Multi-institutional Trial of Non-operative Management of Appendicitis

February 7, 2023 updated by: Peter Minneci, Nationwide Children's Hospital

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

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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Detailed 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.

Study Type

Observational

Enrollment (Actual)

1076

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Ohio
      • Columbus, Ohio, United States, 43205
        • Nationwide Children's Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

8 years to 17 years (CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Sampling Method

Probability Sample

Study Population

Children with uncomplicated appendicitis

Description

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)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Surgery
Surgical management with appendectomy consists of hospital admission with initiation of intravenous antibiotics and urgent appendectomy .
Non-operative
Non-operative management consists of hospital admission for observation with a minimum of 24 hours of intravenous antibiotics and a minimum of 12 hours nil per os (NPO). With clinical improvement, patients are switched to oral antibiotics and discharged home with a prescription for oral antibiotics to complete a total antibiotic course of 7 days (including the duration of intravenous antibiotics).
Other: Non-operative
Patients will receive only antibiotics and will not undergo appendectomy unless they do not improve or their appendicitis recurs
Other Names:
  • Non-operative management with antibiotics alone

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Success rate
Time Frame: 1 year
success rate at 1 year is percent of patients choosing non-operative management who have not undergone an appendectomy
1 year
Disability Days
Time Frame: 1 year
Number of days without normal schedule
1 year

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Perforated appendicitis
Time Frame: 1 year
percent of patients with perforated appendicitis at operation
1 year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Nationwide Children's Hospital

Collaborators

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

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

October 1, 2014

Primary Completion (ACTUAL)

November 1, 2019

Study Completion (ANTICIPATED)

November 1, 2023

Study Registration Dates

First Submitted

October 20, 2014

First Submitted That Met QC Criteria

October 21, 2014

First Posted (ESTIMATE)

October 22, 2014

Study Record Updates

Last Update Posted (ESTIMATE)

February 9, 2023

Last Update Submitted That Met QC Criteria

February 7, 2023

Last Verified

February 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • IRB14-00651
  • 1507-31325 (OTHER_GRANT: PCORI)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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