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. 2025 Aug;72(8):e31802.
doi: 10.1002/pbc.31802. Epub 2025 May 28.

How I Approach Ethical Considerations for Pediatric Phase I CAR T-Cell Trials

Sarah W Peters  1 , Sara K Silbert  1 , Elizabeth M Holland  1 , Annette Rid  2 , Rayne H Rouce  3 , Christine Grady  2 , Nirali N Shah  1
Affiliations

How I Approach Ethical Considerations for Pediatric Phase I CAR T-Cell Trials

Sarah W Peters et al. Pediatr Blood Cancer. 2025 Aug.

Abstract

Phase I trials of chimeric antigen receptor (CAR) T cells have shown remarkable promise in pediatric B-cell malignancies. Such outcomes have shifted expectations for other phase I CAR T-cell trials to have high response rates. The juxtaposition of high expectations, uncertain outcomes, and potential for life-threatening toxicities raises unique ethical considerations. To aid patients and providers considering early-phase CAR T-cell trials, we discuss: (i) considerations for enrollment of pediatric patients, (ii) understanding trial intent and expectations, (iii) weighing risks and benefits, (iv) considerations for informed consent discussions, (v) post CAR T-cell therapy expectations, (vi) barriers to access, and (vii) vulnerability in decision making.

Keywords: chimeric antigen receptor T cells; immunotherapy; pediatrics; phase I trials; relapse.

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References

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