Riluzole use in presence of contraindications in adults affected by amyotrophic lateral sclerosis and its off-label use in other motor neuron diseases: Findings from an Italian multicentre study (the CAESAR project)

Crescioli, Giada; Finocchietti, Marco; Cascini, Silvia; Paoletti, Olga; Franchini, David; Cappello, Emiliano; Valdiserra, Giulia; Sciancalepore, Francesco; Celani, Maria Grazia; Tuccori, Marco; Vannacci, Alfredo; Lombardi, Niccolò; Kirchmayer, Ursula

doi:10.3389/fdsfr.2022.1041275

Cited by 5 publications

(5 citation statements)

References 10 publications

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“…Moreover, it is to be taken into account that our enrolment date may not be the true start of the disease. Of notice, these limitations may have different impacts in the three regions, because traceability of drug claims depends on the administrative procedures underlying drug registration under different reimbursement policies [ 19 , 20 , 33 ]. Furthermore, treatments administered to in-patients (immunomodulation therapies such as intravenous immunoglobulins and plasma exchange) and self-prescribed drugs (i.e., over-the-counter medications) are generally not retrievable at the patient level.…”

Section: Discussionmentioning

confidence: 99%

“…This is a real-world population-based retrospective cohort study, part of a multiregional Italian pharmacovigilance project on the comparative effectiveness and safety of drugs used in rare neuromuscular and neurodegenerative diseases (the CAESAR project) [ 19 , 20 ]. In the period 2013–2019, three Italian regions were involved: Latium, Tuscany, and Umbria.…”

Section: Methodsmentioning

confidence: 99%

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Drug Use Patterns in Myasthenia Gravis: A Real-World Population-Based Cohort Study in Italy

Finocchietti,

Crescioli,

Paoletti

et al. 2024

JCM

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Background: In the context of a comparative study of efficacy and safety of drugs used in rare neuromuscular and neurodegenerative diseases (CAESAR—call AIFA_FV_2012-13-14), we assessed the use patterns of drugs indicated for myasthenia gravis (MG). Methods: A retrospective cohort study was conducted based on administrative healthcare data. For a cohort of MG patients, prevalent and incident use of pyridostigmine (Py) and other indicated drugs in the first year after case identification was evaluated. Prevalent combined use of major therapies (azathioprine (Az), prednisone (Pr), vitamin D (Vd)) stratified by Py use was assessed, and a comparison between therapies at the time of MG identification and during the first year of follow-up was performed. Results: We included 2369 MG patients between 2013 and 2019. Among them, prevalent and incident Py users were 38.4% and 22.0%, respectively. In the first year of follow-up, the use of Pr was observed in 74.5% of Py prevalent users and in 82.0% of Py incident users, respectively; the use of Az was observed in 24.9% and 23.0%, respectively; and the use of Vd was observed in 53.3% and 48.2%, respectively. Among 910 Py prevalent users, 13.1% also used Az, Pr, and Vd, while 15.3% used none of these. Among 938 non-Py users, 2.7% used Az, Pr, and Vd, while 53.8% used none of these. During the first year, an increase in combined therapies was evident in incident Py users. Conclusions: Our results suggest that, for some MG patients, there may be a need for treatments that combine a rapid onset of benefit with long-term and consistent disease control. These issues may be addressed by the new treatments currently being developed. To date, more studies are needed to address the heterogeneity, quality, and generalizability of the existing data and to evaluate patterns of use, efficacy, and safety of new or emerging therapies for MG.

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Section: Discussionmentioning

confidence: 99%

Section: Methodsmentioning

confidence: 99%

Drug Use Patterns in Myasthenia Gravis: A Real-World Population-Based Cohort Study in Italy

Finocchietti,

Crescioli,

Paoletti

et al. 2024

JCM

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“…This is a real-world population-based retrospective cohort study, part of a multiregional Italian pharmacovigilance project on the comparative effectiveness and safety of drugs used in rare neuromuscular and neurodegenerative diseases (the CAESAR project) ( 12 ). In the period 2013–2019, three Italian regions were involved: Latium, Tuscany, and Umbria.…”

Section: Methodsmentioning

confidence: 99%

Potentially inappropriate drug use in myasthenia gravis: a real-world population-based cohort study in Italy

Crescioli,

Finocchietti,

Paoletti

et al. 2023

Front. Neurol.

