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氟达拉滨、环磷酰胺和抗胸腺细胞球蛋白用于严重再生障碍性贫血的匹配相关和无关异体干细胞移植。

Fludarabine, cyclophosphamide, and antithymocyte globulin for matched related and unrelated allogeneic stem cell transplant in severe aplastic anemia.

出版信息

Leuk Lymphoma. 2011 Jan;52(1):137-41. doi: 10.3109/10428194.2010.524328. Epub 2010 Oct 12.

DOI:10.3109/10428194.2010.524328
PMID:20939697
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC4238067/
Abstract

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.

摘要

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。

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本文引用的文献

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