Safety and Efficacy of Gammaplex® in Idiopathic Thrombocytopenic Purpura (ClinicalTrials.gov - NCT00504075)

Abstract

Background and ObjectivesThis multicentre, open-label study investigated the safety and efficacy of Gammaplex, a 5% Intravenous Immunoglobulin (IVIg), in patients with idiopathic (immune) thrombocytopenic purpura (ITP).Materials and MethodsPatients were between the ages of 6 and 70 years; had ITP for at least six months and had a platelet count ≤20×109/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 ≥50×109/L on or before Day 9 after the first dose of Gammaplex.ResultsAll 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. Conclusion: 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.Trial RegistrationClinicalTrials.gov NCT00504075 Clinical Trials Registry India 000016

Tolerability and safety of the intravenous immunoglobulin octagam®10% in patients with immune thrombocytopenia: a post-authorisation safety analysis of two non-interventional phase IV trials

“…Nausea and dizziness were also reported. Such events, together with other minor hypersensitivity reactions involving symptoms such as fevers and chills, are consistent with previously reported studies of octagam® 10% [8], and other ready-to-use liquid preparations of IVIG 10% [9][10][11] and IVIG 5% [12][13][14] in patients with ITP. As was observed with the patients with ITP treated with octagam® 10% in our subgroup analysis, ADRs associated with IVIG are generally transient and of mild-tomoderate severity [8][9][10][11][12][13][14].…”

“…It was also less than the 71.9% response rate achieved with octagam® 5% in patients with ITP seen in supplementary data from the overall analysis [7]. However, this may be explained by the fact that each of the 173 courses of octagam® consisted of only one infusion, which is lower than the recommended two infusions that were administered in the controlled trials [10][11][12]; the mean dose per course (0.9 g/kg) was also lower than in pivotal controlled trials (1.0 g/kg per infusion and 2.0 g/kg per course) [10][11][12]. Furthermore, with regard to the duration of ITP (newly diagnosed or chronic) and treatment history (treatment-naïve or previous IVIG), variable populations of patients may have affected response to octagam® 10% and 5% in the relevant PASS studies.…”

An Idiopathic Thrombocytopenic Purpura Patient Treated With Homeopathy: A Case Report

“…Idiopathic thrombocytopenic purpura (ITP), one of the most common causes of symptomatic thrombocytopenia in children, is an autoimmune-mediated hematologic disorder in which the destruction of platelets occurs resulting in isolated thrombocytopenia, generally defined as a platelet count of less than 100000/mcL, and normal results on a bone marrow examination (except possibly for increased megakaryocytes) [28][29][30]. About 60% of all pediatric cases have a prior history of infection, and most of them experience cutaneous bleeding.…”

“…About 60% of all pediatric cases have a prior history of infection, and most of them experience cutaneous bleeding. ITP is usually a selflimiting, childhood bleeding disorder, and spontaneous recovery is reported in about 50% to 70% of patients [28,30]; however, some cases do require treatment. At present, most treatment protocols concentrate on reducing platelet destruction, and the drugs are usually immunosuppressive [28].…”

“…At present, most treatment protocols concentrate on reducing platelet destruction, and the drugs are usually immunosuppressive [28]. Various treatment modalities applied to treat ITP are corticosteroid therapy [29,31,32], IVIg injection [30,31], splenectomy [29,[31][32][33], thrombopoietin receptor agonists (TPO-RAs) such as romiplostim, [29,31,32,34], and rituximab [29,32,35].…”