Research ArticleAccepted Article

Long-term Efficacy and Safety of Monotherapy Versus Combination Therapy in Systemic Sclerosis-associated Pulmonary Arterial Hypertension: A Retrospective Cohort Study from the Nationwide Spanish Scleroderma Registry (RESCLE)

Melani Pestaña-Fernández, Manuel Rubio-Rivas, Carles Tolosa Vilella, Alfredo Guillén del Castillo, Mayka Freire, Jose Antonio Vargas Hitos, Jose Antonio Todolí Parra, Mónica Rodríguez Carballeira, Adela Marín Ballvé, Gerard Espinosa, Dolores Colunga Argüelles, Norberto Ortego Centeno, Luis Trapiella Martínez, Cristina Carbonell Muñoz, Xavier Pla Salas, Isabel Perales Fraile, Xavier Corbella, Vicent Fonollosa Pla and Carmen Pilar Simeón Aznar on behalf of RESCLE investigators, Autoimmune Diseases Study Group (GEAS).
The Journal of Rheumatology February 2019, jrheum.180595; DOI: https://doi.org/10.3899/jrheum.180595

Abstract

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 efficacy of monotherapy vs. combination therapy in Systemic Sclerosis (SSc)-associated PAH patients.

Methods All patients with SSc-associated PAH from the Spanish Scleroderma Registry (RESCLE) were reviewed. Patients were split up in three groups: monotherapy vs. sequential combination vs. upfront combination. Primary endpoint was death from any cause at 1, 3 and 5 years from PAH diagnosis.

Results 76 patients (4.2%) out of 1817 had SSc-related PAH. 34 patients (45%) were receiving monotherapy [ERA (22, 29%) or PDE5 inhibitors (12, 16%)], 25 (33%) sequential combination, and 17 (22%) upfront combination. A lower FVC/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 mPAP 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 independent predictors of mortality. Sequential combination was found an independent protective factor [HR=0.11 (95%CI 0.03-0.51), p=0.004], while upfront combination 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% vs. 95.8% vs. 94.1% at 1 year, 40.7% vs. 81.5% vs. 51.8% at 3 years and 31.6% vs. 56.5% vs. 34.5% at 5 years (p=0.007). Side effects were not significantly different among groups.

Conclusion Combination sequential therapy improved survival in our cohort.

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