TY - JOUR
T1 - Rituximab as treatment for anti-MuSK myasthenia gravis
AU - Hehir, Michael K.
AU - Hobson-Webb, Lisa D.
AU - Benatar, Michael
AU - Barnett, Carolina
AU - Silvestri, Nicholas J.
AU - Howard, James F.
AU - Howard, DIantha
AU - Visser, Amy
AU - Crum, Brian A.
AU - Nowak, Richard
AU - Beekman, Rachel
AU - Kumar, Aditya
AU - Ruzhansky, Katherine
AU - Chen, I. Hweii Amy
AU - Pulley, Michael T.
AU - Laboy, Shannon M.
AU - Fellman, Melissa A.
AU - Greene, Shane M.
AU - Pasnoor, Mamatha
AU - Burns, Ted M.
N1 - Publisher Copyright:
© 2017 American Academy of Neurology.
PY - 2017/9/5
Y1 - 2017/9/5
N2 - To evaluate the efficacy of rituximab in treatment of anti-muscle-specific kinase (MuSK) myasthenia gravis (MG). Methods: This was a multicenter, blinded, prospective review, comparing anti-MuSK-positive patients with MG treated with rituximab to those not treated with rituximab. The primary clinical endpoint was the Myasthenia Gravis Status and Treatment Intensity (MGSTI), a novel outcome that combines the Myasthenia Gravis Foundation of America (MGFA) postintervention status (PIS) and the number and dosages of other immunosuppressant therapies used. A priori, an MGSTI of level ≤2 was used to define a favorable outcome. Secondary outcomes included modified MGFA PIS of minimal manifestations or better, mean/median prednisone dose, and mean/median doses of other immunosuppressant drugs. Results: Seventy-seven of 119 patients with anti-MuSK MG evaluated between January 1, 2005, and January 1, 2015, at 10 neuromuscular centers were selected for analysis after review of limited clinical data by a blinded expert panel. An additional 22 patients were excluded due to insufficient follow-up. Baseline characteristics were similar between the rituximab-treated patients (n = 24) and the controls (n = 31). Median follow-up duration was >3.5 years. At last visit, 58% (14/24) of rituximab-treated patients reached the primary outcome compared to 16% (5/31) of controls (p = 0.002). Number needed to treat for the primary outcome is 2.4. At last visit, 29% of rituximab-treated patients were taking prednisone (mean dose 4.5 mg/day) compared to 74% of controls (mean dose 13 mg/day) (p = 0.001 and p = 0.005). Classification of evidence: This study provides Class IV evidence that for patients with anti-MuSK MG, rituximab increased the probability of a favorable outcome.
AB - To evaluate the efficacy of rituximab in treatment of anti-muscle-specific kinase (MuSK) myasthenia gravis (MG). Methods: This was a multicenter, blinded, prospective review, comparing anti-MuSK-positive patients with MG treated with rituximab to those not treated with rituximab. The primary clinical endpoint was the Myasthenia Gravis Status and Treatment Intensity (MGSTI), a novel outcome that combines the Myasthenia Gravis Foundation of America (MGFA) postintervention status (PIS) and the number and dosages of other immunosuppressant therapies used. A priori, an MGSTI of level ≤2 was used to define a favorable outcome. Secondary outcomes included modified MGFA PIS of minimal manifestations or better, mean/median prednisone dose, and mean/median doses of other immunosuppressant drugs. Results: Seventy-seven of 119 patients with anti-MuSK MG evaluated between January 1, 2005, and January 1, 2015, at 10 neuromuscular centers were selected for analysis after review of limited clinical data by a blinded expert panel. An additional 22 patients were excluded due to insufficient follow-up. Baseline characteristics were similar between the rituximab-treated patients (n = 24) and the controls (n = 31). Median follow-up duration was >3.5 years. At last visit, 58% (14/24) of rituximab-treated patients reached the primary outcome compared to 16% (5/31) of controls (p = 0.002). Number needed to treat for the primary outcome is 2.4. At last visit, 29% of rituximab-treated patients were taking prednisone (mean dose 4.5 mg/day) compared to 74% of controls (mean dose 13 mg/day) (p = 0.001 and p = 0.005). Classification of evidence: This study provides Class IV evidence that for patients with anti-MuSK MG, rituximab increased the probability of a favorable outcome.
UR - http://www.scopus.com/inward/record.url?scp=85028806889&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85028806889&partnerID=8YFLogxK
U2 - 10.1212/WNL.0000000000004341
DO - 10.1212/WNL.0000000000004341
M3 - Review article
C2 - 28801338
AN - SCOPUS:85028806889
SN - 0028-3878
VL - 89
SP - 1069
EP - 1077
JO - Neurology
JF - Neurology
IS - 10
ER -