Quality of life and functional capacity can be improved in patients with Eisenmenger syndrome with oral sildenafil therapy

Abstract

Background

Patients with Eisenmenger syndrome (ES) have a decreased exercise capacity and poor quality of life (QoL). While patients may survive to middle adulthood, the burden of disease is disabling. Sildenafil seems to improve exercise tolerance and hemodynamics, but there is no data to date on its impact on QoL.

Methods

Eisenmenger patients in New York Heart Association (NYHA) class III were recruited in a prospective study of efficacy and safety of oral sildenafil. The QoL endpoint was assessed using a disease-specific questionnaire (CAMPHOR). Exercise capacity was assessed by means of six minute walk test (6MWT). All patients underwent comprehensive assessment at baseline and after 3 months of treatment.

Results

Twelve patients (mean age was 34.3 ± 10.2, 83% female) with various cardiac anatomies were recruited. No major adverse events during the follow-up or significant drop in resting oxygen saturation were recorded. After 3 months of oral sildenafil therapy, all patients improved to NYHA II with a concomitant improvement in 6MWT distance (347.3 ± 80.7 to 392.5 ± 82.0 m, p = 0.002). All components of the CAMPHOR score, relating to symptoms, activity and QoL, improved significantly resulting in substantial improvement in the total CAMPHOR score (27.6 ± 10.5 to 15.8 ± 10.4, p = 0.002).

Conclusions

Three months of sildenafil therapy in adults with ES was well tolerated and associated with significant improvement in the QoL CAMPHOR questionnaire and in NYHA class and exercise capacity. Larger studies are warranted to assess long term efficacy of oral sildenafil and potential impact on survival.

Introduction

Eisenmenger syndrome (ES) is defined as significant pulmonary arterial hypertension in conjunction with congenital heart disease and reversal of shunt [1]. The majority of patients who develop ES survive to adulthood, and most live many years thereafter. Morbidity and mortality, however, remain high [2]. Multi-system involvement and marked exercise intolerance are common amongst patients with ES and impact not only on outcome but also on quality of life (QoL) [3]. Compared to idiopathic pulmonary arterial hypertension (PAH), patients with ES have better survival prospects and, thus, have to cope with a poor QoL for a significantly longer period of time. Maintaining an adequate QoL is therefore paramount as are interventions improving QoL. Until recently, limited treatment options were available for patients with ES. The advent of advanced therapies for PAH has brought new promise in the management of these patients [4], [5], [6]. Sildenafil, a phosphodiesterase-5 inhibitor, has been an attractive therapeutic option shown to improve functional capacity with limited adverse effects. Available studies on sildenafil have, however, focused primarily on hemodynamic and exercise endpoints (Table 1) [7], [8], [9], [10], [11], with little attention paid to QoL. We assessed the effect of sildenafil on the QoL of patients with ES, exercise capacity and safety.