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Qualitative assessment of the clinical meaningfulness of MDS-UPDRS part III items in early-stage Parkinson's
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DOI：10.1177/1877718x261453177.png)
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Abstract
En 中文
<jats:sec>
<jats:title>Introduction</jats:title>
<jats:p>Developers of disease-modifying therapies (DMTs) for early-stage Parkinson's are in dialogue with regulators to identify the relative merits of the Movement Disorder Society – Unified Parkinson's Disease Rating Scale (MDS-UPDRS) for detecting clinically meaningful changes in patients’ daily functioning during early-stage disease. We report the first qualitative assessment of MDS-UPDRS part III items’ clinical meaningfulness for use in DMT pivotal trials.</jats:p>
</jats:sec>
<jats:sec>
<jats:title>Methods</jats:title>
<jats:p>Semi-structured interviews were conducted with 40 patients and 20 movement disorder neurologists. Evidence was synthesized using an interpretation framework to evaluate items according to four appraisal components.</jats:p>
</jats:sec>
<jats:sec>
<jats:title>Results</jats:title>
<jats:p>
Based on synthesized evidence for each appraisal component:
<jats:list list-type="order">
<jats:list-item>
<jats:p>
Patient feedback indicated all items were at least somewhat
<jats:bold>meaningful to patients</jats:bold>
; 12/18 items were classified as “highly meaningful” given their linkage to motor/daily activities functioning. See
<jats:ext-link xmlns:xlink="http://www.w3.org/1999/xlink" ext-link-type="uri" xlink:href="https://journals.sagepub.com/doi/full/10.1177/1877718X261453177#supplementary-materials-1">
<jats:bold>Interactive Supplement</jats:bold>
</jats:ext-link>
.
</jats:p>
</jats:list-item>
<jats:list-item>
<jats:p>
4/18 items were endorsed by >90% of neurologists as
<jats:bold>clinically relevant in early-stage Parkinson's</jats:bold>
(“highly relevant”).
</jats:p>
</jats:list-item>
<jats:list-item>
<jats:p>
Evidence from neurologists was supportive of 6/18 items in terms of their
<jats:bold>usefulness for capturing disease progression</jats:bold>
(“highly useful”).
</jats:p>
</jats:list-item>
<jats:list-item>
<jats:p>
Neurologist feedback suggested
<jats:bold>response option distinction/assessment specificity</jats:bold>
is inconsistent across items.
</jats:p>
</jats:list-item>
</jats:list>
</jats:p>
<jats:p>Together, qualitative evidence endorsed the value of items 3.3 Rigidity, 3.4 Finger tapping, 3.5 Hand movements, 3.6 Pronation-supination, 3.7 Toe tapping, and 3.8 Leg agility across all four appraisal components.</jats:p>
</jats:sec>
<jats:sec>
<jats:title>Conclusions</jats:title>
<jats:p>A subset of limb-related rigidity/bradykinesia items may be most clinically meaningful for detecting changes in early-stage Parkinson's. Consolidation of quantitative data with evidence from this large qualitative sample will inform final decision-making for a primary efficacy outcome measure in DMT pivotal trials.</jats:p>
</jats:sec>
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