Rosalem Rafael Augusto, Spricigo Mariana Gondim Peixoto, de Oliveira Mateus Boaventura, Adoni Tarso, Apóstolos-Pereira Samira Luísa, Callegaro Dagoberto, Silva Guilherme Diogo
Hospital das Clinicas HCFMUSP, Faculdade de Medicina, Universidade de Sao Paulo, Sao Paulo, SP, BR.
Hospital das Clinicas HCFMUSP, Faculdade de Medicina, Universidade de Sao Paulo, Sao Paulo, SP, BR.
Mult Scler Relat Disord. 2025 Jul 20;103:106627. doi: 10.1016/j.msard.2025.106627.
High efficacy therapy can prevent disability in multiple sclerosis patients, but timing and adherence are imperative. This study assessed the impact of delayed access and non-adherence in the treatment of multiple sclerosis using natalizumab in a tertiary Brazilian center.
We conducted a retrospective, single-center observational study using medical records of multiple sclerosis patients treated at a public tertiary center in São Paulo, Brazil. Data collected included demographics, disease course, treatment history, relapses, clinical and radiological activity, adherence, adverse events, and disability progression. A sample size of 88 was estimated to detect differences in the frequency of Expanded Disability Status Scale (EDSS) ≥ 6 between early and late natalizumab treatment groups, defined by a 48-month cutoff from symptom onset to natalizumab initiation. Statistical analyses included Mann-Whitney, Fisher's, and Chi-squared tests.
A total of 88 patients were included in this study. The majority of patients (n = 69, 78 %) initiated natalizumab late in the disease course. The late-treatment group had a higher frequency of EDSS ≥ 6 at the last follow-up compared to the early-treatment group (32 % vs. 11 %, p = 0.024). One-fourth of patients were non-adherent, with a threefold higher risk of experiencing a relapse during treatment (55 % vs. 17 %, p < 0.001). The primary reason for natalizumab discontinuation was the perceived risk of progressive multifocal leukoencephalopathy (72 %), though no cases were reported.
Enhancing early access and adherence to natalizumab is essential to maximize its real-world benefits for multiple sclerosis patients.
高效疗法可预防多发性硬化症患者出现残疾，但治疗时机和依从性至关重要。本研究在巴西一家三级医疗中心评估了使用那他珠单抗治疗多发性硬化症时延迟治疗和不依从治疗的影响。
我们进行了一项回顾性单中心观察性研究，使用巴西圣保罗一家公立三级医疗中心治疗的多发性硬化症患者的病历。收集的数据包括人口统计学信息、疾病病程、治疗史、复发情况、临床和影像学活动、依从性、不良事件以及残疾进展。估计样本量为88例，以检测早期和晚期那他珠单抗治疗组之间扩展残疾状态量表（EDSS）≥6的频率差异，早期和晚期的定义为从症状出现到开始使用那他珠单抗的时间间隔是否超过48个月。统计分析包括曼-惠特尼检验、费舍尔检验和卡方检验。
本研究共纳入88例患者。大多数患者（n = 69，78%）在疾病病程后期开始使用那他珠单抗。与早期治疗组相比，晚期治疗组在最后一次随访时EDSS≥6的频率更高（32%对11%，p = 0.024）。四分之一的患者不依从治疗，治疗期间复发风险高三倍（55%对17%，p < 0.001）。那他珠单抗停药的主要原因是认为有进行性多灶性白质脑病的风险（72%），尽管未报告病例。
加强早期使用那他珠单抗并提高依从性对于使多发性硬化症患者在现实世界中最大程度获益至关重要。
