A 15-Item modification of the PSP rating scale to improve clinical meaningfulness and statistical performance
Abstract Progressive supranuclear palsy (PSP) is a rare neurodegenerative disorder characterized by physical, cognitive, and behavioral impairments. The PSP Rating Scale (PSPRS) is a widely used and validated, clinical scale to monitor disease progression. Here we show the modification of PSPRS to i...
Saved in:
| Main Authors: | , , , , , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Nature Portfolio
2025-01-01
|
| Series: | Nature Communications |
| Online Access: | https://doi.org/10.1038/s41467-024-55442-0 |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
| _version_ | 1841544458297933824 |
|---|---|
| author | Tien Dam Lili Yang Colin Gillis Yumeng Li John O’Gorman Barbara Kolb Adam L. Boxer Lawrence Golbe Samantha Budd Haeberlein On behalf of the AL-108-231, PROSPERA, and 4RTNI investigators |
| author_facet | Tien Dam Lili Yang Colin Gillis Yumeng Li John O’Gorman Barbara Kolb Adam L. Boxer Lawrence Golbe Samantha Budd Haeberlein On behalf of the AL-108-231, PROSPERA, and 4RTNI investigators |
| author_sort | Tien Dam |
| collection | DOAJ |
| description | Abstract Progressive supranuclear palsy (PSP) is a rare neurodegenerative disorder characterized by physical, cognitive, and behavioral impairments. The PSP Rating Scale (PSPRS) is a widely used and validated, clinical scale to monitor disease progression. Here we show the modification of PSPRS to improve clinical meaningfulness and sensitivity. A conceptual framework was used to select meaningful items. Datasets were analyzed to improve the psychometric properties, understand factor structure, and develop a scoring algorithm. Fifteen items of the PSPRS were selected based on whether the items reflect concepts specific to the disease and those responsive to change over 12 months. Some items were rescored to reflect meaningful changes and to improve discrimination and reliability. The rescored 15-item PSPRS was more interpretable and sensitive to disease progression over 12 months. The approach to develop the 15-item PSPRS provides an example of how to modify an existing clinical outcome assessment for use as a primary endpoint in a clinical trial of a progressive neurological disorder. |
| format | Article |
| id | doaj-art-b21fbf3103904a1a8b8e10ff9cdee8de |
| institution | Kabale University |
| issn | 2041-1723 |
| language | English |
| publishDate | 2025-01-01 |
| publisher | Nature Portfolio |
| record_format | Article |
| series | Nature Communications |
| spelling | doaj-art-b21fbf3103904a1a8b8e10ff9cdee8de2025-01-12T12:31:02ZengNature PortfolioNature Communications2041-17232025-01-011611910.1038/s41467-024-55442-0A 15-Item modification of the PSP rating scale to improve clinical meaningfulness and statistical performanceTien Dam0Lili Yang1Colin Gillis2Yumeng Li3John O’Gorman4Barbara Kolb5Adam L. Boxer6Lawrence Golbe7Samantha Budd Haeberlein8On behalf of the AL-108-231, PROSPERA, and 4RTNI investigatorsBiogen IncBiogen IncBiogen IncBiogen IncBiogen IncBiogen IncMemory and Aging Center, Department of Neurology, University of CaliforniaRutgers Robert Wood Johnson Medical SchoolBiogen IncAbstract Progressive supranuclear palsy (PSP) is a rare neurodegenerative disorder characterized by physical, cognitive, and behavioral impairments. The PSP Rating Scale (PSPRS) is a widely used and validated, clinical scale to monitor disease progression. Here we show the modification of PSPRS to improve clinical meaningfulness and sensitivity. A conceptual framework was used to select meaningful items. Datasets were analyzed to improve the psychometric properties, understand factor structure, and develop a scoring algorithm. Fifteen items of the PSPRS were selected based on whether the items reflect concepts specific to the disease and those responsive to change over 12 months. Some items were rescored to reflect meaningful changes and to improve discrimination and reliability. The rescored 15-item PSPRS was more interpretable and sensitive to disease progression over 12 months. The approach to develop the 15-item PSPRS provides an example of how to modify an existing clinical outcome assessment for use as a primary endpoint in a clinical trial of a progressive neurological disorder.https://doi.org/10.1038/s41467-024-55442-0 |
