International consensus guidelines for the conduct and reporting of CAR T-cell clinical trials in AML

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.

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Publication Details

Journal
Blood Advances
Published
2025-08-19
DOI
https://doi.org/10.1182/bloodadvances.2025017011
Citations
4
Primary Topic
CAR-T cell therapy research
Type
article
Field-Weighted Citation Impact
4.18

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article

International consensus guidelines for the conduct and reporting of CAR T-cell clinical trials in AML

Naomi Taylor, Michael R. Verneris, Marco Becilli, Franco Locatelli et al.
4 citations
Blood Advances
CAR-T cell therapy research
4.18
article

International consensus guidelines for the conduct and reporting of CAR T-cell clinical trials in AML

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

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.

Blood AdvancesVol. 9(23)
Boston Children's Hospital (US), Seattle Children's Hospital (US), National Institutes of Health (US), City Of Hope National Medical Center (US), St. Jude Children's Research Hospital (US), Memorial Sloan Kettering Cancer Center (US), Children's Hospital of Philadelphia (US), University of Wisconsin–Madison (US), Harvard University (US), Johns Hopkins University (US), Baylor College of Medicine (US), Heidelberg University (DE), Johns Hopkins Medicine (US), Great Ormond Street Hospital (GB), University Hospital Heidelberg (DE), LMU Klinikum (DE), Moffitt Cancer Center (US), Kettering University (US), Fred Hutch Cancer Center (US), Gene Therapy Laboratory (FR), Children's Hospital Colorado (US), Ospedale Papa Giovanni XXIII (IT), Seattle Children's Hospital (US), Methodist Hospital (US), City of Hope (US), Federico II University Hospital (IT), Dana-Farber Cancer Institute (US), Bambino Gesù Children's Hospital (IT), Princess Máxima Center (NL), Methodist Hospital (US), National Cancer Institute (US), Texas Children's Hospital (US), Center for Cancer Research (US), Boston Children's Museum (US), Sidney Kimmel Comprehensive Cancer Center (US), University College London (GB), University of Colorado Anschutz Medical Campus (US), University of Milano-Bicocca (IT), University of Naples Federico II (IT), University of Pennsylvania (US), Ludwig-Maximilians-Universität München (DE), University of Colorado Denver (US)
U.S. Department of Health and Human Services, Amgen, Bristol-Myers Squibb, AstraZeneca, Sanofi, Leukemia and Lymphoma Society, Gilead Sciences, Celgene, Children's Hospital of Philadelphia, St. Jude Children's Research Hospital, Jazz Pharmaceuticals, American Lebanese Syrian Associated Charities, bluebird bio, Incyte, Syndax Pharmaceuticals, Swedish Orphan Biovitrum, National Institutes of Health, National Cancer Institute, NIH Clinical Center
Openalex Percentile: Top 5%
CAR-T cell therapy research
4.18
DOIOpen Access PDFOpenAlex
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