075  Top 10 research priorities for patients with primary mitochondrial disease and their carers. Issue 6 (27th May 2022)

• Record Type: Journal Article
• Authors: Graves, Tracey
• Is Part Of: Journal of neurology, neurosurgery and psychiatry. Volume 93:Issue 6(2022)
• Page Start: A35
• Page End: A36
• Publication Date: 2022-05-27
• Subjects: Neurology -- Periodicals; Nervous system -- Surgery -- Periodicals; Psychiatry -- Periodicals; 616.8
• DOI: 10.1136/jnnp-2022-ABN.112
• Languages: English
• ISSNs: 0022-3050

Abstract

Abstract : Primary mitochondrial diseases encompass a number of clinical presentations with a spectrum of severity, lack effective disease-modifying therapies and have high mortality. It is therefore vital to know that research meets the needs of people with mitochondrial disease. Priority setting partnerships are an established collaborative methodology bringing patients, charity representatives and clinicians together to establish the most pressing and unanswered research priorities. We developed a web-based questionnaire, asking patients affected by primary mitochondrial disease, their carers and clinicians to pose their research questions. This yielded 709 questions from 50 patients, 47 carers and 50 clinicians. These were grouped into themes and filtered to exclude unanswerable questions. A literature search was performed to determine which of these questions are unanswered. This process resulted in 42 answerable questions. Patients, carers and clinicians were invited again to prioritise these questions and rank them from 'important' to 'less important' using a web-platform. This identified 24 questions which were then taken forward to a face-to-face workshop attended by a diverse range of patients, carers, charity representatives and clinicians, where the relative merits and strengths of each question was debated to create a definitive 'top 10 of unanswered research questions for primary mito- chondrial disease'. [e-mail]