Disease-modifying treatment

疾病改善治疗
  • 文章类型: Journal Article
    多发性硬化症(MS)是一种自身免疫介导的中枢神经系统退行性疾病,以炎症性脱髓鞘为特征。它主要存在于育龄妇女中,使妊娠成为MS患者和临床医生的重要关注点。为了帮助这些患者实现怀孕的愿望,在怀孕的所有阶段减少MS复发,阻止MS的进展,减轻MS治疗对妊娠过程和结局的影响,以及对妊娠与MS之间关系的透彻了解,以及MS患者在怀孕每个阶段的具体管理和相关药物的应用,是必不可少的。本文提供了MS女性妊娠相关问题的最新信息,包括怀孕每个阶段的一般管理建议。
    Multiple sclerosis (MS) is an autoimmune-mediated degenerative disease of the central nervous system, characterized by inflammatory demyelination. It is primarily found in women of childbearing age, making pregnancy a significant concern for both patients with MS and clinicians. To assist these patients in achieving their desire for pregnancy, reducing MS relapses during all stages of pregnancy, preventing the progression of MS, mitigating the impact of MS treatment on the course and outcome of pregnancy, and a thorough understanding of the relationship between pregnancy and MS, as well as specific management and the application of relevant medications for MS patients at each stage of pregnancy, are essential. This article provides an update on pregnancy-related issues in women with MS, including the general recommendations for management at each stage of pregnancy.
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  • 文章类型: Journal Article
    特立氟胺是中国批准用于治疗复发性多发性硬化症的一线口服免疫调节剂。
    比较特立氟胺和无疾病改善疗法(DMT)治疗(第一年)在多中心真实世界中国多发性硬化症患者中的治疗结果。
    回顾性研究。
    本研究在中国不同地理区域的五家三级医院进行。我们收集了2017年1月1日至2021年8月31日期间接受特立氟胺治疗和未接受DMT治疗(第一年)的患者的临床数据。描述了特立氟胺的有效性。研究了影响特立氟胺有效性的潜在因素。
    总共包括372例接受特立氟胺治疗的患者和148例未接受DMT治疗的患者。共有292例患者接受了至少6个月的特立氟胺治疗,描述为稳定的特立氟胺队列。稳定的特立氟胺组的年复发率明显低于无DMT治疗组(0.23±0.47对0.87±0.67,p<0.001)。稳定特立氟胺队列的平均扩展残疾状态量表(EDSS)评分(1.77±1.62)与非DMT治疗队列(2.09±2.00)略有不同。以前的年化复发率为1,以前的EDSS评分为2,疾病持续时间长5年与更好的临床有效性相关。
    特立氟胺在中国多发性硬化症患者中具有较低的复发率和较少的残疾积累。
    UNASSIGNED: Teriflunomide is a first-line oral immunomodulatory agent approved in China for the treatment of relapsing multiple sclerosis.
    UNASSIGNED: To compare the treatment outcomes of teriflunomide and no disease-modifying therapy (DMT) treatment (in first year) in multi-center real-world Chinese multiple sclerosis patients.
    UNASSIGNED: Retrospective study.
    UNASSIGNED: This study was conducted in five tertiary hospitals in different geographical regions of China. We collected clinical data of patients treated with teriflunomide and no DMT treatment (in first year) between 1 January 2017 and 31 August 2021. The effectiveness of teriflunomide was described. Potential factors influencing the effectiveness of teriflunomide were investigated.
    UNASSIGNED: A total of 372 patients treated with teriflunomide and 148 no DMT treatment patients were included. A total of 292 patients were treated with teriflunomide for at least 6 months, described as a stable teriflunomide cohort. The annualized relapse rate was significantly lower in the stable teriflunomide cohort than in the no DMT treatment cohort (0.23 ± 0.47 versus 0.87 ± 0.67, p < 0.001). The mean Expanded Disability Status Scale (EDSS) score of the stable teriflunomide cohort (1.77 ± 1.62) was slightly different from that of the no DMT treatment cohort (2.09 ± 2.00). A previous annualized relapse rate of ⩾1, a previous EDSS score of ⩾2, and a long disease duration of ⩾5 years were associated with better clinical effectiveness.
