Recombinant Human Hyaluronidase Facilitated Subcutaneous Immunoglobulin Treatment in Pediatric Patients with Primary Immunodeficiencies: Long-Term Efficacy, Safety and Tolerability

• DOI: 10.2217/imt-2016-0066
• Publication Date: 2016-07-28T12:58:46Z
• Authors: Richard L Wasserman; Isaac Melamed; Lisa Kobrynski; Jennifer Puck; Sudhir Gupta; Jennifer Doralt; Marlies Sharkhawy; Werner Engl; Heinz Leibl; David Gelmont; Leman Yel
• Keywords: Tolerability; Primary Immunodeficiency

To assess the long-term efficacy, safety and tolerability of recombinant human hyaluronidase-facilitated subcutaneous infusion immunoglobulin (Ig) (fSCIG; HYQVIA(®); IGHy) in children aged <18 years.Patients with primary immunodeficiency diseases were included studies. IGHy was administered every 3 or 4 weeks.Validated acute serious bacterial infections reported at 0.08/patient-year (four pneumonia episodes three patients). No adverse drug reaction (ADR) reported, rates local systemic ADRs low (0.09/infusion 0.1/infusion). Infection (3.02/patient-year) sustained Ig trough levels (median: 1009 mg/dl). Of 674 infusions, 97.2% required no change administration due to ADR, most (82.5%) one site. patient developed neutralizing anti-rHuPH20 antibodies. Postpivotal study, 100% patients <14 years their caregivers 85.7% 14 expressed preference for compared intravenously subcutaneously.These studies, longest (maximum: 3.3 years) duration any replacement trials diseases, showed infection, along well-tolerated infusions given a single