Grand Rounds February 13, 2026: The Making of the COMPARE-Pediatric IBD Study (Michael D. Kappelman, MD, MPH)

Speaker

Michael D. Kappelman, MD, MPH
Professor, Pediatric Gastroenterology
University of North Carolina at Chapel Hill

Keywords

PCORnet; PCORI; Inflammatory Bowel Disease; Pediatrics; Common Data Model; Study Design

Key Points

  • Inflammatory Bowel Disease (IBD) is a chronic gastrointestinal condition affecting roughly 100,000 youth in the United States. It has a profound impact on nutrition, growth, physical, and psychosocial development. Anti-TNF biologics are the only FDA-approved advanced therapies for children, and approximately 30% of patients experience treatment failure within 2 years. There’s an urgent need for comparative effectiveness research that can guide treatment decisions when anti-TNF fails.
  • COMPARE-Pediatrics IBD, a PCORnet® study, includes 2 parallel multi-center, prospective cohort studies and retrospective cohort studies. The former, developed with multi-stakeholder input, will compare the effectiveness of emerging therapies in children with IBD; the latter will characterize the safety of these treatments and explore the heterogeneity of treatment effects across subgroups.
  • The study is utilizing PCORnet’s® infrastructure, including Prep-to-Research Queries and the PCORnet® Common Data Model (CDM), to inform the study design; identify administrative efficiencies; support recruitment; ease site burden; assess representativeness of the study population; and otherwise bolster their research.

Discussion Themes

Planning a PCORnet® study is a lot of work (and takes time). Start the process early and know that benefits may be on the back-end.

The study team opted not to conduct a randomized pragmatic trial because they anticipated that desperate families would be reluctant “roll the dice” with randomization and because insurance coverage for expensive off-label medications often dictates which therapy a patient can receive.

While the CDM is effective for structured data (like labs and diagnoses), Dr. Kappelman noted it cannot yet capture nuanced interpretations, such as specific MRI findings, which require more advanced AI or manual review.

Grand Rounds February 7, 2025: Improving Symptom Control in Pediatric Cancer Patients With SSPedi and SPARK (Lillian Sung, MD, PhD)

Speaker

Lillian Sung, MD, PhD
Canada Research Chair in Pediatric Oncology Supportive Care
Division of Haematology/Oncology
Chief Clinical Data Scientist
The Hospital for Sick Children
Ontario, Canada

Keywords

Cancer; Pediatrics; Cancer Symptoms; Symptom Control

Key Points

  • Symptom control in pediatric cancer patients is very poor; almost all children undergoing treatment for cancer will experience difficult symptoms, including fatigue, changes in hunger, pain, and nausea or vomiting.
  • The Multinational Association for Supportive Care in Cancer issued ambitious goals for care of cancer patients in 2030, including routine symptom screening to facilitate timely individualized care and pairing symptom identification with evidence-based treatment.
  • With this goal in mind, the research team developed Symptoms Screening and Pediatrics tool, or SSPedi. SSPedi is a validated instrument for measuring self-reported symptoms that is simple, quick, and designed for use in clinical care.
  • They integrated SSPedi with care pathways through SPARK (Supportive care Prioritization Assessment and Recommendations for Kids), a web-based application that the team used to enroll patients and remind them to report their symptoms via SSPedi.
  • The researchers conducted two randomized controlled trials (RCTs): A cluster RCT in the United States, from July 2021 to August 2023, and an inpatient RCT in Canada, from July 2018 to September 2023. In both cases, they randomized cancer patients, aged 8 to 18, to either symptom screening or usual care. In the U.S. study, they worked with the sites to adapt 14 care pathways.
  • Their primary outcome was the patient’s symptom severity, measured by their SSPedi score.
  • In both trials, the research team found a reduction in symptom severity for the intervention group. The benefits were more pronounced in the U.S. trial, with statistically significant improvements for 12 out of 15 symptoms (compared to 8 out of 15 in the Canadian trial).
  • In the U.S. study, symptom-specific interventions (i.e., treatment was clearly provided to address a given symptom) were most common for hurt or pain; changes in hunger; constipation; peripheral neuropathy; feelings of disappointment or sadness; and nausea and vomiting. The first four symptoms were also identified as being documented or treated more often in the Canadian study, in addition to feelings of crankiness or anger.
  • The research team concluded, following both the Canadian and U.S. studies, that symptom screening improves symptom control and that there was strong support for its integration into routine care.
  • There were a couple of additional takeaways from the U.S. study: 1) Care pathway use has independent effects on improving symptoms and 2) symptom screening was associated with increased emergency department visits – likely due to identification of symptoms requiring medical attention.

Discussion Themes

Despite launching right before the COVID-19 pandemic and the labor-intensive process of adapting the care pathways for each individual site, the team completed accrual for the U.S.-based-study about a year ahead of schedule. Dr. Sung attributed this to enthusiastic support from the sites around operationalizing supportive care and a study design which allowed patients to be enrolled remotely and report their symptoms remotely.

Pragmatic trials are a team sport. Having a range of expertise and experience can provide a research team with an advantage that makes all the difference. For example, Dr. Sung’s team had a connection to Epic Systems leadership, which facilitated a partnership between the two groups and allowed them to pilot SSPedi in remote care management.

February 1, 2023: This Week in PCT Grand Rounds, Insights From the NITRIC Trial in Young Children

Headshot of Luregn SchlapbachIn this Friday’s PCT Grand Rounds, Professor Luregn Schlapbach of the University Children’s Hospital Zurich will present “Pragmatic Trials for Children With Congenital Heart Disease: Insights From the NITRIC Trial.”

The Grand Rounds session will be held on Friday, February 3, 2023, at 1:00 pm eastern.

Schlapbach is head of intensive care and neonatology at the University Children’s Hospital Zurich and a researcher at the University of Queensland. He will discuss the design and results of the NITRIC trial, which examined the effect of nitric oxide on children undergoing cardiac surgery on cardiopulmonary bypass.

Join the online meeting.

September 21, 2022: In PCT Grand Rounds, Birth Outcomes of a Nurse Home Visiting Program in a Medicaid-Eligible Population

Headshot of Dr. Margaret McConnell
Dr. Margarat McConnell

In this Friday’s PCT Grand Rounds, Dr. Margaret McConnell of the Harvard T.H. Chan School of Public Health will present “Effect of an Intensive Nurse Home Visiting Program on Adverse Birth Outcomes in a Medicaid-Eligible Population.” The Grand Rounds session will be held on Friday, September 23, 2022, at 1:00 pm eastern.

Dr. McConnell is an associate professor of global health economics at the Harvard Chan School. Her research uses experimental methods and impact evaluation strategies to answer questions about how to improve health outcomes for marginalized populations.

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February 16, 2022: Pediatric Drug Development Is Focus of PCT Grand Rounds This Week

Headshot of Dr. Perdita Taylor Zapata
Dr. Perdita Taylor-Zapata

In this Friday’s PCT Grand Rounds, Dr. Perdita Taylor-Zapata of the National Institute of Child Health and Human Development (NICHD) will present “Building a Resource: The Process of Developing a Trans-Stakeholder Framework to Enable Pediatric Drug Development.” The Grand Rounds session will be held on Friday, February 18, at 1:00 pm eastern.

Dr. Taylor-Zapata, a board-certified pediatrician, leads the NICHD’s Best Pharmaceuticals for Children Act clinical program, a drug development program that sponsors clinical trials to inform and improve pediatric drug labeling.

Join the online meeting.