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

Arquivos de neuro-psiquiatria
Marcos C LangeCarmen M S Bonfim

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).

References

Mar 24, 1994·The New England Journal of Medicine·J O Armitage
Jun 1, 2000·Bone Marrow Transplantation·C Peters, W Krivit
Aug 18, 2000·Journal of Inherited Metabolic Disease·Y SuzukiN Kondo
Jan 5, 2002·Journal of Inherited Metabolic Disease·A Erikson
Apr 4, 2002·Drugs·Raphael Schiffmann, Roscoe O Brady
Aug 27, 2002·Journal of Neurology·A FassasUNKNOWN Autoimmune Disease Working Party of the EBMT (European Group for Blood and Marrow Transplantation)
Mar 7, 2003·Bone Marrow Transplantation·C PetersUNKNOWN Working Party on Inborn Errors, European Bone Marrow Transplant Group
Dec 20, 2003·Pediatric Neurology·J Jeffrey MalatackEliel Bayever
Jan 10, 2004·The Journal of Laboratory and Clinical Medicine·Charles Peters
May 7, 2004·The New England Journal of Medicine·Susan L StabaJoanne Kurtzberg
Aug 31, 2004·Arquivos de neuro-psiquiatria·Orlando G Povoas BarsottiniNelson Hamerschlak

❮ Previous
Next ❯

Citations

Apr 14, 2010·Orphanet Journal of Rare Diseases·Vassili ValayannopoulosSean Turbeville
Sep 26, 2009·The Journal of Pediatrics·Ana Maria MartinsZelita Caldeira Ferreira Guedes
Mar 10, 2009·Brain & Development·Laura Bannach JardimRoberto Giugliani
Apr 9, 2015·Pediatrics International : Official Journal of the Japan Pediatric Society·Fernando AndradeMaría Luz Couce
Nov 24, 2011·Strabismus·Marcel Pm Ten Tusscher
Jun 4, 2011·Genetics and Molecular Biology·Roberto GiuglianiAna Maria Martins
Dec 14, 2018·Frontiers in Physiology·Valeriya V SolovyevaAlbert A Rizvanov
Mar 5, 2021·Journal of Pediatric Hematology/oncology·Shevachut ChavananonThirachit Chotsampancharoen
Dec 5, 2021·Journal of Pediatric Hematology/oncology·Shevachut ChavananonThirachit Chotsampancharoen

❮ Previous
Next ❯

Related Concepts

Related Feeds

Adrenoleukodystrophy

Adrenoleukodystrophy (ALD), the most frequent peroxisomal disorder, is an X-linked disorder caused by a defect in the metabolism of long chain fatty acids leading to demyelination, neurodegeneration, and death. Here is the latest research.

Blood And Marrow Transplantation

The use of hematopoietic stem cell transplantation or blood and marrow transplantation (bmt) is on the increase worldwide. BMT is used to replace damaged or destroyed bone marrow with healthy bone marrow stem cells. Here is the latest research on bone and marrow transplantation.