Paediatric haemophilia with inhibitors: Existing management options, treatment gaps and unmet needs

Elena Santagostino, M. Morfini, G. K H Auerswald, G. M. Benson, S. Z. Šalek, T. Lambert, P. Salaj, V. Jimenez-Yuste, R. C R Ljung

Research output: Contribution to journalArticle

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 Zürich 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.

Original languageEnglish
Pages (from-to)983-989
Number of pages7
JournalHaemophilia
Volume15
Issue number5
DOIs
Publication statusPublished - 2009

Fingerprint

Hemophilia A
Pediatrics
Hemorrhage
Therapeutics
Joints
Hemarthrosis
Factor VIIa
Immune Tolerance
Joint Diseases
Hematology

Keywords

  • Activated prothrombin complex concentrate
  • Haemophilia
  • Inhibitors
  • Paediatric
  • Prophylaxis
  • Recombinant activated factor VII

ASJC Scopus subject areas

  • Hematology
  • Genetics(clinical)

Cite this

Santagostino, E., Morfini, M., Auerswald, G. K. H., Benson, G. M., Šalek, S. Z., Lambert, T., ... Ljung, R. C. R. (2009). Paediatric haemophilia with inhibitors: Existing management options, treatment gaps and unmet needs. Haemophilia, 15(5), 983-989. https://doi.org/10.1111/j.1365-2516.2009.01999.x

Paediatric haemophilia with inhibitors : Existing management options, treatment gaps and unmet needs. / Santagostino, Elena; Morfini, M.; Auerswald, G. K H; Benson, G. M.; Šalek, S. Z.; Lambert, T.; Salaj, P.; Jimenez-Yuste, V.; Ljung, R. C R.

In: Haemophilia, Vol. 15, No. 5, 2009, p. 983-989.

Research output: Contribution to journalArticle

Santagostino, E, Morfini, M, Auerswald, GKH, Benson, GM, Šalek, SZ, Lambert, T, Salaj, P, Jimenez-Yuste, V & Ljung, RCR 2009, 'Paediatric haemophilia with inhibitors: Existing management options, treatment gaps and unmet needs', Haemophilia, vol. 15, no. 5, pp. 983-989. https://doi.org/10.1111/j.1365-2516.2009.01999.x
Santagostino, Elena ; Morfini, M. ; Auerswald, G. K H ; Benson, G. M. ; Šalek, S. Z. ; Lambert, T. ; Salaj, P. ; Jimenez-Yuste, V. ; Ljung, R. C R. / Paediatric haemophilia with inhibitors : Existing management options, treatment gaps and unmet needs. In: Haemophilia. 2009 ; Vol. 15, No. 5. pp. 983-989.
@article{ad336f450c5c406e8a9028087f9afb8f,
title = "Paediatric haemophilia with inhibitors: Existing management options, treatment gaps and unmet needs",
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 Z{\"u}rich 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.",
keywords = "Activated prothrombin complex concentrate, Haemophilia, Inhibitors, Paediatric, Prophylaxis, Recombinant activated factor VII",
author = "Elena Santagostino and M. Morfini and Auerswald, {G. K H} and Benson, {G. M.} and Šalek, {S. Z.} and T. Lambert and P. Salaj and V. Jimenez-Yuste and Ljung, {R. C R}",
year = "2009",
doi = "10.1111/j.1365-2516.2009.01999.x",
language = "English",
volume = "15",
pages = "983--989",
journal = "Haemophilia",
issn = "1351-8216",
publisher = "Wiley-Blackwell Publishing Ltd",
number = "5",

}

TY - JOUR

T1 - Paediatric haemophilia with inhibitors

T2 - Existing management options, treatment gaps and unmet needs

AU - Santagostino, Elena

AU - Morfini, M.

AU - Auerswald, G. K H

AU - Benson, G. M.

AU - Šalek, S. Z.

AU - Lambert, T.

AU - Salaj, P.

AU - Jimenez-Yuste, V.

AU - Ljung, R. C R

PY - 2009

Y1 - 2009

N2 - 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 Zürich 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.

AB - 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 Zürich 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.

KW - Activated prothrombin complex concentrate

KW - Haemophilia

KW - Inhibitors

KW - Paediatric

KW - Prophylaxis

KW - Recombinant activated factor VII

UR - http://www.scopus.com/inward/record.url?scp=69949096687&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=69949096687&partnerID=8YFLogxK

U2 - 10.1111/j.1365-2516.2009.01999.x

DO - 10.1111/j.1365-2516.2009.01999.x

M3 - Article

C2 - 19712172

AN - SCOPUS:69949096687

VL - 15

SP - 983

EP - 989

JO - Haemophilia

JF - Haemophilia

SN - 1351-8216

IS - 5

ER -