Ramdas, Sithara; Della Marina, Adela; Ryan, Monique M; McWilliam, Kenneth; Klein, Andrea; Jacquier, David; Alabaf, Setareh; Childs, Anne-Marie; Parasuraman, Deepak; Beeson, David; Palace, Jacqueline; Jungbluth, Heinz (2022). Rituximab in juvenile myasthenia gravis-an international cohort study and literature review. European journal of paediatric neurology, 40, pp. 5-10. Elsevier 10.1016/j.ejpn.2022.06.009
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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.
Item Type: |
Journal Article (Original Article) |
|---|---|
Division/Institute: |
04 Faculty of Medicine > Department of Gynaecology, Paediatrics and Endocrinology (DFKE) > Clinic of Paediatric Medicine 04 Faculty of Medicine > Department of Gynaecology, Paediatrics and Endocrinology (DFKE) > Clinic of Paediatric Medicine > Neuropaediatrics |
UniBE Contributor: |
Klein, Andrea Katharina |
Subjects: |
600 Technology > 610 Medicine & health |
ISSN: |
1090-3798 |
Publisher: |
Elsevier |
Language: |
English |
Submitter: |
Anette van Dorland |
Date Deposited: |
18 Jul 2022 08:28 |
Last Modified: |
05 Dec 2022 16:21 |
Publisher DOI: |
10.1016/j.ejpn.2022.06.009 |
PubMed ID: |
35835035 |
Uncontrolled Keywords: |
Juvenile myasthenia gravis Myasthenia gravis Paediatric Rituximab |
BORIS DOI: |
10.48350/171324 |
URI: |
https://boris.unibe.ch/id/eprint/171324 |
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