A Randomized Trial of Plasma Exchange vs. IVIG in the Treatment of Myasthenia Gravis
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 87
- 试验地点
- 2
- 主要终点
- Change in Quantitative Myasthenia Gravis Score (QMGS) from baseline to day 14 after treatment
研究概览
简要总结
Immunomodulation is effective in treating patients with myasthenia gravis (MG), but prior studies have not adequately defined if plasma exchange (PLEX) in superior to intravenous immunoglobulin (IVIG) in the treatment of myasthenia gravis. This study aimed to determine if PLEX was superior to IVIG in the treatment of patients with myasthenia gravis.
Patients with MG requiring immunomodulation are randomized to IVIG or PLEX and treated with a full course of immunomodulation. The quantitative myasthenia gravis score (QMGS) will be evaluated as the primary efficacy parameter at day 14 to determine if PLEX is superior to IVIG.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Investigator, Outcomes Assessor)
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
- •>18 years old
- •diagnosis of moderate-severe MG (defined as a Quantitative Myasthenia Gravis Score QMGS >10.5)
- •worsening weakness requiring a change in therapy judged by a neuromuscular expert
排除标准
- •Worsening weakness secondary to concurrent medications (e.g. Aminoglycosides)
- •Worsening weakness secondary to infection
- •Change in corticosteroid dosage in the 2 weeks prior to screening
- •Other disorders causing weakness or fatigue
- •Known absolute IgA deficiency (risk of anaphylactic reaction to IVIG)
- •History of anaphylaxis or severe systemic response to IVIG or albumin
- •Pregnancy or breastfeeding
- •Active renal failure precluding volume of IVIG (risk of volume overload with IVIG) as judged by the investigators
- •Clinically significant cardiac disease precluding IVIG volume as judged by the investigators
- •Known hyperviscosity or hypercoaguable state (risk of stroke with IVIG)
- •Known coagulopathy with bleeding
- •On another current study medication or protocol within 4 weeks of screening
- •Patients with known refractory status to either IVIG or PLEX
- •Poorly controlled or severe hypertension (exacerbation by IVIG)
- •Patient refuses treatment with either IVIG or PLEX
- •Patient refuses follow-up with electrophysiological studies
- •Patient unable or unwilling to give informed consent
结局指标
主要结局
Change in Quantitative Myasthenia Gravis Score (QMGS) from baseline to day 14 after treatment
时间窗: QMGS at day 14, and patients followed to day 60
QMGS is a validated clinical measure of myasthenia gravis ranging from 0 points (no myasthenic weakness) to a maximum of 39 points, with a defined change of 3.4 units required for clinical significance.
次要结局
- QMGS Score change at days 21 and 28 from start of treatment.(28 days)
- Post intervention status(Day 14, 21 and 28)
- Single fiber electromyography: jitter, percent abnormal pair, percent blocking(Days 14 and 28 compared to baseline)
- Repetitive Nerve stimulation studies(Days 14 and 28)
- Acetylcholine Receptor Antibody titers(Day 28 (if positive at baseline))
- AntiMUSK antibody(Day 28 (if positive at baseline))
- Need for ICU admission, ventilation, intubation(60 days)
- Hospitalization(60 days)
- Need for additional myasthenic treatment(Day 60)