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

doi:10.1038/bmt.2010.252

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^*

^*Corresponding author for this work

Research output: Contribution to journal › Review article › peer-review

31 Citations (Scopus)

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

Original language	English
Pages (from-to)	1071-1076
Number of pages	6
Journal	Bone marrow transplantation
Volume	46
Issue number	8
DOIs	https://doi.org/10.1038/bmt.2010.252
Publication status	Published - Aug-2011

Keywords

metachromatic leukodystrophy
allo-SCT
MLD
SCT
review
BONE-MARROW-TRANSPLANTATION
HEMATOPOIETIC-CELL TRANSPLANTATION
HURLER-SYNDROME
NERVE-CONDUCTION
GRAFT FAILURE
OUTCOMES
DISEASE
ARYLSULFATASE
HETEROZYGOTES
RELIABILITY

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de Hosson, L. D., van de Warrenburg, B. P. C., Preijers, F. W. M. B., Blijlevens, N. M. A., van der Reijden, B. A., Kremer, H. P. H., Lefeber, D. J., Allebes, W. A., Al-Ali, H., Niederwieser, D. W., Schaap, N. P. M., & Schattenberg, A. V. M. B. (2011). Adult metachromatic leukodystrophy treated by allo-SCT and a review of the literature. Bone marrow transplantation, 46(8), 1071-1076. https://doi.org/10.1038/bmt.2010.252

@article{7c8c2541db4f4b4cb683d0c8001e0733,

title = "Adult metachromatic leukodystrophy treated by allo-SCT and a review of the literature",

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

keywords = "metachromatic leukodystrophy, allo-SCT, MLD, SCT, review, BONE-MARROW-TRANSPLANTATION, HEMATOPOIETIC-CELL TRANSPLANTATION, HURLER-SYNDROME, NERVE-CONDUCTION, GRAFT FAILURE, OUTCOMES, DISEASE, ARYLSULFATASE, HETEROZYGOTES, RELIABILITY",

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

year = "2011",

month = aug,

doi = "10.1038/bmt.2010.252",

language = "English",

volume = "46",

pages = "1071--1076",

journal = "Bone marrow transplantation",

issn = "0268-3369",

publisher = "Nature Publishing Group",

number = "8",

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de Hosson, LD, van de Warrenburg, BPC, Preijers, FWMB, Blijlevens, NMA, van der Reijden, BA, Kremer, HPH, Lefeber, DJ, Allebes, WA, Al-Ali, H, Niederwieser, DW, Schaap, NPM & Schattenberg, AVMB 2011, 'Adult metachromatic leukodystrophy treated by allo-SCT and a review of the literature', Bone marrow transplantation, vol. 46, no. 8, pp. 1071-1076. https://doi.org/10.1038/bmt.2010.252

TY - JOUR

T1 - Adult metachromatic leukodystrophy treated by allo-SCT and a review of the literature

AU - de Hosson, L. D.

AU - van de Warrenburg, B. P. C.

AU - Preijers, F. W. M. B.

AU - Blijlevens, N. M. A.

AU - van der Reijden, B. A.

AU - Kremer, H. P. H.

AU - Lefeber, D. J.

AU - Allebes, W. A.

AU - Al-Ali, H.

AU - Niederwieser, D. W.

AU - Schaap, N. P. M.

AU - Schattenberg, A. V. M. B.

PY - 2011/8

Y1 - 2011/8

N2 - 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

AB - 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

KW - metachromatic leukodystrophy

KW - allo-SCT

KW - MLD

KW - SCT

KW - review

KW - BONE-MARROW-TRANSPLANTATION

KW - HEMATOPOIETIC-CELL TRANSPLANTATION

KW - HURLER-SYNDROME

KW - NERVE-CONDUCTION

KW - GRAFT FAILURE

KW - OUTCOMES

KW - DISEASE

KW - ARYLSULFATASE

KW - HETEROZYGOTES

KW - RELIABILITY

U2 - 10.1038/bmt.2010.252

DO - 10.1038/bmt.2010.252

M3 - Review article

SN - 0268-3369

VL - 46

SP - 1071

EP - 1076

JO - Bone marrow transplantation

JF - Bone marrow transplantation

IS - 8

ER -

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

Abstract

Keywords

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