Cargando…

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)...

Descripción completa

Detalles Bibliográficos
Autores principales: Coppola, Antonio, Franchini, Massimo, Pappagallo, Giovanni, Borchiellini, Alessandra, De Cristofaro, Raimondo, Molinari, Angelo Claudio, Santoro, Rita Carlotta, Santoro, Cristina, Tagliaferri, Annarita
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
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
_version_ 1784649858168127488
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
work_keys_str_mv AT coppolaantonio currentchoicesandmanagementoftreatmentinpersonswithseverehemophiliaawithoutinhibitorsaminidelphiconsensus
AT franchinimassimo currentchoicesandmanagementoftreatmentinpersonswithseverehemophiliaawithoutinhibitorsaminidelphiconsensus
AT pappagallogiovanni currentchoicesandmanagementoftreatmentinpersonswithseverehemophiliaawithoutinhibitorsaminidelphiconsensus
AT borchiellinialessandra currentchoicesandmanagementoftreatmentinpersonswithseverehemophiliaawithoutinhibitorsaminidelphiconsensus
AT decristofaroraimondo currentchoicesandmanagementoftreatmentinpersonswithseverehemophiliaawithoutinhibitorsaminidelphiconsensus
AT molinariangeloclaudio currentchoicesandmanagementoftreatmentinpersonswithseverehemophiliaawithoutinhibitorsaminidelphiconsensus
AT santororitacarlotta currentchoicesandmanagementoftreatmentinpersonswithseverehemophiliaawithoutinhibitorsaminidelphiconsensus
AT santorocristina currentchoicesandmanagementoftreatmentinpersonswithseverehemophiliaawithoutinhibitorsaminidelphiconsensus
AT tagliaferriannarita currentchoicesandmanagementoftreatmentinpersonswithseverehemophiliaawithoutinhibitorsaminidelphiconsensus