Rituximab in juvenile myasthenia gravis-an international cohort study and literature review


      • Rituximab is an effective treatment option in moderate to severe juvenile myasthenia gravis.
      • Post rituximab treatment there is reduction in hospital admissions related to myasthenia gravis and also need for immunomodulatory treatment.
      • Commonest side effect with rituximab is infusion-related reactions and these respond well to standard symptomatic treatments.


      Juvenile myasthenia gravis (JMG) is a rare, antibody-mediated disorder of the neuromuscular junction. Treatment strategies in JMG are largely informed by adult MG treatments as the pathophysiology is similar. Rituximab is increasingly considered as a treatment option in refractory JMG but has not yet been systematically investigated in this patient group We conducted a retrospective study from five international centres with expertise in paediatric myasthenia. 10 JMG patients treated with rituximab were identified. Following rituximab treatment all patients had a reduction in JMG-related hospital admissions. At 24 month follow up, 6 patients (60%) had achieved complete stable remission or pharmacological remission and 7 patients were able to reduce immunomodulatory treatment(s). The main side-effect was infusion-related reactions (30%) which resolved in all patients with symptomatic treatment. We compared our cohort to previously reported JMG cases treated with rituximab and noted similar response rates but a slightly higher side-effect profile. Rituximab is a safe and effective treatment option in moderate to severe JMG and most patients have an improvement in MG symptoms post treatment.


      To read this article in full you will need to make a payment

      Purchase one-time access:

      Academic & Personal: 24 hour online accessCorporate R&D Professionals: 24 hour online access
      One-time access price info
      • For academic or personal research use, select 'Academic and Personal'
      • For corporate R&D use, select 'Corporate R&D Professionals'


      Subscribe to European Journal of Paediatric Neurology
      Already a print subscriber? Claim online access
      Already an online subscriber? Sign in
      Institutional Access: Sign in to ScienceDirect


        • O'Connell K.
        • Ramdas S.
        • Palace J.
        Management of juvenile myasthenia gravis.
        Front. Neurol. 2020 Jul 24; 11: 743
        • Rodríguez Cruz P.M.
        • Al-Hajjar M.
        • Huda S.
        • Jacobson L.
        • Woodhall M.
        • Jayawant S.
        • et al.
        Clinical features and diagnostic usefulness of antibodies to clustered acetylcholine receptors in the diagnosis of seronegative myasthenia gravis.
        JAMA Neurol. 2015 Jun; 72: 642-649
        • Vecchio D.
        • Ramdas S.
        • Munot P.
        • Pitt M.
        • Beeson D.
        • Knight R.
        • et al.
        Paediatric myasthenia gravis: prognostic factors for drug free remission.
        Neuromuscul. Disord. 2020 Feb; 30: 120-127
        • Pescovitz M.D.
        Rituximab, an anti-CD20 monoclonal antibody: history and mechanism of action.
        Am. J. Transplant. 2006 May; 6: 859-866
        • Tandan R.
        • Hehir M.K.
        • Waheed W.
        • Howard D.B.
        Rituximab treatment of myasthenia gravis: a systematic review.
        Muscle Nerve. 2017; 56: 185-219
        • Di Stefano V.
        • Lupica A.
        • Rispoli M.G.
        • Di Muzio A.
        • Brighina F.
        • Rodolico C.
        Rituximab in AChR subtype of myasthenia gravis: systematic review. Journal of Neurology.
        Neurosurg. Psychiatr. 2020; 91: 392-395
        • Barraud C.
        • Desguerre I.
        • Barnerias C.
        • Gitiaux C.
        • Boulay C.
        • Chabrol B.
        Clinical features and evolution of juvenile myasthenia gravis in a French cohort.
        Muscle Nerve. 2018; 57: 603e609
        • Govindarajan R.
        • Iyadurai S.J.
        • Connolly A.
        • Zaidman C.
        Selective response to rituximab in a young child with MuSK-associated myasthenia gravis.
        Neuromuscul. Disord. 2015; 25: 651e652
        • Koul R.
        • Al-Futaisi A.
        • Abdelrahim R.
        • Mani R.
        • Abdwani R.
        • Al-Asmi A.
        Rituximab treatment in myasthaenia gravis: report of two paediatric cases.
        Sultan Qaboos Univ. Med. J. 2018; 18 (e223ee227)
        • Koul R.
        • Al-Futaisi A.
        • Abdwani R.
        Rituximab in severe seronegative juvenile myasthenia gravis: review of the literature.
        Pediatr. Neurol. 2012 Sep; 47: 209-212
      1. Zingariello CD, Melissa EE, Kang PB. Rituximab as adjunct maintenance therapy for refractory juvenile myasthenia gravis. Pediatr Neurol.

