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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
1   Hemophilia and Congenital Bleeding Disorders Hub Center, University Hospital of Parma, Parma, Italy
,
Massimo Franchini
2   Department of Transfusion Medicine and Hematology, Carlo Poma Hospital, Mantova, Italy
,
Giancarlo Castaman
3   Center for Bleeding Disorders, Careggi University Hospital, Florence, Italy
,
Elena Santagostino
4   Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, IRCCS Ca' Granda Foundation, Maggiore Hospital Policlinico, Milan, Italy
,
Cristina Santoro
5   Hematology Unit, Department of Cellular Biotechnology and Hematology, Sapienza University of Rome, Rome, Italy
,
Rita Carlotta Santoro
6   Hemophilia and Thrombosis Center, Pugliese Ciaccio Hospital, Catanzaro, Italy
,
Massimo Morfini
7   Past President of the Italian Association of Hemophilia Centers (AICE), Italy
,
Giovanni Di Minno
8   Regional Reference Center for Coagulation Disorders, Federico II University Hospital, Naples, Italy
,
Angiola Rocino
9   Hemophilia and Thrombosis Center, Hematology Unit, Ascalesi Hospital, Naples, Italy
,
on behalf of the AICE ad hoc Working Group› Institutsangaben
Funding Dr. Castaman reports personal fees from UniQure B.V., personal fees from Novo Nordisk, personal fees from Shire, personal fees from Sobi, grants and personal fees from CSL Behring, grants and personal fees from Pfizer, personal fees from Roche, personal fees from Kedrion, and personal fees from Bayer, outside the submitted work. Dr. Di Minno reports personal fees from Bayer, personal fees from Boehringer Ingelheim, personal fees from Biotest, personal fees from CSL Behring, personal fees from Eli Lilly, personal fees from Grifols, personal fees from Novo Nordisk, personal fees from Pfizer, personal fees from Sanofi Aventis, and personal fees from Sobi, outside the submitted work. Dr. Santagostino reports personal fees from Bayer, personal fees from Shire, personal fees from NovoNordisk, personal fees from Sobi, personal fees from Pfizer, personal fees from Bioverativ, personal fees from CSL Behring, personal fees from Roche, personal fees from Grifols, personal fees from Kedrion, and personal fees from Octapharma, outside the submitted work. Dr. Santoro has nothing to disclose. Dr. Coppola reports personal fees from Bayer, personal fees from Novo Nordisk, personal fees from Octapharma, personal fees from Kedrion, personal fees from Shire, and personal fees from CSL Behring, outside the submitted work. Dr. Franchini has nothing to disclose. Dr. Morfini reports grants and personal fees from Pfizer, personal fees from Kedrion, personal fees from Octapharma, personal fees from Sobi, personal fees from CSL Behring, personal fees from Baxalta, personal fees from Novo Nordisk, and personal fees from Freeline Therapeutics, outside the submitted work. Dr. Rocino reports personal fees from Bayer, personal fees from Kedrion, personal fees from CSL Behring, personal fees from Novo Nordisk, personal fees from Pfizer, personal fees from Shire, and personal fees from Sobi, outside the submitted work. Dr. Santoro reports personal fees from Bayer, personal fees from CSL Behring, personal fees from Gilead, personal fees from Grifols, personal fees from Kedrion, personal fees from Novartis, personal fees from Novo Nordisk, personal fees from Pfizer, personal fees from Shire, and personal fees from Sobi, outside the submitted work.
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Publikationsdatum:
03. Mai 2018 (online)

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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