Santamaria, Manuel, Neth, Olaf, Douglass, Jo A., Krivan, Gergely, Kobbe, Robin, Bernatowska, Ewa, Grigoriadou, Sofia, Bethune, Claire, Chandra, Anita, Horneff, Gerd, Borte, Michael, Sonnenschein, Anja, Kralickova, Pavlina, Ramon, Silvia Sanchez, Langguth, Daman, Gonzalez-Granado, Luis Ignacio, Alsina, Laia ORCID: 0000-0002-3559-0018, Querolt, Montse, Griffin, Rhonda, Hames, Carrie, Mondou, Elsa, Price, Jeffrey, Sanz, Ana and Lin, Jiang (2022). A Multi-Center, Open-Label, Single-Arm Trial to Evaluate the Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary Immunodeficiency. J. Clin. Immunol., 42 (3). S. 500 - 512. NEW YORK: SPRINGER/PLENUM PUBLISHERS. ISSN 1573-2592

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Abstract

Purpose The purpose of this phase 3 study was to evaluate the efficacy, pharmacokinetics (PK), and safety of Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography Purified (IGSC 20%) in patients with primary immunodeficiency (PI). Methods Immunoglobulin treatment-experienced subjects with PI received 52 weeks of IGSC 20% given weekly at the same dose as the subject's previous IgG regimen (DAF 1:1); the minimum dose was 100 mg/kg/week. The primary endpoint was serious bacterial infections (SBIs [null vs alternative hypothesis: SBI rate per person per year >= 1 vs < 1]). IgG subclasses and specific pathogen antibody levels were also measured. Results Sixty-one subjects (19 children [<= 12 years], 10 adolescents [> 12-16 years], and 32 adults) were enrolled. The rate of SBIs per person per year was 0.017. The 1-sided 99% upper confidence limit was 0.036 (< 1), and the null hypothesis was rejected. The rate of hospitalization due to infection per person per year was 0.017 (2-sided 95% confidence interval: 0.008-0.033) overall. The mean trough total IgG concentrations were comparable to the previous IgG replacement regimen. The average of the individual mean trough ratios (IGSC 20%:previous regimen) was 1.078 (range: 0.83-1.54). The average steady-state mean trough IgG concentrations were 947.64 and 891.37 mg/dL, respectively. Seven subjects had serious treatment-emergent adverse events (TEAEs); none was drug-related. The rate of all TEAEs, including local infusion site reactions, during 3045 IGSC 20% infusions was 0.135. Most TEAEs were mild or moderate. Conclusions IGSC 20% demonstrated efficacy and good safety and tolerability in subjects with PI.

Item Type: Journal Article
Creators:
CreatorsEmailORCIDORCID Put Code
Santamaria, ManuelUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Neth, OlafUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Douglass, Jo A.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Krivan, GergelyUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Kobbe, RobinUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Bernatowska, EwaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Grigoriadou, SofiaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Bethune, ClaireUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Chandra, AnitaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Horneff, GerdUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Borte, MichaelUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Sonnenschein, AnjaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Kralickova, PavlinaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Ramon, Silvia SanchezUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Langguth, DamanUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Gonzalez-Granado, Luis IgnacioUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Alsina, LaiaUNSPECIFIEDorcid.org/0000-0002-3559-0018UNSPECIFIED
Querolt, MontseUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Griffin, RhondaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Hames, CarrieUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Mondou, ElsaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Price, JeffreyUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Sanz, AnaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Lin, JiangUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
URN: urn:nbn:de:hbz:38-696820
DOI: 10.1007/s10875-021-01181-6
Journal or Publication Title: J. Clin. Immunol.
Volume: 42
Number: 3
Page Range: S. 500 - 512
Date: 2022
Publisher: SPRINGER/PLENUM PUBLISHERS
Place of Publication: NEW YORK
ISSN: 1573-2592
Language: English
Faculty: Unspecified
Divisions: Unspecified
Subjects: no entry
Uncontrolled Keywords:
KeywordsLanguage
SUBCUTANEOUS IMMUNOGLOBULIN REPLACEMENT; PRIMARY ANTIBODY DEFICIENCIES; QUALITY-OF-LIFE; IMMUNE GLOBULIN; INTRAVENOUS IMMUNOGLOBULIN; INFUSIONS; THERAPY; HIZENTRA(R); DISEASES; CHILDRENMultiple languages
ImmunologyMultiple languages
URI: http://kups.ub.uni-koeln.de/id/eprint/69682

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