PURPOSE

This study aimed to evaluate how effective and safe a treatment called hyaluronidase-facilitated subcutaneous immunoglobulin (fSCIG) 10% is for children in the U.S. with primary immunodeficiency diseases (PIDDs).

METHODS

The research involved 44 children aged 2 to under 16, who had been receiving immunoglobulin G (IgG) continuously for at least 3 months before the study began. Participants received fSCIG 10% through a gradual increase over 6 weeks and then continued every 3-4 weeks for up to 3 years. The main focus was on the number of serious bacterial infections.

RESULTS

Out of the 44 participants, 34 completed the study. Only one participant had two serious bacterial infections (both were pneumonia). On average, the rate of serious bacterial infections was very low at 0.04 events per participant per year, much lower than the acceptable level of 1.0. Overall, participants experienced about 3.12 infections per year. The treatment maintained stable IgG levels over time. Most side effects were mild or moderate, and no serious immune reactions were observed.

CONCLUSION

The treatment was effective in preventing serious bacterial infections in children with PIDDs and was well-tolerated, showing a good safety profile.

Opportunities Based on Trial Data

Identifying clear goals for the effectiveness, safety, and tolerability of this treatment can help clinics and patients track outcomes more effectively.