Real-World Effectiveness and Safety of Cladribine in Multiple Sclerosis: Longitudinal Data From the Nationwide Registry in Argentina.

IF 0.8 4区医学 Q4 CLINICAL NEUROLOGY

Clinical Neuropharmacology Pub Date : 2024-07-01 Epub Date: 2024-05-22 DOI:10.1097/WNF.0000000000000598

Juan Ignacio Rojas, Ricardo Alonso, Geraldine Luetic, Liliana Patrucco, Magdalena Casas, Berenice Silva, Jimena Miguez, Norma Deri, Carlos Vrech, Susana Liwacki, Raúl Piedrabuena, Emanuel Silva, Verónica Tkachuk, Marcos Burgos, Dario Tavolini, Gisela Zanga, Amelia Alvez Pinheiro, Javier Hryb, Felisa Leguizamon, Eduardo Knorre, Pablo A Lopez, Alejandra Martinez, Adriana Carrá, Marina Alonso Serena, Edgardo Cristiano, Jorge Correale, Orlando Garcea, Nora Fernandez Liguori, Edgar Carnero Contentti

{"title":"Real-World Effectiveness and Safety of Cladribine in Multiple Sclerosis: Longitudinal Data From the Nationwide Registry in Argentina.","authors":"Juan Ignacio Rojas, Ricardo Alonso, Geraldine Luetic, Liliana Patrucco, Magdalena Casas, Berenice Silva, Jimena Miguez, Norma Deri, Carlos Vrech, Susana Liwacki, Raúl Piedrabuena, Emanuel Silva, Verónica Tkachuk, Marcos Burgos, Dario Tavolini, Gisela Zanga, Amelia Alvez Pinheiro, Javier Hryb, Felisa Leguizamon, Eduardo Knorre, Pablo A Lopez, Alejandra Martinez, Adriana Carrá, Marina Alonso Serena, Edgardo Cristiano, Jorge Correale, Orlando Garcea, Nora Fernandez Liguori, Edgar Carnero Contentti","doi":"10.1097/WNF.0000000000000598","DOIUrl":null,"url":null,"abstract":"Objective: The aim was to evaluate patient profiles, effectiveness and safety of cladribine (CLAD) in patients with relapsing-remitting multiple sclerosis in Argentina.Methods: This was a substudy included in RelevarEM (MS and neuromyelitis optica registry in Argentina, NCT03375177). Patients with MS who received CLAD tablets and were followed up for at least 24 months were included. Clinical evaluations every 3 months collect information about: a) clinical relapses; b) progression of physical disability, evaluated through Expanded Disability Status Scale, and c) new lesions found in the magnetic resonance imaging. Lymphopenia was evaluated during the follow-up and defined as grade 1: absolute lymphocyte count (ALC) 800-999/μL; grade 2: ALC 500-799/μL; grade 3: ALC 200-499/μL and grade 4: ALC <200/μL.Results: A total of 240 patients were included from 19 centers from Argentina. The mean annualized relapse rate during the 12-month pre-CLAD initiation was 1.19 ± 0.56 versus 0.22 ± 0.18 at month 12 and 0.19 ± 0.15 at month 24 ( P < 0.001). A total of 142 (59.2%) fulfilled the criteria of disease activity during the 12 months before treatment initiation, whereas 27 (11.3%) fulfilled it at month 12 and 38 (15.8%) at month 24, P < 0.001. Regarding no evidence of disease activity (NEDA), 202 (84.2%) patients achieved NEDA status at month 12 and 185 (77%) at month 24. The most frequent incidence density of lymphopenia for course 2 observed was also for grade 1, 6.1 (95% confidence interval [CI] = 5.5-7.1). The overall incidence density of lymphopenia grade 4 was 0.1 (95% CI = 0.06-0.19).Conclusion: This information will help when choosing the best treatment option for Argentinean patients.","PeriodicalId":10449,"journal":{"name":"Clinical Neuropharmacology","volume":"47 4","pages":"120-127"},"PeriodicalIF":0.8000,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11287052/pdf/","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Clinical Neuropharmacology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1097/WNF.0000000000000598","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/5/22 0:00:00","PubModel":"Epub","JCR":"Q4","JCRName":"CLINICAL NEUROLOGY","Score":null,"Total":0}

引用次数: 0

Abstract

Objective: The aim was to evaluate patient profiles, effectiveness and safety of cladribine (CLAD) in patients with relapsing-remitting multiple sclerosis in Argentina.

