Noseworthy J H, Vandervoort M K, Hopkins M, Ebers G C
Department of Clinical Neurological Sciences, University of Western Ontario, London, Canada.
Neurology. 1989 Jul;39(7):977-81. doi: 10.1212/wnl.39.7.977.
Sixty-two experienced multiple sclerosis (MS) investigators attending a research workshop were asked their opinions about clinical trial design, outcome measures, and treatment. Respondents favored repeated clinical observations of neurologic function (eg, Expanded Disability Status Scale or Ambulation Index) over the opinion of a blinded physician and changes on magnetic resonance imaging. There was agreement that stable MS should not be treated with immunosuppressives. Corticosteroids were rated the most effective treatment for recent disease activity. Total lymphoid irradiation and immunosuppressives were judged more potent for the long-term management of progressive disease. No treatment, however, received the support of more than 11% of the respondents for being of "considerable efficacy" in the long-term management of MS. Most favored a placebo-controlled design in future trials. In order to judge the acceptability of current trial design, participants were asked to act as patient surrogates and to indicate whether they would agree to participate in 4 randomized trials for which they would be eligible. The majority consented to participate in the 3 major trials currently under way, and most refused to enroll in the 1 trial that had never been initiated.
62名参加研究研讨会的经验丰富的多发性硬化症（MS）研究人员被问及他们对临床试验设计、结果指标和治疗的看法。与盲态医生的意见及磁共振成像的变化相比，受访者更倾向于对神经功能进行反复的临床观察（如扩展残疾状态量表或步行指数）。大家一致认为，稳定期MS不应使用免疫抑制剂治疗。皮质类固醇被评为近期疾病活动最有效的治疗方法。全身淋巴照射和免疫抑制剂被认为对进展性疾病的长期管理更有效。然而，在MS的长期管理中，没有一种治疗方法得到超过11%的受访者支持其具有“显著疗效”。大多数人赞成在未来试验中采用安慰剂对照设计。为了判断当前试验设计的可接受性，要求参与者充当患者代理人，并表明他们是否会同意参加4项他们符合条件的随机试验。大多数人同意参加目前正在进行的3项主要试验，并且大多数人拒绝参加1项从未启动的试验。
