Triangulation Method to Detect Meaningful Deterioration in Patient's Autonomy Using the New Multiple Sclerosis Autonomy Scale (MSAS) Questionnaire
Author(s)
Cecile Donze, PhD1, Geraud Paillot, MSc2, Claude Mekies, PhD3, Mikael Cohen, PhD4, Lucie Brechenmacher, PhD5, Alexandre Civet, MSc5, David Pau, MSc5, Ella Lukasiewicz, BSc5, Catherine Mouzawak, MSc6, Patrick Vermersch, PhD7.
1Hospital saint Philibert, Lille, France, 2Aventure Hustive, Saint Malo, France, 3RAMSAY Clinique des Cèdres, Toulouse, France, 4CHU Nice Pasteur, Nice, France, 5Roche, Boulogne-Billancourt, France, 6Structure régionale neuro SEP SYNAPSE, Le Vesinet, France, 7Univ Lille, Lille, France.
1Hospital saint Philibert, Lille, France, 2Aventure Hustive, Saint Malo, France, 3RAMSAY Clinique des Cèdres, Toulouse, France, 4CHU Nice Pasteur, Nice, France, 5Roche, Boulogne-Billancourt, France, 6Structure régionale neuro SEP SYNAPSE, Le Vesinet, France, 7Univ Lille, Lille, France.
OBJECTIVES: We evaluated the Multiple Sclerosis Autonomy Scale (MSAS), a new PRO designed to assess MS patient autonomy. Establishing a minimal clinically important deterioration is essential, given that even small shifts in autonomy can necessitate adjustments to patient care. Our objective is to determine a meaningful deterioration in patients’ autonomy using the MSAS.
METHODS: FOCAL-MS2 is a 1-year longitudinal prospective study (2024-2025) evaluating patients' autonomy. Anchored and distribution-based approaches were used to assess the Meaningful Changes Threshold (MCT).Three anchors:Anchor_1: Overall perceived autonomy; Anchor_2: clinical EDSS score; Anchor_3:Recent impactful event affecting MSwere assessed at baseline and 1 year. Change from baseline was categorized into three groups: no change, deterioration and amelioration. For each group, the Minimum Clinically Important Differences (MCID) was calculated using minimum detectable change MDC90 (90%CI) , MDC95 (95%CI), and Mean Change From Baseline. MCIDs were derived from Global Score changes (quantifying overall autonomy by averaging impact scores across dimensions), along with confidence intervals and sample sizes.MSAS score ranges from 0 to 100 and an increased score indicates a patient's autonomy deterioration.A triangulation approach, integrating weights from the three anchors, was employed to determine Minimum Clinically Threshold (MCT), ensuring it surpassed the standard error of measurement (SEM).
RESULTS: 158 patients answered the questionnaires at baseline and Day_360. Anchor-based methods estimated deterioration in 38 patients (24.1%) for anchor_1, 22 (13.9%) for anchor_2, and 31 (19.6%) for anchor_3. MCT found for each calculated MCID were: 1.6 for MDC90 2.8 for MDC95 5.7 for the mean change from baseline. The Standard Error of the Mean (SEM) at 5.1 was only surpassed by the mean change from baseline MCT, reached by 49 patients of the cohort (31%).
CONCLUSIONS: This triangulation approach provided a 5.7 clinically relevant threshold (MCID) for detecting deterioration in MS patient autonomy, guiding adaptive patient management.
METHODS: FOCAL-MS2 is a 1-year longitudinal prospective study (2024-2025) evaluating patients' autonomy. Anchored and distribution-based approaches were used to assess the Meaningful Changes Threshold (MCT).Three anchors:Anchor_1: Overall perceived autonomy; Anchor_2: clinical EDSS score; Anchor_3:Recent impactful event affecting MSwere assessed at baseline and 1 year. Change from baseline was categorized into three groups: no change, deterioration and amelioration. For each group, the Minimum Clinically Important Differences (MCID) was calculated using minimum detectable change MDC90 (90%CI) , MDC95 (95%CI), and Mean Change From Baseline. MCIDs were derived from Global Score changes (quantifying overall autonomy by averaging impact scores across dimensions), along with confidence intervals and sample sizes.MSAS score ranges from 0 to 100 and an increased score indicates a patient's autonomy deterioration.A triangulation approach, integrating weights from the three anchors, was employed to determine Minimum Clinically Threshold (MCT), ensuring it surpassed the standard error of measurement (SEM).
RESULTS: 158 patients answered the questionnaires at baseline and Day_360. Anchor-based methods estimated deterioration in 38 patients (24.1%) for anchor_1, 22 (13.9%) for anchor_2, and 31 (19.6%) for anchor_3. MCT found for each calculated MCID were: 1.6 for MDC90 2.8 for MDC95 5.7 for the mean change from baseline. The Standard Error of the Mean (SEM) at 5.1 was only surpassed by the mean change from baseline MCT, reached by 49 patients of the cohort (31%).
CONCLUSIONS: This triangulation approach provided a 5.7 clinically relevant threshold (MCID) for detecting deterioration in MS patient autonomy, guiding adaptive patient management.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
PCR252
Topic
Methodological & Statistical Research, Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
Neurological Disorders