THE MULTIDIMENSIONAL BURDEN OF ADULT OCULAR MYASTHENIA GRAVIS: A SYSTEMATIC LITERATURE REVIEW
Author(s)
Jordy van Enkhuizen, PhD, MS1, Hicham Benhaddi, MD2, Ritesh Dubey, PharmD3, Barinder Singh, RPh4, April Betts, PhD1.
1UCB Pharma, Slough, United Kingdom, 2UCB Pharma, Paris, France, 3Pharmacoevidence, Mohali, India, 4Pharmacoevidence, London, United Kingdom.
1UCB Pharma, Slough, United Kingdom, 2UCB Pharma, Paris, France, 3Pharmacoevidence, Mohali, India, 4Pharmacoevidence, London, United Kingdom.
OBJECTIVES: Ocular myasthenia gravis (oMG) is an autoimmune neuromuscular disorder and imposes a measurable burden on patients and healthcare systems. However, this burden remains incompletely characterised in published literature. A systematic literature review (SLR) aimed to explore the clinical (including disease generalisation), humanistic, and economic burden among adult patients with oMG.
METHODS: A PRISMA-compliant SLR searched MEDLINE, Embase, Cochrane Library, and NHS EED (2010-2025) for interventional and observational studies (including surveys, registries, and databases) reporting clinical, humanistic, or economic outcomes in adult oMG patients. Citations were screened using Pharmacoevidence's metaSLR tool in accordance with Makhija et al. (2025).
RESULTS: Overall, 28 studies (52 publications) were included, reporting evidence on clinical (n=21), humanistic (n=9) and economic burden (n=2). At oMG presentation, high rates of diplopia (7.5%-89.8%), and ptosis (14.3%-89.8%) were reported, while clinically relevant fatigue was reported by 50.8% of patients. The reported rates of oMG generalisation ranged from 15.7% to 62.0%. Median progression typically occurred within 6 to 16 months post-onset, with ≥70% of generalisation events occurring within the first two years. AChR seropositivity was the strongest prognostic factor for generalisation (ORadj=8.86, p=0.04; HRadj=5.03, p=0.001). Despite relatively high generic utility scores (EQ-5D: 0.89; SF-6D: 0.81), substantial psychosocial burden was observed, including anxiety (23.2%), and depression (16.8%). Half of the patients (50.4%) held a disabled person’s pass, and 25.7% experienced employment restrictions, including unemployment (9.0%), disability (8.4%), and incapacity to work (8.0%), with overlap between categories.
CONCLUSIONS: oMG imposes substantial clinical and psychosocial burden, with impacts on fatigue, mental health, disability, and employment, while also carrying a considerable risk of progression to generalised disease. The discordance between relatively high generic utility scores and substantial patient-reported burden suggests that generic preference-based utilities may lack sensitivity to capture the full impact of oMG, highlighting the need for oMG-specific preference-based measures to inform value assessments.
METHODS: A PRISMA-compliant SLR searched MEDLINE, Embase, Cochrane Library, and NHS EED (2010-2025) for interventional and observational studies (including surveys, registries, and databases) reporting clinical, humanistic, or economic outcomes in adult oMG patients. Citations were screened using Pharmacoevidence's metaSLR tool in accordance with Makhija et al. (2025).
RESULTS: Overall, 28 studies (52 publications) were included, reporting evidence on clinical (n=21), humanistic (n=9) and economic burden (n=2). At oMG presentation, high rates of diplopia (7.5%-89.8%), and ptosis (14.3%-89.8%) were reported, while clinically relevant fatigue was reported by 50.8% of patients. The reported rates of oMG generalisation ranged from 15.7% to 62.0%. Median progression typically occurred within 6 to 16 months post-onset, with ≥70% of generalisation events occurring within the first two years. AChR seropositivity was the strongest prognostic factor for generalisation (ORadj=8.86, p=0.04; HRadj=5.03, p=0.001). Despite relatively high generic utility scores (EQ-5D: 0.89; SF-6D: 0.81), substantial psychosocial burden was observed, including anxiety (23.2%), and depression (16.8%). Half of the patients (50.4%) held a disabled person’s pass, and 25.7% experienced employment restrictions, including unemployment (9.0%), disability (8.4%), and incapacity to work (8.0%), with overlap between categories.
CONCLUSIONS: oMG imposes substantial clinical and psychosocial burden, with impacts on fatigue, mental health, disability, and employment, while also carrying a considerable risk of progression to generalised disease. The discordance between relatively high generic utility scores and substantial patient-reported burden suggests that generic preference-based utilities may lack sensitivity to capture the full impact of oMG, highlighting the need for oMG-specific preference-based measures to inform value assessments.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
CO59
Topic
Clinical Outcomes, Epidemiology & Public Health, Real World Data & Information Systems
Topic Subcategory
Clinical Outcomes Assessment
Disease
Mental Health (including addiction), Neurological Disorders, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)