Neurology Service, Department of Neurosciences, Hospital de Santa Maria, Lisbon, Portugal.
Clinical Pharmacology Unit, Instituto de Medicina Molecular, Lisbon, Portugal.
Mov Disord. 2017 Oct;32(10):1356-1364. doi: 10.1002/mds.27080. Epub 2017 Aug 7.
Multiple system atrophy (MSA) is a rare neurodegenerative disease of undetermined cause. Although many clinical trials have been conducted, there is still no treatment that cures the disease or slows its progression. We sought to assess the clinical trials, methodology, and quality of reporting of clinical trails conducted in MSA patients. We conducted a systematic review of all trials with at least 1 MSA patient subject to any pharmacological/nonpharmacological interventions. Two independent reviewers evaluated the methodological characteristics and quality of reporting of trials. A total of 60 clinical trials were identified, including 1375 MSA patients. Of the trials, 51% (n = 31) were single-arm studies. A total of 28% (n = 17) had a parallel design, half of which (n = 13) were placebo controlled. Of the studies, 8 (13.3%) were conducted in a multicenter setting, 3 of which were responsible for 49.3% (n = 678) of the total included MSA patients. The description of primary outcomes was unclear in 60% (n = 40) of trials. Only 10 (16.7%) clinical trials clearly described the randomization process. Blinding of the participants, personnel, and outcome assessments were at high risk of bias in the majority of studies. The number of dropouts/withdrawals was high (n = 326, 23.4% among the included patients). Overall, the design and quality of reporting of the reviewed studies is unsatisfactory. The most frequent clinical trials were small and single centered. Inadequate reporting was related to the information on the randomization process, sequence generation, allocation concealment, blinding of participants, and sample size calculations. Although improved during the recent years, methodological quality and trial design need to be optimized to generate more informative results. © 2017 International Parkinson and Movement Disorder Society.
多系统萎缩症（MSA）是一种病因不明的罕见神经退行性疾病。尽管已经进行了许多临床试验，但仍没有治愈该疾病或减缓其进展的治疗方法。我们旨在评估 MSA 患者进行的临床试验的方法学、临床试验设计和报告质量。我们对所有至少有 1 例 MSA 患者接受任何药物/非药物干预的试验进行了系统评价。两名独立的审查员评估了试验的方法学特征和报告质量。共确定了 60 项临床试验，其中包括 1375 例 MSA 患者。这些试验中，51%（n = 31）为单臂研究。共有 28%（n = 17）为平行设计，其中一半（n = 13）为安慰剂对照。在这些研究中，8 项（13.3%）为多中心研究，其中 3 项研究负责纳入的 MSA 患者总数的 49.3%（n = 678）。60%（n = 40）的试验中，主要结局的描述不明确。仅有 10 项（16.7%）临床试验明确描述了随机化过程。在大多数研究中，参与者、人员和结局评估的盲法存在高偏倚风险。脱落/撤回的人数很多（n = 326，占纳入患者的 23.4%）。总的来说，所审查研究的设计和报告质量不尽如人意。最常见的临床试验规模较小且为单中心。报告不充分与随机化过程、序列生成、分配隐藏、参与者盲法和样本量计算的信息有关。尽管近年来有所改善，但方法学质量和试验设计需要优化，以产生更有信息的结果。© 2017 国际帕金森病和运动障碍学会。