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IntroductionTo evaluate the use of pyridostigmine in presence of contraindications, and the use of concomitant potentially contraindicated drugs in a cohort of patients affected by Myasthenia Gravis (MG) in the Italian Regions of Lazio, Tuscany, and Umbria.MethodsThis is a retrospective cohort study. A multivariate logistic regression model was used to evaluate the determinants of pyridostigmine and of potentially contraindicated drugs use in MG patients.ResultsAmong 591 incident pyridostigmine users affected by MG, 91 (15.4%) had at least one of the contraindications considered at the first prescription of pyridostigmine. Patients prescribed with pyridostigmine in presence of contraindications were more frequently affected by diabetes, obesity, and renal diseases. Age 75+ years (odds ratio, OR 4.94, 95% confidence interval, CI 1.60–15.22 for Latium; OR 3.78, 95%CI: 1.26–11.34 for Tuscany; OR 5.83, 95%CI 1.19–28.52 for Umbria), the presence of at least one specific comorbidity (OR 3.93; 95%CI 1.68–9.17 for Latium), and polytherapy (6+ drugs, OR 4.90, 95%CI: 1.35–17.85 for Tuscany) were found to be significantly associated with pyridostigmine use in presence of contraindications. Among patients affected by MG, 1,483 (62.6%) were treated with potentially contraindicated drugs in the first year of follow-up (67.06.9% in Latium; 59% in Tuscany; 57.6% in Umbria). Patients aged 75+ years, those with at least one specific complication or comorbidity, and those exposed to polytherapy were more likely to be treated with a potential contraindicated drug.ConclusionAmong incident users of pyridostigmine, more than 15% of patients have at least one of the contraindications considered, and among patients diagnosed with MG, in the first year of follow-up >60% of subjects were treated with potentially contraindicated drugs.

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“…Moreover, selected characteristics (demographic information, complications, comorbidities, pharmacological and non‐pharmacological therapy, healthcare service use) were explored as potential predictors of adherence. The study is part of the CAESAR project (‘Comparative evaluation of the efficacy and safety of drugs used in rare neuromuscular and neurodegenerative diseases’, AIFA call FV 2012‐13‐14) 21 …”

Section: Introductionmentioning

confidence: 99%

“…The study is part of the CAESAR project ('Comparative evaluation of the efficacy and safety of drugs used in rare neuromuscular and neurodegenerative diseases', AIFA call FV 2012-13-14). 21…”

Section: Introductionmentioning

confidence: 99%

Adherence to riluzole therapy in patients with amyotrophic lateral sclerosis in three Italian regions—The CAESAR study

Giometto,

Finocchietti,

Paoletti

et al. 2023

Pharmacoepidemiology and Drug

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PurposeAmyotrophic lateral sclerosis (ALS) is a rare neurodegenerative disease. Riluzole may increase survival and delay the need for mechanical ventilation. The CAESAR project (‘Comparative evaluation of the efficacy and safety of drugs used in rare neuromuscular and neurodegenerative diseases’, FV AIFA project 2012‐2013‐2014) involves evaluating prescribing patterns, and analysing effectiveness and comparative safety of drugs, in patients with neurodegenerative diseases. The aim of this study is to evaluate adherence to riluzole in patients with ALS during the first year of use, identifying adherence clusters.MethodsA retrospective cohort study was conducted using administrative data from Latium, Tuscany, and Umbria. We identified subjects with a new diagnosis of ALS between 2014 and 2019, with the first dispensation of riluzole within 180 days of diagnosis. We considered a two‐year look‐back period for the characterization of patients, and we followed them from the date of first dispensing of riluzole for 1 year. We calculated 12 monthly adherence measures, through a modified version of the Medication Possession Ratio, estimating drug coverage with Defined Daily Dose. Adherence trajectories were identified using a three‐step method: (1) calculation of statistical measures; (2) principal component analysis; (3) cluster analysis. Patient characteristics at baseline and during follow‐up were described and compared between adherence groups identified.ResultsWe included 264 ALS patients as new users of riluzole in Latium, 344 in Tuscany, and 63 in Umbria. We observed a higher frequency of males (56.2%) and a mean age of 67.4 (standard deviation, SD, 10.4) in the overall population. We identified two clusters in all regions: one more numerous, including adherent patients (60%, 74%, 88%, respectively), and another one including patients who discontinued therapy (40%, 26%, 12%, respectively). In Tuscany patients discontinuing riluzole more frequently died (28.6% vs. 15.4%, p‐value <0.01). Additionally, low‐adherers had a higher frequency of central nervous system disorders (69.0% vs. 52.5%, p‐value 0.01), and a greater use of non‐pharmacological treatments (p‐values ≤0.01 for invasive ventilation and tracheostomy). We did not observe any differences in Lazio, whereas in Umbria we observed a higher use of drugs for dementia‐related psychiatric problems among low‐adherers (57.1% vs. 7.8%, respectively, p‐value <0.01), although with small numbers.ConclusionMost ALS patients who start riluzole adhere to therapy during the first year. Patients who discontinue therapy early show greater fragility and mortality.

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Riluzole use in presence of contraindications in adults affected by amyotrophic lateral sclerosis and its off-label use in other motor neuron diseases: Findings from an Italian multicentre study (the CAESAR project)

Cited by 5 publications

References 10 publications

Drug Use Patterns in Myasthenia Gravis: A Real-World Population-Based Cohort Study in Italy

Drug Use Patterns in Myasthenia Gravis: A Real-World Population-Based Cohort Study in Italy

Potentially inappropriate drug use in myasthenia gravis: a real-world population-based cohort study in Italy

Adherence to riluzole therapy in patients with amyotrophic lateral sclerosis in three Italian regions—The CAESAR study

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