TY - JOUR
T1 - High efficacy of rituximab for myasthenia gravis
T2 - a comprehensive nationwide study in Austria
AU - Topakian, Raffi
AU - Zimprich, Fritz
AU - Iglseder, Stephan
AU - Embacher, Norbert
AU - Guger, Michael
AU - Stieglbauer, Karl
AU - Langenscheidt, Dieter
AU - Rath, Jakob
AU - Quasthoff, Stefan
AU - Simschitz, Philipp
AU - Wanschitz, Julia
AU - Windisch, David
AU - Müller, Petra
AU - Oel, Dierk
AU - Schustereder, Günther
AU - Einsiedler, Stefan
AU - Eggers, Christian
AU - Löscher, Wolfgang
N1 - Publisher Copyright:
© 2019, Springer-Verlag GmbH Germany, part of Springer Nature.
PY - 2019/3/1
Y1 - 2019/3/1
N2 - BACKGROUND: Most patients with myasthenia gravis (MG) need long-term immunosuppressive therapy. However, conventional agents may have intolerable side effects, take too long or fail to achieve disease control. Rituximab (RTX) has emerged as an off-label treatment for refractory MG, but data on its use are still sparse.METHODS: We conducted a retrospective nationwide study contacting all Austrian neurologists to provide anonymized data of all adult MG patients treated with RTX and minimum follow-up of 3 months. The Myasthenia Gravis Foundation of America Postintervention Status scale was used to assess outcomes.RESULTS: 34 (60.7%) of a total of 56 patients were women. Median (IQR) age at diagnosis of MG and start of RTX were 41.5 (24.3; 65.8) and 47.5 (33; 71) years, respectively. Antibodies (ab) against acetylcholine receptor (AchR) and muscle-specific tyrosine kinase (MuSK) were present in 69.6% and 25% of patients, respectively (seronegative: 5.4%). Before RTX, 47 (83.9%) patients had had plasma exchange, immune adsorption or immunoglobulins. Three months after RTX, 14 of 53 (26.4%) patients were in remission. At last follow-up after a median of 20 (10; 53) months, remission was present in 42.9% of patients and another 25% had minimal manifestations. Remission was more frequent in patients with MuSK ab vs. those with AchR ab (71.4% vs. 35.9%, p = 0.022). RTX was safe. The presence of MuSK ab independently predicted remission after RTX.CONCLUSION: In this retrospective study on RTX for MG, the largest to date, RTX appeared safe, efficacious and fast acting. Benefit from RTX was greatest in MuSK ab + MG.
AB - BACKGROUND: Most patients with myasthenia gravis (MG) need long-term immunosuppressive therapy. However, conventional agents may have intolerable side effects, take too long or fail to achieve disease control. Rituximab (RTX) has emerged as an off-label treatment for refractory MG, but data on its use are still sparse.METHODS: We conducted a retrospective nationwide study contacting all Austrian neurologists to provide anonymized data of all adult MG patients treated with RTX and minimum follow-up of 3 months. The Myasthenia Gravis Foundation of America Postintervention Status scale was used to assess outcomes.RESULTS: 34 (60.7%) of a total of 56 patients were women. Median (IQR) age at diagnosis of MG and start of RTX were 41.5 (24.3; 65.8) and 47.5 (33; 71) years, respectively. Antibodies (ab) against acetylcholine receptor (AchR) and muscle-specific tyrosine kinase (MuSK) were present in 69.6% and 25% of patients, respectively (seronegative: 5.4%). Before RTX, 47 (83.9%) patients had had plasma exchange, immune adsorption or immunoglobulins. Three months after RTX, 14 of 53 (26.4%) patients were in remission. At last follow-up after a median of 20 (10; 53) months, remission was present in 42.9% of patients and another 25% had minimal manifestations. Remission was more frequent in patients with MuSK ab vs. those with AchR ab (71.4% vs. 35.9%, p = 0.022). RTX was safe. The presence of MuSK ab independently predicted remission after RTX.CONCLUSION: In this retrospective study on RTX for MG, the largest to date, RTX appeared safe, efficacious and fast acting. Benefit from RTX was greatest in MuSK ab + MG.
KW - Adult
KW - Aged
KW - Austria
KW - Cohort Studies
KW - Dose-Response Relationship, Drug
KW - Female
KW - Humans
KW - Immunologic Factors/therapeutic use
KW - Logistic Models
KW - Male
KW - Middle Aged
KW - Myasthenia Gravis/drug therapy
KW - Receptor Protein-Tyrosine Kinases/immunology
KW - Receptors, Cholinergic/immunology
KW - Rituximab/therapeutic use
KW - Treatment Outcome
UR - http://www.scopus.com/inward/record.url?scp=85060184544&partnerID=8YFLogxK
U2 - 10.1007/s00415-019-09191-6
DO - 10.1007/s00415-019-09191-6
M3 - Journal article
C2 - 30649616
SN - 0340-5354
VL - 266
SP - 699
EP - 706
JO - Journal of Neurology
JF - Journal of Neurology
IS - 3
ER -