title: IGIV-C, a novel intravenous immunoglobulin: evaluation of safety, efficacy, mechanisms of action, and impact on quality of life.
abstract: The general safety and efficacy of intravenous immunoglobulin (IGIV) as treatment for idiopathic thrombocytopenic purpura (ITP) has been well-studied. The current study compares the safety and efficacy of a novel IGIV (IGIV-C; Gamunex, 10%) with a licensed solvent/detergent-treated product (IGIV-S/D; GamimuneN, 10%) in treatment of ITP. Ninety-seven pediatric and adult patients with acute and chronic ITP were treated in a multi-center, prospective, randomized, double-blind parallel group, non-inferiority trial at 26 international sites. Baseline data (age, duration of ITP, platelet counts, previous treatment) were comparable between groups. Patients were treated with 1 g/kg/day of IGIV-C or IGIV-S/D for 2 days. The primary end-point, proportion of patients whose platelet counts increased from >/=20 x 10(9)/L to >/=50 x 10(9)/L within 7 days after dosing, was achieved by 35/39 (90%) and 35/42 (83%) of patients treated with IGIV-C and IGIV-S/D, respectively. A secondary endpoint, maintaining platelet counts >/=50 x 10(9)/L for >/=7 days, was achieved by 29/39 (74%) of IGIV-C and 25/42 (60%) IGIV-S/D treated patients. Compared with IGIV-S/D, fewer patients treated with IGIV-C received corticosteroids beyond day 7 (p = 0.02). Efficacy was independent of the presence of isoantibodies or blood type, supporting mechanisms of effect different from anti-D treatments. Adverse events were generally mild and occurred with similar frequency in each group. Viral safety monitoring for HIV, HCV, HBV and Parvovirus B19 showed no seroconversions on study. In conclusion, IGIV-C is as safe and efficacious as IGIV-S/D in treatment of ITP.
authors.affiliation: New York Presbyterian Hospital, Payson 695, 525 East 68th Street, New York, New York 10021-4885, USA. jbussel@med.cornell.edu
authors.forename: James B
authors.initials: JB
authors.lastname: Bussel
authors.forename: Amiram
authors.initials: A
authors.lastname: Eldor
authors.forename: John G
authors.initials: JG
authors.lastname: Kelton
authors.forename: David
authors.initials: D
authors.lastname: Varon
authors.forename: Benjamin
authors.initials: B
authors.lastname: Brenner
authors.forename: Shmuel
authors.initials: S
authors.lastname: Gillis
authors.forename: Anne
authors.initials: A
authors.lastname: Angiolillo
authors.forename: Roshni
authors.initials: R
authors.lastname: Kulkarni
authors.forename: Thomas C
authors.initials: TC
authors.lastname: Abshire
authors.forename: Jack
authors.initials: J
authors.lastname: Kelleher
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D002210:Caprylates / Q000008:administration & dosage / Q000627:therapeutic use / Q000633:toxicity; D002648:Child; D002675:Child, Preschool; D004311:Double-Blind Method; D005260:Female; D006801:Humans; D016756:Immunoglobulins, Intravenous / Q000008:administration & dosage* / Q000627:therapeutic use / Q000633:toxicity; D015994:Incidence; D007223:Infant; D008297:Male; D008875:Middle Aged; D010976:Platelet Count; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000188:drug therapy*; D011788:Quality of Life; D013242:Sterilization; D016896:Treatment Outcome; D014777:Virus Diseases / Q000635:transmission
chemical_list: D002210:Caprylates; D016756:Immunoglobulins, Intravenous