10th Military Hospital Bydgoszcz, Powstańców Warszawy 5, 85-001 Bydgoszcz, Poland.
Neurol Neurochir Pol. 2020;54(1):54-61. doi: 10.5603/PJNNS.a2019.0069. Epub 2020 Jan 10.
To compare the clinical and neuroradiological efficacy of mitoxantrone (MTX) in various forms of multiple sclerosis (MS), to ascertain whether there is a new place for the drug in the treatment regimen of the disease, as well as to determine its safety profile.
Due to the increasing availability of new immunomodulatory therapies in multiple sclerosis (MS), there is a strong need to re-identify clinical variants and stages of the disease in which mitoxantrone (MTX) can be the most effective form of treatment.
This was a retrospective, non-randomised, observational study evaluating a cohort of 100 MS patients (36 relapsing-remitting - RRMS, 36 secondary progressive - SPMS, and 28 primary progressive - PPMS). 59% of the RRMS patients had discontinued immunomodulatory therapies (IMTs) within the two years preceding MTX infusion. Patients' disability levels, based on the Kurtzke Expanded Disability Status Scale (EDSS) as well as haematological and echocardiographic parameters, were assessed at baseline and before every infusion. Magnetic resonance imaging (MRI) were performed at entry and after termination of treatment.
We observed a decrease in the median EDSS score from 4.0 at baseline to 3.5 at the end of MTX infusion in the RRMS subgroup, an increase from 4.5 to 5.25 in the PPMS subgroup, and a stable value of 5 points in the SPMS subgroup (p < 0.0001). During the treatment period, 97% of patients with initial RRMS were free of exacerbations. The baseline EDSS in the RRMS subgroup, as well as the ineffectiveness of previous IMTs, suggested the beginning of conversion to SPMS. We found an 86% decrease in the proportion of patients with gadolinium-enhancing lesions on MRI after MTX infusions. There were no lifethreatening adverse events of MTX during the period of evaluation.
Mitoxantrone can be considered as a valuable therapeutic option for patients who are on the borderline of RRMS and SPMS.
比较米托蒽醌（MTX）在多种形式多发性硬化症（MS）中的临床和神经放射学疗效，确定该药物在疾病治疗方案中是否有新的地位，并确定其安全性概况。
由于多发性硬化症（MS）中新型免疫调节疗法的可用性不断增加，因此强烈需要重新确定疾病的临床变异和阶段，在这些阶段中，米托蒽醌（MTX）可能是最有效的治疗形式。
这是一项回顾性、非随机、观察性研究，评估了 100 名 MS 患者（36 名复发缓解型-MS，36 名继发进展型-MS，28 名原发进展型-MS）的队列。在 MTX 输注前的两年内，59%的 RRMS 患者已停止免疫调节治疗（IMTs）。根据 Kurtzke 扩展残疾状况量表（EDSS）以及血液学和超声心动图参数评估患者的残疾程度，在基线和每次输注前进行评估。在入组时和治疗结束时进行磁共振成像（MRI）检查。
我们观察到 RRMS 亚组的中位 EDSS 评分从基线时的 4.0 降至 MTX 输注结束时的 3.5，PPMS 亚组从 4.5 增至 5.25，SPMS 亚组稳定在 5 分（p<0.0001）。在治疗期间，97%的初始 RRMS 患者无恶化。RRMS 亚组的基线 EDSS 以及先前 IMTs 的无效性提示开始向 SPMS 转化。我们发现，MTX 输注后 MRI 上钆增强病变的患者比例下降了 86%。在评估期间，没有 MTX 导致的危及生命的不良事件。
米托蒽醌可被视为 RRMS 和 SPMS 边缘患者的有价值的治疗选择。
