PMID: 9161789Mar 1, 1997Paper

Bone marrow transplantation for inherited diseases

Current Opinion in Oncology
A S O'Marcaigh, M J Cowan

Abstract

Allogeneic bone marrow transplantation has been used successfully for the treatment of a variety of inherited diseases. The goal of transplantation in this setting is to provide a sufficient degree of sustained marrow engraftment to allow longterm amelioration of the inherited disease phenotype. Many factors influence the likelihood of achieving this goal, including donor availability, conditioning regimen, marrow processing, and the nature and extent of progression of the disease. For many inherited diseases early diagnosis is imperative because the outcome of transplantation is more favorable when performed prior to the development of significant organ damage from the disease, its complications, or treatment. Although the results of bone marrow transplantation are good for some inherited diseases and are improving for others, significant problems remain such as donor availability, conditioning regimen toxicity, graft failure, and graft-versus-host disease. This review describes some of the unique features of bone marrow transplantation for inherited diseases and discusses recent advances in this area.

Citations

Jun 23, 1999·Proceedings of the National Academy of Sciences of the United States of America·Z HouG S Stein
Jun 27, 2002·Proceedings of the National Academy of Sciences of the United States of America·Edwin M HorwitzTed Hofmann
Mar 30, 2007·Acta Paediatrica. Supplement·Mark E Haskins
May 18, 1999·The Journal of Allergy and Clinical Immunology·A P Knutsen, D A Wall
Apr 7, 2005·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Crystal Y KohWilliam J Murphy
Jul 20, 2002·Blood·John MilledgeJohn Christodoulou
Jul 11, 2000·British Journal of Haematology·M I GonzálezJ F San Miguel

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