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Current Choices and Management of Treatment in Persons with Severe Hemophilia A without Inhibitors: A Mini-Delphi Consensus
Background. Regular treatment to prevent bleeding and consequent joint deterioration (prophylaxis) is the standard of care for persons with severe hemophilia A, traditionally based on intravenous infusions of the deficient clotting FVIII concentrates (CFCs). In recent years, extended half-life (EHL)...
Autores principales: | , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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MDPI
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8837169/ https://www.ncbi.nlm.nih.gov/pubmed/35160253 http://dx.doi.org/10.3390/jcm11030801 |
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author | Coppola, Antonio Franchini, Massimo Pappagallo, Giovanni Borchiellini, Alessandra De Cristofaro, Raimondo Molinari, Angelo Claudio Santoro, Rita Carlotta Santoro, Cristina Tagliaferri, Annarita |
author_facet | Coppola, Antonio Franchini, Massimo Pappagallo, Giovanni Borchiellini, Alessandra De Cristofaro, Raimondo Molinari, Angelo Claudio Santoro, Rita Carlotta Santoro, Cristina Tagliaferri, Annarita |
author_sort | Coppola, Antonio |
collection | PubMed |
description | Background. Regular treatment to prevent bleeding and consequent joint deterioration (prophylaxis) is the standard of care for persons with severe hemophilia A, traditionally based on intravenous infusions of the deficient clotting FVIII concentrates (CFCs). In recent years, extended half-life (EHL) CFCs and the non-replacement agent emicizumab, subcutaneously administered, have reduced the treatment burden. Methods. To compare and integrate the opinions on the different therapies available, eight hemophilia specialists were involved in drafting items of interest and relative statements through the Estimate-Talk-Estimate (ETE) method (“mini-Delphi”), in this way reaching consensus. Results. Eighteen items were identified, then harmonized to 10, and a statement was generated for each. These statements highlight the importance of personalized prophylaxis regimens. CFCs, particularly EHL products, seem more suitable for this, despite the challenging intravenous (i.v.) administration. Limited real-world experience, particularly in some clinical settings, and the lack of evidence on long-term safety and efficacy of non-replacement agents, require careful individual risk/benefit assessment and multidisciplinary data collection. Conclusions. The increased treatment options extend the opportunities of personalized prophylaxis, the mainstay of modern management of hemophilia. Close, long-term clinical and laboratory follow-up of patients using newer therapeutic approaches by specialized hemophilia treatment centers is needed. |
format | Online Article Text |
id | pubmed-8837169 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2022 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
spelling | pubmed-88371692022-02-12 Current Choices and Management of Treatment in Persons with Severe Hemophilia A without Inhibitors: A Mini-Delphi Consensus Coppola, Antonio Franchini, Massimo Pappagallo, Giovanni Borchiellini, Alessandra De Cristofaro, Raimondo Molinari, Angelo Claudio Santoro, Rita Carlotta Santoro, Cristina Tagliaferri, Annarita J Clin Med Article Background. Regular treatment to prevent bleeding and consequent joint deterioration (prophylaxis) is the standard of care for persons with severe hemophilia A, traditionally based on intravenous infusions of the deficient clotting FVIII concentrates (CFCs). In recent years, extended half-life (EHL) CFCs and the non-replacement agent emicizumab, subcutaneously administered, have reduced the treatment burden. Methods. To compare and integrate the opinions on the different therapies available, eight hemophilia specialists were involved in drafting items of interest and relative statements through the Estimate-Talk-Estimate (ETE) method (“mini-Delphi”), in this way reaching consensus. Results. Eighteen items were identified, then harmonized to 10, and a statement was generated for each. These statements highlight the importance of personalized prophylaxis regimens. CFCs, particularly EHL products, seem more suitable for this, despite the challenging intravenous (i.v.) administration. Limited real-world experience, particularly in some clinical settings, and the lack of evidence on long-term safety and efficacy of non-replacement agents, require careful individual risk/benefit assessment and multidisciplinary data collection. Conclusions. The increased treatment options extend the opportunities of personalized prophylaxis, the mainstay of modern management of hemophilia. Close, long-term clinical and laboratory follow-up of patients using newer therapeutic approaches by specialized hemophilia treatment centers is needed. MDPI 2022-02-02 /pmc/articles/PMC8837169/ /pubmed/35160253 http://dx.doi.org/10.3390/jcm11030801 Text en © 2022 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/). |
spellingShingle | Article Coppola, Antonio Franchini, Massimo Pappagallo, Giovanni Borchiellini, Alessandra De Cristofaro, Raimondo Molinari, Angelo Claudio Santoro, Rita Carlotta Santoro, Cristina Tagliaferri, Annarita Current Choices and Management of Treatment in Persons with Severe Hemophilia A without Inhibitors: A Mini-Delphi Consensus |
title | Current Choices and Management of Treatment in Persons with Severe Hemophilia A without Inhibitors: A Mini-Delphi Consensus |
title_full | Current Choices and Management of Treatment in Persons with Severe Hemophilia A without Inhibitors: A Mini-Delphi Consensus |
title_fullStr | Current Choices and Management of Treatment in Persons with Severe Hemophilia A without Inhibitors: A Mini-Delphi Consensus |
title_full_unstemmed | Current Choices and Management of Treatment in Persons with Severe Hemophilia A without Inhibitors: A Mini-Delphi Consensus |
title_short | Current Choices and Management of Treatment in Persons with Severe Hemophilia A without Inhibitors: A Mini-Delphi Consensus |
title_sort | current choices and management of treatment in persons with severe hemophilia a without inhibitors: a mini-delphi consensus |
topic | Article |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8837169/ https://www.ncbi.nlm.nih.gov/pubmed/35160253 http://dx.doi.org/10.3390/jcm11030801 |
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