Objective: To analyze the clinical efficacy of haploidentical hematopoietic stem cell transplantation (haplo-HSCT) by using parental donors on thalassemia patients.
Methods: The 13 thalassemia patients treated by haplo-HSCT using parental donors in our hospital from July 1, 2016, to July 1, 2020 were retrospectively reviewed. Hematopoiesis reconstitution, the incidence of GVHD, infections and the long-term survival of the patients were analyzed.
Results: Twelve of the 13 patients were successfully implanted, the success rate of implantation was 92.3%. The median time of neutrophil and platelet engraftment was 12.5 days (range, 9-22 days) and 21 days (range,12-34 days), respectively. One patient achieved primary graft failure. Three (25%) patients developed to acute GVHD (aGVHD) and achieved complete remission after treatment. Chronic GVHD developed in three (25%) patients, one of them was extensive and under treatment, while one patient developed to severe bacterial infection (7.7%). CMV viremia was diagnosed in two patients (15.4%). There were no patients developed to CMV disease. Three (23.1%) patients achieved EB viremia after transplantation, one of them developed to EBV-related lymphocytic proliferative disease, while there were no patients showed invasive fungal infection. At the last follow-up, all patients survived, twelve of them were free from transfusion dependency. There were no transplant-related deaths. Projected overall and thalassemia-free survival at three years was 100% and 92.3%, respectively.
Conclusion: The transplant protocol of haplo-HSCT by using parental donors in patients with thalassemia has reliable source of donors, high incidence of successful implantation and low incidence of GVHD, which can be used as an effective way to increase the source of donors in children with thalassemia.
题目: 以父母为供者的单倍体造血干细胞移植治疗地中海贫血患者的临床疗效.
目的: 探讨以父母为供者的单倍体造血干细胞移植治疗重型地中海贫血患者的临床疗效.
方法: 回顾性分析2016年7月1日至2020年7月1日于厦门大学附属中山医院完成以父母为供者的单倍体造血干细胞移植治疗重型地中海贫血患者13例，对植入情况、GVHD发生率、感染等并发症及长期生存情况进行分析.
结果: 13例患者中，12例成功植入，移植成功率92.3%，中位粒细胞植入时间为12.5（9-22）d，中位血小板植入时间为21（12-34）d，1例原发性植入失败。3例aGVHD（25%），经治疗完全缓解。3例cGVHD（25%），其中1例为广泛型，正在治疗中。1例合并重症细菌感染（7.7%），2例移植后发生CMV血症（15.4%），未发现CMV靶器官受累证据。3例发生 EB病毒血症（23.1%），其中1例移植后出现EB病毒相关性淋巴细胞增殖性疾病。未发现侵袭性真菌感染病例。至随访结束，13例患者全部存活，12例脱离输血，无移植相关死亡病例。预期3年总生存率100%，3年无地中海贫血生存率92.3.
结论: 以父母为供者的单倍体移植模式治疗地中海贫血，供体来源可靠，植入成功率高，GVHD发生率低，可作为解决地中海贫血儿童供者来源问题的有效方法.
Keywords: complication; conditioning regimen; haploidentical; stem cell transplantation; thalassemia.