Brune Mats, Kiss Thomas, Anderson Harald, Nicklasson Malin, Delage Robert, Finke Jürgen, Gedde-Dahl Tobias, Hébert Josée, Höglund Martin, Kaare Ain, Lazarevic Vladimir, Möllgård Lars, Remes Kari, Ritchie David, Spyridonidis Alexandros, Sabloff Mitchell, Spearing Ruth, Wallhult Elisabeth, Ljungman Per
Section of Hematology and Coagulation, Department of Specialist Medicine, Sahlgrenska University Hospital, Gothenburg, Sweden.
Hopital Maisonneuve-Rosemont, Division of Hematology, Oncology, Hematopoietic Cell Transplant and Cellular therapy, Université de Montréal, Montréal, QC, Canada.
Bone Marrow Transplant. 2024 Dec;59(12):1676-1682. doi: 10.1038/s41409-024-02408-x. Epub 2024 Sep 2.
The aim of this prospective, international multicenter, pseudorandomized study comparing RICT HCT to standard-of-care chemotherapy in intermediate- or high-risk AML patients 50-70 years using the donor versus no-donor concept. Part 1 included only patients with potential family donors (RD) at the date of HLA-typing of the first potential sibling or CR-date, if later. Part 2 allowed the inclusion of patients without a possible sibling donor using the start of an unrelated donor (URD) search as inclusion date. 360 patients were registered and 309 analyzed. The median follow-up was 47 months (1-168). There was no difference in overall survival (OS) between the RD (n = 124) and the Control (n = 77) groups (p = 0.50, 3-year OS RD: 0.41(95% CI; 0.32-0.50); Controls: 0.49 (95% CI; 0.37-0.59)). The main cause of death was relapse (67% RD; 88% Controls). In Part 2, the 3-year OS was 0.60 (95% CI 0.50-0.70) for URD-HCT (n = 86) and 0.37 (95% CI 0.13-0.62) for Controls (n = 20), respectively (p = 0.10). When analyzing transplanted patients (Part 2), the OS at 3-years was higher for URD-HCT than RD-HCT (0.67 (0.55-0.76) vs. 0.42 (0.26-0.57; p = 0.005). This study doesn't support elderly HLA-identical siblings as donors for older AML patients undergoing a RICT allogeneic HCT in first CR.
本前瞻性、国际多中心、伪随机研究采用有供者与无供者的概念，比较50至70岁中高危急性髓系白血病（AML）患者接受减低预处理强度异基因造血干细胞移植（RICT HCT）与标准护理化疗的效果。第1部分仅纳入在首次潜在同胞HLA分型日期或CR日期（若更晚）时有潜在家族供者（RD）的患者。第2部分允许纳入没有可能的同胞供者的患者，以开始无关供者（URD）搜索的日期作为纳入日期。共登记360例患者，309例进行分析。中位随访时间为47个月（1至168个月）。RD组（n = 124）和对照组（n = 77）的总生存期（OS）无差异（p = 0.50，3年OS，RD组：0.41（95%CI；0.32 - 0.50）；对照组：0.49（95%CI；0.37 - 0.59））。主要死亡原因是复发（RD组67%；对照组88%）。在第2部分中，URD - HCT组（n = 86）的3年OS为0.60（95%CI 0.50 - 0.70），对照组（n = 20）为0.37（95%CI 0.13 - 0.62）（p = 0.10）。分析移植患者（第2部分）时发现，URD - HCT的3年OS高于RD - HCT（0.67（0.55 - 0.76）对0.42（0.26 - 0.57；p = 0.005）。本研究不支持老年HLA相同的同胞作为首次完全缓解期接受RICT异基因HCT的老年AML患者的供者。
