Choudhury Dharma, Kumar Meet, Sharma Sanjeev, Khandelwal Vipin, Doval Divya, Dadu Tina, Setia Rasika, Handoo Anil
Department of Hematology and BMT, BLK Super Specialty Hospital, New Delhi.
Department of Hematology, BLK Super Specialty Hospital, New Delhi.
Blood Cell Ther. 2020 May 25;3(2):32-36. doi: 10.31547/bct-2019-018.
Stem cell transplantation is the cornerstone of therapy for transplant-eligible patients with severe aplastic anemia.
Patients with severe aplastic anemia undergoing stem cell transplantation (including matched haplo-identical related donors) with a standard conditioning regimen and graft-versus-host disease (GVHD) prophylaxis were analyzed. High-risk patients were identified as having undergone >20 pre-transplant transfusions, having febrile neutropenia at the time of transplantation, or having undergone failed immunosuppressive therapy.
A total of 111 patients underwent stem cell transplantation, with a median age of 17 years. Seventy-six patients received matched related donor (MRD) transplants, and 35 received haplo-identical donor (HID) transplants. Among all patients, 65.7% were high-risk patients, with a significantly higher proportion among those receiving HID transplants (38% for MRD vs. 83% for HID). Acute GVHD grades 2-4 was observed in 9% of patients, and chronic GVHD in 16.2% of patients. Primary graft rejection was more common in 9.9% of patients (21% for HID, 5% for MRD). The 2-year overall survival and disease-free survival were 67% and 66%, respectively, with better outcomes for MRD and low-risk HID transplants than for high-risk HID transplants. The most common cause of mortality was sepsis-related death (accounting for 27% of the total deaths). Sepsis-related early deaths were significantly more common among high-risk patients who received HID transplants.
We conclude that MRDs remain the preferred donor source for allogeneic stem cell transplants in patients with aplastic anemia; however, HIDs can be considered as a life-saving treatment for patients with aplastic anemia.
干细胞移植是适合移植的重型再生障碍性贫血患者治疗的基石。
对接受标准预处理方案和预防移植物抗宿主病（GVHD）的重型再生障碍性贫血患者进行干细胞移植（包括单倍体相合相关供者）分析。高危患者被定义为移植前输血超过20次、移植时发生发热性中性粒细胞减少或免疫抑制治疗失败。
共有111例患者接受干细胞移植，中位年龄17岁。76例患者接受了相合相关供者（MRD）移植，35例接受了单倍体相合供者（HID）移植。所有患者中，65.7%为高危患者，接受HID移植的患者中这一比例显著更高（MRD为38%，HID为83%）。9%的患者发生2-4级急性GVHD，16.2%的患者发生慢性GVHD。9.9%的患者发生原发性移植物排斥（HID为21%，MRD为5%）。2年总生存率和无病生存率分别为67%和66%，MRD和低危HID移植的结局优于高危HID移植。最常见的死亡原因是脓毒症相关死亡（占总死亡人数的27%）。脓毒症相关早期死亡在接受HID移植的高危患者中显著更常见。
我们得出结论，MRD仍然是再生障碍性贫血患者异基因干细胞移植的首选供者来源；然而，HID可被视为再生障碍性贫血患者的一种挽救生命的治疗方法。
