Prospective analysis of the port-wine stain patient population in the Netherlands in light of novel treatment modalities

Abstract

Background: Pulsed dye laser is the gold standard for port-wine stain (PWS) treatment. However, pulsed dye lasers achieve suboptimal clinical results in a majority of patients. Patient demand for novel therapies and willingness to participate in clinical studies is currently unknown, yet, imperative for steering R&D activity. The objective of this study was to evaluate these two factors in relation to PWS patient demographics. Methods: A questionnaire was used to assess patient and PWS characteristics, treatment history, efficacy, and satisfaction, stress level, willingness to travel and pay for an effective treatment, participation in clinical studies, and amenability to intravenous drug administration. Descriptive statistics and correlation analysis were performed. Results: Of the respondents (N = 108), 65% would participate in clinical studies and 49% would accept intravenous drugs. For an effective treatment, 58% was prepared to pay over €2,000 and 48% would travel more than 6 h. Travel time was inversely correlated with age, clearance rate, and satisfaction. Facial PWS patients had undergone more treatments, were less satisfied, and less willing to participate in studies or accept intravenous drugs. Stress levels were higher in females. Conclusion: There is considerable demand for new PWS therapies, and a substantial proportion of patients are willing to participate in clinical studies.