1. BT595, a 10% Human Normal Immunoglobulin, for Replacement Therapy of Primary Immunodeficiency Disease: Results of a Subcohort Analysis in Children
- Author
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Gergely Kriván, Michael Borte, Pere Soler-Palacin, Joseph A. Church, Ildiko Csurke, James B. Harris, Jay A. Lieberman, Isaac R. Melamed, James N. Moy, Reka Simon, Silke Aigner, Stephan Lentze, Christiane Staiger, Institut Català de la Salut, [Kriván G] Department of Pediatric Hematology and Stem Cell Transplantation, United St. Istvan and St Laszlo Hospital, Budapest, Hungary. [Borte M] ImmunoDefciency Center Leipzig (IDCL) at Klinikum St. Georg gGmbH, Leipzig, Germany. [Soler-Palacin P] Vall d’Hebron Hospital Universitari, Barcelona, Spain. [Church JA] Children’s Hospital Los Angeles, Los Angeles, CA, USA. [Csurke I] Szabolcs-Szatmar-Bereg Megyei Korhazak és Egyetemi Oktatokorhaz, Nyíregyháza, Hungary. [Harris JB] The South Bend Clinic, South Bend, IN, USA, and Vall d'Hebron Barcelona Hospital Campus
- Subjects
enfermedades del sistema inmune::síndromes de inmunodeficiencia [ENFERMEDADES] ,Amino Acids, Peptides, and Proteins::Proteins::Blood Proteins::Immunoproteins::Immunoglobulins::Antibodies::Immunoglobulin Isotypes::Immunoglobulin G::Immunoglobulins, Intravenous [CHEMICALS AND DRUGS] ,aminoácidos, péptidos y proteínas::proteínas::proteínas sanguíneas::inmunoproteínas::inmunoglobulinas::anticuerpos::isotipos de inmunoglobulinas::inmunoglobulina G::inmunoglobulinas intravenosas [COMPUESTOS QUÍMICOS Y DROGAS] ,Otros calificadores::/uso terapéutico [Otros calificadores] ,Immunology ,Immune System Diseases::Immunologic Deficiency Syndromes [DISEASES] ,Immunology and Allergy ,Otros calificadores::Otros calificadores::/farmacoterapia [Otros calificadores] ,Other subheadings::/therapeutic use [Other subheadings] ,Immunoglobulines - Ús terapèutic ,Other subheadings::Other subheadings::/drug therapy [Other subheadings] ,Síndromes de deficiència immunitària - Tractament - Abstract
Purpose To assess the efficacy, pharmacokinetics, and safety of a new, highly purified 10% IVIg (BT595, Yimmugo®) administered in children with PID. Methods This was an open-label, prospective, uncontrolled, multicenter Phase III pivotal trial. Among the 67 subjects in the trial were 18 pediatric patients aged 2 to 17 years with diagnosis of PID included in this analysis. They received doses between 0.2 and 0.8 g/kg body weight for approximately 12 months at intervals of either 3 or 4 weeks. Dosage and dosing interval were based on each patient’s pre-trial infusion schedule. The rates of acute serious bacterial infections (SBI), secondary efficacy, safety, and pharmacokinetic outcomes were evaluated. Results No SBI occurred in the pediatric population. Two hundred sixty infusions were administered to the 18 pediatric patients. The mean (SD) IgG trough level was 8.55 (1.67) g/L at baseline and 8.84 (2.17) g/L at the follow-up visit after the last BT595 infusion. At the single infusions respectively, the average mean IgG trough levels ranged between 8.52 and 10.58 g/L. More than 85% of all infusions administered were not associated with any infusional AE (start during or within 72 h post-infusion). None of the severe or serious AEs were related to the investigational medicinal product (IMP). No premedication was used. Thirteen children reached a maximum infusion rate between > 2.0 and 8 mL/kg/h; no AE with an onset during the infusion occurred at these infusion rates. Conclusion BT595 is effective, convenient, well tolerated, and safe for the treatment of children with PID. Trial registration EudraCT: 2015–003652-52; NCT02810444, registered June 23, 2016.
- Published
- 2023