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在接受利鲁唑治疗的 MND 患者队列中,临床和人口统计学特征。片剂和口服混悬剂之间的差异。

Clinical and demographical characteristics in a cohort of MND patients treated with riluzole. Differences between tablets and oral suspension.

机构信息

Functional Motor Neuron Unit (UFMN), Department of Neurology, Bellvitge University Hospital, L'Hospitalet de Llobregat, Barcelona, Spain.

IDIBELL (Institut d'Investigació Biomèdica de Bellvitge), L'Hospitalet de Llobregat, Barcelona, Spain.

出版信息

Amyotroph Lateral Scler Frontotemporal Degener. 2023 Aug;24(5-6):369-382. doi: 10.1080/21678421.2023.2192247. Epub 2023 Apr 3.

Abstract

To describe the clinical and demographic characteristics of patients with MND treated with riluzole by comparing two dosage forms (oral suspension and tablets), as well as the impact on survival in patients with and without dysphagia according to the form of dosage. Retrospective and prospective cohort of patients diagnosed with MND at the multidisciplinary functional unit of Motor Neuron Disease in our center in the period between 1 of January 2011 and 31 of December 2020 ( = 742). A descriptive analysis (univariate and bivariate) was carried out and survival curves were estimated. During the follow-up period, 402 males (54.18%) and 340 females (45.82%) were diagnosed with MND. Of these patients, 632 (97.23%) were being treated with 100mg riluzole: 282 (54.55%) patients took this in tablet form and 235 (45.45%) oral suspension. Riluzole in tablet form is taken more frequently by men than women, in younger age ranges, and mostly without dysphagia (78.31%). Also, it is the predominant dosage form for classic spinal ALS and respiratory phenotypes. Dosages via oral suspension are taken by patients in the older age ranges (over 64.8 years), mostly with dysphagia (53.67%) and more frequently with bulbar phenotypes such as classic bulbar ALS and PBP. Because of this, patients using oral suspension (most of them with dysphagia) had a poorer survival rate (at 90% CI) than patients using tablets (most of them without dysphagia). The most appropriate dosage form should be given according to the patient's needs at each stage of the disease and, furthermore, oral suspension could improve adherence to treatment because it avoids having to change from one form (tablet) to the other (suspension) when swallowing disorders appear.

摘要

描述在我们中心的运动神经元疾病多学科功能单位诊断为 MND 的患者中,使用两种剂型(口服混悬液和片剂)的利鲁唑治疗的患者的临床和人口统计学特征,以及根据剂型评估吞咽困难患者和无吞咽困难患者的生存影响。2011 年 1 月 1 日至 2020 年 12 月 31 日期间在我们中心的运动神经元疾病多学科功能单元诊断为 MND 的患者的回顾性和前瞻性队列( = 742)。进行了描述性分析(单变量和双变量),并估计了生存曲线。在随访期间,诊断出 402 名男性(54.18%)和 340 名女性(45.82%)患有 MND。其中 632 名患者(97.23%)接受了 100mg 利鲁唑治疗:282 名患者(54.55%)以片剂形式服用,235 名患者(45.45%)以口服混悬液形式服用。与女性相比,男性更频繁地服用片剂形式的利鲁唑,年龄更小,且大多数无吞咽困难(78.31%)。此外,它也是经典脊髓性 ALS 和呼吸表型的主要剂型。口服混悬液剂型用于年龄较大的患者(64.8 岁以上),大多数有吞咽困难(53.67%),且更频繁地出现延髓表型,如经典延髓性 ALS 和 PBP。因此,使用口服混悬液的患者(大多数有吞咽困难)的生存率(90%CI)比使用片剂的患者(大多数无吞咽困难)差。应根据患者在疾病的每个阶段的需求给予最合适的剂型,此外,当出现吞咽障碍时,口服混悬液可以改善治疗依从性,因为它避免了从一种剂型(片剂)变为另一种剂型(混悬液)。

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