Can patient self-evaluation of functional status be used for evaluation of impairment of motor function in Guillain-Barré syndrome? Mapping clinician- and patient-reported outcomes in a phase 3 study of eculizumab in Japan
Background and purposeGuillain-Barré syndrome (GBS) is an autoimmune neurological disorder characterized by muscle weakness. In clinical trials, treatment benefit and disease severity are typically measured using clinician-reported outcome measures like the Hughes Functional Grading Scale (HFGS). Ho...
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Frontiers Media S.A.
2025-02-01
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| Series: | Frontiers in Neurology |
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| Online Access: | https://www.frontiersin.org/articles/10.3389/fneur.2025.1463938/full |
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| author | Antoine Regnault Angély Loubert Stéphane Quéré Qun Lin Glen Frick Hirokazu Ishida Yuko Abeta Helene Chevrou-Severac |
| author_facet | Antoine Regnault Angély Loubert Stéphane Quéré Qun Lin Glen Frick Hirokazu Ishida Yuko Abeta Helene Chevrou-Severac |
| author_sort | Antoine Regnault |
| collection | DOAJ |
| description | Background and purposeGuillain-Barré syndrome (GBS) is an autoimmune neurological disorder characterized by muscle weakness. In clinical trials, treatment benefit and disease severity are typically measured using clinician-reported outcome measures like the Hughes Functional Grading Scale (HFGS). However, patient-reported outcome measures, such as the Rasch-built Overall Disability Scale (R-ODS) may provide additional insight into the patient experience during treatment. In this study, exploratory analyses of clinical trial data were performed to investigate how existing clinician-reported outcomes and patient-reported outcomes can help to assess disease progression by providing an accurate measurement of functional status.MethodsData were collected as part of a phase 3 study to assess the safety and efficacy of eculizumab in patients in Japan with severe GBS. The association between HFGS score and R-ODS total centile score (linear measure of limitations; 0, most severe activity and social participation limitations and 100, no limitations) was assessed using the Spearman rank-order correlation coefficient. Threshold values of R-ODS total centile score that could differentiate between patients with an HFGS score of ≤ 1 and > 1 were determined using receiver-operating characteristic curve analyses and mapping (Rasch measurement theory). A triangulation approach was used to establish a proposed value for R-ODS total centile score equivalent to an HFGS score of ≤ 1 or > 1.ResultsOverall, 57 patients were included in this analysis. These exploratory analyses revealed good correlation between R-ODS total centile and HFGS scores. Using the Rasch model, mapping of HFGS to R-ODS scores showed a good fit. Evaluation of the R-ODS threshold that could approximate the functional motor symptom categories based on HFGS (score of 0 or 1) revealed a range of values from 60 to 80. Based on a trial sample, a threshold of 60 was found to have 100% sensitivity and 87% specificity at week 4, and 93.8% sensitivity and 77.8% specificity at week 24.ConclusionThis study established thresholds for the R-ODS total centile score that could approximate classification of functional impairment in GBS based on the HFGS score. Given that the R-ODS reflects the patient perspective, it may be used to capture a more complete picture of GBS severity. |
| format | Article |
| id | doaj-art-89a0dee0a6b74a6f94a4fd4e34be6ccb |
| institution | OA Journals |
| issn | 1664-2295 |
| language | English |
| publishDate | 2025-02-01 |
| publisher | Frontiers Media S.A. |
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| series | Frontiers in Neurology |
| spelling | doaj-art-89a0dee0a6b74a6f94a4fd4e34be6ccb2025-08-20T02:11:09ZengFrontiers Media S.A.Frontiers in Neurology1664-22952025-02-011610.3389/fneur.2025.14639381463938Can patient self-evaluation of functional status be used for evaluation of impairment of motor function in Guillain-Barré syndrome? Mapping clinician- and patient-reported outcomes in a phase 3 study of eculizumab in JapanAntoine Regnault0Angély Loubert1Stéphane Quéré2Qun Lin3Glen Frick4Hirokazu Ishida5Yuko Abeta6Helene Chevrou-Severac7Modus Outcomes, A THREAD Company, Lyon, FranceModus Outcomes, A THREAD Company, Lyon, FranceModus Outcomes, A THREAD Company, Lyon, FranceAlexion, AstraZeneca Rare Disease, Boston, MA, United StatesAlexion, AstraZeneca Rare Disease, Boston, MA, United StatesAlexion, AstraZeneca Rare Disease, Tokyo, JapanAlexion, AstraZeneca Rare Disease, Tokyo, JapanAlexion, AstraZeneca Rare