title: Comparison of the efficacy of IGIV-C, 10% (caprylate/chromatography) and IGIV-SD, 10% as replacement therapy in primary immune deficiency. A randomized double-blind trial.
abstract: A novel method of large-scale chromatography has been developed to improve recovery and purity of immunoglobulin G (IgG) from pooled plasma. The current study compares safety, toxicity and efficacy of two intravenous immunoglobulin products: a novel formulation, IGIV caprylate/chromatography (IGIV-C; Gamunex, 10%) and a licensed solvent/detergent-treated product, Gamimune N, 10% (IGIV-SD). The study, a randomized, double-blind, parallel group, therapeutic equivalence trial, was conducted at 25 treatment centers in Canada and the United States. Patients (n=172) having confirmed chronic primary immunodeficiency (PID), aged 1-75 years, and receiving IGIV therapy were enrolled. For 9 months, patients were treated with IGIV-C or IGIV-SD in accordance with the patient's individualized treatment regimen utilized before study entry. The primary endpoint was the proportion of patients with >or=1 validated acute sinopulmonary infection during the treatment period. Secondary endpoints included the proportion of patients with all infections, time to first infection, annual infection rates, lung function parameters, infusion-related safety and viral safety. The annual validated infection rate in the IGIV-C group was 0.18 compared to 0.43 in the IGIV-SD group (p=0.023). Nine patients receiving IGIV-C experienced validated infections, compared to 17 patients in IGIV-SD group (p=0.06). Acute sinusitis (validated plus clinically defined) was less frequent in the IGIV-C group (p=0.012). Presence of bronchiectasis did not affect efficacy. Adverse reactions were similar in frequency and severity in both groups. No evidence of viral transmission was observed. IGIV-C appears to be superior to IGIV-SD in preventing validated sinopulmonary infections, especially acute sinusitis, in patients with PID.
authors.affiliation: Division of Immunology/Allergy, Hospital for Sick Children, Toronto, ON, Canada M5G 1X8. croifman@sickkids.on.ca
authors.forename: Chaim M
authors.initials: CM
authors.lastname: Roifman
authors.forename: Harry
authors.initials: H
authors.lastname: Schroeder
authors.forename: Melvin
authors.initials: M
authors.lastname: Berger
authors.forename: Ricardo
authors.initials: R
authors.lastname: Sorensen
authors.forename: Mark
authors.initials: M
authors.lastname: Ballow
authors.forename: Rebecca H
authors.initials: RH
authors.lastname: Buckley
authors.forename: Anita
authors.initials: A
authors.lastname: Gewurz
authors.forename: Phillip
authors.initials: P
authors.lastname: Korenblat
authors.forename: Gordon
authors.initials: G
authors.lastname: Sussman
authors.forename: Georg
authors.initials: G
authors.lastname: Lemm
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D002210:Caprylates / Q000009:adverse effects / Q000302:isolation & purification / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D002852:Chromatography, Ion Exchange / Q000379:methods; D003902:Detergents; D004198:Disease Susceptibility; D004311:Double-Blind Method; D005260:Female; D006801:Humans; D016756:Immunoglobulins, Intravenous / Q000009:adverse effects / Q000302:isolation & purification / Q000627:therapeutic use*; D007153:Immunologic Deficiency Syndromes / Q000150:complications / Q000628:therapy*; D015994:Incidence; D007223:Infant; D017053:Infection Control; D007239:Infections / Q000453:epidemiology / Q000276:immunology; D008297:Male; D008875:Middle Aged; D011014:Pneumonia / Q000453:epidemiology / Q000276:immunology / Q000517:prevention & control; D012852:Sinusitis / Q000453:epidemiology / Q000276:immunology / Q000517:prevention & control; D012997:Solvents; D016896:Treatment Outcome
chemical_list: D002210:Caprylates; D003902:Detergents; D016756:Immunoglobulins, Intravenous; D012997:Solvents