title: Safety and efficacy of Gammaplex® in idiopathic thrombocytopenic purpura (ClinicalTrials.gov--NCT00504075).
abstract: OBJECTIVE
  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).
  
  
  METHODS
  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.
  
  
  RESULTS
  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.
  
  
  CONCLUSIONS
  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.
  
  
  BACKGROUND
  ClinicalTrials.gov NCT00504075 Clinical Trials Registry India 000016.
authors.affiliation: Medical Department, Bio Products Laboratory Limited (Ltd.), Elstree, Hertfordshire, United Kingdom.
authors.forename: Clive H
authors.initials: CH
authors.lastname: Dash
authors.affiliation: Medical Department, Bio Products Laboratory Limited (Ltd.), Elstree, Hertfordshire, United Kingdom.
authors.forename: Kate R
authors.initials: KR
authors.lastname: Gillanders
authors.affiliation: Medical Department, Bio Products Laboratory Limited (Ltd.), Elstree, Hertfordshire, United Kingdom.
authors.forename: Margaret E
authors.initials: ME
authors.lastname: Stratford Bobbitt
authors.affiliation: Medical Department, Bio Products Laboratory Limited (Ltd.), Elstree, Hertfordshire, United Kingdom.
authors.forename: Ernie W
authors.initials: EW
authors.lastname: Gascoigne
authors.affiliation: Medical Department, Bio Products Laboratory Limited (Ltd.), Elstree, Hertfordshire, United Kingdom.
authors.forename: Samantha J
authors.initials: SJ
authors.lastname: Leach
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D002648:Child; D003710:Demography; D004305:Dose-Response Relationship, Drug; D005260:Female; D006470:Hemorrhage / Q000139:chemically induced; D006801:Humans; D016756:Immunoglobulins, Intravenous / Q000008:administration & dosage / Q000009:adverse effects* / Q000493:pharmacokinetics / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D010976:Platelet Count; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000097:blood / Q000188:drug therapy*; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D016756:Immunoglobulins, Intravenous