Original article

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A multicenter, real-world experience with recombinant FXIII for the treatment of patients with FXIII deficiency: from pharmacokinetics to clinical practice. The Italian FXIII Study

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Key words: FXIII deficiency, recombinant FXIII, NovoThrirteen, rare bleeding disorders, pharmacokinetics
Publication Date: 2022-11-18

Abstract

Background - Congenital factor XIII (FXIII) deficiency is a rare coagulation disorder characterized by muscular or mucocutaneous bleeding with life-threatening intracranial hemorrhages (ICHs), especially in cases with severe disease. The best treatment is the use of prophylactic plasma-derived or recombinant FXIII (rFXIII). Few data on the use of rFXIII in the real-world scenario are available. The main goal of this study was to assess the efficacy and safety of catridecacog (NovoThirteen®) in a population of patients with FXIII deficiency. Other objectives were to compare the different pharmacokinetic (PK) profiles of each patient and to use them to create a tailored prophylaxis regimen.
Materials and methods - We collected and analyzed all pharmacokinetic and clinical data in our registry of the patients with congenital FXIII deficiency treated with rFXIII at eleven Italian hemophilia centers. Data were collected from January 2019 to December 2020.
Results - Overall, data on 20 patients with FXIII deficiency were collected, 16 of whom presented with severe disease. Pharmacokinetics was assessed in 18 cases before starting prophylaxis. Prophylaxis was subsequently started in these patients using a wide range of dosages (25.0-80.0 IU/kg; mean 33.8 IU/kg) and infusion intervals (3.0-8.0 weeks). During a mean follow up of 47 months, two minor bleeds and one ICH in a severe patient who had remained under on-demand treatment were reported.
Discussion - Efficacy and safety of rFXIII were proven in all patients. The dosage and infusion timing for the treated patients sometimes differed to those reported in the MENTOR pivotal studies, thus underlying the importance of tailored management in a real-world scenario.

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Authors

Ezio Zanon - General Medicine, Padua University Hospital, Padua, Italy

Samantha Pasca - Biological Sciences Department (DSB), Padua University Hospital, Padua, Italy

Gianluca Sottilotta - Department of Onco-Hematology and Radioterapy, Hospital of Reggio Calabria, Reggio Calabria, Italy

Angelo C. Molinari - Thrombosis and Hemostasis Unit, "Gianna Gaslini" Children Hospital, Genoa, Italy

Antonietta Ferretti - Haemorrhagic and Thrombotic Diseases Unit, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy

Patrizia Di Gregorio - Transfusion Medicine Department, "SS. Annunziata" Hospital, Chieti, Italy

Berardino Pollio - Transfusion Medicine, Dept. of Diagnostic, "Regina Margherita" Children Hospital, Turin, Italy

Michele Pizzuti - Hematology Department, "San Carlo" Hospital, Potenza, Italy

Lucia Dora Notarangelo - Pediatric Onco-Hematology, ASST Spedali Civili , Brescia, Italy

Chiara Biasoli - Transfusion Medicine, Department of Clinical Pathology, Hospital of Cesena, Cesena, Italy

Piergiorgio Cojutti - SSD Clinical Pharmacology, IRCCS Azienda Ospedaliero Universitaria , Bologna, Italy

Federico Pea - SSD Clinical Pharmacology, IRCCS Azienda Ospedaliero Universitaria , Bologna, Italy

Paolo Simioni - General Medicine, Padua University Hospital, Padua, Italy

Flora Peyvandi - Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy

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