Safety and efficacy of CD33-targeted CAR-NK cell therapy for relapsed/refractory AML: preclinical evaluation and phase I trial
2025

Safety and Efficacy of CD33-Targeted CAR-NK Cell Therapy for Relapsed/Refractory AML

Sample size: 10 publication 10 minutes Evidence: moderate

Author Information

Author(s): Huang Ruihao, Wang Xiaoqi, Yan Hongju, Tan Xu, Ma Yingying, Wang Maihong, Han Xiao, Liu Jia, Gao Li, Gao Lei, Jing Guangjun, Zhang Cheng, Wen Qin, Zhang Xi

Primary Institution: Xinqiao Hospital of Army Medical University

Hypothesis

Can CD33-targeted CAR-NK cell therapy improve outcomes for patients with relapsed/refractory acute myeloid leukemia?

Conclusion

CD33 CAR-NK cells demonstrated primary efficacy and safety for patients with relapsed/refractory AML.

Supporting Evidence

  • Six of ten patients achieved minimal residual disease-negative complete remission by day 28.
  • No grade 3-4 adverse events were observed, except for manageable bone marrow suppression.
  • The median number of prior treatments for patients was five.

Takeaway

This study tested a new treatment for a type of blood cancer using special immune cells that target cancer cells while being safer than previous methods.

Methodology

The study involved a phase I trial with 10 patients receiving CD33 CAR-NK cell infusions after a preconditioning regimen.

Potential Biases

Potential biases may arise from the small sample size and lack of a control group.

Limitations

The study had a small sample size and a short follow-up period.

Participant Demographics

Patients aged 18-65 with relapsed/refractory AML, median age 42.5 years.

Statistical Information

P-Value

p<0.05

Statistical Significance

p<0.05

Digital Object Identifier (DOI)

10.1186/s40164-024-00592-6

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