在二次进展性多发性硬化症 (EXPAND) 中,西波尼莫德与安慰剂对比:双盲,随机,第三阶段研究
Ludwig Kappos1, Amit Bar-Or2, Bruce A C Cree3
1Neurologic Clinic and Policlinic, Departments of Medicine, Clinical Research, Biomedicine, and Biomedical Engineering, University Hospital, University of Basel, Basel, Switzerland.
Lancet (London, England)
|March 27, 2018
概括
在患有二次渐进性多发性硬化症 (SPMS) 的患者中,西波尼莫德显著降低了残疾进展. 这种选择性基酸盐 (S1P) 受体调节器为SPMS患者提供了新的治疗选择.
科学领域:
- 神经学
- 免疫学
- 药理学
背景情况:
- 二次渐进性多发性硬化症 (SPMS) 的特点是神经障碍不断恶化.
- 目前,没有任何治疗方法能够稳定地减缓SPMS的进展.
研究的目的:
- 评估选择性酸盐 (S1P) 受体调节剂siponimod在SPMS患者中减缓残疾进展的疗效和安全性.
主要方法:
- 这是一项双盲,随机,安慰剂控制的3期试验,涉及31个国家的1651名SPMS患者.
- 患者被分配接受口服西波尼莫德 (每日2毫克) 或安慰剂,长达3年.
- 主要终点是确认残疾进展的时间到3个月 (CDP).
主要成果:
- 与安慰剂相比,在3个月的CDP中,西波尼莫德治疗导致21%的相对风险降低 (危险比率为0. 79; p=0. 013).
- 使用西波尼莫德的不良反应更为频繁,包括淋巴缺血,肝酶升高和心肌梗塞,但严重事件和死亡事件没有差异.
- 初始剂量定位有效减轻心脏影响.
结论:
- 在SPMS中,西波尼莫德在减少残疾进展方面表现出显著的有效性.
- 这种药物具有有利的安全性,与其他S1P调节剂相比,使其成为SPMS的有前途的治疗选择.
相关概念视频
Blind Procedures
13.7K
Ideally, the people who observe and record the children’s behavior are unaware of who was assigned to the experimental or control group, in order to control for experimenter bias. Experimenter bias refers to the possibility that a researcher’s expectations might skew the results of the study. Remember, conducting an experiment requires a lot of planning, and the people involved in the research project have a vested interest in supporting their hypotheses. If the observers knew which...
13.7K
The Placebo Effect
6.9K
The placebo effect occurs when people's expectations or beliefs influence or determine their experience in a given situation. In other words, simply expecting something to happen can actually make it happen.
6.9K
Blinding
4.0K
Blinding is a commonly used method of not telling participants which treatment a subject is receiving. Blinding is a critical part of a randomized control trial or RCT. It reduces the bias that affects the results. In an RCT, blinding is used in the form of a placebo. A placebo effect occurs when untreated subjects falsely believe they have received the treatment and report improved symptoms. A placebo or a dummy treatment is administered to subjects to negate the bias caused by such an effect.
4.0K
Secondary Active Transport
138.2K
One example of how cells use the energy contained in electrochemical gradients is demonstrated by glucose transport into cells. The ion vital to this process is sodium (Na+), which is typically present in higher concentrations extracellularly than in the cytosol. Such a concentration difference is due, in part, to the action of an enzyme “pump” embedded in the cellular membrane that actively expels Na+ from a cell. Importantly, as this pump contributes to the high concentration of...
138.2K
Bioavailability Study Design: Single Versus Multiple Dose Studies
251
Bioavailability studies are essential for understanding how a drug is absorbed, distributed, metabolized, and excreted in the body. These studies assess the extent and rate at which the active pharmaceutical agent becomes available at the site of action. The design of bioavailability studies can involve single-dose or multiple-dose regimens, each with distinct advantages and limitations.Single-dose studies are the preferred approach due to their simplicity and reduced drug exposure for...
251
Secondary Healthcare System
2.1K
Secondary healthcare is offered by a specialist, generally in hospitals or clinics for patients referred by primary healthcare providers. It occurs when a person has an illness or injury that requires specific medical care. Secondary care is often referred to as acute care. Secondary care can range from uncomplicated care to repair a minor laceration or treat a strep throat infection to more complicated emergent care, such as treating a head injury sustained in an automobile accident. Whatever...
2.1K