Methods: This was a substudy included in RelevarEM (MS and neuromyelitis optica registry in Argentina, NCT03375177). Patients with MS who received CLAD tablets and were followed up for at least 24 months were included. Clinical evaluations every 3 months collect information about: a) clinical relapses; b) progression of physical disability, evaluated through Expanded Disability Status Scale, and c) new lesions found in the magnetic resonance imaging. Lymphopenia was evaluated during the follow-up and defined as grade 1: absolute lymphocyte count (ALC) 800-999/μL; grade 2: ALC 500-799/μL; grade 3: ALC 200-499/μL and grade 4: ALC <200/μL.

Results: A total of 240 patients were included from 19 centers from Argentina. The mean annualized relapse rate during the 12-month pre-CLAD initiation was 1.19 ± 0.56 versus 0.22 ± 0.18 at month 12 and 0.19 ± 0.15 at month 24 ( P < 0.001). A total of 142 (59.2%) fulfilled the criteria of disease activity during the 12 months before treatment initiation, whereas 27 (11.3%) fulfilled it at month 12 and 38 (15.8%) at month 24, P < 0.001. Regarding no evidence of disease activity (NEDA), 202 (84.2%) patients achieved NEDA status at month 12 and 185 (77%) at month 24. The most frequent incidence density of lymphopenia for course 2 observed was also for grade 1, 6.1 (95% confidence interval [CI] = 5.5-7.1). The overall incidence density of lymphopenia grade 4 was 0.1 (95% CI = 0.06-0.19).

Conclusion: This information will help when choosing the best treatment option for Argentinean patients.

查看原文本刊更多论文

克拉利宾治疗多发性硬化症的实际效果和安全性：阿根廷全国登记处的纵向数据。

目的目的是评估阿根廷复发缓解型多发性硬化症患者的概况、克拉德里滨（CLAD）的有效性和安全性：这是 RelevarEM（阿根廷多发性硬化症和视神经脊髓炎登记，NCT03375177）的一项子研究。研究对象包括接受 CLAD 药片治疗并随访至少 24 个月的多发性硬化症患者。每 3 个月进行一次临床评估，收集以下信息：a) 临床复发；b) 身体残疾进展（通过残疾状况扩展量表进行评估）；c) 磁共振成像中发现的新病灶。随访期间对淋巴细胞减少症进行了评估，并将其定义为 1 级：绝对淋巴细胞计数（ALC）800-999/μL；2 级：ALC 500-799/μL；3 级：ALC 200-499/μL；4 级：ALC 结果：阿根廷的 19 个中心共纳入 240 名患者。CLAD 前 12 个月的平均年复发率为 1.19 ± 0.56，而第 12 个月为 0.22 ± 0.18，第 24 个月为 0.19 ± 0.15（P < 0.001）。在开始治疗前的 12 个月中，共有 142 人（59.2%）符合疾病活动标准，而在第 12 个月和第 24 个月分别有 27 人（11.3%）和 38 人（15.8%）符合标准，P < 0.001。关于无疾病活动证据（NEDA），202 名（84.2%）患者在第 12 个月时达到了 NEDA 状态，185 名（77%）患者在第 24 个月时达到了 NEDA 状态。在第 2 个疗程中，淋巴细胞减少症最常见的发病密度也是 1 级，为 6.1（95% 置信区间 [CI] = 5.5-7.1）。淋巴细胞减少症 4 级的总体发病密度为 0.1（95% 置信区间 = 0.06-0.19）：这些信息将有助于阿根廷患者选择最佳治疗方案。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

求助全文

约1分钟内获得全文求助全文

来源期刊

Clinical Neuropharmacology 医学-临床神经学

CiteScore

1.20

自引率

10.00%

发文量

审稿时长

6-12 weeks

期刊介绍： Clinical Neuropharmacology is a peer-reviewed journal devoted to the pharmacology of the nervous system in its broadest sense. Coverage ranges from such basic aspects as mechanisms of action, structure-activity relationships, and drug metabolism and pharmacokinetics, to practical clinical problems such as drug interactions, drug toxicity, and therapy for specific syndromes and symptoms. The journal publishes original articles and brief reports, invited and submitted reviews, and letters to the editor. A regular feature is the Patient Management Series: in-depth case presentations with clinical questions and answers.