Effectiveness and Safety of Efgartigimod in the Acute Phase of Neuromyelitis Optica Spectrum Disorders-a Multicentric, Controlled, Retrospective, Real-Word Study
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- 入组人数
- 24
- 主要终点
- Change in Expanded Disability Status Scale (EDSS) score from baseline.
研究概览
简要总结
This study aims to retrospective investigate the safety and effectiveness of Efgartigimod in the acute phase of neuromyelitis optica spectrum disorders (NMOSD) patients.
详细描述
This is a multicentre, controlled, retrospective, real-world study which aims to compare the safety and effectiveness of Intravenous methylprednisolone (IVMP) with Efgartigimod injection add-on treatment with IVMP treatment in acute NMOSD patients. Twenty-four patients from 6 centres in China will be enrolled.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≥ 18 years with anti-AQP4-IgG seropositive NMOSD as defined by 2015 NMOSD diagnostic criteria by IPND (International Panel for NMO Diagnosis).
- •Patients in the acute phase of NMOSD (definition of acute phase: new neurological symptoms or aggravation of existing symptoms within 30 days before received treatment, lasting at least 24 hours without fever), who had poor response to IVMP and without having received second-line therapies such as plasma exchange or intravenous immunoglobulin consequencely (Poor response is defined as a reduction in EDSS score of: I. <1.0 from the baseline EDSS score when the baseline score was <=5.5 II. < 0.5 when the baseline EDSS score > 5.5).
- •Patients who were approved for Efgartigimod treatment would be enrolled in the exposed group.
- •Expanded disability status scale (EDSS) score ≤ 8 and ≥ 2.5 before treatment.
- •Patients have given their written informed consent.
排除标准
- •Lactating and pregnant females before treatment.
- •Participated in other interventional studies within 30 days before treatment.
- •Received plasma exchange, immunoadsorption, or intravenous immunoglobulin (IVIG） therapy within 1 month before treatment.
- •History of malignancies.
- •Combined with severe mental disorders and other conditions that unable to cooperate with follow-up.
- •After being evaluated by experts, patients with active hepatitis, active tuberculosis, or other special conditions which were ineligible to participate in this study.
研究组 & 干预措施
Exposed group
Intravenous methylprednisolone (IVMP) plus Efgartigimod
干预措施: Intravenous methylprednisolone (IVMP) and Efgartigimod (Drug)
Control group
IVMP
干预措施: IVMP (Drug)
结局指标
主要结局
Change in Expanded Disability Status Scale (EDSS) score from baseline.
时间窗: 1 month
Change in Expanded Disability Status Scale (EDSS) score from baseline to 1 month after treatment (EDSS: Minimum Score 1, Maximum score 10, higher scores mean a worse outcome).
次要结局
- Change in retinal nerve fibre layer (RNFL) loss from baseline(1 month,6 months)
- Percentage of Participants with Disability Improvement(1 month, 3 months, 6 months)
- Number of NMOSD attacked during follow-up(6 months)
- Time to first relapse(6 months)
- Number of New, and/or Enlarging T2 Hyperintense Lesions Detected by Magnetic Resonance Imaging (MRI)(6 months)
- Change in serum NfL levels from baseline(1 month, 6 months)
- Change in timed 25 Foot Walk Test from baseline.(1 month, 3 months, 6 months)
- Change in serum GFAP levels from baseline(1 month, 6 months)
- Change in AQP4-ab titres from baseline(1month, 6 months)
- Adverse reactions during treatment and follow-up(6 months)
- Change in Expanded Disability Status Scale (EDSS) score from baseline.(3 months, 6 months)
- Change in modified Rankin score (mRS) from baseline.(1 month, 3 months, 6 months)
- Change in Visual Acuity (VA) from baseline(1 month, 6 months)
- Changes in EQ-5D-5L scores from baseline(6 months)
研究者
Feng Jinzhou
Associate professor
First Affiliated Hospital of Chongqing Medical University