Childhood pulmonary arterial hypertension: Insights from REVEAL

Background: The 55-center, observational, US Registry to EValuate Early And Long-term pulmonary arterial hypertension (PAH) Disease Management (REVEAL) examines demographics, clinical course and management of patients (pts) diagnosed with PAH. Methods: Pts aged ≥3 months and ≤18 years at time of confirmatory diagnostic catheterization were followed at 26 sites. Data were analyzed to explore demographics, treatment and outcomes in the following PAH subgroups: idiopathic PAH (IPAH), familial PAH (FPAH), and PAH associated with congenital heart disease (APAH-CHD). Results: 3518 pts enrolled in REVEAL between 03/2006 and 12/2009; 77 APAH-CHD and 122 IPAH/FPAH had childhood-onset PAH; mean ± SD age at PAH diagnosis 6±6 years and 9±6 years ( P = 0.002), respectively. APAH-CHD had similar functional class (FC) at diagnosis compared with IPAH/FPAH (FC: I, 5%; II, 51%; III, 32%; and IV, 11% vs FC: I, 7%; II, 40%; III, 43%; and IV, 10%, respectively; P = 0.64). Hemodynamics were also similar at diagnosis. Similar proportions were treated with endothelin receptor antagonists (APAH-CHD, 40%; IPAH/FPAH, 45%; P = 0.53), though phosphodiesterase 5 inhibitors (45% vs 64%, respectively; P = 0.011) and prostacyclin (28% vs 50%, respectively; P = 0.003) analogs were used less in APAH-CHD. Five-year survival from diagnosis was similar for APAH-CHD and IPAH/FPAH (71±14% vs 75±7% vs, respectively; P = 0.53). Conclusion: Childhood-onset APAH-CHD and IPAH/FPAH have similar hemodynamics and FC and similar 5-year survival from diagnosis. REVEAL provides observational data on treatment and outcomes in childhood PAH that should prove useful in identifying prognostic parameters, thereby helping clinicians improve outcomes.