Reduced intensity conditioning haematopoietic stem cell transplantation with mesenchymal stromal cells infusion for the treatment of metachromatic leukodystrophy: a case report.
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.Case ReportsJournal Articleinfo:eu-repo/semantics/publishe
Sciences bio-médicales et agricoles, Adult, Bone Marrow Cells -- cytology, Bone Marrow Transplantation, Cerebroside-Sulfatase -- biosynthesis, Female, Graft Survival, Hematopoietic Stem Cell Transplantation -- methods, Humans, Leukodystrophy, Metachromatic -- blood, Leukodystrophy, Metachromatic -- therapy, Magnetic Resonance Imaging, Mesoderm -- metabolism, Stromal Cells -- cytology, Stromal Cells -- pathology, Transplantation Conditioning -- methods, Treatment Outcome
Publisher: 'Ferrata Storti Foundation (Haematologica)'
DOI identifier: 10.3324/haematol.11802
Sorry, we are unable to provide the full text but you may find it at the