Data from: Adverse events associated with risdiplam use
Data files
Mar 13, 2024 version files 1.02 MB
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1.Demographics_of_the_target_drug_population.xls
32.77 KB
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2.MedDRA_signal_calculations_by_level.xlsx
118.70 KB
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3.Number_of_PT_signals_at_SOC.xlsx
17.48 KB
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4.Calculation_of_signals_in_each_MedDRA_tier_stratified_by_gender.xlsx
256.32 KB
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5.Calculation_of_signals_in_each_MedDRA_tier_stratified_by_age_group.xlsx
285.95 KB
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6.Calculation_of_signals_in_each_MedDRA_tier__stratified_by_reporting_country.xlsx
305.85 KB
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README.md
1.99 KB
Abstract
Risdiplam is a new drug for treating spinal muscular atrophy (SMA). However, pharmacovigilance analyses are necessary to objectively evaluate its safety—a crucial step in preventing severe adverse events (AEs). Accordingly, the primary objective of the current study was to examine the AEs associated with risdiplam use based on real-world data obtained from the US Food and Drug Administration Adverse Event Reporting System (FAERS) database. More specifically, we examined incidents reported between the third quarter of 2020 and the second quarter of 2023. The imbalance of risdiplam-related AEs was evaluated by computing the reporting odds ratio. A total of 5,406,334 reports were thoroughly reviewed. By removing duplicate reports, we identified 1588 reports in which risdiplam was the main suspected drug whose use was accompanied by 3470 associated AEs. Among the included AEs, 703 were categorized as serious and 885 as non-serious. Risdiplam use induced AEs across 18 organ systems, resulting in 130 positive signals. Notably, we detected new AE signals, including cardiac arrest, nephrolithiasis, tachycardia, loss of libido, and elevated hepatic enzyme activities; however, no ophthalmologic toxicity was reported. Although these new adverse reaction signals associated with risdiplam have been defined, long-term clinical studies are needed to confirm these findings. Nevertheless, our findings provide a valuable reference for improving the clinical management of SMA.
README: Data on risdiplam-related adverse events
https://doi.org/10.5061/dryad.573n5tbfv
We examined incidents reported between the third quarter of 2020 and the second quarter of 2023. The imbalance of risdiplam-related AEs was evaluated by computing the reporting odds ratio. A total of 5,406,334 reports were thoroughly reviewed. By removing duplicate reports, we identified 1588 reports in which risdiplam was the main suspected drug whose use was accompanied by 3470 associated AEs. Among the included AEs, 703 were categorized as serious and 885 as non-serious. Risdiplam use induced AEs across 18 organ systems, resulting in 130 positive signals.
Description of the data and file structure
Table 1 counts the demographic information related to risdiplam;
List of Abbreviations: DEMO, patient demographics; DRUG, administration drug details; REAC, records of adverse events.
Table 2 summarizes the RORs and confidence intervals of the adverse event signals; Adverse events with the highest number of occurrences and the highest signal strength can be observed.
List of Abbreviations: Reporting odds ratio (ROR), confidence interval (CI), preferred term (PT)
Table 3 summarizes the number of adverse event signals ,and Contains the number of PT signals and the composition ratio of adverse events at each organ level.
Tables 4, 5, and 6 count the RORs and confidence intervals of the adverse event signals stratified into subgroups according to sex, age, and country of reporting, respectively.
Importantly, a portion of the RORs and confidence intervals corresponding to adverse events inside Tables 2-4 are blank, which is due to the fact that the denominator is zero in the formula and cannot be calculated.
Sharing/Access information
Data was derived from the following sources:
Methods
The data used in this study were obtained from the publicly available FAERS database. The FAERS data files contained seven datasets—patient demographics and administration (DEMO), drug details (DRUG), records of AEs (REAC), patient outcomes (OUTC), sources of reports (RPSR), start and end dates of therapy for the reported drugs (THER), and indications for drug usage (INDI). To conduct this study, we extracted all data in the ASCII format, encompassing the timeframe from Q3 of 2020 to Q2 of 2023. Subsequently, we imported the data into the SAS 9.4 software for cleaning and analysis.