Haploidentical BMT and post-transplant Cy for severe aplastic anemia: a multicenter retrospective study
Haploidentical BMT and post-transplant Cy for severe aplastic anemia: a multicenter retrospective study
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DOI：
10.1038/bmt.2015.20
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发表时间：
2015-05-01
影响因子：
4.8
通讯作者：
Hamerschlak, N.
中科院分区：
文献类型：
--
作者：
Esteves, I.;Bonfim, C.;Hamerschlak, N.
难治性严重再生障碍性贫血（SAA）患者缺乏匹配的兄弟姐妹或非亲属供体需要新的治疗方法。使用不匹配或单倍体相同的相关供体的造血干细胞移植（HSCT）在过去已经被使用，但与GVHD和死亡率的显著风险相关。最近，移植后使用环磷酰胺（Cy）已被证明是预防单倍体造血干细胞移植受体GVHD的有效策略，但大多数报道都集中在血液学恶性肿瘤患者身上。我们描述了16例接受单倍体移植的患者的结果，他们使用移植后Cy的低强度调理方案，干细胞来源是BM （N = 13）或PBSCs （N = 3）。中性粒细胞着床率为94%，血小板着床率为75%。两名患者继发移植失败，并通过另一次移植成功挽救。两名患者中有三名发展为急性GVHD，等级为2-4级。5例患者死亡，1年OS为67.1%（95%可信区间：36.5-86.4%）。在我们的小范围研究中，在复发/难治性SAA患者中，单倍体BMT移植后使用低强度调节与高植入率和低GVHD风险相关。
Patients with refractory severe aplastic anemia (SAA) who lack a matched sibling or unrelated donor need new therapeutic approaches. Hematopoietic SCT (HSCT) using mismatched or haploidentical related donors has been used in the past, but was associated with a significant risk of GVHD and mortality. Recently, the use of post-transplant cyclophosphamide (Cy) has been shown to be an effective strategy to prevent GVHD in recipients of haploidentical HSCT, but the majority of reports have focused on patients with hematology malignancies. We describe the outcome of 16 patients who underwent haploidentical transplantation using a reduced-intensity conditioning regimen with post-transplant Cy. Stem cell sources were BM (N = 13) or PBSCs (N = 3). The rate of neutrophil engraftment was 94% and of platelet engraftment was 75%. Two patients had secondary graft failure and were successfully salvaged with another transplant. Three patients developed acute GVHD being grades 2-4 in two. Five patients have died and the 1-year OS was 67.1% (95% confidence interval: 36.5-86.4%). In our small series, the use of a reduced-intensity conditioning with post-transplant Cy in haploidentical BMT was associated with high rates of engraftment and low risk of GVHD in patients with relapsed/refractory SAA.