Canakinumab exerts lasting effects in colchicine-resistant familial Mediterranean fever

Patients with colchicine-resistant familial Mediterranean fever (crFMF) benefit from long-term treatment with canakinumab, preventing flares and controlling disease activity without increasing adverse events (AEs), according to the results of a phase III study.

The analysis included 60 crFMF patients who entered the Epoch 4 (weeks 41 to 113) phase of the CLUSTER trial, with 57 completing the study. These patients had been randomized to receive open-label canakinumab 150 or 300 mg every 4 or 8 weeks during a 72-week period. Overall, 44 patients received <2,700-mg canakinumab and 16 received ≥2,700 mg.

Median age of the population was 18 years, with three patients aged <6 years. Fourteen (23 percent) patients had body weight <40 kg and therefore received weight-adjusted doses. There were 15 participants previously treated with anakinra. All patients had frequent flares prior to entering the trial, with a median of 17.5 flares per year.

Over the 72-week period, 35 (58.3 percent) participants had no disease flares, while 23 (38.3 percent) had one flare. Physician global assessment scores indicated no disease activity in the majority of the population.

Median C-reactive protein concentrations remained <10 mg/L, while median serum amyloid A levels stayed above the limit of normal (10 mg/L) but under the 30-mg/L threshold.

No new or unexpected adverse events (AEs) were reported. The median duration of exposure to canakinumab in Epoch 4 was 511.5 days. Most AEs (90.6 percent) were mild to moderate in severity, and the most common were symptoms of FMF flares, such as abdominal pain and fever.