Leuk Lymphoma. 2011 Jan;52(1):137-41. doi: 10.3109/10428194.2010.524328. Epub 2010 Oct 12.
Twenty severe aplastic anemia (SAA) patients underwent allogeneic stem cell transplantation (allo-SCT) with fludarabine (FLU), cyclophosphamide and antithymocyte globulin from a matched related (n=7, age ≥ 40) or unrelated donor (n=13, any age). Median age was 34 years (range 1–59). Median time from diagnosis to allo-SCT was 12 months (range 2–244). Seventeen out of 19 evaluable patients engrafted (90%). There were two secondary graft failures (10%). Median time to neutrophil recovery was 15 days (range 8–30). Chimerism studies indicated ≥90% donor-derived engraftment in 16/19 evaluable patients (75%). Four out of 20 patients (20%) developed acute (grade II–IV) GVHD, and 6/16 evaluable patients (37%) developed chronic GVHD. We observed EBV reactivation and viremia in seven patients, which was successfully treated with rituximab in all but one instance (where it was self-limiting). Thirteen patients (62%) are alive (including eight of the last nine treated) with a median follow-up of 30 months (range 3–112). Seven patients expired (graft rejection n=1, GVHD n=1, multiorgan failure n=1, infection n=2, EBV post-transplant lymphoproliferative disorder/PTLD n=2). Total body irradiation-free, FLU-based conditioning for matched related and unrelated allo-SCT is feasible with high engraftment rates. EBV PTLD remains a drawback of this approach.
20 例重型再生障碍性贫血(SAA)患者接受了亲缘全相合(n=7,年龄≥40 岁)或无关供者(n=13,任何年龄)来源的氟达拉滨(FLU)、环磷酰胺和抗胸腺细胞球蛋白的异基因造血干细胞移植(allo-SCT)。中位年龄为 34 岁(范围 1-59 岁)。从诊断到 allo-SCT 的中位时间为 12 个月(范围 2-244 天)。19 例可评估患者中 17 例(90%)植活。有 2 例(10%)二次移植物失败。中性粒细胞恢复的中位时间为 15 天(范围 8-30 天)。嵌合状态研究表明,16 例可评估患者中有 16 例(75%)获得了≥90%的供者嵌合。20 例患者中有 4 例(20%)发生了急性(Ⅱ-Ⅳ级)GVHD,16 例可评估患者中有 6 例(37%)发生了慢性 GVHD。我们观察到 7 例患者发生 EBV 再激活和病毒血症,除 1 例外(为自限性),所有患者均成功接受利妥昔单抗治疗。13 例患者(62%)存活(包括最后 9 例治疗中的 8 例),中位随访时间为 30 个月(范围 3-112 个月)。7 例患者死亡(移植物排斥 1 例,GVHD 1 例,多器官衰竭 1 例,感染 2 例,EBV 移植后淋巴增殖性疾病/PTLD 2 例)。无全身照射、基于 FLU 的亲缘全相合和无关 allo-SCT 预处理方案是可行的,具有较高的植活率。EBV PTLD 仍然是该方法的一个缺点。