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

Publikation: Beitrag in FachzeitschriftForschungsartikelBeigetragenBegutachtung

Beitragende

  • Maria Fasshauer - , Universität Leipzig (Autor:in)
  • Michael Borte - , Universität Leipzig (Autor:in)
  • Michaela Bitzenhofer - , Universität Bern (Autor:in)
  • Christine Pausch - , Technische Universität Dresden, GWT-TUD GmbH (Autor:in)
  • David Pittrow - , Technische Universität Dresden, Institut für Klinische Pharmakologie, GWT-TUD GmbH (Autor:in)
  • Michelle Park - , Takeda Pharmaceutical Company GmbH (Autor:in)
  • André Gladiator - , Takeda Pharmaceutical Company GmbH (Autor:in)
  • Peter Jandus - , Hôpitaux universitaires de Genève (Autor:in)

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.

Details

OriginalspracheEnglisch
Seiten (von - bis)5168-5187
Seitenumfang20
FachzeitschriftAdvances in therapy
Jahrgang40
Ausgabenummer12
PublikationsstatusVeröffentlicht - Dez. 2023
Peer-Review-StatusJa
Extern publiziertJa

Externe IDs

PubMed 37751025

Schlagworte

Ziele für nachhaltige Entwicklung

ASJC Scopus Sachgebiete

Schlagwörter

  • Antibody deficiency syndromes, Ig20Gly, Immunoglobulin replacement therapy, Inborn errors of immunity, Observational, Primary immunodeficiency diseases, SCIG, Subcutaneous immunoglobulin