Pediatric Crohn Disease Activity Index: Responsive to Short-term Change

Kundhal, Pavi S.; Critch, Jeff; Zachos, Mary; Otley, Anthony; Stephens, Derek; Griffiths, Anne M.

doi:10.1097/00005176-200301000-00016

Cited by 54 publications

(51 citation statements)

References 21 publications

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“…Responsiveness of DAIBD change was assessed by calculating effect size statistics, correlation with external measure of change (delta PGA), and the diagnostic utility test. 8,18 The effect size was measured by the difference between mean baseline scores and follow-up scores, divided by the standard deviation of baseline scores. A higher effect size indicated a greater change effect, and as a general rule, a score > 0.8 was considered a large effect, a score of 0.5-0.8 moderate, and a score of 0.2-0.5 small.…”

Section: Discussionmentioning

confidence: 99%

Development, validation, and responsiveness of a novel disease activity index for intestinal Behcetʼs disease

Cheon

Han

Park

et al. 2011

Inflammatory Bowel Diseases

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Section: Discussionmentioning

confidence: 99%

Development, validation, and responsiveness of a novel disease activity index for intestinal Behcetʼs disease

Cheon

Han

Park

et al. 2011

Inflammatory Bowel Diseases

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“…The diagnosis of Crohn disease was established before inclusion with the Porto criteria. 16 Clinical activity was measured by the Pediatric Crohn Disease Activity Index (PCDAI), a validated internationally used index. 16,17 The PCDAI score ranges from 0 to 95, with a score greater than 10 indicating active disease and a score of 30 or more indicating moderate to severe disease.…”

Section: Patientsmentioning

confidence: 99%

“…The primary efficacy endpoints were clinical remission at week 8, measured with the PCDAI and defined by a PCDAI score of 10 or less 16,17 and a reduction in PCDAI score of 25% or greater or 75% or greater, measured at weeks 4 and 8. Secondary outcomes measured at weeks 4 and 8 included mean PCDAI score, C-reactive protein level, erythrocyte sedimentation rate, body mass index, weight for age, the Physician Global Assessment score, and incidence of adverse effects.…”

Section: Primary and Secondary End Pointsmentioning

confidence: 99%

Effect of Thalidomide on Clinical Remission in Children and Adolescents With Refractory Crohn Disease

Lazzerini

Martelossi

Magazzù

et al. 2013

JAMA

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IMPORTANCE Pediatric-onset Crohn disease is more aggressive than adult-onset disease, has high rates of resistance to existing drugs, and can lead to permanent impairments. Few trials have evaluated new drugs for refractory Crohn disease in children.OBJECTIVE To determine whether thalidomide is effective in inducing remission in refractory pediatric Crohn disease. DESIGN, SETTING, AND PATIENTS Multicenter, double-blind, placebo-controlled, randomized clinical trial of 56 children with active Crohn disease despite immunosuppressive treatment, conducted August 2008-September 2012 in 6 pediatric tertiary care centers in Italy. INTERVENTIONS Thalidomide, 1.5 to 2.5 mg/kg per day, or placebo once daily for 8 weeks. In an open-label extension, nonresponders to placebo received thalidomide for an additional 8 weeks. All responders continued to receive thalidomide for an additional minimum 52 weeks. MAIN OUTCOMES AND MEASURES Primary outcomes were clinical remission at week 8, measured by Pediatric Crohn Disease Activity Index (PCDAI) score and reduction in PCDAI by Ն25% or Ն75% at weeks 4 and 8. Primary outcomes during the open-label follow-up were clinical remission and 75% response. RESULTS Twenty-eight children were randomized to thalidomide and 26 to placebo. Clinical remission was achieved by significantly more children treated with thalidomide (13/28 [46.4%] vs 3/26 [11.5%]; risk ratio [RR], 4.0 [95% CI, 1.2-12.5]; P = .01; number needed to treat [NNT], 2.86). Responses were not different at 4 weeks, but greater improvement was observed at 8 weeks in the thalidomide group (75% response, 13/28 [46.4%] vs 3/26 [11.5%]; RR, 4.0 [95% CI, 1.2-12.5]; NNT = 2.86; P = .01; and 25% response, 18/28 [64.2%] vs 8/26 [30.8%]; RR, 2.1 [95% CI,]; NNT = 2.99; P = .01). Of the nonresponders to placebo who began receiving thalidomide, 11 of 21 (52.4%) subsequently reached remission at week 8 (RR, 4.5 [95% CI, 1.4-14.1]; NNT = 2.45; P = .01). Overall, 31 of 49 children treated with thalidomide (63.3%) achieved clinical remission, and 32 of 49 (65.3%) achieved 75% response. Mean duration of clinical remission in the thalidomide group was 181.1 weeks (95% CI, 144.53-217.76) vs 6.3 weeks (95% CI, 3.51-9.15) in the placebo group (P < .001). Cumulative incidence of severe adverse events was 2.1 per 1000 patient-weeks, with peripheral neuropathy the most frequent severe adverse event.CONCLUSIONS AND RELEVANCE In children and adolescents with refractory Crohn disease, thalidomide compared with placebo resulted in improved clinical remission at 8 weeks of treatment and longer-term maintenance of remission in an open-label follow-up. These findings require replication to definitively determine clinical utility of this treatment.

