N-of-1 Trial Data on On-Demand Sildenafil for Raynaud Phenomenon

38 patients completed a series of randomized, double-blind n-of-1 trials assessing on-demand sildenafil for Raynaud phenomenon. The dataset captures repeated crossover treatment blocks comparing placebo, 40 mg, and 80 mg sildenafil doses. Individual and aggregated efficacy was analyzed using mixed-effects models in a Bayesian framework.

Use Cases

• Analyze heterogeneity in patient response by comparing Raynaud Condition Score (RCS) outcomes across placebo, 40 mg, and 80 mg sildenafil doses.
• Model the relationship between skin blood flow measurements from laser speckle contrast imaging and the daily duration of Raynaud attacks.
• Apply Bayesian mixed-effects models to aggregated data to estimate the probability of sildenafil's superiority over placebo for attack frequency.
• Identify patient subgroups where sildenafil at 40 mg or 80 mg demonstrated clinically relevant efficacy versus placebo.

Strengths

• Data originates from a formal clinical trial series with 38 participants, providing a structured experimental basis.
• Study design features repeated crossover blocks (2 to 5 per patient) comparing three distinct treatment conditions.
• Includes multiple clinical outcome measures: Raynaud Condition Score, attack frequency, attack duration, and objective skin blood flow.

Limitations

• Small sample size of 38 patients limits statistical power for detecting subgroup effects or generalizing findings.
• Substantial heterogeneity in individual patient response to sildenafil was reported as a key study limitation.
• The aggregated effect size for sildenafil was not found to be clinically relevant despite statistical significance.

Provenance

Source

Dryad digital repository, from a clinical trial (NCT02050360).

Collection Method

Series of randomized, double-blind, n-of-1 trials with multiple crossover treatment blocks.

Time Range

Trial period not specified; data published in 2020.

Freshness

Data reflects a trial completed and published by 2020.

Geography

Outpatients at a French university hospital.