Multi-institutional Trial of Non-operative Management of Appendicitis

Appendicitis Clinical Trial

Official title:

Multi-institutional Trial of Non-operative Management of Uncomplicated Pediatric Appendicitis

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT02271932
• Other study ID #: IRB14-00651
• Secondary ID: 1507-31325
• Status: Active, not recruiting
• Start date: October 1, 2014
• Est. completion date: November 1, 2023

Study information

• Verified date: February 2023
• Source: Nationwide Children's Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

A successful non-operative management strategy for early appendicitis will decrease the number of children requiring surgery and may improve the quality of care related to the treatment of appendicitis. To account for the child-family perspective and treatment preferences, the investigators will perform a study in which patients and their families choose between antibiotics alone (Non-operative group) or appendectomy (Surgery group) at ten U.S. hospitals. This study will determine the effectiveness of non-operative management of early appendicitis with antibiotics alone in children and compare differences in morbidity, disability, quality of life, satisfaction, and cost between families choosing surgery or non-operative management.

Description:

Several randomized controlled trials comparing appendectomy to antibiotics alone in adults with acute appendicitis have been reported from non-U.S. countries. These studies reveal that non-operative management of appendicitis is a safe treatment for appendicitis and is associated with a significantly lower risk of complications than appendectomy. We have recently completed the first study in the U.S. examining the feasibility of non-operative management of uncomplicated appendicitis in children. In our pilot study, patients and their families chose between urgent appendectomy or antibiotics alone for treatment of uncomplicated appendicitis. The success rate of non-operative management was 90% at 30 days and 80% at a median follow-up of 8 months. In addition, patients in the non-operative group demonstrated a significantly faster return to normal activity and reported higher quality of life scores. The current proposal seeks to further investigate the effectiveness of non-operative management with antibiotics alone as a treatment option for children with uncomplicated appendicitis. When randomized trials are not feasible due to strong treatment preferences or when only a small proportion of patients will accept randomization, a parallel group non-randomized study where participants are allocated to their preferred treatment is a valuable alternative. Based on feedback from a multi-disciplinary stakeholder group that was convened to assess the potential impact of family preferences on study outcomes, recruitment, and generalizability, we propose a multi-institutional trial in which patients and their families choose between antibiotics alone (Non-operative group) or appendectomy (Surgery group). We hypothesize that non-operative management will be successful in 75% of patients at 1 year follow-up and will be associated with fewer disability days, higher quality of life scores, and lower costs than appendectomy. This study will enroll 795 patients, age 8-17 years, with uncomplicated appendicitis at 10 children's hospitals. The primary outcomes are: 1) differences in treatment-related disability between non-operative management and surgery in children with uncomplicated appendicitis across 10 children's hospitals; and 2) the success rate of non-operative management of uncomplicated appendicitis at 10 children's hospitals, defined as the percent of patients treated non-operatively who do not undergo an appendectomy. Secondary outcomes include differences between the groups in rates of complicated appendicitis, post-treatment related complications, disability days of the parent, quality of life and healthcare satisfaction measures, and total costs and the incremental cost-effectiveness of non-operative management relative to appendectomy. The proposed study will expand upon our previous pilot study by including patients from both freestanding and non-freestanding children's hospitals with both urban and rural demographies to determine the success rate and morbidity associated with non-operative management in a population that better represents the overall population of children in the U.S. Successful completion of this study may provide evidence for the effectiveness of non-operative management of uncomplicated appendicitis as an alternative first line therapy in children.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 1076
• Est. completion date: November 1, 2023
• Est. primary completion date: November 1, 2019
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 8 Years to 17 Years

Eligibility

Inclusion Criteria:

• English and non-English speaking patients

• Age : 8-17 years

• US or CT confirmed early appendicitis with US showing hyperemia, = 1.1 cm in diameter, compressible or non-compressible, no abscess, no fecalith, no phlegmon or CT showing hyperemia, fat stranding, = 1.1 cm in diameter, no abscess, no fecalith, no phlegmon

• White Blood Cell count > 5,000/µL and = 18,000/µL

• Abdominal pain = 48hours prior to receiving antibiotics

Exclusion Criteria:

• History of chronic intermittent abdominal pain

• Pain > 48 hours prior to first antibiotic dose

• Diffuse peritonitis

• Positive urine pregnancy test

• White Blood Cell = 5,000/µL or = 18,000/µL

• Presence of a fecalith on imaging

• Evidence on imaging studies concerning for evolving perforated appendicitis including abscess or phlegmon

• Communication difficulties (e.g. severe developmental delay)

Related Conditions & MeSH terms

• Appendicitis

Intervention

Other:
• Non-operative
Patients will receive only antibiotics and will not undergo appendectomy unless they do not improve or their appendicitis recurs

Locations

Country	Name	City	State
United States	Nationwide Children's Hospital	Columbus	Ohio

Sponsors (10)

Lead Sponsor	Collaborator
Nationwide Children's Hospital	American Family Children's Hospital, Ann & Robert H Lurie Children's Hospital of Chicago, C.S. Mott Children's Hospital, Children's Hospital and Health System Foundation, Wisconsin, Children's Hospital Medical Center, Cincinnati, Comer Children' Hospital, James Whitcomb Riley Hospital for Children, Kosair Children' Hospital, St. Louis Children's Hospital

Country where clinical trial is conducted

United States, 

References & Publications (3)

Gonzalez DO, Deans KJ, Minneci PC. Role of non-operative management in pediatric appendicitis. Semin Pediatr Surg. 2016 Aug;25(4):204-7. doi: 10.1053/j.sempedsurg.2016.05.002. Epub 2016 May 10. — View Citation

Minneci PC, Mahida JB, Lodwick DL, Sulkowski JP, Nacion KM, Cooper JN, Ambeba EJ, Moss RL, Deans KJ. Effectiveness of Patient Choice in Nonoperative vs Surgical Management of Pediatric Uncomplicated Acute Appendicitis. JAMA Surg. 2016 May 1;151(5):408-15. doi: 10.1001/jamasurg.2015.4534. — View Citation

Minneci PC, Sulkowski JP, Nacion KM, Mahida JB, Cooper JN, Moss RL, Deans KJ. Feasibility of a nonoperative management strategy for uncomplicated acute appendicitis in children. J Am Coll Surg. 2014 Aug;219(2):272-9. doi: 10.1016/j.jamcollsurg.2014.02.031. Epub 2014 Apr 13. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Success rate	success rate at 1 year is percent of patients choosing non-operative management who have not undergone an appendectomy	1 year
Primary	Disability Days	Number of days without normal schedule	1 year
Secondary	Perforated appendicitis	percent of patients with perforated appendicitis at operation	1 year