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A New Immunosuppressive Therapy for Very Severe Aplastic Anemia in Children with Autoantibodies

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Abstract

Objective

At present, a number of very severe aplastic anemia (VSAA) patients cannot receive hematopoietic stem cell transplantation (HSCT) or standard immunosuppressive therapy (IST) due to the high cost of therapy, shortage of sibling donors, and lack of resources to support the HSCT. In addition, some VSAA patients with autoantibodies have no life-threatening infections or bleeding at the time of initial diagnosis. Considering the disease condition, economics and other factors, the present study designed a new and relatively mild treatment strategy: cyclosporine A plus pulsed high-dose prednisone (CsA+HDP).

Methods

The present study retrospectively analyzed 11 VSAA patients, who were treated with CsA+HDP in our hospital from August 2017 to August 2019.

Results

The median follow-up time for these patients was 24.9 months. The overall response rate was 54.5% (6/11) at six months after the initiation of IST and 81.8% (9/11) at deadline. Five patients achieved complete remission and four patients met the criteria for partial response at the last follow-up. The median time to response for responders was 110 days. Three patients underwent HSCT due to the poor effect of CsA+HDP or to find a suitable transplant donor. Recurrence and clonal evolution were not found in any of these patients. The estimated 3-year overall survival rate and 3-year failure-free survival rate were 100.0% and 72.7%, respectively. In addition, the results revealed that the cyclosporine-prednisone-associated toxicity was mild and well-tolerated by most patients.

Conclusion

The novel CsA+HDP regimen has good therapeutic effect and safety for VSAA patients with autoantibodies, who have no serious life-threatening infections or bleeding at the time of initial diagnosis.

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Authors and Affiliations

  1. Department of Pediatrics, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, 430022, China

    Zhong-jian Wang, Hong-bo Chen, Fen Zhou, Hui Yu, Xiao-yan Wu, Ya-qing Shen, Yi-ning Qiu & Run-ming Jin

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  1. Zhong-jian Wang
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  2. Hong-bo Chen
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  3. Fen Zhou
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Corresponding authors

Correspondence to Yi-ning Qiu or Run-ming Jin.

Ethics declarations

Author Run-ming JIN is a member of the Editorial Board for [Current Medical Science]. The paper was handled by the other editor and has undergone rigorous peer review process. Author Run-ming JIN was not involved in the journal’s review of, or decisions related to, this manuscript.

Additional information

This work was supported by a grant from the National Natural Science Foundation of China (No. 21906061).

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Wang, Zj., Chen, Hb., Zhou, F. et al. A New Immunosuppressive Therapy for Very Severe Aplastic Anemia in Children with Autoantibodies. CURR MED SCI 42, 379–386 (2022). https://doi.org/10.1007/s11596-022-2519-2

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  • DOI: https://doi.org/10.1007/s11596-022-2519-2

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