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Allografting

Adult metachromatic leukodystrophy treated by allo-SCT and a review of the literature

Bone Marrow Transplantation volume 46, pages 1071–1076 (2011)Cite this article

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Abstract

Five patients with adult-onset metachromatic leukodystrophy (MLD) underwent allo-SCT. Conditioning was reduced in intensity and grafts were obtained from voluntary unrelated donors. All but one graft were depleted of T-lymphocytes. Patient age at transplantation varied from 18 to 29 (median, 27) years. Two patients rejected their graft and MLD progressed. The recipient of the unmanipulated graft converted to complete donor chimerism with normalization of arylsulphatase A (ARSA) levels. Despite ARSA normalization, he deteriorated. Another patient was a mixed chimera. Following escalated doses of donor lymphocyte infusions he converted to complete donor chimerism. His levels of ARSA correlated positively with the percentage of donor cells and MLD was not progressive. The fifth patient died after 35 days from complications associated with GVHD. We conclude that results of allo-SCT in symptomatic MLD patients are poor. However, allo-SCT may stop progression of MLD in selected patients.

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Authors and Affiliations

  1. Department of Hematology, Radboud University Nijmegen Medical Centre, Nijmegen, The Netherlands

    L D de Hosson, N M A Blijlevens, N P M Schaap & A V M B Schattenberg

  2. Department of Neurology, Radboud University Nijmegen Medical Centre, Nijmegen, The Netherlands

    B P C van de Warrenburg & H P H Kremer

  3. Laboratory of Hematology, Radboud University Nijmegen Medical Centre, Nijmegen, The Netherlands

    F W M B Preijers & B A van der Reijden

  4. Department of Neurology, University of Groningen Medical Centre, Groningen, The Netherlands

    H P H Kremer

  5. Laboratory of Pediatrics and Neurology, Radboud University Nijmegen Medical Centre, Nijmegen, The Netherlands

    D J Lefeber

  6. Department of Laboratory Medicine, Laboratory of Medical Immunology, Radboud University Nijmegen Medical Centre, Nijmegen, The Netherlands

    W A Allebes

  7. Department of Hematology and Oncology, University of Leipzig, Leipzig, Germany

    H Al-Ali & D W Niederwieser

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  1. L D de Hosson
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Correspondence to A V M B Schattenberg.

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de Hosson, L., van de Warrenburg, B., Preijers, F. et al. Adult metachromatic leukodystrophy treated by allo-SCT and a review of the literature. Bone Marrow Transplant 46, 1071–1076 (2011). https://doi.org/10.1038/bmt.2010.252

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