title: Efficacy and safety of a nanofiltered liquid intravenous immunoglobulin product in patients with primary immunodeficiency and idiopathic thrombocytopenic purpura.
abstract: OBJECTIVE
  In the production process of a new 5% liquid intravenous immunoglobulin (IVIG-L) product (Nanogam(®) ), a combined pepsin/pH 4·4 treatment/15-nm filtration (pH 4·4/15NF) step and a solvent-detergent (SD) treatment step were incorporated to improve the virus inactivating/reducing capacity of the manufacturing process. Two prospective uncontrolled multicentre studies were performed to evaluate the safety and efficacy of this product.
  
  
  METHODS
  Efficacy, including pharmacokinetics, of IVIG-L was studied for 6 months in 18 primary immunodeficiency (PID) patients, succeeded by a long-term follow-up study (mean 2·2 years, n=17). Second, in 24 patients with idiopathic thrombocytopenic purpura (ITP), IVIG-L was studied for efficacy for 14 days. In both studies, adverse events and vital signs were recorded to study safety.
  
  
  RESULTS
  In PID patients treated with IVIG-L, 0·60 and 0·38 severe infections per patient per year were reported during, respectively, the short-term and long-term follow-up. Pharmacokinetic studies resulted in an IgG half-life of 30·9 ± 11·3 days and a mean IgG trough level of 6·8 ± 1·2 g/l. In the ITP study, all patients showed an increase in platelet counts after infusion with IVIG-L, and 20/24 patients responded with a platelet count >50 × 10(9) /l (83·3%) within 1 week. IVIG-L infusions did not cause clinical relevant changes in laboratory parameters or vital signs.
  
  
  CONCLUSIONS
  In clinical studies, IVIG-L (Nanogam®) demonstrated to be efficacious, well tolerated and safe.
authors.affiliation: Department of General Internal Medicine, Radboud University Nijmegen Medical Centre, Nijmegen, the Netherlands.
authors.forename: J W M
authors.initials: JW
authors.lastname: van der Meer
authors.forename: R T
authors.initials: RT
authors.lastname: van Beem
authors.forename: T
authors.initials: T
authors.lastname: Robak
authors.forename: A
authors.initials: A
authors.lastname: Deptala
authors.forename: P F W
authors.initials: PF
authors.lastname: Strengers
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D005260:Female; D006801:Humans; D016756:Immunoglobulins, Intravenous / Q000008:administration & dosage* / Q000009:adverse effects / Q000737:chemistry / Q000493:pharmacokinetics; D007153:Immunologic Deficiency Syndromes / Q000188:drug therapy* / Q000378:metabolism; D008297:Male; D008875:Middle Aged; D036103:Nanotechnology / Q000379:methods; D011446:Prospective Studies; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000188:drug therapy* / Q000378:metabolism; D055815:Young Adult
chemical_list: D016756:Immunoglobulins, Intravenous