The Effects of Chimeric Antigen Receptor T-Cells for the Treatment of Acute Myeloid Leukemia: A Systematic Review and Meta-Analysis
Author(s)
Bunchai Chongmelaxme, PhD1, Kok Pim Kua, MSc2.
1Social and Administrative Pharmacy, Chulalongkorn University, Bangkok, Thailand, 2Sultan Idris Shah Hospital, Selangor, Malaysia.
1Social and Administrative Pharmacy, Chulalongkorn University, Bangkok, Thailand, 2Sultan Idris Shah Hospital, Selangor, Malaysia.
OBJECTIVES: Although several studies have examined the effects of chimeric antigen receptor (CAR) T-cell therapy, its overall clinical impact remains less clear. This study aims to investigate its clinical effects among patients with acute myeloid leukemia (AML).
METHODS: We conducted a comprehensive search of PubMed, Cochrane CENTRAL, EMBASE, ClinicalTrial.gov, and Scopus from inception to January 31, 2025 to identify studies assessing the efficacy and safety of CAR T-cells in AML. The outcomes evaluated included complete response (CR), partial response (PR), overall response rate (ORR), relapse, overall survival (OS), stable disease (SD), progressive disease (PD), persistence, post-hematopoietic stem cell transplantation (post-HSCT), myelosuppression, cytokine release syndrome (CRS), and immune effector cell-associated neurotoxicity syndrome (ICANS). We performed meta-analyses using a random-effects model.
RESULTS: Twenty-three studies involving patients with relapsed or refractory (R/R) AML were included. Meta-analyses revealed that 68% (0.68 [95% CI: 0.52, 0.84]) of patients achieved a CR to CAR T-cells, while none (0.00 [95% CI: 0.00, 0.08]) showed a PR. The ORR was 73% (0.73 [95% CI: 0.57, 0.86]); however, approximately two-thirds of the patients (41% [0.41 [95% CI: 0.25, 0.58]) experienced relapse, and the OS was 59% (0.59 [95% CI: 0.41, 0.77]). Additionally, 31% (0.31 [95% CI: 0.20, 0.42]) of patients exhibited PD, while 27% (0.27 [95% CI: 0.11, 0.45]) demonstrated SD. CAR T-cell persistence was reported in nearly all patients (97%) (0.97 [95% CI: 0.79, 1.00]), and 40% (0.40 [95% CI: 0.23, 0.58]) received post-HSCT. Myelosuppression was observed in most patients (95%) (0.95 [95% CI: 0.75, 1.00]), and CRS occurred in 91% (0.91 [95% CI: 0.73, 1.00]), while ICANS was detected in 5% (0.05 [95% CI: 0.00, 0.13]).
CONCLUSIONS: While CAR T-cell therapy in R/R AML shows some initial clinical benefits, the occurrence of adverse effects, such as myelosuppression and CRS highlight the limitations and safety concerns of this treatment.
METHODS: We conducted a comprehensive search of PubMed, Cochrane CENTRAL, EMBASE, ClinicalTrial.gov, and Scopus from inception to January 31, 2025 to identify studies assessing the efficacy and safety of CAR T-cells in AML. The outcomes evaluated included complete response (CR), partial response (PR), overall response rate (ORR), relapse, overall survival (OS), stable disease (SD), progressive disease (PD), persistence, post-hematopoietic stem cell transplantation (post-HSCT), myelosuppression, cytokine release syndrome (CRS), and immune effector cell-associated neurotoxicity syndrome (ICANS). We performed meta-analyses using a random-effects model.
RESULTS: Twenty-three studies involving patients with relapsed or refractory (R/R) AML were included. Meta-analyses revealed that 68% (0.68 [95% CI: 0.52, 0.84]) of patients achieved a CR to CAR T-cells, while none (0.00 [95% CI: 0.00, 0.08]) showed a PR. The ORR was 73% (0.73 [95% CI: 0.57, 0.86]); however, approximately two-thirds of the patients (41% [0.41 [95% CI: 0.25, 0.58]) experienced relapse, and the OS was 59% (0.59 [95% CI: 0.41, 0.77]). Additionally, 31% (0.31 [95% CI: 0.20, 0.42]) of patients exhibited PD, while 27% (0.27 [95% CI: 0.11, 0.45]) demonstrated SD. CAR T-cell persistence was reported in nearly all patients (97%) (0.97 [95% CI: 0.79, 1.00]), and 40% (0.40 [95% CI: 0.23, 0.58]) received post-HSCT. Myelosuppression was observed in most patients (95%) (0.95 [95% CI: 0.75, 1.00]), and CRS occurred in 91% (0.91 [95% CI: 0.73, 1.00]), while ICANS was detected in 5% (0.05 [95% CI: 0.00, 0.13]).
CONCLUSIONS: While CAR T-cell therapy in R/R AML shows some initial clinical benefits, the occurrence of adverse effects, such as myelosuppression and CRS highlight the limitations and safety concerns of this treatment.
Conference/Value in Health Info
2025-09, ISPOR Real-World Evidence Summit 2025, Tokyo, Japan
Value in Health Regional, Volume 49S (September 2025)
Code
RWD191
Topic Subcategory
Distributed Data & Research Networks
Disease
SDC: Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)