杜氏肌营养不良症的临床研究。一种基于自然病程对照的治疗试验方法。

Clinical investigation of Duchenne muscular dystrophy. A methodology for therapeutic trials based on natural history controls.

作者信息

Mendell J R, Province M A, Moxley R T, Griggs R C, Brooke M H, Fenichel G M, Miller J P, Kaiser K K, King W, Robison J

出版信息

Arch Neurol. 1987 Aug;44(8):808-11. doi: 10.1001/archneur.1987.00520200012009.

DOI:10.1001/archneur.1987.00520200012009

Abstract

Between 1979 and 1987 we documented the natural history of Duchenne muscular dystrophy in 170 patients, aged from 3 to 23 years, by making serial measurements in over 5000 individual evaluations. This database makes it possible to design and conduct therapeutic trials using natural history controls. Such trials do not replace the need for randomized placebo-controlled trials of promising agents but they do require fewer patients, are cost-effective, and permit the use of high-risk therapy where toxicity monitoring may be important. Natural history-controlled trials, therefore, may serve as a screening method for new therapeutic agents. Drugs showing a significant benefit can then be evaluated in a randomized controlled trial.

摘要

1979年至1987年间，我们通过对170名年龄在3至23岁的杜氏肌营养不良症患者进行5000多次个体评估的系列测量，记录了该病的自然史。该数据库使得利用自然史对照来设计和开展治疗试验成为可能。此类试验并不能取代对有前景药物进行随机安慰剂对照试验的必要性，但它们所需患者较少，具有成本效益，并且在毒性监测可能很重要的情况下允许使用高风险疗法。因此，自然史对照试验可作为新治疗药物的筛选方法。然后可以在随机对照试验中对显示出显著疗效的药物进行评估。