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Fludarabine- and low-dose cyclophosphamide-based conditioning regimens provided favorable survival and engraftment for unmanipulated hematopoietic cell transplantation from unrelated donors and matched siblings in patients with Fanconi anemia: results from the CBMTR

Bone Marrow Transplantation volume 58, pages 106–108 (2023)Cite this article

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Hematopoietic cell transplantation (HCT) is the only option to cure hematological complications in FA patients. In addition to HCT from a matched sibling donor (MSD), which remains the best strategy, matched unrelated donors (MUD) and mismatched unrelated donors (MMUD) are important alternatives [1].

Graft failure (GF) and severe graft-versus-host disease (GvHD) were two primary obstacles to successful HCT. The addition of fludarabine (Flu) has decreased GF [2], and the use of serotherapy and T-cell depleted grafts has decreased GvHD [3, 4]. Recently, the long-term side effects of HCT prompted the elimination of irradiation [5]. However, experience with unmanipulated HCT for FA is relatively limited in the modern era, which is still of great importance because graft manipulation is unavailable in many countries and regions. Furthermore, the optimal conditioning regimen, specifically for unmanipulated HCT, has not yet been established. We analyzed the results of ninety-eight unmanipulated HCT from unrelated donors and matched siblings based on the Chinese Blood and Marrow Transplant Registry (CBMTR).

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Fig. 1: The probabilities of overall survival (OS) and event-free survival (EFS) in patients with different conditioning regimens.

Data availability

The data analyzed in this study would be available from the corresponding author on reasonable request.

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Acknowledgements

On behalf of the CMBTR Group, we thank all patients who agreed to participate in this study.

Funding

This work was supported by the National Key Research and Development Program of China (No. 2017YFA0104500), Innovative Research Groups of the National Natural Science Foundation of China (No. 81621001), and Key Program of the National Natural Science Foundation of China (No. 81530046).

Author information

Author notes

  1. These authors contributed equally: Lanping Xu, Yue Lu, Jing Chen.

  2. A list of members and their affiliations appears in the Supplementary Information.

Authors and Affiliations

  1. Peking University People’s Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Collaborative Innovation Center of Hematology, Beijing Key Laboratory of Hematopoietic Stem Cell Transplant, Beijing, China

    Lanping Xu, Fan Lin & Xiaojun Huang

  2. Hebei Yanda Lu Daopei Hospital & Beijing Ludaopei Hospital, Langfang & Beijing, China

    Yue Lu & Peihua Lu

  3. Shanghai Children’s Medical Center, Shanghai, China

    Jing Chen & Chengjuan Luo

  4. West China Second University Hospital, Sichuan University/ West China Women’s and Children’s Hospital, Chengdu, China

    Shuwen Sun

  5. Children’s Hospital of Soochow University, Soochow, China

    Shaoyan Hu

  6. Guangzhou First People’s Hospital, Guangzhou, China

    Shunqing Wang

  7. Nanfang Hospital, Southern Medical University, Guangzhou, China

    Xuedong Wu

  8. Beijing Jingdu Children’s Hospital, Beijing, China

    Yuan Sun

  9. The First Affiliated Hospital of Zhengzhou University, Zhengzhou, China

    Dingming Wan

  10. Xiangya Hospital, Central South University, Changsha, China

    Yajing Xu

  11. Shanghai Children’s Hospital, Shanghai, China

    Hui Jiang

  12. Nanfang-Chunfu Children’s Institute of Hematology & Oncology, Taixin Hospital, Dongguan, China

    Chunfu Li

  13. The People’s Hospital of Guangxi Zhuang Autonomous Region, Guangxi Academy of Medical Sciences, Nanning, China

    Mei Lan

  14. Institute of Hematology & Blood Diseases hospital, Chinese Academy of Medical Sciences, Tianjin, China

    Erlie Jiang

  15. The First Affiliated Hospital of Nanchang University, Nanchang, China

    Fei Li

  16. Shenzhen Children’s Hospital, Shenzhen, China

    Sixi Liu

  17. The Children’s Hospital of Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou, China

    Yongmin Tang

  18. Peking-Tsinghua Centre for Life Sciences, School of Life Sciences, Peking University, Beijing, China

    Xiaojun Huang

Authors
  1. Lanping Xu
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  2. Yue Lu
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  17. Yongmin Tang
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  18. Fan Lin
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  19. Peihua Lu
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  20. Chengjuan Luo
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  21. Xiaojun Huang
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Consortia

on behalf of the Chinese Blood and Marrow Transplant Registry Group (CBMTR)

Contributions

LX and XH designed the study. The data analysis and manuscript development were led by LX, YL, and JC. All authors contributed to providing clinical data and approved the final version for submission.

Corresponding author

Correspondence to Xiaojun Huang.

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The authors declare no competing interests.

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Xu, L., Lu, Y., Chen, J. et al. Fludarabine- and low-dose cyclophosphamide-based conditioning regimens provided favorable survival and engraftment for unmanipulated hematopoietic cell transplantation from unrelated donors and matched siblings in patients with Fanconi anemia: results from the CBMTR. Bone Marrow Transplant 58, 106–108 (2023). https://doi.org/10.1038/s41409-022-01838-9

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  • DOI: https://doi.org/10.1038/s41409-022-01838-9

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