Pediatr Int. 2022 Sep 24:e15362. doi: 10.1111/ped.15362. Online ahead of print.
BACKGROUND: Subcutaneous immunoglobulin (SCIG) is one of the standard treatments for hypogammaglobulinemia in primary immunodeficiencies (PID) worldwide. In Japan, IgPro20 (Hizentra®; L-proline-stabilized 20% human SCIG) is approved for agammaglobulinemia or hypogammaglobulinemia due to PID or secondary immunodeficiency (SID); however, its safety and effectiveness had not been assessed in a real-world setting.
METHODS: This multicenter, open label post-marketing surveillance (PMS) study was conducted between January 2014 and March 2019. Patients who received IgPro20 due to PID or SID were included after informed consent. Physicians completed a case report form for each patient. Safety was determined from reported adverse events (AEs), adverse drug reactions (ADRs), and serious AEs (SAEs); effectiveness was assessed by infection rates after the first IgPro20 dose.
RESULTS: Among 85 patients receiving IgPro20 in the safety analysis, 39 developed AEs (45.9%; PID n=28, SID n=11). At least one ADR was observed in 27 patients (31.8%; PID n=21, SID n=6); the most common was injection site reactions (n=17, 20.0%). Four patients (PID n=3, SID n=1) reported SAEs but two were unrelated to IgPro20 administration. The infection rate decreased from 0.54 per patient during the 6 months before IgPro20 to 0.39 per patient during IgPro20 treatment. Serious bacterial infections occurred in six patients before IgPro20 (7.9%; PID n=2; SID n=4), but in only one patient with SID during IgPro20 treatment (1.2%).
CONCLUSIONS: IgPro20 was considered safe and effective among patients with agammaglobulinemia or hypogammaglobulinemia due to PID or SID in Japan.
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