Description of the goods or services required
19/120 Relapsing polymyalgia rheumatica
The aim of the HTA Programme is to ensure that high quality research information on the effectiveness, costs and broader impact of health technology is produced in the most efficient way for those who use, manage, provide care in or develop policy for the NHS. Topics for research are identified and prioritised to meet the needs of the NHS. Health technology assessment forms a substantial portfolio of work within the National Institute for Health Research and each year about fifty new studies are commissioned to help answer questions of direct importance to the NHS. The studies include both primary research and evidence synthesis.
What are the optimal management options for patients with polymyalgia rheumatica who have relapsed during corticosteroid therapy?
1.Intervention: Options could include non-biologic disease-modifying anti-rheumatic drugs as add-on therapies (e.g. methotrexate, leflunomide). Applicants to define and justify choice and dosing of treatment regimen(s).
2.Patient group: Patients with polymyalgia rheumatica with recurrence during corticosteroid therapy (to be defined and justified by applicants).
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: Primary and secondary care, taking into account shared care protocols.
4.Control: Usual care as defined and justified by applicants.
5.Study design: Randomised controlled trial with an internal pilot phase to test the ability to recruit and randomise. Clear stop/go criteria should be provided to inform progression from pilot to full trial. Applicants to give consideration to study designs that would allow the comparison of different therapeutic pathways.
6.Important outcomes: Disease relapse (pain, stiffness); sustained discontinuation of corticosteroids; cumulative steroid dose; time to disease remission; physical function; corticosteroid-related adverse events; short-term serious adverse effects and withdrawals due to adverse events.
Other outcomes: Quality of life; work participation; cost-effectiveness; development of giant cell arteritis.
7.Minimum duration of follow-up: Applicants to define and justify.
Longer-term follow up: If appropriate, researchers should consider obtaining consent from participants to allow future follow up through efficient means (such as routine data) as part of a separately funded study.
Polymyalgia rheumatica is an inflammatory condition affecting the muscles, particularly in the shoulders, neck and pelvic girdle. It most commonly occurs in older people of Scandinavian/northern European descent. Patients have severe pain and stiffness in the muscles as well as non-specific symptoms such as fatigue and loss of appetite. Increases in acute phase reactants such as C-reactive protein are common. The aetiology of polymyalgia rheumatica is unclear.
Polymyalgia rheumatica is usually treated with corticosteroids which act to rapidly reduce inflammation, improving symptoms in the majority of patients. However, relapse is common following reduction of corticosteroid dose and patients are often treated for up to two years. As a result, they are at risk of adverse effects such as increased blood pressure, stomach ulcers and osteoporosis, the treatment of which potentially requires further use of NHS resources.
Management of polymyalgia rheumatica with steroid-sparing agents such as disease-modifying anti-rheumatic drugs is therefore an attractive option. However, there is limited high quality evidence to demonstrate a benefit of using such agents in this condition. The addition of methotrexate to corticosteroids has been investigated in a few randomised studies that have reported mixed results. Evidence showing a benefit of treatment with leflunomide is limited to small case series. The HTA programme would therefore like to commission primary research to address this evidence gap and to inform clinical practice in this area.
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 firstname.lastname@example.org.