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Pediatric Transplants

Hematopoietic cell transplantation for Chediak–Higashi syndrome

Bone Marrow Transplantation volume 39, pages 411–415 (2007)Cite this article

Abstract

We reviewed outcomes after allogeneic hematopoietic cell transplantation (HCT) in 35 children with Chediak–Higashi syndrome (CHS). Twenty-two patients had a history of the life-threatening accelerated phase of CHS before HCT and 11 were in accelerated phase at transplantation. Thirteen patients received their allograft from an human leukocyte antigen (HLA)-matched sibling, 10 from an alternative related donor and 12 from an unrelated donor. Eleven recipients of HLA-matched sibling donor, three recipients of alternative related donor and eight recipients of unrelated donor HCT are alive. With a median follow-up of 6.5 years, the 5-year probability of overall survival is 62%. Mortality was highest in those with accelerated phase disease at transplantation and after alternative related donor HCT. Only four of 11 patients with active disease at transplantation are alive. Seven recipients of alternative related donor HCT had active disease at transplantation and this may have influenced the poor outcome in this group. Although numbers are limited, HCT appears to be effective therapy for correcting and preventing hematologic and immunologic complications of CHS, and an unrelated donor may be a suitable alternative for patients without an HLA-matched sibling. Early referral and transplantation in remission after accelerated phase disease may improve disease-free survival.

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Acknowledgements

This study was supported by Public Health Service Grant U24-CA76518-08 from the National Cancer Institute, the National Institute of Allergy and Infectious Diseases, and the National Heart, Lung and Blood Institute.

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Authors and Affiliations

  1. Department of Medicine, Statistical Center, Center for International Blood and Marrow Transplant Registry, Medical College of Wisconsin, Milwaukee, WI, USA

    M Eapen

  2. Division of General and Community Pediatrics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA

    C A DeLaat & A H Filipovich

  3. Department of Pediatrics, University of Minnesota Medical School, Minneapolis, MN, USA

    K S Baker

  4. Department of Pediatrics, Columbia University, New York, NY, USA

    M S Cairo

  5. Pediatric BMT Program, University of California Medical Center, San Francisco, CA, USA

    M J Cowan

  6. Department of Pediatrics, Duke University Medical Center, Durham, NC, USA

    J Kurtzberg

  7. Department of Pediatric Oncology/BMT, Bristol Royal Hospital for Children, Bristol, UK

    C G Steward

  8. BMT Service, Great Ormond Street Hospital for Children NHS Trust, London, UK

    P A Veys

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  1. M Eapen
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  2. C A DeLaat
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  3. K S Baker
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  4. M S Cairo
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  5. M J Cowan
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  6. J Kurtzberg
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  7. C G Steward
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  8. P A Veys
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  9. A H Filipovich
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Correspondence to A H Filipovich.

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Eapen, M., DeLaat, C., Baker, K. et al. Hematopoietic cell transplantation for Chediak–Higashi syndrome. Bone Marrow Transplant 39, 411–415 (2007). https://doi.org/10.1038/sj.bmt.1705600

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  • DOI: https://doi.org/10.1038/sj.bmt.1705600

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