    UNASSIGNED: Teriflunomide is associated with a lower relapse rate and less disability accumulation in Chinese patients with multiple sclerosis.
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  • 文章类型: Journal Article
    背景:冠状病毒病(COVID-19)大流行给慢性病患者的护理带来了挑战。我们确定了中国Duchenne型肌营养不良(DMD)患者在大流行期间面临的挑战。
    方法:在2021年3月27日至6月30日之间进行了在线横断面调查。
    结果:在2105份有效问卷答复中,2,056名患者来自非封锁地区。在这些地区,外部日常活动减少42.8%,康复服务使用量减少49.4%,39.7%推迟定期随访,和40.8%报告加速运动功能下降。来自封锁区的患者的这些数字普遍增加-日常活动外减少了67.3%,康复服务使用量减少44.9%,79.6%推迟定期随访,55.1%的人报告运动功能加速下降。步行损失最常见于2020年之前的9月和3月;然而,这一趋势在2020年没有出现。关于欧洲和美国的疾病改善药物的知情价格,86.7%的人最多可以负担得起二十分之一的价格,8.0%的人可以负担得起十分之一的价格,只有0.6%的病人能负担得起全价。
    结论:在中国实施DMD标准化护理与全球实践一致,COVID-19大流行影响了全世界慢性病患者的护理,特别是在封锁地区。远程医疗是为此类患者提供医疗保健的有效模式。医护人员应协助患者并建立更强大的慢性病管理系统。政府实体和非政府实体之间的合作可以解决中国和其他发展中国家改善疾病药物的成本问题。
    BACKGROUND: The coronavirus disease (COVID-19) pandemic has presented challenges in the care of patients with chronic diseases. We identified the challenges faced by Chinese patients with Duchenne muscular dystrophy (DMD) during the pandemic.
    METHODS: An online cross-sectional survey was conducted between March 27 and June 30, 2021.
    RESULTS: Of the 2105 valid questionnaire responses, 2,056 patients were from non-lockdown areas. In these areas, 42.8% reduced outside daily activities, 49.4% reduced rehabilitation service use, 39.7% postponed regular follow-ups, and 40.8% reported accelerated motor function decline. These figures generally increased for patients from lockdown areas-67.3% reduced outside daily activities, 44.9% reduced rehabilitation service use, 79.6% postponed regular follow-ups, and 55.1% reported accelerated motor function decline. Ambulation loss was most commonly reported in September and March before 2020; however, this trend was absent in 2020. Regarding the informed prices of disease-modifying drugs in Europe and the United States, 86.7% could afford a maximum of one-twentieth of the prices, 8.0% could afford one-tenth of the prices, and only 0.6% of the patients could afford the full prices.
    CONCLUSIONS: Implementation of standardized care for DMD in China is consistent with global practices, and the COVID-19 pandemic has affected the care of patients with chronic diseases worldwide, particularly in lockdown areas. Telemedicine is an effective model for providing healthcare to such patients. Healthcare workers should assist patients and establish more robust chronic disease management systems. Collaboration between governmental and non-governmental entities could address the cost of disease-modifying drugs in China and other developing countries.
