Arquivos de Neuro-Psiquiatria | |
Bone marrow transplantation in patients with storage diseases: a developing country experience | |
Marcos C. Lange2  Hélio A.g. Teive2  André R. Troiano2  Marco Bitencourt1  Vaneuza A.m. Funke1  Daniela C. Setúbal1  José Zanis Neto1  Carlos R. Medeiros1  Lineu C. Werneck2  Ricardo Pasquini1  Carmen M.s. Bonfim1  | |
[1] ,Universidade Federal do Paraná Hospital de Clínicas Internal Medicine DepartmentCuritiba PR ,Brazil | |
关键词: storage diseases; bone marrow transplantation; genetic neurological diseases; mucopolysaccharidosis; adrenoleukodystrophy; Gaucher disease; doenças de acúmulo; transplante de medula óssea; doenças neurológicas genéticas; mucopolissacaridose; adrenoleucodistrofia; doença de Gaucher; | |
DOI : 10.1590/S0004-282X2006000100001 | |
来源: SciELO | |
【 摘 要 】
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).
【 授权许可】
CC BY
All the contents of this journal, except where otherwise noted, is licensed under a Creative Commons Attribution License
【 预 览 】
Files | Size | Format | View |
---|---|---|---|
RO202103040009644ZK.pdf | 148KB | download |