Therapeutic management and costs of severe haemophilia A patients with inhibitors in Italy

F. Abbonizio, A. Giampaolo, A. Coppola, R. Arcieri, H. J. Hassan, Arianna Accorsi, Pietro Cosimo Ettorre, Paola Giordano, Giuseppina Rodorigo, Lelia Valdre, Lucia Notarangelo, Anna Brigida Aru, Dorina Cultrera, Piergiorgio Iannaccaro, Biasoli Chiara, Patrizia Di Gregorio, Vincenza Rossi, Sophie Testa, Maria Luisa Serino, Massimo MorfiniAngelo Claudio Molinari, G. Grazia Delios, Carlo Ciabatta, Flora Peyvandi, Elena Santagostino, Marco Marietta, Giovanni Di Minno, Corrado Perricone, Michele Schiavulli, Angiola Rocino, Ezio Zanon, Fabio Gagliano, Sergio Siragusa, Annarita Tagliaferri, Gianna Franca Rivolta, Gabriella Gamba, Paolo Gresele, Emily Oliovecchio, Alfredo Dragani, Maria Cristina Arbasi, Silvia Macchi, Patrizia Albertini, Caterina Latella, Marco D'Incà, Matteo Luciani, Raimondo De Cristofaro, Maria Gabriella Mazzucconi, Cristina Santoro, Gavino Piseddu, Mario Schiavoni, Pasquale Spagnuolo, Pier Carla Schinco, Maria Messina, Gina Rossetti, Attilio Fabio Cristallo, Giovanni Barillari, Giulio Feola, Simone Cesaro, Elisa Bonetti, Giorgio Gandini, Giancarlo Castaman, Francesco Rodeghiero

Research output: Contribution to journalArticle

11 Citations (Scopus)

Abstract

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.

Original languageEnglish
JournalHaemophilia
Volume20
Issue number4
DOIs
Publication statusPublished - 2014

Fingerprint

Hemophilia A
Italy
Immune Tolerance
Costs and Cost Analysis
Therapeutics
Registries

Keywords

  • aPCC
  • Bypassing agents
  • Haemophilia A
  • Immune tolerance induction
  • Inhibitors
  • rFVIIa

ASJC Scopus subject areas

  • Hematology
  • Genetics(clinical)
  • Medicine(all)

Cite this

Abbonizio, F., Giampaolo, A., Coppola, A., Arcieri, R., Hassan, H. J., Accorsi, A., ... Rodeghiero, F. (2014). Therapeutic management and costs of severe haemophilia A patients with inhibitors in Italy. Haemophilia, 20(4). https://doi.org/10.1111/hae.12456

Therapeutic management and costs of severe haemophilia A patients with inhibitors in Italy. / Abbonizio, F.; Giampaolo, A.; Coppola, A.; Arcieri, R.; Hassan, H. J.; Accorsi, Arianna; Ettorre, Pietro Cosimo; Giordano, Paola; Rodorigo, Giuseppina; Valdre, Lelia; Notarangelo, Lucia; Aru, Anna Brigida; Cultrera, Dorina; Iannaccaro, Piergiorgio; Chiara, Biasoli; Di Gregorio, Patrizia; Rossi, Vincenza; Testa, Sophie; Serino, Maria Luisa; Morfini, Massimo; Molinari, Angelo Claudio; Delios, G. Grazia; Ciabatta, Carlo; Peyvandi, Flora; Santagostino, Elena; Marietta, Marco; Di Minno, Giovanni; Perricone, Corrado; Schiavulli, Michele; Rocino, Angiola; Zanon, Ezio; Gagliano, Fabio; Siragusa, Sergio; Tagliaferri, Annarita; Rivolta, Gianna Franca; Gamba, Gabriella; Gresele, Paolo; Oliovecchio, Emily; Dragani, Alfredo; Arbasi, Maria Cristina; Macchi, Silvia; Albertini, Patrizia; Latella, Caterina; D'Incà, Marco; Luciani, Matteo; De Cristofaro, Raimondo; Mazzucconi, Maria Gabriella; Santoro, Cristina; Piseddu, Gavino; Schiavoni, Mario; Spagnuolo, Pasquale; Schinco, Pier Carla; Messina, Maria; Rossetti, Gina; Cristallo, Attilio Fabio; Barillari, Giovanni; Feola, Giulio; Cesaro, Simone; Bonetti, Elisa; Gandini, Giorgio; Castaman, Giancarlo; Rodeghiero, Francesco.

In: Haemophilia, Vol. 20, No. 4, 2014.

