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February 16, 2021; 96 (7) ArticleOpen Access

Association of GBA Genotype With Motor and Functional Decline in Patients With Newly Diagnosed Parkinson Disease

Jodi Maple-Grødem, Ingvild Dalen, Ole-Bjørn Tysnes, Angus D. Macleod, Lars Forsgren, Carl E. Counsell, Guido Alves
First published December 21, 2020, DOI: https://doi.org/10.1212/WNL.0000000000011411
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Figures

  • Figure 1
    Figure 1 Flowchart of Study Participants

    Overview of patient inclusion from baseline until the 7-year visit. The number of patients in the study at each visit is shown. Withdrawals and deaths between visits are shown in dashed boxes. The number of patients carrying a GBA polymorphism/mutation is shown in bold. The flowchart is simplified for readability.

  • Figure 2
    Figure 2 Prediction of Unified Parkinson’s Disease Rating Scale (UPDRS) Motor Score Over Time

    Average predicted UPDRS motor scores with confidence bands for the first 7 years after diagnosis of PD for carriers of GBA variants (red, diamonds) and noncarriers (blue, triangles).

  • Figure 3
    Figure 3 Reduced Trial Size in GBA-Targeted Clinical Trials Compared to the Traditional “All-Comer” Design

    Required sample size for clinical trials enrolling only carriers of GBA variants (red diamonds) or an “all-comer” design with nonselected patients with Parkinson disease (PD) (black squares) across varying levels of power to detect a between–within subjects interaction effect. A trial recruiting only PD carriers of a GBA variant could reduce the size required by threefold for an intervention indicated to halve the rate of motor decline, measured by the Unified Parkinson’s Disease Rating Scale (UPDRS) motor score.

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