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Congenital heart disease
Original research article
Subcutaneous treprostinil in congenital heart disease-related pulmonary arterial hypertension
  1. Nika Skoro-Sajer1,
  2. Christian Gerges1,
  3. Olga Hajnalka Balint2,
  4. Dora Kohalmi2,
  5. Monika Kaldararova3,
  6. Iveta Simkova3,
  7. Johannes Jakowitsch1,
  8. Harald Gabriel1,
  9. Helmut Baumgartner4,
  10. Mario Gerges1,
  11. Roela Sadushi-Kolici1,
  12. David S Celermajer5,
  13. Irene Marthe Lang1
  1. 1Department of Internal Medicine II, Division of Cardiology, Vienna General Hospital, Medical University of Vienna, Vienna, Austria
  2. 2György Gottsegen, Hungarian Institute of Cardiology, Budapest, Hungary
  3. 3Department of Cardiology and Angiology, Slovak Medical University and National Institute of Cardiovascular Diseases, Bratislava, Slovakia
  4. 4Division of Adult Congenital and Valvular Heart Disease, Department of Cardiovascular Medicine, University Hospital Muenster, Muenster, Germany
  5. 5Sydney Medical School, University of Sydney, Sydney, Australia
  1. Correspondence to Dr Irene Marthe Lang, Department of Internal Medicine II, Division of Cardiology, Medical University of Vienna, Vienna 1090, Austria; irene.lang{at}meduniwien.ac.at

Abstract

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.

  • secondary pulmonary hypertension

This is an open access article distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/

https://doi.org/10.1136/heartjnl-2017-312143

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    Footnotes

    • Contributors NS-S and IML designed, wrote the protocol, submitted ethics application, included patients and treated patients. HG, RS-K and HB have included and treated the patients. OHB, DK, MK and IS submitted ethics applications, included patients and treated patients. CG, MG and JJ have prepared statistical analyses and the figures. NS-S, DC and IML have drafted the work.

    • Competing interests NS-S reports personal fees from GlaxoSmithKline, grants and personal fees from AOPOrphan Pharmaceuticals, grants and personal fees from Actelion, personal fees from Bayer AG, personal fees from Pfizer, personal fees from United Therapeutics, outside the submitted work.

    • Patient consent Obtained.

    • Ethics approval The databases used in this study were approved by the Ethics Committee of the Medical University of Vienna (# 972/2009), the Ethics Committee of the Gottsegen György Hungarian Institute of Cardiology and the Ethics Committee of the Slovak Medical University.

    • Provenance and peer review Not commissioned; externally peer reviewed.