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Clinical Efficacy, Safety and Tolerability of a New Subcutaneous Immunoglobulin 16.5% (Octanorm [Cutaquig®]) in the Treatment of Patients With Primary Immunodeficiencies

Introduction: Subcutaneously administered immunoglobulin (SCIG) is increasingly used to treat patients with primary immunodeficiencies (PIDs). Octanorm (marketed as cutaquig® in USA and Canada) is a new 16.5% solution of human SCIG, manufactured by a process based on that of the intravenous preparat...

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Autores principales: Kobayashi, Roger H., Gupta, Sudhir, Melamed, Isaac, Mandujano, J. Fernando, Kobayashi, Ai Lan, Ritchie, Bruce, Geng, Bob, Atkinson, Thomas Prescott, Rehman, Syed, Turpel-Kantor, Eva, Litzman, Jiří
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6369354/
https://www.ncbi.nlm.nih.gov/pubmed/30778345
http://dx.doi.org/10.3389/fimmu.2019.00040
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author Kobayashi, Roger H.
Gupta, Sudhir
Melamed, Isaac
Mandujano, J. Fernando
Kobayashi, Ai Lan
Ritchie, Bruce
Geng, Bob
Atkinson, Thomas Prescott
Rehman, Syed
Turpel-Kantor, Eva
Litzman, Jiří
author_facet Kobayashi, Roger H.
Gupta, Sudhir
Melamed, Isaac
Mandujano, J. Fernando
Kobayashi, Ai Lan
Ritchie, Bruce
Geng, Bob
Atkinson, Thomas Prescott
Rehman, Syed
Turpel-Kantor, Eva
Litzman, Jiří
author_sort Kobayashi, Roger H.
collection PubMed
description Introduction: Subcutaneously administered immunoglobulin (SCIG) is increasingly used to treat patients with primary immunodeficiencies (PIDs). Octanorm (marketed as cutaquig® in USA and Canada) is a new 16.5% solution of human SCIG, manufactured by a process based on that of the intravenous preparation (IVIG) octagam®. Objectives: To investigate the efficacy, safety and tolerability of octanorm in a prospective, open-label, single-arm phase 3 study involving adult and pediatric patients with PIDs (NCT01888484; clinicaltrials.gov/ct2/show/NCT01888484). Methods: Patients who were previously treated with IVIG received a total of 64 weekly SCIG infusions, including 12 weekly infusions during the wash-in/wash-out period, followed by 52 weekly infusions during the evaluation period. Results: A total of 61 patients aged 2–73 years received 3,497 infusions of octanorm. The mean dose per patient was 0.175 g/kg/infusion. The mean calculated dose conversion factor from the patients' previous IVIG dose for octanorm was 1.37. No serious bacterial infections developed during the study. The rate of other infections per person-year during the primary observation period was 3.43 (upper 95% CI 4.57). All but one non-bacterial infection were mild or moderate in intensity. IgG trough levels were constant during the course of the study. Eleven patients (18.0%) experienced 14 mild or moderate systemic adverse events (AEs) related to octanorm. The rate of related AEs per infusion was 0.004. In 76.7% of infusions, no infusion site reactions were observed and only two (0.3%) reactions were deemed severe. The incidence of site reactions decreased with successive infusions. Conclusion: The new 16.5% SCIG octanorm was shown to be efficacious in preventing infections in PIDs, and was well tolerated.
