METHODS: A 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.
RESULTS: We 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.
CONCLUSIONS: Most ALS patients who start riluzole adhere to therapy during the first year. Patients who discontinue therapy early show greater fragility and mortality.
方法:使用Latium的管理数据进行了一项回顾性队列研究,托斯卡纳,和翁布里亚.我们在2014年至2019年之间确定了新诊断为ALS的受试者,在诊断后180天内首次分配了利鲁唑。我们考虑了两年的回顾期,用于患者的表征,我们从第一次分配利鲁唑的日期开始跟踪他们1年。我们计算了12个月的坚持措施,通过修改版本的药物持有率,用确定的每日剂量估计药物覆盖率。使用三步方法识别粘附轨迹:(1)统计度量的计算;(2)主成分分析;(3)聚类分析。描述基线和随访期间的患者特征,并在确定的依从组之间进行比较。
结果:我们纳入了264名ALS患者作为Latium利鲁唑的新使用者,344在托斯卡纳,和63在翁布里亚。我们观察到男性的频率更高(56.2%),平均年龄为67.4岁(标准差,SD,10.4)在总人口中。我们在所有地区都确定了两个集群:还有一个,包括粘附患者(60%,74%,88%,分别),另一个包括停止治疗的患者(40%,26%,12%,分别)。在托斯卡纳,停止利鲁唑的患者更频繁地死亡(28.6%vs.15.4%,p值<0.01)。此外,低用户的中枢神经系统疾病发生率较高(69.0%vs.52.5%,p值0.01),以及更多地使用非药物治疗(有创通气和气管造口术的p值≤0.01)。我们没有观察到拉齐奥的任何差异,而在翁布里亚,我们观察到在低信奉者中,治疗痴呆相关精神问题的药物使用率较高(57.1%vs.7.8%,分别,p值<0.01),虽然数字很小。
结论:大多数开始使用利鲁唑的ALS患者在第一年坚持治疗。早期停止治疗的患者表现出更大的脆弱性和死亡率。