Disease, Baar, SwitzerlandBackground and purposeGuillain-Barré syndrome (GBS) is an autoimmune neurological disorder characterized by muscle weakness. In clinical trials, treatment benefit and disease severity are typically measured using clinician-reported outcome measures like the Hughes Functional Grading Scale (HFGS). However, patient-reported outcome measures, such as the Rasch-built Overall Disability Scale (R-ODS) may provide additional insight into the patient experience during treatment. In this study, exploratory analyses of clinical trial data were performed to investigate how existing clinician-reported outcomes and patient-reported outcomes can help to assess disease progression by providing an accurate measurement of functional status.MethodsData were collected as part of a phase 3 study to assess the safety and efficacy of eculizumab in patients in Japan with severe GBS. The association between HFGS score and R-ODS total centile score (linear measure of limitations; 0, most severe activity and social participation limitations and 100, no limitations) was assessed using the Spearman rank-order correlation coefficient. Threshold values of R-ODS total centile score that could differentiate between patients with an HFGS score of ≤ 1 and > 1 were determined using receiver-operating characteristic curve analyses and mapping (Rasch measurement theory). A triangulation approach was used to establish a proposed value for R-ODS total centile score equivalent to an HFGS score of ≤ 1 or > 1.ResultsOverall, 57 patients were included in this analysis. These exploratory analyses revealed good correlation between R-ODS total centile and HFGS scores. Using the Rasch model, mapping of HFGS to R-ODS scores showed a good fit. Evaluation of the R-ODS threshold that could approximate the functional motor symptom categories based on HFGS (score of 0 or 1) revealed a range of values from 60 to 80. Based on a trial sample, a threshold of 60 was found to have 100% sensitivity and 87% specificity at week 4, and 93.8% sensitivity and 77.8% specificity at week 24.ConclusionThis study established thresholds for the R-ODS total centile score that could approximate classification of functional impairment in GBS based on the HFGS score. Given that the R-ODS reflects the patient perspective, it may be used to capture a more complete picture of GBS severity.https://www.frontiersin.org/articles/10.3389/fneur.2025.1463938/fullGuillain-Barré syndromeclinician-reported outcomespatient-reported outcomestreatment outcomesdisease progressioneculizumab |
| spellingShingle | Antoine Regnault Angély Loubert Stéphane Quéré Qun Lin Glen Frick Hirokazu Ishida Yuko Abeta Helene Chevrou-Severac Can patient self-evaluation of functional status be used for evaluation of impairment of motor function in Guillain-Barré syndrome? Mapping clinician- and patient-reported outcomes in a phase 3 study of eculizumab in Japan Frontiers in Neurology Guillain-Barré syndrome clinician-reported outcomes patient-reported outcomes treatment outcomes disease progression eculizumab |
| title | Can patient self-evaluation of functional status be used for evaluation of impairment of motor function in Guillain-Barré syndrome? Mapping clinician- and patient-reported outcomes in a phase 3 study of eculizumab in Japan |
| title_full | Can patient self-evaluation of functional status be used for evaluation of impairment of motor function in Guillain-Barré syndrome? Mapping clinician- and patient-reported outcomes in a phase 3 study of eculizumab in Japan |
| title_fullStr | Can patient self-evaluation of functional status be used for evaluation of impairment of motor function in Guillain-Barré syndrome? Mapping clinician- and patient-reported outcomes in a phase 3 study of eculizumab in Japan |
| title_full_unstemmed | Can patient self-evaluation of functional status be used for evaluation of impairment of motor function in Guillain-Barré syndrome? Mapping clinician- and patient-reported outcomes in a phase 3 study of eculizumab in Japan |
| title_short | Can patient self-evaluation of functional status be used for evaluation of impairment of motor function in Guillain-Barré syndrome? Mapping clinician- and patient-reported outcomes in a phase 3 study of eculizumab in Japan |
| title_sort | can patient self evaluation of functional status be used for evaluation of impairment of motor function in guillain barre syndrome mapping clinician and patient reported outcomes in a phase 3 study of eculizumab in japan |
| topic | Guillain-Barré syndrome clinician-reported outcomes patient-reported outcomes treatment outcomes disease progression eculizumab |
| url | https://www.frontiersin.org/articles/10.3389/fneur.2025.1463938/full |
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