PMID- 37849499 OWN - NLM STAT- PubMed-not-MEDLINE LR - 20231020 IS - 2296-2360 (Print) IS - 2296-2360 (Electronic) IS - 2296-2360 (Linking) VI - 11 DP - 2023 TI - Real-world experience with CLAIRYG(R) 50 mg/mL (intravenous immunoglobulin) in children under 12 years with primary immunodeficiency or immmune thrombocytopenia: a post-approval safety study. PG - 1260296 LID - 10.3389/fped.2023.1260296 [doi] LID - 1260296 AB - INTRODUCTION: This study presents the results of a real-life, multicenter, prospective, post-approval safety evaluation of Clairyg(R) 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(R), 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(R) 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(R) 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(R) 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. CI - (c) 2023 Mahlaoui, Fouyssac, Mazingue, Mallebranche, Barthez-Toullec, Denti, Ruhier, Andre-Bonnet, Marie-Cardine, Aladjidi and Stephan. FAU - Mahlaoui, Nizar AU - Mahlaoui N AD - Pediatric Immunology Hematology and Rheumatology Unit, Necker University Hospital, Assistance Publique-Hopitaux de Paris (AP-HP), Paris, France. AD - French National Reference Center for Primary Immune Deficiencies (CEREDIH), Necker University Hospital, Assistance Publique-Hopitaux de Paris (AP-HP), Paris, France. FAU - Fouyssac, Fanny AU - Fouyssac F AD - Pediatric Oncology and Hematology Unit, Children Hospital, Vandoeuvre-les-Nancy, France. FAU - Mazingue, Francoise AU - Mazingue F AD - Department of Pediatric Hematology-Oncology, CHRU Lille, Lille, France. FAU - Mallebranche, Coralie AU - Mallebranche C AD - Pediatric Immuno-Hemato-Oncology Unit, Angers University Hospital, Angers, France. FAU - Barthez-Toullec, Malika AU - Barthez-Toullec M AD - Clinical Development and Medical Affairs Unit, Scientific, Medical and Regulatory Affairs Department, Laboratoire Francais du Fractionnement et des Biotechnologies (LFB), Les Ulis, France. FAU - Denti, Lamia AU - Denti L AD - Pharmacovigilance Unit, Scientific, Medical and Regulatory Affairs Department, Laboratoire Francais du Fractionnement et des Biotechnologies (LFB), Les Ulis, France. FAU - Ruhier, Kalaivani AU - Ruhier K AD - Clinical Development and Medical Affairs Unit, Scientific, Medical and Regulatory Affairs Department, Laboratoire Francais du Fractionnement et des Biotechnologies (LFB), Les Ulis, France. FAU - Andre-Bonnet, Marie-Helene AU - Andre-Bonnet MH AD - Clinical Development and Medical Affairs Unit, Scientific, Medical and Regulatory Affairs Department, Laboratoire Francais du Fractionnement et des Biotechnologies (LFB), Les Ulis, France. FAU - Marie-Cardine, Aude AU - Marie-Cardine A AD - Department of Pediatric Hematology and Oncology, Rouen University Hospital, Rouen, France. FAU - Aladjidi, Nathalie AU - Aladjidi N AD - Pediatric Oncology Hematology Unit, University Hospital, Bordeaux, France. FAU - Stephan, Jean-Louis AU - Stephan JL AD - Department of Pediatric Oncology, University Hospital of Saint Etienne, North Hospital, Saint Etienne, France. LA - eng PT - Journal Article DEP - 20231002 PL - Switzerland TA - Front Pediatr JT - Frontiers in pediatrics JID - 101615492 PMC - PMC10577179 OTO - NOTNLM OT - Clairyg(R) OT - immune thrombocytopenia OT - immunoglobulin OT - pediatrics OT - post-approval safety study OT - primary immunodeficiency OT - real-world experience COIS- CEREDIH receives unrestricted grant from the following pharmaceutical companies: LFB biomedicaments, Grifols, Takeda, CSL Behring, Binding Site, Octapharma, Pharming, LVL medical. NM received honorarium for advisory boards, participation to symposia or production of educational material from LFB biomedicaments, Grifols, Takeda, CSL Behring, Octapharma, Pharming, LVL medical, X4 Pharma. MBT, LD, KR and MHAB are employees of LFB. The remaining authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest. EDAT- 2023/10/18 06:42 MHDA- 2023/10/18 06:43 PMCR- 2023/10/02 CRDT- 2023/10/18 03:51 PHST- 2023/07/17 00:00 [received] PHST- 2023/09/12 00:00 [accepted] PHST- 2023/10/18 06:43 [medline] PHST- 2023/10/18 06:42 [pubmed] PHST- 2023/10/18 03:51 [entrez] PHST- 2023/10/02 00:00 [pmc-release] AID - 10.3389/fped.2023.1260296 [doi] PST - epublish SO - Front Pediatr. 2023 Oct 2;11:1260296. doi: 10.3389/fped.2023.1260296. eCollection 2023.