Rituximab as treatment for anti-MuSK myasthenia gravis

Michael K. Hehir, Lisa D. Hobson-Webb, Michael G Benatar, Carolina Barnett, Nicholas J. Silvestri, James F. Howard, DIantha Howard, Amy Visser, Brian A. Crum, Richard Nowak, Rachel Beekman, Aditya Kumar, Katherine Ruzhansky, I. Hweii Amy Chen, Michael T. Pulley, Shannon M. Laboy, Melissa A. Fellman, Shane M. Greene, Mamatha Pasnoor, Ted M. Burns

Research output: Contribution to journalReview article

28 Citations (Scopus)

Abstract

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.

Original languageEnglish (US)
Pages (from-to)1069-1077
Number of pages9
JournalNeurology
Volume89
Issue number10
DOIs
StatePublished - Sep 5 2017

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Myasthenia Gravis
Phosphotransferases
Muscles
Therapeutics
Immunosuppressive Agents
Prednisone
Numbers Needed To Treat
Rituximab
Pharmaceutical Preparations

ASJC Scopus subject areas

  • Clinical Neurology

Cite this

Hehir, M. K., Hobson-Webb, L. D., Benatar, M. G., Barnett, C., Silvestri, N. J., Howard, J. F., ... Burns, T. M. (2017). Rituximab as treatment for anti-MuSK myasthenia gravis. Neurology, 89(10), 1069-1077. https://doi.org/10.1212/WNL.0000000000004341

Rituximab as treatment for anti-MuSK myasthenia gravis. / Hehir, Michael K.; Hobson-Webb, Lisa D.; Benatar, Michael G; Barnett, Carolina; Silvestri, Nicholas J.; Howard, James F.; Howard, DIantha; Visser, Amy; Crum, Brian A.; Nowak, Richard; Beekman, Rachel; Kumar, Aditya; Ruzhansky, Katherine; Chen, I. Hweii Amy; Pulley, Michael T.; Laboy, Shannon M.; Fellman, Melissa A.; Greene, Shane M.; Pasnoor, Mamatha; Burns, Ted M.

In: Neurology, Vol. 89, No. 10, 05.09.2017, p. 1069-1077.

Research output: Contribution to journalReview article

Hehir, MK, Hobson-Webb, LD, Benatar, MG, Barnett, C, Silvestri, NJ, Howard, JF, Howard, DI, Visser, A, Crum, BA, Nowak, R, Beekman, R, Kumar, A, Ruzhansky, K, Chen, IHA, Pulley, MT, Laboy, SM, Fellman, MA, Greene, SM, Pasnoor, M & Burns, TM 2017, 'Rituximab as treatment for anti-MuSK myasthenia gravis', Neurology, vol. 89, no. 10, pp. 1069-1077. https://doi.org/10.1212/WNL.0000000000004341
Hehir MK, Hobson-Webb LD, Benatar MG, Barnett C, Silvestri NJ, Howard JF et al. Rituximab as treatment for anti-MuSK myasthenia gravis. Neurology. 2017 Sep 5;89(10):1069-1077. https://doi.org/10.1212/WNL.0000000000004341
Hehir, Michael K. ; Hobson-Webb, Lisa D. ; Benatar, Michael G ; Barnett, Carolina ; Silvestri, Nicholas J. ; Howard, James F. ; Howard, DIantha ; Visser, Amy ; Crum, Brian A. ; Nowak, Richard ; Beekman, Rachel ; Kumar, Aditya ; Ruzhansky, Katherine ; Chen, I. Hweii Amy ; Pulley, Michael T. ; Laboy, Shannon M. ; Fellman, Melissa A. ; Greene, Shane M. ; Pasnoor, Mamatha ; Burns, Ted M. / Rituximab as treatment for anti-MuSK myasthenia gravis. In: Neurology. 2017 ; Vol. 89, No. 10. pp. 1069-1077.
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abstract = "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.",
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AU - Hehir, Michael K.

AU - Hobson-Webb, Lisa D.

AU - Benatar, Michael G

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.

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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.

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