Therapeutic management and costs of severe haemophilia A patients with inhibitors in Italy. (19th May 2014)

• Record Type: Journal Article
• Authors: Abbonizio, F.; Giampaolo, A.; Coppola, A.; Arcieri, R.; Hassan, H. J.
• Other Names: Accorsi Arianna investigator.; Ettorre Pietro Cosimo investigator.; Giordano Paola investigator.; Rodorigo Giuseppina investigator.; Valdrè Lelia investigator.; Notarangelo Lucia investigator.; Aru Anna Brigida investigator.; Cultrera Dorina investigator.; Iannaccaro Piergiorgio investigator.; Biasoli Chiara investigator.; Gregorio Patrizia Di investigator.; Rossi Vincenza investigator.; Testa Sophie investigator.; Serino Maria Luisa investigator.; Morfini Massimo investigator.; Molinari Angelo Claudio investigator.; Delios Grazia investigator.; Ciabatta Carlo investigator.; Peyvandi Flora investigator.; Santagostino Elena investigator.; Marietta Marco investigator.; Minno Giovanni Di investigator.; Perricone Corrado investigator.; Schiavulli Michele investigator.; Rocino Angiola investigator.; Zanon Ezio investigator.; Gagliano Fabio investigator.; Siragusa Sergio investigator.; Tagliaferri Annarita investigator.; Rivolta Gianna Franca investigator.; Gamba Gabriella investigator.; Gresele Paolo investigator.; Oliovecchio Emily investigator.; Dragani Alfredo investigator.; Arbasi Maria Cristina investigator.; Macchi Silvia investigator.; Albertini Patrizia investigator.; Latella Caterina investigator.; D'Incà Marco investigator.; Luciani Matteo investigator.; De Cristofaro Raimondo investigator.; Mazzucconi Maria Gabriella investigator.; Santoro Cristina investigator.; Piseddu Gavino investigator.; Schiavoni Mario investigator.; Spagnuolo Pasquale investigator.; Schinco Pier Carla investigator.; Messina Maria investigator.; Rossetti Gina investigator.; Cristallo Attilio Fabio investigator.; Barillari Giovanni investigator.; Feola Giulio investigator.; Cesaro Simone investigator.; Bonetti Elisa investigator.; Gandini Giorgio investigator.; Castaman Giancarlo investigator.; Rodeghiero Francesco investigator.;
• Is Part Of: Haemophilia. Volume 20:Number 4(2014:Jul.)
• Page Start: e243
• Page End: e250
• Publication Date: 2014-05-19
• Subjects: aPCC -- bypassing agents -- haemophilia A -- immune tolerance induction -- inhibitors -- rFVIIa; Hemophilia -- Periodicals; 616.1572005
• DOI: 10.1111/hae.12456
• Languages: English
• ISSNs: 1351-8216

Abstract

Summary: Haemophilia A (HA) patients with high responding inhibitors require therapies with bypassing agents to control bleedings or Immune Tolerance Induction (ITI) to attempt inhibitor eradication and restore FVIII therapy. The aim of this study was to assess the therapeutic management and product consumption of HA inhibitor patients and the relative costs in Italy. A retrospective survey was performed utilizing data from the National Registry of Congenital Coagulopathies and from a specific questionnaire on product consumption of HA inhibitor patients over the year 2011. Among HA patients, 10% had currently detectable inhibitors; 24% of patients were undergoing ITI (mostly children) and 76% utilized bypassing agents. Patients on ITI consumed 45 000 000 IU of FVIII (median consumption/patient of 1 200 000 IU year −1 ). Patients receiving bypassing agents utilized 21 000 000 IU of aPCC (median consumption/patient of 360 000 IU year −1 ), and 38 000 mg of rFVIIa (median consumption/patient of 440 mg year −1 ). The annual cost/patient on ITI and on bypassing agents therapy was analysed. Recombinant products represen‐ted the product of choice for children therapies in >90% of the cases. FVIII prophylaxis of severe HA patients without inhibitor costs about half than therapy with bypassing agents and is three times less expensive than prophylaxis with such agents. Therefore, the possibility to restore FVIII prophylaxis, having eradicated the inhibitor through ITI, can justify the high costs of ITI treatment needed in the short term. Consistent with this notion, over the last years a 50% increase in the number of patients undergoing ITI in Italy was registered.