Abstract
Pulmonary hypertension (PH) in children is a life-threatening disease of complex underlying causes and poor prognosis. Oral therapy with sildenafil added to conventional treatments has improved patient outcomes in terms of exercise capacity and hemodynamics. This study aimed to address the efficacy of using oral sildenafil in children with PH and assess the short-term survival after therapy initiation.
This is an observational study of 167 children aged 3 months to 17 years with pulmonary hypertension who were admitted to the Cardiology Department of Assiut University Children Hospital. The children were followed for 12 weeks between November 2014 and May 2016. The therapeutic effects of adding oral sildenafil plus conventional therapy were compared with those on conventional therapy alone. The main outcome measure was the change in the estimated mean pulmonary arterial pressure (mPAP) measured by echocardiography from the baseline to week 12 after initiation of therapy. The secondary endpoints were 12-week overall survival and improvement in oxygen saturation.
All children were treatment-naïve with median age of 1 year. After 12 weeks of treatment, the mPAP was significantly reduced in the sildenafil-treated children compared to the conventional group (−17.4 ± 16.4 mmHg versus −7.9 ± 15.1 mmHg; p < .001). Multivariate regression analysis demonstrated a significant influence of adding sildenafil (p < .01) on the mPAP at 12-week follow up. Sildenafil was not associated with an increased risk of death, [Hazard ratio (HR): 1.16; 95% confidence intervals (CI), 0.68–2.00, p = .39]. Reduced ejection fraction heart failure and transposition of great arteries were significant factors associated with worse survival [HR: 2.46; 95% CI: 1.08–5.58, p = .03] and HR: 3.69; 95% CI: 1.04–13.11, p = .04, respectively].
Addition of sildenafil to conventional therapy led to significant improvements in mPAP and oxygen saturation. However, it did not lower the mortality risk in children with pulmonary hypertension.
•Adding oral sildenafil to the conventional therapy improved the mean arterial blood pressure•Sildenafil did not improve the short-term survival of children•Physicians added oral sildenafil to the conventional therapy for critically ill children.