单药治疗与联合治疗在系统性硬化症相关肺动脉高压中的长期疗效和安全性：一项回顾性RESCLE注册研究
Longterm Efficacy and Safety of Monotherapy versus Combination Therapy in Systemic Sclerosis-associated Pulmonary Arterial Hypertension: A Retrospective RESCLE Registry Study.
作者信息
Pestaña-Fernández Melani, Rubio-Rivas Manuel, Tolosa-Vilella Carles, Guillén-Del-Castillo Alfredo, Freire Mayka, Vargas-Hitos Jose Antonio, Todolí-Parra Jose Antonio, Rodríguez-Carballeira Mónica, Marín-Ballvé Adela, Espinosa Gerard, Colunga-Argüelles Dolores, Ortego-Centeno Norberto, Trapiella-Martínez Luis, Carbonell-Muñoz Cristina, Pla-Salas Xavier, Perales-Fraile Isabel, Corbella Xavier, Fonollosa-Pla Vicent, Simeón-Aznar Carmen Pilar
出版信息
J Rheumatol. 2020 Jan;47(1):89-98. doi: 10.3899/jrheum.180595. Epub 2019 Feb 15.
OBJECTIVE
Monotherapy is an option as first-line therapy for pulmonary arterial hypertension (PAH). However, combination therapy is a beneficial alternative. Our objective was to evaluate the efficacy of monotherapy versus combination therapy in patients with systemic sclerosis (SSc)-associated PAH.
METHODS
All patients with SSc-associated PAH from the Spanish Scleroderma Registry (RESCLE) were reviewed. Patients were split into 3 groups: monotherapy versus sequential combination versus upfront combination therapy. The primary endpoint was death from any cause at 1, 3, and 5 years from PAH diagnosis.
RESULTS
Seventy-six patients (4.2%) out of 1817 had SSc-related PAH. Thirty-four patients (45%) were receiving monotherapy [endothelin receptor antagonist (n = 22; 29%) or phosphodiesterase-5 inhibitors (n = 12; 16%)], 25 (33%) sequential combination, and 17 (22%) upfront combination therapy. A lower forced vital capacity/DLCO in the sequential combination group was reported (2.9 ± 1.1 vs 1.8 ± 0.4 vs 2.3 ± 0.8; p = 0.085) and also a higher mean pulmonary arterial pressure in combination groups (37.2 ± 8.7 mmHg vs 40.8 ± 8.8 vs 46 ± 15.9; p = 0.026) at baseline. Treatment regimen (p = 0.017) and functional class (p = 0.007) were found to be independent predictors of mortality. Sequential combination therapy was found to be an independent protective factor (HR 0.11, 95% CI 0.03-0.51; p = 0.004), while upfront combination therapy showed a trend (HR 0.68, 95% CI 0.23-1.97; p = 0.476). Survival from PAH diagnosis among monotherapy, sequential, and upfront combination groups was 78% versus 95.8% versus 94.1% at 1 year, 40.7% versus 81.5% versus 51.8% at 3 years, and 31.6% versus 56.5% versus 34.5% at 5 years (p = 0.007), respectively. Side effects were not significantly different among groups.
CONCLUSION
Combination sequential therapy improved survival in our cohort.
目的
单药治疗是肺动脉高压（PAH）一线治疗的一种选择。然而，联合治疗是一种有益的替代方案。我们的目的是评估单药治疗与联合治疗在系统性硬化症（SSc）相关PAH患者中的疗效。
方法
对西班牙硬皮病注册中心（RESCLE）中所有SSc相关PAH患者进行了回顾。患者被分为3组：单药治疗组、序贯联合治疗组和初始联合治疗组。主要终点是PAH诊断后1年、3年和5年的任何原因导致的死亡。
结果
1817例患者中有76例（4.2%）患有SSc相关PAH。34例患者（45%）接受单药治疗[内皮素受体拮抗剂（n = 22；29%）或磷酸二酯酶-5抑制剂（n = 12；16%）]，25例（33%）接受序贯联合治疗，17例（22%）接受初始联合治疗。序贯联合治疗组的用力肺活量/一氧化碳弥散量较低（分别为2.9±1.1、1.8±0.4和2.3±0.8；p = 0.085），联合治疗组基线时平均肺动脉压也较高（分别为37.2±8.7 mmHg、40.8±8.8和46±15.9；p = 0.026）。治疗方案（p = 0.017）和功能分级（p = 0.007）是死亡率的独立预测因素。序贯联合治疗是一个独立的保护因素（风险比0.11，95%置信区间0.03 - 0.51；p = 0.004），而初始联合治疗显示出一种趋势（风险比0.68，95%置信区间0.23 - 1.97；p = 0.476）。单药治疗组、序贯联合治疗组和初始联合治疗组从PAH诊断开始的1年生存率分别为78%、95.8%和94.1%，3年生存率分别为40.7%、81.5%和51.8%，5年生存率分别为31.6%、56.5%和34.5%（p = 0.007）。各组间副作用无显著差异。
结论
序贯联合治疗提高了我们队列中的生存率。
Zotero 插件