Background: Although curative, appendectomy is a major intra-abdominal operation with associated perioperative risks, pain, and disability. A nonoperative management strategy with antibiotics alone has the potential to treat the disease effectively with less negative impact on the child and the family.
Objectives: To determine the effectiveness of nonoperative management by characterizing its success rate and comparing treatment-related disability, health-related quality of life (HRQOL), health care satisfaction, and treatment-associated complications of nonoperative management vs surgery in children with uncomplicated appendicitis.
Methods: We conducted a multi-institutional trial with patient choice between antibiotics alone (nonoperative group) and appendectomy (surgery group) in children aged 7 to 17 years with uncomplicated appendicitis. Uncomplicated appendicitis was defined by focal abdominal pain ≤48 hours before receiving antibiotics, a white blood cell count of ≤18 000 cells/μL, and an ultrasound or computed tomography (CT) scan diagnosis of acute appendicitis (appendiceal diameter ≤1.1 cm, with no phlegmon, abscess, or appendicolith). All patients and their caregivers were counseled on each treatment option, and then chose either nonoperative management or surgery. Nonoperative management consisted of hospital admission with at least 24 hours of intravenous (IV) antibiotics, followed by oral antibiotics at home to complete a total antibiotic course of 7 days. Surgical management consisted of hospital admission with IV antibiotics and urgent laparoscopic appendectomy. The 2 primary outcomes assessed at 1 year were disability days (expected difference, 5 days) and success rate of nonoperative management, defined as the proportion of patients who were initially managed nonoperatively and who did not undergo appendectomy by 1 year (lowest acceptable success rate ≥70% based on surgeon input). Secondary outcomes included differences in caregiver disability days, HRQOL, health care satisfaction, rates of complicated appendicitis, and posttreatment-related complications. Outcomes were assessed at 30 days and 1 year. Inverse probability of treatment weighting (IPTW) was used to adjust for differences between treatment groups for all outcome comparative assessments. In addition, IPTW was used to account for differences between treatment groups to create an “unbiased” cohort for the single-group outcome of the success of nonoperative management.
Results: We enrolled 1068 patients from May 2015 through November 2018 across 10 tertiary care children's hospitals, with 370 patients choosing nonoperative management and 698 choosing surgery. Patients choosing nonoperative management were younger, more often non-White (Black or other race), had caregivers with higher education (college or higher), and were more likely to have undergone an ultrasound rather than a CT scan. The success rate of nonoperative management was analyzed in all 370 patients so treated, and disability days were analyzed in 806 of the 1098 participating patients (follow-up rates: nonoperative, 284/370 [77%]; surgery, 522/698 [75%]).
Primary outcomes: After IPTW, the success rate of nonoperative management at 1 year was 67.1% (96% CI, 61.5%-72.3%; P = .86 using lowest acceptable success rate ≥70%). Nonoperative management was associated with significantly fewer patient disability days at 1 year than with surgery (mean, 6.6 vs 10.9 days, respectively; difference = −4.3 days (99% CI, −6.17 to −2.43).
Secondary outcomes: Health care satisfaction scores at 30 days were not different between groups. Although differences were observed in decision satisfaction scores at 30 days and 1 year, these are not likely clinically meaningful differences, as both groups had scores indicative of very high satisfaction with their decision. HRQOL scores reported by the child and caregiver at 30 days were higher in the nonoperative management group than in the surgery group (child-reported difference = 2.73 [95% CI, 1.00-4.46]; parent proxy difference = 3.20 [95% CI, 1.60-4.81]).
Conclusions: Among children with uncomplicated appendicitis, an initial nonoperative management strategy with antibiotics alone had a success rate of 67.1%, and, compared with urgent surgery, was associated with fewer cumulative disability days at 1 year. However, the prespecified threshold for an acceptable success rate of nonoperative management of ≥70% was not met. Health care satisfaction scores were not different between families choosing surgery and those choosing nonoperative management.
Limitations: The generalizability of these results may be limited because this study was performed specifically at tertiary children's hospitals and because of substantial rates of incomplete follow-up. In addition, there may be residual treatment selection bias despite using robust inferential methods to aid in accounting for treatment confounding bias.
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