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Pediatric Transplants

Unrelated donor hematopoietic cell transplantation for hemophagocytic lymphohistiocytosis

Bone Marrow Transplantation volume 42, pages 175–180 (2008)Cite this article

Abstract

We report outcomes after unrelated donor hematopoietic cell transplantation (HCT) for 91 patients with hemophagocytic lymphohistiocytosis (HLH) transplanted in the US in 1989–2005. Fifty-one percent were <1 year at HCT and 29% had Lansky performance scores <90%. Most (80%) were conditioned with BU, CY, and etoposide (VP16) with or without anti-thymocyte globulin. Bone marrow was the predominant graft source. Neutrophil recovery was 91% at day-42. The probabilities of grades 2–4 acute GVHD at day-100 and chronic GVHD at 5 years were 41 and 23%, respectively. The overall mortality rate was higher in patients who did not receive BU/CY/VP16-conditioning regimen (RR 1.95, P=0.035). The 5-year probability of overall survival was 53% in patients who received BU/CY/VP16 compared to 24% in those who received other regimens. In the subset of patients with known disease-specific characteristics, only one of five patients with active disease at HCT is alive. For those in clinical remission at HCT (n=46), the 5-year probability of overall survival was 49%. Early mortality rates after HCT were high, 35% at day-100. These data demonstrate that a BU/CY/VP16-conditioning regimen provides cure in approximately 50% of patients and future studies should explore strategies to lower early mortality.

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Acknowledgements

The CIBMTR is supported by Public Health Service Grant U24-CA76518 from the National Cancer Institute, the National Institute of Allergy and Infectious Diseases, and the National Heart, Lung and Blood Institute; Office of Naval Research; Health Resources and Services Administration (DHHS); and grants from AABB; Aetna; American International Group Inc.; American Society for Blood and Marrow Transplantation; Amgen Inc.; Anonymous donation to the Medical College of Wisconsin; Astellas Pharma US Inc.; Baxter International Inc.; Bayer HealthCare Pharmaceuticals; BioOne Corporation; BloodCenter of Wisconsin; Blue Cross and Blue Shield Association; Bone Marrow Foundation; Bristol-Myers Squibb Company; Cangene Corporation; Celgene Corporation; CellGenix, GmbH; Cerus Corporation; Cubist Pharmaceuticals; Cylex Inc.; CytoTherm; DOR BioPharma Inc.; Dynal Biotech, an Invitrogen Company; EKR Therapeutics; Enzon Pharmaceuticals Inc.; Gambro BCT Inc.; Gamida Cell, Ltd.; Genzyme Corporation; Gift of Life Bone Marrow Foundation; GlaxoSmithKline Inc.; Histogenetics Inc.; HKS Medical Information Systems; Hospira Inc.; Infectious Diseases Society of America; Kiadis Pharma; Kirin Brewery Co., Ltd.; Merck & Company; The Medical College of Wisconsin; MGI Pharma Inc.; Millennium Pharmaceuticals Inc.; Miller Pharmacal Group; Milliman USA Inc.; Miltenyi Biotec Inc.; MultiPlan Inc.; National Marrow Donor Program; Nature Publishing Group; Oncology Nursing Society; Osiris Therapeutics Inc.; Pall Life Sciences; PDL BioPharma Inc; Pfizer Inc; Pharmion Corporation; Roche Laboratories; Schering Plough Corporation; Society for Healthcare Epidemiology of America; StemCyte, Inc.; StemSoft Software, Inc.; SuperGen, Inc.; Sysmex; Teva Pharmaceutical Industries; The Marrow Foundation; THERAKOS Inc.; University of Colorado Cord Blood Bank; ViaCell Inc.; Vidacare Corporation; ViraCor Laboratories; ViroPharma Inc.; and Wellpoint Inc. The views expressed in this article do not reflect the official policy or position of the National Institute of Health, the Department of the Navy, the Department of Defense, or any other agency of the US Government.

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Authors and Affiliations

  1. University of Minnesota Medical Center, Minneapolis, MN, USA

    K S Baker

  2. Cincinnati's Children's Hospital Medical Center, Cincinnati, OH, USA

    A H Filipovich

  3. Columbus Children's Hospital, Columbus, Ohio, USA

    T G Gross

  4. Children's Hospital of Wisconsin, Milwaukee, WI, USA

    W J Grossman

  5. St Jude Children's Research Hospital, Memphis, TN, USA

    G A Hale

  6. St Louis Children's Hospital, St Louis, Missouri, USA

    R J Hayashi

  7. Children's National Medical Center, Washington, DC, USA

    N R Kamani

  8. City of Hope National Medical Center, Duarte, CA, USA

    S Kurian

  9. Children's Hospital of Los Angeles, Los Angeles, CA, USA

    N Kapoor

  10. Karolinska University Hospital, Stockholm, Sweden

    O Ringdén

  11. Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, WI, USA

    M Eapen

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  1. K S Baker
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  2. A H Filipovich
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  3. T G Gross
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  4. W J Grossman
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  5. G A Hale
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  10. O Ringdén
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  11. M Eapen
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Correspondence to M Eapen.

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Baker, K., Filipovich, A., Gross, T. et al. Unrelated donor hematopoietic cell transplantation for hemophagocytic lymphohistiocytosis. Bone Marrow Transplant 42, 175–180 (2008). https://doi.org/10.1038/bmt.2008.133

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