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http://www.ncbi.nlm.nih.gov/pmc/articles/PMC11657668PMC
http://dx.doi.org/10.1016/j.ijcchd.2022.100353DOI Listing

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Pulmonary arterial hypertension (PAH) is a long-term condition characterised by increased resistance to blood flow in the pulmonary circulation. The disease has a progressive course and is associated with a poor prognosis. Without treatment, PAH is associated with mortality in <3 years.

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Pulmonary arterial hypertension (PAH) is a rare and potentially fatal condition characterized by progressive increases in blood pressure in the arteries of the lungs. Oral selexipag, approved by the Food and Drug Administration (FDA) in 2015 for the treatment of PAH, targets prostacyclin receptors on pulmonary arterial vascular smooth muscle and endothelial cells to improve blood flow through the lungs and reduce pulmonary vascular resistance. Oral selexipag is effective, but may be discontinued due to factors like side effects, emergency conditions, or inability to take oral medication, potentially leading to severe adverse events, such as rebound pulmonary hypertension and right heart failure.

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Background: Selexipag, an oral prostacyclin (PGI) receptor agonist, is approved for adult patients with pulmonary arterial hypertension (PAH). This study evaluated the efficacy and safety of selexipag for Japanese pediatric patients with PAH.

Methods And Results: The study enrolled 6 patients who received selexipag twice daily at an individualized dose based on body weight; maintenance doses were determined for each patient by 12 weeks after starting administration.

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A wide range of substances is currently available for the treatment of patients with pulmonary arterial hypertension. The current recommendations for initial drug therapy are based on the patient's risk profile. For patients at high risk, an initial triple combination therapy with different substances including prostanoids is recommended.

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Article Synopsis
  • Selexipag is an oral medication used to treat pulmonary arterial hypertension (PAH) in adults, but there are currently no approved treatments targeting this pathway for children.
  • The researchers aimed to establish appropriate dosing for pediatric patients by using a pharmacokinetic model derived from adult data, creating weight-based dosing groups with specific starting and maximum doses for different age brackets.
  • A clinical study involving 63 pediatric PAH patients showed that the adjusted doses for children resulted in similar drug exposure levels to the adult population, confirming the dosing regimen's safety and efficacy for kids aged 2 to under 18.
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