Induction and Maintenance Therapy With Infliximab for Children With Moderate to Severe Ulcerative Colitis

Hyams, Jeffrey S.; Damaraju, Lakshmi; Blank, Marion; Johanns, Jewel; Guzzo, Cynthia; Winter, Harland S.; Kugathasan, Subra; Cohen, Stanley A.; Markowitz, James; Escher, Johanna C.; Veereman-Wauters, G.; Crandall, Wallace; Baldassano, Robert N.; Griffiths, Anne M.

doi:10.1016/j.cgh.2011.11.026

Cited by 245 publications

(194 citation statements)

References 19 publications

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“…Conversely, anti-tumor necrosis factor therapy, which is effective in most cases of severe IBD, 8,9 resulted in only partial remission of gastrointestinal disorders caused by MKD. Possible efficacy of IL-1 agents to treat and prevent attacks in some patients with MKD has been highlighted.…”

Section: Figurementioning

confidence: 99%

Severe Early-Onset Colitis Revealing Mevalonate Kinase Deficiency

Levy

Arion²,

Berrebi

et al. 2013

Pediatrics

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Hyperimmunoglobulinemia D is the less severe form of mevalonate kinase deficiency (MKD) caused by recessive inherited mutation in the mevalonate kinase gene. Hyperimmunoglobulinemia D is characterized by febrile attacks, often associated with transient digestive manifestations, such as abdominal pain, diarrhea, and vomiting. Here we report for the first time 2 patients with MKD revealed by severe neonatal colitis. Both patients had chronic bloody diarrhea and failure to thrive; 1 patient since the age of 1 month and the other since the age of 12 days. Total parenteral nutrition was required. A marked elevation of acute phase reactants was present, and no evidence of infection was found. In patient 1, ileocolonoscopy revealed ulcerative colitis at the age of 5 months. Patient 2 suffered from enterocolitis and shock, associated with multiple bowel adhesions at age 5 weeks; the rectosigmoidoscopy showed aphtoid lesions of the sigmoid colon. Pathologic findings of colonic biopsies revealed a dense polymorph inflammatory infiltrate associated with deep ulcerations. Febrile attacks occurred 2 months after the onset of digestive symptoms in patient 1, and at onset of disease in patient 2. Genomic sequencing of the mevalonate kinase gene revealed compound heterozygous mutations in both patients. Anti-interleukin-1 agent produced longterm remission of all digestive features and laboratory parameters. This report emphasizes that MKD may be the cause of severe earlyonset inflammatory colitis, and must be considered by physicians, even in the absence of fever, after ruling out infections. Antiinterleukin-1 therapy may result in a dramatic improvement of MKD-related inflammatory bowel disease. Pediatrics 2013;132:e779-e783 AUTHORS:

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Section: Figurementioning

confidence: 99%

Severe Early-Onset Colitis Revealing Mevalonate Kinase Deficiency

Levy

Arion²,

Berrebi

et al. 2013

Pediatrics

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“…Hyams et al confirmed the efficacy and safety of infliximab in the treatment of moderate-to-severe pediatric UC. 21 After the induction, clinical response was induced in 73.3% of patients, and 28.6% of children were in clinical remission in the 54 th week. Szychta et al in 2012 presented the results of the first in Poland infliximab therapy of severe pediatric UC.…”

Section: Discussionmentioning

confidence: 99%

Induction and Maintenance Infliximab Therapy in Children with Moderate to Severe Ulcerative Colitis. Retrospective, Multicenter Study

Iwańczak¹,

Kierkuś²,

Ryżko³

et al. 2017

Adv Clin Exp Med

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“…46,52 Eight out of the 10 trials (see Figure 3) were considered to be at low risk of performance bias because there was reporting to indicate that participants and personnel were blinded to participants' treatment allocation. Two trials were considered at unclear risk of bias for this domain; one because there was no clear statement in the trial report 52 and one because the treatment regimen differed for non-responders at week 8 in AZA arm which could break the blinding. 51 Blinding of the outcome assessment was reported by five trials, 44,45,47,48,50 all of which were considered at low risk of bias for this domain.…”

Section: Quality Of Included Evidencementioning

confidence: 99%

“…A total of 239 participants were randomised to IFX, AZA or combination therapy (with no PBO group included). Outcomes were assessed at weeks 8 and 16. Population: children and adolescents aged 6-17 years (inclusive) with severely active ulcerative colitis who have had an inadequate response to conventional therapy including corticosteroids and mercaptopurine or azathioprine, or who are intolerant to, or have medical contraindications against, such therapies A single Phase III open-label RCT was identified for the paediatric population (T72 52 ) which evaluated the use of IFX in maintenance therapy. All patients received the licensed IFX induction regimen before being randomised to one of two IFX maintenance regimens.…”

