Minneci PC et al. Association of Nonoperative management Using Antibiotic Therapy vs Laparoscopic Appendectomy With Treatment Success and Disability Days in Children with Uncomplicated Appendicitis. JAMA. 2020; doi: https://doi.org/10.1001/jama.2020.10888
John S. Schieffelin, MD, Associate Professor of Pediatrics, Section of Infectious Diseases, Tulane University School of Medicine
Appendicitis remains one of the most common indications for surgery in children with tens of thousands of appendectomies performed in the U.S. each year. This procedure is associated with the risk of anesthesia as well as post-operative pain and post-operative complications in up to 15% of patients. On the other hand, non-operative management is associated with less post-treatment disability and is successful in up to 75% of patients at one year. Most children and adolescents with appendicitis undergo appendectomies rather than non-operative management with antibiotics alone. However, it is unknown if one treatment modality over the other is preferred by patients and their families when the potential risks and benefits of both are known. This manuscript looks at the success rate of non-operative management for appendicitis and compares the differences in disability days and complications. More interestingly, it also compares the patient and family satisfaction between surgical and non-surgical management of appendicitis.
This prospective, nonrandomized, multi-institutional study was conducted at ten children’s hospitals in the U.S. The hypothesis was that non-operative management would have a success rate of 75% and would be associated with fewer disability days and complications than urgent appendectomy. This Patient-Centered Outcomes Research Institute (PCORI)-funded project sought input from patients and their families. Therefore, the authors tested a second hypothesis: quality of life and health care satisfaction as scored by the patients and their families would be significantly different at one year between the surgical and nonsurgical groups. Because a large multi-disciplinary team felt that patients and their families would have strong preferences for either surgery or antibiotic management, patients were not randomized.
Children aged 7 to 17 years old who were diagnosed with uncomplicated appendicitis were eligible for enrollment. A standardized script was read to patients and their families about the two treatment options. Patients and their families then chose into which arm of the study they were enrolled: non-operative group or surgical group. The non-operative group received standardized intravenous antibiotics and, when clinical improvement was recognized, their diet was advanced and they were switched to oral antibiotics. The operative group had urgent laparoscopic appendectomy with standardized, intravenous antibiotics. Failure of non-operative management was defined as lack of improvement after 24 hours of IV antibiotics or clinical deterioration. At one year, success rate of non-operative management was defined as not undergoing appendectomy within one year of enrollment. Outcomes were assessed with regression analysis using inverse probability of treatment weighting to adjust for differences between treatment groups for all outcome assessments as patients were not randomized.
1068 children were enrolled. Of these 35% of families chose non-operative management. Although non-randomized, the two groups were very similar. However, white patients were over-represented in the operative group. During the initial hospitalization, the success rate of non-operative management was 85%. However, at one year, this success rate decreased to 67%. Sixteen patients (4.3%) in the non-operative group, requested surgery during their initial hospitalization. When those patients were excluded from the analysis, the success rate of non-operative management increased to 89% at 30 days and 70% at one year. The number of reported disability days was significantly fewer in the non-operative group than the operative group at both 30 days (3.3 vs 6.5 days; p<.001) and at one year (6.6 vs 10.9 days; p<.001). Patients and family members in the non-operative group reported higher health-related quality of life scores in the non-operative group at 30 days but not at one year. Healthcare satisfaction scores at 30 days were not significantly different between the two groups at either time point. However, satisfaction with decision scores were significantly lower among the non-operative group at both 30 days (27.6 vs 28.7; p<.001) and one year (27.7 vs 28.5; p=.006).
This study highlights several interesting aspects of patient care related to a specific diagnosis which in this case was acute appendicitis. First, while a success rate of 63% may appear low, it was still associated with significantly fewer disability days in both the short and long term. Surgery results in a prolonged recovery time when compared to antibiotic management alone. Even though there is a 33% risk of failure, the shorter recovery time associated with non-operative management remains significant when considering all children. Second, although the difference was small, the health-related quality of life scores at 30 days were significantly higher in the non-operative group, but there was no difference in healthcare satisfaction scores. Third, despite having fewer disability days and higher quality of life scores, satisfaction with decision scores were lower among the non-operative group. These last two points demonstrate that complex emotions are involved in illness and healthcare. This study demonstrated that non-operative management results in fewer disability days than operative management and yet family members were not more satisfied with their decisions or with the healthcare their children received.
While this study design was very well thought out and included input from many different stakeholders, several limitations remain. First, their loss to follow-up was 23% at 30 days and 25% at one year. This lack of data could result in significant bias. Second, there may be bias in treatment selection. The authors provide a sound justification for not using a randomized approach and, by using a scripted approach, minimize the risk of investigators influencing parental decision-making. However, the risk of bias remains. Additionally, the authors note that these results may only apply to a small proportion of children with acute, uncomplicated appendicitis. Nevertheless, it provides important information about the success of non-operative management of appendicitis and how patients and family members view their decisions, care, recovery time and outcomes.
Randomized controlled trials evaluate patient outcomes at the population level. However, individual patients and their families view the results of their healthcare on an individual and personal level. Medical decision making, especially when it pertains to a pediatric population, is very complex. Physicians must balance the concerns and perspectives of patients and their family members with standard of care treatment and data from recent literature. In addition, physicians from different specialties may have their own preferred practice and biases. PCORI studies such as this one shed light on many of these challenges.