Henslee-Downey P J
Department of Medicine and Pediatrics, University of South Carolina, Columbia.
Am J Pediatr Hematol Oncol. 1993 May;15(2):150-61. doi: 10.1097/00043426-199305000-00002.
Bone marrow transplantation (BMT) can be curative for patients with hematological malignancies, marrow failure syndromes, and certain metabolic disorders. However, fewer than half of the patients who could benefit have a donor who is either an HLA-matched sibling or a phenotypically similar unrelated volunteer.
The opportunity for allogeneic bone marrow transplantation is significantly increased by the use of partially mismatched, haploidentical related donors. The likelihood of finding a donor within the family is dependent on the acceptable degree of mismatch on the unshared chromosome. The degree of mismatch is expected to be approximately 10% for a one-antigen mismatch, 20-30% for a two-antigen mismatch, and > 95% for a three-antigen mismatch. Improvements in transplant techniques designed to enhance engraftment and to prevent the development of severe acute and chronic graft-versus-host disease have increased the feasibility of utilizing this readily available donor pool. Obviously, the ability to use a three-antigen mismatched related donor would make bone marrow transplantation available for almost every patient.
Recent studies combining in vitro and in vivo immunomodulation of donor lymphocytes have resulted in consistent engraftment and control of acute graft-versus-host disease.
These findings provide encouragement that there may need be no restriction on the availability of allogeneic marrow transplants as a therapeutic option for appropriate candidates.
骨髓移植(BMT)对血液系统恶性肿瘤、骨髓衰竭综合征及某些代谢紊乱患者可能具有治愈作用。然而,能够从中获益的患者中,不到一半有人类白细胞抗原(HLA)匹配的同胞供者或表型相似的无关志愿供者。
使用部分不匹配的单倍体相合相关供者可显著增加异基因骨髓移植的机会。在家族中找到供者的可能性取决于非共享染色体上可接受的不匹配程度。单抗原不匹配时,不匹配程度预计约为10%;双抗原不匹配时为20% - 30%;三抗原不匹配时则> 95%。旨在提高植入率并预防严重急性和慢性移植物抗宿主病发生的移植技术改进,增加了利用这一现成供者库的可行性。显然,使用三抗原不匹配相关供者的能力将使几乎每位患者都能接受骨髓移植。
近期将供者淋巴细胞的体外和体内免疫调节相结合的研究,已实现持续植入并控制急性移植物抗宿主病。
这些发现令人鼓舞,提示对于合适的候选者,作为一种治疗选择的异基因骨髓移植在可及性方面可能无需受限。