Objectives:

1. Identify current paradigms in genomic classification for AML and potential therapeutic implications of such classification.
2. Recognize the importance of and methods to detect minimal residual AML in patients after therapy.
3. Discuss emerging therapeutic approaches for high-risk AML including molecularly targeted and immune-based therapies.

Recent advances in risk stratification for AML based on genomics and the presence of minimal residual disease after therapy allow for the identification of patients with a very high likelihood of relapse after standard therapy. Allogeneic hematopoietic stem cell transplantation has traditionally been used after initial chemotherapy in high risk AML. This session will describe current genomic classification for AML and methods to measure minimal residual disease. The potentially changing role for HSCT in the modern AML risk-classification era will also be presented. Finally, the potential for immunotherapeutic approaches to improve outcomes for such high risk patients will be discussed.

No relevant financial relationships to disclose and no discussion of off-label drug use: Terry Fry; Soheil Meshinchi; Sarah K. Tasian
Relevant financial relationships to disclose; discussion of off-label drug use: Todd E. Druley - Canopy Biosciences: Licensee, Other Financial Benefit

Objectives: 

1. Identify the biology and mechanisms of graft-versus-host disease (GvHD).
2. Examine the biological differences of GvHD in children compared to adults.
3. Recognize all of the available and future forms of treatment of GvHD.

Graft-versus-host disease (GvHD) remains a major source of mortality and late effects in children and adolescent receiving a hematopoietic cell transplantation (HCT). Recently, studies have helped to better understand the biology, biomarkers, and treatment of GvHD. While we have known that the frequency of GvHD is lower and different in the pediatric population compared to the adults, only recently have we gained an understanding of the different mechanisms. This session will give participants state-of-the-art knowledge in pediatric GvHD as well as the most recent therapeutic advances. This improved understanding will lead to more personalized and targeted therapies for pediatric GVHD.

No relevant relationships and no discussion of off-label drug use: Kirk R. Schultz 

No relevant relationships; discussion of off-label drug use: Jennifer Whangbo

Relevant relationships; discussion of off-label drug use: Leslie Kean- Bristol Myers Squibb: Consultant, Honoraria; Magenta: Advisory Board Member, No Compensation Received; Regeneron, Kymab, Bristol Myers Squibb, Jazz: Principal Investigator, Research Grant to my Institution

No CME provided for this session.  

Relevant relationships and no discussion of off-label drug use: John Levine-Viracor: Intellectual Property/Patent, Royalties

Adoptive T-Cell Therapy for Acute Lymphoblastic Leukemia Targeting Multiple Tumor Associated Antigens
Swati Naik, MD

N-803 (IL-15 Superagonist) And Dinutuximab Significantly Enhance Expanded Natural Killer Cell Activity against GD2+ Pediatric Solid Tumors (ST)
Yaya Chu, PhD

Hexaviral Specific T-Cells Targeting Hpiv3, Cmv, Ebv, Adv, Hhv6 And Bkv After Stem Cell Transplant
Katherine Harris, MD

No relevant financial relationships and no discussion of off-label drug use: Swati Naik; Yaya Chu; Katherine Harris

Objectives: 

1. List current outcomes following cord blood and haploidentical stem cell transplants for pediatric malignancies.
2. Recall advantages and disadvantages of each transplant modality.
3. Examine the new areas of discovery with each transplant method as efforts continue to improve upon past experience.

This session will examine umbilical cord blood and haploidentical stem cell transplantation in pediatric malignancies. Each stem cell source has risks and benefits. Umbilical cord blood has the advantage of providing adequate cells in pediatric donors, is easily accessible, can now be expanded, and does not need to be HLA-identical for good outcomes. Cost, graft rejection risks, and delayed immune reconstitution are barriers. Haploidentical donors are easily available, economically viable, and transplant success with immune manipulation is promising. Transplant methods and technologic advances will be reviewed for each modality.

No relevant relationships and no discussion of off-label drug use: Alice Bertaina; Shalini Shenoy
Relevant relationships and no discussion of off-label drug use: Vanderson Rocha- Hematology Department of Medical Clinical Division I of ICHC: Honorarium, speaker

Transplant Associated Thrombotic Microangiopathy after Transplant: A PBMTC/PALISI Multi-Center Collaborative
Christopher Dandoy, MD

Blinatumomab Reduces Minimal Residual Disease Prior To Allogeneic Stem Cell Transplant for Children with Acute Lymphoblastic Leukemia
Amy Keating, MD

Immunotoxins Target Stem Cells in Fanconi Anemia to Avoid Leukemogenesis and Facilitate Engraftment
Meera Srikanthan, MD

No relevant financial relationships and no discussion of off-label drug use: Christopher Dandoy; Amy Keating; Meera Srikanthan

Objectives:

1. Describe the efficacy of using a video (digital format) for discharge teaching to parents/caregivers.
2. Report study results if using a discharge teaching video together with standard 1 to 1 verbal teaching increased parent knowledge.
3. Describe how learning style of parent/caregivers affects the comprehension of discharge teaching provided.

