What is the clinical evidence from this study?

Study design: Observational. Population: Pulmonary arterial hypertension (IPAH, HPAH, CTD-PAH). Intervention: First-line combination therapy vs. First-line single therapy. Primary outcome: Survival, treatment escalation, and haemodynamics.

What does this research mean for the field?

In patients with pulmonary arterial hypertension, first-line combination therapy improves hemodynamics more effectively than single therapy, and the MRAP/CI ratio serves as a novel prognostic marker for outcomes. Novelty: ClaimNovelty.CONFIRMATORY. Consensus alignment: ConsensusAlignment.SUPPORTS_CONSENSUS.

June 10, 2014

Prognosis and response to first-line single and combination therapy in pulmonary arterial hypertension

Key Result

First-line combination therapy for pulmonary arterial hypertension more greatly decreased pulmonary vascular resistance index (p=0.017) and improved cardiac index (p=0.042) than single therapy.

Study Design

Type

Observational

Multicenter

Structured PICO

Does first-line combination therapy improve hemodynamics and survival compared to first-line single therapy in patients with PAH?

Population

Patients with idiopathic (IPAH), hereditary (HPAH), and connective tissue disease-associated (CTD-PAH) pulmonary arterial hypertension (PAH) treated at Skåne University Hospital 2000-2011

Intervention

First-line combination therapy

Comparator

First-line single therapy

Outcome

Survival at 1, 2, and 3 yearshard clinical

First-line combination therapy in PAH may more potently improve hemodynamics compared to single therapy, though overall survival remains poor and early treatment escalation is frequently needed.

Abstract

OBJECTIVES: To investigate survival, treatment escalation, effects of first-line single- and first-line combination therapy and prognostic markers in idiopathic- (IPAH), hereditary- (HPAH) and connective tissue disease-associated (CTD-PAH) pulmonary arterial hypertension (PAH). DESIGN: Retrospective analysis of medical journals from PAH patients at Skåne University Hospital 2000-2011. RESULTS: 1-, 2- and 3-year survival was 87%, 67%, and 54%, respectively, for the entire population, but worse (p = 0.003) in CTD-PAH than IPAH/HPAH. After 1, 2 and 3 years, 58%, 41% and 24% of patients starting on single therapy were alive on single therapy. 37.5% of patients on first-line single therapy received escalated treatment at first follow-up. First-line combination therapy more greatly decreased pulmonary vascular resistance index (PVRI, p = 0.017) than first-line single therapy. Only first-line combination therapy improved (p = 0.042) cardiac index (CI). Higher mean right atrial pressure (MRAP, p = 0.018), MRAP/CI (p = 0.021) and WHO functional class (p < 0.001) and lower 6-min walking distance (6MWD, p = 0.001) at baseline, and higher PVRI (p = 0.008) and lower 6MWD (p = 0.004) at follow-up were associated with worse outcome. CONCLUSIONS: We confirm improved survival with PAH-targeted therapies. Survival is still poor and early treatment escalation frequently needed. First-line combination therapy may more potently improve haemodynamics. MRAP/CI may represent a new prognostic marker in PAH.

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