Producing a novel iMCD Symptom Burden Scale (ISBUS)
Research type
Research Study
Full title
Producing a novel Symptom Burden Scale for people living with Idiopathic Multicentric Castleman Disease (ISBUS)
IRAS ID
332875
Contact name
Philip Powell
Contact email
Sponsor organisation
University of Sheffield
Clinicaltrials.gov Identifier
ClinicalTrials.gov ID: NCT05995834, Trial registration; iMCD-EUSA-NI-604, RRD protocol Number code
Duration of Study in the UK
1 years, 9 months, 2 days
Research summary
Idiopathic Multicentric Castleman Disease (iMCD) is a rare lymphoproliferative disorder associated with systemic inflammation and organ dysfunction. The high symptom burden of iMCD is associated with varied impacts on daily life for people living with the condition, including in work/education, social life, travel, mobility, personal relationships, and sexual functioning. The disease has an estimated prevalence of between 6.9 and 9.7 people per million. The primary approach to iMCD treatment is symptom control and preventing serious complications.\n\nCurrently, no validated condition-specific measure exists to assess symptom burden in iMCD. A condition-specific symptom burden scale for iMCD would have significant value in routine care and during clinical trials, for assessing disease burden at diagnosis, during progression, and for treatment efficacy. \nThis mixed methods project will develop a novel, valid and reliable symptom burden patient reported outcome measure (PROM) for iMCD. The international project includes the UK, US, Canada, Australia, New Zealand and Brazil, and involves close collaboration between multiple stakeholders, including patients, clinicians, industry representatives and researchers. Important: This application relates to the UK arm of the study. Separate ethics and governance applications will be submitted for data collection in other countries.\nThe research involves developing the content of the PROM using existing literature and incorporating expert opinions. We will assess how well the PROM covers all relevant elements of iMCD symptom burden, through online interviews with people living with iMCD. Resulting revisions to the PROM will be made in consultation with patient and expert advisors. The revised PROM will then be administered to people with iMCD in an online survey to evaluate its performance and inform final decisions on PROM content. The PROM will be readministered to observe change in symptom burden over time. This will be complemented with qualitative interviews to estimate a minimally clinically important difference for the PROM.
REC name
West of Scotland REC 1
REC reference
23/WS/0160
Date of REC Opinion
18 Dec 2023
REC opinion
Further Information Favourable Opinion