阵发性睡眠性血红蛋白尿症的造血干细胞移植：意大利骨髓移植组（GITMO）的一项回顾性研究的长期结果。
Hematopoietic stem cell transplantation for paroxysmal nocturnal hemoglobinuria: long-term results of a retrospective study on behalf of the Gruppo Italiano Trapianto Midollo Osseo (GITMO).
机构信息
BMT Center, Ospedale Civile, Via Fonte Romana 8, Pescara 65125, Italy.
出版信息
Haematologica. 2010 Jun;95(6):983-8. doi: 10.3324/haematol.2009.017269. Epub 2009 Dec 8.
BACKGROUND
Paroxysmal nocturnal hemoglobinuria is an acquired clonal disorder of the hemopoietic stem cells for which the only curative treatment is allogeneic hematopoietic stem cell transplantation.
DESIGN AND METHODS
The aim of this retrospective study was to assess the long-term clinical and hematologic results in 26 paroxysmal nocturnal hemoglobinuria patients who received hematopoietic stem cell transplantation in Italy between 1988 and 2006. The patients were aged 22 to 60 years (median 32 years). Twenty-three donors were HLA-identical (22 siblings and one unrelated) and 3 were HLA-mismatched (2 related and one unrelated).
RESULTS
Fifteen patients received a myeloablative conditioning consisting of busulfan and cyclophosphamide (in all cases from identical donor) and 11 were given a reduced intensity conditioning (8 from identical donor and 3 from mismatched donor). The cumulative incidence of graft failure was 8% (4% primary and 4% secondary graft failure). Transplant-related mortality for all patients was 42% (26% and 63% for patients transplanted following myeloablative or reduced intensity conditioning, respectively). As of October 31, 2009, 15 patients (11 in the myeloablative conditioning group and 4 in the reduced intensity conditioning group) are alive with complete hematologic recovery and no evidence of paroxysmal nocturnal hemoglobinuria following a median follow-up of 131 months (range 30-240). The 10-year Kaplan-Meier probability of disease-free survival was 57% for all patients: 65% for 23 patients transplanted from identical donor and 73% for 15 patients transplanted with myeloablative conditioning. No thromboembolic event nor recurrence of the disease were reported following transplant.
CONCLUSIONS
The findings of this study confirm that most patients with paroxysmal nocturnal hemoglobinuria may be definitively cured with hematopoietic stem cell transplantation.
背景
阵发性睡眠性血红蛋白尿是一种后天性造血干细胞克隆性疾病，唯一的治愈方法是异基因造血干细胞移植。
设计与方法
本回顾性研究的目的是评估 26 例在意大利于 1988 年至 2006 年期间接受造血干细胞移植的阵发性睡眠性血红蛋白尿患者的长期临床和血液学结果。患者年龄为 22 至 60 岁（中位年龄 32 岁）。23 名供者为 HLA 完全匹配（22 名亲属和 1 名无关供者），3 名供者为 HLA 不完全匹配（2 名亲属和 1 名无关供者）。
结果
15 名患者接受了以白消安和环磷酰胺为基础的清髓性预处理（均来自于完全匹配的供者），11 名患者接受了强度降低的预处理（8 名来自完全匹配的供者，3 名来自不完全匹配的供者）。移植物失败的累积发生率为 8%（4%为原发性，4%为继发性）。所有患者的移植相关死亡率为 42%（清髓性预处理组为 26%，强度降低预处理组为 63%）。截至 2009 年 10 月 31 日，15 名患者（清髓性预处理组 11 例，强度降低预处理组 4 例）在接受中位随访 131 个月（30-240 个月）后完全血液学恢复，且无阵发性睡眠性血红蛋白尿证据而存活。所有患者的 10 年无病生存率为 57%：23 名接受完全匹配供者移植的患者为 65%，15 名接受清髓性预处理的患者为 73%。移植后无血栓栓塞事件或疾病复发报告。
结论
本研究结果证实，大多数阵发性睡眠性血红蛋白尿患者可以通过造血干细胞移植得到根治。
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