| spellingShingle | Tien Dam Lili Yang Colin Gillis Yumeng Li John O’Gorman Barbara Kolb Adam L. Boxer Lawrence Golbe Samantha Budd Haeberlein On behalf of the AL-108-231, PROSPERA, and 4RTNI investigators A 15-Item modification of the PSP rating scale to improve clinical meaningfulness and statistical performance Nature Communications |
| title | A 15-Item modification of the PSP rating scale to improve clinical meaningfulness and statistical performance |
| title_full | A 15-Item modification of the PSP rating scale to improve clinical meaningfulness and statistical performance |
| title_fullStr | A 15-Item modification of the PSP rating scale to improve clinical meaningfulness and statistical performance |
| title_full_unstemmed | A 15-Item modification of the PSP rating scale to improve clinical meaningfulness and statistical performance |
| title_short | A 15-Item modification of the PSP rating scale to improve clinical meaningfulness and statistical performance |
| title_sort | 15 item modification of the psp rating scale to improve clinical meaningfulness and statistical performance |
| url | https://doi.org/10.1038/s41467-024-55442-0 |
| work_keys_str_mv | AT tiendam a15itemmodificationofthepspratingscaletoimproveclinicalmeaningfulnessandstatisticalperformance AT liliyang a15itemmodificationofthepspratingscaletoimproveclinicalmeaningfulnessandstatisticalperformance AT colingillis a15itemmodificationofthepspratingscaletoimproveclinicalmeaningfulnessandstatisticalperformance AT yumengli a15itemmodificationofthepspratingscaletoimproveclinicalmeaningfulnessandstatisticalperformance AT johnogorman a15itemmodificationofthepspratingscaletoimproveclinicalmeaningfulnessandstatisticalperformance AT barbarakolb a15itemmodificationofthepspratingscaletoimproveclinicalmeaningfulnessandstatisticalperformance AT adamlboxer a15itemmodificationofthepspratingscaletoimproveclinicalmeaningfulnessandstatisticalperformance AT lawrencegolbe a15itemmodificationofthepspratingscaletoimproveclinicalmeaningfulnessandstatisticalperformance AT samanthabuddhaeberlein a15itemmodificationofthepspratingscaletoimproveclinicalmeaningfulnessandstatisticalperformance AT onbehalfoftheal108231prosperaand4rtniinvestigators a15itemmodificationofthepspratingscaletoimproveclinicalmeaningfulnessandstatisticalperformance AT tiendam 15itemmodificationofthepspratingscaletoimproveclinicalmeaningfulnessandstatisticalperformance AT liliyang 15itemmodificationofthepspratingscaletoimproveclinicalmeaningfulnessandstatisticalperformance AT colingillis 15itemmodificationofthepspratingscaletoimproveclinicalmeaningfulnessandstatisticalperformance AT yumengli 15itemmodificationofthepspratingscaletoimproveclinicalmeaningfulnessandstatisticalperformance AT johnogorman 15itemmodificationofthepspratingscaletoimproveclinicalmeaningfulnessandstatisticalperformance AT barbarakolb 15itemmodificationofthepspratingscaletoimproveclinicalmeaningfulnessandstatisticalperformance AT adamlboxer 15itemmodificationofthepspratingscaletoimproveclinicalmeaningfulnessandstatisticalperformance AT lawrencegolbe 15itemmodificationofthepspratingscaletoimproveclinicalmeaningfulnessandstatisticalperformance AT samanthabuddhaeberlein 15itemmodificationofthepspratingscaletoimproveclinicalmeaningfulnessandstatisticalperformance AT onbehalfoftheal108231prosperaand4rtniinvestigators 15itemmodificationofthepspratingscaletoimproveclinicalmeaningfulnessandstatisticalperformance |