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Comparison of haploidentical–allogeneic hematopoietic stem cell transplantation and intensive immunosuppressive therapy for patients with severe aplastic anemia with an absolute neutrophil count of zero: a retrospective study

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Abstract

A retrospective analysis was conducted based on the clinical data from 60 patients older than 16 years from January 2016 to January 2021. All the patients were newly diagnosed with severe aplastic anemia (SAA) with an absolute neutrophil count (ANC) of zero. We compared the hematological response and survival of haploidentical–allogeneic hematopoietic stem cell transplantation (HID-HSCT) (n = 25) and intensive immunosuppressive therapy (IST) (n = 35) treatments. At six months, the overall response rate and complete response were significantly higher in the HID-HSCT group than those in the IST group (84.0% vs. 40.0%, P = 0.001; 80.0% vs. 17.1%, P = 0.001). With a median follow-up of 18.5 months (4.3~30.8 months), patients in the HID-HSCT group had longer overall survival and event-free survival (80.0% vs. 47.9%, P = 0.0419; 79.2% vs. 33.5%, P = 0.0048). These data suggested that HID-HSCT might be an effective alternative treatment option for adult patients with SAA with an ANC of zero, which requires further validation in an additional prospective study.

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Data availability

The datasets used and/or analyzed during the current study are available from the corresponding author upon reasonable request.

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Author information

Author notes

  1. Li-qiang Wu and Li-fang Huang contributed equally to this work.

Authors and Affiliations

  1. Department of Hematology, The First Affiliated Hospital of Zhejiang Chinese Medical University (Zhejiang Provincial Hospital of Chinese Medicine), Hangzhou, China

    Li-qiang Wu, Bao-dong Ye, Jian-ping Sheng, Qing-hong Yu & Sheng-yun Lin

  2. Department of Hematology, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China

    Li-fang Huang & Dong-hua Zhang

  3. Department of Hematology, The First Affiliated Hospital of Nanjing Medical University, Jiangsu Province Hospital, Collaborative Innovation Center for Cancer Personalized Medicine, Nanjing, China

    Hui Yang, Guang-sheng He & Jian-yong Li

  4. Department of Hematology, The First Hospital of Jilin University, Changchun, China

    Yan Yang

  5. Peking University People’s Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Research Unit of Key Technique for Diagnosis and Treatments of Hematologic Malignancies, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China

    Jin-song Jia

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Contributions

S.L. and G.H. conceived and designed the analysis. H.Y, B.Y, J.S., and Q.Y. recruited patients. Y.Y., D.Z., J.J., and J.L. collected and analyzed the data. L.W. and L.H. wrote the original draft.

Corresponding authors

Correspondence to Sheng-yun Lin or Guang-sheng He.

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Ethics approval and consent to participate

Informed consent was obtained from patients. Our study complies with the Declaration of Helsinki and was approved by the hospital ethics committee (ChiCTR2100045895).

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The authors declare no competing interests.

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Wu, Lq., Huang, Lf., Yang, H. et al. Comparison of haploidentical–allogeneic hematopoietic stem cell transplantation and intensive immunosuppressive therapy for patients with severe aplastic anemia with an absolute neutrophil count of zero: a retrospective study. Ann Hematol 102, 2015–2023 (2023). https://doi.org/10.1007/s00277-023-05256-9

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