Related Experiment Video
Updated: Sep 17, 2026
Assessment of Chimeric Antigen Receptor T Cell-Associated Toxicities Using an Acute Lymphoblastic Leukemia Patient-Derived Xenograft Mouse Model
Published on: February 10, 2023
Incidence Rates of Immune-related Adverse Events and Their Correlation With Response in Hematological Malignancies
Zihao Li1, Wuyuan Tan1, Shaorong Lei2
1Department of Plastic and Cosmetic Surgery, Xiangya Hospital, Central South University, Changsha, China; National Clinical Research Center for Geriatric Diseases, Xiangya Hospital, Central South University, Changsha, China.
Abstract:
Chimeric antigen receptor T (CAR-T) cell therapy provides breakthrough efficacy for relapsed/refractory hematological malignancies, yet the role of cytokine release syndrome (CRS) as an efficacy predictor remains controversial and lacks large-scale validation. This pooled-analysis aimed to systematically evaluate the incidence of CAR-T-related adverse events (TRAEs) and their correlation with therapeutic efficacy. We searched 4 major databases up to December 10, 2025, and included 222 prospective trials enrolling 7538 patients. Pooled analyses were performed using a random‑effects model, and Pearson correlation was used to assess the association between TRAE incidences and key efficacy endpoints. The pooled rates of all‑grade and grade ≥3 CRS were 78.07% and 7.67%, with objective response rate (ORR) 83.26% and complete response rate (CRR) 67.46%. CRS only showed a weak correlation with short‑term remission, while specific TRAEs had moderate‑to‑strong correlations with efficacy (r = .6099 to .9677, P < .05). CAR-T therapy exhibits remarkable efficacy and manageable toxicity in hematological malignancies. CRS is not a reliable efficacy marker; grade ≥3 dyspnea/hypotension/tremor and all‑grade encephalopathy/fatigue are potential early efficacy predictors.
