Trial testing rapamycin as treatment for fibrofolliculomas – results published

Earlier this week, the results of a clinical trial testing the effectiveness of topical rapamycin as a treatment for BHD were published in PLOS ONE. The study was performed in Professor Dr Maurice van Steensel’s lab at Maastricht University Medical Centre, and funded by the Myrovlytis Trust.

19 BHD patients were able to complete the trial and were given two creams to use daily; a placebo cream, and a cream containing 0.1% Sirolimus. The study was double-blind, randomised, facial left-right controlled, so patients and doctors did not know which side of their face was being treated with the drug. Patients were then followed up at 3 and 6 months. The primary outcome measure was cosmetic improvement as measured by doctors and patients, and changes in the number and size of fibrofolliculomas were also measured.

Doctors reported that 2 patients’ fibrofolliculomas showed cosmetic improvement on both sides of their faces. 3 patients reported that their fibrofolliculomas showed cosmetic improvement on the placebo treated side only; 7 reported improvement on the treated side only; and 2 patients reported improvement on both sides of their face. Reduction in the number of fibrofolliculomas was observed on the rapamycin treated side only in 2 patients; on the placebo treated side only in 3 patients; and on both facial halves in 4 patients. Fibrofolliculomas were shown to grow more on rapamycin treated than placebo treated facial sides, but this finding was not statistically significant.

13 out of 19 patients reported side effects of rapamycin treatment, including a burning sensation, erythema, dryness and itching.

Thus, the researchers conclude that topical rapamycin does not improve the cosmetic appearance of fibrofolliculomas.

This study is freely available to download from the BHD Articles Library: Clinical Research.

To find out more, the latest version of the database is available to download here.