Idiopathic Pulmonary Arterial Hypertension in Paediatrics Represents Still a Serious Challenge: A Case Series Study

Introduction: Paediatric pulmonary hypertension (PH) represents a heterogeneous illness that is responsible for high morbidity and mortality if left without treatment. Idiopathic pulmonary arterial hypertension (IPAH) is a subtype of PAH rarely seen in paediatrics. Limited long-term data are available. Methods: Over a period of 20 years, 10 paediatric patients were enrolled at two tertiary centres. Their clinical, echocardiographic, and right heart catheterisation (RHC) features and outcome were evaluated. Results: The mean age at first diagnosis was 5.7 +/- 5.7 years. The age at the last follow-up was 12.4 +/- 6.1 years. The average follow-up was 6.6 +/- 0.8 years. There was a female prevalence of 60% (p < 0.05) in this case series. Regarding the NYHA functional class, 80% of IPAH subjects were in class III or IV. The mean saturation was 91 +/- 5%. In this regard, 70% of the patients were on a combination of three drugs, with sildenafil (90%) included. On echocardiography, longitudinal right ventricular contractility (TAPSE) was slightly reduced (13.4 +/- 2.6 mm), whilst RVSP was severely elevated (101 +/- 19 mmHg). The RHC data showed that mPAP was 61.8 +/- 23.1 mmHg (p = 0.0017 with RVSP on echocardiography), mRAP was 10.7 +/- 3.8 mmHg, CI was 2.6 +/- 1 L center dot min(-1)center dot m(-2), PVRi was 16.8 +/- 12.6 WU center dot m(2), and SVO2 was 63.6 +/- 14.8%. Regarding the outcome, two male IPAH patients (20%) died, and 50% underwent lung transplant or were on transplant assessment or already on the waiting list for lung transplantation. One patient underwent a ductus arteriosus stenting (reverse Potts shunt) and another underwent atrial septostomy and stenting. Conclusions: Notwithstanding the progress in medical therapy, IPAH continues to represent a serious challenge, particularly in the paediatric population, with the need for lung transplantation and significant mortality.