fSCIG 10% in pediatric primary immunodeficiency diseases: a European post-authorization safety study

Area of Science:

• Immunology
• Pediatrics
• Pharmacology

Background:

• Assessed the safety, tolerability, and immunogenicity of hyaluronidase-facilitated subcutaneous immunoglobulin (fSCIG) 10% in pediatric patients with primary immunodeficiency diseases (PIDs).
• fSCIG 10% combines human immunoglobulin 10% with recombinant human hyaluronidase (rHuPH20).

Purpose of the Study:

• To evaluate the safety and tolerability of fSCIG 10% in children diagnosed with PIDs.
• To determine the incidence of noninfectious treatment-related adverse events (AEs) in pediatric PID patients receiving fSCIG 10%.

Main Methods:

• Phase 4, prospective, interventional, multicenter study (NCT03116347) involving 42 pediatric patients (aged 2 to <18 years) with PIDs.
• Patients either initiated fSCIG 10% with dose ramp-up or were pretreated.
• Primary outcome: number and rate of noninfectious treatment-related serious and severe adverse events (AEs).

Main Results:

• 49 related noninfectious adverse events (TEAEs) occurred in 15 patients, mostly mild (87.8%).
• No serious treatment-related TEAEs were reported; two severe TEAEs (infusion site pain, emotional distress) occurred in one new starter.
• Local TEAE rates were lower in pretreated patients (0.1/patient-year) vs. new starters (1.3/patient-year); no anti-rHuPH20 antibodies detected.

Conclusions:

• fSCIG 10% demonstrated a favorable safety profile in children with PIDs, with no identified safety signals.
• The incidence of local adverse events decreased over the treatment duration.
• The study supports the long-term safety of fSCIG 10% for pediatric PID patients.