AN INTEGRATED DISEASE CONCEPTUAL MODEL OF MULTIPLE SCLEROSIS (MS) FROM THE PATIENTS' PERSPECTIVE: A MULTINATIONAL QUALITATIVE STUDY
Author(s)
Dorsa Khazaei, MSc1, Katja Rudell, PhD2, Cécile Gousset, Pharm D3, Lita Araujo, PhD4, Nele Von Horsten, MSc5, Lisa Emrich, DMA6, Tommi Tervonen, PhD7, Guillaume Montagu, MSc3.
1Kielo Research, London, United Kingdom, 2Kielo Research, Cambridge, United Kingdom, 3Sanofi, Gentilly, France, 4Sanofi, Cambridge, MA, USA, 5Patient advocate, Pirna, Germany, 6Patient advocate, Accelerated Cure Project, Waltham, MA, USA, 7Kielo Research, Zug, Switzerland.
1Kielo Research, London, United Kingdom, 2Kielo Research, Cambridge, United Kingdom, 3Sanofi, Gentilly, France, 4Sanofi, Cambridge, MA, USA, 5Patient advocate, Pirna, Germany, 6Patient advocate, Accelerated Cure Project, Waltham, MA, USA, 7Kielo Research, Zug, Switzerland.
OBJECTIVES: MS is a chronic neurological disease with heterogeneous symptoms. Existing MS conceptual models don’t comprehensively capture patients’ experience across relapsing-remitting MS (RRMS), non-relapsing secondary progressive MS (nrSPMS), and primary progressive MS (PPMS). This qualitative study aimed to develop an integrated, patient-grounded conceptual model integrating symptoms and impacts across all MS subtypes.
METHODS: Semi-structured interviews (50-90 minutes) were conducted remotely with N=45 adults across the US, UK, and Germany (15 RRMS, 15 nrSPMS, 15 PPMS; 5 per subtype per country). Interviews explored symptoms, their impacts, and disease progression perspectives. Transcripts were coded deductively and inductively in Atlas.ti; Sankey diagrams were generated in R to represent symptom-to-impact causal relationships. Patient-Determined Disease Steps (PDDS) captured disability severity.
RESULTS: Participants median age: 50 years (range 30-76); 29/45 female. Mean PDDS scores: 2.9 (RRMS), 5.3 (PPMS), 5.7 (nrSPMS).
The most frequently endorsed symptoms were limbs weakness/heaviness (44/45) and balance/stability (44/45), followed by fatigue/low energy (42/45) and cognitive difficulties (40/45). Among the top 3 most bothersome symptoms, fatigue ranked first (21/45), followed by limbs weakness (20/45) and pain (14/45).
Patients report symptoms with wide-ranging consequences spanning multiple life domains. Among them, the most impactful were:
• Motor functions (limb weakness, spasticity, stiffness, foot drop) spread across physical activities, personal care, work productivity, and role/social functioning.
• Fatigue and cognitive difficulties linked to work productivity, role/social functioning, relationships, physical activities, cognitive impacts, and emotional well-being.
Distinct patterns appeared: in RRMS, cerebral functions dominated impact pathways; in nrSPMS, pyramidal and cerebral functions contributed equally; in PPMS, pyramidal functions were predominant, cerebral secondary. Physical activities and role/social functioning were the most impacted domains overall.
CONCLUSIONS: This study provides the first integrated patient-grounded MS conceptual model, addressing a knowledge gap. It reflects a disease continuum, driven by motor symptoms, fatigue and cognition, with subtype-specific variations in pathway dominance, rather than strictly separate entities.
METHODS: Semi-structured interviews (50-90 minutes) were conducted remotely with N=45 adults across the US, UK, and Germany (15 RRMS, 15 nrSPMS, 15 PPMS; 5 per subtype per country). Interviews explored symptoms, their impacts, and disease progression perspectives. Transcripts were coded deductively and inductively in Atlas.ti; Sankey diagrams were generated in R to represent symptom-to-impact causal relationships. Patient-Determined Disease Steps (PDDS) captured disability severity.
RESULTS: Participants median age: 50 years (range 30-76); 29/45 female. Mean PDDS scores: 2.9 (RRMS), 5.3 (PPMS), 5.7 (nrSPMS).
The most frequently endorsed symptoms were limbs weakness/heaviness (44/45) and balance/stability (44/45), followed by fatigue/low energy (42/45) and cognitive difficulties (40/45). Among the top 3 most bothersome symptoms, fatigue ranked first (21/45), followed by limbs weakness (20/45) and pain (14/45).
Patients report symptoms with wide-ranging consequences spanning multiple life domains. Among them, the most impactful were:
• Motor functions (limb weakness, spasticity, stiffness, foot drop) spread across physical activities, personal care, work productivity, and role/social functioning.
• Fatigue and cognitive difficulties linked to work productivity, role/social functioning, relationships, physical activities, cognitive impacts, and emotional well-being.
Distinct patterns appeared: in RRMS, cerebral functions dominated impact pathways; in nrSPMS, pyramidal and cerebral functions contributed equally; in PPMS, pyramidal functions were predominant, cerebral secondary. Physical activities and role/social functioning were the most impacted domains overall.
CONCLUSIONS: This study provides the first integrated patient-grounded MS conceptual model, addressing a knowledge gap. It reflects a disease continuum, driven by motor symptoms, fatigue and cognition, with subtype-specific variations in pathway dominance, rather than strictly separate entities.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
PCR72
Topic
Patient-Centered Research
Disease
Neurological Disorders, No Additional Disease & Conditions/Specialized Treatment Areas