Salas Maria Queralt, Atenafu Eshetu G, Lam Wilson, Law Arjun Datt, Kim Dennis Dong Hwan, Michelis Fotios V, Al-Shaibani Zeyad, Gerbitz Armin, Lipton Jeffrey Howard, Viswabandya Auro, Mattsson Jonas, Gupta Vikas, Kumar Rajat
University of Toronto, Dept of Medicine, Section of Medical Oncology and Hematology.
Hans Messner Allogeneic Blood and Marrow Transplantation Program, Division of Medical Oncology and Hematology, Princess Margaret Cancer Centre, University Health Network, Toronto, Ontario, Canada.
Blood Cell Ther. 2020 Jul 14;3(3):48-58. doi: 10.31547/bct-2020-001. eCollection 2020 Aug 25.
We report a single-center experience of allogeneic hematopoietic stem cell transplant for patients with severe aplastic anemia over 13 years of age. Fifty-eight patients were included, and T-cell depletion was used in all cases. Fifty-one (88%) received alemtuzumab and 7 (12%) were given rabbit anti-thymocyte globulin. The median follow-up period was 6 years (range: 0-13.5). Data was collected retrospectively and updated in April 2019. The median age was 31 years (range: 18-67). Forty (69%) recipients received grafts from related donors and 18 (31%) received them from unrelated donors. Peripheral blood grafts were infused in 12 (20.7%) patients. Five-year overall survival (OS) was 80.7%. Five-year graft-versus-host disease (GVHD)/rejection-free survival was 56%. Eight (13.8%) patients experienced graft failure. The cumulative incidence of grade II-IV acute GVHD at day 100 was 14% and that of chronic GVHD at 1 year was 7%. The selection of unrelated donors and the use of peripheral blood grafts were not significant risk factors for clinically relevant GVHD or for lower OS. Recipients older than 40 years showed significantly worse OS, as observed from the results of univariate analysis. T-cell depletion in severe aplastic anemia shows low rates of GVHD and high OS, but older patients remain a group with higher risk of mortality. Long-term complications were mainly autoimmune in character.
我们报告了13岁以上重型再生障碍性贫血患者接受异基因造血干细胞移植的单中心经验。纳入了58例患者,所有病例均采用T细胞去除法。51例(88%)接受了阿仑单抗治疗,7例(12%)接受了兔抗胸腺细胞球蛋白治疗。中位随访期为6年(范围:0 - 13.5年)。数据为回顾性收集,并于2019年4月更新。中位年龄为31岁(范围:18 - 67岁)。40例(69%)受者接受了来自相关供者的移植物,18例(31%)接受了来自无关供者的移植物。12例(20.7%)患者输注了外周血移植物。5年总生存率(OS)为80.7%。5年无移植物抗宿主病(GVHD)/无排斥生存率为56%。8例(13.8%)患者发生移植物失败。100天时II - IV级急性GVHD的累积发生率为14%,1年时慢性GVHD的累积发生率为7%。选择无关供者和使用外周血移植物并非临床相关GVHD或较低OS的显著危险因素。单因素分析结果显示,年龄大于40岁的受者OS明显较差。重型再生障碍性贫血患者的T细胞去除法显示GVHD发生率低且OS高,但老年患者仍然是死亡率较高的群体。长期并发症主要具有自身免疫性。