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

TY - JOUR

T1 - Rituximab in juvenile myasthenia gravis-an international cohort study and literature review

AU - Ramdas, Sithara

AU - Della Marina, Adela

AU - Ryan, Monique M.

AU - McWilliam, Kenneth

AU - Klein, Andrea

AU - Jacquier, David

AU - Alabaf, Setareh

AU - Childs, Anne Marie

AU - Parasuraman, Deepak

AU - Beeson, David

AU - Palace, Jacqueline

AU - Jungbluth, Heinz

N1 - Publisher Copyright: © 2022

PY - 2022/9

Y1 - 2022/9

N2 - 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.

AB - 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.

KW - Juvenile myasthenia gravis

KW - Myasthenia gravis

KW - Paediatric

KW - Rituximab

UR - http://www.scopus.com/inward/record.url?scp=85133924829&partnerID=8YFLogxK

U2 - 10.1016/j.ejpn.2022.06.009

DO - 10.1016/j.ejpn.2022.06.009

M3 - Article

AN - SCOPUS:85133924829

SN - 1090-3798

VL - 40

SP - 5

EP - 10

JO - European Journal of Paediatric Neurology

JF - European Journal of Paediatric Neurology

ER -