehringer Ingelheim Pharma Gmbh & Co. KG, Allergan Inc., Amgen Inc., E.R. Squibb & Sons, L.L.C., EMD Serono, Inc., Astrazeneca Pharmceuticals LP, Genentech, Inc, Leo Pharma, Inc, Seattle Genetics, Bayer, Manner SAS, Lutris, Pierre Fabre, Paxman Coolers, Adjucare, Dignitana, Biotechspert, Teva Mexico, Parexel. OnQuality Pharmaceuticals Ltd, Novartis, Our Brain Bank, Takeda Millenium: Consultant, Honoraria; Veloce, US Biotest, Berg, Bristol-Myers Squibb, Lutris, Paxman, Novocure: Research Funding
(D23) Leukemia Predisposition Syndromes: Who, What, Why and When to Test?
Emerging research indicates that approximately 5% of children with leukemia harbor germline mutations in cancer predisposition genes, including in newly identified leukemia predisposition genes. However, identifying children with leukemia predisposition syndromes and directing their management remains challenging. Furthermore, the underlying pathophysiology of some of these syndromes is only beginning to come to light.
Pediatric oncologists need to be knowledgeable about the presentation and genetics of leukemia predisposition syndromes, the unique challenges related to counseling and testing children for these disorders, and be able to consider how such a diagnosis impacts care of the patient, as well as relatives.
Objectives:
- Identify children who may have a genetic predisposition to leukemia and recognize the challenges associated with counselling and testing patients and families with these disorders.
- Develop clinical management strategies for children with cancer predisposition syndromes.
- Discuss emerging data on the pathophysiology of specific leukemia predisposition syndromes.
Moderator: Christopher C. Porter, MD
Presenters:
- Sarah Bannon, MS
- Kim Nichols, MD
- Sinisa Dovat, MD PhD
No relevant financial relationships and no discussion of off-label drug use: Christopher C. Porter, Sarah Bannon
No relevant financial relationships; discussion of off-label drug use: Sinisa Dovat
Relevant financial relationships; no discussion of off-label drug use: Kim Nichols - Incyte: Principal Investigator, Research Grant to my Institution
(D24) Treatment of Neurofibromatosis Type 1 Related Tumors
There is now a growing number of new options for the treatment of Neurofibromatosis Type 1 (NF1) tumors. Certain optic gliomas can be safely observed, while others will respond to treatment with less toxic BRAF/MEK inhibitors. Previously, plexiform neurofibromas only had surgical treatment options but are now responding to oral MEK inhibitors. Malignant Peripheral Nerve Sheath tumors are still a major source of NF1 mortality, but potential for new therapies are emerging for this tumor type. Many oncologists care for patients with NF1, and familiarity with the newest available therapies will ensure they continue to deliver state of the art care to their patients with NF1.
Objectives:
- Describe the use and preliminary efficacy of using BRAF/MEK inhibitors in the treatment of NF1 optic pathway gliomas.
- Summarize the current knowledge of the natural history of NF1 optic gliomas with advice of when to treat versus observe.
- Describe the use and preliminary efficacy of using MEK inhibitors in the treatment of plexiform neurofibromas.
- Discuss potential new ways to improve the treatment of Malignant Peripheral Nerve Sheath tumors.
Moderator: Adam J. Esbenshade, MD MSCI
Presentations:
- Optic Pathway Gliomas in NF1: Management Challenges, Visual Outcomes, and Potential Biomarkers
Michael J. Fisher, MD - MEK Inhibition in the Treatment of Plexiform Neurofibromas
Brigitte W. Widemann, MD - Malignant Peripheral Nerve Sheath Tumors and How to Identify Better Therapies?
AeRang Kim, MD PhD
No relevant financial relationships and no discussion of off-label drug use: Brigitte W. Widemann
No relevant financial relationships; discussion of off-label drug use: Adam J. Esbenshade, AeRang Kim
Relevant financial relationships; discussion of off-label drug use: Michael J. Fisher - AztraZeneca, SpringWorks: Consultant, No Compensation Received
11:15 am–12:45 pm
Paper Session with Luminary Investigator—Oncology and HSCT/Cellular Therapy
E. Anders Kolb, MD, will present followed by four 15-minute presentations of scientific research from selected oncology and HSCT/cellular therapy related abstracts.
Moderators: TBD
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.
(2024) DISCOVERY OF A NOVEL RAL PATHWAY DISORDER (“RALOPATHY”) WITH EARLY ONSET MYELOPROLIFERATIVE DISEASE
Harry Lesmana, MD
(2025) GUT DECONTAMINATION ALTERS THE INTESTINAL MICROBIOTA DURING ALLOGENIC BONE MARROW TRANSPLANT
Christopher J. Severyn, MD, PhD
(2026) PROSPECTIVE TRIAL OF IBRUTINIB IN CHILDREN WITH CHRONIC GRAFT VERSUS HOST DISEASE
Sonali Chaudhury, MD
(2027) CD30-DIRECTED CAR T-CELLS FOR THE TREATMENT OF PEDIATRIC HODGKIN LYMPHOMA AND NON-HODGKIN LYMPHOMA
George E. Hucks Jr., MD
No relevant financial relationships and no discussion of off-label drug use: E. Anders Kolb, Harry Lesmana, Christopher J. Severyn, George E. Hucks Jr.
Relevant financial relationships; discussion of off-label drug use: Sonali Chaudhury - Novartis: Consultant, honoraria; Bristol-Myers Squibb, Abatacept: Research funding
Paper Session with Luminary Investigator - Basic Science and Clinical Outcomes in Hemostasis, Thrombosis, and Red Cell Pathology
Jorge Di Paola, MD, will present followed by four 15-minute presentations of scientific research from selected hemostasis, thrombosis, and red cell pathology related abstracts.
Moderators: TBD
- 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.
(2028) MIR29B MEDIATES EPIGENETIC MECHANISMS OF HBG GENE ACTIVATION IN SICKLE CELL DISEASE
Ashley M. Fitzgerald, PhD
(2029) OUTCOMES OF RIGHT ATRIAL THROMBI IN CHILDREN; AN OBSERVATIONAL COHORT STUDY
Benjamin T. Barnes, MD
(2030) LONG TERM NEUROLOGICAL OUTCOMES IN PEDIATRIC THROMBOTIC THROMBOCYTOPENIC PURPURA
Sara Graciaa, CPNP
(2031) RESTORING THE SECRETION AND ACTIVITY OF FVIII MUTANTS BY A PROTEOSTASIS REGULATOR
Wei Wei, MD PhD
No relevant financial relationships and no discussion of off-label drug use: Jorge Di Paola, Ashley M. Fitzgerald, Sara Graciaa, Wei Wei
Relevant financial relationships and no discussion of off-label drug use: Benjamin T. Barnes - NIH: Trainee, Educational Grant