同种异体人类间充质干细胞疗法(remestemcel-L Prochymal)为救援的代理在儿科患者严重难治性急性移植物抗宿主病。
文章的细节
-
引用
复制到剪贴板 -
Kurtzberg J, Prockop年代,Teira P, Bittencourt H,路易斯V,陈千瓦,角B, Yu L, Talano是的,她也是E,米尔斯CR,乔杜里年代
同种异体人类间充质干细胞疗法(remestemcel-L Prochymal)为救援的代理在儿科患者严重难治性急性移植物抗宿主病。
血液骨髓移植杂志。2014年2月,20 (2):229 - 35。doi: 10.1016 / j.bbmt.2013.11.001。Epub 2013年11月8日。
- PubMed ID
-
24216185 (在PubMed]
- 文摘
-
严重steroid-refractory急性移植物抗宿主病(aGVHD)是相关重要的同种异体干细胞移植后死亡率和发病率。早期的临床试验治疗与人类间充质干细胞(hMSCs)在小儿患者严重aGVHD耐多种免疫抑制剂表现出不错的效果。在这项研究中,我们评估的风险/收益情况remestemcel-L (Prochymal),第三方,hMSCs的现成的来源,作为难治性aGVHD的救援代理在儿科患者。年级儿童罪犯aGVHD类固醇和失败,在大多数情况下,其他免疫抑制剂是负责招生的资格。患者接受8两周一次的输液注入2 x 10 (6) hMSCs /公斤4周,与一个额外的4周后注入天+ 28的患者取得部分或混合反应。登记病人构成一个非常具有挑战性的人口患有严重疾病,停止响应的标准治疗,88%的患者出现严重aGVHD (C或D级),七十五名患者(平均年龄8年;男性58.7%;和61.3%的白种人)治疗研究。六十四名患者(85.3%)接受了一个不相关的造血干细胞移植,和28个病人(37.3%)接受脐带血移植。在基线,aGVHD的分布等级B, C, D为12.0%,28.0%,和60.0%,分别。 The median duration of aGVHD before enrollment was 30 d (range, 2 to 1639 d), and patients failed a median of 3 immunosuppressive agents. Organ involvement at baseline was 86.7% gastrointestinal, 54.7% skin, and 36.0% liver. Thirty-six patients (48.0%) had 2 organs involved, and 11 patients (14.7%) had all 3 organs involved. When stratified by aGVHD grade at baseline, the rate of overall response (complete and partial response) at day +28 was 66.7% for aGVHD grade B, 76.2% for grade C, and 53.3% for grade D. Overall response for individual organs at day +28 was 58.5% for the gastrointestinal system, 75.6% for skin, and 44.4% for liver. Collectively, overall response at day +28 for patients treated for severe refractory aGVHD was 61.3%, and this response was correlated with statistically significant improved survival at day +100 after hMSC infusion. Patients who responded to therapy by day +28 had a higher Kaplan-Meier estimated probability of 100-d survival compared with patients who did not respond (78.1% versus 31.0%; P < .001). Prochymal infusions were generally well tolerated, with no evidence of ectopic tissue formation.
DrugBank数据引用了这篇文章
- 药物