Section: Act1mentioning

confidence: 99%

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Infliximab, adalimumab and golimumab for treating moderately to severely active ulcerative colitis after the failure of conventional therapy (including a review of TA140 and TA262): clinical effectiveness systematic review and economic model

Archer

Tappenden

Ren

et al. 2016

Health Technol Assess

143

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BackgroundUlcerative colitis (UC) is the most common form of inflammatory bowel disease in the UK. UC can have a considerable impact on patients’ quality of life. The burden for the NHS is substantial.ObjectivesTo evaluate the clinical effectiveness and safety of interventions, to evaluate the incremental cost-effectiveness of all interventions and comparators (including medical and surgical options), to estimate the expected net budget impact of each intervention, and to identify key research priorities.Data sourcesPeer-reviewed publications, European Public Assessment Reports and manufacturers’ submissions. The following databases were searched from inception to December 2013 for clinical effectiveness searches and from inception to January 2014 for cost-effectiveness searches for published and unpublished research evidence: MEDLINE, EMBASE, Cumulative Index to Nursing and Allied Health Literature, The Cochrane Library including the Cochrane Systematic Reviews Database, Cochrane Controlled Trials Register, Database of Abstracts of Reviews of Effects, the Health Technology Assessment database and NHS Economic Evaluation Database; ISI Web of Science, including Science Citation Index, and the Conference Proceedings Citation Index-Science and Bioscience Information Service Previews. The US Food and Drug Administration website and the European Medicines Agency website were also searched, as were research registers, conference proceedings and key journals.Review methodsA systematic review [including network meta-analysis (NMA)] was conducted to evaluate the clinical effectiveness and safety of named interventions. The health economic analysis included a review of published economic evaluations and the development of a de novo model.ResultsTen randomised controlled trials were included in the systematic review. The trials suggest that adult patients receiving infliximab (IFX) [Remicade®, Merck Sharp & Dohme Ltd (MSD)], adalimumab (ADA) (Humira®, AbbVie) or golimumab (GOL) (Simponi®, MSD) were more likely to achieve clinical response and remission than those receiving placebo (PBO). Hospitalisation data were limited, but suggested more favourable outcomes for ADA- and IFX-treated patients. Data on the use of surgical intervention were sparse, with a potential benefit for intervention-treated patients. Data were available from one trial to support the use of IFX in paediatric patients. Safety issues identified included serious infections, malignancies and administration site reactions. Based on the NMA, in the induction phase, all biological treatments were associated with statistically significant beneficial effects relative to PBO, with the greatest effect associated with IFX. For patients in response following induction, all treatments except ADA and GOL 100 mg at 32–52 weeks were associated with beneficial effects when compared with PBO, although these were not significant. The greatest effects at 8–32 and 32–52 weeks were associated with 100 mg of GOL and 5 mg/kg of IFX, respectively. For patients in remission following induction, all treatments except ADA at 8–32 weeks and GOL 50 mg at 32–52 weeks were associated with beneficial effects when compared with PBO, although only the effect of ADA at 32–52 weeks was significant. The greatest effects were associated with GOL (at 8–32 weeks) and ADA (at 32–52 weeks). The economic analysis suggests that colectomy is expected to dominate drug therapies, but for some patients, colectomy may not be considered acceptable. In circumstances in which only drug options are considered, IFX and GOL are expected to be ruled out because of dominance, while the incremental cost-effectiveness ratio for ADA versus conventional treatment is approximately £50,300 per QALY gained.LimitationsThe health economic model is subject to several limitations: uncertainty associated with extrapolating trial data over a lifetime horizon, the model does not consider explicit sequential pathways of non-biological treatments, and evidence relating to complications of colectomy was identified through consideration of approaches used within previous models rather than a full systematic review.ConclusionsAdult patients receiving IFX, ADA or GOL were more likely to achieve clinical response and remission than those receiving PBO. Further data are required to conclusively demonstrate the effect of interventions on hospitalisation and surgical outcomes. The economic analysis indicates that colectomy is expected to dominate medical treatments for moderate to severe UC.Study registrationThis study is registered as PROSPERO CRD42013006883.FundingThe National Institute for Health Research Health Technology Assessment programme.

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Induction and Maintenance Therapy With Infliximab for Children With Moderate to Severe Ulcerative Colitis

Cited by 245 publications

References 19 publications

Severe Early-Onset Colitis Revealing Mevalonate Kinase Deficiency

Severe Early-Onset Colitis Revealing Mevalonate Kinase Deficiency

Induction and Maintenance Infliximab Therapy in Children with Moderate to Severe Ulcerative Colitis. Retrospective, Multicenter Study

Infliximab, adalimumab and golimumab for treating moderately to severely active ulcerative colitis after the failure of conventional therapy (including a review of TA140 and TA262): clinical effectiveness systematic review and economic model

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