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Sequential haplo-identical conditioning transplant regimen for pediatric patients with relapsed or refractory hemophagocytic lymphohistiocytosis

Bone Marrow Transplantation volume 59, pages 513–517 (2024)Cite this article

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Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) currently stands as the sole remedy for individuals afflicted with hemophagocytic lymphohistiocytosis (HLH). In this study, we retrospectively evaluated how pediatric patients with relapsed or refractory (R/R) HLH responded to our institution’s cocktail conditioning regimen. The disease was diagnosed according to criteria applicable to patients with familial/genetic, relapsing, or severe/persistent HLH. All donors were HLA haplo-identical family donors. In our cohort, sixty-five patients (P-HLH), including 28 with familial/genetic HLH, 36 with secondary HLH, and 1 with an unknown cause, underwent haplo-identical family donor HSCT. The conditioning regimen consisted of intravenous administration of etoposide (VP-16), busulfan, fludarabine, rabbit anti-human thymocyte globulin (r-ATG), and cyclophosphamide (Cy). Tacrolimus and mycophenolate mofetil were used for graft-versus-host disease (GvHD) prevention. We observed that the median time for neutrophil recovery was 11 days (range, 8–24), and for platelet counts to exceed 20 × 109/L, it was 14 days (range, 7–130). There were 5 patients (7.7%) who experienced grades III to IV acute GvHD, and 6 patients (9.2%) developed extensive chronic GvHD. The estimated 3- and 5-year overall survival rates were 78.1% (95% CI, 65.8–84.6%) and 74.9% (95% CI, 61.2–84.4%), respectively. The estimated 3- and 5-year event-free survival rates were 73.5% (95% CI, 60.8–82.6%) and 70.3% (95% CI, 56.4–80.5%), respectively. Our findings demonstrate that our innovative conditioning regimen is both effective and safe, offering valuable insights for healthcare professionals evaluating the merits of existing therapies.

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Fig. 1: Cocktail conditioning regimens for patients with p-HLH.
Fig. 2: Survival analysis for patients with p-HLH after HSCT.

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Detailed data are available upon request to corresponding author.

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Author notes

  1. These authors contributed equally: Yan Yue, Shifen Fan.

Authors and Affiliations

  1. Department of Hematology and Oncology, Hospital of Beijing Jingdu Pediatrics, Beijing, China

    Yan Yue, Shifen Fan, Zhouyang Liu, Fan Jiang, Jiao Chen & Yuan Sun

  2. Department of Medical Affairs, Acornmed Biotechnology Co., Ltd., Tianjin, China

    Jiayue Qin

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Contributions

YY, SF and YS designed, wrote and revised the manuscript. YY, ZL, YS, FJ, SF collected data and provided clinical care. YY, JC and JQ analyzed the clinical data. All authors approved the final manuscript for publication.

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Correspondence to Yuan Sun.

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Competing interests

Author JQ was employed by Acornmed Biotechnology Co., Ltd. The remaining authors declare that the research was conducted without any commercial or financial relationships that could be considered potential conflicts of interest.

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Informed consent was obtained from the patients/participants included in the study. All procedures conducted in studies involving human participants were in accordance with the ethical standards of the institutional and/or national research committee, and they were conducted following the Helsinki Declaration and its later amendments or comparable ethical standards.

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Yue, Y., Fan, S., Liu, Z. et al. Sequential haplo-identical conditioning transplant regimen for pediatric patients with relapsed or refractory hemophagocytic lymphohistiocytosis. Bone Marrow Transplant 59, 513–517 (2024). https://doi.org/10.1038/s41409-024-02212-7

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