New needs-based quality-of-life measure for manifest Huntington’s disease (HD-mQoL) published in the Journal of Neurology
Background
Huntington’s Disease (HD) is a rare inherited neurodegenerative disease which can present with progressive motor, cognitive and psychiatric symptoms, all of which have significant impacts on QoL. Existing Patient Reported Outcome Measures (PROMs) currently used in HD research focus on symptoms, functioning and health-related QoL (HR-QoL); whilst useful for assessing disease status, these measures tend to overlook the lived experience of HD patients.
The HD-mQoL has been developed to produce a holistic assessment of patient value. The unidimensional measure is not only patient-centric but produces a composite score that is effective for monitoring change over time. Thus, the HD-mQoL addresses the limitations of existing measures in order to enhance the quality of patient value data collection in clinical trials, real-world studies, routine clinical care, and wider research.
Project overview
The HD-mQoL was developed as part of the multinational HealthE-RND study. The aim was to develop five disease-specific QoL measures for five different populations impacted by HD: manifest HD patients (HD-mQoL), premanifest gene-expansion carriers (HD-pQoL), partners of manifest HD patient (HD-mQoL-par) and partners of pre-manifest patients (HD-pQoL-par). The latter three measures are nearing completion, alongside a proxy measure for the manifest stage of HD (HD-mQoL-prx). The suite of measures is designed to capture QoL deficits and change across a range of patients and carers affected by the disease.
Three stages were involved in the HD-mQoL development and validation, each of which incorporated patients: (1) Qualitative interviewing (n=51) to generate questionnaire content and item translations (2) Testing face and content validity of the draft measure (n=62) and (3) Validation survey (n=238) and data analysis, using both Rasch Measurement Theory (RMT) and Classical Test Theory (CTT) frameworks.
Measure Profile
|
Attribute |
Detail |
|
Full name (Abbreviation) |
Huntington’s Disease Manifest Quality of Life measure (HD-mQoL) |
|
Development and validation patient population |
HD patients with motor symptoms Age ≥ 18 CAG ≥ 36 Diagnostic Confidence Level of 4
|
|
Measure structure |
23-item, unidimensional, interval-level scale. |
|
Response format |
Dichotomous. Response options: True/Not true |
|
Administration method |
Validated in paper (ePRO version can be approved) |
|
Scaling method |
Rasch analysis demonstrated good model fit (item–trait interaction χ² = 0.391), confirmed unidimensionality, no differential item functioning, no local dependency |
|
Internal consistency (alpha) |
Cronbach’s α = 0.91 |
|
Test-retest reliability |
Test-retest r = 0.87 |
|
Translation versions available |
UK English, Italian, German, Czech, and Dutch (requires additional validation work) |
Click here for the full paper.
For more information about the measure or licensing enquiries please contact gr@galen-research.com
Galen Research Ltd. is a UK based company specialising in the development, validation and translation of disease-specific PROMs, with 35 existing measures adapted for use in up to 75 countries. For nearly 40 years, Galen has developed measures fundamentally underpinned by the patients’ perspective, favouring a single model of QoL (needs-based approach), allowing for the construction of unidimensional scales by applying Rasch analysis. The measures are used globally in clinical trials, HTA and market access submissions, as well as routine clinical care and wider research.
LinkedIn: https://www.linkedin.com/company/galen-research-ltd
Contact: gr@galen-research.com