title: Recombinant human hyaluronidase facilitated subcutaneous immunoglobulin treatment in pediatric patients with primary immunodeficiencies: long-term efficacy, safety and tolerability.
abstract: To assess the long-term efficacy, safety and tolerability of recombinant human hyaluronidase-facilitated subcutaneous infusion of immunoglobulin (Ig) (fSCIG; HYQVIA(®); IGHy) in children aged <18 years.
  
  
  
  Patients with primary immunodeficiency diseases were included in the studies. IGHy was administered every 3 or 4 weeks.
  
  
  
  Validated acute serious bacterial infections were reported at 0.08/patient-year (four pneumonia episodes in three patients). No serious adverse drug reaction (ADR) was reported, and rates of local and systemic ADRs were low (0.09/infusion and 0.1/infusion). Infection rates were low (3.02/patient-year) with sustained Ig trough levels (median: 1009 mg/dl). Of 674 IGHy infusions, 97.2% required no change of administration due to ADR, in most (82.5%) with one infusion site. No patient developed neutralizing anti-rHuPH20 antibodies. Postpivotal study, 100% of patients aged <14 years or their caregivers and 85.7% of patients aged 14 to <18 years expressed preference for IGHy compared with Ig administered intravenously or Ig administered subcutaneously.
  
  
  
  These studies, with the longest (maximum: 3.3 years) duration of any reported Ig replacement trials in children with primary immunodeficiency diseases, showed low infection, local and systemic reaction rates along with well-tolerated infusions given in a single site.
authors.affiliation: Allergy Partners of North Texas Research, Dallas, TX, USA.
authors.forename: Richard L
authors.initials: RL
authors.lastname: Wasserman
authors.affiliation: IMMUNOe Health Centers, Centennial, CO, USA.
authors.forename: Isaac
authors.initials: I
authors.lastname: Melamed
authors.affiliation: Emory University, Atlanta, GA, USA.
authors.forename: Lisa
authors.initials: L
authors.lastname: Kobrynski
authors.affiliation: University of California, San Francisco, San Francisco, CA, USA.
authors.forename: Jennifer
authors.initials: J
authors.lastname: Puck
authors.affiliation: University of California, Irvine, Irvine, CA, USA.
authors.forename: Sudhir
authors.initials: S
authors.lastname: Gupta
authors.affiliation: Baxalta Innovations GmbH, now part of Shire, Vienna, Austria.
authors.forename: Jennifer
authors.initials: J
authors.lastname: Doralt
authors.affiliation: Baxalta Innovations GmbH, now part of Shire, Vienna, Austria.
authors.forename: Marlies
authors.initials: M
authors.lastname: Sharkhawy
authors.affiliation: Baxalta Innovations GmbH, now part of Shire, Vienna, Austria.
authors.forename: Werner
authors.initials: W
authors.lastname: Engl
authors.affiliation: Baxalta Innovations GmbH, now part of Shire, Vienna, Austria.
authors.forename: Heinz
authors.initials: H
authors.lastname: Leibl
authors.affiliation: Baxalta US Inc., now part of Shire, Cambridge, MA, USA.
authors.forename: David
authors.initials: D
authors.lastname: Gelmont
authors.affiliation: University of California, Irvine, Irvine, CA, USA.|Baxalta US Inc., now part of Shire, Cambridge, MA, USA.
authors.forename: Leman
authors.initials: L
authors.lastname: Yel
mesh_terms: D000293:Adolescent; D000951:Antigens, Neoplasm / Q000009:adverse effects / Q000235:genetics / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D005260:Female; D051548:Histone Acetyltransferases / Q000009:adverse effects / Q000235:genetics / Q000627:therapeutic use*; D006801:Humans; D006821:Hyaluronoglucosaminidase / Q000009:adverse effects / Q000235:genetics / Q000627:therapeutic use*; D007136:Immunoglobulins / Q000627:therapeutic use*; D007153:Immunologic Deficiency Syndromes / Q000276:immunology / Q000628:therapy*; D007279:Injections, Subcutaneous; D008297:Male; D011994:Recombinant Proteins / Q000235:genetics; D013997:Time Factors; D014481:United States
chemical_list: D000951:Antigens, Neoplasm; D007136:Immunoglobulins; D011994:Recombinant Proteins; D051548:Histone Acetyltransferases; C115866:OGA protein, human; D006821:Hyaluronoglucosaminidase
keywords: IVIG; PIDD; home infusion; hyaluronidase; immunoglobulin; primary immunodeficiency disease