A proposed objective way to assess results of randomized prospective clinical trials with acute graft-versus-host disease as an outcome of interest
A proposed objective way to assess results of randomized prospective clinical trials with acute graft-versus-host disease as an outcome of interest
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DOI：
10.1046/j.1365-2141.2001.02706.x
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发表时间：
2001-05-01
影响因子：
6.5
通讯作者：
Martin, PJ
中科院分区：
文献类型：
--
作者：
Al-Ghamdi, H;Leisenring, W;Martin, PJ
作为临床试验终点的移植物抗宿主病(GVHD)的评估需要主观判断，以区分GVHD引起的发病率和其他并发症引起的发病率。我们开发了一种基于顺序回归的方法，用于纵向评估涉及皮肤、肝脏和肠道的发病率，无论原因如何，作为GVHD感兴趣的随机预期第三阶段治疗或预防试验的客观终点。这种方法在治疗研究中得到了验证，结果表明，在II-IV级GVHD患者中，发病率比那些没有GVHD的患者更严重。在一项随机的III期临床试验中，我们发现30名接受外周血干细胞移植的患者和37名接受骨髓移植的患者在被诊断为GVHD后，皮肤、肝脏和肠道的发病率没有差异。这些初步结果表明，客观终点可用于GVHD治疗的随机临床试验。还需要进一步的研究来确定类似的方法是否可以用于预防移植物抗宿主病的随机临床试验。
The assessment of graft-versus-host disease (GVHD) as an end-point in clinical trials requires subjective judgement to distinguish morbidity caused by GVHD from morbidity caused by other complications. We developed a method based on ordinal regression for longitudinal assessment of morbidity involving the skin, liver and gut, regardless of cause as an objective end-point in randomized prospective phase III treatment or prevention trials for which GVHD is an outcome of interest. This method was validated for treatment studies by showing that morbidity was more severe among patients with grade II-IV GVHD than among those who did not have GVHD. We found no differences in morbidity involving the skin, liver and gut after the diagnosis of GVHD in a group of 30 patients who received peripheral blood stem cells and a group of 37 who received marrow in a randomized phase III clinical trial. These preliminary results suggest that objective end-points could be used in randomized clinical trials for treatment of GVHD. Further studies will be needed to determine if similar methods could be used in randomized clinical trials for prevention of GVHD.