International Consensus Guidelines for the Conduct and Reporting of Car T-Cell Clinical Trials in AML

Authors

Swati Naik
Richard Aplenc
Susanne H C Baumeister
Marco Becilli
Anand S Bhagwat
Challice L Bonifant
Lihua E Budde
Christopher D Chien
Kevin J Curran
Anthony F Daniyan
Alexandra Dreyzin
Rebecca A Gardner
Sara Ghorashian
Stephen Gottschalk
LaQuisa C Hill
M Eric Kohler
Adam Lamble
Franco Locatelli
Maksim Mamonkin
Bilal Omer
Jae H Park
Concetta Quintarelli
Benedetta Rambaldi
Rebecca M Richards
David A Sallman
Tim Sauer
Nirali N Shah
Marion Subklewe
Corinne Summers
Sarah K Tasian
Naomi Taylor
Sarah Tettamanti
Michael R Verneris
M Paulina Velasquez
Saar I Gill

Language

English

Publication Date

12-9-2025

Journal

Blood Advances

DOI

10.1182/bloodadvances.2025017011

PMID

40829117

PMCID

PMC12686697

PubMedCentral® Posted Date

8-21-2025

PubMedCentral® Full Text Version

Post-print

Abstract

Early clinical experience with the use of chimeric antigen receptor (CAR) T-cell therapies for patients with acute myeloid leukemia (AML) has been beset by high rates of toxicities and low rates of response. We convened an international workshop with the goal of bringing investigators in the field of AML-directed CAR T-cell therapy together to facilitate discussion of challenges and to brainstorm potential solutions. Based on discussions at the workshop, it was evident that (1) treating and targeting AML with CAR T cells is associated with unique clinical challenges, and (2) variability in clinical trial design, definitions of toxicities, correlative data collection, and reporting methods hinders the field's ability to compare study outcomes and to develop best practices. Further, details of fundamental CAR T-cell attributes and key correlates of efficacy and toxicity were not uniformly reported in published studies, limiting understanding of barriers to success. These observations led to a concerted team effort in which experts in basic/translational science and clinical investigation from pediatric and adult centers worked together to streamline key attributes of clinical trial design and reporting. Consensus criteria were discussed and agreed upon, leading to the creation of a white paper. These guidelines aim to bolster the overall quality of AML-directed CAR T-cell research, allow for comparisons across trials, and inform the next phase of development of AML-directed CAR T-cell therapies that we hope will improve patient outcomes.

Keywords

Humans, Leukemia, Myeloid, Acute, Immunotherapy, Adoptive, Clinical Trials as Topic, Receptors, Chimeric Antigen, Consensus, T-Lymphocytes, Research Design

Published Open-Access

yes

Recommended Citation

Naik, Swati; Aplenc, Richard; Baumeister, Susanne H C; et al., "International Consensus Guidelines for the Conduct and Reporting of Car T-Cell Clinical Trials in AML" (2025). Faculty and Staff Publications. 5668.
https://digitalcommons.library.tmc.edu/baylor_docs/5668