Joint Rheumatology Program, 1st Department of Propedeutic Internal Medicine-Rheumatology Unit, National and Kapodistrian University of Athens, School of Medicine, Laikon General Hospital, 17 Agiou Thoma str., 115 27, Athens, Greece.
Rheumatology Clinic, Department of Internal Medicine, Medical School, University of Ioannina, Ioannina, Greece.
Arthritis Res Ther. 2020 Mar 23;22(1):56. doi: 10.1186/s13075-020-2140-3.
European data indicate that systemic sclerosis (SSc)-related death rates are increasing, thus raising concerns about SSc's optimal management. Herein, we describe current treatment modalities and drug survival in a real-life SSc cohort.
Details on immunosuppressive/antiproliferative (methotrexate, mycophenolate, cyclophosphamide, azathioprine, rituximab, tocilizumab) and vasoactive agent [(endothelin receptor antagonists (ERAs), sildenafil, iloprost, and calcium channel blockers (CCB)] administration during the disease course (11.8 ± 8.4 years, mean + SD) of 497 consecutive patients examined between 2016 and 2018 were retrospectively recorded. Drug survival was assessed by Kaplan-Meier analysis.
Methotrexate was the most frequently administered immunosuppressive/antiproliferative agent (53% of patients), followed by cyclophosphamide (26%), mycophenolate (12%), and azathioprine (11%). Regarding vasoactive agents, CCB had been ever administered in 68%, ERAs in 38%, iloprost in 7%, and sildenafil in 7% of patients; 23% of patients with pulmonary fibrosis had never received immunosuppressive/antiproliferative agents, 33% of those with digital ulcers had never received ERAs, iloprost, or sildenafil, whereas 19% of all patients had never received either immunosuppressive/antiproliferative or other than CCB vasoactive agents. Survival rates of methotrexate, cyclophosphamide, and mycophenolate differed significantly, being 84/75%, 59/43%, and 74/63% at 12/24 months, respectively, with inefficacy being the most frequent discontinuation cause. Conversely, CCB, ERAs, and sildenafil had high and comparable retention rates of 97/91%, 88/86%, and 80/80%, respectively.
Existing therapeutic limitations indicate that more evidence-based treatment is warranted for successful management of SSc. Vasculopathy seems to be managed more rigorously, but the low retention rates of immunosuppressive/antiproliferative drugs suggest that effective and targeted disease-modifying agents are warranted.
欧洲数据表明，系统性硬化症（SSc）相关死亡率正在上升，这引发了人们对 SSc 最佳治疗方法的担忧。在此，我们描述了真实 SSc 患者队列中的当前治疗方法和药物生存情况。
回顾性记录了 2016 年至 2018 年间检查的 497 例连续患者在疾病过程中（11.8±8.4 年，平均值+标准差）接受免疫抑制/抗增殖药物（甲氨蝶呤、霉酚酸酯、环磷酰胺、硫唑嘌呤、利妥昔单抗、托珠单抗）和血管活性剂[内皮素受体拮抗剂（ERAs）、西地那非、伊洛前列素和钙通道阻滞剂（CCB）]的使用情况。通过 Kaplan-Meier 分析评估药物生存情况。
甲氨蝶呤是最常使用的免疫抑制/抗增殖药物（53%的患者），其次是环磷酰胺（26%）、霉酚酸酯（12%）和硫唑嘌呤（11%）。在血管活性剂方面，CCB 曾被 68%的患者使用，ERAs 被 38%的患者使用，伊洛前列素被 7%的患者使用，西地那非被 7%的患者使用；23%的肺纤维化患者从未接受过免疫抑制/抗增殖药物治疗，33%的手指溃疡患者从未接受过 ERAs、伊洛前列素或西地那非治疗，而 19%的所有患者从未接受过免疫抑制/抗增殖药物或除 CCB 以外的血管活性剂治疗。甲氨蝶呤、环磷酰胺和霉酚酸酯的生存率差异显著，分别为 12/24 个月时的 84/75%、59/43%和 74/63%，药物无效是最常见的停药原因。相反，CCB、ERAs 和西地那非的保留率较高且相似，分别为 97/91%、88/86%和 80/80%。
现有治疗方法的局限性表明，需要更多基于证据的治疗方法来成功管理 SSc。血管病变的治疗似乎更加严格，但免疫抑制/抗增殖药物的保留率较低表明，需要有效的靶向疾病修饰药物。
