迷你剂量甲状腺素与甲基普雷迪尼索隆结合用于急性GVHD的初始治疗:一个多中心的随机试验
Yu Wang1, Qi-Fa Liu2, De-Pei Wu3
1Peking University People's Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Collaborative Innovation Center of Hematology, Peking University, Beijing, China.
BMC medicine
|April 25, 2024
概括
在干细胞移植后,将微剂量甲醇 (MTX) 添加到皮质类固醇显著改善了急性移植对宿主疾病 (aGVHD) 的整体反应率. 这种组合疗法耐受性良好,并且在aGVHD患者中改善了无失败生存率.
科学领域:
- 血液学 血液学 血液学
- 免疫学 免疫学 免疫学
- 在瘤学瘤学.
背景情况:
- 急性移植对宿主疾病 (aGVHD) 呈现出超出标准皮质类固醇治疗后的有效初始治疗的未满足需求,即异构血造干细胞移植 (allo-HSCT).
- 开发新的治疗策略对于改善alo-HSCT患者的治疗结果至关重要.
研究的目的:
- 为了评估在aGVHD的初始治疗中添加微剂量甲状腺 (MTX) 加入标准皮质类固醇治疗的疗效和安全性.
- 为了确定这种组合治疗是否与单独使用皮质类固醇相比,改善整体反应率 (ORR) 和无失败生存率.
主要方法:
- 一项多中心的,开放的,随机的第3期研究招募了157名15岁或以上的157名患有GVHD后合并HSCT的患者,这些患者之前没有接受过aGVHD治疗.
- 患者被随机分配1:1接受单独皮质类固醇或皮质类固醇与小剂量MTX (5 mg/m2在第1,3和第8天) 结合.
- 主要终点是第10天的整体反应率 (ORR).
主要成果:
- 10日ORR在MTX加上皮质固醇组 (97%) 与皮质固醇单独组 (81%) 相比显著更高 (p=0.005).
- 在患有轻度aGVHD的患者中,10日ORR为100%的MTX加上皮质类固醇,与仅使用皮质类固醇的86%相比 (p=0.001).
- 据估计,MTX组的1年无故障生存率为69%,对照组为41% (p=0.002),治疗相关不良事件没有显著差异.
结论:
- 迷你剂量MTX与皮质类固醇结合显著改善ORR和aGVHD患者的无故障生存率,并且耐受性良好.
- 虽然没有达到预规定的20%的改善目标,但MTX的添加为初始aGVHD管理提供了有前途的治疗选择.
- 这种组合疗法代表了对aGVHD后合金-HSCT治疗武器库的宝贵补充.
相关概念视频
01:22Bone Marrow Sampling and Transplants
326
Bone marrow transplant is a potential cure for several diseases, including cancer and specific genetic disorders. Notably, this procedure is applicable for patients suffering from aplastic anemia, certain types of leukemia, severe combined immunodeficiency disease (SCID), Hodgkin's disease, non-Hodgkin's lymphoma, multiple myeloma, thalassemia, sickle-cell disease, and certain cancers.
The transplant begins with high doses of chemotherapy and radiation treatment, which aim to destroy...
The transplant begins with high doses of chemotherapy and radiation treatment, which aim to destroy...
326
01:29Drugs for Treatment of Crohn's Disease in IBD Using Immunomodulatory Agents
160
Crohn's disease is an inflammatory bowel disorder marked by chronic inflammation of the GI tract. Various treatment strategies for Crohn's disease are employed, such as immunomodulatory agents, glucocorticoids, and biologics or anti-TNF therapy. Azathioprine (Imuran), a commonly used immunomodulatory drug for Crohn's disease, is converted in the body to mercaptopurine, which inhibits purine biosynthesis and cell proliferation. Both are utilized in severe cases of Inflammatory Bowel...
160