A Snapshot of IPAH from a National Australian and New Zealand Paediatric Registry

Background: Idiopathic pulmonary arterial hypertension (IPAH) in children is an uncommon and progressive condition with a poor natural history. Currently available therapies have not been trialled in children and there is little information about long-term survival in treated children. Methods: A multicentre, web-based Australian and New Zealand registry for children with PAH was established to better understand the profile of children with IPAH, aiming to describe demographics, presenting features, clinical course and outcomes of children aged three months to 18 years at time of PAH diagnosis, from 1/1/2002. A group of historic controls with IPAH diagnosed between 1970 and 1990 were used for comparison. Results: Of the 24 patients enrolled with IPAH, 54% are female and 75% Caucasian. The mean (SD) age at diagnosis was 9.3 (4.90) years and 50% were WHO functional class III at presentation. Baseline cardiac catheterisation data for both groups is shown. During follow-up, 20 (83%) subjects were treated with an endothelin receptor antagonist (exclusively Bosentan monohydrate), 17 (71%) with a phosphodiesterase 5 inhibitor (sildenafil) and 10 (42%) with either epoprostenol or iloprost; 19 (79%) are anticoagulated with warfarin. The mean duration of follow-up was 3.45 years, with an annualised mortality of 6%. Study end-points of death/transplant occurred in 6/24, compared to 10/12 controls (p = .001). Severity of PAH from cardiac catheterisation was similar between patients and controls. Conclusions: Multiple medical therapies have resulted in improved survival for children with IPAH diagnosed during the current era. A better understanding of childhood IPAH will facilitate medical care, permit standardisation of therapeutic guidelines, and allow better representation in the healthcare arena.