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Pediatric Transplants

Allogeneic blood SCT for children with Hurler's syndrome: results from the German multicenter approach MPS-HCT 2005

Bone Marrow Transplantation volume 43, pages 375–381 (2009)Cite this article

Abstract

Hurler's syndrome is an inborn error of mucopolysaccharide metabolism leading to premature death in childhood. Allogeneic hematopoietic SCT can achieve long-term survival by correcting the enzymatic deficiency. In an attempt to improve long-term engraftment and to reduce regimen-related toxicity (RRT), a prospective multicenter approach was initiated in Germany using a fludarabine-based radiation-free preparative regimen. Between 2001 and 2008, 12 children were enrolled. Median age at SCT was 14 months (range, 4–31 months). The conditioning regimen contained fludarabine, BU, melphalan and antithymocyte globulin. CD34 positively selected PBSC were used in 10 children with a matched unrelated donor. Median cell dose was 24.6 × 106 CD34+ cells per kg (range 10.0–54.8). Two children with a matched sibling donor received non-manipulated BM. Donor lymphocyte infusions were given in 6/12 children for mixed hematopoietic chimerism. At a median follow-up of 29 months (range 2–85 months), all children engrafted and have either stabilized or improved neurological function. In total, 12/12 patients showed donor-derived engraftment with 9/12 having full and 3/12 having mixed hematopoiesis. One developed acute GVHD ⩾grade II. RRT ⩾grade II was observed in two patients.

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Authors and Affiliations

  1. Department of Pediatric Hematology/Oncology, Hannover Medical School, Hannover, Germany

    M Sauer, B Meissner, A Beilken, K Welte, L Grigull & K W Sykora

  2. Department of Pediatric Hematology and Oncology, Friedrich-Schiller-University Jena, Jena, Germany

    D Fuchs & B Gruhn

  3. Department of Pediatric Hematology and Oncology, University Children's Hospital Hamburg-Eppendorf, Hamburg, Germany

    H Kabisch & R Erttmann

  4. Department of Pediatric Hematology and Oncology, Carl Gustav Carus University, Dresden

    M Suttorp

  5. Germany

    M Suttorp

  6. Department of Pediatric Neurology, Hannover Medical School, Hannover, Germany

    T Luecke

Authors
  1. M Sauer
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  2. B Meissner
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  9. T Luecke
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  10. K Welte
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  11. L Grigull
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Correspondence to M Sauer.

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Sauer, M., Meissner, B., Fuchs, D. et al. Allogeneic blood SCT for children with Hurler's syndrome: results from the German multicenter approach MPS-HCT 2005. Bone Marrow Transplant 43, 375–381 (2009). https://doi.org/10.1038/bmt.2008.328

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  • DOI: https://doi.org/10.1038/bmt.2008.328

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