University Medical Centre, Department of Neurology, Erasmus MC, Room Ee-2230, PO Box 2040, 3000 CA Rotterdam, The Netherlands.
Expert Opin Emerg Drugs. 2011 Mar;16(1):105-20. doi: 10.1517/14728214.2011.531699.
Intravenous immunoglobulin (IVIg) and plasma exchange are proven effective treatments for Guillain-Barré syndrome (GBS). However, this treatment is insufficient for many patients as 1 - 5% die, 25% need artificial respiration, 20% are still unable to walk unaided after 6 months and 85% have residual symptoms, such as fatigue and pain.
Strategies to design and conduct trials with new compounds and individualized regimens of IVIg are discussed. The development of specific immunomodulators is set against a background of recent insights in the pathophysiological mechanisms of GBS. Patients with poor prognosis can be identified in the early phase of disease using predictors such as high age, severe disability, preceding diarrhea and possibly low increase in serum IgG after standard IVIg treatment. An ongoing trial with a second IVIg dose in this group and the preclinical development of potential new treatments and their mode of action are discussed.
GBS is a heterogeneous disease with considerable short- and long-term disability for which more effective and individualized treatments are required. Under investigation are new treatment strategies with adapted IVIg dosages based on prognostic factors and more specific immunomodulation, including complement inhibitors.
静脉注射免疫球蛋白（IVIg）和血浆置换已被证实可有效治疗格林-巴利综合征（GBS）。然而，对于许多患者来说，这种治疗并不足够，因为有 1-5%的患者死亡，25%需要人工呼吸，20%在 6 个月后仍无法独立行走，85%有残留症状，如疲劳和疼痛。
讨论了设计和进行新化合物和 IVIg 个体化方案临床试验的策略。特定免疫调节剂的开发是基于 GBS 病理生理机制的最新见解。使用预测指标（如高龄、严重残疾、先前腹泻以及标准 IVIg 治疗后血清 IgG 升高幅度可能较低）可以在疾病早期识别预后不良的患者。正在进行一项针对该组患者的第二剂 IVIg 试验，以及潜在新治疗方法及其作用模式的临床前开发。
GBS 是一种异质性疾病，患者存在严重的短期和长期残疾，需要更有效和个体化的治疗方法。正在研究的是新的治疗策略，包括根据预后因素调整 IVIg 剂量和更具特异性的免疫调节，包括补体抑制剂。
