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The burden of inhibitors in haemophilia patients

Christopher E Walsh, Víctor Jiménez-Yuste1, Guenter Auerswald

  • 1Victor Jiménez-Yuste, Hospital Universitario La Paz - Hematology, Paseo de la Castellana 261 Apostol Santiago 61 1 J, Madrid 28017, Spain, Tel.: +34 619452698, Fax: +34 917277226,

Thrombosis and Haemostasis
|August 17, 2016
PubMed

Insights

Achieving immune tolerance induction (ITI) is crucial for haemophilia patients with inhibitors. Using von Willebrand factor (VWF)-containing factor VIII (FVIII) concentrates in ITI may improve inhibitor eradication success rates.

Area of Science:

  • Hematology
  • Immunology
  • Pharmacology

Background:

  • Haemophilia patients with inhibitors face increased morbidity, mortality, and reduced quality of life.
  • The presence of inhibitors significantly increases the societal cost of care.
  • Immune tolerance induction (ITI) is the most critical step for managing inhibitor patients.

Purpose of the Study:

  • To evaluate the effectiveness of different factor VIII (FVIII) products in immune tolerance induction (ITI) regimens.
  • To determine the optimal FVIII product for achieving high inhibitor eradication success rates in haemophilia patients.

Main Methods:

  • Review of in vitro and retrospective clinical evidence.
  • Analysis of ITI regimens utilizing various factor VIII (FVIII) products.
  • Comparison of outcomes based on the type of FVIII concentrate used.

Main Results:

  • Evidence supports the use of plasma-derived von Willebrand factor (VWF)-containing FVIII concentrates in ITI.
  • These specific FVIII products may lead to early and high success rates in inhibitor eradication.
  • The choice of FVIII product can influence the efficacy of ITI.

Conclusions:

  • Successful ITI is paramount for improving outcomes in haemophilia patients with inhibitors.
  • Plasma-derived VWF-containing FVIII concentrates show promise for effective ITI.
  • Further research may clarify optimal product selection for ITI regimens.

Keywords:
FVIIIHaemophiliaimmune tolerance inductioninhibitorvon Willebrand factor

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