10:20 AM - 11:50 AM
[I-S10-02] 小児期発症の遺伝性肺動脈性肺高血圧における臨症状、治療反応性、予後の違い
Clinical differences in disease severities, treatment responses, and outcomes between idiopathic and heritable pulmonary arterial hypertension in children
Shinichi Takatsuki MD (lecturer), Satoshi Ikehara MD, Tomotaka Nakayama MD, Hiroyuki Matsuura MD, and Tsutomu Saji MD
Department of Pediatrics, Toho University Omori Medical Center, Tokyo
Abstract
The previous study reported that pediatric heritable pulmonary arterial hypertension (HPAH) with BMPR2 and ALK 1 mutation has worse 5-year survival rate compared with idiopathic PAH (IPAH). However, the causal factors of the different prognosis between HPAH and IPAH have not been fully evaluated. We review the survival rate and clinical differences between HPAH and IPAH in pediatric populations.
This is retrospective cohort study using clinical data from 81 pediatric patients (≤20 years at diagnosis) who were managed at Toho University Omori Medical Center between 1992 and 2015. A positive response to acute vasoreactivity testing (AVT) is defined as a reduction of mean pulmonary arterial pressure ≥20% and unchanged or improved CI and Rp/Rs ratio. Composite outcomes include hospitalization due to heart failure, lung transplantation, and cardiac mortality.
We have 18 HPAH (9 BMPRII, 6 ALK-1, 5 others) and 62 IPAH children. Among HPAH, 11 children have familial history. There were no significant differences in demographic data at diagnosis including gender, age, symptoms, brain natriuretic peptide, NYHA functional class, and hemodynamics. During AVT, 15% IPAH patients were positive responders, whereas none of HPAH patients had positive response to vasodilators. Although 85% HPAH patients received epoprostenol therapy (IPAH; 65%), HPAH had worse prognosis compared with IPAH (adverse events; 70% vs 40%, respectively) during follow-up. However, cardiac mortality were not significant different between HAPAH and IPAH (30% vs 20%, respectively). Response to vasodilators and composite outcomes in children with HPAH are not favorable compared with IPAH, whereas mortality is not significantly different.
Shinichi Takatsuki MD (lecturer), Satoshi Ikehara MD, Tomotaka Nakayama MD, Hiroyuki Matsuura MD, and Tsutomu Saji MD
Department of Pediatrics, Toho University Omori Medical Center, Tokyo
Abstract
The previous study reported that pediatric heritable pulmonary arterial hypertension (HPAH) with BMPR2 and ALK 1 mutation has worse 5-year survival rate compared with idiopathic PAH (IPAH). However, the causal factors of the different prognosis between HPAH and IPAH have not been fully evaluated. We review the survival rate and clinical differences between HPAH and IPAH in pediatric populations.
This is retrospective cohort study using clinical data from 81 pediatric patients (≤20 years at diagnosis) who were managed at Toho University Omori Medical Center between 1992 and 2015. A positive response to acute vasoreactivity testing (AVT) is defined as a reduction of mean pulmonary arterial pressure ≥20% and unchanged or improved CI and Rp/Rs ratio. Composite outcomes include hospitalization due to heart failure, lung transplantation, and cardiac mortality.
We have 18 HPAH (9 BMPRII, 6 ALK-1, 5 others) and 62 IPAH children. Among HPAH, 11 children have familial history. There were no significant differences in demographic data at diagnosis including gender, age, symptoms, brain natriuretic peptide, NYHA functional class, and hemodynamics. During AVT, 15% IPAH patients were positive responders, whereas none of HPAH patients had positive response to vasodilators. Although 85% HPAH patients received epoprostenol therapy (IPAH; 65%), HPAH had worse prognosis compared with IPAH (adverse events; 70% vs 40%, respectively) during follow-up. However, cardiac mortality were not significant different between HAPAH and IPAH (30% vs 20%, respectively). Response to vasodilators and composite outcomes in children with HPAH are not favorable compared with IPAH, whereas mortality is not significantly different.