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How I Approach Ethical Considerations for Pediatric Phase I CAR T-cell Trials
  • +4
  • Sarah W. Peters,
  • Sara Silbert,
  • Elizabeth M. Holland,
  • Annette Rid,
  • Rayne H. Rouce,
  • Christine Grady,
  • Nirali Shah
Sarah W. Peters
National Cancer Institute Pediatric Oncology Branch
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Sara Silbert
National Cancer Institute Pediatric Oncology Branch

Corresponding Author:[email protected]

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Elizabeth M. Holland
National Cancer Institute Pediatric Oncology Branch
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Annette Rid
National Institutes of Health Clinical Center Department of Bioethics
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Rayne H. Rouce
Baylor College of Medicine Department of Pediatrics
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Christine Grady
National Institutes of Health Clinical Center Department of Bioethics
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Nirali Shah
National Cancer Institute Pediatric Oncology Branch
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Abstract

Chimeric antigen receptor (CAR) T-cells have shown remarkable promise in children, adolescents and young adults with B-cell malignancies. Such outcomes have shifted expectations for all phase I CAR T-cell trials to be equally successful. The juxtaposition of high expectations, uncertain outcomes, and potential for life-threatening toxicities raises unique ethical challenges. To aid patients and providers considering enrollment on early phase CAR T-cell trials, we discuss: (1) considerations for enrollment of pediatric patients, (2) understanding trial intent and expectations, (3) weighing risks and benefits, (4) considerations for informed consent discussions, (5) post CAR T-cell therapy expectations, and (6) barriers to access.