O'Marcaigh A S, Cowan M J
Division of Pediatric Bone Marrow Transplantation, University of California at San Francisco 94143, USA.
Curr Opin Oncol. 1997 Mar;9(2):126-30.
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.
异基因骨髓移植已成功用于治疗多种遗传性疾病。在这种情况下，移植的目标是实现足够程度的持续骨髓植入，以使遗传性疾病表型得到长期改善。许多因素会影响实现这一目标的可能性，包括供体的可获得性、预处理方案、骨髓处理以及疾病进展的性质和程度。对于许多遗传性疾病来说，早期诊断至关重要，因为在疾病、其并发症或治疗导致显著器官损害之前进行移植，结果会更有利。尽管骨髓移植对某些遗传性疾病效果良好，对其他疾病的效果也在改善，但仍存在一些重大问题，如供体可获得性、预处理方案毒性、移植失败和移植物抗宿主病。本文综述描述了遗传性疾病骨髓移植的一些独特特征，并讨论了该领域的最新进展。
