[II-JCKAP3-2] Pharmacological treatment for pediatric patients with congenital heart disease with pulmonary hypertension: Insights from nationwide registry in Japan
Keywords:CHD-PAH, pharmacological treatment, efficacy
Background: The efficacy of pharmacological treatment for pediatric patients with congenital heart disease with pulmonary hypertension (CHD-PH) remains unclear. Methods: The multi-center registry study on CHD-PH in Japan began in August 2021. From this registry, patients aged 15 years old or younger at the time of diagnosis were selected, while those with a single ventricle were excluded. Results: 215 patients were included in this study. The median age of patients at enrollment was 8.5 years old. Approximately 60% of the enrolled patients had some form of comorbidity, including trisomy 21. Pharmacological treatments received by the enrolled patients were as follows: no medication in 74 cases (34.4%), monotherapy in 61 cases (28.4%), and combination therapy in 80 cases (37.2%). The medication used predominantly were endothelin receptor blocker and PDE-5 inhibitors, each being administered to approximately half of the patients. In the 85 cases where past and registration catheter results were comparable, 19 patients (22.4%) showed an increase in mPAP of over 20%. In logistic regression analysis, even after adjusting for age at diagnosis, mPAP at diagnosis, and clinical classification, the odds ratio for worsening mPAP remained high in the group not receiving medication.Conclusion: In pediatric patients with CHD-PH, pharmacological treatment for pulmonary hypertension is essential.