Core Outcome Measures in Effectiveness Trials

Development of a Core Outcome Set (COS) for the optimisation of treatment for patients with an anorectal malformation (ARM): The ARM and Outcome Review (the ARMOUR-project)

General Information

Summary:
Introduction: An anorectal malformation (ARM) is a congenital abnormality in which the anus and rectum have not developed properly. It amounts to an overall incidence of around 1 in 5000 live born children. The primary treatment of ARM is the surgical restoration of the anatomy. Despite early operative intervention, these children experience many problems during the course of their lives. Faecal or urinary incontinence, obstipation, sexual dysfunction, and socio-emotional and psychological problems are frequent issues encountered by patients with an ARM. Hence, long-term follow-up, including transition into adulthood is required by an experienced team of (para)medical professionals. Suitable care pathways for ARM treatment are currently lacking in the Netherlands and Germany. Such care pathways need to be based on sound medical aspects, clinical evidence, and also on the needs of patients. The aim of the ARMOUR-study is to identify the outcomes and themes concerning ARM that are important from both a medical perspective and from the patients’ view. We then develop a Core Outcome Set (COS) that can support individual ARM management decisions and can be used in future studies evaluating the effectiveness of the management of ARM.

Methods/Design: Firstly, the ARMOUR-study will conduct a systematic review of the literature to identify a list of outcomes that already have been reported in studies concerning ARM. Secondly, semi-structured qualitative interviews with patients of different age groups and their caregivers will be carried out to ensure the inclusion of outcomes and issues that are considered relevant for patients with an ARM. Thirdly, an international Delphi study, with a panel of medical experts and patients, will be conducted to identify the most important outcomes to be included in the ARM care-pathway and the COS. A stakeholder meeting will be conveyed to reach final consensus on the COS. Finally, the abovementioned phases of the study will lead to the selection and, if appropriate, the development of, patient-reported outcome measures (PROMs), which can be implemented at specified time points during follow-up in order to direct treatment towards patient specific needs. This COS should be assessed and reported in future interventional studies for ARM in order to enhance the availability of comparable data to further facilitate evidence-based patient care.

Discussion: A COS represents the minimum of outcomes that should be measured in interventional studies for a specific area of health. The ARMOUR study will aim to identify a COS that could be used in clinical practice and in future studies of the management and life-long treatment of ARM, thereby promoting better treatment evaluation across hospitals and countries and evidence-based medicine in this clinical area.

Keywords: Anorectal malformation, Core outcome set, Delphi, Consensus method

Contributors:
CBM Rosenberg
SAM Cortenraad
ML Kimman
WG Van Gemert
RGJ Visschers

Further Study Information

Current Stage:
Ongoing
Date:
October 2019 - October 2023
Funding source(s):
To be confirmed

Health Area

Disease Category
Gastroenterology

Disease Name
Anorectal malformation (ARM)

Target Population

Age Range
0 - 100

Sex
Either


Nature / type of Intervention
Surgery

Method(s)

Consensus meeting
Delphi process
Interview
Systematic review

Firstly, the ARMOUR-study will conduct a systematic review of the literature to identify a list of outcomes that already have been reported in studies concerning ARM. Secondly, semi-structured qualitative interviews with patients of different age groups and their caregivers will be carried out to ensure the inclusion of outcomes and issues that are considered relevant for patients with an ARM. Thirdly, an international Delphi study, with a panel of medical experts and patients, will be conducted to identify the most important outcomes to be included in the ARM care-pathway and the COS. A stakeholder meeting will be conveyed to reach final consensus on the COS. Finally, the abovementioned phases of the study will lead to the selection and, if appropriate, the development of, patient-reported outcome measures (PROMs), which can be implemented at specified time points during follow-up in order to direct treatment towards patient specific needs. This COS should be assessed and reported in future interventional studies for ARM in order to enhance the availability of comparable data to further facilitate evidence-based patient care.


Stakeholders Involved

Clinical experts
Epidemiologists
Families
Patient/ support group representatives
Researchers

Study Type

COS for clinical trials or clinical research
COS for practice
Recommendations for outcome measures (measurement/how)

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