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  • 文章类型: Journal Article
    目的:报告多发性硬化症(MS)患者皮下使用克拉屈滨(Litak®)的安全性和有效性。
    方法:向MS患者提供Litak®,无论病程如何。在第1周期间施用Litak®10mg持续3-4天。根据第4周的淋巴细胞计数,患者在第5周接受了另外的0-3次剂量。11个月后进行第二个疗程。随访包括不良事件,复发,扩展残疾状况量表(EDSS),9孔钉和定时25英尺步行测试,无疾病活动证据(NEDA),没有进展或活跃疾病的证据(新伙伴关系),MRI,脑脊液(CSF)神经丝轻链(NfL),和淋巴细胞计数。
    结果:总而言之,208名患者接受了至少一个疗程的治疗。基线年龄为44(17-72)岁,EDSS为0-8.5。克拉屈滨一般耐受性良好。一次心肌梗塞,一个乳腺癌,发生了三次严重的皮肤反应,没有长期后遗症。两名患者死亡(一名肺炎,一种脑炎)。3级淋巴细胞减少发生率为5%,4级淋巴细胞减少发生率为0.5%。在两个疗程后的基线和随访(BaFU)数据的116个pwMS中有94个,EDSS保持稳定或改善。18个月时,64%的复发MS和BaFU患者(n=39)患有NEDA。19个月时,62%的进展性MS和BaFU患者(n=13)有新伙伴关系。在n=13例患者中,基线时CSF-NfL升高,77%在12个月内恢复正常。
    结论:Litak®耐受性良好。复发性MS的有效性与克拉屈滨片相似,在进行性MS中令人鼓舞。我们的数据表明克拉屈滨在MS患者中可能是安全有效的,无论其疾病阶段如何。
    OBJECTIVE: To report on safety and effectiveness of subcutaneous cladribine (Litak®) in multiple sclerosis (MS) patients.
    METHODS: Litak® was offered to MS-patients irrespective of disease course. Litak® 10 mg was administered for 3-4 days during week 1. Based on lymphocyte count at week 4, patients received another 0-3 doses at week 5. A second course was administered 11 months later. Follow-up included adverse events, relapses, expanded disability status scale (EDSS), 9-hole-peg and Timed-25-foot-walking tests, no-evidence-of-disease-activity (NEDA), no-evidence-of-progression-or-active-disease (NEPAD), MRI, cerebrospinal fluid (CSF) neurofilament light chain (NfL), and lymphocyte counts.
    RESULTS: In all, 208 patients received at least one course of treatment. Age at baseline was 44 (17-72) years and EDSS 0-8.5. Cladribine was generally well tolerated. One myocardial infarction, one breast cancer, and three severe skin reactions occurred without long-term sequelae. Two patients died (one pneumonia, one encephalitis). Lymphopenia grade 3 occurred in 5% and grade 4 in 0.5%. In 94 out of 116 pwMS with baseline and follow-up (BaFU) data after two treatment courses, EDSS remained stable or improved. At 18 months, 64% of patients with relapsing MS and BaFU data (n = 39) had NEDA. At 19 months, 62% of patients with progressive MS and BaFU data (n = 13) had NEPAD. Of n = 13 patients whose CSF-NfL at baseline was elevated, 77% were normalised within 12 months.
    CONCLUSIONS: Litak® was well tolerated. Effectiveness in relapsing MS appeared similar to cladribine tablets and was encouraging in progressive MS. Our data suggest cladribine may be safe and effective in MS-patients irrespective of their disease stage.
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  • 文章类型: Journal Article
    Multiple sclerosis (MS) is an autoimmune chronic inflammatory disease of the central nervous system with a wide range of symptoms, like executive function defect, cognitive dysfunction, blurred vision, decreased sensation, spasticity, fatigue, and other symptoms. This neurological disease is characterized by the destruction of the blood-brain barrier, loss of myelin, and damage to neurons. It is the result of immune cells crossing the blood-brain barrier into the central nervous system and attacking self-antigens. Heretofore, many treatments proved that they can retard the progression of the disease even though there is no cure. Therefore, treatments aimed at improving patients\' quality of life and reducing adverse drug reactions and costs are essential. In this review, the treatment approaches to alleviate the progress of MS include the following: pharmacotherapy, antibody therapy, cell therapy, gene therapy, and surgery. The current treatment methods of MS are described in terms of the prevention of myelin shedding, the promotion of myelin regeneration, and the protection of neurons.
