New guidelines on the management of amyotrophic lateral sclerosis (ALS) were presented at the EAN 2023 meeting.
New guidelines on the management of amyotrophic lateral sclerosis (ALS) were presented at the EAN 2023 meeting and are expected to be published before the end of 2023. The guidelines are a joint effort of the EAN in collaboration with ERN Euro-NMD. Three new drugs soon to be evaluated may necessitate an update of these guidelines soon.
The new ALS guidelines were presented at EAN 2023 by Philip van Damme, MD, PhD. This version replaces the previous guidelines dating back to 2012. Dr. Van Damme explained that 26 research questions were identified using the PICO framework. Of these, eight were recommendations adapted from the British NICE guidelines (NICE guideline NG42: Motor neurone disease); the other 16 were de novo recommendations.
A cornerstone of ALS management is multidisciplinary care; the evidence is based mostly on observational studies. A team ideally consists of an ALS neurologist, a respiratory specialist, a nursing professional, a mental health professional, a social worker, a speech/language pathologist, a dietician, and a physical therapist.
The main recommendations concerning disease-modifying therapies are the following:
- Riluzole should be offered lifelong to all ALS patients at diagnosis. “A single daily dose of 50 mg can already be effective,” added Dr. Van Damme.
- Cell-based therapies are not recommended outside the context of clinical trials until positive phase 3 trial data are available.
- Edaravone and AMX0035 both receive temporary recommendations; the guideline committee wants to await the phase 3 trial results before issuing a final recommendation.
- Tofersen should be offered as first-line treatment in patients with progressive ALS caused by mutations in superoxide dismutase 1 (SOD1). The possibility of serious adverse events should be discussed with the patient.
Randomized controlled trials for nutritional interventions are ongoing, and there are none for gastrostomy. Yet, the benefits of early placement and the possible risks of a late gastrostomy should be discussed at an early stage and thereafter, the guidelines advise.
In conclusion, Dr. Van Damme expressed hope that an update will be necessary soon, as three additional drugs are likely to be evaluated by the European Medicines Agency in the coming year.
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