The interleukin 1 inhibitor rilonacept in treatment of chronic gouty arthritis: results of a placebo-controlled, monosequence crossover, non-randomised, single-blind pilot study.
BACKGROUND
Recent studies suggest that blockade of the NLRP3 (cryopyrin) inflammasome interleukin 1beta (IL1beta) pathway may offer a new treatment strategy for gout.


OBJECTIVE
To explore the potential utility of rilonacept (IL1 Trap) in patients with chronic active gouty arthritis in a proof-of-concept study.


METHODS
This 14-week, multicentre, non-randomised, single-blind, monosequence crossover study of 10 patients with chronic active gouty arthritis included a placebo run-in (2 weeks), active rilonacept treatment (6 weeks) and a 6-week post-treatment follow-up.


RESULTS
Rilonacept was generally well tolerated. No deaths and no serious adverse events occurred during the study. One patient withdrew owing to an injection-site reaction. Patients' self-reported median pain visual analogue scale scores significantly decreased from week 2 (after the placebo run-in) to week 4 (2 weeks of rilonacept) (5.0 to 2.8; p<0.049), with sustained improvement at week 8 (1.3; p<0.049); 5 of 10 patients reported at least a 75% improvement. Median symptom-adjusted and severity-adjusted joint scores were significantly decreased. High-sensitivity C-reactive protein levels fell significantly.


CONCLUSIONS
This proof-of-concept study demonstrated that rilonacept is generally well tolerated and may offer therapeutic benefit in reducing pain in patients with chronic refractory gouty arthritis, supporting the need for larger, randomised, controlled studies of IL1 antagonism such as with rilonacept for this clinical indication.
Terkeltaub
R
R
VAMC Rheumatology, 111K, 3350 La Jolla Village Drive, San Diego, CA 92161, USA. rterkeltaub@ucsd.edu
Sundy
J S
JS
Schumacher
H R
HR
Murphy
F
F
Bookbinder
S
S
Biedermann
S
S
Wu
R
R
Mellis
S
S
Radin
A
A
D000368:Aged; D015210:Arthritis, Gouty / Q000097:blood / Q000188:drug therapy*; D002097:C-Reactive Protein / Q000378:metabolism; D002908:Chronic Disease; D004812:Epidemiologic Methods; D005260:Female; D006074:Gout Suppressants / Q000009:adverse effects / Q000627:therapeutic use*; D006801:Humans; D053583:Interleukin-1beta / Q000037:antagonists & inhibitors*; D008297:Male; D008875:Middle Aged; D010147:Pain Measurement / Q000379:methods; D011993:Recombinant Fusion Proteins / Q000009:adverse effects / Q000627:therapeutic use*; D016896:Treatment Outcome
D006074:Gout Suppressants; D053583:Interleukin-1beta; D011993:Recombinant Fusion Proteins; C531377:rilonacept; D002097:C-Reactive Protein