Diacerein 1% may benefit patients with severe epidermolysis bullosa simplex
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Key takeaways:
- Diacerein 1% did not outperform vehicle in study endpoints.
- Among the severe disease subgroup, an IGA of 0 or 1 was achieved by 46.2% of diacerein- treated patients vs. 15.4% of vehicle-treated patients.
Diacerein 1% ointment was comparable to vehicle in study endpoints but did outperform vehicle when stratified by disease severity in the treatment of patients with epidermolysis bullosa simplex, according to a study.
"Epidermolysis bullosa simplex (EBS), the most common type of epidermolysis bullosa, is a rare genetic skin condition, diagnosed in approximately eight per 1 million births,” Joyce Teng, MD, PhD, of Stanford Medicine, and colleagues wrote. “There are no approved treatments for EBS. Therapy is currently limited to local wound care, pain and itch management, and treatment of infection.”
Diacerein 1% ointment is an interleukin-1 beta inhibitor that may be able to manage EBS. In the double-blind, randomized, vehicle-controlled trial, Teng and colleagues evaluated the outcomes of diacerein 1% on blistering, pain and pruritus compared with vehicle.
A total of 54 patients with EBS were randomly assigned to receive diacerein 1% (n = 28) or vehicle (n = 26) to be applied once daily for 8 weeks, with eight follow-ups during the 16 weeks following treatment.
Results showed that there was no statistical significance between diacerein 1% and vehicle outcomes. A slightly higher, although not significant, proportion of diacerein-treated patients achieved the primary endpoint of a greater than or equal to 60% reduction in body surface area of EBS lesions compared with vehicle (57.1% vs. 53.8%).
Similarly, a higher proportion of diacerein-treated patients achieved the secondary endpoint of a greater than or equal to 2-point reduction on the IGA scale compared with vehicle (42.9% vs 26.9%), but this finding was also not statistically significant.
Treatment-emergent adverse events were also similar between groups with 78.6% occurring in the diacerein groups and 80.8% occurring in the vehicle group.
Two serious adverse events were reported in the vehicle group, whereas diacerein 1% had no serious events and was considered generally well tolerated and safe.
A post hoc analysis stratified by EBS subtypes showed that IGA success was higher in patients with severe EBS compared with moderate EBS. Among those with severe EBS, an IGA score of 0 or 1 was reported in six of 13 (46.2%) of the diacerein-treated patients compared with two of 13 (15.4%) of the vehicle group (relative risk = 3.08; 95% CI, 0.71-13.4). As a result, diacerein 1% may be more beneficial for patients with higher disease severity, according to the researchers.
“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,” Teng and colleagues concluded.
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