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  • 文章类型: Journal Article
    背景:相当数量的多发性硬化症(pwMS)患者生活在低收入和中等收入国家(LMIC),缺乏资源对获得有效的疾病改善治疗产生不利影响。
    目的:本评论的目的是为LMIC中的pwMS提出一种有用的具有成本效益的疾病修饰治疗方案,对pwMS和治疗医师具有潜在的高疗效和高便利性。观点:我们建议使用通用的2-氯-2'-脱氧腺苷(克拉屈滨),一种被许可用于治疗毛细胞白血病的小分子,作为解决这一重大股权失衡的解决方案。克拉屈滨已在II期和III期试验中被证明是一种非常有效的改善pwMS疾病的治疗方法,其不利影响与目前在高收入经济体中获得许可的任何DMT相当,在高收入经济体中,口服制剂最近已获得欧洲药品管理局的许可。
    结论:我们的观点考虑了我们在过去三年中收集的使用通用克拉屈滨治疗pwMS的经验。虽然在这里我们专注于MS,克拉屈滨在其他可能受益于其作用机制的条件下具有巨大的潜力。
    BACKGROUND: A considerable number of people with multiple sclerosis (pwMS) live in low- and middle-income countries (LMIC), where lack of resource adversely affects access to effective disease-modifying treatment.
    OBJECTIVE: The objective of this commentary is to propose a useful cost-effective disease-modifying treatment option for pwMS in LMIC with potential high efficacy and high convenience to the pwMS and treating physician.Viewpoint: We propose using generic 2-chloro-2\'-deoxyadenosine (cladribine), a small molecule licensed for treatment of people with hairy cell leukaemia, as a solution of this significant equity imbalance. Cladribine has been shown in phase II and III trials to be a highly effective disease-modifying treatment for pwMS, and its adverse effect profile is comparable with any DMT currently licensed in high-income economies where an oral preparation has recently been licensed by the European Medicines Agency.
    CONCLUSIONS: Our viewpoint takes into account experience we have gathered over the past three years in the use of generic cladribine to treat pwMS. Whilst here we focus on MS, there is significant potential for use of cladribine in other conditions that could benefit from its mechanism of action.
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  • 文章类型: Journal Article
    背景:多发性硬化症(MS)是一种慢性免疫介导的中枢神经系统脱髓鞘疾病,表现出性二态性,并可能受益于性别特异性治疗。探讨性别对MS患者免疫调节治疗效果的潜在影响,我们对已发表的研究进行了全面分析,这些研究检查了MS疾病改善治疗(DMT)效果的性别差异。
    方法:PubMed,科克伦图书馆,我们在WebofScience数据库中搜索涉及正在接受DMT的MS患者的临床研究。如果他们调查了DMT结果的性别差异,则纳入研究。
    结果:纳入了14项研究,11,425名参与者;其中11项是随机对照试验,和3个是队列研究。尽管这些研究偶尔显示接受DMT的MS患者的某些临床结局存在性别差异,亚组分析设计的局限性使得很难就性别效应的方向或程度得出结论。
    结论:迄今为止,对DMT的反应没有明显的基于性别的差异。需要更多的研究来更好地阐明性别差异对DMT影响的存在。
    BACKGROUND: Multiple sclerosis (MS) is a chronic immune mediated demyelinating disease of the central nervous system that exhibits sexual dimorphism and may benefit from sex-specific treatment. To investigate a potential influence of sex on immunomodulatory therapeutic effects in patients with MS, we performed a comprehensive analysis of published studies examining sex differences in the effects of disease-modifying treatments (DMTs) for MS.
    METHODS: PubMed, Cochrane Library, and Web of Science databases were searched for clinical studies involving patients with MS who were undergoing DMTs. Studies were included if they investigated sex differences in DMT outcomes.
    RESULTS: Fourteen studies with 11,425 participants were included; 11 of these studies were randomized controlled trials, and 3 were cohort studies. Although the studies did occasionally show sex-specific differences for some clinical outcomes in patients with MS who received DMTs, the limitation of subgroup analysis design made it difficult to draw conclusions on the direction or the extent of the sex-based effect.
    CONCLUSIONS: No clear sex-based differences in response to DMTs have been documented to date. More studies will be needed to better elucidate the presence of sex differences on the DMT effects.
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