Phase 3 clinical trial of steroids/mycophenolate mofetil vs steroids/placebo as therapy for acute GVHD: BMT CTN 0802
Phase 3 clinical trial of steroids/mycophenolate mofetil vs steroids/placebo as therapy for acute GVHD: BMT CTN 0802
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DOI：
10.1182/blood-2014-06-577023
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发表时间：
2014-11-20
期刊：
影响因子：
20.3
通讯作者：
Ho, Vincent T.
中科院分区：
文献类型：
--
作者：
Bolanos-Meade, Javier;Logan, Brent R.;Ho, Vincent T.
皮质类固醇是公认的急性移植物抗宿主病(GVHD)的主要治疗方法，但只有大约一半的患者看到持久的反应。血液和骨髓移植临床试验网络(BMT CTN)0802是一项3期多中心随机双盲试验，旨在测试霉酚酸酯(MMF)联合皮质类固醇作为急性GVHD的初始治疗是否优于单独使用皮质类固醇。新诊断的急性GVHD患者如果需要全身治疗，则符合条件。患者随机接受泼尼松与MMF或安慰剂的治疗。主要终点是治疗开始后第56天的急性或慢性无移植物抗宿主病生存期。在372名患者中的235名患者入选后，一项计划中的中期分析满足了第56天无移植物抗宿主病存活的无效规则：116名MMF，119名安慰剂。治疗组之间的基线特征很好地平衡，包括GVHD的分级和器官分布。56天无移植物抗宿主病生存率、12个月慢性移植物抗宿主病累积发生率、总存活率、Epstein-Barr病毒重新激活、严重的危及生命的感染、12个月后复发和生活质量相似。与单独使用皮质类固醇相比，将MMF加入皮质类固醇作为急性GVHD的初始治疗并不能提高无GVHD的存活率。这项试验在www.Clinicaltrials.gov上注册为#NCT01002742。
Corticosteroids are the accepted primary therapy for acute graft-versus-host disease (GVHD), but durable responses are seen in only about half of the patients. Blood and Marrow Transplant Clinical Trials Network (BMT CTN) 0802, a phase 3 multicenter randomized double-blinded trial, was designed to test whether mycophenolate mofetil (MMF) plus corticosteroids was superior to corticosteroids alone as initial therapy for acute GVH D. Patients with newly diagnosed acute GVHD were eligible if they required systemic therapy. Patients were randomized to receive prednisone with either MMF or placebo. The primary end point was acute or chronic GVHD-free survival at day 56 after initiation of therapy. A futility rule for GVHD-free survival at day 56 was met at a planned interim analysis after 235 patients (of 372) were enrolled: 116 MMF, 119 placebo. Baseline characteristics were well balanced between treatment groups including grade and organ distribution of GVHD. GVHD-free survival at day 56, cumulative incidence of chronic GVHD at 12 months, overall survival, Epstein-Barr virus reactivation, severe, life-threatening infections, relapse at 12 months, and quality of life were similar. The addition of MMF to corticosteroids as initial therapy for acute GVHD does not improve GVHD-free survival compared with corticosteroids alone. This trial was registered at www.clinicaltrials.gov as #NCT01002742.