Abstract 4386: Single-Center Experience in Large Cohort of Patients with Pulmonary Arterial Hypertension Related to Congenital Heart Disease over 15 years: Effect of New Therapies
Introduction: The aim of this study was to define the epidemiology, clinical parameters and 15-year follow-up of a large single-center cohort of patients with pulmonary arterial hypertension (PAH) related to congenital heart disease (CHD) and to evaluate the effect of emerging therapeutic strategies in this population.
Methods: All patients diagnosed with PAH related to CHD between 1993 and 2008 were studied and clinical, echocardiographic, maximal and submaximal exercise as well as hemodynamic data were recorded.
Results: A total of 60 pts aged 21 ± 14 (0.1–51) years at diagnosis were included in the study. Of these, 44 (73%) had unrestrictive mixing lesions and Eisenmenger syndrome and the rest non-mixing lesions, mainly repaired CHD. Of this population, 24 (40%) were <18yrs and the remainder adults at diagnosis at our institution. Seven (12%) pts in WHO Class II received no therapy, 45 (75%) pts in Class II-III monotherapy (34 bosentan with 1 discontinuation after corrective surgery, 6 sildenafil, 5 sitaxsentan) with clinical and exercise improvement. Due to deterioration, 6 (10%) pts received addition of sildenafil after 3±1 years of bosentan monotherapy and 2 (3%) pts addition of epoprostenol after 1 year of the above combination therapy. Over 6±4 (1–15) years of follow-up, 49 (82%) pts remained stable on no or monotherapy, 4 pts (7%) deteriorated after 1–5 years monotherapy but stabilized on combination therapy and 7 (12%) pts died (3 from intractable right heart failure despite escalation of therapy and 4 suddenly while on therapy). The unrestrictive mixing group had 2 (4%) and the non-mixing group 5 (33%) deaths respectively.
Conclusions: Long-term improvement and stabilization is possible in pts with PAH related to CHD with either monotherapy or combination therapy in case of deterioration overtime while on treatment. Death in this population may be sudden or due to intractable right heart failure, while survival is significantly better in pts with unrestrictive mixing. Larger long-term registry studies are needed to evaluate survival and clinical condition of these pts in the current treatment era and to improve medical management of this population.