Research output: Contribution to journalArticle

Abbonizio, F, Giampaolo, A, Coppola, A, Arcieri, R, Hassan, HJ, Accorsi, A, Ettorre, PC, Giordano, P, Rodorigo, G, Valdre, L, Notarangelo, L, Aru, AB, Cultrera, D, Iannaccaro, P, Chiara, B, Di Gregorio, P, Rossi, V, Testa, S, Serino, ML, Morfini, M, Molinari, AC, Delios, GG, Ciabatta, C, Peyvandi, F, Santagostino, E, Marietta, M, Di Minno, G, Perricone, C, Schiavulli, M, Rocino, A, Zanon, E, Gagliano, F, Siragusa, S, Tagliaferri, A, Rivolta, GF, Gamba, G, Gresele, P, Oliovecchio, E, Dragani, A, Arbasi, MC, Macchi, S, Albertini, P, Latella, C, D'Incà, M, Luciani, M, De Cristofaro, R, Mazzucconi, MG, Santoro, C, Piseddu, G, Schiavoni, M, Spagnuolo, P, Schinco, PC, Messina, M, Rossetti, G, Cristallo, AF, Barillari, G, Feola, G, Cesaro, S, Bonetti, E, Gandini, G, Castaman, G & Rodeghiero, F 2014, 'Therapeutic management and costs of severe haemophilia A patients with inhibitors in Italy', Haemophilia, vol. 20, no. 4. https://doi.org/10.1111/hae.12456
Abbonizio F, Giampaolo A, Coppola A, Arcieri R, Hassan HJ, Accorsi A et al. Therapeutic management and costs of severe haemophilia A patients with inhibitors in Italy. Haemophilia. 2014;20(4). https://doi.org/10.1111/hae.12456
Abbonizio, F. ; Giampaolo, A. ; Coppola, A. ; Arcieri, R. ; Hassan, H. J. ; Accorsi, Arianna ; Ettorre, Pietro Cosimo ; Giordano, Paola ; Rodorigo, Giuseppina ; Valdre, Lelia ; Notarangelo, Lucia ; Aru, Anna Brigida ; Cultrera, Dorina ; Iannaccaro, Piergiorgio ; Chiara, Biasoli ; Di Gregorio, Patrizia ; Rossi, Vincenza ; Testa, Sophie ; Serino, Maria Luisa ; Morfini, Massimo ; Molinari, Angelo Claudio ; Delios, G. Grazia ; Ciabatta, Carlo ; Peyvandi, Flora ; Santagostino, Elena ; Marietta, Marco ; Di Minno, Giovanni ; Perricone, Corrado ; Schiavulli, Michele ; Rocino, Angiola ; Zanon, Ezio ; Gagliano, Fabio ; Siragusa, Sergio ; Tagliaferri, Annarita ; Rivolta, Gianna Franca ; Gamba, Gabriella ; Gresele, Paolo ; Oliovecchio, Emily ; Dragani, Alfredo ; Arbasi, Maria Cristina ; Macchi, Silvia ; Albertini, Patrizia ; Latella, Caterina ; D'Incà, Marco ; Luciani, Matteo ; De Cristofaro, Raimondo ; Mazzucconi, Maria Gabriella ; Santoro, Cristina ; Piseddu, Gavino ; Schiavoni, Mario ; Spagnuolo, Pasquale ; Schinco, Pier Carla ; Messina, Maria ; Rossetti, Gina ; Cristallo, Attilio Fabio ; Barillari, Giovanni ; Feola, Giulio ; Cesaro, Simone ; Bonetti, Elisa ; Gandini, Giorgio ; Castaman, Giancarlo ; Rodeghiero, Francesco. / Therapeutic management and costs of severe haemophilia A patients with inhibitors in Italy. In: Haemophilia. 2014 ; Vol. 20, No. 4.
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AU - Abbonizio, F.

AU - Giampaolo, A.

AU - Coppola, A.

AU - Arcieri, R.

AU - Hassan, H. J.

AU - Accorsi, Arianna

AU - Ettorre, Pietro Cosimo

AU - Giordano, Paola

AU - Rodorigo, Giuseppina

AU - Valdre, Lelia

AU - Notarangelo, Lucia

AU - Aru, Anna Brigida

AU - Cultrera, Dorina

AU - Iannaccaro, Piergiorgio

AU - Chiara, Biasoli

AU - Di Gregorio, Patrizia

AU - Rossi, Vincenza

AU - Testa, Sophie

AU - Serino, Maria Luisa

AU - Morfini, Massimo

AU - Molinari, Angelo Claudio

AU - Delios, G. Grazia

AU - Ciabatta, Carlo

AU - Peyvandi, Flora

AU - Santagostino, Elena

AU - Marietta, Marco

AU - Di Minno, Giovanni

AU - Perricone, Corrado

AU - Schiavulli, Michele

AU - Rocino, Angiola

AU - Zanon, Ezio

AU - Gagliano, Fabio

AU - Siragusa, Sergio

AU - Tagliaferri, Annarita

AU - Rivolta, Gianna Franca

AU - Gamba, Gabriella

AU - Gresele, Paolo

AU - Oliovecchio, Emily

AU - Dragani, Alfredo

AU - Arbasi, Maria Cristina

AU - Macchi, Silvia

AU - Albertini, Patrizia

AU - Latella, Caterina

AU - D'Incà, Marco

AU - Luciani, Matteo

AU - De Cristofaro, Raimondo

AU - Mazzucconi, Maria Gabriella

AU - Santoro, Cristina

AU - Piseddu, Gavino

AU - Schiavoni, Mario

AU - Spagnuolo, Pasquale

AU - Schinco, Pier Carla

AU - Messina, Maria

AU - Rossetti, Gina

AU - Cristallo, Attilio Fabio

AU - Barillari, Giovanni

AU - Feola, Giulio

AU - Cesaro, Simone

AU - Bonetti, Elisa

AU - Gandini, Giorgio

AU - Castaman, Giancarlo

AU - Rodeghiero, Francesco

PY - 2014

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AB - 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.

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