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21/586 Management of Perthes’ disease in children commissioning brief

Contents

Introduction

The aim of the HTA Programme is to ensure that high quality research information on the clinical effectiveness, cost-effectiveness and broader impact of healthcare treatments and tests are produced in the most efficient way for those who plan, provide or receive care from NHS and social care services. The commissioned workstream invites applications in response to calls for research on specific questions which have been identified and prioritised for their importance to the NHS, patients and social care.

Research question

What is the clinical and cost-effectiveness of containment surgery compared with best conservative care in the treatment of Perthes’ disease of the hip?

  1. Intervention: Surgical containment versus conservative care (applicants to define and justify).
  2. Patient groups: Children aged above five years newly diagnosed with Perthes’ disease of the hip, stratified by age (<8 years old and >8 years old) in whom surgery is being considered. Applications are encouraged which include recruitment from geographic populations with high disease burden which have been historically underserved by research activity in this field.
  3. Setting: Secondary care.
  4. Comparator: Interventions to be compared against each other.
  5. Study design: A randomised controlled trial with an internal pilot phase to test key trial processes such as recruitment and adherence. Clear stop/go criteria should be provided to inform progression from pilot to full trial. Applicants may want to suggest an alternative study design if appropriate, which must be fully defined and justified.
  6. Important outcomes: Function (PROMIS Mobility Score) at 3 years; quality of life; patient and carer acceptability.
  7. Other outcomes: Need for further surgery; days lost from usual activities. Existing Core Outcomes should be included amongst the list of outcomes, unless a good rationale is provided to do otherwise. Applicants are encouraged to report recruitment and findings disaggregated by sex (and other demographic factors where relevant).
  8. Minimum duration of follow-up: Three years.
  9. Longer-term follow up: If appropriate, researchers should consider obtaining consent from participants to allow potential future follow up through efficient means (such as routine data) as part of a separately funded study.  

Rationale

Perthes’ disease (also known as Legg-Calvé-Perthes’ disease) is a condition that causes hip pain and disability in children. In the UK around 1,200 children have Perthes’ disease, most commonly in those aged between four and nine years of age, and it affects four times as many boys as girls. It is currently unknown what causes Perthes’ disease. Usually it occurs in only one hip, although approximately 15% of children will have it in both hips.

In children with Perthes’ disease there is a temporary disruption to the blood supply to the head of the femur (the ‘ball’ of the hip’s ‘ball and socket’ joint) and this causes bone cells in the affected area to die, which in turn causes the bone to soften and it can become distorted or fracture.  The child will develop symptoms such as pain in the hip and a limp. 

Over a period of several months, blood supply will return to the bone tissue, generating new tissue which allows the head of the femur to reform and regrow.  However, a deformity may have developed during this process which can lead to difficulties walking, restricted mobility, and pain. Perthes’ disease can have significant negative impacts on the child’s ability to fully participate in school and family activities, and it also increases the risk of the child developing osteoarthritis in the affected hip in the future.

The main aim of treatment for Perthes’ disease is to try to protect the hip joint so that the head of the femur can heal with as little deformity as possible, whilst also reducing pain for the child. Treatment options include conservative treatments such as physiotherapy, walking aids, and watchful waiting, and surgical treatment to cut and realign the bone so it fits better within the pelvis. However, the variation in treatment options and the lack of good quality evidence from research studies means it is not known whether surgical or conservative care leads to superior long-term outcomes for children with Perthes’ disease. As such, both the Royal College of Surgeons and a James Lind Alliance Priority Setting Partnership have highlighted this uncertainty as one of their highest research priorities, the results of which would help inform future practice.

Additional commissioning brief background information

A background document is available that provides further information to support applicants for this call. It is intended to summarise what prompted the call and the existing evidence base, including relevant work from the HTA and wider NIHR research portfolio. It was researched and written on the basis of information from a search of relevant sources and databases, and in consultation with a number of experts in the field. If you would like a copy please email htaresearchers@nihr.ac.uk.

Making an application

If you wish to submit a Stage 1 application for this call, the online application form can be found on the funding opportunities page. To select this call, use the filters on the right of the screen or search using the call name and/or number.

Your application must be submitted on-line no later than 1pm on the 30 March 2022. Applications will be considered by the HTA Funding Committee at its meeting in May 2022.

Guidance notes and supporting information for HTA Programme applications are available.

Important: Shortlisted Stage 1 applicants will be given eight weeks to submit a Stage 2 application. The Stage 2 application will be considered at the Funding Committee in September 2022.

Applications received electronically after 1300 hours on the due date will not be considered.

For commissioned topics, the Programme strongly discourages the practice of the same co-applicant joining more than one competing team. There may be unusual circumstances where the same person could be included on more than on application eg a lead from a named charity or a unique national expert in a condition.

For such exceptions (i) each application needs to state the case as to why the same person is included (ii) the shared co-applicant should not divulge application details between teams and (iii) both teams should acknowledge in their application that they are aware that one of their co-applicants is part of a competing application and that study details have not been shared.

Should you have any queries please contact us by email: htacommissioning@nihr.ac.uk