Paediatric haemophilia with inhibitors: existing management options, treatment gaps and unmet needs
Santagostino, E. ; Morfini, M. ; Auerswald, G. K. -H. ; Benson, G. M. ; Salek, S. Z. ; Lambert, T. ; Salaj, P. ; Jimenez-Yuste, V. and Ljung, Rolf LU
(2009)
In Haemophilia
15(5).
p.983-989
- Abstract
- Development of inhibitors is a severe complication of haemophilia posing many management challenges. While a long-term goal in inhibitor patients is eradication of inhibitors through immune tolerance induction, bypassing agents such as recombinant activated factor VII (rFVIIa) and activated prothrombin complex concentrate (aPCC) are essential for control of bleeding episodes. Paediatric patients with haemophilia and inhibitors are at particular risk of recurrent haemarthroses, and management of these patients should seek to avoid joint damage and support the child's full social and physical development. Current options for management of bleeding complications include on-demand treatment of acute bleeding episodes, secondary prophylaxis to... (More)
- Development of inhibitors is a severe complication of haemophilia posing many management challenges. While a long-term goal in inhibitor patients is eradication of inhibitors through immune tolerance induction, bypassing agents such as recombinant activated factor VII (rFVIIa) and activated prothrombin complex concentrate (aPCC) are essential for control of bleeding episodes. Paediatric patients with haemophilia and inhibitors are at particular risk of recurrent haemarthroses, and management of these patients should seek to avoid joint damage and support the child's full social and physical development. Current options for management of bleeding complications include on-demand treatment of acute bleeding episodes, secondary prophylaxis to avoid recurrent bleeds and surgery to treat affected joints. There is also a rationale for adopting prophylactic approaches to prevent bleeding in inhibitor patients, allowing this group similar opportunities for protection against arthropathy development as are given to non-inhibitor patients. This paper, based on a roundtable meeting of haematology experts at the first Zurich Haemophilia Forum in May 2008, reviews the current evidence supporting more intense and prophylactic approaches to manage bleeding risk in paediatric haemophilia patients with inhibitors, and highlights the need for investigations of primary prophylaxis in this vulnerable patient group, to support best long-term outcome. (Less)
Please use this url to cite or link to this publication:
https://lup.lub.lu.se/record/1476316
- author
- Santagostino, E.
; Morfini, M.
; Auerswald, G. K. -H.
; Benson, G. M.
; Salek, S. Z.
; Lambert, T.
; Salaj, P.
; Jimenez-Yuste, V.
and Ljung, Rolf
LU
- organization
- publishing date
- 2009
- type
- Contribution to journal
- publication status
- published
- subject
- keywords
- paediatric, inhibitors, activated prothrombin complex concentrate, haemophilia, prophylaxis, recombinant activated factor VII
- in
- Haemophilia
- volume
- 15
- issue
- 5
- pages
- 983 - 989
- publisher
- Wiley-Blackwell
- external identifiers
-
- wos:000269367800001
- scopus:69949096687
- pmid:19712172
- ISSN
- 1351-8216
- DOI
- 10.1111/j.1365-2516.2009.01999.x
- language
- English
- LU publication?
- yes
- id
- f70116c1-c3ad-4f6c-aa15-0a2d87dbb55d (old id 1476316)
- date added to LUP
- 2016-04-01 11:43:51
- date last changed
- 2022-04-20 20:57:51
@article{f70116c1-c3ad-4f6c-aa15-0a2d87dbb55d,
abstract = {{Development of inhibitors is a severe complication of haemophilia posing many management challenges. While a long-term goal in inhibitor patients is eradication of inhibitors through immune tolerance induction, bypassing agents such as recombinant activated factor VII (rFVIIa) and activated prothrombin complex concentrate (aPCC) are essential for control of bleeding episodes. Paediatric patients with haemophilia and inhibitors are at particular risk of recurrent haemarthroses, and management of these patients should seek to avoid joint damage and support the child's full social and physical development. Current options for management of bleeding complications include on-demand treatment of acute bleeding episodes, secondary prophylaxis to avoid recurrent bleeds and surgery to treat affected joints. There is also a rationale for adopting prophylactic approaches to prevent bleeding in inhibitor patients, allowing this group similar opportunities for protection against arthropathy development as are given to non-inhibitor patients. This paper, based on a roundtable meeting of haematology experts at the first Zurich Haemophilia Forum in May 2008, reviews the current evidence supporting more intense and prophylactic approaches to manage bleeding risk in paediatric haemophilia patients with inhibitors, and highlights the need for investigations of primary prophylaxis in this vulnerable patient group, to support best long-term outcome.}},
author = {{Santagostino, E. and Morfini, M. and Auerswald, G. K. -H. and Benson, G. M. and Salek, S. Z. and Lambert, T. and Salaj, P. and Jimenez-Yuste, V. and Ljung, Rolf}},
issn = {{1351-8216}},
keywords = {{paediatric; inhibitors; activated prothrombin complex concentrate; haemophilia; prophylaxis; recombinant activated factor VII}},
language = {{eng}},
number = {{5}},
pages = {{983--989}},
publisher = {{Wiley-Blackwell}},
series = {{Haemophilia}},
title = {{Paediatric haemophilia with inhibitors: existing management options, treatment gaps and unmet needs}},
url = {{http://dx.doi.org/10.1111/j.1365-2516.2009.01999.x}},
doi = {{10.1111/j.1365-2516.2009.01999.x}},
volume = {{15}},
year = {{2009}},
}