title: Bone marrow transplantation for late infantile metachromatic leukodystrophy: pathogenic investigation for graft rejection.
abstract: A Japanese boy aged 2 years 11 months with late infantile metachromatic leukodystrophy underwent bone marrow transplantation (BMT) from his human leukocyte antigen (HLA) identical but mixed lymphocyte culture reactive father. Chimerism and increased arylsulfatase A activities of leukocytes had been observed with retarded progression of neurological deterioration during the first 3 months post-BMT. Graft rejection gradually occurred and donor cells were almost completely eliminated from the patient at 1 year after BMT. The process of neurodegeneration progressed clinically and neuroradiologically. Three possible reasons for the pathogenesis of graft rejection are: (i) T cell depletion of donor marrow cells as graft-versus-host disease (GVHD) prophylaxis; (ii) a slightly weak conditioning regimen: and (iii) a small number of marrow cells transplanted. It is stressed that as BMT is still a preliminary therapy for metachromatic leukodystrophy indications, conditioning, and GVHD prophylaxis for BMT should be considered individually.
authors.affiliation: Department of Neurology, Tokyo Metropolitan Kiyose Children's Hospital, Japan.
authors.forename: T
authors.initials: T
authors.lastname: Nagai
authors.forename: T
authors.initials: T
authors.lastname: Kaneko
authors.forename: H
authors.initials: H
authors.lastname: Shichijou
authors.forename: T
authors.initials: T
authors.lastname: Karato
authors.forename: A
authors.initials: A
authors.lastname: Maruyama
authors.forename: Y
authors.initials: Y
authors.lastname: Tsuchiya
mesh_terms: D016026:Bone Marrow Transplantation; D002675:Child, Preschool; D003241:Consanguinity; D016057:Evoked Potentials, Auditory, Brain Stem; D005190:Family; D006084:Graft Rejection / Q000473:pathology*; D006801:Humans; D007966:Leukodystrophy, Metachromatic / Q000175:diagnosis / Q000628:therapy*; D008279:Magnetic Resonance Imaging; D008297:Male; D010375:Pedigree; D014057:Tomography, X-Ray Computed