Joffe M M, Love L A, Leff R L, Fraser D D, Targoff I N, Hicks J E, Plotz P H, Miller F W
National Institute of Arthritis and Musculoskeletal and Skin Diseases, National Institutes of Health, Bethesda, Maryland.
Am J Med. 1993 Apr;94(4):379-87. doi: 10.1016/0002-9343(93)90148-i.
To identify factors associated with responses to treatment with prednisone, methotrexate, or azathioprine in patients with idiopathic inflammatory myopathy, and to compare the efficacy of these drugs.
Data were collected on 113 adult patients meeting criteria for definite idiopathic inflammatory myopathy in this retrospective cohort study. Patients were categorized as responding completely, partially, or not at all to each therapeutic trial based upon clinical and laboratory criteria.
Clinical group, presence of certain myositis-specific autoantibodies, and time from disease onset to diagnosis influenced rates of complete clinical response to these therapeutic agents. Patients with inclusion body myositis responded comparatively poorly to prednisone and the other drugs: 43% had no clinical response to prednisone and none responded completely to any medication. Patients with autoantibodies to aminoacyl-tRNA synthetases or to signal recognition particle proteins were likely to respond partially, but not completely, to prednisone. No patient with a long delay to diagnosis (greater than 18 months) responded completely, compared with 34% of those with a short delay (less than 3 months). A patient's response to the first course of prednisone predicted subsequent responses to prednisone and to azathioprine better than response to methotrexate. Men responded to methotrexate better than women. Among certain subgroups of patients, responses to methotrexate were better than to either azathioprine or retreatment with prednisone.
Determining the clinical group, autoantibody status, and time from disease onset to diagnosis of patients with myositis provides useful information in predicting clinical responses to therapy, and these factors should be considered in designing future therapeutic trials. Methotrexate therapy may be superior to either azathioprine or further steroid treatment alone in certain patients who do not respond completely to an initial adequate course of prednisone.
确定与特发性炎性肌病患者对泼尼松、甲氨蝶呤或硫唑嘌呤治疗反应相关的因素，并比较这些药物的疗效。
在这项回顾性队列研究中，收集了113例符合确诊特发性炎性肌病标准的成年患者的数据。根据临床和实验室标准，将患者在每次治疗试验中的反应分为完全缓解、部分缓解或无缓解。
临床分组、某些肌炎特异性自身抗体的存在以及从疾病发作到诊断的时间影响了对这些治疗药物的完全临床缓解率。包涵体肌炎患者对泼尼松和其他药物的反应相对较差：43%的患者对泼尼松无临床反应，且无一例对任何药物完全缓解。抗氨酰tRNA合成酶或信号识别颗粒蛋白自身抗体阳性的患者可能对泼尼松有部分反应，但非完全反应。诊断延迟较长（超过18个月）的患者无完全缓解者，而诊断延迟较短（少于3个月）的患者完全缓解率为34%。患者对泼尼松首个疗程的反应比其对甲氨蝶呤的反应更能预测其随后对泼尼松和硫唑嘌呤的反应。男性对甲氨蝶呤的反应优于女性。在某些患者亚组中，对甲氨蝶呤的反应优于对硫唑嘌呤或泼尼松再治疗的反应。
确定肌炎患者的临床分组、自身抗体状态以及从疾病发作到诊断的时间，可为预测治疗的临床反应提供有用信息，在设计未来治疗试验时应考虑这些因素。对于初始足量泼尼松疗程治疗未完全缓解的某些患者，甲氨蝶呤治疗可能优于硫唑嘌呤或单纯进一步的类固醇治疗。
