Outcome of allogeneic hematopoietic stem cell transplantation in adult patients with paroxysmal nocturnal hemoglobinuria
Outcome of allogeneic hematopoietic stem cell transplantation in adult patients with paroxysmal nocturnal hemoglobinuria
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DOI：
10.1007/s12185-020-02982-y
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发表时间：
2020-09-05
影响因子：
2.1
通讯作者：
Mori, Takehiko
中科院分区：
文献类型：
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作者：
Nakamura, Yukinori;Takenaka, Katsuto;Mori, Takehiko
异基因造血干细胞移植（HSCT）治疗阵发性睡眠性血红蛋白尿（PNH）的安全性和有效性仍不清楚。因此，我们利用日本造血细胞移植学会的注册数据库回顾性分析了 42 例接受同种异体 HSCT 的成人 PNH 患者的结果。患者中位年龄为 32.5 岁。 19 名患者的浓缩红细胞 (PRC) 输注次数 < 20 次，16 名患者的输注次数 >= 20 次； 7 名患者数据缺失。干细胞来源是来自相关捐赠者的骨髓（N = 15）或外周血（N = 13）或来自无关捐赠者的骨髓（N = 11）和脐带血（N = 3）。第 40 天时中性粒细胞植入的累积发生率为 81%。 6 名患者在移植前死亡，6 年总生存率 (OS) 为 74%。移植前 PRC 输血次数 < 20 次的患者的 OS 显着高于移植前 PRC 输血次数 >= 20 次的患者（95% vs. 63%；P < 0.05）。此外，年龄< 30岁患者的OS显着高于年龄>= 30岁患者（90% vs. 59%；P < 0.05）。 PNH 的同种异体 HSCT 可以提供良好的生存率；然而，在决定同种异体造血干细胞移植的时机时，应考虑移植前的输血负担和患者年龄。
The safety and efficacy of allogeneic hematopoietic stem cell transplantation (HSCT) for paroxysmal nocturnal hemoglobinuria (PNH) remain unclear. Therefore, we retrospectively analyzed the outcomes of 42 adult patients with PNH who underwent allogeneic HSCT using the registry database of the Japan Society for Hematopoietic Cell Transplantation. The median patient age was 32.5 years. The number of packed red cell (PRC) transfusions was < 20 times in 19 patients and >= 20 times in 16; 7 patients had missing data. Stem cell sources were bone marrow (N = 15) or peripheral blood (N = 13) from a related donor or bone marrow (N = 11) and cord blood (N = 3) from an unrelated donor. The cumulative incidence of neutrophil engraftment at day 40 was 81%. Six patients died before engraftment, and the 6-year overall survival (OS) was 74%. The OS of patients with < 20 pretransplant PRC transfusions was significantly higher than that of patients with >= 20 pretransplant PRC transfusions (95% vs. 63%;P < 0.05). Moreover, the OS of patients aged < 30 years was significantly higher than that of patients aged >= 30 years (90% vs. 59%;P < 0.05). Allogeneic HSCT for PNH could provide favorable survival; however, pretransplant transfusion burden and patient age should be considered when deciding the timing of allogeneic HSCT.