title: Quality of life, safety and efficacy profile of thermostable flolan in pulmonary arterial hypertension.
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.
  
  
  CONCLUSIONS
  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.
  
  
  BACKGROUND
  ClinicalTrials.gov NCT01462565.
authors: Pulmonary Hypertension Research Group, Centre de recherche de l'Institut Universitaire de cardiologie et de pneumologie de Québec, Université Laval, Québec, Québec, Canada.
authors: Steeve
authors: S
authors: Provencher
authors: GlaxoSmithKline R&D, 980 Great West Road, Brentford, United Kingdom.
authors: Patrap
authors: P
authors: Paruchuru
authors: GlaxoSmithKline R&D, 980 Great West Road, Brentford, United Kingdom.
authors: Andrea
authors: A
authors: Spezzi
authors: GlaxoSmithKline R&D, 980 Great West Road, Brentford, United Kingdom.
authors: Brian
authors: B
authors: Waterhouse
authors: University of Chicago Medical Centre, 5841 S Maryland Ave, MC5403, Rm L08, Chicago, Illinois, United States of America.
authors: Mardi
authors: M
authors: Gomberg-Maitland
mesh_terms: D023381:Endpoint Determination; D011464:Epoprostenol / Q000009:adverse effects / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D006976:Hypertension, Pulmonary / Q000188:drug therapy*; D008297:Male; D008875:Middle Aged; D011788:Quality of Life; D011795:Surveys and Questionnaires; D013696:Temperature; D016896:Treatment Outcome
chemical_list: D011464:Epoprostenol