Randomised and clinical controlled trials reporting the efficacy of IL-1 inhibitors (anakinra, canakinumab and rilonacept) in sJIA and AOSD
| First author | Study design | Patients (N) | Intervention | CID (%) | Other outcomes (%) | Risk* |
| Quartier45 | Double-blind randomised placebo-controlled | sJIA (12) | ANK | na | M1: ACRPed 70 5 (42%) | |
| sJIA (12) | PBO | na | M1: ACRPed 70 0 | |||
| LTE | sJIA (22) | ANK | 5/16 (31%)* | na | ||
| Nordstrom46 | Open-label randomised | AOSD (12) | ANK | M6: 6/12 (50%) | na | |
| AOSD (10) | csDMARDs | M6: 2/10 (20%) | na | |||
| LTE | AOSD (17) | ANK | M12: 7/14 (50%) | na | ||
| Ruperto† 52 | Double-blind randomised single dose | sJIA (43) | CAM | M1: 13 (30%) | M1: ACR70 29/43 (67%) ACR90 20/43 (47%) | |
| sJIA (41) | PBO | M1: 0 | M1 aACR70 1/41 (2%) aACR90 1/41 (2%) | |||
| Ruperto52 | Open-label lead-in phase | sJIA (177) | CAM | 55/176 (31) | aACR70 113/175 (65%) aACR90 90/175 (51%) | |
| Randomised withdrawal | sJIA (50) | CAM | 31/50 (62) | aACR70 41/50 (82%) aACR90 38/50 (76%) | ||
| sJIA (50) | PBO | 17/50 (34) | aACR70 31/50 (62%) aACR90 28/50 (56%) | |||
| Ruperto53 | LTE from NCT00886769, NCT00889863, NCT00426218 and NCT00891046 open-label single arm | sJIA (144) | CAM | M6: 58/177 (33) M24: 69/177 (40) | M6: aJIA-ACR 70 116 (65%) aJIA-ACR 90 92 (52%) At 3 years: aJIA-ACR 70 95 (54%) aJIA-ACR 90 87 (50%) CRM: 33/177 (19%) at 6M | |
| Nishimura54 | Open label-single arm | sJIA (19) | CAM | M11: 9/12 (75) | M11: ACRPed70 16 (100%) ACRPed90 12/14 (87%) | |
| Quartier55 | sJIA (98) | CAM | na | M6: CRM: 49/98 (50%) | ||
| Lovell65 | Randomised double blind | sJIA (17) | RIL | na | M1: ACR 70 3/17 (18%) | |
| sJIA (7) | PBO | na | M1: ACR 70 1/7 (14%) | |||
| LTE | sJIA (23) | RIL | 2/23 (8%) | M12: ACR70 19 (83%) | ||
| Ilowite66 | Randomised double blind | sJIA (36) | RIL | M3: 4/33 (12%) M6: 11/55 (20%) | M1: ACRPed70 14/35 (40%) M3: ACRPed70 23/33 (70%) | |
| sJIA (35) | PBO | na‡ | M1: ACRPed70 4/33 (12%) | |||
| Kedor60 | Double-blind randomised | AOSD (18) | CAM | M3: 5/18 (33%) | M3: ACR70 5 (28%) ACR90 2 (11%) DAS28-ESR<2.6 5 (33%)§ DAS28-CRP 12 (67%) | |
| AOSD (17) | PBO | M3: 2/17 (12%) | M3: ACR70 2 (12%) ACR90 1 (6%) DAS28-ESR<2.6 1 (12%)§ DAS28-CRP 7 (41%) | |||
| LTE | AOSD (23) | CAM | 4/23 (17%) at M5 | na |
The RoB was assessed with the Rob2 tool. Red=high, yellow=intermediate and green=low.
*In the long-term open-label phase, 16 patients reached month 12; among seven responders, five of them had inactive disease.
†The principal outcome of Trial-1 was the proportion of patients who achieved adapted ACR30 response; the open-label phase determined if at least 25% of patients treated with GCs were able to have their dose tapered; in the withdrawal phase (Trial-2) the objective was to show that the time to flare was longer with CAM than placebo. In Trial-2, patients were also evaluated for higher levels of improvement including adapted JIA-ACR50.
‡The PBO group received RIL after the 4-week double-blind phase, therefore CID is not available for the initial PBO group.
§Data are referred to as per-protocol population.
ACR, American College of Rheumatology; ANK, anakinra; AOSD, adult-onset Still’s disease; CAM, canakinumab; CID, clinical inactive disease; CRM, clinical remission on medications; CRP, C reactive protein; csDMARDs, conventional synthetic disease antirheumatic modifying drugs; DAS, disease activity score; ESR, erythrocyte sedimentation rate; LTE, long-term extension; M, month; na, not available; OLE, open-label extension; PBO, placebo; RIL, rilonacept; sJIA, systemic juvenile idiopathic arthritis.