Quality of Life, Safety and Efficacy Profile of Thermostable Flolan in Pulmonary Arterial Hypertension

Overview

Journal PLoS One

Specialties General Medicine
Science

Date 2015 Mar 21

PMID 25793960

Citations 6

Authors

Steeve Provencher

Patrap Paruchuru

Andrea Spezzi

Brian Waterhouse

Mardi Gomberg-Maitland

Affiliations

Soon will be listed here.

Abstract

Background: Flolan (epoprostenol sodium) is most commonly prescribed to patients with severe pulmonary arterial hypertension (PAH) owing to the requirement that the drug be delivered by continuous intravenous infusion and the reconstituted solution may only be administered up to 24 hours when it is maintained between a temperature of 2°C and 8°C. The aim of this single-arm, open label study was to describe the effects of the new thermostable formulation of Flolan on health-related quality of life (HRQoL) and ease of administration in subjects switching from the currently marketed Flolan to the reformulated product.

Methods: Following a 4-week run-in period and after 4 weeks of treatment with the reformulated product, patients completed the SF-36 HRQoL questionnaire and a study-specific questionnaire evaluating ease of administration, along with World Health Organization (WHO) functional class, six-minute walked distance (6MWD) and N-terminal-pro B-type natriuretic peptide (NT-proBNP) assessment.

Results: 16 participants completed the study. The SF-36 scores remained unchanged from baseline to Week 4. Conversely, there were small improvements for the majority of the study-specific questionnaire items and 14 (88%) subjects preferred the reformulated product to the currently marketed Flolan. There was no significant change in the dose of reformulated product, 6MWD, Borg dyspnoea index, WHO functional class and mean NT-proBNP levels. No significant changes in haemodynamic parameters were seen from baseline to 2 hours post transition in a subset of patients undergoing catheterization.

Conclusion: The reformulated product was not associated with significant improvement in HRQoL compared with the currently marketed Flolan as measured by the SF-36. However, most subjects preferred the reformulated product to the currently marketed Flolan. Moreover, the 2 formulations of Flolan had similar safety and efficacy profiles.

Trial Registration: ClinicalTrials.gov NCT01462565.

Citing Articles

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The Transition From Ambrisentan to Macitentan in Patients With Pulmonary Arterial Hypertension: A Real-word Prospective Study.

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Investigation of safety and efficacy of the new more thermostable formulation of Flolan (epoprostenol) in Japanese patients with pulmonary arterial hypertension (PAH)-An open-label, single-arm study.

Mihara K, Ogawa A, Matsubara H, Terao T, Ichikawa Y PLoS One. 2018; 13(4):e0195195.

PMID: 29608587 PMC: 5880383. DOI: 10.1371/journal.pone.0195195.

A systematic review of transition studies of pulmonary arterial hypertension specific medications.

Sofer A, Ryan M, Tedford R, Wirth J, Fares W Pulm Circ. 2017; 7(2):326-338.

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Epoprostenol and pulmonary arterial hypertension: 20 years of clinical experience.

Sitbon O, Vonk Noordegraaf A Eur Respir Rev. 2017; 26(143).

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