Department of Rheumatology, Renji Hospital, Shanghai Jiao Tong University School of Medicine, 145 Shandong C Rd, Shanghai 200001, China.
Clin Rheumatol. 2012 Sep;31(9):1395-400. doi: 10.1007/s10067-012-2032-2. Epub 2012 Aug 9.
Systemic sclerosis (SSc) is a progressive fibrotic disorder with no legitimate effective treatment. Several clinical trials had investigated imatinib mesylate with a target dose of 400∼600 mg/day on SSc, and the efficacy is controversial with a generally poor tolerability. We herein reported six female Chinese patients with SSc administered with low-dose imatinib (200 mg/day) for a median of 23 months (10∼30 months). Patients displayed a decreased modified Rodman skin scores (mRSS) by a mean of 6.29 points after 6 months of treatment. Three patients who completed 2 years of treatment achieved a reduction of mRSS by 8, 18, and 30.5 points, respectively. Pulmonary function was improved or stabilized in two patients with interstitial lung disease. Severe gastrointestinal involvement in one patient was attenuated in terms of discontinuation of total parenteral nutrition and restoration of the serum albumin level. Imatinib was well-tolerated in general, although there were two severe adverse events: a bone fracture and a cerebral hemorrhage in two individuals. Both the adverse events were probably not directly related to imatinib and were recovered uneventfully. Our limited data, along with the review of the literature, suggested that low-dose imatinib might be effective and better tolerated in severe SSc that deserves further study.
系统性硬化症（SSc）是一种进行性纤维化疾病，目前尚无合法有效的治疗方法。多项临床试验已经研究了甲磺酸伊马替尼治疗 SSc，目标剂量为 400∼600mg/天，但疗效存在争议，通常耐受性较差。我们在此报告了 6 例接受低剂量伊马替尼（200mg/天）治疗的 SSc 中国女性患者，中位治疗时间为 23 个月（10∼30 个月）。治疗 6 个月后，患者的改良 Rodman 皮肤评分（mRSS）平均降低了 6.29 分。3 例完成 2 年治疗的患者 mRSS 分别降低了 8、18 和 30.5 分。2 例间质性肺病患者的肺功能得到改善或稳定。1 例严重胃肠道受累患者的总胃肠外营养停止，血清白蛋白水平恢复正常，病情得到缓解。伊马替尼总体耐受性良好，尽管有 2 例严重不良事件：2 例患者分别发生骨折和脑出血。这两起不良事件可能与伊马替尼无关，均已顺利恢复。我们的有限数据以及文献复习表明，低剂量伊马替尼可能对严重 SSc 有效且耐受性更好，值得进一步研究。
