RT Journal Article
SR Electronic
T1 Subcutaneous treprostinil in congenital heart disease-related pulmonary arterial hypertension
JF Heart
JO Heart
FD BMJ Publishing Group Ltd and British Cardiovascular Society
SP heartjnl-2017-312143
DO 10.1136/heartjnl-2017-312143
A1 Nika Skoro-Sajer
A1 Christian Gerges
A1 Olga Hajnalka Balint
A1 Dora Kohalmi
A1 Monika Kaldararova
A1 Iveta Simkova
A1 Johannes Jakowitsch
A1 Harald Gabriel
A1 Helmut Baumgartner
A1 Mario Gerges
A1 Roela Sadushi-Kolici
A1 David S Celermajer
A1 Irene Marthe Lang
YR 2018
UL http://heart.bmj.com/content/early/2018/02/06/heartjnl-2017-312143.abstract
AB Objective To assess the efficacy and safety of subcutaneous treprostinil in adult patients with congenital heart disease (CHD)-associated pulmonary arterial hypertension (PAH) after 12 months of treatment.Methods Consecutive adult patients with CHD–PAH received subcutaneous treprostinil to maximum tolerated doses in an observational study.Results Advanced CHD–PAH patients with WHO class III or IV disease (n=32, age 40±10 years, 20 females) received treprostinil for suboptimal response to bosentan (n=12), WHO functional class IV disease (FC, n=7) or prior to bosentan approval (n=13). In the multivariate mixed model, mean increase in 6 min walk distance (6-MWD) from baseline to 12 months was 114 m (76; 152) (P<0.001). WHO FC improved significantly (P=0.001) and B-type brain natriuretic peptide decreased from 1259 (375; 2368) pg/mL to 380 (144; 1468) pg/mL (P=0.02). In those 14 patients who had haemodynamic data before and after initiation of treprostinil, pulmonary vascular resistance decreased significantly (from 18.4±11.1 to 12.6±7.9 Wood units, P=0.003). The most common adverse events were infusion-site erythema and pain. One patient stopped treatment because of intolerable infusion-site pain after 8 months of treatment. No other major treatment-related complications were observed. Five patients died during early follow-up, having experienced a decrease in their 6-MWD prior.Conclusions Subcutaneous treprostinil therapy is generally safe and effective for at least 12 months and may be used in CHD-related PAH class III and IV.