PReS-FINAL-2180: Efficacy and safety of tocilizumab (TCZ) in patients with polyarticular-course juvenile idiopathic arthritis (pcJIA): 2-year data from CHERISH

Benedetti, Fabrizio; Ruperto, Nicolino; Żuber, Zbigniew; Cuttica, Rubén; Xavier, Ricardo Machado; Calvo, Ignacio; Rubio, Nadina; Алексеева, Е. И.; Chasnyk, Vyacheslav; Chávez, José; Horneff, Gerd; Opoka-Winiarska, Violetta; Quartier, Pierre; Spindler, Alberto; Keane, Caroline; Bharucha, Kamal N.; Wang, J; Lovell, Daniel J.; Martini, Alberto; Brunner, HI

doi:10.1186/1546-0096-11-s2-o15

Cited by 2 publications

(6 citation statements)

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“…d n calculated by reviewer (155 completed 104 weeks; 82 TCZ group completed 104 weeks). e Two abstracts 69,74 contain a table with a footnote to indicate patients who withdrew were excluded; however, in the third abstract 71 the table footnote states that patients who withdrew owing to non-safety reasons are non-responders, whereas patients who withdrew owing to safety are included using LOCF. f No active joints, no active uveitis, ESR < 20 mm/hour and PGA VAS ≤ 10.…”

Section: Results: Open-label Extensionsmentioning

confidence: 99%

“…69,71,74 Only participants who achieved at least ACR Pedi-30 during the open-label phase and who then continued into the double-blind RCT phase of the trial were eligible to enter the OLE, either after a JIA flare or when they completed the double-blind RCT phase. One-hundred and sixty (96%) of the 166 participants eligible to enter the OLE did so and 155 (97%) completed 104 weeks of follow-up (16-week open-label plus 24 weeks double-blind RCT plus 64 weeks OLE).…”

Section: Tocilizumab 68mentioning

confidence: 99%

“…Studies included (n = 4) (described by 9 full papers and 12 conference abstracts) [65][66][67] ) and one RCT evaluated tocilizumab [68][69][70][71][72][73][74][75][76] (the CHERISH trial). For the sake of brevity, generally only the key reference for each RCT is cited in the main text of this report.…”

Section: Quantity and Quality Of Available Researchmentioning

confidence: 99%

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The clinical effectiveness and cost-effectiveness of abatacept, adalimumab, etanercept and tocilizumab for treating juvenile idiopathic arthritis: a systematic review and economic evaluation

Shepherd

Cooper²,

Harris³

et al. 2016

Health Technol Assess

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BackgroundJuvenile idiopathic arthritis (JIA) is characterised by joint pain, swelling and a limitation of movement caused by inflammation. Subsequent joint damage can lead to disability and growth restriction. Treatment commonly includes disease-modifying antirheumatic drugs (DMARDs), such as methotrexate. Clinical practice now favours newer drugs termed biologic DMARDs where indicated.ObjectiveTo assess the clinical effectiveness and cost-effectiveness of four biologic DMARDs [etanercept (Enbrel®, Pfizer), abatacept (Orencia®, Bristol-Myers Squibb), adalimumab (Humira®, AbbVie) and tocilizumab (RoActemra®, Roche) – with or without methotrexate where indicated] for the treatment of JIA (systemic or oligoarticular JIA are excluded).Data sourcesElectronic bibliographic databases including MEDLINE, EMBASE, The Cochrane Library and the Database of Abstracts of Reviews of Effects were searched for published studies from inception to May 2015 for English-language articles. Bibliographies of related papers, systematic reviews and company submissions were screened and experts were contacted to identify additional evidence.Review methodsSystematic reviews of clinical effectiveness, health-related quality of life and cost-effectiveness were undertaken in accordance with the Preferred Reporting Items for Systematic Reviews and Meta-Analyses statement. A cost–utility decision-analytic model was developed to compare the estimated cost-effectiveness of biologic DMARDs versus methotrexate. The base-case time horizon was 30 years and the model took a NHS perspective, with costs and benefits discounted at 3.5%.ResultsFour placebo-controlled randomised controlled trials (RCTs) met the inclusion criteria for the clinical effectiveness review (one RCT evaluating each biologic DMARD). Only one RCT included UK participants. Participants had to achieve an American College of Rheumatology Pediatric (ACR Pedi)-30 response to open-label lead-in treatment in order to be randomised. An exploratory adjusted indirect comparison suggests that the four biologic DMARDs are similar, with fewer disease flares and greater proportions of ACR Pedi-50 and -70 responses among participants randomised to continued biologic DMARDs. However, confidence intervals were wide, the number of trials was low and there was clinical heterogeneity between trials. Open-label extensions of the trials showed that, generally, ACR responses remained constant or even increased after the double-blind phase. The proportions of adverse events and serious adverse events were generally similar between the treatment and placebo groups. Four economic evaluations of biologic DMARDs for patients with JIA were identified but all had limitations. Two quality-of-life studies were included, one of which informed the cost–utility model. The incremental cost-effectiveness ratios (ICERs) for adalimumab, etanercept and tocilizumab versus methotrexate were £38,127, £32,526 and £38,656 per quality-adjusted life year (QALY), respectively. The ICER for abatacept versus methotrexate as a second-line biologic was £39,536 per QALY.LimitationsThe model does not incorporate the natural history of JIA in terms of long-term disease progression, as the current evidence is limited. There are no head-to-head trials of biologic DMARDs, and clinical evidence for specific JIA subtypes is limited.ConclusionsBiologic DMARDs are superior to placebo (with methotrexate where permitted) in children with (predominantly) polyarticular course JIA who have had an insufficient response to previous treatment. Randomised comparisons of biologic DMARDs with long-term efficacy and safety follow-up are needed to establish comparative effectiveness. RCTs for JIA subtypes for which evidence is lacking are also required.Study registrationThis study is registered as PROSPERO CRD42015016459.FundingThe National Institute for Health Research Health Technology Assessment programme.

