title: Safety and efficacy of self-administered subcutaneous immunoglobulin in patients with primary immunodeficiency diseases.
abstract: Intravenous immunoglobulin (IVIg) infusions at 3-4 week intervals are currently standard therapy in the United States for patients with primary immune deficiency diseases (PIDD). To evaluate alternative modes of immunoglobulin administration we have designed an open-label study to investigate the efficacy and safety of a subcutaneously administered immunoglobulin preparation (16% IgG) in patients with PIDD. After their final IVIg infusion, 65 patients entered a 3-month, wash-in/wash-out phase, designed to bring patients to steady-state with subcutaneously administered immunoglobulin. This was followed by 12 months of weekly SCIg infusions, at a dose determined in a pharmacokinetic substudy to provide noninferior intravascular exposure. This resulted in a mean weekly dose of 158 mg/kg, calculated to equal 137% of the previous intravenous dose. Two patients (4%) each reported 1 serious bacterial infection (pneumonia), an annual rate of 0.04 per patient-year. There were 4.43 infections of any type per patient-year. Mean trough serum IgG levels increased from 786 to 1040 mg/dL during the study, a mean increase of 39%. The most frequent treatment-related adverse event was infusion-site reaction, reported by 91% of patients; this was predominantly mild or moderate, and the incidence decreased over time. No treatment-related serious adverse events were reported. We conclude that subcutaneous administration of 16% SCIg is a safe and effective alternative to IVIg for replacement therapy of PIDD.
authors.affiliation: Department of Pediatrics, University of Washington School of Medicine, Seattle, Washington 98107, USA. allgau@u.washington.edu
authors.forename: Hans D
authors.initials: HD
authors.lastname: Ochs
authors.forename: Sudhir
authors.initials: S
authors.lastname: Gupta
authors.forename: Peter
authors.initials: P
authors.lastname: Kiessling
authors.forename: Uwe
authors.initials: U
authors.lastname: Nicolay
authors.forename: Melvin
authors.initials: M
authors.lastname: Berger
mesh_terms: D000293:Adolescent; D000328:Adult; D000361:Agammaglobulinemia / Q000188:drug therapy / Q000276:immunology*; D000368:Aged; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D007074:Immunoglobulin G / Q000097:blood; D007136:Immunoglobulins / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D007153:Immunologic Deficiency Syndromes / Q000188:drug therapy / Q000276:immunology*; D007279:Injections, Subcutaneous; D008297:Male; D008875:Middle Aged
chemical_list: D007074:Immunoglobulin G; D007136:Immunoglobulins; C510539:Vivaglobin