Key result
The addition of oral selexipag in a 15-year-old girl with severe idiopathic pulmonary arterial hypertension improved her WHO functional class from IV to II and decreased pulmonary artery systolic pressure from 104.8 to 78 mmHg over 3 months.
Why the study?
There is a lack of childhood data regarding the use of the oral prostacyclin pathway drug selexipag in pediatric patients with pulmonary arterial hypertension.
Does the addition of oral selexipag improve symptoms, quality of life, and exercise capacity in a teenage patient with severe idiopathic PAH failing dual therapy?
Case Report (n=1)
No
Does the addition of oral selexipag improve symptoms, quality of life, and exercise capacity in a teenage patient with severe idiopathic PAH failing dual therapy?
Oral selexipag may be a safe and effective add-on therapy for pediatric patients with severe PAH who are deteriorating on dual oral therapy, improving functional class and hemodynamics.
May support add-on selexipag in severe pediatric IPAH; leaves open need for controlled trials before practice change.
Pulmonary arterial hypertension (PAH) is a chronic, progressive, multifactorial disease. Currently, combination therapy is an attractive option for PAH management because three pathways (endothelin, nitric oxide, and prostacyclin) are involved in this disease. Selexipag is a novel oral prostacyclin pathway drug and is a highly selective IP prostacyclin receptor agonist with vasodilatory and antiproliferative effects. We report the case of a teenage patient with idiopathic PAH who presented in World Health Organization functional Class IV and showed no clinical improvement with dual therapy. We added oral selexipag to the treatment regimen and observed substantial improvement in her quality of life at the short-time follow-up. Despite the lack of childhood data regarding the use of selexipag in pediatric patients with PAH, the use of this drug in the current teenage patient improved her quality of life and exercise capacity.
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Bravo‐Valenzuela et al. (2020) conducted a case report in Idiopathic pulmonary arterial hypertension (n=1). Selexipag was evaluated on WHO functional class and pulmonary artery systolic pressure (PASP). The addition of oral selexipag in a 15-year-old girl with severe idiopathic pulmonary arterial hypertension improved her WHO functional class from IV to II and decreased pulmonary artery systolic pressure from 104.8 to 78 mmHg over 3 months.
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