Using multiple imputation of real-world data to estimate clinical remission in pediatric inflammatory bowel disease.

To evaluate the performance of the multiple imputation (MI) method for estimating clinical effectiveness in pediatric Crohn's disease in the ImproveCareNow registry; to address the analytical challenge of missing data. Simulation studies were performed by creating missing datasets based on fully observed data from patients with moderate-to-severe Crohn's disease treated with non-ustekinumab biologics. MI was used to impute sPCDAI remission statuses in each simulated dataset.

Clinical Outcome Assessments in Pediatric Patients With Ulcerative Colitis and Crohn's Disease Receiving Biologics: A Retrospective Cohort Study.

Background: To assess disease activity, steroid-free remission, and other clinical outcome assessments among pediatric patients with ulcerative colitis (UC) and Crohn's disease (CD) in the ImproveCareNow (ICN) registry.

Methods: Patients aged 2-17 years diagnosed with UC or CD between June 1, 2013 and December 31, 2019 were enrolled if they initiated a biologic after enrollment in the ICN registry and completed at least 12 months follow-up after first maintenance dose. Baseline (at biologic initiation) demographics were summarized using descriptive statistics.

Validating the Simplified Endoscopic Mucosal Assessment for Crohn's Disease: A Novel Method for Assessing Disease Activity.

Background: To demonstrate treatment efficacy in Crohn's disease (CD), regulatory authorities require that trials include an endoscopic remission/response end point; however, standardized endoscopic assessment of disease activity, such as the Simple Endoscopic Score for Crohn's Disease (SES-CD), is not typically recorded by clinicians in practice or outside of clinical trials. The novel Simplified Endoscopic Mucosal Assessment for Crohn's Disease (SEMA-CD) was developed to be easy to use in routine clinical practice and as a trial end point. We conducted a study to assess and validate the reliability and feasibility of SEMA-CD as a measure of endoscopic disease activity.

Quantification of Mucosal Activity from Colonoscopy Reports via the Simplified Endoscopic Mucosal Assessment for Crohn's Disease.

Background: Endoscopic mucosal healing is the gold standard for evaluating Crohn's disease (CD) treatment efficacy. Standard endoscopic indices are not routinely used in clinical practice, limiting the quality of retrospective research. A method for retrospectively quantifying mucosal activity from documentation is needed.

Quality Items Required for Running a Paediatric Inflammatory Bowel Disease Centre: An ECCO Paper.

Background: The importance of a holistic approach with a comprehensive multidisciplinary team, including nutritional and psychosocial support, is becoming well recognised as a key contributor to optimal care in paediatric inflammatory bowel disease [IBD]. The Paediatric committee of ECCO [P-ECCO] aimed to determine important components that would contribute to quality of care in a paediatric IBD centre [henceforth 'quality items'].

Methods: First, a list of items has been generated by a Delphi group of 111 international paediatric IBD experts.

Infliximab Is Not Associated With Increased Risk of Malignancy or Hemophagocytic Lymphohistiocytosis in Pediatric Patients With Inflammatory Bowel Disease.

Background And Aims: Immunosuppressive therapy for inflammatory bowel disease (IBD) in pediatric patients is thought to increase the risk of malignancy and lymphoproliferative disorders, including hemophagocytic lymphohistiocytosis (HLH). We compared unadjusted incidence rates of malignancy and HLH in pediatric patients with IBD exposed to infliximab (IFX) with patients not exposed to biologics and calculated standardized incidence ratios (SIRs).

Methods: We collected and analyzed data from 5766 participants in a prospective study of long-term outcomes of pediatric patients with IBD (NCT00606346), from May 31, 2007 through June 30, 2016.

A Digital Architecture for a Network-Based Learning Health System: Integrating Chronic Care Management, Quality Improvement, and Research.

Introduction: We collaborated with the ImproveCareNow Network to create a proof-of-concept architecture for a network-based Learning Health System. This collaboration involved transitioning an existing registry to one that is linked to the electronic health record (EHR), enabling a "data in once" strategy. We sought to automate a series of reports that support care improvement while also demonstrating the use of observational registry data for comparative effectiveness research.

Health Insurance Paid Costs and Drivers of Costs for Patients With Crohn's Disease in the United States.

Objectives: The cost of medical care for Crohn's disease (CD) and comorbidities in the era of biologics is unclear. We examined insurance claims data from US health plans to understand this relationship.

Methods: Longitudinal CD patient data and reimbursement information from 11 health plans engaged with Accordant Health Services between 2011 and 2013 were analyzed.

Feasibility and validity of the pediatric ulcerative colitis activity index in routine clinical practice.

Objectives: The Pediatric Ulcerative Colitis Activity Index (PUCAI) is a noninvasive disease activity index developed as a clinical trial endpoint. More recently, practice guidelines have recommended the use of PUCAI in routine clinical care. We therefore sought to evaluate the feasibility, validity, and responsiveness of PUCAI in a large, diverse collection of pediatric gastroenterology practices.

Routine use of thiopurines in maintaining remission in pediatric Crohn's disease.

Aim: To evaluate the effectiveness of thiopurines in maintaining steroid-free remission in routine clinical practice.

