A recent study evaluating the efficacy of diacerein 1% in patients with epidermolysis bullosa simplex (EBS) revealed that it did not outperform vehicle in study endpoints.
The double-blind, randomized study consisted of 54 individuals with EBS. Patients were randomly assigned to receive either diacerein 1% or vehicle to be applied once daily for a total of eight weeks. Each patient then received eight follow-ups during a 16-week period following treatment. The study aimed to evaluate the outcomes of diacerein 1% on blistering, pain, and pruritus when compared with vehicle.
Results of the study indicated that there was no major statistical significance between diacerein 1% and vehicle.
A slightly higher percentage of diacerein-treated patients achieved the primary endpoint of a greater than or equal to 60% reduction in body surface area of EBS lesions, but not by a significant amount. Results showed 57.1% for diacerein-treated patients compared to 53.8% for vehicle.
As for the secondary endpoint of a greater than or equal to a 2-point reduction on the IGA scale, diacerein-treated patients did also achieve a slightly higher proportion compared to vehicle (42.9% compared to 26.9%). The findings were not deemed statistically significant.
During the study, reported treatment-emergent adverse events were also similar between the groups. A total of 78.6% occurred in the diacerein-treated group and 80.8% in the vehicle group. Two serious adverse events were reported in the vehicle group and none were reported in the diacerein 1% group. It was considered generally well-tolerated and safe.
A post hoc analysis revealed that the Investigator’s Global Assessment (IGA) success rate was higher in patients experiencing severe EBS compared to those with a moderate form of the disease. Among those with severe EBS, an IGA score of zero or one was reported in six of the 13 (46.2%) diacerein-treated patients compared with only two of 13 (15.4%) of the vehicle group. According to researchers, therefore, it is believed that diacerein 1% may be more beneficial for patients with higher disease severity.
The study concluded that “Although we can’t rule out the potential clinical benefits of increased concentrations of diacerein or longer treatment duration on EBS, future studies should focus on patients with severe EBS with more extensive involvement of lesion areas to demonstrate efficacy.”