APHERESIS IN THE TREATMENT OF POLYMYOSITIS/DERMATOMYOSITIS

血液分离术治疗多发性肌炎/皮肌炎

• 批准号: 3874316
• 负责人: S F LEITMAN
• 金额: --
• 依托单位: CLINICAL CENTER
• 依托单位国家: 美国
• 资助国家: 美国
• 起止时间: 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/3874316
• 关键词: aldehyde lyase; autoimmune disorder; azathioprine; biopsy; cyclophosphamide; dermatomyositis; drug administration rate /duration; drug adverse effect; enzyme mechanism; human subject; human therapy evaluation; leukapheresis; methotrexate; muscle disorder chemotherapy; muscle function; muscle metabolism; plasmapheresis; polymyositis; prednisone

Polymyositis and dermatomyositis are uncommon diseases of autoimmune etiology. Most patients have disease responsive to prednisone, however, approximately 20% of PM/DM patients either do not respond to steroids, respond incompletely, or have intolerable adverse effects of therapy. In these individuals, cytotoxic drugs such as azathioprine or methotrexate are often given. The rate of adverse reactions to cytotoxic agents is high, however, and a significant proportion of patients will remain nonresponsive or intolerant of therapy. A number of anecdotal reports describe improvement in symptoms of PM/DM in response to either plasma exchange or leukapheresis. In the majority of these cases, therapy with steroids and/or cytotoxic agents was given simultaneously, and the effect of apheresis could not be analyzed apart from that of other therapies. The purpose of this study was to define the role of apheresis in the treatment if PM/DM in a randomized, sham-controlled, double-blind fashion. Patients non-responsive or intolerant of high-dose prednisone were randomized to either: (1) plasma exchange, (2) leukapheresis, or (3) sham apheresis. The apheresis procedures were performed 3 times weekly for 4 weeks. The patients and the investigators assessing response to therapy were kept blinded as to the procedures performed. Patients who did not improve with apheresis were subsequently given cyclophosphamide 1.0 g/M2 IV monthly for 6 months. Forty-two patients have entered the study and the study closed to further patient accrual in June, 1990. Analyses of responses in muscle strength, CPK, aldolase, LDH, SGOT, and muscle biopsy in the 42 participants are currently in progress.

多发性肌炎和皮肌炎是少见的自身免疫性疾病 病因学然而，大多数患者患有对泼尼松有反应的疾病， 大约20%的PM/DM患者对类固醇没有反应， 反应不完全，或有无法忍受的治疗副作用。在 对于这些个体，细胞毒性药物如硫唑嘌呤或甲氨蝶呤 经常给。细胞毒性药物的不良反应率很高， 然而，很大一部分患者将保持无反应， 或者对治疗不耐受 一些轶事报道描述了PM/DM症状的改善， 对血浆置换或白细胞去除术的反应。在大多数 在这些病例中，给予类固醇和/或细胞毒性药物治疗 同时，不能单独分析单采的效果 与其他疗法的区别本研究的目的是确定 如果PM/DM在随机分组中， 假对照双盲实验 对大剂量泼尼松无反应或不耐受的患者， 随机分配至：（1）血浆置换，（2）白细胞去除术，或（3）假手术 单采。单采程序每周进行3次，持续4周。 周患者和研究者评估对治疗的反应 对所进行的程序保持盲态。无效的患者 经单采改善者随后给予环磷酰胺1.0 g/M2 IV 每月6个月。42名患者已进入研究， 研究于1990年6月关闭，以进一步招募患者。分析 肌力、CPK、醛缩酶、LDH、SGOT和肌肉活检的反应 42名参与者目前正在进行中。