講演情報
[I-OR05-01]Treprostinil effectiveness in higher-risk pediatric patients with Idiopathic and Heritable Pulmonary Arterial Hypertension
○Yuan He, Qiangqiang Li, Chen Zhang, Hong Gu (Department of Pediatric Cardiology, Beijing Anzhen Hospital, China)
キーワード：
pediatric PAH、treatment、genetic testing
Introduction: Little is known about the effectiveness of treprostinil in higher-risk pediatric patients with various PAH genotypes. This study was designed to investigate the prognosis of higher-risk pediatric patients with IPAH and HPAH (IPAH/HPAH) following treprostinil therapy.Methods: Children with IPAH/HPAH who were stratified as higher-risk and treated with treprostinil in our center were included as study cohort. Meanwhile, those who only received oral medications were included as reference cohort. All patients in the study cohort received PAH-related genotyping. Results: Forty-nine children (median age 7.7 (IQR 4.2, 11.5) years, female 65.3%) were included in the study cohort and forty-eight children were included in the reference cohort. 84% of the study cohort had genetic disorders after genetic testing with a dominance of BMPR2 and ACVRL1 mutations. After a median therapy duration of 5.56 (IQR 2.66, 11.12) months, all patients were alive with significant improvements in clinical characteristics. One-, two- and three-year survival rate were 91% ,84% and 69% respectively with a median follow-up duration of 19.17 (IQR 9.7, 29.79) months, which was significant superior to the reference cohort (P=0.038). Multivariate cox regression analysis identified WHO-FC after therapy as a predictor for survival. There was no significant difference in survival among patients with different genotypes.Conclusion: Treprostinil can significantly improve the prognosis in children with IPAH/HPAH who are in higher-risk despite genetic backgrounds.