Schedule of Events

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

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

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

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

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

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

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

Objectives:

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

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

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

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

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

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

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

Objectives: 

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

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

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

Objectives:

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

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

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

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

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

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

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

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

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

Objectives:

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

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

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

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

Objectives:

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

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

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

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

Surveillance for DICER1-related Conditions
Junne Kamihara, MD PhD

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

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

No CME will be offered for this session.

Objectives:

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

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

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

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

Recommended by the Professional Development Committee

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

Objectives:

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

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

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

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

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

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

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

Objectives:

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

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

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

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

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

Objectives:

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

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

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

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

Diversity in Medicine

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

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

Objectives:

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

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

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

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

Objectives: 

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

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

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

Pediatric Radiosurgery
Erin Murphy, MD

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

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

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

Objectives: 

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

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

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

Objectives:

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

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

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

Pediatric Stroke: A Hematologist's Perspective
Nihal Bakeer, MD

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

Stroke in Special Pediatric Populations
Yaser Diab, MBBS

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

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

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

Objectives:

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

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

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

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

Objectives:

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

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

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

Teens with Haemophilia
Vicky R. Breakey, MD

AYA with Cancer
Jennifer Stinson, PhD RN

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

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

Objectives:

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

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

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

Recommended by the Professional Development Committee.

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

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

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

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

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

Cell and Gene Therapy
Erica Esrick, MD

Pharmaceutical Industry
Kerri Nottage, MD

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

Recommended by the Professional Development Committee.

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

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

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

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

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

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

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

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

Development and Clinical Translation of Engineered Microsystems for Hematologic Applications

Objectives

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

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

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

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

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

Objectives:

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

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

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

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

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

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

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

Objectives:

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

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

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

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

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

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

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

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

Objectives:

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

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

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

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

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

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

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

Objectives: 

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

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

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

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

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

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

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

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