REAL-WORLD CHARACTERISTICS OF MYASTHENIA GRAVIS (MG) AND GENERALIZED MG (MG-RENIRA): A SINGLE-CENTER RETROSPECTIVE STUDY IN POLAND
Author(s)
Małgorzata Bilińska, MD, PhD1, Julia Bogdan, MSc1, Kamil Pytlak, MSc2, Izabela Lenart, PhD3, Szymon Drygała, PhD3, Pawel Dubiela, PhD3, Dorota Giersz, MSc4, Bartosz Karaszewski, MD, PhD1.
1Department of Adult Neurology, Faculty of Medicine, Medical University of Gdansk, Gdansk, Poland, 2Transition Technologies Science Sp. z o.o., Warsaw, Poland, 3AstraZeneca Pharma Poland Sp. z o.o., Warsaw, Poland, 4AstraZeneca Pharma Poland Sp. z o.o., Warszawa, Poland.
1Department of Adult Neurology, Faculty of Medicine, Medical University of Gdansk, Gdansk, Poland, 2Transition Technologies Science Sp. z o.o., Warsaw, Poland, 3AstraZeneca Pharma Poland Sp. z o.o., Warsaw, Poland, 4AstraZeneca Pharma Poland Sp. z o.o., Warszawa, Poland.
OBJECTIVES: Myasthenia gravis (MG) often progresses to generalized disease (gMG), yet real-world data on patient characteristics and management remain limited. This study aimed to characterize patients with MG/gMG at a Polish tertiary center, including demographics, clinical profile, treatment patterns, and healthcare resource utilization (HCRU).
METHODS: This retrospective single-center study analyzed electronic health records from University Clinical Center Gdańsk, Poland (2001-June 2025). Adults with clinically confirmed MG (ICD-10 G70.0) were included. Index date was the earliest recorded encounter confirming MG diagnosis. gMG was defined as Myasthenia Gravis Foundation of America class ≥IIa at any time. All analyses were descriptive with domain-specific analytic windows.
RESULTS: Among 235 patients with MG, 184 (78.3%) met gMG criteria. In the gMG cohort, 66% were female (mean [SD] age at diagnosis, 48 [18] years). Comorbidities were common at baseline and throughout the median 7.0-year follow-up (IQR: 3.1-11.6). Treatment exposure in the gMG group (n=146) included pyridostigmine (115, 79%), systemic glucocorticoids (90, 62%), azathioprine (57, 39%), mycophenolate mofetil(27, 18%), methotrexate (18, 12%), intravenous immunoglobulin (IVIg; 19, 13%), and plasma exchange (6, 4.1%). Mean (SD) daily glucocorticoid dose was 21 (15) mg overall but 39 (25) mg during the first 12 months. Among glucocorticoid-exposed patients, 33% received treatment more than 6 months post-index; 87% of these received additional nonsteroidal immunosuppression, with up to four treatment lines observed. Compared with the overall cohort, patients with gMG had higher outpatient visit rates (1.36 vs 0.32 per patient-year) and hospitalization rates (0.21 vs 0.02 per patient- year). Intensive care unit utilization was 0.12 days per patient-year (107 days during the last 12 months), and inpatient IVIg administration rate was 0.13 per patient-year.
CONCLUSIONS: Generalized disease was frequent and associated with substantial glucocorticoid exposure, immunosuppressive escalation, and higher HCRU, highlighting the need for durable disease control with reduced long-term treatment burden.
METHODS: This retrospective single-center study analyzed electronic health records from University Clinical Center Gdańsk, Poland (2001-June 2025). Adults with clinically confirmed MG (ICD-10 G70.0) were included. Index date was the earliest recorded encounter confirming MG diagnosis. gMG was defined as Myasthenia Gravis Foundation of America class ≥IIa at any time. All analyses were descriptive with domain-specific analytic windows.
RESULTS: Among 235 patients with MG, 184 (78.3%) met gMG criteria. In the gMG cohort, 66% were female (mean [SD] age at diagnosis, 48 [18] years). Comorbidities were common at baseline and throughout the median 7.0-year follow-up (IQR: 3.1-11.6). Treatment exposure in the gMG group (n=146) included pyridostigmine (115, 79%), systemic glucocorticoids (90, 62%), azathioprine (57, 39%), mycophenolate mofetil(27, 18%), methotrexate (18, 12%), intravenous immunoglobulin (IVIg; 19, 13%), and plasma exchange (6, 4.1%). Mean (SD) daily glucocorticoid dose was 21 (15) mg overall but 39 (25) mg during the first 12 months. Among glucocorticoid-exposed patients, 33% received treatment more than 6 months post-index; 87% of these received additional nonsteroidal immunosuppression, with up to four treatment lines observed. Compared with the overall cohort, patients with gMG had higher outpatient visit rates (1.36 vs 0.32 per patient-year) and hospitalization rates (0.21 vs 0.02 per patient- year). Intensive care unit utilization was 0.12 days per patient-year (107 days during the last 12 months), and inpatient IVIg administration rate was 0.13 per patient-year.
CONCLUSIONS: Generalized disease was frequent and associated with substantial glucocorticoid exposure, immunosuppressive escalation, and higher HCRU, highlighting the need for durable disease control with reduced long-term treatment burden.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HSD60
Topic
Clinical Outcomes, Health Service Delivery & Process of Care
Disease
Neurological Disorders, Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)