Bone marrow transplantation in patients with storage diseases: a developing country experience.
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).
Lange
Marcos C
MC
Neurology Division, Internal Medicine Department, Hospital de Clínicas, Universidade Federal do Paraná, Curitiba, PR, Brazil.
Teive
Hélio A G
HA
Troiano
André R
AR
Bitencourt
Marco
M
Funke
Vaneuza A M
VA
Setúbal
Daniela C
DC
Zanis Neto
José
J
Medeiros
Carlos R
CR
Werneck
Lineu C
LC
Pasquini
Ricardo
R
Bonfim
Carmen M S
CM
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