Sirolimus and mycophenolate mofetil as GVHD prophylaxis in myeloablative, matched-related donor hematopoietic cell transplantation
Sirolimus and mycophenolate mofetil as GVHD prophylaxis in myeloablative, matched-related donor hematopoietic cell transplantation
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DOI：
10.1038/bmt.2011.104
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发表时间：
2012-04-01
影响因子：
4.8
通讯作者：
Negrin, R.
中科院分区：
文献类型：
--
作者：
Johnston, L.;Florek, M.;Negrin, R.
我们研究了西罗莫司和霉酚酸酯（MMF）作为GVHD预防晚期恶性血液病患者接受清髓性造血细胞移植（HCT）从HLA相同的同胞供体。根据研究前停止规则，在入组11例成人患者后，试验停止入组。11例患者中有7例接受了含BU的准备方案。3例患者因严重窦阻塞综合征、门静脉血栓形成、精神状态改变等毒性相关事件而停用西罗莫司，1例患者因伤口愈合不良风险而停用西罗莫司。总之，11例患者中有6例在HCT后中位数15.5天发生了II-IV级急性GVHD（AGVHD）。2/3例IV级AGVHD患者在HCT后9天停用西罗莫司。所有患者均对AGVHD治疗有反应，无GVHD相关死亡。有2例非复发和2例复发相关死亡。在中位随访38个月（2-47个月）时，11例患者中有7例存活，无疾病。MMF和西罗莫司预防GVHD并不能降低AGVHD的风险，但是，没有GVHD相关的死亡。接受含BU的准备方案的患者中的严重毒性限制了西罗莫司和MMF用于预防AGVHD的继续使用。骨髓移植（2012）47，581-588; doi：10.1038/bmt.2011.104; 2011年5月9日在线发表
We investigated sirolimus and mycophenolate mofetil (MMF) as GVHD prophylaxis in patients with advanced hematological malignancies receiving myeloablative hematopoietic cell transplantation (HCT) from HLA-identical sibling donors. On the basis of pre-study stopping rules, the trial was closed to accrual after enrollment of 11 adult patients. In all, 7 of the 11 patients received BU-containing preparative regimens. Sirolimus was discontinued in three patients because of the toxicity-related events of severe sinusoidal obstructive syndrome, portal vein thrombosis, altered mental status and in one patient because of the risk of poor wound healing. In all, 6 of the 11 patients developed grade II-IV acute GVHD (AGVHD) a median of 15.5 days post HCT. Two of three patients with grade IV AGVHD had sirolimus discontinued by 9 days post HCT. All patients responded to AGVHD therapy without GVHD-related deaths. There were two non-relapse-and two relapse-related deaths. At a median follow-up of 38 months (2-47 months), 7 of 11 patients were alive without disease. MMF and sirolimus GVHD prophylaxis did not reduce the risk of AGVHD, however, there were no GVHD-related deaths. The severe toxicities in the patients receiving the BU-containing preparative regimens limited the continued use of sirolimus and MMF for the prevention of AGVHD. Bone Marrow Transplantation (2012) 47, 581-588; doi:10.1038/bmt.2011.104; published online 9 May 2011