神经脊髓炎视网膜病变早期利妥昔单抗与升级疗法的对比:成本与生活质量分析。

IF 2.9 3区 医学 Q2 CLINICAL NEUROLOGY
Guilherme Diogo Silva , Samira Luísa Apóstolos-Pereira , Mateus Boaventura , Renata Barbosa Paolilo , Aline Matos , Milena Sales Pitombeira , Tarso Adoni , Douglas K Sato , Dagoberto Callegaro
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引用次数: 0

摘要

简介:利妥昔单抗是一种抗 CD20 单克隆抗体,在减少神经脊髓炎视网膜频谱疾病(NMOSD)患者的疾病复发和因复发导致的残疾累积方面显示出了疗效。然而,与口服免疫抑制剂相比,它的成本较高,这就引起了人们对其成本效益的质疑,尤其是在中低收入国家。本研究旨在比较 NMOSD 患者早期利妥昔单抗治疗与升级治疗的成本效益:我们对巴西圣保罗一家医院在 2015 年至 2019 年间发病头五年接受利妥昔单抗治疗的 NMOSD 患者进行了一项回顾性研究。早期治疗组包括接受利妥昔单抗一线治疗的NMOSD患者。升级治疗组包括在口服免疫抑制剂(主要是硫唑嘌呤)期间出现疾病活动后接受利妥昔单抗治疗的患者。该研究采用了基于扩展残疾状态评分(EDSS)转换的经济模型来评估成本效益。成本和效用数据来自以往的研究,并对不同的支付意愿(WTP)阈值和从口服免疫抑制剂升级到利妥昔单抗的患者比例进行了敏感性分析:共纳入 30 名 NMOSD 患者。与基线相比,早期治疗组中达到最高 EDSS 状态(6.5 或以上)的患者比例在五年内有所下降。相比之下,升级疗法组的这一比例在同期有所上升。在我们的主要分析中,支付意愿(WTP)为20-80,000欧元的患者获得了成本效益,而在我们的敏感性分析中,这一成本效益得以维持:结论:利妥昔单抗的早期治疗有可能降低医疗成本并提高 NMOSD 患者的生活质量,因此支持尽早使用利妥昔单抗进行预防性治疗。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Early rituximab versus escalating therapy in neuromyelitis optica: A cost and quality of life analysis

Introduction

Rituximab, an anti-CD20 monoclonal antibody, has shown effectiveness in reducing disease relapses and disability accrual through relapses in patients with neuromyelitis optica spectrum disorders (NMOSD). However, its higher cost compared to oral immunosuppressants raises questions about its cost-effectiveness, particularly in low and middle-income countries. This study aimed to compare the cost-effectiveness of early rituximab treatment versus escalation treatment in NMOSD patients.

Methods

We conducted a retrospective study of NMOSD patients treated with rituximab in the first five years of disease at a hospital in São Paulo, Brazil, from 2015 to 2019. The Early Group consisted of NMOSD patients who received rituximab as a first-line treatment. The Escalating Therapy Group included patients who were prescribed rituximab after experiencing disease activity while on oral immunosuppressants, primarily azathioprine. An economic model based on Expanded Disability Status Score (EDSS) transitions was used to assess cost-effectiveness. Cost and utility data were derived from previous studies, and sensitivity analyses for different willingness to pay (WTP) thresholds and percentage of patients upscaling from oral immunossupressants to rituximab were performed.

Results

: Thirty NMOSD patients were included. In the Early Group, the proportion of patients reaching the highest EDSS states (6.5 or more) decreased over five years compared to baseline. In contrast, the Escalating Therapy Group experienced an increase in this proportion over the same period. Cost-effectiveness was achieved for willingness to pay (WTP) of €20–80,000 in our main analysis, sustained in our sensitivity analysis.

Conclusion

: Early treatment with rituximab has the potential to lower healthcare costs and enhance quality of life for NMOSD patients, supporting its early prescription for preventive treatment.
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来源期刊
CiteScore
5.80
自引率
20.00%
发文量
814
审稿时长
66 days
期刊介绍: Multiple Sclerosis is an area of ever expanding research and escalating publications. Multiple Sclerosis and Related Disorders is a wide ranging international journal supported by key researchers from all neuroscience domains that focus on MS and associated disease of the central nervous system. The primary aim of this new journal is the rapid publication of high quality original research in the field. Important secondary aims will be timely updates and editorials on important scientific and clinical care advances, controversies in the field, and invited opinion articles from current thought leaders on topical issues. One section of the journal will focus on teaching, written to enhance the practice of community and academic neurologists involved in the care of MS patients. Summaries of key articles written for a lay audience will be provided as an on-line resource. A team of four chief editors is supported by leading section editors who will commission and appraise original and review articles concerning: clinical neurology, neuroimaging, neuropathology, neuroepidemiology, therapeutics, genetics / transcriptomics, experimental models, neuroimmunology, biomarkers, neuropsychology, neurorehabilitation, measurement scales, teaching, neuroethics and lay communication.
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