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

L. D. de Hosson, B. P. C. van de Warrenburg, F. W. M. B. Preijers, N. M. A. Blijlevens, B. A. van der Reijden, H. P. H. Kremer, D. J. Lefeber, W. A. Allebes, H. Al-Ali, D. W. Niederwieser, N. P. M. Schaap, A. V. M. B. Schattenberg*

*Bijbehorende auteur voor dit werk

    OnderzoeksoutputAcademicpeer review

    20 Citaten (Scopus)


    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. Bone Marrow Transplantation (2011) 46, 1071-1076; doi: 10.1038/bmt.2010.252; published online 1 November 2010

    Originele taal-2English
    Pagina's (van-tot)1071-1076
    Aantal pagina's6
    TijdschriftBone marrow transplantation
    Nummer van het tijdschrift8
    StatusPublished - aug-2011

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