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HLA-matched HSCT using targeted busulfan-based conditioning in children with primary hemophagocytic lymphohistiocytosis

Bone Marrow Transplantation volume 56, pages 3097–3099 (2021)Cite this article

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To the Editor:

Hematopoietic stem cell transplantation (HSCT) cures primary hemophagocytic lymphohistiocytosis (HLH). HSCT using reduced-intensity conditioning (RIC) in HLH is associated with low transplant-related mortality; however, there is an increased risk of graft failure. A successful RIC regimen has to lead to a satisfactory and durable donor engraftment. A recent paper by Felber et al. reported an excellent outcome in 25 primary HLH patients who underwent RIC HSCT from HLA-matched donors using targeted busulfan (45–65 mg/L X h), fludarabine, and serotherapy. There was stable donor chimerism, no graft rejection, low risk of GVHD, and no mortality among all patients [1]. We would like to report our successful experience using similar targeted busulfan-based RIC HSCT from HLA-matched donors in primary HLH including patients with mutated PRF1, STX11, RAB27A, STXBP2, LYST, and UNC13D. Felber et al. study did not include patients with PRF1, STX11, or RAB27A pathogenic mutations [1].

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References

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Authors and Affiliations

  1. Department of Pediatric Hematology/Oncology, King Abdullah Specialist Children’s Hospital, Ministry of National Guard Health Affairs, Riyadh, Saudi Arabia

    Mohammed F. Essa, Enas Elbashir, Reem Alsudairy & Ali Alomari

  2. College of Medicine, King Saud bin Abdulaziz University for Health Sciences, Riyadh, Saudi Arabia

    Mohammed F. Essa

  3. King Abdullah International Medical Research Center, National Guard Health Affairs, Riyadh, Saudi Arabia

    Mohammed F. Essa

  4. Department of Nursing, King Abdullah Specialist Children’s Hospital, Ministry of National Guard Health Affairs, Riyadh, Saudi Arabia

    Rodina Abujoub

  5. Department of Pediatrics, College of Medicine, King Saud University, Riyadh, Saudi Arabia

    Abdulrahman Alsultan

  6. Oncology Center, King Saud University Medical City, Riyadh, Saudi Arabia

    Abdulrahman Alsultan

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  6. Abdulrahman Alsultan
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Contributions

All authors have contributed significantly to the submitted work and approve all aspects of the letter. Mohammed Essa: Data collection, data analysis, and manuscript writing. Rodina Abujoub: Data collection and manuscript review. Enas Elbashir: Data interpretation and manuscript review. Reem Alsudairy: Data interpretation and manuscript review. Ali Alomari: Data interpretation and manuscript review. Abdulrahman Alsultan: Data analysis, manuscript writing, and review.

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Correspondence to Mohammed F. Essa.

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Essa, M.F., Abujoub, R., Elbashir, E. et al. HLA-matched HSCT using targeted busulfan-based conditioning in children with primary hemophagocytic lymphohistiocytosis. Bone Marrow Transplant 56, 3097–3099 (2021). https://doi.org/10.1038/s41409-021-01468-7

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