Background: This analysis describes the use of riluzole in amyotrophic lateral sclerosis (ALS) individuals with contraindications and off-label use for subjects with other motor neuron diseases (o-MND) in the Italian regions of Latium, Tuscany and Umbria.Methods: A cohort of adults with ALS prescribed with riluzole during the years 2016–2019 was enrolled from administrative healthcare databases, excluding subjects with o-MND in the preceding 5 years. Being affected by ALS for more than 5 years, presence of tracheostomy, renal or hepatic failure were considered as contraindications to the use of riluzole. A cohort of adults with o-MND was enrolled in 2016–2019 for whom off-label use of riluzole was retrieved up to 4 years, analysing over the time differences related to sex.Results: Among 206 ALS individuals prescribed with riluzole in Latium, 336 in Tuscany and 60 in Umbria, less than 1% were diagnosed with ALS for more than 5 years. Less than 2% were tracheotomised or affected by hepatic failure. Renal failure was documented for 1.9%, 2.7%, and 5.0% of ALS individuals in Latium, Tuscany and Umbria. The o-MND cohort comprised 264 subjects in Latium, 222 in Tuscany, and 66 in Umbria. Non-negligible off-label riluzole use was observed: 8.5%, 33.0%, and 4.2% in females, and 19.9%, 26.5% and 2.4% in males in Latium, Tuscany and Umbria.Discussion: Riluzole use in ALS individuals in the presence of contraindications is rare, with slightly higher numbers in presence of renal failure. Off-label use in o-MND was found to be non-negligible, with variations between sexes.

Background Studies of the epidemiology of amyotrophic lateral sclerosis (ALS) are numerous, with incidence and prevalence estimates varying widely depending on population characteristics, geographical areas, and available data sources. The aim of this study was to estimate prevalence and incidence of ALS in three Italian regions (Latium, Tuscany, and Umbria) using health administrative databases. Methods ALS patients residing in Latium, Umbria and Tuscany were identified through an algorithm based on data from three different health administrative databases: hospital discharges, exemptions from health care co-payment, and emergency departments (study period 2014-2019). We calculated crude, age- and gender specific prevalence estimates on December 31, 2019 and standardised incidence rates of ALS by region, year, and sex between 2014-2019. Moreover, using a clinical dataset available in the Lazio region, we calculated the proportion of individuals correctly identified as ALS cases by the algorithm in this region. Results A total of 1,031 persons affected by ALS, aged ≥ 18 years at the prevalence day were identified: 408 cases in Tuscany, 546 in Latium, and 77 in Umbria. Diseases specific co-payment exemptions accounted for the biggest contribution for case detection, ranging between 24.7% in Umbria and 30.6% in Latium. The algorithm produced standardised overall ALS prevalence rates similar between the three regions, varying between 12.31/100,000 in Tuscany, 11.52/100,000 in Latium and 9.90/100,000 in Umbria. The 5-year crude rates were higher in men, and in people aged 65-79 years. Among 310 patients included in the clinical dataset, 263 (84.8%) were identified by the algorithm based on health administrative databases. Conclusions ALS prevalence and incidence in three regions of Central Italy are rather similar but slightly higher than those previously reported. This finding is plausible given that previous results relate to at least ten years ago and evidenced of increasing trends. Overall, our results encourage the use of administrative data to produce occurrence estimates, useful to both, epidemiological surveillance and research and healthcare policies.