中国系统性硬化症治疗现状——风湿科和皮肤科医师调查问卷  

Current status of systemic sclerosis treatment in China-a survey from rheumatologists and dermatologists

作　　者：赵力 姜莉[2] 梁敏锐 于一云 万伟国 邹和建 薛愉 ZHAO Li;JIANG Li;LIANG Minrui;YU Yiyun;WAN Weiguo;ZOU Hejian;XUE Yu(Department of Rheumatology,Huashan Hospital,Fudan University,Shanghai 200040,China;Department of Rheumatology,Linyi People’s Hospital,LinYi 276034,China;International Network of Scleroderma Clinic and Research(InSCAR),Shanghai 200040,China)

机构地区：[1]复旦大学附属华山医院风湿科,上海200040 [2]临沂市人民医院风湿科,山东临沂276034 [3]国际硬皮病临床和研究协作网,上海200040

出　　处：《内科理论与实践》2020年第4期236-244,共9页Journal of Internal Medicine Concepts & Practice

摘　　要：目的:了解中国系统性硬化症(systemic sclerosis,SSc)治疗的现状。方法:通过向风湿科和皮肤科医师发放电子问卷,收集有关SSc治疗的信息。结果:286名医师完成了问卷。74.5%的受访者在SSc治疗中使用了肾上腺糖皮质激素(glucocorticoid,GC),并支持低剂量长期维持GC使用。75.5%的受访者将环磷酰胺(cyclophosphamide,CTX)作为皮肤累及和间质性肺病(interstitial lung disease,ILD)的一线免疫抑制剂,而甲氨蝶呤(methotrexate,MTX)则被33.6%的受访者选择。38.8%受访者将静脉注射免疫球蛋白(intravenous immunoglobulin,IVIG)作为SSc-ILD中最常用的免疫疗法。13.6%的受访者将抗白介素-6(interleukin 6,IL-6)受体抗体作为SSc-ILD中生物制剂的首选。约半数(47.9%~61.2%)受访者使用靶向药物磷酸二酯酶-5抑制剂、内皮素受体拮抗剂作为一线或联合治疗药物来治疗指/趾端血管病变和肺动脉高压(pulmonary arterial hypertension,PAH)。此外,有31.5%的受访者曾尝试使用吡非尼酮治疗SSc-ILD。造血干细胞移植在皮肤受累、SSc-ILD和指/趾端血管病变方面进行了尝试,但在胃肠道(gastrointestinal,GI)受累(1.4%)和SSc-PAH(1.0%)方面尝试较少。受访者的背景对SSc-ILD、SSc-PAH、指/趾端血管病变和皮肤受累的一线免疫抑制剂的选择产生了影响,但对长期GC维持影响较小,职称比科别影响更大。结论:除了使用GC外,免疫抑制剂和靶向药物被广泛用于治疗SSc;并在难治性病例中尝试了生物制剂、IVIG和造血干细胞移植。风湿科和皮肤科医师在药物选择上存在差异。Objectives To understand the current status of systemic sclerosis(SSc)treatment in China.Methods Rheumatologists and dermatologists were sent a electronic questionnaire to collect information on SSc treatment.Results A total of 286 doctors responded the questionnaire.Majority respondents(74.5%)used glucocorticoid(GC)in management of SSc and continued the treatment for long-term GC maintenance at lower dosage.Methotrexate(MTX)and cyclophos-phamide(CTX)were adopted by 33.6%and 75.5%responders as first-line immunosuppressants for skin and interstitial lung disease(ILD).Intravenous immunoglobulin(IVIG)was the most commonly used immunotherapy by the responders(38.8%)in SSc-ILD.The anti-interleukin 6(IL-6)receptor antibody was the first choice of biologics(13.6%)in SSc-ILD.About half of the respondents(47.9%-61.2%)had used the targeted drugs(phosphodiesterase-5 inhibitor,endothelin receptor antagonists)in managing digital vasculopathy and SSc-pulmonary arterial hypertention(PAH)as a first-line or combined therapy.In addition,31.5%respondents had tried pirfenidone to manage SSc-ILD.Stem cell transplantation was tried more in skin involvement,SSc-ILD and digital vasculopathy,but less in gastrointestinal(GI)involvement and SSc-PAH.Background of respondents in department and title influenced the choice of first-line immunosuppressant more on SSc-ILD,SSc-PAH,digital vasculopathy and skin involvement in sequence,but less on long-term GC maintenance.The decision was more relevant to the clinical experience of the responder rather than their specialty.Conclusions Besides GC,the immunosuppressive and targeted treatment are widely used in manage SSc in China.Biotherapuetics,IVIG and stem cell transplantation are reserved only for refractory cases.Discrepancies in drug choices occur among rheumatologists and dermatologists.

关 键 词：系统性硬化症 肾上腺糖皮质激素 免疫抑制剂 生物制剂 干细胞移植 

分 类 号：R593.25[医药卫生—内科学]