Extended half-life recombinant factor VIII treatment of hemophilia A in Brazil: an expert consensus statement
Introduction: Treatment of hemophilia A in Brazil is offered to all patients at no cost. However, several unmet medical needs exist. Method: In this study, we applied the Delphi method to discuss with seven hemophilia A specialists the challenges that patients and the health system face regarding he...
| Main Authors: | , , , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Elsevier
2024-01-01
|
| Series: | Hematology, Transfusion and Cell Therapy |
| Subjects: | |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2531137922014705 |
| _version_ | 1827324740934565888 |
|---|---|
| author | Margareth Castro Ozelo Sandra Vallin Antunes Paula Ribeiro Villaca Luciana Correa Oliveira Ieda Solange Pinto Claúdia Santos Lorenzato Alessandra Nunes Loureiro Prezotti Renato Mantelli Picoli |
| author_facet | Margareth Castro Ozelo Sandra Vallin Antunes Paula Ribeiro Villaca Luciana Correa Oliveira Ieda Solange Pinto Claúdia Santos Lorenzato Alessandra Nunes Loureiro Prezotti Renato Mantelli Picoli |
| author_sort | Margareth Castro Ozelo |
| collection | DOAJ |
| description | Introduction: Treatment of hemophilia A in Brazil is offered to all patients at no cost. However, several unmet medical needs exist. Method: In this study, we applied the Delphi method to discuss with seven hemophilia A specialists the challenges that patients and the health system face regarding hemophilia A treatment and opportunities for improvement. Results: A consensus was obtained regarding the number of weekly infusions and patient adherence to treatment. The bleeding profile, unfavourable pharmacokinetics (PKs), low adherence and high daily activity were patient profiles that would benefit from using the extended half-life (EHL) recombinant factor VIII (rFVIII). The advantages of treatment with the EHL rFVIII were the lower number of infusions per week, which could increase patient adherence and decrease the risk of bleeds, due to a more constant plasma level, a lower value. Additionally, the EHL rFVIII could improve quality of life, especially in patients with high daily activity, such as adolescents and young adults. The panelists mentioned that EHL rFVIII, if available, could be offered first to the priority group (adolescents between 12 and 19 years old), followed by adults (20 to 64 years old) and elderly people (over 65 years old). Conclusion: In summary, the EHL rFVIII offers the optimal prophylaxis by decreasing the dose frequency, increasing the treatment adherence and improving the QoL, without compromising safety and efficacy. |
| first_indexed | 2024-03-07T14:00:25Z |
| format | Article |
| id | doaj.art-c06a34d679284488a5f40c55467d372d |
| institution | Directory Open Access Journal |
| issn | 2531-1379 |
| language | English |
| last_indexed | 2024-03-07T14:00:25Z |
| publishDate | 2024-01-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Hematology, Transfusion and Cell Therapy |
| spelling | doaj.art-c06a34d679284488a5f40c55467d372d2024-03-07T05:28:57ZengElsevierHematology, Transfusion and Cell Therapy2531-13792024-01-014613641Extended half-life recombinant factor VIII treatment of hemophilia A in Brazil: an expert consensus statementMargareth Castro Ozelo0Sandra Vallin Antunes1Paula Ribeiro Villaca2Luciana Correa Oliveira3Ieda Solange Pinto4Claúdia Santos Lorenzato5Alessandra Nunes Loureiro Prezotti6Renato Mantelli Picoli7Universidade Estadual de Campinas (Unicamp), Campinas, SP, BrazilUniversidade Federal de Sao Paulo (USP), São Paulo, SP, BrazilHospital das Clínicas da Universidade de São Paulo (HC USP), São Paulo, SP, BrazilHospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo (HCFMRP/USP), Ribeirão Preto, SP, BrazilFundação Centro de Hematologia e Hemoterapia do Pará (Hemopa), Belém, PA, BrazilCentro de Hematologia e Hemoterapia do Paraná (Hemopar), Londrina, PR, BrazilCentro de Hematologia e Hemoterapia do Espírito Santo (Hemoes), Vitória, ES, BrazilCerner Enviza, São Paulo, SP, Brazil; Corresponding author.Introduction: Treatment of hemophilia A in Brazil is offered to all patients at no cost. However, several unmet medical needs exist. Method: In this study, we applied the Delphi method to discuss with seven hemophilia A specialists the challenges that patients and the health system face regarding hemophilia A treatment and opportunities for improvement. Results: A consensus was obtained regarding the number of weekly infusions and patient adherence to treatment. The bleeding profile, unfavourable pharmacokinetics (PKs), low adherence and high daily activity were patient profiles that would benefit from using the extended half-life (EHL) recombinant factor VIII (rFVIII). The advantages of treatment with the EHL rFVIII were the lower number of infusions per week, which could increase patient adherence and decrease the risk of bleeds, due to a more constant plasma level, a lower value. Additionally, the EHL rFVIII could improve quality of life, especially in patients with high daily activity, such as adolescents and young adults. The panelists mentioned that EHL rFVIII, if available, could be offered first to the priority group (adolescents between 12 and 19 years old), followed by adults (20 to 64 years old) and elderly people (over 65 years old). Conclusion: In summary, the EHL rFVIII offers the optimal prophylaxis by decreasing the dose frequency, increasing the treatment adherence and improving the QoL, without compromising safety and efficacy.http://www.sciencedirect.com/science/article/pii/S2531137922014705Hemophilia AFactor VIIIHemophilia replacement treatmentDelphi method |
| spellingShingle | Margareth Castro Ozelo Sandra Vallin Antunes Paula Ribeiro Villaca Luciana Correa Oliveira Ieda Solange Pinto Claúdia Santos Lorenzato Alessandra Nunes Loureiro Prezotti Renato Mantelli Picoli Extended half-life recombinant factor VIII treatment of hemophilia A in Brazil: an expert consensus statement Hematology, Transfusion and Cell Therapy Hemophilia A Factor VIII Hemophilia replacement treatment Delphi method |
| title | Extended half-life recombinant factor VIII treatment of hemophilia A in Brazil: an expert consensus statement |
| title_full | Extended half-life recombinant factor VIII treatment of hemophilia A in Brazil: an expert consensus statement |
| title_fullStr | Extended half-life recombinant factor VIII treatment of hemophilia A in Brazil: an expert consensus statement |
| title_full_unstemmed | Extended half-life recombinant factor VIII treatment of hemophilia A in Brazil: an expert consensus statement |
| title_short | Extended half-life recombinant factor VIII treatment of hemophilia A in Brazil: an expert consensus statement |
| title_sort | extended half life recombinant factor viii treatment of hemophilia a in brazil an expert consensus statement |
| topic | Hemophilia A Factor VIII Hemophilia replacement treatment Delphi method |
| url | http://www.sciencedirect.com/science/article/pii/S2531137922014705 |
| work_keys_str_mv | AT margarethcastroozelo extendedhalfliferecombinantfactorviiitreatmentofhemophiliaainbrazilanexpertconsensusstatement AT sandravallinantunes extendedhalfliferecombinantfactorviiitreatmentofhemophiliaainbrazilanexpertconsensusstatement AT paularibeirovillaca extendedhalfliferecombinantfactorviiitreatmentofhemophiliaainbrazilanexpertconsensusstatement AT lucianacorreaoliveira extendedhalfliferecombinantfactorviiitreatmentofhemophiliaainbrazilanexpertconsensusstatement AT iedasolangepinto extendedhalfliferecombinantfactorviiitreatmentofhemophiliaainbrazilanexpertconsensusstatement AT claudiasantoslorenzato extendedhalfliferecombinantfactorviiitreatmentofhemophiliaainbrazilanexpertconsensusstatement AT alessandranunesloureiroprezotti extendedhalfliferecombinantfactorviiitreatmentofhemophiliaainbrazilanexpertconsensusstatement AT renatomantellipicoli extendedhalfliferecombinantfactorviiitreatmentofhemophiliaainbrazilanexpertconsensusstatement |