The outcomes of Perthes’ disease: Development of a core outcomes set for clinical trials in Perthes’ disease

AIMS:
To identify a suite of the key physical, emotional, and social outcomes to be employed in clinical practice and research concerning Perthes' disease in children.

METHODS:
The study follows the guidelines of the COMET-Initiative (Core Outcome Measures in Effectiveness Trials). A systematic review of the literature was performed to identify a list of outcomes reported in previous studies, which was supplemented by a qualitative study exploring the experiences of families affected by Perthes' disease. Collectively, these outcomes formed the basis of a Delphi survey (two rounds), where 18 patients with Perthes' disease, 46 parents, and 36 orthopaedic surgeons rated each outcome for importance. The International Perthes Study Group (IPSG) (Dallas, Texas, USA (October 2018)) discussed outcomes that failed to reach any consensus (either 'in' or 'out') before a final consensus meeting with representatives of surgeons, patients, and parents.

RESULTS:
In total, 23 different outcome domains were identified from the systematic review, and a further ten from qualitative interviews. After round one of the Delphi survey, participants suggested five further outcome domains. A total of 38 outcomes were scored in round two of the Delphi. Among these, 16 outcomes were scored over the prespecified 70% threshold for importance (divided into six main categories: adverse events; life impact; resource use; pathophysiological manifestations; death; and technical considerations). Following the final consensus meeting, 14 outcomes were included in the final Core Outcome Set (COS).

CONCLUSION:
Core Outcome Sets (COSs) are important to improve standardization of outcomes in clinical research and to aid communication between patients, clinicians, and funding bodies. The results of this study should be a catalyst to develop high-quality clinical research in order to determine the optimal treatments for children with Perthes' disease. Cite this article: Bone Joint J 2020;102-B(5):611-617.

Contributors

Donato G. Leo
Helen Jones
Rebecca Murphy
Justin Wei Leong
Tina Gambling
Andrew F. Long
Jennifer Laine
Daniel C. Perry

Publication

Journal: The Bone and Joint Journal
Volume: 102
Issue: B(5)
Pages: 611 - 617
Year: 2020
DOI: 10.1302/0301-620X.102B5.BJJ-2020-0072

Further Study Information

Current Stage: Completed
Date: June 2017 - February 2018
Funding source(s): This work is funded by the Liverpool John Moores University Faculty of Engineering & Technology (FET) - Faculty of Sciences (SCS) Joint PhD Studentships 2016/17.

Health Area

Disease Category: Orthopaedics & trauma

Disease Name: Perthes' Disease

Target Population

Age Range: 2 - 16

Sex: Either

Nature of Intervention: Surgery

Stakeholders Involved

- Clinical experts
- Consumers (caregivers)
- Consumers (patients)

Study Type

- COS for clinical trials or clinical research
- COS for practice

Method(s)

- Consensus meeting
- Delphi process
- Interview
- Systematic review

The study follows the guidelines of the COMET-Initiative (Core Outcome Measures in Effectiveness Trials). A systematic review of the literature was performed to identify a list of outcomes reported in previous studies, which was supplemented by a qualitative study exploring the experiences of families affected by Perthes’ disease. Collectively, these outcomes formed the basis of a Delphi survey (two rounds), where 18 patients with Perthes’ disease, 46 parents, and 36 orthopaedic surgeons rated each outcome for importance. The International Perthes Study Group (IPSG) (Dallas, Texas, USA (October 2018)) discussed outcomes that failed to reach any consensus (either ‘in’ or ‘out’) before a final consensus meeting with representatives of surgeons, patients, and parents.

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