Dash Clive H, Gillanders Kate R, Stratford Bobbitt Margaret E, Gascoigne Ernie W, Leach Samantha J
Medical Department, Bio Products Laboratory Limited (Ltd.), Elstree, Hertfordshire, United Kingdom.
PLoS One. 2014 Jun 3;9(6):e96600. doi: 10.1371/journal.pone.0096600. eCollection 2014.
This multicentre, open-label study investigated the safety and efficacy of Gammaplex, a 5% Intravenous Immunoglobulin (IVIg), in patients with idiopathic (immune) thrombocytopenic purpura (ITP).
Patients were between the ages of 6 and 70 years; had ITP for at least six months and had a platelet count ≤ 20 × 10(9)/L. Eligible patients were dosed with 1 g/kg of Gammaplex on two consecutive days, followed by assessment of safety and efficacy on Days 3, 5, 9, 14, 21, 32 and 90. Response was defined as the increase in platelet count to a threshold of ≥ 50 × 10(9)/L on or before Day 9 after the first dose of Gammaplex.
All 35 patients received at least one infusion of Gammaplex. Twenty-nine (83%) patients responded to Gammaplex, similar to the historical control, with a 95% lower one-sided confidence interval of 68.9%. Median duration of response was 10.0 days, with an overall reduction in bleeding episodes. Gammaplex provided supranormal concentrations of total IgG; mean peak concentration (Cmax) of 45.3 g/L (4.53 g/dL), with a mean half-life of 28.5 days. Fifteen patients reported 63 Adverse Drug Reactions (ADRs); the most common were headache (10 patients), vomiting (6 patients) and pyrexia (5 patients). Five of these ADRs were considered serious, one patient had three concurrent Serious Adverse Events (SAEs); these were vomiting, dehydration and headache. Two other patients each had one SAE (headache). There were no unexpected Adverse Events (AEs) or thromboembolic episodes and no significant changes in vital signs, biochemical, haematological and virology results.
Gammaplex achieved a very high concentration of serum IgG but was well-tolerated and effective in the treatment of ITP with a similar degree of efficacy to the pre-determined historical control group and the pre-set statistical criteria.
ClinicalTrials.gov NCT00504075 Clinical Trials Registry India 000016.
本多中心、开放标签研究调查了5%静脉注射免疫球蛋白（IVIg）Gammaplex治疗特发性（免疫性）血小板减少性紫癜（ITP）患者的安全性和有效性。
患者年龄在6至70岁之间；患有ITP至少6个月，血小板计数≤20×10⁹/L。符合条件的患者连续两天接受1g/kg的Gammaplex给药，随后在第3、5、9、14、21、32和90天评估安全性和有效性。反应定义为在首次给予Gammaplex后第9天或之前血小板计数增加至≥50×10⁹/L的阈值。
所有35例患者至少接受了一次Gammaplex输注。29例（83%）患者对Gammaplex有反应，与历史对照相似，单侧95%置信区间下限为68.9%。反应的中位持续时间为10.0天，出血事件总体减少。Gammaplex提供了超正常浓度的总IgG；平均峰值浓度（Cmax）为45.3g/L（4.53g/dL），平均半衰期为28.5天。15例患者报告了63例药物不良反应（ADR）；最常见的是头痛（10例患者）、呕吐（6例患者）和发热（5例患者）。其中5例ADR被认为是严重的，1例患者同时发生了3起严重不良事件（SAE）；这些是呕吐、脱水和头痛。另外2例患者各有1起SAE（头痛）。没有意外不良事件（AE）或血栓栓塞事件，生命体征、生化、血液学和病毒学结果也没有显著变化。
Gammaplex达到了非常高的血清IgG浓度，但耐受性良好，在治疗ITP方面有效，疗效程度与预先确定的历史对照组和预设的统计标准相似。
ClinicalTrials.gov NCT00504075 印度临床试验注册000016。
