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...
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| Format: | Article |
| Language: | English |
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Thieme Revinter Publicações
2006-01-01
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| Series: | Arquivos de Neuro-Psiquiatria |
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| Online Access: | http://www.scielo.br/scielo.php?script=sci_arttext&pid=S0004-282X2006000100001 |
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| author | Lange Marcos C. Teive Hélio A.G. Troiano André R. Bitencourt Marco Funke Vaneuza A.M. Setúbal Daniela C. Zanis Neto José Medeiros Carlos R. Werneck Lineu C. Pasquini Ricardo Bonfim Carmen M.S. |
| author_facet | Lange Marcos C. Teive Hélio A.G. Troiano André R. Bitencourt Marco Funke Vaneuza A.M. Setúbal Daniela C. Zanis Neto José Medeiros Carlos R. Werneck Lineu C. Pasquini Ricardo Bonfim Carmen M.S. |
| author_sort | Lange Marcos C. |
| collection | DOAJ |
| description | 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). |
| format | Article |
| id | doaj-art-31d64b7bdb3249ba9a45b827944dc4a4 |
| institution | OA Journals |
| issn | 0004-282X 1678-4227 |
| language | English |
| publishDate | 2006-01-01 |
| publisher | Thieme Revinter Publicações |
| record_format | Article |
| series | Arquivos de Neuro-Psiquiatria |
| spelling | doaj-art-31d64b7bdb3249ba9a45b827944dc4a42025-08-20T02:01:40ZengThieme Revinter PublicaçõesArquivos de Neuro-Psiquiatria0004-282X1678-42272006-01-0164114Bone marrow transplantation in patients with storage diseases: a developing country experienceLange Marcos C.Teive Hélio A.G.Troiano André R.Bitencourt MarcoFunke Vaneuza A.M.Setúbal Daniela C.Zanis Neto JoséMedeiros Carlos R.Werneck Lineu C.Pasquini RicardoBonfim Carmen M.S.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).http://www.scielo.br/scielo.php?script=sci_arttext&pid=S0004-282X2006000100001storage diseasesbone marrow transplantationgenetic neurological diseasesmucopolysaccharidosisadrenoleukodystrophyGaucher disease |
| spellingShingle | Lange Marcos C. Teive Hélio A.G. Troiano André R. Bitencourt Marco Funke Vaneuza A.M. Setúbal Daniela C. Zanis Neto José Medeiros Carlos R. Werneck Lineu C. Pasquini Ricardo Bonfim Carmen M.S. Bone marrow transplantation in patients with storage diseases: a developing country experience Arquivos de Neuro-Psiquiatria storage diseases bone marrow transplantation genetic neurological diseases mucopolysaccharidosis adrenoleukodystrophy Gaucher disease |
| title | Bone marrow transplantation in patients with storage diseases: a developing country experience |
| title_full | Bone marrow transplantation in patients with storage diseases: a developing country experience |
| title_fullStr | Bone marrow transplantation in patients with storage diseases: a developing country experience |
| title_full_unstemmed | Bone marrow transplantation in patients with storage diseases: a developing country experience |
| title_short | Bone marrow transplantation in patients with storage diseases: a developing country experience |
| title_sort | bone marrow transplantation in patients with storage diseases a developing country experience |
| topic | storage diseases bone marrow transplantation genetic neurological diseases mucopolysaccharidosis adrenoleukodystrophy Gaucher disease |
| url | http://www.scielo.br/scielo.php?script=sci_arttext&pid=S0004-282X2006000100001 |
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