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

被引：11

作者：

Pestana-Fernandez, Melani ^{[1
]}

Rubio-Rivas, Manuel ^{[1
]}

Tolosa-Vilella, Carles ^{[2
]}

Guillen-Del-Castillo, Alfredo ^{[3
]}

Freire, Mayka ^{[4
]}

Antonio Vargas-Hitos, Jose ^{[5
]}

Antonio Todoli-Parra, Jose ^{[6
]}

Rodriguez-Carballeira, Monica ^{[7
]}

Marin-Ballv, Adela ^{[8
]}

Espinosa, Gerard ^{[9
]}

Colunga-Argueelles, Dolores ^{[10
]}

Ortego-Centeno, Norberto ^{[11
]}

Trapiella-Martinez, Luis ^{[12
]}

Carbonell-Munoz, Cristina ^{[13
]}

Pla-Salas, Xavier ^{[14
]}

Perales-Fraile, Isabel ^{[15
]}

Corbella, Xavier ^{[1
,16
]}

Fonollosa-Pla, Vicent ^{[3
]}

Pilar Simeon-Aznar, Carmen ^{[3
]}

机构：

[1] Bellvitge Univ Hosp, Dept Internal Med, Autoimmune Dis Unit, Lhospitalet De Llobregat, Spain

[2] Corp Sanitaria Univ Parc Tauli, Dept Internal Med, Barcelona, Spain

[3] Hosp Univ Vall dHebron, Dept Internal Med, Unit Autoimmune Dis, Barcelona, Spain

[4] Complejo Hosp Univ Vigo, Dept Internal Med, Unit Syst Autoimmune Dis & Thrombosis, Vigo, Spain

[5] Hosp Univ Virgen de las Nieves, Dept Internal Med, Granada, Spain

[6] Hosp Univ & Politecn La Fe, Dept Internal Med, Valencia, Spain

[7] Hosp Univ Mutua Terrassa, Dept Internal Med, Barcelona, Spain

[8] Hosp Clin Univ Lozano Blesa, Dept Internal Med, Unit Autoimmune Dis, Zaragoza, Spain

[9] Hosp Univ Clin, Clin Inst Med & Dermatol, Dept Syst Autoimmune Dis, Barcelona, Spain

[10] Hosp Univ Cent Asturias, Dept Internal Med, Oviedo, Spain

[11] Complejo Univ Granada, Hosp Campus Salud, Dept Internal Med, Unit Syst Autoimmune Dis, Granada, Spain

[12] Hosp Cabuenes, Dept Internal Med, Unit Syst Autoimmune Dis, Gijon, Asturias, Spain

[13] Complejo Asistencial Univ Salamanca, Dept Internal Med, Salamanca, Spain

[14] Consorci Hosp Vic, Dept Internal Med, Unit Syst Autoimmune Dis, Barcelona, Spain

[15] Hosp Univ Rey Juan Carlos, Dept Internal Med, Madrid, Spain

[16] Univ Int Catalunya, Fac Med & Hlth Sci, Barcelona, Spain

来源：

JOURNAL OF RHEUMATOLOGY | 2020年 / 47卷 / 01期

关键词：

SYSTEMIC SCLEROSIS; PULMONARY ARTERIAL HYPERTENSION; SURVIVAL ANALYSIS; CONNECTIVE-TISSUE DISEASE; PROGNOSTIC-FACTORS; SURVIVAL; BOSENTAN; SILDENAFIL; TADALAFIL; MORTALITY; AMBRISENTAN; PREVALENCE; PREDICTORS;

D O I：

10.3899/jrheum.180595

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

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.

引用

页码：89 / 98

页数：10

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