Cargando…

Real-world experience with CLAIRYG® 50 mg/mL (intravenous immunoglobulin) in children under 12 years with primary immunodeficiency or immmune thrombocytopenia: a post-approval safety study

INTRODUCTION: This study presents the results of a real-life, multicenter, prospective, post-approval safety evaluation of Clairyg® 50 mg/mL, a 5% intravenous immunoglobulin (IVIg) liquid, in 59 children (aged < 12 years) with primary immunodeficiency diseases (PID) (n = 32) or immune thrombocyto...

Descripción completa

Detalles Bibliográficos
Autores principales: Mahlaoui, Nizar, Fouyssac, Fanny, Mazingue, Françoise, Mallebranche, Coralie, Barthez-Toullec, Malika, Denti, Lamia, Ruhier, Kalaivani, André-Bonnet, Marie-Hélène, Marie-Cardine, Aude, Aladjidi, Nathalie, Stephan, Jean-Louis
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10577179/
https://www.ncbi.nlm.nih.gov/pubmed/37849499
http://dx.doi.org/10.3389/fped.2023.1260296
_version_ 1785121268980252672
author Mahlaoui, Nizar
Fouyssac, Fanny
Mazingue, Françoise
Mallebranche, Coralie
Barthez-Toullec, Malika
Denti, Lamia
Ruhier, Kalaivani
André-Bonnet, Marie-Hélène
Marie-Cardine, Aude
Aladjidi, Nathalie
Stephan, Jean-Louis
author_facet Mahlaoui, Nizar
Fouyssac, Fanny
Mazingue, Françoise
Mallebranche, Coralie
Barthez-Toullec, Malika
Denti, Lamia
Ruhier, Kalaivani
André-Bonnet, Marie-Hélène
Marie-Cardine, Aude
Aladjidi, Nathalie
Stephan, Jean-Louis
author_sort Mahlaoui, Nizar
collection PubMed
description INTRODUCTION: This study presents the results of a real-life, multicenter, prospective, post-approval safety evaluation of Clairyg® 50 mg/mL, a 5% intravenous immunoglobulin (IVIg) liquid, in 59 children (aged < 12 years) with primary immunodeficiency diseases (PID) (n = 32) or immune thrombocytopenia (ITP) (n = 27) in France. METHODS: The primary objective of the study was to assess the safety and tolerability of Clairyg®, recording all serious and non-serious adverse events (AEs), whether related (rAEs) or not related to the product. Secondary objectives aimed at evaluating the administration of Clairyg® under routine conditions and the available efficacy data to better document the benefit/risk ratio in this pediatric population. An exploratory objective was added to evaluate the potential factors associated with the occurrence of rAEs. Patients received Clairyg® according to the approved dosage under normal conditions of prescriptions over a median follow-up period of 11.8 months. RESULTS: A total of 549 infusions (PID: n = 464 and ITP: n = 85), were administered, of which 58.8% were preceded by premedication. The most frequent rAEs were headache, vomiting, and pyrexia in both indications. Most of them were considered non-serious and mild or moderate in intensity. A severe single rAE was observed (aseptic meningitis) in a 4-year-old girl presenting with chronic ITP. The exploratory multivariate analysis of potential co-factors showed that the occurrence of rAEs is significantly linked to high IVIg doses and possibly to female gender. The annualized rate of serious bacterial infections was 0.11 for patients with PID. For patients with ITP, 74.1% experienced at least one bleeding episode during the follow-up, mostly a cutaneous one, and none had gastrointestinal, genitourinary, or central nervous system bleeding. CONCLUSION: Clairyg® was well tolerated and allowed for control of serious bacterial infection in PID and serious bleeding in ITP, which are the main complications in these respective pediatric disorders. No new safety signal was detected in children less than 12 years-old in real-life conditions of use.
format Online
Article
Text
id pubmed-10577179
institution National Center for Biotechnology Information
language English
publishDate 2023
publisher Frontiers Media S.A.
record_format MEDLINE/PubMed
spelling pubmed-105771792023-10-17 Real-world experience with CLAIRYG® 50 mg/mL (intravenous immunoglobulin) in children under 12 years with primary immunodeficiency or immmune thrombocytopenia: a post-approval safety study Mahlaoui, Nizar Fouyssac, Fanny Mazingue, Françoise Mallebranche, Coralie Barthez-Toullec, Malika Denti, Lamia Ruhier, Kalaivani André-Bonnet, Marie-Hélène Marie-Cardine, Aude Aladjidi, Nathalie Stephan, Jean-Louis Front Pediatr Pediatrics INTRODUCTION: This study presents the results of a real-life, multicenter, prospective, post-approval safety evaluation of Clairyg® 50 mg/mL, a 5% intravenous immunoglobulin (IVIg) liquid, in 59 children (aged < 12 years) with primary immunodeficiency diseases (PID) (n = 32) or immune thrombocytopenia (ITP) (n = 27) in France. METHODS: The primary objective of the study was to assess the safety and tolerability of Clairyg®, recording all serious and non-serious adverse events (AEs), whether related (rAEs) or not related to the product. Secondary objectives aimed at evaluating the administration of Clairyg® under routine conditions and the available efficacy data to better document the benefit/risk ratio in this pediatric population. An exploratory objective was added to evaluate the potential factors associated with the occurrence of rAEs. Patients received Clairyg® according to the approved dosage under normal conditions of prescriptions over a median follow-up period of 11.8 months. RESULTS: A total of 549 infusions (PID: n = 464 and ITP: n = 85), were administered, of which 58.8% were preceded by premedication. The most frequent rAEs were headache, vomiting, and pyrexia in both indications. Most of them were considered non-serious and mild or moderate in intensity. A severe single rAE was observed (aseptic meningitis) in a 4-year-old girl presenting with chronic ITP. The exploratory multivariate analysis of potential co-factors showed that the occurrence of rAEs is significantly linked to high IVIg doses and possibly to female gender. The annualized rate of serious bacterial infections was 0.11 for patients with PID. For patients with ITP, 74.1% experienced at least one bleeding episode during the follow-up, mostly a cutaneous one, and none had gastrointestinal, genitourinary, or central nervous system bleeding. CONCLUSION: Clairyg® was well tolerated and allowed for control of serious bacterial infection in PID and serious bleeding in ITP, which are the main complications in these respective pediatric disorders. No new safety signal was detected in children less than 12 years-old in real-life conditions of use. Frontiers Media S.A. 2023-10-02 /pmc/articles/PMC10577179/ /pubmed/37849499 http://dx.doi.org/10.3389/fped.2023.1260296 Text en © 2023 Mahlaoui, Fouyssac, Mazingue, Mallebranche, Barthez-Toullec, Denti, Ruhier, André-Bonnet, Marie-Cardine, Aladjidi and Stephan. 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) (https://creativecommons.org/licenses/by/4.0/) . 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 Pediatrics
Mahlaoui, Nizar
Fouyssac, Fanny
Mazingue, Françoise
Mallebranche, Coralie
Barthez-Toullec, Malika
Denti, Lamia
Ruhier, Kalaivani
André-Bonnet, Marie-Hélène
Marie-Cardine, Aude
Aladjidi, Nathalie
Stephan, Jean-Louis
Real-world experience with CLAIRYG® 50 mg/mL (intravenous immunoglobulin) in children under 12 years with primary immunodeficiency or immmune thrombocytopenia: a post-approval safety study
title Real-world experience with CLAIRYG® 50 mg/mL (intravenous immunoglobulin) in children under 12 years with primary immunodeficiency or immmune thrombocytopenia: a post-approval safety study
title_full Real-world experience with CLAIRYG® 50 mg/mL (intravenous immunoglobulin) in children under 12 years with primary immunodeficiency or immmune thrombocytopenia: a post-approval safety study
title_fullStr Real-world experience with CLAIRYG® 50 mg/mL (intravenous immunoglobulin) in children under 12 years with primary immunodeficiency or immmune thrombocytopenia: a post-approval safety study
title_full_unstemmed Real-world experience with CLAIRYG® 50 mg/mL (intravenous immunoglobulin) in children under 12 years with primary immunodeficiency or immmune thrombocytopenia: a post-approval safety study
title_short Real-world experience with CLAIRYG® 50 mg/mL (intravenous immunoglobulin) in children under 12 years with primary immunodeficiency or immmune thrombocytopenia: a post-approval safety study
title_sort real-world experience with clairyg® 50 mg/ml (intravenous immunoglobulin) in children under 12 years with primary immunodeficiency or immmune thrombocytopenia: a post-approval safety study
topic Pediatrics
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10577179/
https://www.ncbi.nlm.nih.gov/pubmed/37849499
http://dx.doi.org/10.3389/fped.2023.1260296
work_keys_str_mv AT mahlaouinizar realworldexperiencewithclairyg50mgmlintravenousimmunoglobulininchildrenunder12yearswithprimaryimmunodeficiencyorimmmunethrombocytopeniaapostapprovalsafetystudy
AT fouyssacfanny realworldexperiencewithclairyg50mgmlintravenousimmunoglobulininchildrenunder12yearswithprimaryimmunodeficiencyorimmmunethrombocytopeniaapostapprovalsafetystudy
AT mazinguefrancoise realworldexperiencewithclairyg50mgmlintravenousimmunoglobulininchildrenunder12yearswithprimaryimmunodeficiencyorimmmunethrombocytopeniaapostapprovalsafetystudy
AT mallebranchecoralie realworldexperiencewithclairyg50mgmlintravenousimmunoglobulininchildrenunder12yearswithprimaryimmunodeficiencyorimmmunethrombocytopeniaapostapprovalsafetystudy
AT bartheztoullecmalika realworldexperiencewithclairyg50mgmlintravenousimmunoglobulininchildrenunder12yearswithprimaryimmunodeficiencyorimmmunethrombocytopeniaapostapprovalsafetystudy
AT dentilamia realworldexperiencewithclairyg50mgmlintravenousimmunoglobulininchildrenunder12yearswithprimaryimmunodeficiencyorimmmunethrombocytopeniaapostapprovalsafetystudy
AT ruhierkalaivani realworldexperiencewithclairyg50mgmlintravenousimmunoglobulininchildrenunder12yearswithprimaryimmunodeficiencyorimmmunethrombocytopeniaapostapprovalsafetystudy
AT andrebonnetmariehelene realworldexperiencewithclairyg50mgmlintravenousimmunoglobulininchildrenunder12yearswithprimaryimmunodeficiencyorimmmunethrombocytopeniaapostapprovalsafetystudy
AT mariecardineaude realworldexperiencewithclairyg50mgmlintravenousimmunoglobulininchildrenunder12yearswithprimaryimmunodeficiencyorimmmunethrombocytopeniaapostapprovalsafetystudy
AT aladjidinathalie realworldexperiencewithclairyg50mgmlintravenousimmunoglobulininchildrenunder12yearswithprimaryimmunodeficiencyorimmmunethrombocytopeniaapostapprovalsafetystudy
AT stephanjeanlouis realworldexperiencewithclairyg50mgmlintravenousimmunoglobulininchildrenunder12yearswithprimaryimmunodeficiencyorimmmunethrombocytopeniaapostapprovalsafetystudy