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spelling pubmed-63693542019-02-18 Clinical Efficacy, Safety and Tolerability of a New Subcutaneous Immunoglobulin 16.5% (Octanorm [Cutaquig®]) in the Treatment of Patients With Primary Immunodeficiencies Kobayashi, Roger H. Gupta, Sudhir Melamed, Isaac Mandujano, J. Fernando Kobayashi, Ai Lan Ritchie, Bruce Geng, Bob Atkinson, Thomas Prescott Rehman, Syed Turpel-Kantor, Eva Litzman, Jiří Front Immunol Immunology Introduction: Subcutaneously administered immunoglobulin (SCIG) is increasingly used to treat patients with primary immunodeficiencies (PIDs). Octanorm (marketed as cutaquig® in USA and Canada) is a new 16.5% solution of human SCIG, manufactured by a process based on that of the intravenous preparation (IVIG) octagam®. Objectives: To investigate the efficacy, safety and tolerability of octanorm in a prospective, open-label, single-arm phase 3 study involving adult and pediatric patients with PIDs (NCT01888484; clinicaltrials.gov/ct2/show/NCT01888484). Methods: Patients who were previously treated with IVIG received a total of 64 weekly SCIG infusions, including 12 weekly infusions during the wash-in/wash-out period, followed by 52 weekly infusions during the evaluation period. Results: A total of 61 patients aged 2–73 years received 3,497 infusions of octanorm. The mean dose per patient was 0.175 g/kg/infusion. The mean calculated dose conversion factor from the patients' previous IVIG dose for octanorm was 1.37. No serious bacterial infections developed during the study. The rate of other infections per person-year during the primary observation period was 3.43 (upper 95% CI 4.57). All but one non-bacterial infection were mild or moderate in intensity. IgG trough levels were constant during the course of the study. Eleven patients (18.0%) experienced 14 mild or moderate systemic adverse events (AEs) related to octanorm. The rate of related AEs per infusion was 0.004. In 76.7% of infusions, no infusion site reactions were observed and only two (0.3%) reactions were deemed severe. The incidence of site reactions decreased with successive infusions. Conclusion: The new 16.5% SCIG octanorm was shown to be efficacious in preventing infections in PIDs, and was well tolerated. Frontiers Media S.A. 2019-02-04 /pmc/articles/PMC6369354/ /pubmed/30778345 http://dx.doi.org/10.3389/fimmu.2019.00040 Text en Copyright © 2019 Kobayashi, Gupta, Melamed, Mandujano, Kobayashi, Ritchie, Geng, Atkinson, Rehman, Turpel-Kantor and Litzman. https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
spellingShingle Immunology
Kobayashi, Roger H.
Gupta, Sudhir
Melamed, Isaac
Mandujano, J. Fernando
Kobayashi, Ai Lan
Ritchie, Bruce
Geng, Bob
Atkinson, Thomas Prescott
Rehman, Syed
Turpel-Kantor, Eva
Litzman, Jiří
Clinical Efficacy, Safety and Tolerability of a New Subcutaneous Immunoglobulin 16.5% (Octanorm [Cutaquig®]) in the Treatment of Patients With Primary Immunodeficiencies
title Clinical Efficacy, Safety and Tolerability of a New Subcutaneous Immunoglobulin 16.5% (Octanorm [Cutaquig®]) in the Treatment of Patients With Primary Immunodeficiencies
title_full Clinical Efficacy, Safety and Tolerability of a New Subcutaneous Immunoglobulin 16.5% (Octanorm [Cutaquig®]) in the Treatment of Patients With Primary Immunodeficiencies
title_fullStr Clinical Efficacy, Safety and Tolerability of a New Subcutaneous Immunoglobulin 16.5% (Octanorm [Cutaquig®]) in the Treatment of Patients With Primary Immunodeficiencies
title_full_unstemmed Clinical Efficacy, Safety and Tolerability of a New Subcutaneous Immunoglobulin 16.5% (Octanorm [Cutaquig®]) in the Treatment of Patients With Primary Immunodeficiencies
title_short Clinical Efficacy, Safety and Tolerability of a New Subcutaneous Immunoglobulin 16.5% (Octanorm [Cutaquig®]) in the Treatment of Patients With Primary Immunodeficiencies
title_sort clinical efficacy, safety and tolerability of a new subcutaneous immunoglobulin 16.5% (octanorm [cutaquig®]) in the treatment of patients with primary immunodeficiencies
topic Immunology
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6369354/
https://www.ncbi.nlm.nih.gov/pubmed/30778345
http://dx.doi.org/10.3389/fimmu.2019.00040
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