Abstract
The aim of this study was to evaluate the efficacy, safety, and steroid-sparing effect of tacrolimus in patients with autoimmune cytopenia, including immune thrombocytopenia (ITP), autoimmune hemolytic anemia (AIHA), and Evans syndrome (ES). Patients in the tacrolimus group were treated with tacrolimus in combination with steroids, and the control group received only steroids. Of the 318 patients finally enrolled, 87 (27.4%) were males, with a median age of 45 (14–90) years. The tacrolimus group comprised 144 patients, including 120 ITP, 19 AIHA, and 5 ES patients, and the control group comprised 174 patients, including 141 ITP, 25 AIHA, and 8 ES patients. The optimal ORR of the tacrolimus group was comparable to that of the control group, and the optimal CRR was higher (p < 0.05). Patients receiving tacrolimus had a decreased relapse rate and prolonged relapse-free survival (p < 0.05) compared with the controls for both the whole cohort and the ITP and AIHA subgroups. Compared with the control group, the tacrolimus group had a lower cumulative steroid dosage and earlier discontinuation of steroids (p < 0.05), which resulted in a decreased incidence of steroid-related adverse events (p < 0.05) although the total side effects were similar between the two groups. Similar drug expenses were observed between the tacrolimus and control groups at the 18-month follow-up. In conclusion, the early addition of tacrolimus had a similar ORR, better CRR, lower relapse rate, and prolonged relapse-free survival compared to steroids alone, with reduced steroid-related adverse events.
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Acknowledgements
We thank all patients who consented to disclose their medical records and answered our review calls. Their cooperation and helpful comments throw light upon our data collection and statistical analysis.
Funding
This study was supported by the CAMS innovation fund for medical sciences (CIFMS 2021-I2M-1-003), Peking Union Medical Colleague Hospital Youth Research Foundation (pumch201912367), and National Natural Science Foundation of China (81970106).
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R.Z. and B.H. designed the study and wrote the manuscript. R.Z. and M.C. collected and analyzed the clinical data. C.Y. helped to analyze the date and provide critical suggestions. All authors reviewed the manuscript finally and approved the final submission of the manuscript.
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This study was approved by the ethics committee of Peking Union Medical College Hospital and was conducted in accordance with the Declaration of Helsinki.
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Zhang, R., Chen, M., Yang, C. et al. Efficacy and steroid-sparing effect of tacrolimus in patients with autoimmune cytopenia. Ann Hematol 101, 2421–2431 (2022). https://doi.org/10.1007/s00277-022-04967-9
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DOI: https://doi.org/10.1007/s00277-022-04967-9