Inhibitors: A Need for Eradication?

Elena Santagostino, Guy Young, Carmen Escuriola Ettingshausen, Victor Jimenez-Yuste, Manuel Carcao

Research output: Contribution to journalReview article

1 Citation (Scopus)

Abstract

The development of inhibitors against factor VIII (FVIII) concentrates represents a significant treatment complication for hemophilia. Immune tolerance induction (ITI) therapy eradicates inhibitors in 60-80% of patients, resulting in a normal FVIII response. This article, based on presentations at the 6th International Coagulation Meeting, held in Barcelona, Spain, in September 2017, provides an overview of management approaches for patients with inhibitors and briefly tabulates four cases of ITI therapy (first-line or rescue ITI therapy in pediatric and adult patients) with successful outcomes. Switching FVIII product from recombinant FVIII to plasma-derived FVIII/VWF concentrate may be helpful in eradicating inhibitors. The rate of decline of inhibitor titer in the initial stages of ITI therapy is a good indicator of the success or failure of therapy, although prognostic biomarkers are needed. The development of the bispecific monoclonal antibody emicizumab, which was recently shown to reduce bleeding in inhibitor patients, offers a potential alternative therapeutic option. However, the benefits of inhibitor eradication, including a wider choice of cheaper therapeutic products for preventing and treating bleeds, suggest that at least one attempt of ITI therapy should be offered to patients who develop inhibitors.

Original languageEnglish
Pages (from-to)151-155
Number of pages5
JournalActa Haematologica
Volume141
Issue number3
DOIs
Publication statusPublished - Apr 1 2019

Fingerprint

Immune Tolerance
Factor VIII
Therapeutics
Bispecific Antibodies
Hemophilia A
Spain
Biomarkers
Pediatrics
Hemorrhage

Keywords

  • Factor VIII concentrate
  • Hemophilia
  • Immune tolerance induction
  • Inhibitors
  • Von Willebrand factor

ASJC Scopus subject areas

  • Hematology

Cite this

Santagostino, E., Young, G., Escuriola Ettingshausen, C., Jimenez-Yuste, V., & Carcao, M. (2019). Inhibitors: A Need for Eradication? Acta Haematologica, 141(3), 151-155. https://doi.org/10.1159/000495454

Inhibitors : A Need for Eradication? / Santagostino, Elena; Young, Guy; Escuriola Ettingshausen, Carmen; Jimenez-Yuste, Victor; Carcao, Manuel.

In: Acta Haematologica, Vol. 141, No. 3, 01.04.2019, p. 151-155.

Research output: Contribution to journalReview article

Santagostino, E, Young, G, Escuriola Ettingshausen, C, Jimenez-Yuste, V & Carcao, M 2019, 'Inhibitors: A Need for Eradication?', Acta Haematologica, vol. 141, no. 3, pp. 151-155. https://doi.org/10.1159/000495454
Santagostino E, Young G, Escuriola Ettingshausen C, Jimenez-Yuste V, Carcao M. Inhibitors: A Need for Eradication? Acta Haematologica. 2019 Apr 1;141(3):151-155. https://doi.org/10.1159/000495454
Santagostino, Elena ; Young, Guy ; Escuriola Ettingshausen, Carmen ; Jimenez-Yuste, Victor ; Carcao, Manuel. / Inhibitors : A Need for Eradication?. In: Acta Haematologica. 2019 ; Vol. 141, No. 3. pp. 151-155.
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