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

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

doi:10.1182/bloodadvances.2025017011

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

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

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, …

Blood advances, Vol.9(23), pp.6047-6058

09/12/2025

DOI: https://doi.org/10.1182/bloodadvances.2025017011

PMID: 40829117

Résumé

Hematology

Life Sciences & Biomedicine

Science & Technology

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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Détails

Titre: International consensus guidelines for the conduct and reporting of CAR T-cell clinical trials in AML
Créateurs - sans rôle: Swati Naik - St Jude Childrens Res Hosp, Dept Bone Marrow Transplantat & Cellular Therapy, 262 Danny Thomas Pl, Memphis, TN 38105 USA
Richard Aplenc - Childrens Hosp Philadelphia, Ctr Childhood Canc Res, Div Oncol, Philadelphia, PA USA
Susanne H. C. Baumeister - Dana Farber Canc Inst, Dept Pediat Oncol, Boston, MA USA
Marco Becilli - IRCCS Bambino Gesu Childrens Hosp, Dept Hematol Oncol & Cell & Gene Therapy, Rome, Italy
Anand S. Bhagwat - St Jude Childrens Res Hosp, Dept Bone Marrow Transplantat & Cellular Therapy, 262 Danny Thomas Pl, Memphis, TN 38105 USA
Challice L. Bonifant - Johns Hopkins Univ, Sch Med, Sidney Kimmel Comprehens Canc Ctr, Dept Oncol, Baltimore, MD USA
Lihua E. Budde - Beckman Res Inst, City Hope, Duarte, CA USA
Christopher D. Chien - NCI, NIH, Ctr Canc Res, Pediat Oncol Branch, Bethesda, MD USA
Kevin J. Curran - Mem Sloan Kettering Canc Ctr, Dept Pediat, New York, NY USA
Anthony F. Daniyan - Mem Sloan Kettering Canc Ctr, New York, NY USA
Alexandra Dreyzin - NCI, NIH, Ctr Canc Res, Pediat Oncol Branch, Bethesda, MD USA
Rebecca A. Gardner - St Jude Childrens Res Hosp, Dept Oncol, Memphis, TN USA
Sara Ghorashian - UCL Great Ormond St Inst Child Hlth, London, England
Stephen Gottschalk - St Jude Childrens Res Hosp, Dept Bone Marrow Transplantat & Cellular Therapy, 262 Danny Thomas Pl, Memphis, TN 38105 USA
Laquisa C. Hill - Baylor Coll Med, Ctr Cell & Gene Therapy, Houston, TX USA
M. Eric Kohler - Univ Colorado Anschutz Med Campus, Childrens Hosp Colorado, Dept Pediat & Neurol, Aurora, CO USA
Adam Lamble - Seattle Childrens Hosp, Seattle, WA USA
Franco Locatelli - IRCCS Bambino Gesu Childrens Hosp, Dept Hematol Oncol & Cell & Gene Therapy, Rome, Italy
Maksim Mamonkin - Baylor Coll Med, Ctr Cell & Gene Therapy, Houston, TX USA
Bilal Omer - Baylor Coll Med, Ctr Cell & Gene Therapy, Houston, TX USA
Jae H. Park - Mem Sloan Kettering Canc Ctr, Dept Med, Leukemia Serv, New York, NY USA
Concetta Quintarelli - IRCCS Bambino Gesu Childrens Hosp, Dept Hematol Oncol & Cell & Gene Therapy, Rome, Italy
Benedetta Rambaldi - Azienda Sociosanit Terr Papa Giovanni XXIII, Azienda Socio Sanit Territoriale Papa Giovanni23, Bergamo, Italy
Rebecca M. Richards - Univ Wisconsin Madison, Dept Pediat, Madison, WI USA
David A. Sallman - H Lee Moffitt Canc Ctr & Res Inst, Dept Malignant Hematol, Tampa, FL USA
Tim Sauer - Univ Hosp Heidelberg, Dept Hematol Oncol & Rheumatol, D-69120 Heidelberg, Germany
Nirali N. Shah - NCI, NIH, Ctr Canc Res, Pediat Oncol Branch, Bethesda, MD USA
Marion Subklewe - LMU Gene Ctr, Lab Translat Canc Immunol, Munich, Germany
Corinne Summers - Seattle Childrens Hosp, Seattle, WA USA
Sarah K. Tasian - Childrens Hosp Philadelphia, Ctr Childhood Canc Res, Div Oncol, Philadelphia, PA USA
Naomi Taylor - NCI, NIH, Ctr Canc Res, Pediat Oncol Branch, Bethesda, MD USA
Sarah Tettamanti - Univ Milano Bicocca, Tettamanti Res Ctr, Dept Pediat, Fdn MBBM, Monza, Italy
Michael R. Verneris - Univ Colorado Anschutz Med Campus, Childrens Hosp Colorado, Dept Pediat & Neurol, Aurora, CO USA
M. Paulina Velasquez - St Jude Childrens Res Hosp, Dept Bone Marrow Transplantat & Cellular Therapy, 262 Danny Thomas Pl, Memphis, TN 38105 USA
Saar I. Gill - Univ Penn, Dept Med, Div Hematol Oncol, Philadelphia, PA 19104 USA
Détails de publication: Blood advances, Vol.9(23), pp.6047-6058
Éditeur: Elsevier
Nombre de pages: 12
Note de subvention: American Lebanese Syrian Associated Charities; American Lebanese Syrian Associated Charities (ALSAC) Intramural Research Program of the National Institutes of Health (NIH); United States Department of Health & Human Services; National Institutes of Health (NIH) - USA Center of Excellence for Pediatric Immuno-Oncology at St. Jude Children's Research Hospita
Identifiants: 99153950609311
Unité académique: Institut de Génétique Moléculaire de Montpellier - IGMM
Langue: English
Type de ressource: Journal article