Clinical-scientists at Nationwide Children’s Hospital have been approved for a $2.9 million funding award by the Patient-Centered Outcomes Research Institute (PCORI) for a multi-institutional trial of non-operative management of appendicitis.
The study will determine the effectiveness of non-operative management as an alternative first-line therapy for children with uncomplicated acute appendicitis across 10 pediatric institutions and across subgroups based on age, race, ethnicity, socioeconomic status, and rural and urban demography. If non-operative management is proven effective, then more than 60,000 patients affected with uncomplicated acute appendicitis per year could be offered a treatment choice that would allow them to avoid surgery.
The research is an expansion of a study at Nationwide Children’s, which found that three out of four children with uncomplicated acute appendicitis had been successfully treated with antibiotics alone at one year follow-up. In that study, compared to urgent appendectomy, non-operative management was associated with less recovery time, lower health care costs and no difference in the rate of complications at one year.
“Treating appendicitis with antibiotics alone instead of surgery may be preferred by patients and families given its potential for less negative impact on the child and family,” said Peter C. Minneci, MD, co-director of the Center for Surgical Outcomes Research and principal investigator in the Center for Innovation in Pediatric Practice in The Research Institute at Nationwide Children’s. “We will measure the amount of time the child misses from everyday activities and the success of non-operative management at one year. Other important results include disability for the caregiver, quality of life after treatment, satisfaction with care, the percent of patients with complicated appendicitis and treatment complications.”
“Our research team is comprised of various stakeholders, each of which represents a different perspective and are an integral part of our research team. Pediatric patients, families, primary care practitioners, specialized physicians, payers, and health educators from various demographics all agreed we should perform a patient choice trial that allows for families to choose the best treatment option for their child,” said Katherine J. Deans, MD, co-director of the Center for Surgical Outcomes Research and principal investigator in the Center for Innovation in Pediatric Practice in The Research Institute at Nationwide Children’s. “Our stakeholders felt this would respect the family’s preferences for a particular treatment, make patients and families from different backgrounds feel comfortable to participate in the study, and allow our results to be easily used at other hospitals.”
Drs. Minneci and Deans, both assistant professors of surgery and pediatrics in The Ohio State University College of Medicine, say stakeholder partners also helped them choose which results to measure to provide patients and families with the information they need to make an informed decision between non-operative management and surgery.
“This project was selected for PCORI funding not only for its scientific merit and commitment to engaging patients and other stakeholders, but also for its potential to fill an important gap in our health knowledge and give people information to help them weigh the effectiveness of their care options,” said PCORI Executive Director Joe Selby, MD, MPH. “We look forward to following the study’s progress and working with Nationwide Children’s to share the results.”
Nationwide Children’s study was selected for PCORI funding through a highly competitive review process in which patients, clinicians and other stakeholders joined clinical scientists to evaluate the proposals. Applications were assessed for scientific merit, how well they will engage patients and other stakeholders, and their methodological rigor among other criteria.
Nationwide Children’s award has been approved pending completion of a business and programmatic review by PCORI staff and issuance of a formal award contract.
PCORI is an independent, nonprofit organization authorized by Congress in 2010. Its mission is to fund research that will provide patients, their caregivers, and clinicians with the evidence-based information needed to make better-informed healthcare decisions. For more information about PCORI’s funding, visit www.pcori.org.