Methods: The multi-center Pediatric Inflammatory Bowel Disease Network (PIBDNet) cohort study prospectively collected data on thiopurine naïve patients initiating mercaptopurine (6MP) or azathioprine. Patients with a diagnosis of Crohn's disease (CD) were included in our study upon entering remission as determined by physician global assessment (PGA) within 365 d of initiation of thiopurines.

PEDSnet: how a prototype pediatric learning health system is being expanded into a national network.

Except for a few conditions, pediatric disorders are rare diseases. Because of this, no single institution has enough patients to generate adequate sample sizes to produce generalizable knowledge. Aggregating electronic clinical data from millions of children across many pediatric institutions holds the promise of producing sufficiently large data sets to accelerate knowledge discovery.

Effectiveness of anti-TNFα for Crohn disease: research in a pediatric learning health system.

Objectives: ImproveCareNow (ICN) is the largest pediatric learning health system in the nation and started as a quality improvement collaborative. To test the feasibility and validity of using ICN data for clinical research, we evaluated the effectiveness of anti-tumor necrosis factor-α (anti-TNFα) agents in the management of pediatric Crohn disease (CD).

Methods: Data were collected in 35 pediatric gastroenterology practices (April 2007 to March 2012) and analyzed as a sequence of nonrandomized trials.

Appropriateness of emergency department use in pediatric inflammatory bowel disease: a quality improvement opportunity.

Objectives: We sought to characterize emergency department (ED) encounters for pediatric inflammatory bowel disease (IBD) to identify areas for prevention.

Methods: Retrospective chart review of 5 consecutive ED encounters at 7 centers was performed.

Results: Of 35 unique encounters by 32 patients, 3 main factors contributed to ED utilization: disease severity or course, day or time of care, and physician instruction.

Seasonality and pediatric inflammatory bowel disease.

Objectives: Seasonal and geographic variations of inflammatory bowel disease (IBD) exacerbations have been described in adults, with inconsistent findings. We sought to determine whether disease activity in pediatric-onset IBD is associated with a seasonal pattern.

Methods: We examined children with Crohn disease (CD) and ulcerative colitis (UC) using data from the ImproveCareNow Collaborative between December 2008 and November 2010.

Implementable strategies and exploratory considerations to reduce costs associated with anti-TNF therapy in inflammatory bowel disease.

A health care system is needed where care is based on the best available evidence and is delivered reliably, efficiently, and less expensively (best care at lower cost). In gastroenterology, anti-tumor necrosis factor agents represent the most effective medical therapeutic option for patients with moderate-to-severe inflammatory bowel disease (IBD), but are very expensive and account for nearly a quarter of the cost of IBD care, representing a major area of present and future impact in direct health care costs. The ImproveCareNow Network, consisting of over 55 pediatric IBD centers, seeks ways to improve the value of care in IBD, curtailing unnecessary costs and promoting better health outcomes through systematic and incremental quality improvement initiatives.

Exemplar pediatric collaborative improvement networks: achieving results.

A number of pediatric collaborative improvement networks have demonstrated improved care and outcomes for children. Regionally, Cincinnati Children's Hospital Medical Center Physician Hospital Organization has sustained key asthma processes, substantially increased the percentage of their asthma population receiving "perfect care," and implemented an innovative pay-for-performance program with a large commercial payor based on asthma performance measures. The California Perinatal Quality Care Collaborative uses its outcomes database to improve care for infants in California NICUs.

What's in a name generator? Choosing the right name generators for social network surveys in healthcare quality and safety research.

Background: Interest in the use of social network analysis (SNA) in healthcare research has increased, but there has been little methodological research on how to choose the name generators that are often used to collect primary data on the social connection between individuals for SNA.

Objective: We sought to determine a minimum set of name generators sufficient to distinguish the social networks of a target population of physicians active in quality improvement (QI).

Methods: We conducted a pilot survey including 8 name generators in a convenience sample of 25 physicians active in QI to characterize their social networks.

Role of sex in the treatment and clinical outcomes of pediatric patients with inflammatory bowel disease.

Objective: To examine sex differences in medical therapy and clinical outcomes in pediatric patients with inflammatory bowel disease (IBD).

Methods: We performed a cross-sectional analysis of children with Crohn disease (CD) and ulcerative colitis (UC) using data from the ImproveCareNow Network collected between May 2007 and May 2010. Clinical remission, disease severity, body mass index (BMI) z scores, normal height velocity, and medication use were analyzed by sex and age.

Safety and efficacy of adalimumab for moderate to severe Crohn's disease in children.

Background & Aims: The IMAgINE 1 study (NCT00409682) evaluated the safety and efficacy of adalimumab double-blind maintenance dosing regimens following open-label induction for pediatric patients with moderate to severe Crohn's disease (CD).

Methods: We studied 192 patients with Pediatric Crohn's Disease Activity Index (PCDAI) scores >30 for whom conventional treatment was unsuccessful. Patients received open-label induction therapy with subcutaneous adalimumab at weeks 0 and 2 (160 mg and 80 mg, or 80 mg and 40 mg, for body weight ≥40 kg or <40 kg).