Myasthenia gravis

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Real-life pharmacovigilance identifies unreported side effects of eculizumab and alglucosidase alpha

Two Chinese teams looked at the real-life side-effects of eculizumab in the treatment of myasthenia and alglucosidase alfa in the treatment of Pompe disease. Analysis of data from the FDA’s pharmacovigilance system, FAERS (FDA Adverse Event Reporting System), revealed : 46,316 side-effect reports for eculizumab between 2007 and 2023, grouped under 461 preferred terms; 4,326 … [Read more]

Positive phase III results for nipocalimab in generalised myasthenia gravis

The Vivacity-MG3 phase III trial included 153 adults with generalised myasthenia to evaluate nipocalimab, a monoclonal antibody targeting the neonatal Fc receptor (FcRn). Of all the patients, 88% had anti-RACh autoantibodies, 11% had anti-MuSK autoantibodies and 2% had anti-LRP4 autoantibodies. Patients were randomised into two groups: 77 received nipocalimab and 76 a placebo, every fortnight … [Read more]

A majority of patients with refractory myasthenia gravis experience durable remission after autologous haematopoietic stem cell transplantation

Following an initial report of three cases in 2022, a Canadian team has published the results of a retrospective study of 21 patients with severe and refractory Myasthenia Gravis with anti-MusK or anti-RACh, treated by autologous haematopoietic stem cell transplantation 1.9 to 21.8 years after diagnosis. Out of 18 evaluable patients : 16 achieved and … [Read more]

Searching for markers of myasthenia gravis in French National Health Insurance data

Over the period 2013-2020, 14,459 people in France received reimbursements for care related to autoimmune myasthenia gravis, including 6,354 for the first time. Of these, : 34.6% were hospitalised at least once in intensive care, 2,817 patients received IVIG and 432 received plasma exchange, these events, which indicate poor control of the disease, mainly occurred … [Read more]

An algorithm to assist diagnostic for seronegative autoimmune myasthenia gravis

At the 275th workshop of the European Neuromuscular Centre (ENMC), held in February 2024 in the Netherlands, experts and patient representatives met to review the diagnosis and management of seronegative autoimmune myasthenia gravis in the light of the latest advances. They concluded that : seronegative autoimmune myasthenia (undetectable autoantibodies) affects 10 to 15% of patients … [Read more]

From diagnosis to treatment, antisynthetase syndrome is attracting international attention

In France, the National Data Bank for Rare Diseases lists 1,156 patients followed up in Centres of Reference or Competence for an anti-synthetase syndrome (SAS), an entity associated with overlapping myositis, which until now has lacked a consensual definition. The members of the international project Classification Criteria for Anti-synthetase Syndrome (Class) : have identified clinical … [Read more]

Post-hoc news on efgartigimod in anti-RACh myasthenia gravis

The phase III placebo-controlled Adapt trial and its open-label extension Adapt+ evaluated the efficacy of efgartigimod, an anti-FcRN, in generalised autoimmune myasthenia gravis. They ended in 2020 and 2022 respectively. Their results were the subject of a post hoc analysis, which showed that, in participants with anti-RACh : a significantly higher percentage of patients treated … [Read more]

Stamina analyses the treatment of myasthenia in ‘real life’ in France

Based on data from the French National Health Data System (SNDS), the Stamina study reviewed the management of two groups of adults with autoimmune myasthenia gravis: the ‘prevalent’ population, of 22,079 patients diagnosed before 31 December 2019, the ‘incident’ population, comprising 2,661 patients diagnosed in 2012 or 2013. A study of their follow-up shows that … [Read more]

Clinical and biological markers to differentiate between ocular and generalised forms of myasthenia gravis

Danish clinicians reviewed 350 records of patients with ocular or generalised forms of autoimmune myasthenia : clinical, electrophysiological and serological data were compiled and compared, 15.7% of patients had ocular myasthenia, the onset was later than in generalised forms, with lower levels of autoantibodies against the acetylcholine receptor (ARch), diagnostic odyssey was also longer, and … [Read more]

Ventilatory weaning and myasthenic crisis: the experience of Raymond Poincaré University Hospital

Clinicians in the western Ile-de-France region have retrospectively analyzed the clinical and paraclinical data of 126 myasthenia gravis patients who presented with a first myasthenic episode during the course of their disease, over the period 2001-2018: 18 of the 126 patients (i.e. 14%) could not be weaned off their ventilator after a first extubation attempt, … [Read more]