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Reduced intensity conditioning haematopoietic stem cell transplantation with mesenchymal stromal cells infusion for the treatment of metachromatic leukodystrophy: a case report

¹ Department of Clinical and Experimental Hematology, Institut Jules Bordet, Université Libre de Bruxelles (ULB)
² Department of Neurology ,Centre Hospitalier Molière Longchamp, Brussels, Belgium

Correspondence: Dr. N. Meuleman, Department of Hematology, Institut Jules Bordet, 1 rue Heger Bordet, 1000 Bruxelles, Belgium. Phone: +32.2.541-32-37. Fax: +32.2.544-02-57. E-mail: nathalie.meuleman{at}bordet.be

We report the case of a 23-year-old woman who presented with an adult form of metachromatic leukodystrophy (MLD) evolving over one year with a progressive neurological deterioration. A non-myeloablative matched related haematopoietic stem cell transplantation (HSCT) with concomitant mesenchymal stromal cells (MSCs) infusion was performed. Engraftment occurred rapidly with no significant toxicity or side effects following the MSC infusion. At a follow up of 40 months, the patient had a stabilisation of all neurological manifestations of her disease. This case report suggests the feasibility and the potential efficacy of reduced intensity conditioning (RIC) allogeneic HSCT combined with MSC infusion for patients with the adult form of MLD.

Key words: MSC, stromal cell, metachromatic leukodystrophy, transplantation.

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Reply to: Reduced intensity conditioning haematopoietic stem cell transplantation with mesenchymal stromal cells infusion for the treatment of metachromatic leukodystrophy: a case report. Haematologica 2008; 93:e11–13

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				L. Charnas, F. Eichler, A. Kohlschuetter, J. Tolar, and P.J. Orchard Reply to: Reduced intensity conditioning haematopoietic stem cell transplantation with mesenchymal stromal cells infusion for the treatment of metachromatic leukodystrophy: a case report. Haematologica 2008; 93:e11-13 Haematologica, May 1, 2008; 93(5): e47 - e47. [Full Text] [PDF]

				N. Meuleman, T. Tondreau, G. Vanhaelen, L. Lagneaux, and D. Bron Reduced intensity conditioning haematopoietic stem cell transplantation with mesenchymal stromal cells infusion for the treatment of metachromatic leukodystrophy: a case report Haematologica, May 1, 2008; 93(5): e48 - e48. [Full Text] [PDF]