title: Reduced intensity conditioning haematopoietic stem cell transplantation with mesenchymal stromal cells infusion for the treatment of metachromatic leukodystrophy: a case report.
abstract: 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.
authors.affiliation: Department of Clinical and Experimental Hematology, Institut Jules Bordet, Université Libre de Bruxelles (ULB), Bruxelles, Belgium. nathalie.meuleman@bordet.be
authors.forename: N
authors.initials: N
authors.lastname: Meuleman
authors.forename: G
authors.initials: G
authors.lastname: Vanhaelen
authors.forename: T
authors.initials: T
authors.lastname: Tondreau
authors.forename: P
authors.initials: P
authors.lastname: Lewalle
authors.forename: J
authors.initials: J
authors.lastname: Kwan
authors.forename: J
authors.initials: J
authors.lastname: Bennani
authors.forename: P
authors.initials: P
authors.lastname: Martiat
authors.forename: L
authors.initials: L
authors.lastname: Lagneaux
authors.forename: D
authors.initials: D
authors.lastname: Bron
mesh_terms: D000328:Adult; D001854:Bone Marrow Cells / Q000166:cytology; D016026:Bone Marrow Transplantation; D002553:Cerebroside-Sulfatase / Q000096:biosynthesis; D005260:Female; D006085:Graft Survival; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D007966:Leukodystrophy, Metachromatic / Q000097:blood* / Q000628:therapy*; D008279:Magnetic Resonance Imaging; D008648:Mesoderm / Q000378:metabolism*; D017154:Stromal Cells / Q000166:cytology* / Q000473:pathology*; D019172:Transplantation Conditioning / Q000379:methods*; D016896:Treatment Outcome
chemical_list: D002553:Cerebroside-Sulfatase