[OR20-1] セレキシパグの小児肺動脈性肺高血圧症に対する安全性と効果：初の多施設共同研究
Keywords:肺高血圧症, セレキシパグ, 多施設共同研究
Background: EPPVDN investigated the safety and efficacy of add-on selexipag in the largest, exploratory pediatric cohort to date.
Methods: This is a prospective observational study of 15 consecutive children with PAH, treated with oral add-on selexipag at 3 centers. Most patients underwent cardiac catheterizations at baseline and median 8 months follow-up. All patients had clinical, echocardiographic and NTproBNP studies, including the novel EPPVDN pediatric PH risk score.
Results: There was no death during selexipag use. Two of 15 patients ultimately underwent lung transplantation. One patient with heritable PAH died on intravenous treprostinil (off selexipag). Mean right atrial pressure, the ratio of pulmonary artery to systemic artery pressure (mPAP/mSAP -17%; p＜0.05), and transpulmonary pressure gradients (mean TPG -17%; p＜0.01; diastolic TPG -6 mmHg; p＜0.05) improved. Selexipag therapy was associated with better TAPSE and functional class. Non-invasive and combined-invasive PH risk scores improved (lower risk +18-22%, higher risk －35-37%; p＜0.05). Overall, the efficacy of selexipag was variable, often with better response in less sick patients.
Conclusion: Oral selexipag use in children with PAH is well tolerated and safe when closely monitored. Add-on selexipag therapy improved several outcome-relevant variables in about half, and prevented disease progression in additional 27% of patients. The novel EPPVDN pediatric PH risk score indicated these drug effects properly, can be useful in clinical follow-up, and should be validated in larger prospective studies.
Methods: This is a prospective observational study of 15 consecutive children with PAH, treated with oral add-on selexipag at 3 centers. Most patients underwent cardiac catheterizations at baseline and median 8 months follow-up. All patients had clinical, echocardiographic and NTproBNP studies, including the novel EPPVDN pediatric PH risk score.
Results: There was no death during selexipag use. Two of 15 patients ultimately underwent lung transplantation. One patient with heritable PAH died on intravenous treprostinil (off selexipag). Mean right atrial pressure, the ratio of pulmonary artery to systemic artery pressure (mPAP/mSAP -17%; p＜0.05), and transpulmonary pressure gradients (mean TPG -17%; p＜0.01; diastolic TPG -6 mmHg; p＜0.05) improved. Selexipag therapy was associated with better TAPSE and functional class. Non-invasive and combined-invasive PH risk scores improved (lower risk +18-22%, higher risk －35-37%; p＜0.05). Overall, the efficacy of selexipag was variable, often with better response in less sick patients.
Conclusion: Oral selexipag use in children with PAH is well tolerated and safe when closely monitored. Add-on selexipag therapy improved several outcome-relevant variables in about half, and prevented disease progression in additional 27% of patients. The novel EPPVDN pediatric PH risk score indicated these drug effects properly, can be useful in clinical follow-up, and should be validated in larger prospective studies.