On-Demand Sildenafil as a Treatment for Raynaud Phenomenon: A Series of n-of-1 Trials.

摘要: Background Treatment of Raynaud phenomenon (RP) with phosphodiesterase-5 inhibitors has shown moderate efficacy. Adverse effects decrease the risk-benefit profile these drugs, and patients may not be willing to receive long-term treatment. On-demand single doses before or during exposure cold a good alternative. Objective To assess efficacy safety on-demand sildenafil in RP. Design Series randomized, double-blind, n-of-1 trials. (ClinicalTrials.gov: NCT02050360). Setting Outpatients at French university hospital. Participants Patients primary secondary Intervention Each trial consisted multiple crossover study patient. Repeated blocks 3 periods treatment were evaluated: 1 week placebo, 40 mg per dose, 80 maximum 2 daily. Measurements Condition Score (RCS) frequency daily duration attacks. Skin blood flow response cooling also was assessed laser speckle contrast imaging. Mixed-effects models used parameters estimated Bayesian framework determine individual aggregated Results 38 completed 5 blocks. On basis data, probability that more effective than placebo greater 90% for all outcomes (except RCS sildenafil, mg). However, effect size clinically relevant. Yet, substantial heterogeneity sildenafil's observed among participants, relevant some patients. Limitation The substantially heterogeneous Conclusion Despite high is superior patient results did show In this context, use trials an original approach Primary Funding Source GIRCI (Groupement Interregional de Recherche Clinique et d'Innovation) Auvergne Rhone-Alpes (academic funding) Pfizer.