Recommendations for the management of myasthenia gravis in Belgium.

International guidelines on the treatment of myasthenia gravis (MG) have been published but are not tailored to the Belgian situation. This publication presents recommendations from a group of Belgian MG experts for the practical management of MG in Belgium. It includes recommendations for treatment of adult patients with generalized myasthenia gravis (gMG) or ocular myasthenia gravis (oMG). Depending on the MG-related antibody a treatment sequence is suggested with therapies that can be added on if the treatment goal is not achieved. Selection of treatments was based on the level of evidence of efficacy, registration and reimbursement status in Belgium, common daily practice and the personal views and experiences of the authors. The paper reflects the situation in February 2024. In addition to the treatment considerations, other relevant aspects in the management of MG are addressed, including comorbidities, drugs aggravating disease symptoms, pregnancy, and vaccination. As many new treatments might potentially come to market, a realistic future perspective on the impact of these treatments on clinical practice is given. In conclusion, these recommendations intend to be a guide for neurologists treating patients with MG in Belgium.

A retrospective survey of patients with hereditary transthyretin-mediated (hATTR) amyloidosis treated with patisiran in real-world clinical practice in Belgium.

INTRODUCTION: Hereditary transthyretin-mediated (hATTR) amyloidosis, a genetic disease caused by mutations in the transthyretin gene, leads to progressive sensory and autonomic neuropathy and/or cardiomyopathy and is associated with renal and ophthalmologic manifestations and a poor prognosis. METHODS: This is a retrospective study based on data collected from the medical records of patients with hATTR amyloidosis treated with patisiran between 01 July 2018 and 01 February 2021. Six Belgian neuromuscular reference centers participated, covering all patisiran-treated hATTR amyloidosis patients at the study time. This study was conducted to collect data requested in the context of the reimbursement of patisiran in Belgium. RESULTS: Thirty-one patients were diagnosed with hATTR amyloidosis with polyneuropathy, Coutinho stage 1 or 2, and eligible for active treatment during the data collection period. Of the hATTR amyloidosis patients treated with patisiran (n = 12), seven and five had polyneuropathy stages 1 and 2, respectively. Six patients had cardiac symptoms (New York Heart Association class 2 or above). Follow-up information was available for nine patients. Following patisiran treatment, eight patients showed stable or improved assessments for most neurological or cardiological parameters. Only one patient presented with worsening statuses at the end of the data collection period. CONCLUSIONS: The patients with hATTR amyloidosis in Belgium have similar baseline demographics and disease characteristics to those studied in the patisiran APOLLO study and show a similar therapeutic response in the real-world, altering the expected disease progression in most patients.

Brain adaptation following various unilateral vocal fold paralysis treatments: A magnetic resonance imaging based longitudinal case series.

Aim: Examination of central compensatory mechanisms following peripheral vocal nerve injury and recovery is essential to build knowledge about plasticity of the neural network underlying phonation. The objective of this prospective multiple-cases longitudinal study is to describe brain activity in response to unilateral vocal fold paralysis (UVFP) management and to follow central nervous system adaptation over time in three patients with different nervous and vocal recovery profiles. Materials and methods: Participants were enrolled within 3 months of the onset of UVFP. Within 1 year of the injury, the first patient did not recover voice or vocal fold mobility despite voice therapy, the second patient recovered voice and mobility in absence of treatment and the third patient recovered voice and vocal fold mobility following an injection augmentation with hyaluronic acid in the paralyzed vocal fold. These different evolutions allowed comparison of individual outcomes according to nervous and vocal recovery. All three patients underwent functional magnetic resonance imaging (fMRI task and resting-state) scans at three (patient 1) or four (patients 2 and 3) time points. The fMRI task included three conditions: a condition of phonation and audition of the sustained [a:] vowel for 3 s, an audition condition of this vowel and a resting condition. Acoustic and aerodynamic measures as well as laryngostroboscopic images and laryngeal electromyographic data were collected. Results and conclusion: This study highlighted for the first time two key findings. First, hyperactivation during the fMRI phonation task was observed at the first time point following the onset of UVFP and this hyperactivation was related to an increase in resting-state connectivity between previoulsy described phonatory regions of interest. Second, for the patient who received an augmentation injection in the paralyzed vocal fold, we subsequently observed a bilateral activation of the voice-related nuclei in the brainstem. This new observation, along with the fact that for this patient the resting-state connectivity between the voice motor/sensory brainstem nuclei and other brain regions of interest correlated with an aerodynamic measure of voice, support the idea that there is a need to investigate whether the neural recovery process can be enhanced by promoting the restoration of proprioceptive feedback.