Qualitative measures that assess functional disability and quality of life in ALS
Abstract Background Selection of appropriate trial endpoints and outcome measures is particularly important in rare disease and rapidly progressing disease such as amyotrophic lateral sclerosis (ALS) where the challenges to conducting clinical trials, are substantial: patient and disease heterogenei...
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| Format: | Article |
| Language: | English |
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BMC
2022-01-01
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| Series: | Health and Quality of Life Outcomes |
| Subjects: | |
| Online Access: | https://doi.org/10.1186/s12955-022-01919-9 |
| _version_ | 1819260910986854400 |
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| author | Susan L. Hartmaier Thomas Rhodes Suzanne F. Cook Courtney Schlusser Chao Chen Steve Han Neta Zach Venkatesha Murthy Shreya Davé |
| author_facet | Susan L. Hartmaier Thomas Rhodes Suzanne F. Cook Courtney Schlusser Chao Chen Steve Han Neta Zach Venkatesha Murthy Shreya Davé |
| author_sort | Susan L. Hartmaier |
| collection | DOAJ |
| description | Abstract Background Selection of appropriate trial endpoints and outcome measures is particularly important in rare disease and rapidly progressing disease such as amyotrophic lateral sclerosis (ALS) where the challenges to conducting clinical trials, are substantial: patient and disease heterogeneity, limited understanding of exact disease pathophysiology, and lack of robust and available biomarkers. To address these challenges in ALS, the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised version (ALSFRS-R) was developed and has become a key primary endpoint in ALS clinical trials to assess functional disability and disease progression, often replacing survival as a primary outcome. However, increased understanding of the ALS disease journey and improvements in assistive technology for ALS patients have exposed issues with the ALSFRS-R, including non-linearity, multidimensionality and floor and ceiling effects that could challenge its continued utility as a primary outcome measure in ALS clinical trials. Recently, other qualitative scale measures of functioning disability have been developed to help address these issues. With this in mind, we conducted a literature search aimed at identifying both established and promising new measures for potential use in clinical trials. Methods We searched PubMed, Google, Google Scholar, and the reference sections of key studies to identify papers that discussed qualitative measures of functional status for potential use in ALS studies. We also searched clinicaltrials.gov to identify functional status and health-related quality of life (HRQoL) measures that have been used in ALS interventional studies. Results In addition to the ALSFRS-R, we identified several newer qualitative scales including ALSFRS-EX, ALS-MITOS, CNS-BFS, DALS-15, MND-DS, and ROADS. Strengths and limitations of each measure were identified and discussed, along with their potential to act as a primary or secondary outcome to assess patient functional status in ALS clinical trials. Conclusion This paper serves as a reference guide for researchers deciding which qualitative measures to use as endpoints in their ALS clinical trials to assess functional status. This paper also discusses the importance of including ALS HRQoL and ALS cognitive screens in future clinical trials to assess the value of a new ALS therapy more comprehensively. |
| first_indexed | 2024-12-23T19:33:25Z |
| format | Article |
| id | doaj.art-7406e7e395ae4fc9b61346944fb7f530 |
| institution | Directory Open Access Journal |
| issn | 1477-7525 |
| language | English |
| last_indexed | 2024-12-23T19:33:25Z |
| publishDate | 2022-01-01 |
| publisher | BMC |
| record_format | Article |
| series | Health and Quality of Life Outcomes |
| spelling | doaj.art-7406e7e395ae4fc9b61346944fb7f5302022-12-21T17:33:51ZengBMCHealth and Quality of Life Outcomes1477-75252022-01-0120111610.1186/s12955-022-01919-9Qualitative measures that assess functional disability and quality of life in ALSSusan L. Hartmaier0Thomas Rhodes1Suzanne F. Cook2Courtney Schlusser3Chao Chen4Steve Han5Neta Zach6Venkatesha Murthy7Shreya Davé8CERobs Consulting, LLCCERobs Consulting, LLCCERobs Consulting, LLCCERobs Consulting, LLCTakeda Development Center Americas, Inc.Takeda Development Center Americas, Inc.Takeda Development Center Americas, Inc.Takeda Development Center Americas, Inc.Takeda Development Center Americas, Inc.Abstract Background Selection of appropriate trial endpoints and outcome measures is particularly important in rare disease and rapidly progressing disease such as amyotrophic lateral sclerosis (ALS) where the challenges to conducting clinical trials, are substantial: patient and disease heterogeneity, limited understanding of exact disease pathophysiology, and lack of robust and available biomarkers. To address these challenges in ALS, the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised version (ALSFRS-R) was developed and has become a key primary endpoint in ALS clinical trials to assess functional disability and disease progression, often replacing survival as a primary outcome. However, increased understanding of the ALS disease journey and improvements in assistive technology for ALS patients have exposed issues with the ALSFRS-R, including non-linearity, multidimensionality and floor and ceiling effects that could challenge its continued utility as a primary outcome measure in ALS clinical trials. Recently, other qualitative scale measures of functioning disability have been developed to help address these issues. With this in mind, we conducted a literature search aimed at identifying both established and promising new measures for potential use in clinical trials. Methods We searched PubMed, Google, Google Scholar, and the reference sections of key studies to identify papers that discussed qualitative measures of functional status for potential use in ALS studies. We also searched clinicaltrials.gov to identify functional status and health-related quality of life (HRQoL) measures that have been used in ALS interventional studies. Results In addition to the ALSFRS-R, we identified several newer qualitative scales including ALSFRS-EX, ALS-MITOS, CNS-BFS, DALS-15, MND-DS, and ROADS. Strengths and limitations of each measure were identified and discussed, along with their potential to act as a primary or secondary outcome to assess patient functional status in ALS clinical trials. Conclusion This paper serves as a reference guide for researchers deciding which qualitative measures to use as endpoints in their ALS clinical trials to assess functional status. This paper also discusses the importance of including ALS HRQoL and ALS cognitive screens in future clinical trials to assess the value of a new ALS therapy more comprehensively.https://doi.org/10.1186/s12955-022-01919-9ALSALSFRS-RClinical trialsQualitative outcome measuresHRQoLALS cognitive screens |
| spellingShingle | Susan L. Hartmaier Thomas Rhodes Suzanne F. Cook Courtney Schlusser Chao Chen Steve Han Neta Zach Venkatesha Murthy Shreya Davé Qualitative measures that assess functional disability and quality of life in ALS Health and Quality of Life Outcomes ALS ALSFRS-R Clinical trials Qualitative outcome measures HRQoL ALS cognitive screens |
| title | Qualitative measures that assess functional disability and quality of life in ALS |
| title_full | Qualitative measures that assess functional disability and quality of life in ALS |
| title_fullStr | Qualitative measures that assess functional disability and quality of life in ALS |
| title_full_unstemmed | Qualitative measures that assess functional disability and quality of life in ALS |
| title_short | Qualitative measures that assess functional disability and quality of life in ALS |
| title_sort | qualitative measures that assess functional disability and quality of life in als |
| topic | ALS ALSFRS-R Clinical trials Qualitative outcome measures HRQoL ALS cognitive screens |
| url | https://doi.org/10.1186/s12955-022-01919-9 |
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