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

Frontiers in Pediatrics

Pediatrics

Marie-Hélène André-Bonnet¹  Kalaivani Ruhier¹  Malika Barthez-Toullec¹  Aude Marie-Cardine²  Françoise Mazingue³  Jean-Louis Stephan⁴  Coralie Mallebranche⁵  Nizar Mahlaoui⁶  Nathalie Aladjidi⁷  Fanny Fouyssac⁸  Lamia Denti⁹ 

[1] Clinical Development and Medical Affairs Unit, Scientific, Medical and Regulatory Affairs Department, Laboratoire Français du Fractionnement et des Biotechnologies (LFB), Les Ulis, France;Department of Pediatric Hematology and Oncology, Rouen University Hospital, Rouen, France;Department of Pediatric Hematology-Oncology, CHRU Lille, Lille, France;Department of Pediatric Oncology, University Hospital of Saint Etienne, North Hospital, Saint Etienne, France;Pediatric Immuno-Hemato-Oncology Unit, Angers University Hospital, Angers, France;Pediatric Immunology Hematology and Rheumatology Unit, Necker University Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France;French National Reference Center for Primary Immune Deficiencies (CEREDIH), Necker University Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France;Pediatric Oncology Hematology Unit, University Hospital, Bordeaux, France;Pediatric Oncology and Hematology Unit, Children Hospital, Vandoeuvre-les-Nancy, France;Pharmacovigilance Unit, Scientific, Medical and Regulatory Affairs Department, Laboratoire Français du Fractionnement et des Biotechnologies (LFB), Les Ulis, France;

关键词: immunoglobulin;    primary immunodeficiency;    Clairyg®;    post-approval safety study;    real-world experience;    pediatrics;    immune thrombocytopenia;   

DOI  :  10.3389/fped.2023.1260296

 received in 2023-07-17, accepted in 2023-09-12,  发布年份 2023

来源: Frontiers

【 摘 要 】

IntroductionThis 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.MethodsThe 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.ResultsA 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.ConclusionClairyg® 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.

【 授权许可】

Unknown   
© 2023 Mahlaoui, Fouyssac, Mazingue, Mallebranche, Barthez-Toullec, Denti, Ruhier, André-Bonnet, Marie-Cardine, Aladjidi and Stephan.