The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, John Hopkins University, Baltimore, MD, United States of America.
Hematology and Bone Marrow Transplant Unit, Fondazione Universitario Policlinico Gemelli IRCCS, Rome, Italy.
Blood Rev. 2023 Nov;62:100990. doi: 10.1016/j.blre.2022.100990. Epub 2022 Jul 14.
Post-transplant cyclophosphamide (PTCy) allows safe and effective partially matched donor allogeneic blood or marrow transplantation (alloBMT), so that almost everyone in need of the procedure now has a donor. Moreover, PTCy and other recent advances have lowered alloBMT mortality rates to less than half of that seen before the turn of the century, at costs that are substantially less than most newly approved anticancer agents. These advances also make tailoring BMT based on patients' unique diseases and characteristics now feasible for further improving outcomes. Personalizing every aspect of alloBMT, including conditioning, donor, graft type, and post-transplant maintenance is now possible. For example, alloBMT's antitumor activity historically was restricted to the allogeneic graft-versus-tumor effect directed against histocompatibility antigens. However, replacing exhausted immune systems with healthy non-exhausted, non-tolerant ones likely can enhance the activity of novel targeted therapies. The impressive results seen with tyrosine kinase inhibitors after alloBMT for patients with both Ph+ acute lymphoblastic leukemia and FLT/ITD+ acute myeloid leukemia herald the potential of precision BMT.
移植后环磷酰胺（PTCy）可安全有效地进行部分匹配供体同种异体血液或骨髓移植（alloBMT），因此现在几乎每个有需要的人都有供体。此外，PTCy 和其他最近的进展将 alloBMT 的死亡率降低到本世纪初之前的一半以下，成本大大低于大多数新批准的抗癌药物。这些进展还使得根据患者独特的疾病和特征来定制 BMT 成为可能，从而进一步提高治疗效果。现在可以个性化 alloBMT 的各个方面，包括预处理、供体、移植物类型和移植后维持治疗。例如，alloBMT 的抗肿瘤活性历史上仅限于针对组织相容性抗原的同种异体移植物抗肿瘤效应。然而，用健康的、非衰竭的、非耐受的免疫系统替代衰竭的免疫系统，可能会增强新型靶向治疗的活性。在 alloBMT 后接受酪氨酸激酶抑制剂治疗 Ph+急性淋巴细胞白血病和 FLT/ITD+急性髓细胞白血病的患者取得了令人印象深刻的结果，预示着精准 BMT 的潜力。
