Long-Term Efficacy and Safety of Oleogel-S10 (Birch Triterpenes) for Pediatric Patients With Epidermolysis Bullosa.

BACKGROUND/

OBJECTIVES

Pediatric patients with epidermolysis bullosa (EB) experience lifelong complications, and wound healing is an important treatment goal. In the phase III EASE study (NCT03068780), Oleogel-S10 accelerated wound healing in EB. This prespecified subgroup analysis evaluated the long-term efficacy and safety of Oleogel-S10 in patients aged < 18 years from EASE.

METHODS

EASE comprised a 90-day double-blind phase (DBP) and 24-month open-label phase (OLP). In the DBP, patients were randomized 1:1 to receive Oleogel-S10 or control gel; all patients could enter the OLP and receive Oleogel-S10. Endpoints in this analysis included wound healing, total body wound burden, dressing change burden, and safety.

RESULTS

Overall, 156 pediatric patients were enrolled into the DBP (Oleogel-S10: n = 74; Control gel: n = 82); 147 entered the OLP. Oleogel-S10 resulted in 44.6% of pediatric patients with first complete target wound closure within 45 days, versus 25.6% with control gel (relative risk [95% CI]: 1.70 [1.11, 2.60], p = 0.012). Mean absolute body surface area percentage scores were 12.5% at DBP baseline and 5.3% at OLP Month 24 for the Oleogel-S10 group. Significantly more patients treated with Oleogel-S10 (38%) no longer required daily dressing changes versus control gel (9%) at DBP Day 90. Similar proportions of patients experienced adverse events in the Oleogel-S10 and control gel groups in the DBP, with most mild or moderate.

CONCLUSIONS

These analyses of pediatric patients demonstrated accelerated wound healing, reduced frequency of dressing changes, and sustained reduction in wound burden up to 27 months. Long-term safety was consistent with the overall EASE population.

TRIAL REGISTRATION: NCT03068780, EudraCT 2016-002066-32; registered 3 March 2017; https://clinicaltrials.gov/study/NCT03068780 and https://www.clinicaltrialsregister.eu/ctr-search/trial/2016-002066-32/GB.