For parents/caregivers, the responsibility of care for a child following HSCT in the home setting is challenging. Effective discharge education and ensuring the parents/caregivers are ready for the complex care is essential to successfully transitioning to the home. Unfortunately, there is limited research that has tested the various discharge education modes in the pediatric HSCT setting. This study evaluated the feasibility and effectiveness of a discharge video intervention used in conjunction with standard discharge teaching.

No relevant relationships and no discussion of off-label drug use: Gail Covington

Objectives:

1. Describe the molecular basis of Biphenotypic Leukemia.
2. Assess how targeted therapies can be successful in curing the disorder.
3. Discuss current therapeutic options for Biphenotypic Leukemia.

This session will focus on the unique entity of Biphenotypic leukemia. After a case presentation, we will review the pathophysiology of this disorder and how targeted therapies can be developed and directed for future therapies. We will also discuss how current treatment strategies can be effective for this disease. Current therapeutic options will be discussed and future directions in therapy will be summarized.

No relevant relationships and no discussion of off-label drug use: Thomas Alexander; Etan Orgel
Relevant relationships and no discussion of off-label drug use: Michael Nieder - Jazz Pharmaceuticals: Consultant and Speaker's bureau, honorarium

Reduced Intensity Conditioning For Pediatric Therapy-Related Myelodysplastic Syndrome/Acute Myeloid Leukemia Is Associated With Reduced Non-Relapse
Akshay Sharma, MD

Regulation Of Cytokines/Chemokines Release And Anti-Tumor Effect Of Expanded Natural Killer (NK) Cells By A Novel Fusion Of N-820 (2B8T2M), AN IL-15 Superagonist With 4 Single-Chain Anti-CD20 Antibody Domains, Against Rituximab Resistant Burkitt Lymphoma (BL)
Yaya Chu, PhD

No relevant financial relationships and no discussion of off-label drug use: Yaya Chu; Akshay Sharma

Objectives:

1. Describe a general overview of Targeted Immunotherapy.
2. Describe a general overview of CART Therapy.
3. Compare CART products available.
4. Recognize CART Therapy patients side effects.

Chimeric antigen receptor (CAR) T-cell therapy is an immunotherapy in which the patient’s own T cells or donor T Cells are isolated in the lab, redirected with a synthetic receptor to recognize a particular antigen or protein, and re-infused into the patient. These cells leverage the patient’s immune system to target, reduce and/or eliminate malignant disease. This therapy has been extensively studied and utilized in children with refractory or relapsed ALL in goals to induce remission or minimal residual disease and/or in preparation for HSCT. This therapy may have varied side effects based on the patient’s various immune responses for which there are specific patient care interventions recommended.

No relevant relationships and no discussion of off-label drug use: Rita Secola

Objectives:

1. Recognize the distinct biology of hypodiploid ALL, allowing them to decide how best to treat this disease using chemotherapy, transplant and CAR T-cell approaches.
2. Define TKI-resistant/intolerant CML and decide when third generation TKIs should be used or HCT considered.
3. Articulate the role and outcomes of checkpoint inhibitors, auto/allo HCT, and innovative immune/cell therapy for treating primary refractory or relapsed/refractory Hodgkin Disease.

With targeted molecules, immune/cell therapeutics, and check point inhibitors now being used routinely in pediatric diseases, we have tools that allow us to manage challenging malignancies. But sorting out when and how best to employ these tools is challenging. This session will discuss three difficult to treat disease situations—hypodiploid ALL, TKI resistant CML, and refractory Hodgkin Disease. Presenters will discuss the latest genetic and biological understanding of the diseases followed by a review of how and when to employ targeted, immune/cell, and transplantation therapies therapies.

No relevant relationships and no discussion of off-label drug use: Jennifer L. McNeer; Sonali Chaudhury
No relevant relationships; discussion of off-label drug use: Kirsten M. Williams
Relevant relationships; discussion of off-label drug use: Michael A. Pulsipher - Novartis: Consulting, Study Steering Committee, and Speaker, honorarium; Adaptive: Consultant and study support, honorarium