Efficacy and safety of tocilizumab in patients with polyarticular-course juvenile idiopathic arthritis: results from a phase 3, randomised, double-blind withdrawal trial.
Efficacy and safety of tocilizumab in patients with polyarticular-course juvenile idiopathic arthritis: results from a phase 3, randomised, double-blind withdrawal trial.
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Tocilizumab对多节幼年特发性关节炎患者的疗效和安全性：第3期，随机，双盲戒断试验的结果。
DOI：
10.1136/annrheumdis-2014-205351
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发表时间：
2015-06
影响因子：
27.4
通讯作者：
Pediatric Rheumatology Collaborative Study Group (PRCSG)
中科院分区：
文献类型：
--
作者：
Brunner HI;Ruperto N;Zuber Z;Keane C;Harari O;Kenwright A;Lu P;Cuttica R;Keltsev V;Xavier RM;Calvo I;Nikishina I;Rubio-Pérez N;Alexeeva E;Chasnyk V;Horneff G;Opoka-Winiarska V;Quartier P;Silva CA;Silverman E;Spindler A;Baildam E;Gámir ML;Martin A;Rietschel C;Siri D;Smolewska E;Lovell D;Martini A;De Benedetti F;Paediatric Rheumatology International Trials Organisation PRINTO;Pediatric Rheumatology Collaborative Study Group (PRCSG)
评价白细胞介素6受体抑制剂托珠单抗治疗多关节型幼年特发性关节炎（pcJIA）的疗效。这项三部分、随机、安慰剂对照、双盲退出研究（NCT 00988221）纳入了活动性pcJIA ≥6个月且对甲氨蝶呤应答不足的患者。 在第1部分，患者每4周接受一次开放标签托珠单抗（体重（BW）<30 kg为8或10 mg/kg; BW ≥30 kg为8 mg/kg）。 在第16周，改善≥ JIA-美国流变学学会（ACR）30的患者在1：1随机分配至安慰剂组或托珠单抗组（按甲氨蝶呤和类固醇背景治疗分层）后进入24周双盲第2部分，以评价主要终点：与第16周相比的JIA发作。正在发作或完成第2部分的患者接受开放标签托珠单抗治疗。在第1部分，188例患者接受托珠单抗治疗（<30 kg：10 mg/kg（n=35）或8 mg/kg（n=34）; ≥30 kg：n=119）。 在第2部分，163例患者接受托珠单抗（n=82）或安慰剂（n=81）。安慰剂组48.1%的患者发生JIA发作，而托珠单抗组25.6%的患者发生JIA发作（分层调整后的均值差异：-0.21; 95%CI-0.35至-0.08; p=0.0024）。在第2部分结束时，64.6%和45.1%接受托珠单抗治疗的患者分别有JIA-ACR 70和JIA-ACR 90应答。不良事件（AE）和严重AE（SAE）的发生率/100患者年（PY）分别为480和12.5;感染是最常见的SAE（4.9/100 PY）。托珠单抗治疗导致pcJIA体征和症状的显著改善，并随时间推移而维持，其安全性特征与成人类风湿性关节炎一致。NCT00988221。
To evaluate the interleukin-6 receptor inhibitor tocilizumab for the treatment of patients with polyarticular-course juvenile idiopathic arthritis (pcJIA). This three-part, randomised, placebo-controlled, double-blind withdrawal study (NCT00988221) included patients who had active pcJIA for ≥6 months and inadequate responses to methotrexate. During part 1, patients received open-label tocilizumab every 4 weeks (8 or 10 mg/kg for body weight (BW) <30 kg; 8 mg/kg for BW ≥30 kg). At week 16, patients with ≥JIA-American College of Rheumatology (ACR) 30 improvement entered the 24-week, double-blind part 2 after randomisation 1:1 to placebo or tocilizumab (stratified by methotrexate and steroid background therapy) for evaluation of the primary end point: JIA flare, compared with week 16. Patients flaring or completing part 2 received open-label tocilizumab. In part 1, 188 patients received tocilizumab (<30 kg: 10 mg/kg (n=35) or 8 mg/kg (n=34); ≥30 kg: n=119). In part 2, 163 patients received tocilizumab (n=82) or placebo (n=81). JIA flare occurred in 48.1% of patients on placebo versus 25.6% continuing tocilizumab (difference in means adjusted for stratification: −0.21; 95% CI −0.35 to −0.08; p=0.0024). At the end of part 2, 64.6% and 45.1% of patients receiving tocilizumab had JIA-ACR70 and JIA-ACR90 responses, respectively. Rates/100 patient-years (PY) of adverse events (AEs) and serious AEs (SAEs) were 480 and 12.5, respectively; infections were the most common SAE (4.9/100 PY). Tocilizumab treatment results in significant improvement, maintained over time, of pcJIA signs and symptoms and has a safety profile consistent with that for adults with rheumatoid arthritis. NCT00988221.
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影响因子：
158.5
作者：
De Benedetti, Fabrizio;Brunner, Hermine I.;Martini, Alberto
通讯作者：
Martini, Alberto
影响因子：
27.4
作者：
Otten, Marieke H.;Prince, Femke H. M.;van Suijlekom-Smit, Lisette W. A.
通讯作者：
van Suijlekom-Smit, Lisette W. A.
影响因子：
8
作者：
Daniels, Stephen R.;Greer, Frank R.
通讯作者：
Greer, Frank R.
影响因子：
168.9
作者：
Ruperto, Nicolino;Lovell, Daniel J.;Giannini, Edward H.
通讯作者：
Giannini, Edward H.
DOI：
10.1136/bmj.c332
发表时间：
2010-03-23
期刊：
BMJ (Clinical research ed.)
影响因子：
--
作者：
Schulz KF;Altman DG;Moher D;CONSORT Group
通讯作者：
CONSORT Group