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Section: Results: Open-label Extensionsmentioning

confidence: 99%

Section: Tocilizumab 68mentioning

confidence: 99%

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The clinical effectiveness and cost-effectiveness of abatacept, adalimumab, etanercept and tocilizumab for treating juvenile idiopathic arthritis: a systematic review and economic evaluation

Shepherd

Cooper²,

Harris³

et al. 2016

Health Technol Assess

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“…Позже была установлена роль интерлейкина (Interleukin, IL) 6 в развитии не только системного ЮИА [8], но и ЮИА без системных проявлений [9]. В 2009 г. к использованию в детской практике при системном варианте заболевания был одобрен блокатор IL6 тоцилизумаб.…”

Section: обоснованиеunclassified

“…В 2009 г. к использованию в детской практике при системном варианте заболевания был одобрен блокатор IL6 тоцилизумаб. В последующем в многоцентровом плацебоконтролируемом исследовании CHERISH была доказана эффективность моноклональных антител к рецептору IL6 при всех вариантах ЮИА: 71% пациентов с ЮИА без системных проявлений за 2 года достигли 70% улучшения, а 58% -90% улучшения по критериям Американской коллегии ревматологов для детей (АКР педи ; American College of Rheumatology, ACR) [9]. С 2013 г. ILAR включила в клинические рекомендации использование тоцилизумаба как ГИБП первой линии при назначении пациентам с ЮИА без системных проявлений [4].…”

Section: обоснованиеunclassified

Prognostic Factors for the Response to Tocilizumab Therapy in Patients With Juvenile Idiopathic Arthritis Without Systemic Manifestations: A Cohort Study

Алексеева¹,

Дворяковская²,

Исаева³

et al. 2018

Vopr. sovr. pediatr.

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Background. To assign genetically engineered biologic drugs, we need data on the predictors for response to therapy. Prognostic factors for the response to tocilizumab in patients with juvenile idiopathic arthritis (JIA) without systemic symptoms are poorly studied.Objective. Our aim was to reveal early predictors for the response to tocilizumab therapy in patients with JIA without systemic symptoms.Methods. A retrospective cohort study enrolled patients with JIA without systemic symptoms who received tocilizumab therapy between July 2009 and August 2017. We assessed the association between the initial demographic, clinical, and laboratory parameters in patients and the best response (according to the ACR90 criteria) to treatment after a year.Results. The study included 95 (girls 85%) patients; the mean age was 10.3 (6.0; 13.6). During the first year of therapy, 71 (75%), 55 (58%), 38 (40%), and 22 (23%) patients achieved the improvement according to ACR30/50/70/90 criteria, respectively; 22 (23%) patients reached disease inactive stage according to the Wallace criteria. When performing multivariate analysis, the following improvement predictors were revealed based on the ACR90 criteria after a year of treatment: decrease in serum C-reactive protein level during the first month of therapy [odds ratio (OR) 1.024; 95% confidence interval (CI) 1.007–1.051], decrease in disease activity score on the visual analogue scale according to the parent/patient assessment (OR 1.048; 95% CI 1.005–1.105), early onset of the disease (OR 0.38; 95% CI 0.16–0.72), persistent oligoarthritis according to the ILAR (OR 9.9; 95% CI 1.5–109.3). During the first year of tocilizumab administration, neutropenia was registered in one patient, leukopenia — in three cases, and urticaria — in one case.Conclusion. The variant of JIA, the age at the disease onset, and the disease course pattern in the first month of tocilizumab therapy are the predictors of treatment efficacy throughout the year.

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PReS-FINAL-2180: Efficacy and safety of tocilizumab (TCZ) in patients with polyarticular-course juvenile idiopathic arthritis (pcJIA): 2-year data from CHERISH

Cited by 2 publications

References 0 publications

The clinical effectiveness and cost-effectiveness of abatacept, adalimumab, etanercept and tocilizumab for treating juvenile idiopathic arthritis: a systematic review and economic evaluation

The clinical effectiveness and cost-effectiveness of abatacept, adalimumab, etanercept and tocilizumab for treating juvenile idiopathic arthritis: a systematic review and economic evaluation

Prognostic Factors for the Response to Tocilizumab Therapy in Patients With Juvenile Idiopathic Arthritis Without Systemic Manifestations: A Cohort Study

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