title: Bone marrow transplantation in patients with storage diseases: a developing country experience.
abstract: Bone marrow transplantation (BMT) is a therapeutic option for patients with genetic storage diseases. Between 1979 and 2002, eight patients, four females and four males (1 to 13 years old) were submitted to this procedure in our center. Six patients had mucopolysaccharidosis (MPS I in 3; MPS III in one and MPS VI in 2), one had adrenoleukodystrophy (ALD) and one had Gaucher disease. Five patients had related and three unrelated BMT donor. Three patients developed graft versus host disease (two MPS I and one MPS VI) and died between 37 and 151 days after transplantation. Five patients survived 4 to 16 years after transplantation. Three patients improved (one MPS I; one MPS VI and the Gaucher disease patient), one patient had no disease progression (ALD) and in one patient this procedure did not change the natural course of the disease (MPS III).
authors.affiliation: Neurology Division, Internal Medicine Department, Hospital de Clínicas, Universidade Federal do Paraná, Curitiba, PR, Brazil.
authors.forename: Marcos C
authors.initials: MC
authors.lastname: Lange
authors.forename: Hélio A G
authors.initials: HA
authors.lastname: Teive
authors.forename: André R
authors.initials: AR
authors.lastname: Troiano
authors.forename: Marco
authors.initials: M
authors.lastname: Bitencourt
authors.forename: Vaneuza A M
authors.initials: VA
authors.lastname: Funke
authors.forename: Daniela C
authors.initials: DC
authors.lastname: Setúbal
authors.forename: José
authors.initials: J
authors.lastname: Zanis Neto
authors.forename: Carlos R
authors.initials: CR
authors.lastname: Medeiros
authors.forename: Lineu C
authors.initials: LC
authors.lastname: Werneck
authors.forename: Ricardo
authors.initials: R
authors.lastname: Pasquini
authors.forename: Carmen M S
authors.initials: CM
authors.lastname: Bonfim
mesh_terms: D000293:Adolescent; D000326:Adrenoleukodystrophy / Q000601:surgery; D016026:Bone Marrow Transplantation / Q000401:mortality; D001938:Brazil / Q000453:epidemiology; D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D005776:Gaucher Disease / Q000601:surgery; D006086:Graft vs Host Disease; D006801:Humans; D007223:Infant; D016464:Lysosomal Storage Diseases / Q000401:mortality / Q000601:surgery*; D008297:Male; D009083:Mucopolysaccharidoses / Q000601:surgery; D016019:Survival Analysis; D016896:Treatment Outcome