Treatment Regimens with Bypassing Agents in Patients with Hemophilia A and Inhibitors: A Survey from the Italian Association of Hemophilia Centers (AICE)

Antonio Coppola; Massimo Franchini; Giancarlo Castaman; Elena Santagostino; Cristina Santoro; Rita Carlotta Santoro; Massimo Morfini; Giovanni Di Minno; Angiola Rocino; on behalf of the AICE ad hoc Working Group

doi:10.1055/s-0038-1648230

Seminars in Thrombosis and Hemostasis, Table of Contents

Semin Thromb Hemost 2018; 44(06): 551-560
DOI: 10.1055/s-0038-1648230

Review Article

Thieme Medical Publishers 333 Seventh Avenue, New York, NY 10001, USA.

Treatment Regimens with Bypassing Agents in Patients with Hemophilia A and Inhibitors: A Survey from the Italian Association of Hemophilia Centers (AICE)

Antonio Coppola

¹Hemophilia and Congenital Bleeding Disorders Hub Center, University Hospital of Parma, Parma, Italy

,

Massimo Franchini

²Department of Transfusion Medicine and Hematology, Carlo Poma Hospital, Mantova, Italy

,

Giancarlo Castaman

³Center for Bleeding Disorders, Careggi University Hospital, Florence, Italy

,

Elena Santagostino

⁴Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, IRCCS Ca' Granda Foundation, Maggiore Hospital Policlinico, Milan, Italy

,

Cristina Santoro

⁵Hematology Unit, Department of Cellular Biotechnology and Hematology, Sapienza University of Rome, Rome, Italy

,

Rita Carlotta Santoro

⁶Hemophilia and Thrombosis Center, Pugliese Ciaccio Hospital, Catanzaro, Italy

,

Massimo Morfini

⁷Past President of the Italian Association of Hemophilia Centers (AICE), Italy

,

Giovanni Di Minno

⁸Regional Reference Center for Coagulation Disorders, Federico II University Hospital, Naples, Italy

,

Angiola Rocino

⁹Hemophilia and Thrombosis Center, Hematology Unit, Ascalesi Hospital, Naples, Italy

,

on behalf of the AICE ad hoc Working Group

› Author Affiliations

Abstract

The development of neutralizing antibodies (inhibitors) against infused factor VIII currently represents the main complication of replacement therapy in patients with severe hemophilia A. Inhibitors, indeed, particularly high-titer inhibitors (>5 BU/mL), greatly complicate the management of bleeding, exposing patients to an increased morbidity and mortality risk, thus representing a significant burden for physicians of Hemophilia Treatment Centers (HTCs). Although bypassing agents (i.e., activated prothrombin complex concentrate [APCC] and recombinant activated factor VII [rFVIIa]) are available for the treatment and prevention of bleeding in inhibitor patients, their efficacy, safety, and cost–benefit outcomes are poorly known in the long term and should be further improved. In the frame of the update of recommendations for the management of inhibitor patients by the Italian Association of Hemophilia Centers (AICE), to collect more information on real-life therapeutic approaches with bypassing agents in this setting, a survey was conducted among the Directors of the Italian HTCs. From questionnaires returned by 55% of them, data on the use of rFVIIa and APCC in children, adolescent, and adult patients with hemophilia A and inhibitors were obtained and are summarized in this article, including information about the implementation of prophylaxis with both bypassing agents, the adopted regimens, and reasons for starting, adjusting, and interrupting such a therapeutic approach.

Keywords

hemophilia A - inhibitors - prophylaxis - bypassing agents - activated prothrombin complex concentrate - recombinant activated factor VII

Full Text

References

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