6月26日，吴德沛教授课题组在《Leukemia& Lymphoma》发表题为“Haploidentical hematopoietic stem celltransplant in paroxysmal nocturnal hemoglobinuria”的研究论文，总结半相合移植治疗阵发性睡眠性血红蛋白尿。吴德沛教授为该文的通讯作者。

阵发性睡眠性血红蛋白尿（PNH）是一种少见的，以反复发生溶血性贫血、易于形成血栓和血细胞减少为特征的疾病，亚洲人群易并发骨髓衰竭的症状。2007年起美国和欧洲率先采用Eculizumab治疗PNH，使PNH的治疗进人了补体抑制剂时代，可是靶向治疗并不能对所有患者起效果，而且Eculizumab并不能改善PNH患者的骨髓衰竭，而且考虑到经济、疗效等各方面因素，对于难治的PNH患者行异基因造血干细胞移植是一个上佳选择。吴教授课题组总结了难治性高危组PNH患者行半相合移植的资料，结果肯定了半相合移植的疗效，比较发现半相合患者与全相合患者移植后长期生存相似，同时，还指出移植前检测HLA抗体对预防移植后并发症有一定的意义。

原文摘要：Eighteenpatients with paroxysmal nocturnal hemoglobinuria (PNH) receiving allogeneichematopoietic stem cell transplant (allo-HSCT), either from HLA-haploidenticaldonors (HRD; n?=?10) or HLA-matched donors (n?=?5 from siblings and n?=?3 fromunrelated donors), were retrospectively evaluated. One showed primary graftfailure following unrelated-donor HSCT. He was given a second HRD-HSCT, butdied from cytomegalovirus pneumonia after achieving hematopoietic recovery. Theother 17 patients achieved sustained engraftment and full-donor chimerism. Fourin the HRD-HSCT group experienced grade II/III acute graft-versus-host disease(aGVHD), and five in the HLA-matched HSCT group developed grade II aGVHD. Amongall 18 patients, 10 developed chronic GVHD (cGVHD), only one patient receivingHRD-HSCT developed extensive cGVHD. Nine in the HRD-HSCT group and all those inthe HLA-matched HSCT group were alive and transfusion-independent at lastfollow-up. Our findings suggest that allo-HSCT is a promising treatment forPNH, and HRD-HSCT is a viable option for patients with PNH who lack HLA-matcheddonors.

原文链接：http://dx.doi.org/10.3109/10428194.2015.1068309