Alternate Endpoints and Clinical Outcome Assessments in Pediatric Ulcerative Colitis Registration Trials

Sun, Haiming; Lee, Jessica J.; Papadopoulos, Elektra J.; Lee, Catherine S.; Nelson, Robert M.; Sachs, Hari Cheryl; Rodríguez, William J.; Mulberg, Andrew E.

doi:10.1097/mpg.0000000000000186

Cited by 9 publications

(14 citation statements)

References 16 publications

(40 reference statements)

Supporting

Mentioning

Contrasting

Order By: Relevance

“…Recently, physicians at regulatory agencies have suggested that the PUCAI may not be an optimal measure of disease activity in clinical trials[8]. The PUCAI is a clinician reported outcome rather than patient reported outcome.…”

Section: Introductionmentioning

confidence: 99%

Correlation of endoscopic disease severity with pediatric ulcerative colitis activity index score in children and young adults with ulcerative colitis

Kerur¹,

Litman²,

Stern³

et al. 2017

WJG

View full text Add to dashboard Cite

AIMTo investigate of pediatric ulcerative colitis activity index (PUCAI) in ulcerative colitis correlate with mucosal inflammation and endoscopic assessment of disease activity (Mayo endoscopic score).METHODSWe reviewed charts from ulcerative colitis patients who had undergone both colonoscopy over 3 years. Clinical assessment of disease severity within 35 d (either before or after) the colonoscopy were included. Patients were excluded if they had significant therapeutic interventions (such as the start of corticosteroids or immunosuppressive agents) between the colonoscopy and the clinical assessment. Mayo endoscopic score of the rectum and sigmoid were done by two gastroenterologists. Inter-observer variability in Mayo score was assessed.RESULTSWe identified 99 patients (53% female, 74% pancolitis) that met inclusion criteria. The indications for colonoscopy included ongoing disease activity (62%), consideration of medication change (10%), assessment of medication efficacy (14%), and cancer screening (14%). Based on PUCAI scores, 33% of patients were in remission, 39% had mild disease, 23% had moderate disease, and 4% had severe disease. There was “moderate-substantial” agreement between the two reviewers in assessing rectal Mayo scores (kappa = 0.54, 95%CI: 0.41-0.68).CONCLUSIONEndoscopic disease severity (Mayo score) assessed by reviewing photographs of pediatric colonoscopy has moderate inter-rater reliability, and agreement was less robust in assessing patients with mild disease activity. Endoscopic disease severity generally correlates with clinical disease severity as measured by PUCAI score. However, children with inflamed colons can have significant variation in their reported clinical symptoms. Thus, assessment of both clinical symptoms and endoscopic disease severity may be required in future clinical studies.

show abstract

Section: Introductionmentioning

confidence: 99%

Correlation of endoscopic disease severity with pediatric ulcerative colitis activity index score in children and young adults with ulcerative colitis

Kerur¹,

Litman²,

Stern³

et al. 2017

WJG

View full text Add to dashboard Cite

show abstract

“…The challenge encountered with these conditions is that the pathophysiology is often less well-understood and pediatric validated clinical endpoints are often lacking. 16,17 …”

Section: Discussionmentioning

confidence: 99%

Characteristics and Changes of Pediatric Therapeutic Trials under the Best Pharmaceuticals for Children Act

Avant

Wharton

Murphy

2018

The Journal of Pediatrics

View full text Add to dashboard Cite

“…However, consensus on the definition of successful treatment outcome (clinical response and/or remission) and col-laboration in the development of well-defined and reliable measures of signs and symptoms for use in conjunction with endoscopic parameters of mucosal healing has become necessary to facilitate pediatric IBD drug development [12]. Endoscopy in pediatric IBD provides a more definitive diagnosis and disease extent evaluation, assesses therapeutic efficacy, and leads to a targeted therapy [13].…”

Section: Clinical Outcome Assessmentmentioning

confidence: 99%

Precision medicine for pediatric inflammatory bowel disease: a perspective

Choe

2020

Precis Future Med

View full text Add to dashboard Cite

There has been a growing need to utilize the concept of "precision medicine" in the treatment of inflammatory bowel disease (IBD). In practice, precision medicine for IBD comprises prediction and personalizing therapies. Prediction is divided into two subcategories: pre-treatment investigation and monitoring response to therapy. Pre-treatment prediction includes clinical outcome assessment, investigation of factors for poor outcome, magnetic resonance enterography, and measurement of TPMT, NUDT15, and other biologic markers. Mucosal healing or transmural healing is considered the "treat-to-target" approach in IBD. To achieve "treat-to-target," the "top-down strategy" is recommended in patients with pediatric Crohn's disease, because the therapeutic window of opportunity may be shorter than that generally believed. We are unable to state that we are truly utilizing biologics in a perfect manner until both "treat-to-target" and "top-down strategy" are supported by "therapeutic drug monitoring (TDM)," a key component of personalizing therapy for IBD. Proactive TDM, or more precisely, model-based proactive dosing with point-of-care assay will soon emerge as the new standard for IBD treatment. It is speculated that "treat-to-target," "top-down strategy," and "TDM" may aid personalized therapy to achieve outstanding improvement in patients with pediatric IBD.

show abstract

Alternate Endpoints and Clinical Outcome Assessments in Pediatric Ulcerative Colitis Registration Trials

Cited by 9 publications

References 16 publications

Correlation of endoscopic disease severity with pediatric ulcerative colitis activity index score in children and young adults with ulcerative colitis

Correlation of endoscopic disease severity with pediatric ulcerative colitis activity index score in children and young adults with ulcerative colitis

Characteristics and Changes of Pediatric Therapeutic Trials under the Best Pharmaceuticals for Children Act

Precision medicine for pediatric inflammatory bowel disease: a perspective

Contact Info

Product

Resources

About