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September 10, 20250 citationsOpen Access

Efficacy and Safety of CAR T-cell Therapy in Relapsed/Refractory B-cell Acute Lymphoblastic Leukemia: A Systematic Review and Meta-analysis

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RKRakhshanda KhanSOSai Manohar Raju ObbaniSTSanjana Thota

Key Points

  • CAR T-cell therapy demonstrates a significant remission rate in relapsed/refractory B-cell ALL.
  • The pooled relapse rate was found to be 0.39, with CD19 and CD22 therapies showing no significant difference.
  • The systematic review included data from 32 studies involving over 155,000 patients and used random effects models.
  • This research highlights the potential of CAR T-cell therapy while emphasizing the need for further refinements in treatment approaches.

Abstract

Introduction: Acute lymphoblastic leukemia (ALL) is the most common childhood cancer, with rising global incidence. The prognosis for patients with relapsed or refractory B-cell ALL (r/r B-ALL) remains poor, necessitating novel therapies. Chimeric Antigen Receptor T-cell (CAR T-cell) therapy has shown promise in treating r/r B-ALL, offering significant improvements in remission rates. Methods: A comprehensive literature search was conducted across PubMed, Embase, and Cochrane Library for studies evaluating CAR T-cell therapy in r/r B-ALL. Randomized controlled trials, cohort, and case-control studies were included, focusing on efficacy and safety outcomes. Data were extracted and pooled using random effects models. The risk of bias was assessed with the New Ottawa Scale. Results: A total of 32 studies were included, involving 1,55,365 patients. The pooled relapse rate was 0.39 (95% CI: 0.29–0.49), with no significant difference between CD19 and CD22 CAR T-cell therapies (p = 0.88). Co-stimulatory agents like 4-1BB showed the most favorable relapse rate of 0.38 (95% CI: 0.27–0.49). The overall Cytokine Release Syndrome (CRS) rate was 0.63 (95% CI: 0.53–0.73), and neurotoxicity occurred at a rate of 0.32 (95% CI: 0.24–0.41). Conclusion: CAR T-cell therapy is effective in treating r/r B-ALL, with high remission rates and manageable adverse events. The choice of co-stimulatory agent and antigen target influences relapse outcomes. Further research is needed to refine CAR T-cell constructs and optimize patient-specific treatments.

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Cite This Study

Khan et al. (2025) studied this question.

synapsesocial.com/papers/68c1824b9b7b07f3a060e8d8https://doi.org/10.1101/2025.08.31.25334789
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