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Improved transplant outcomes with myeloablative conditioning for hemophagocytic lymphohistiocytosis in HLA-matched and mismatched donors: a national multicenter retrospective study

Bone Marrow Transplantation volume 56, pages 2088–2096 (2021)Cite this article

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Abstract

We report the results of national retrospective study of 45 children with hemophagocytic lymphohistiocytosis (HLH) who underwent allogeneic hematopoietic stem-cell transplantation (HSCT) in Israel between the years 2000–2018. Donors were either HLA-matched (n = 26), partially mismatched (n = 7), haploidentical (n = 8), or cord-blood (n = 4). Myeloablative conditioning (MAC) was used in 20 procedures, and reduced-intensity conditioning (RIC) in 25. Forty-two patients engrafted, two had primary graft failure (one successfully retransplanted), one died prior to engraftment, and two developed secondary graft failure. Of the eight patients who had mixed donor chimerism at day 30 (5–95%), five achieved stable mixed or full donor chimerism. The 5-year probabilities of overall survival and event-free survival (EFS) were 86% and 82%, respectively. Five-year EFS was lower for patients receiving RIC compared to MAC (72% vs. 100%, p = 0.018) and following alternative-donor transplant (68% vs. 92% for HLA-matched donors, p = 0.034), mostly due to increased transplant-related mortality (TRM). Thus, both HLA-matched and alternative donor transplant procedures may benefit form a myeloablative conditioning regimen.

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Fig. 1: Engraftment following HSCT for patients with HLH.
Fig. 2: Outcome of patients transplanted for HLH.

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Author information

Author notes

  1. These authors contributed equally: Yarden Greental Ness, Amir A Kuperman

  2. These authors jointly supervised this work: Bella Bielorai, Elad Jacoby

Authors and Affiliations

  1. Azrieli Faculty of Medicine, Bar Ilan University, Safed, Israel

    Yarden Greental Ness & Amir A. Kuperman

  2. Blood Coagulation Service and Pediatric Hematology Clinic, Galilee Medical Center, Nahariya, Israel

    Amir A. Kuperman

  3. Hematology-Oncology Division, Schneider Children’s Medical Center of Israel, Petach Tikvah, Israel

    Jerry Stein & Joanne Yacobovich

  4. Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel

    Jerry Stein, Joanne Yacobovich, Amos Toren, Bella Bielorai & Elad Jacoby

  5. Faculty of Medicine, Hebrew University of Jerusalem, Israel, Department of Bone Marrow Transplantation and Cancer Immunotherapy, Hadassah Medical Center, Jerusalem, Israel

    Ehud Even-Or, Irina Zaidman & Polina Stepensky

  6. Division of Pediatric Hematology-Oncology, Ruth Rappaport Children’s Hospital, Rambam Medical Center, Haifa, Israel

    Aharon Gefen & Neta Nevo

  7. Biostatistics & Biomathematics, The Gertner Institute for Epidemiology and Health Policy Research, Tel Aviv, Israel

    Bernice Oberman

  8. Division of Pediatric Hematology, Oncology and BMT, The Edmond and Lily Safra Children’s Hospital, Sheba Medical Center, Ramat Gan, Israel

    Amos Toren, Bella Bielorai & Elad Jacoby

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Greental Ness, Y., Kuperman, A.A., Stein, J. et al. Improved transplant outcomes with myeloablative conditioning for hemophagocytic lymphohistiocytosis in HLA-matched and mismatched donors: a national multicenter retrospective study. Bone Marrow Transplant 56, 2088–2096 (2021). https://doi.org/10.1038/s41409-021-01290-1

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