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Pediatric Hematological Malignancies: CARs for Kids - Live Q&A

Program: Education Program
Monday, December 7, 2020: 11:30 AM-12:15 PM

Description:
The remarkable success of CD19-targeted CAR-T cells for pediatric Pre-B acute lymphoblastic leukemia (B-ALL) has not only changed how practitioners approach relapsed, refractory disease, but also has raised several important, if controversial, questions. For decades, allogeneic hematopoietic stem cell transplant (allo-HSCT) has been the gold standard salvage therapy for pediatric hematological malignancies. With continuing improvements to efficacy and the accumulation of more data on safety, CAR-T cells have emerged as a probable, less toxic replacement for allo-HSCT. This educational session will explore current thinking regarding use of CAR T cells earlier in therapy for patients with poor prognostic features who would historically have been relegated to allo-HSCT in first remission. Importantly, despite their resounding successes in B-ALL, whether CD19 CAR T cells will have similarly striking results in pediatric patients with mature B-cell lymphomas including Burkitt Lymphoma remains an open area of investigation. Beyond CD19, we will discuss current clinical and pre-clinical CAR-T cell strategies targeting an array of other antigens for pediatric patients with lymphoid and myeloid malignancies, including T-lineage malignancies.

Dr. Maude will outline the evidence for use of CD19 CAR T cells as an alternative to allo-HSCT, both in patients with relapsed, refractory Pre-B ALL and in patients with poor-prognostic features who have achieved first remission. She will discuss evidence suggesting CAR T cells are a feasible alternative to allo-HSCT, as well as how patient and clinical characteristics impact clinical decision-making.

Dr. Rouce will discuss the use of CAR T cells for mature B cell and Burkitt lymphoma, reviewing current clinical trials testing different CD19 CAR constructs in this patient population, as well as special challenges in treating these diseases.  She will review efficacy data and discuss broader application and best practices that can be extrapolated from adult data.  

Dr. Gardner will discuss the challenges preventing the rapid translation of CAR T cell strategies for T lineage and myeloid malignancies, as well as strategies to overcome them. She will provide a comprehensive overview of CAR targets for these malignancies, as well as safety concerns for CAR T cell therapies for this pediatric patient population and strategies to overcome them.

Chair:
Rayne H. Rouce, MD, Texas Children's Hospital
Disclosures:
Rouce: Novartis: Honoraria; Tessa Therapeutics: Other, Research Funding.
Panelists:
Shannon L. Maude, MD, PhD, Children's Hospital of Philadelphia , Rayne H. Rouce, MD, Texas Children's Hospital and Rebecca A Gardner, MD, Seattle Children's Hospital
Disclosures:
Maude: Novartis Pharmaceuticals: Consultancy; Kite Pharma: Consultancy. Rouce: Novartis: Honoraria; Tessa Therapeutics: Other, Research Funding. Gardner: Novartis: Honoraria.
The remarkable success of CD19-targeted CAR-T cells for pediatric Pre-B acute lymphoblastic leukemia (B-ALL) has not only changed how practitioners approach relapsed, refractory disease, but also has raised several important, if controversial, questions. For decades, allogeneic hematopoietic stem cell transplant (allo-HSCT) has been the gold standard salvage therapy for pediatric hematological malignancies. With continuing improvements to efficacy and the accumulation of more data on safety, CAR-T cells have emerged as a probable, less toxic replacement for allo-HSCT. This educational session will explore current thinking regarding use of CAR T cells earlier in therapy for patients with poor prognostic features who would historically have been relegated to allo-HSCT in first remission. Importantly, despite their resounding successes in B-ALL, whether CD19 CAR T cells will have similarly striking results in pediatric patients with mature B-cell lymphomas including Burkitt Lymphoma remains an open area of investigation. Beyond CD19, we will discuss current clinical and pre-clinical CAR-T cell strategies targeting an array of other antigens for pediatric patients with lymphoid and myeloid malignancies, including T-lineage malignancies.

Dr. Maude will outline the evidence for use of CD19 CAR T cells as an alternative to allo-HSCT, both in patients with relapsed, refractory Pre-B ALL and in patients with poor-prognostic features who have achieved first remission. She will discuss evidence suggesting CAR T cells are a feasible alternative to allo-HSCT, as well as how patient and clinical characteristics impact clinical decision-making.

Dr. Rouce will discuss the use of CAR T cells for mature B cell and Burkitt lymphoma, reviewing current clinical trials testing different CD19 CAR constructs in this patient population, as well as special challenges in treating these diseases.  She will review efficacy data and discuss broader application and best practices that can be extrapolated from adult data.  

Dr. Gardner will discuss the challenges preventing the rapid translation of CAR T cell strategies for T lineage and myeloid malignancies, as well as strategies to overcome them. She will provide a comprehensive overview of CAR targets for these malignancies, as well as safety concerns for CAR T cell therapies for this pediatric patient population and strategies to overcome them.

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