Final Research Report
View this project's final research report.
Article Highlight: More than 70,000 children in the United States have surgery each year to treat appendicitis. As reported in JAMA, a PCORI-funded study found that among children with uncomplicated appendicitis, nonsurgical treatment with antibiotics was successful in more than half of those patients, and compared with surgery initiated shortly after hospital admission, was associated with significantly fewer disability days. However, the results after one year did not meet the threshold success rate specified at the outset of the study.
Results of This Project
Related Journal Citations
Peer review of PCORI-funded research helps make sure the report presents complete, balanced, and useful information about the research. It also assesses how the project addressed PCORI’s Methodology Standards. During peer review, experts read a draft report of the research and provide comments about the report. These experts may include a scientist focused on the research topic, a specialist in research methods, a patient or caregiver, and a healthcare professional. These reviewers cannot have conflicts of interest with the study.
The peer reviewers point out where the draft report may need revision. For example, they may suggest ways to improve descriptions of the conduct of the study or to clarify the connection between results and conclusions. Sometimes, awardees revise their draft reports twice or more to address all of the reviewers’ comments.
Peer reviewers commented and the researchers made changes or provided responses. Those comments and responses included the following:
- The reviewers expressed concern about the inconsistent acceptable threshold for establishing the efficacy of nonsurgical treatment; at one point in the report the researchers indicated that the threshold would be a 70 percent success rate for nonsurgical treatment, but in their conclusions the researchers wrote that nonsurgical treatment should be offered when the success rate was more than 50 percent. The researchers explained that in the absence of prior research establishing an acceptable threshold, they used the 70 percent success rate to interest surgeons in study participation. The researchers changed the conclusions to say that the data do not support a success rate that exceeds the preset 70 percent threshold.
- The reviewers questioned the report’s description of success and failure at the one-year follow-up point in the study. The reviewers could not identify whether the researchers had included recurrent appendicitis within one year as part of the failure group even though it was more likely that an appendectomy between initial hospitalization and one year postdischarge was caused by recurrent appendicitis rather than the initial condition. The researchers confirmed that their definition of failure of nonoperative treatment at one year included initial in-hospital failures and suspected recurrent appendicitis. The researchers also added data to the report on negative appendectomy rate, which came from not identifying appendicitis at surgery by histology.
- The reviewers noted that there was an unexpectedly high rate of loss to follow-up in the study and asked for what might explain this and whether the high rate of dropping out posed a risk for selection bias that might have affected the outcomes. The researchers said the high dropout rate may reflect the fact that appendicitis tends to be a time-limited disease and families participating in the study may have lost interest in participating further once the condition resolved. The researchers acknowledged that the high dropout rate could have introduced selection bias but said they believed the bias would equally affect both arms of the study.
- A reviewer questioned whether it was necessary to have a nonrandomized trial design and asked the researchers to justify conducting a patient-choice trial in this population. The researchers acknowledged that other groups have performed randomized controlled trials even with patients who have treatment preferences, but said that in studies of children with appendicitis, less than 40 percent of eligible patients enrolled and those who enrolled disproportionately represented those who preferred a nonsurgical intervention, which was not offered outside the trial setting. The researchers felt that their pragmatic study design enrolled a broader population than otherwise would have participated and therefore leads to more useful estimates of the effectiveness of nonsurgical versus surgical treatment of appendicitis.
Conflict of Interest Disclosures
Patient / Caregiver Partners
- William Hawke
- Nicholas Hawke
- Rebeccah Abanukam
- Trinity Patten
- Amanda Monroe
- Alyssa Gillman
- Diana Godwin
- William Blake Godwin
- Darcy Moulin
- Lorelei Moulin
- Melissa Blom
- Maxwell Blom
- Iluminado Castellano
- Tanner Goodman
- Joshua Montalvo
- Aubrey Gibson
- Jason Gibson
- Aria Gibson
- Liz Sullivan
- Kaleb Boyd
- Luanne Farr
- Nolan Chehak
- Mrs. Caldwell
- Rheya Maurilio Valdes
- Nicolas Valdes
- Lisa Valdes
- Lisa Shrader
Other Stakeholder Partners
- Alisa McQueen, MD, Physician C.
- Stephen Baum, MD, Pediatrician
- Kathryn E. Nuss, MD, Physician
- Sean Gleeson, MD, President of Partners for Kids
- Paul Seese, RN, MSN Home Health Manager
- Robert T. Rohloff, MD, Physician and Director of Quality and Patient Safety
- Lawrence Moss, MD, Surgeon-in-Chief
- Dana Schinasi MD, Pediatric Emergency Medicine
- Darryl Robbins, DO, Physician
- Michael Levas, MD, MS, Assistant Professor of Pediatrics
- Courtney Porter, RN, MS, Clinical Leader at Canal Winchester Urgent
- Gian Musarra, MD, Assistant Professor
- Has Results