Department of Hematology and Hematopoietic Cell Transplantation and.
Department of Computational Quantitative Medicine-Beckman Research Institute, City of Hope National Medical Center, Duarte, CA.
Blood Adv. 2020 May 12;4(9):2073-2083. doi: 10.1182/bloodadvances.2020001499.
We compared outcomes of 1461 adult patients with acute lymphoblastic leukemia (ALL) receiving hematopoietic cell transplantation (HCT) from a haploidentical (n = 487) or matched unrelated donor (MUD; n = 974) between January 2005 and June 2018. Graft-versus-host disease (GVHD) prophylaxis was posttransplant cyclophosphamide (PTCy), calcineurin inhibitor (CNI), and mycophenolate mofetil (MMF) for haploidentical, and CNI with MMF or methotrexate with/without antithymoglobulin for MUDs. Haploidentical recipients were matched (1:2 ratio) with MUD controls for sex, conditioning intensity, disease stage, Philadelphia-chromosome status, and cytogenetic risk. In the myeloablative setting, day +28 neutrophil recovery was similar between haploidentical (87%) and MUD (88%) (P = .11). Corresponding rates after reduced-intensity conditioning (RIC) were 84% and 88% (P = .47). The 3-month incidence of grade II-IV acute GVHD (aGVHD) and 3-year chronic GVHD (cGVHD) was similar after haploidentical compared with MUD: myeloablative conditioning, 33% vs 34% (P = .46) for aGVHD and 29% vs 31% for cGVHD (P = .58); RIC, 31% vs 30% (P = .06) for aGVHD and 24% vs 29% for cGVHD (P = .86). Among patients receiving myeloablative regimens, 3-year probabilities of overall survival were 44% and 51% with haploidentical and MUD (P = .56). Corresponding rates after RIC were 43% and 42% (P = .6). In this large multicenter case-matched retrospective analysis, despite the limitations of a registry-based study (ie, unavailability of key elements such as minimal residual disease testing), our analysis indicated that outcomes of patients with ALL undergoing HCT from a haploidentical donor were comparable with 8 of 8 MUD transplantations.
我们比较了 2005 年 1 月至 2018 年 6 月期间，接受亲缘半相合（n=487）或匹配无关供者（MUD；n=974）造血细胞移植（HCT）的 1461 例成人急性淋巴细胞白血病（ALL）患者的结局。移植物抗宿主病（GVHD）预防方案为亲缘半相合组用环磷酰胺（PTCy）、钙调神经磷酸酶抑制剂（CNI）和霉酚酸酯（MMF），MUD 组用 CNI 联合 MMF 或甲氨蝶呤联合/不联合抗胸腺球蛋白。亲缘半相合组与 MUD 对照组按性别、预处理强度、疾病分期、费城染色体状态和细胞遗传学风险 1:2 匹配。在清髓性预处理条件下，亲缘半相合组（87%）和 MUD 组（88%）的+28 天中性粒细胞恢复情况相似（P=0.11）。在减低强度预处理条件下，相应的恢复率分别为 84%和 88%（P=0.47）。亲缘半相合组与 MUD 组比较，+3 个月时 II-IV 级急性 GVHD（aGVHD）和 3 年慢性 GVHD（cGVHD）的发生率相似：清髓性预处理，aGVHD 为 33%比 34%（P=0.46），cGVHD 为 29%比 31%（P=0.58）；减低强度预处理，aGVHD 为 31%比 30%（P=0.06），cGVHD 为 24%比 29%（P=0.86）。在接受清髓性方案的患者中，亲缘半相合组和 MUD 组的 3 年总生存率分别为 44%和 51%（P=0.56）。相应的 RIC 后生存率分别为 43%和 42%（P=0.6）。在这项大型多中心病例匹配回顾性分析中，尽管存在注册研究的局限性（即无法获得微小残留病检测等关键要素），但我们的分析表明，接受亲缘半相合供者 HCT 的 ALL 患者的结局与 8 例 MUD 移植相当。