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“…The measure has good reliability, with validity mainly determined by comparison with a global assessment by pediatric gastroenterologists. It also is responsive to clinical change among pediatric patients with CD (25,26). We found no comparable measure of disease activity among pediatric patients with UC, and thus the pediatric gastroenterologists in the consortium agreed on a similar measure, based in part on a published activity index for adult UC (27).…”

Section: Generic Quality Of Lifementioning

confidence: 75%

Measuring quality of life in pediatric patients with inflammatory bowel disease: psychometric and clinical characteristics

2009

Primary Health Care

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Objective-To extend development of a pediatric inflammatory bowel disease (IBD) healthrelated quality of life (HRQoL) measure by determining its factor structure and associations of factors with generic HRQoL measures and clinical variables.Patients and Methods-Cross-sectional survey of children and adolescents ages 8 years to 18 years and their parents attending any of 6 US IBD centers, recruited from either existing registry of age-eligible subjects or visits to participating centers. The survey included generic (Pediatric Quality of Life Inventory) and IBD-specific (Impact Questionnaire) quality of life measures, disease activity, and other clinical indicators. We carried out factor analysis of Impact responses, comparing resulting factors with results on the generic HRQoL and the clinical measures.Results-We included 220 subjects (161 with Crohn disease and 59 with ulcerative colitis). Initial confirmatory factor analysis did not support the 6 proposed Impact domains. Exploratory factor analysis indicated 4 factors with good to excellent reliability for IBD responses: general well-being and symptoms, emotional functioning, social interactions, and body image. Two items did not load well on any factor. The 4 factors correlated well with the Pediatric Quality of Life Inventory and subscales. Children with higher disease activity scores and other indicators of clinical activity reported lower HRQoL.Conclusions-This study provides further characteristics of a HRQoL measure specific to pediatric IBD and indicates ways to score the measure based on the resulting factor structure. The measure correlates appropriately with generic HRQoL measures and clinical severity indicators. KeywordsCrohn disease; Ulcerative colitis; Well-being; Disease activity; Functional status © 2008 by European Society for Pediatric Gastroenterology, Hepatology, and Nutrition and North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition Address correspondence and reprint requests to James Perrin, MD, 50 Staniford Street, Suite 901, Boston, MA 02114 (jperrin@partners.org). The authors report no conflicts of interest. The goals of this study, involving a consortium of pediatric IBD centers, were to extend evaluation of the Impact Questionnaire in a larger population and through factor analysis. We expected that confirmatory factor analysis would support the original 6 domains proposed by the developers of the measure. In addition, we compared the IBD-specific QoL findings with those on a more generic HRQoL measure and with measures of severity of IBD, as further validation of the measure. We hypothesized that the IBD measure would correlate well with broader measures of HRQoL. Furthermore, we expected that children and adolescents with more severe or active disease would report lower QoL. Based on previous research, we also expected that subjects with CD would report worse HRQoL. NIH Public Access PATIENTS AND METHODSThis study sampled children diagnosed with UC or CD followed at 1 of 6 pediatric gastroenterolog...

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Pediatric Crohn Disease Activity Index: Responsive to Short-term Change

Abstract: The PCDAI is responsive to improvement in disease activity in Crohn disease patients over a short interval. As such, the PCDAI is an appropriate instrument to use in pediatric acute treatment trials.

Cited by 54 publications

References 21 publications

Development, validation, and responsiveness of a novel disease activity index for intestinal Behcetʼs disease

Development, validation, and responsiveness of a novel disease activity index for intestinal Behcetʼs disease

Effect of Thalidomide on Clinical Remission in Children and Adolescents With Refractory Crohn Disease

Measuring quality of life in pediatric patients with inflammatory bowel disease: psychometric and clinical characteristics

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