Portal de Periódicos da CAPES

Artigo Acesso aberto Produção Nacional Revisado por pares

BIBTEX RIS

Bone marrow transplantation in patients with storage diseases: a developing country experience

2006; Thieme Medical Publishers (Germany); Volume: 64; Issue: 1 Linguagem: Inglês

10.1590/s0004-282x2006000100001

1678-4227

Marcos Christiano Lange, Hélio Afonso Ghizoni Teive, André R. Troiano, Marco A. Bitencourt, Vaneuza Araújo Moreira Funke, D.C. Setubal, José Zanis Neto, Carlos Roberto de Medeiros, Lineu César Werneck, Ricardo Pasqüini, Carmen Bonfim,

Carbohydrate Chemistry and Synthesis

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).