        • Wylam M.E.
        • Anderson P.M.
        • Kuntz N.L.
        • Rodriguez V.
        Successful treatment of refractory myasthenia gravis using rituximab: a pediatric case report.
        J. Pediatr. 2003 Nov; 143: 674-677
        • Tzaribachev N.
        • Koetter I.
        • Kuemmerle-Deschner J.B.
        • Schedel J.
        Rituximab for the treatment of refractory pediatric autoimmune diseases: a case series.
        Cases J. 2009; 2: 6609
        • Skjei K.J.
        • Lennon V.A.
        • Kuntz N.L.
        Muscle specific kinase autoimmune myasthenia gravis in children: a case series.
        Neuromuscul. Disord. 2013; 23: 874-882
        • Weger S.
        • Appendino J.P.
        • Clark I.H.
        Longstanding and refractory anti-muscle specific tyrosine kinase antibody-associated myasthenia gravis (Anti-MuSK-MG) in a child successfully treated with rituximab.
        J. Binocul. Vis. Ocul. Motil. 2019; 69: 26-29
        • Lindberg C.
        • Bokarewa M.
        Rituximab for severe myasthenia gravis–experience from five patients.
        Acta Neurol. Scand. 2010 Oct; 122: 225-228
        • Maddison P.
        • McConville J.
        • Farrugia M.E.
        • Davies N.
        • Rose M.
        • Norwood F.
        • Jungbluth H.
        • Robb S.
        • Hilton-Jones D.
        The use of rituximab in myasthenia gravis and Lambert-Eaton myasthenic syndrome.
        J. Neurol. Neurosurg. Psychiatry. 2011 Jun; 82: 671-673
        • Jaretzki 3rd, A.
        • Barohn R.J.
        • Ernstoff R.M.
        • et al.
        Myasthenia gravis: recommendations for clinical research standards. Task force of the medical scientific advisory board of the myasthenia gravis foundation of America.
        Neurology. 2000; 55: 16-23
        • Nowak R.J.
        • Coffey C.
        • Goldstein J.A.A.N.
        2018 Emerging science abstracts: B-cell targeted treatment in myasthenia gravis (BeatMG) - a phase 2 trial of rituximab in myasthenia gravis: topline results.
        Neurology. 2018; 90: e2182-e2194
        • Kemper M.J.
        • Gellermann J.
        • Habbig S.
        • Krmar R.T.
        • Dittrich K.
        • Jungraithmayr T.
        • et al.
        Long-term follow-up after rituximab for steroid-dependent idiopathic nephrotic syndrome.
        Nephrol. Dial. Transplant. 2012 May; 27: 1910-1915
        • Mahmoud I.
        • Jellouli M.
        • Boukhris I.
        • Charfi R.
        • Tekaya A.B.
        • Saidane O.
        • et al.
        Efficacy and safety of rituximab in the management of pediatric systemic lupus erythematosus: a systematic review.
        J. Pediatr. 2017 Aug; 187: 213-219
        • Qu M.
        • Zhou J.
        • Yang S.J.
        • Zhou Z.P.
        Efficacy and safety of rituximab for minors with immune thrombocytopenia: a systematic review and meta-analysis.
        J. Int. Med. Res. 2020 Oct; 48 (300060520962348)
        • Olfat M.
        • Silverman E.D.
        • Levy D.M.
        Rituximab therapy has a rapid and durable response for refractory cytopenia in childhood-onset systemic lupus erythematosus.
        Lupus. 2015; 24: 966-972
        • Khojah A.M.
        • Miller M.L.
        • Klein-Gitelman M.S.
        • Curran M.L.
        • Hans V.
        • Pachman L.M.
        • Fuleihan R.L.
        Rituximab-associated Hypogammaglobulinemia in pediatric patients with autoimmune diseases.
        Pediatr. Rheumatol. Online J. 2019 Aug 28; 17: 61
        • Deyà-Martínez A.
        • Gordón Y.
        • Molina-Anguita C.
        • Vlagea A.
        • Piquer M.
        • Juan M.
        • et al.
        Single-cycle rituximab-induced immunologic changes in children: enhanced in neuroimmunologic disease?.
        Neurol. Neuroimmunol. Neuroinflamm. 2020 May 6; 7: e724