Longterm Efficacy and Safety of Monotherapy versus Combination Therapy in Systemic Sclerosis-associated Pulmonary Arterial Hypertension: A Retrospective RESCLE Registry Study

• Published in: Journal of rheumatology Vol. 47; no. 1; p. 89
• Main Authors: 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
• Format: Journal Article
• Language: English
• Published: Canada 01.01.2020
• Subjects: Adult; Aged; Aged, 80 and over; Antihypertensive Agents - adverse effects; Drug Therapy, Combination; Endothelin Receptor Antagonists - adverse effects; Female; Follow-Up Studies; Humans; Kaplan-Meier Estimate; Male; Middle Aged; Phosphodiesterase 5 Inhibitors - adverse effects; Pulmonary Arterial Hypertension - drug therapy; Pulmonary Arterial Hypertension - epidemiology; Pulmonary Arterial Hypertension - etiology; Pulmonary Arterial Hypertension - mortality; Registries; Retrospective Studies; Risk Factors; Scleroderma, Systemic - complications; Scleroderma, Systemic - epidemiology; Spain - epidemiology; Survival Rate; Treatment Outcome; Vital Capacity; Spain; PULMONARY ARTERIAL HYPERTENSION; SYSTEMIC SCLEROSIS; SURVIVAL ANALYSIS
• ISSN: 0315-162X; 1499-2752
• DOI: 10.3899/jrheum.180595

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. 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. 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. Combination sequential therapy improved survival in our cohort.