Bone Marrow Transplantation Department, Hospital Israelita Albert Einstein, Guaramomis 480/64, São Paulo, SP, CEP 04076-010, Brazil.
Bone Marrow Transplantation Department, Instituto Nacional de Cancer, Rio de Janeiro, Brazil.
Ann Hematol. 2022 Aug;101(8):1795-1802. doi: 10.1007/s00277-022-04865-0. Epub 2022 May 16.
Allogeneic hematopoietic cell transplantation (HCT) is a potentially curative therapy for several malignant hematologic diseases and alternative donors, including haploidentical, play a significant role in HCT. Despite the increasing use of haplo-HCT with PTCy, some questions remain open. The objective of the present study was to investigate risk factors for adverse outcomes after haplo-HCT with PTCy. This is a retrospective study conducted at two Brazilian centers. A total of 103 patients with hematologic malignancies who underwent first allogeneic, haploidentical HCT with PTCy were included. Risk factors for death were age at transplant (HR = 1.03 for each year; p = 0.002) and high/very high disease risk index (DRI; HR = 2.77; p = 0.0007) and mother as the donor compared with other donors (HR = 3.53; p = 0.005). In multivariate analysis, PFS was significantly poorer for older patients (HR = 1.02; p = 0.006), high/very high DRI (HR = 2.39; p = 0.003), and mother as the donor compared with other donors (HR = 3.18; p = 0.006). Relapse rate was higher for high/very high DRI (HR = 4.01; p = 0.002) and mother as the donor compared with other donors (HR = 2.52; p = 0.05). NRM was higher for older patients (HR = 1.03 for each year; p = 0.03). Tacrolimus was a protective factor for grades II-IV aGVHD (HR = 0.46; p = 0.04) compared with cyclosporine. Peripheral blood (PBSC) was a risk factor for cGVHD (HR = 3.48; p = 0.006), while tacrolimus was protective (HR = 0.30; p = 0.009). Mother as the donor compared with other donors was also a risk factor for poorer OS, PFS, and relapse, suggesting that this combination should be avoided. Tacrolimus was protective for both grades II-IV aGVHD and cGVHD, suggesting that tacrolimus may be more effective than cyclosporine in preventing GVHD. PBSC was a risk factor for cGVHD without any impact on relapse. Prospective studies comparing tacrolimus with cyclosporine are awaited.
异基因造血细胞移植（HCT）是多种恶性血液病的潜在治愈疗法，供者包括单倍体相合，在 HCT 中发挥重要作用。尽管随着 PTCy 的应用，单倍体-HCT 的使用有所增加，但仍存在一些悬而未决的问题。本研究的目的是研究 PTCy 后单倍体-HCT 不良结局的危险因素。这是在巴西的两个中心进行的回顾性研究。共纳入 103 例接受异基因、单倍体 HCT 伴 PTCy 的血液系统恶性肿瘤患者。移植时年龄（每增加 1 岁 HR=1.03；p=0.002）和高/极高疾病风险指数（DRI；HR=2.77；p=0.0007）以及母亲作为供者与其他供者相比是死亡的危险因素（HR=3.53；p=0.005）。多变量分析显示，年龄较大（HR=1.02；p=0.006）、高/极高 DRI（HR=2.39；p=0.003）和母亲作为供者与其他供者相比（HR=3.18；p=0.006）的患者，PFS 明显较差。高/极高 DRI（HR=4.01；p=0.002）和母亲作为供者与其他供者相比（HR=2.52；p=0.05）的患者复发率较高。年龄较大（HR=1.03 每年；p=0.03）的患者 NRM 较高。与环孢素相比，他克莫司是 II-IV 级移植物抗宿主病（GVHD）的保护因素（HR=0.46；p=0.04）。外周血（PBSC）是 cGVHD 的危险因素（HR=3.48；p=0.006），而他克莫司具有保护作用（HR=0.30；p=0.009）。与其他供者相比，母亲作为供者也是 OS、PFS 和复发较差的危险因素，提示应避免这种组合。他克莫司对 II-IV 级 aGVHD 和 cGVHD 均有保护作用，提示他克莫司在预防 GVHD 方面可能比环孢素更有效。PBSC 是 cGVHD 的危险因素，但对复发无影响。期待进行比较他克莫司与环孢素的前瞻性研究。
