Abstract
Introduction: The development of inhibitors against factor VIII (FVIII) replacement therapy remains the most important challenge for clinicians in the treatment of hemophilia patients. This review focusses on risk factors and management of FVIII inhibitors, particularly in light of SIPPET study findings and subsequent analyses. Areas covered: A brief history and evolution of hemophilia therapies is provided, including an overview of conventional and new (including investigational) therapeutic approaches for the treatment of hemophilia. The SIPPET study, the first randomized clinical trial to demonstrate a lower incidence of inhibitors in previously untreated patients treated with plasma-derived FVIII products compared with recombinant FVIII products, has generated much debate. We review the SIPPET trial and reactions, in addition to preliminary observations from a single center’s experience, the cost impact of inhibitors, recent findings from SIPPET subanalyses, and inhibitor development in previously-treated patients. Expert commentary: Despite recent advances in potential new treatment options for hemophilia, conventional factor replacement concentrates currently remain the cornerstone of treatment. It is paramount that clinicians familiarize themselves with the findings from the SIPPET trial and substudies, in order to better inform their patients and families on inhibitor risk factors and to aid the treatment decision-making process.
| Original language | English |
|---|---|
| Pages (from-to) | 87-97 |
| Number of pages | 11 |
| Journal | Expert Review of Hematology |
| Volume | 11 |
| Issue number | 2 |
| DOIs | |
| Publication status | Published - Feb 1 2018 |
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Keywords
- Factor VIII
- hemophilia
- inhibitors
- plasma-derived FVIII
- recombinant FVIII
- SIPPET
ASJC Scopus subject areas
- Hematology
Cite this
A contemporary look at FVIII inhibitor development : still a great influence on the evolution of hemophilia therapies. / Santagostino, Elena; Young, Guy; Carcao, Manuel; Mannucci, Pier Mannuccio; Halimeh, Susan; Austin, Steve.
In: Expert Review of Hematology, Vol. 11, No. 2, 01.02.2018, p. 87-97.Research output: Contribution to journal › Review article
}
TY - JOUR
T1 - A contemporary look at FVIII inhibitor development
T2 - still a great influence on the evolution of hemophilia therapies
AU - Santagostino, Elena
AU - Young, Guy
AU - Carcao, Manuel
AU - Mannucci, Pier Mannuccio
AU - Halimeh, Susan
AU - Austin, Steve
PY - 2018/2/1
Y1 - 2018/2/1
N2 - Introduction: The development of inhibitors against factor VIII (FVIII) replacement therapy remains the most important challenge for clinicians in the treatment of hemophilia patients. This review focusses on risk factors and management of FVIII inhibitors, particularly in light of SIPPET study findings and subsequent analyses. Areas covered: A brief history and evolution of hemophilia therapies is provided, including an overview of conventional and new (including investigational) therapeutic approaches for the treatment of hemophilia. The SIPPET study, the first randomized clinical trial to demonstrate a lower incidence of inhibitors in previously untreated patients treated with plasma-derived FVIII products compared with recombinant FVIII products, has generated much debate. We review the SIPPET trial and reactions, in addition to preliminary observations from a single center’s experience, the cost impact of inhibitors, recent findings from SIPPET subanalyses, and inhibitor development in previously-treated patients. Expert commentary: Despite recent advances in potential new treatment options for hemophilia, conventional factor replacement concentrates currently remain the cornerstone of treatment. It is paramount that clinicians familiarize themselves with the findings from the SIPPET trial and substudies, in order to better inform their patients and families on inhibitor risk factors and to aid the treatment decision-making process.
AB - Introduction: The development of inhibitors against factor VIII (FVIII) replacement therapy remains the most important challenge for clinicians in the treatment of hemophilia patients. This review focusses on risk factors and management of FVIII inhibitors, particularly in light of SIPPET study findings and subsequent analyses. Areas covered: A brief history and evolution of hemophilia therapies is provided, including an overview of conventional and new (including investigational) therapeutic approaches for the treatment of hemophilia. The SIPPET study, the first randomized clinical trial to demonstrate a lower incidence of inhibitors in previously untreated patients treated with plasma-derived FVIII products compared with recombinant FVIII products, has generated much debate. We review the SIPPET trial and reactions, in addition to preliminary observations from a single center’s experience, the cost impact of inhibitors, recent findings from SIPPET subanalyses, and inhibitor development in previously-treated patients. Expert commentary: Despite recent advances in potential new treatment options for hemophilia, conventional factor replacement concentrates currently remain the cornerstone of treatment. It is paramount that clinicians familiarize themselves with the findings from the SIPPET trial and substudies, in order to better inform their patients and families on inhibitor risk factors and to aid the treatment decision-making process.
KW - Factor VIII
KW - hemophilia
KW - inhibitors
KW - plasma-derived FVIII
KW - recombinant FVIII
KW - SIPPET
UR - http://www.scopus.com/inward/record.url?scp=85041200904&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85041200904&partnerID=8YFLogxK
U2 - 10.1080/17474086.2018.1419862
DO - 10.1080/17474086.2018.1419862
M3 - Review article
AN - SCOPUS:85041200904
VL - 11
SP - 87
EP - 97
JO - Expert Review of Hematology
JF - Expert Review of Hematology
SN - 1747-4086
IS - 2
ER -