Long-term efficacy, safety, and tolerability of a subcutaneous immunoglobulin 16.5% (cutaquig (R)) in the treatment of patients with primary immunodeficiencies

Authors: Kobayashi, RH; Litzman, J; Melamed, I; Mandujano, JF; Kobayashi, AL; Ritchie, B; Geng, B; Atkinson, TP; Rehman, S; Hoeller, S; Turpel-Kantor, E; Kreuwel, H; Speer, JC; Gupta, S

Affiliations: UCLA, Sch Med, Los Angeles, CA 90095 USA. Masaryk Univ, Fac Med, St Annes Univ Hosp Brno, Dept Clin Immunol & Allergol, Brno, Czech Republic. IMMUNOe Res Ctr, Centennial, CO USA. Pediat Pulm Associates North Texas, Frisco, TX USA. Midlands Pediat, Papillion, NE USA. Univ Alberta Hosp, Dept Med, Div Hematol, Edmonton, AB, Canada. Univ Calif San Diego, Div Allergy, La Jolla, CA 92093 USA. Univ Calif San Diego, Div Immunol, La Jolla, CA 92093 USA. Univ Alabama Birmingham, Dept Pediat Allergy Asthma & Immunol, Birmingham, AL USA. Allergy & Asthma Ctr Inc, Toledo, OH USA. Octapharma Pharmazeut Prod GesmbH, Vienna, Austria. Octapharma US, Paramus, NJ USA. Univ Calif Irvine, Div Basic & Clin Immunol, Irvine, CA 92717 USA.

Publication: Clinical and experimental immunology , November 2022

Abstract: A prospective study and its long-term extension examined whether weekly treatment of patients with primary immunodeficiencies (PIDs) with a 16.5% subcutaneous immunoglobulin (SCIg; cutaquig (R)) confers acceptable efficacy, safety, and tolerability over a follow-up of up to 238 weeks (>4 years). Seventy-five patients received 4462 infusions during up to 70 weeks of follow-up in the main study and 27 patients received 2777 infusions during up to 168 weeks of follow-up in the extension. In the main study, there were no serious bacterial infections (SBIs), and the annual rate of other infections was 3.3 (95% CI 2.4, 4.5). One SBI was recorded in the extension, for an SBI rate of 0.02 (upper 99% CI 0.19). The annual rate of all infections over the duration of the extension study was 2.2 (95% CI 1.2, 3.9). Only 15.0% (1085) of 7239 infusions were associated with infusion site reactions (ISRs), leaving 85.0% (6153) of infusions without reactions. The majority of ISRs were mild and transient. ISR incidence decreased over time, from 36.9% to 16% during the main study and from 9% to 2.3% during the extension. The incidence of related systemic adverse events was 14.7% in the main study and 74% in the extension. In conclusion, this prospective, long-term study with cutaquig showed maintained efficacy and low rates of local and systemic adverse reactions in PID patients over up to 238 weeks of follow-up.