title: Efficacy and safety of home-based subcutaneous immunoglobulin replacement therapy in paediatric patients with primary immunodeficiencies.
abstract: Subcutaneous immunoglobulin infusions are effective, safe and well tolerated in the treatment of primary immunodeficiencies, but only limited data on the treatment of children are available. We investigated the efficacy, safety and pharmacokinetics of home therapy with a 16% liquid human immunoglobulin G preparation (Vivaglobin®) when administered subcutaneously in children with primary immunodeficiencies. Data were analysed from 22 children (2-<12 years) who participated in two prospective, open-label studies (one in Europe/Brazil, one in North America). All children had previously received intravenous immunoglobulins. They started weekly subcutaneous immunoglobulin infusions with an approximately 3-month wash-in/wash-out period, followed by a 6-month (Europe/Brazil) or 12-month (North America) efficacy evaluation period. In Europe/Brazil, subcutaneous doses generally equalled the previous weekly equivalent intravenous doses. In North America, subcutaneous doses during the efficacy evaluation period were 126% (median) of the previous weekly equivalent intravenous doses. Efficacy end-points in both studies included the occurrence of serious bacterial infections and any infections, and serum immunoglobulin G trough levels. Median serum immunoglobulin G trough levels exceeded those during previous intravenous therapy by 13% (North America) and 16% (Europe/Brazil). During the efficacy evaluation period of both studies, none of the children had a serious bacterial infection; the mean overall infection rate/patient year was 4·7 in Europe/Brazil and 5·6 in North America, concurring with previous reports in adults. The adverse event profile was comparable to previous reports in adults. Both studies confirmed the efficacy and safety of subcutaneous immunoglobulin therapy with Vivaglobin in children with primary immunodeficiencies.
authors.affiliation: Municipal Hospital St Georg Leipzig, Academic Teaching Hospital of the University of Leipzig, Germany. michael.borte@sanktgeorg.de
authors.forename: M
authors.initials: M
authors.lastname: Borte
authors.forename: E
authors.initials: E
authors.lastname: Bernatowska
authors.forename: H D
authors.initials: HD
authors.lastname: Ochs
authors.forename: C M
authors.initials: CM
authors.lastname: Roifman
mesh_terms: D001938:Brazil; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D005060:Europe; D005260:Female; D005500:Follow-Up Studies; D018718:Home Infusion Therapy; D006801:Humans; D007136:Immunoglobulins / Q000008:administration & dosage* / Q000009:adverse effects; D007153:Immunologic Deficiency Syndromes / Q000188:drug therapy* / Q000276:immunology / Q000503:physiopathology; D007239:Infections; D007279:Injections, Subcutaneous; D008297:Male; D009656:North America
chemical_list: D007136:Immunoglobulins; C510539:Vivaglobin