Choy E H S, Isenberg D A
Academic Department of Rheumatology, Division of Medicine, Guy's, King's and St Thomas' Hospitals School of Medicine, King's College Hospital (Dulwich), East Dulwich Grove, London SE22 8PT, UK.
Rheumatology (Oxford). 2002 Jan;41(1):7-13. doi: 10.1093/rheumatology/41.1.7.
Since idiopathic inflammatory myositis is relatively uncommon, randomized placebo controlled trials are rare. Although corticosteroids have not been tested in randomized controlled trials, general clinical consensus among physicians has accepted it as effective therapy. However, corticosteroid toxicity leads to significant disability in many patients. For patients with refractory dermatomyositis, intravenous immunoglobulin is an effective short-term treatment but its long-term effect remains unknown. Immunosuppressants are commonly used in refractory inflammatory myositis; evidence for their efficacy, with very few exceptions, has been derived from case reports and open studies with small numbers of patients. Even in randomized trials, the lack of validated and generally accepted outcome measures makes it difficult to compare the effect of interventions in different studies. Although the balance of evidence suggests that immunosuppressants are equally effective in dermatomyositis and polymyositis, there are no randomized controlled trials to show if any of these drugs, individually or in combination, is best. For uncommon diseases, such as inflammatory myositis, only multicentre randomized controlled trials involving rheumatologists and neurologists will define the optimal therapy.
由于特发性炎性肌病相对不常见,随机安慰剂对照试验很少见。尽管皮质类固醇尚未在随机对照试验中进行测试,但医生之间的普遍临床共识已将其接受为有效治疗方法。然而,皮质类固醇毒性在许多患者中导致严重残疾。对于难治性皮肌炎患者,静脉注射免疫球蛋白是一种有效的短期治疗方法,但其长期效果尚不清楚。免疫抑制剂常用于难治性炎性肌病;除极少数例外,其疗效证据均来自病例报告和少量患者的开放性研究。即使在随机试验中,缺乏经过验证且普遍接受的结局指标也使得难以比较不同研究中干预措施的效果。尽管证据平衡表明免疫抑制剂在皮肌炎和多发性肌炎中同样有效,但尚无随机对照试验表明这些药物中的任何一种单独使用或联合使用是否最佳。对于炎性肌病等罕见疾病,只有涉及风湿病学家和神经科医生的多中心随机对照试验才能确定最佳治疗方法。