LONGITUDINAL ANALYSIS OF AUTONOMY LOSS USING THE WIN RATIO (WR) METHOD:APPLICATION TO PATIENT-REPORTED OUTCOMES (PROS) IN THE FOCAL-MS2 STUDY
Author(s)
Laurent Chardaire, PharmD, MSc1, Cecile Donze, MD2, Geraud Paillot, MSc3, Claude Mekies, MD4, Mikael Cohen, MD5, Lucie Brechenmacher, MSc1, Alexandre Civet, MSc1, David Pau, MSc1, Catherine Mouzawak, MD6, Patrick Vermersch, MD7.
1Roche SAS, Boulogne-Billancourt, France, 2Hôpital saint Philibert, Groupement des Hôpitaux de l'Institut Catholique de Lille, Faculté de médecine et de maïeutique de Lille, Lomme, France, 3Association Aventure Hustive, Saint-Malo, France, 4RAMSAY Clinique des Cèdres, Neurologie, Toulouse, France, 5Université Nice Cote d’Azur, UR2CA-URRIS CRCSEP CHU Nice Pasteur, Service de Neurologie, Nice, France, 6Structure régionale neuro SEP SYNAPSE, Le Vésinet, France, 7Univ. Lille, INSERM UMR1172 LilNCog, CHU Lille, FHU Precise, Lille, France.
1Roche SAS, Boulogne-Billancourt, France, 2Hôpital saint Philibert, Groupement des Hôpitaux de l'Institut Catholique de Lille, Faculté de médecine et de maïeutique de Lille, Lomme, France, 3Association Aventure Hustive, Saint-Malo, France, 4RAMSAY Clinique des Cèdres, Neurologie, Toulouse, France, 5Université Nice Cote d’Azur, UR2CA-URRIS CRCSEP CHU Nice Pasteur, Service de Neurologie, Nice, France, 6Structure régionale neuro SEP SYNAPSE, Le Vésinet, France, 7Univ. Lille, INSERM UMR1172 LilNCog, CHU Lille, FHU Precise, Lille, France.
OBJECTIVES: The Multiple Sclerosis Autonomy Scale (MSAS) is a new PRO that aims at evaluating patient autonomy in multiple sclerosis (MS). This research aims to evaluate and rank the trajectories of autonomy decline across six clinico-demographic profiles over a one-year period, identifying profiles exhibiting slower autonomy deterioration.
METHODS: FOCAL-MS2 is a 1-year longitudinal prospective study (2024-2025) evaluating autonomy, which was assessed using global and 10-dimensions scores derived from the MSAS. The event of interest was a clinically significant deterioration(CSD) in autonomy, defined by reaching a CSD threshold of ≥5.7 points. Applied to monthly measurement time points, the hierarchical WR method compared patient trajectories chronologically on a month-by-month basis, thereby inherently penalizing profiles (clusters) that experienced an earlier degradation in autonomy. Six clusters with different clinical profiles were identified in previous research (DOI:10.1186/s41687-026-01074-5): cluster 1 (C1): sick-leave [50-60]yo, C2: walking-assistance [40-50]yo, C3: walking-assistance [50-60]yo, C4: retired >60yo with leisure, C5: inactive <40yo, C6: active women <40yo. A 'win' was therefore defined as a cluster experiencing a clinically significant degradation earlier than another one.
RESULTS: Among the 199 included patients, exploratory global autonomy degradation comparisons against the rest of the cohort yielded a single nominally significant result: C4 demonstrated a slower degradation rate (WR=0.54,IC95%=[0.3-0.97]). Conversely, trajectories for the five other clusters did not reach statistical significance compared to the overall cohort: C1 showed a trend towards faster degradation (WR=1.44,IC95%=[0.89-2.32]), followed by C6 (WR=1.15,IC95%=[0.75-1.76]), C2 (WR=1.12,IC95%=[0.69-1.81]), C5 (WR=1.07,IC95%=[0.53-2.14]), and C3 (WR=0.84,IC95%=[0.53-1.33]). The majority of wins occurred as early as M1(62.5%), with subsequent degradations distributed as follows: M2(1.7%), M3(8%), M4(8.1%), M5(4.3%), M6(3%), M7(2%), M8(1.8%), M9(1.5%), M10(1.4%), M11(1.3%), and M12(4.4%).
CONCLUSIONS: Exploratory WR analysis highlighted a patient profile exhibiting slower autonomy deterioration.
METHODS: FOCAL-MS2 is a 1-year longitudinal prospective study (2024-2025) evaluating autonomy, which was assessed using global and 10-dimensions scores derived from the MSAS. The event of interest was a clinically significant deterioration(CSD) in autonomy, defined by reaching a CSD threshold of ≥5.7 points. Applied to monthly measurement time points, the hierarchical WR method compared patient trajectories chronologically on a month-by-month basis, thereby inherently penalizing profiles (clusters) that experienced an earlier degradation in autonomy. Six clusters with different clinical profiles were identified in previous research (DOI:10.1186/s41687-026-01074-5): cluster 1 (C1): sick-leave [50-60]yo, C2: walking-assistance [40-50]yo, C3: walking-assistance [50-60]yo, C4: retired >60yo with leisure, C5: inactive <40yo, C6: active women <40yo. A 'win' was therefore defined as a cluster experiencing a clinically significant degradation earlier than another one.
RESULTS: Among the 199 included patients, exploratory global autonomy degradation comparisons against the rest of the cohort yielded a single nominally significant result: C4 demonstrated a slower degradation rate (WR=0.54,IC95%=[0.3-0.97]). Conversely, trajectories for the five other clusters did not reach statistical significance compared to the overall cohort: C1 showed a trend towards faster degradation (WR=1.44,IC95%=[0.89-2.32]), followed by C6 (WR=1.15,IC95%=[0.75-1.76]), C2 (WR=1.12,IC95%=[0.69-1.81]), C5 (WR=1.07,IC95%=[0.53-2.14]), and C3 (WR=0.84,IC95%=[0.53-1.33]). The majority of wins occurred as early as M1(62.5%), with subsequent degradations distributed as follows: M2(1.7%), M3(8%), M4(8.1%), M5(4.3%), M6(3%), M7(2%), M8(1.8%), M9(1.5%), M10(1.4%), M11(1.3%), and M12(4.4%).
CONCLUSIONS: Exploratory WR analysis highlighted a patient profile exhibiting slower autonomy deterioration.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
PCR71
Topic
Methodological & Statistical Research, Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
Neurological Disorders