Abstract
Many patients with primary immunodeficiency (PID) require immunoglobulin G (IgG) replacement therapy, delivered as intravenous IgG (IVIG) or subcutaneous IgG (SCIG). We aim to identify trends in efficacy and safety that would not be evident in individual studies of small patient numbers. Seven open-label, Phase 3, prospective, multicenter studies of the efficacy and safety of Hizentra® (a SCIG), conducted in Japan, Europe, and the US were summarized. Overall, 125 unique patients received 15,013 weekly infusions during a total observation period of 250.9 patient-years. Mean weekly doses of Hizentra® were 83.22–221.3 mg/kg body weight; infusion rates per patient (total body rate) were 25.2–49.3 mL/h across studies. The rates of infections and serious bacterial infections were 3.10 and 0.03 events per patient/year, respectively. Annualized rates of days hospitalized due to infection, out of work/school, and prophylactic antibiotic use were 0.95, 5.14, and 36.78 per patient, respectively. For the equivalent monthly dose, weekly Hizentra® SCIG administration resulted in expectedly-increased serum IgG trough levels in patients switching from IVIG, and maintained levels in patients switching from previous SCIG. Adverse events (AEs) totaled 5039 (events/infusion 0.094–0.773), almost all of which were mild/moderate. Three thousand one hundred ninety-seven were considered treatment-related, the most common of which were injection site reactions (2919 events; 0.001–0.592 AEs per infusion). Systemic AEs were very uncommon. The results from these seven studies indicate that Hizentra® therapy was both efficacious and well tolerated during long-term treatment. This is particularly important in patients with PID, who may require lifelong IgG replacement therapy.
Highlights
The majority of patients with primary immunodeficiency (PID), including common variable immunodeficiency (CVID) and X-linked agammaglobulinemia (XLA), require immunoglobulin G (IgG) replacement therapy [1,2,3]
Total serum IgG trough levels during Hizentra® therapy were compared with those achieved during the mandatory Intravenous IgG (IVIG) treatment periods by calculating the geometric mean ratios (GMR) and respective 90% confidence intervals (CIs)
This review of seven Phase 3 clinical trials in 125 patients who received 15,013 weekly infusions for a total observation period of 250.9 patient years supports that subcutaneous IgG (SCIG) Hizentra® administration as a treatment is effective against infections, serious bacterial infection (SBI), in a broad age range of patients with PID
Summary
The majority of patients with primary immunodeficiency (PID), including common variable immunodeficiency (CVID) and X-linked agammaglobulinemia (XLA), require immunoglobulin G (IgG) replacement therapy [1,2,3]. SCIG delivery permits the patient flexibility with their treatment schedule, and enables home-based self-administration for many patients [1, 7]. Five published clinical trials provide evidence that Hizentra® is efficacious and well tolerated by patients with PID [12,13,14,15]. We summarize results from those trials and two additional unpublished extension studies to further define long-term efficacy and safety in a global context. This integrated summary sought to identify trends in efficacy and safety that might not be evident in individual trials of small numbers of patients, as often occurs with clinical trials in PID
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