Real-World Use, Safety, and Patient Experience of 20% Subcutaneous Immunoglobulin for Primary Immunodeficiency Diseases.

Abstract

INTRODUCTION:

The CORE study aimed to provide a detailed understanding of real-world immune globulin subcutaneous (human) 20% solution (Ig20Gly) utilization in patients with primary immunodeficiency diseases (PIDs) in Germany and Switzerland.

METHODS:

Patients with PIDs receiving a stable dose of any subcutaneous immunoglobulin for ≥ 3 months before enrollment were eligible for this multicenter (n = 5), phase 4, non-interventional, prospective, longitudinal cohort study. Besides baseline demographics and clinical characteristics, Ig20Gly utilization and safety data, and patient-reported outcomes (Life Quality Index/Treatment Satisfaction Questionnaire for Medication) were collected at baseline, 6 and 12 months. Statistical analysis was descriptive.

RESULTS:

Overall, 36 patients provided data at baseline [69.4% female; mean age: 41.6 years (7-78 years)]. Totals of 23 and 26 patients attended 6- and 12-month visits, respectively; 16 attended all three visits. One patient withdrew consent before 6-month follow-up. Median maximum infusion rates of Ig20Gly at baseline, 6 months, and 12 months were 26.7, 24.5, and 40.0 mL/h, respectively (10-60 mL/h). Infusion and dosing parameters remained consistent across time points: patients used a median of two infusion sites, primarily the abdomen, and all patients used an infusion pump; all but one infused at home and most self-administered Ig20Gly (80.8-83.3%) at once-weekly intervals (69.2-73.9%). During follow-up, 10 adverse events were reported: none were rated serious, while 2 were considered probably related to Ig20Gly. Total patient-reported outcome scores remained high throughout the study.

CONCLUSION:

The CORE study provides real-world evidence of the flexibility, feasibility, safety, and tolerability of Ig20Gly infusions, at mostly weekly intervals, over 1 year in patients with PIDs.

TRIAL REGISTRATION:

German Clinical Trials Register, DRKS00014562. Registered April 9, 2018, https://drks.de/search/en/trial/DRKS00014562.

• Authors: Maria Fasshauer, Michael Borte, Michaela Bitzenhofer, Christine Pausch, David Pittrow, Michelle Park, André Gladiator, Peter Jandus
• Journal: Advances in therapy (Adv Ther) Vol. 40 Issue 12 Pg. 5168-5187 (12 2023) ISSN: 1865-8652 [Electronic] United States
• PMID: 37751025 (Publication Type: Multicenter Study, Journal Article, Research Support, Non-U.S. Gov't)
• Copyright: © 2023. The Author(s).

Chemical References

• Immunoglobulin G

Topics

• Humans
• Female
• Adult
• Male
• Immunologic Deficiency Syndromes (chemically induced, drug therapy)
• Prospective Studies
• Longitudinal Studies
• Immunoglobulin G
• Infusions, Subcutaneous
• Primary Immunodeficiency Diseases (drug therapy)
• Patient Outcome Assessment