10.5061/DRYAD.C670TQ2
Roustit, Matthieu
Grenoble Alpes University
Giai, Joris
Hospices Civils de Lyon
Gaget, Olivier
Grenoble Alpes University
Khouri, Charles
Grenoble Alpes University
Mouhib, Myriam
Grenoble Alpes University
Lotito, Adrien
Grenoble Alpes University
Blaise, Sophie
Grenoble Alpes University
Seinturier, Christophe
Grenoble Alpes University
Subtil, Fabien
Hospices Civils de Lyon
Paris, Adeline
Grenoble Alpes University
Cracowski, Claire
Grenoble Alpes University
Imbert, Bernard
Grenoble Alpes University
Carpentier, Patrick
Grenoble Alpes University
Vohra, Sunita
University of Alberta
Cracowski, Jean-Luc
Grenoble Alpes University
Data from: On-demand sildenafil as a treatment for Raynaud phenomenon: a
series of n-of-1 trials
Dryad
dataset
2019
Raynaud
sildenafil
n-of-1 trial
2019-01-25T15:09:06Z
2019-01-25T15:09:06Z
en
https://doi.org/10.7326/m18-0517
498012 bytes
1
CC0 1.0 Universal (CC0 1.0) Public Domain Dedication
Background: Treatment of Raynaud phenomenon (RP) with phosphodiesterase-5
inhibitors has shown moderate efficacy. Adverse effects decrease the
risk–benefit profile of these drugs, and patients may not be willing to
receive long-term treatment. On-demand single doses before or during
exposure to cold may be a good alternative. Objective: To assess the
efficacy and safety of on-demand sildenafil in RP. Design: Series of
randomized, double-blind, n-of-1 trials. (ClinicalTrials.gov:
NCT02050360). Setting: Outpatients at a French university hospital.
Participants: Patients with primary or secondary RP. Intervention: Each
trial consisted of a multiple crossover study in a single patient. Repeat
blocks of 3 periods of on-demand treatment were evaluated: 1 week of
placebo, 1 week of sildenafil at 40 mg per dose, and 1 week of sildenafil
at 80 mg per dose, with a maximum of 2 doses daily. Measurements: Raynaud
Condition Score (RCS) and frequency and daily duration of attacks. Skin
blood flow in response to cooling also was assessed with laser speckle
contrast imaging. Mixed-effects models were used and parameters were
estimated in a Bayesian framework to determine individual and aggregated
efficacy. Results: 38 patients completed 2 to 5 treatment blocks. On the
basis of aggregated data, the probability that sildenafil at 40 mg or 80
mg was more effective than placebo was greater than 90% for all outcomes
(except for RCS with sildenafil, 80 mg). However, the aggregated effect
size was not clinically relevant. Yet, substantial heterogeneity in
sildenafil's efficacy was observed among participants, with
clinically relevant efficacy in some patients. Limitation: The response to
sildenafil was substantially heterogeneous among patients. Conclusion:
Despite a high probability that sildenafil is superior to placebo,
substantial heterogeneity was observed in patient response and aggregated
results did not show that on-demand sildenafil has clinically relevant
efficacy. In this context, the use of n-of-1 trials may be an original and
relevant approach in RP.
PROFIL_DATAThe data in plain text format with variables names as header
2306 observations on 50 variablesmodel_1Final modelmodel_3Final model with
informative priors for the effect of sildenafilmodel_4Final model with
interaction between treatment and primary or secondary RP
Grenoble
France