Adult Bone Marrow Transplantation Service, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY.
Department of Hematology/Oncology, Dana-Farber Cancer Institute, Boston, MA.
J Clin Oncol. 2024 Oct;42(28):3277-3286. doi: 10.1200/JCO.24.00184. Epub 2024 Jul 17.
PURPOSEAccess to allogeneic hematopoietic cell transplantation (HCT) remains limited among persons of non-European ancestry if human leukocyte antigen (HLA) matching is required. We evaluated whether post-transplant cyclophosphamide (PTCy)-based graft-versus-host disease (GVHD) prophylaxis improved HCT outcomes with HLA-matched unrelated donor (MUD) and mismatched unrelated donor (MMUD) HCT when compared with calcineurin inhibitor (CNI)-based prophylaxis.METHODSThree-year overall survival (OS) and GVHD-free, relapse-free survival (GRFS) were compared between adult recipients undergoing initial MUD or single HLA locus MMUD HCT with either PTCy- or CNI-based prophylaxis who were reported to the Center for International Blood and Marrow Transplant Research between 2017 and 2021.RESULTSIncluded were 10,025 HCT recipients (7,272 recipients of MUD with CNI, 1,681 MUD with PTCy, 613 MMUD with CNI, and 459 MMUD with PTCy) who underwent HCT for acute leukemia (70.9%) or myelodysplastic syndromes (29.2%). Median patient age was 60.7 years (range, 18.0-82.7) and median follow-up was 36.6 (range, 3.0-77.8) months. When compared with MUD HCT with PTCy, MMUD HCT with PTCy had similar OS (hazard ratio [HR], 0.96 [95% CI, 0.823 to 1.11]; = .60) and GRFS (HR, 0.90 [0.79 to 1.02]; = .1). When compared with MUD HCT with CNI, OS was improved after MUD HCT with PTCy (HR, 0.88 [0.80 to 0.96]; = .004) and GRFS was improved with PTCy after either MUD (HR, 0.61 [0.57 to 0.66]; < .0001) or MMUD (HR, 0.68 [0.60 to 0.76]; < .0001) HCT. Benefit from PTCy was independent of patient ancestry. Global registry level analysis demonstrated that inclusion of MMUD increased donor availability regardless of recipient ancestry.CONCLUSIONUse of PTCy results in comparable OS and GRFS using either MUD or MMUD HCT, expanding access to HCT for patients from all racial and ethnic ancestry groups.
如果需要人类白细胞抗原（HLA）匹配，那么非欧洲血统的人获得同种异体造血细胞移植（HCT）的机会仍然有限。我们评估了在与 HLA 匹配的无关供体（MUD）和不匹配的无关供体（MMUD）HCT 中，与钙调神经磷酸酶抑制剂（CNI）为基础的预防相比，使用环磷酰胺（PTCy）为基础的移植物抗宿主病（GVHD）预防是否改善了 HCT 结果。
2017 年至 2021 年期间，向国际血液和骨髓移植研究中心报告的接受初始 MUD 或单 HLA 位点 MMUD HCT 的成年受者中，比较了接受 PTCy 或 CNI 为基础的预防的受者的 3 年总生存率（OS）和无 GVHD、无复发生存（GRFS）。
共纳入 10025 例 HCT 受者（7272 例 MUD 接受 CNI，1681 例 MUD 接受 PTCy，613 例 MMUD 接受 CNI，459 例 MMUD 接受 PTCy），他们因急性白血病（70.9%）或骨髓增生异常综合征（29.2%）接受 HCT。中位患者年龄为 60.7 岁（范围，18.0-82.7），中位随访时间为 36.6 个月（范围，3.0-77.8）。与 MUD HCT 联合 PTCy 相比，MMUD HCT 联合 PTCy 的 OS 相似（危险比[HR]，0.96[95%CI，0.823 至 1.11]；=.60）和 GRFS（HR，0.90[0.79 至 1.02]；=.1）。与 MUD HCT 联合 CNI 相比，MUD HCT 联合 PTCy 后的 OS 得到改善（HR，0.88[0.80 至 0.96]；=.004），并且在接受 MUD（HR，0.61[0.57 至 0.66]；<.0001）或 MMUD（HR，0.68[0.60 至 0.76]；<.0001）HCT 后，PTCy 也改善了 GRFS。PTCy 的获益与患者的种族无关。全球注册水平分析表明，无论受者的种族如何，纳入 MMUD 均可增加供者的可用性。
使用 PTCy 可获得与使用 MUD 或 MMUD HCT 相似的 OS 和 GRFS，扩大了 HCT 机会，使所有种族和族裔背景的患者受益。
