@article {Pesta{\~n}a-Fern{\'a}ndezjrheum.180595, author = {Melani Pesta{\~n}a-Fern{\'a}ndez and Manuel Rubio-Rivas and Carles Tolosa-Vilella and Alfredo Guill{\'e}n-Del-Castillo and Mayka Freire and Jose Antonio Vargas-Hitos and Jose Antonio Todol{\'\i}-Parra and M{\'o}nica Rodr{\'\i}guez-Carballeira and Adela Mar{\'\i}n-Ballv{\'e} and Gerard Espinosa and Dolores Colunga-Arg{\"u}elles and Norberto Ortego-Centeno and Luis Trapiella-Mart{\'\i}nez and Cristina Carbonell-Mu{\~n}oz and Xavier Pla-Salas and Isabel Perales-Fraile and Xavier Corbella and Vicent Fonollosa-Pla and Carmen Pilar Sime{\'o}n-Aznar}, title = {Longterm Efficacy and Safety of Monotherapy versus Combination Therapy in Systemic Sclerosis{\textendash}associated Pulmonary Arterial Hypertension: A Retrospective RESCLE Registry Study}, elocation-id = {jrheum.180595}, year = {2019}, doi = {10.3899/jrheum.180595}, publisher = {The Journal of Rheumatology}, 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 the efficacy of monotherapy versus combination therapy in patients with systemic sclerosis (SSc){\textendash}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 {\textpm} 1.1 vs 1.8 {\textpm} 0.4 vs 2.3 {\textpm} 0.8; p = 0.085) and also a higher mean pulmonary arterial pressure in combination groups (37.2 {\textpm} 8.7 mmHg vs 40.8 {\textpm} 8.8 vs 46 {\textpm} 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{\textendash}0.51; p = 0.004), while upfront combination therapy showed a trend (HR 0.68, 95\% CI 0.23{\textendash}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.}, issn = {0315-162X}, URL = {https://www.jrheum.org/content/early/2019/07/23/jrheum.180595}, eprint = {https://www.jrheum.org/content/early/2019/07/23/jrheum.180595.full.pdf}, journal = {The Journal of Rheumatology} }