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

Ramdas, Sithara; Marina, Adela Della; Ryan, Monique M.; McWilliam, Kenneth; Klein, Andrea; Jacquier, David; Alabaf, Setareh; Childs, Anne‐Marie; Parasuraman, Deepak; Beeson, David; Palace, J; Jungbluth, Heinz

doi:10.1016/j.ejpn.2022.06.009

Cited by 9 publications

(6 citation statements)

References 24 publications

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“…Exposure to rituximab over longer periods increases the risk of severe infections, but not of cancer [44,45]. There are no randomized studies of rituximab in children with MG, but the drug should be considered if moderate and severe symptoms [46], especially if newly onset.…”

Section: Rituximabmentioning

confidence: 99%

Generalized myasthenia gravis with acetylcholine receptor antibodies: A guidance for treatment

Gilhus,

Andersen,

Andersen

et al. 2024

Euro J of Neurology

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BackgroundGeneralized myasthenia gravis (MG) with antibodies against the acetylcholine receptor is a chronic disease causing muscle weakness. Access to novel treatments warrants authoritative treatment recommendations. The Nordic countries have similar, comprehensive health systems, mandatory health registers, and extensive MG research.MethodsMG experts and patient representatives from the five Nordic countries formed a working group to prepare treatment guidance for MG based on a systematic literature search and consensus meetings.ResultsPyridostigmine represents the first‐line symptomatic treatment, while ambenonium and beta adrenergic agonists are second‐line options. Early thymectomy should be undertaken if a thymoma, and in non‐thymoma patients up to the age of 50–65 years if not obtaining remission on symptomatic treatment. Most patients need immunosuppressive drug treatment. Combining corticosteroids at the lowest possible dose with azathioprine is recommended, rituximab being an alternative first‐line option. Mycophenolate, methotrexate, and tacrolimus represent second‐line immunosuppression. Plasma exchange and intravenous immunoglobulin are used for myasthenic crises and acute exacerbations. Novel complement inhibitors and FcRn blockers are effective and fast‐acting treatments with promising safety profiles. Their use depends on local availability, refunding policies, and cost–benefit analyses. Adapted physical training is recommended. Planning of pregnancies with optimal treatment, information, and awareness of neonatal MG is necessary. Social support and adaptation of work and daily life activities are recommended.ConclusionsSuccessful treatment of MG rests on timely combination of different interventions. Due to spontaneous disease fluctuations, comorbidities, and changes in life conditions, regular long‐term specialized follow‐up is needed. Most patients do reasonably well but there is room for further improvement. Novel treatments are promising, though subject to restricted access due to costs.

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Section: Rituximabmentioning

confidence: 99%

Generalized myasthenia gravis with acetylcholine receptor antibodies: A guidance for treatment

Gilhus,

Andersen,

Andersen

et al. 2024

Euro J of Neurology

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“…Nevertheless, the lack of precise demographic data and the small sample size limit the generalizability of these findings. However, pediatric neuromuscular disorders are rare, and a small sample size is not unusual for this population [17][18][19][20][21][22][23][24]. Other limitations include possible selection bias, in particular sampling and volunteer bias subtypes.…”

Section: The Overall Psychosocial Impact Of Covid-19 On Children and ...mentioning

confidence: 99%

The Impact of COVID-19 on Families With Pediatric Muscular Dystrophy Patients

Zubair¹,

Scharer²,

Ionita³

et al. 2023

Cureus

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The coronavirus disease 2019 (COVID-19) pandemic resulted in unprecedented changes in daily activities and healthcare services. In the United States, stay-at-home orders and social distancing measures were put, and school closures impacted many students. The psychological impact of the COVID-19 pandemic has been shown to have wide-ranging and long-term effects. With school closures and limitations in in-person visits and provider care, we hypothesized that the patients with pediatric muscular dystrophies and neuromuscular conditions were more vulnerable to the restriction posed by this pandemic. This surveybased study examined the psychosocial impact of this pandemic on pediatric patients with neuromuscular disorders and caregiver burden through chart review and self-reports via survey administration using a validated tool (COVID-19 Exposure and Family Impact Scales {CEFIS}). The majority of families reported that they had a stay-at-home order (91.7%), schools/childcare centers were closed (87.5%), their children's education was disrupted (83.3%), and they were unable to visit or care for a family member (58.3%). Parents/caregivers felt that the COVID-19 pandemic made parenting a little bit worse (mean = 2.6 ± 0.96) and made it more difficult to care for the elderly or those with disabilities in the family (mean = 2.6 ± 0.95) and for their child with a neuromuscular disability (mean = 2.6 ± 0.91). Our data highlights the significant impact of the COVID-19 pandemic on the lives of families and caregivers of pediatric patients with muscular dystrophies.

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Section: Myasthenia Gravis (Mg)mentioning

confidence: 99%

“…Treatment is mainly derived from adult guidelines, given the analogous pathobiological mechanisms and the absence of pediatric clinical trials [136]. Pyridostigmine is usually the first line of therapy, although additive immunosuppressants (corticosteroids, steroid-sparing agents, and anti-B cell agents), and/or thymectomy are often required [136]. HD-IVIG and/or PLEX can induce a prompt short-lasting response in patients with myasthenic crises or severe motor impairment but can also be useful in maintaining remission through monthly cycles, in cases of refractoriness or intolerance to other treatment options [136,137].…”

Section: Myasthenia Gravis (Mg)mentioning

confidence: 99%

Anti-Inflammatory and Immunomodulatory Effect of High-Dose Immunoglobulins in Children: From Approved Indications to Off-Label Use

Conti,

Moratti,

Leonardi

et al. 2023

Cells

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Background: The large-scale utilization of immunoglobulins in patients with inborn errors of immunity (IEIs) since 1952 prompted the discovery of their key role at high doses as immunomodulatory and anti-inflammatory therapy, in the treatment of IEI-related immune dysregulation disorders, according to labelled and off-label indications. Recent years have been dominated by a progressive imbalance between the gradual but constant increase in the use of immunoglobulins and their availability, exacerbated by the SARS-CoV-2 pandemic. Objectives: To provide pragmatic indications for a need-based application of high-dose immunoglobulins in the pediatric context. Sources: A literature search was performed using PubMed, from inception until 1st August 2023, including the following keywords: anti-inflammatory; children; high dose gammaglobulin; high dose immunoglobulin; immune dysregulation; immunomodulation; immunomodulatory; inflammation; intravenous gammaglobulin; intravenous immunoglobulin; off-label; pediatric; subcutaneous gammaglobulin; subcutaneous immunoglobulin. All article types were considered. Implications: In the light of the current imbalance between gammaglobulins’ demand and availability, this review advocates the urgency of a more conscious utilization of this medical product, giving indications about benefits, risks, cost-effectiveness, and administration routes of high-dose immunoglobulins in children with hematologic, neurologic, and inflammatory immune dysregulation disorders, prompting further research towards a responsible employment of gammaglobulins and improving the therapeutical decisional process.

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

Cited by 9 publications

References 24 publications

Generalized myasthenia gravis with acetylcholine receptor antibodies: A guidance for treatment

Generalized myasthenia gravis with acetylcholine receptor antibodies: A guidance for treatment

The Impact of COVID-19 on Families With Pediatric Muscular Dystrophy Patients

Anti-Inflammatory and Immunomodulatory Effect of High-Dose Immunoglobulins in Children: From Approved Indications to Off-Label Use

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