Current view and outcome of ITI therapy: A change over time?

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Current view and outcome of ITI therapy : A change over time? / Holstein, K.; Batorova, A.; Carvalho, M.; Fijnvandraat, K.; Holme, P.; Kavakli, K.; Lambert, T.; Rocino, A.; Jiménez-Yuste, V.; Astermark, J.

I: Thrombosis Research, Vol. 148, 12.2016, s. 38-44.

Forskningsoutput: TidskriftsbidragArtikel i vetenskaplig tidskrift

Harvard

Holstein, K, Batorova, A, Carvalho, M, Fijnvandraat, K, Holme, P, Kavakli, K, Lambert, T, Rocino, A, Jiménez-Yuste, V & Astermark, J 2016, 'Current view and outcome of ITI therapy: A change over time?', Thrombosis Research, vol. 148, s. 38-44. https://doi.org/10.1016/j.thromres.2016.10.015

APA

Holstein, K., Batorova, A., Carvalho, M., Fijnvandraat, K., Holme, P., Kavakli, K., Lambert, T., Rocino, A., Jiménez-Yuste, V., & Astermark, J. (2016). Current view and outcome of ITI therapy: A change over time? Thrombosis Research, 148, 38-44. https://doi.org/10.1016/j.thromres.2016.10.015

CBE

Holstein K, Batorova A, Carvalho M, Fijnvandraat K, Holme P, Kavakli K, Lambert T, Rocino A, Jiménez-Yuste V, Astermark J. 2016. Current view and outcome of ITI therapy: A change over time?. Thrombosis Research. 148:38-44. https://doi.org/10.1016/j.thromres.2016.10.015

MLA

Vancouver

Holstein K, Batorova A, Carvalho M, Fijnvandraat K, Holme P, Kavakli K et al. Current view and outcome of ITI therapy: A change over time? Thrombosis Research. 2016 dec;148:38-44. https://doi.org/10.1016/j.thromres.2016.10.015

Author

Holstein, K. ; Batorova, A. ; Carvalho, M. ; Fijnvandraat, K. ; Holme, P. ; Kavakli, K. ; Lambert, T. ; Rocino, A. ; Jiménez-Yuste, V. ; Astermark, J. / Current view and outcome of ITI therapy : A change over time?. I: Thrombosis Research. 2016 ; Vol. 148. s. 38-44.

RIS

TY - JOUR

T1 - Current view and outcome of ITI therapy

T2 - A change over time?

AU - Holstein, K.

AU - Batorova, A.

AU - Carvalho, M.

AU - Fijnvandraat, K.

AU - Holme, P.

AU - Kavakli, K.

AU - Lambert, T.

AU - Rocino, A.

AU - Jiménez-Yuste, V.

AU - Astermark, J.

PY - 2016/12

Y1 - 2016/12

N2 - Introduction Inhibitor development in people with haemophilia is a serious complication that may require intensive and costly interventions. The goal of inhibitor management should be permanent inhibitor eradication through immune tolerance induction (ITI), but well-designed studies are lacking and the management of patients is therefore defined by the experience and views of the clinician. Objectives To explore the current clinical practice and outcome of ITI therapy in Europe and how this may have changed over the last decade, as well as to provide consensus recommendations to guide clinicians in their clinical practice. Methods A survey was conducted among 16 European haemophilia comprehensive care centres to evaluate current ITI treatment regimens and success rates in severe and mild/moderate haemophilia A and haemophilia B. In addition, an updated literature review was performed as guidance for providing recommendations. Results We demonstrated successful inhibitor treatment in 86% of severe haemophilia A patients with low responding (LR) and 59% of patients with high responding (HR) inhibitors. Some new trends in the management of patients with inhibitors were identified, including a tendency to use low-dose regimens (< 50 IU/kg/d) in both children and adults with HR inhibitors possibly based on similar success rates demonstrated in the I-ITI study compared to a high-dose protocol. Data on ITI therapy in mild and moderate haemophilia as well as haemophilia B were limited. Conclusions The outcome of ITI therapy seems to be stable over time, and treatment regimens remain heterogeneous. The use of low dose regimens however is considered more frequently.

AB - Introduction Inhibitor development in people with haemophilia is a serious complication that may require intensive and costly interventions. The goal of inhibitor management should be permanent inhibitor eradication through immune tolerance induction (ITI), but well-designed studies are lacking and the management of patients is therefore defined by the experience and views of the clinician. Objectives To explore the current clinical practice and outcome of ITI therapy in Europe and how this may have changed over the last decade, as well as to provide consensus recommendations to guide clinicians in their clinical practice. Methods A survey was conducted among 16 European haemophilia comprehensive care centres to evaluate current ITI treatment regimens and success rates in severe and mild/moderate haemophilia A and haemophilia B. In addition, an updated literature review was performed as guidance for providing recommendations. Results We demonstrated successful inhibitor treatment in 86% of severe haemophilia A patients with low responding (LR) and 59% of patients with high responding (HR) inhibitors. Some new trends in the management of patients with inhibitors were identified, including a tendency to use low-dose regimens (< 50 IU/kg/d) in both children and adults with HR inhibitors possibly based on similar success rates demonstrated in the I-ITI study compared to a high-dose protocol. Data on ITI therapy in mild and moderate haemophilia as well as haemophilia B were limited. Conclusions The outcome of ITI therapy seems to be stable over time, and treatment regimens remain heterogeneous. The use of low dose regimens however is considered more frequently.

KW - Haemophilia

KW - Immune tolerance induction

KW - Immunosuppression

KW - Inhibitors

KW - ITI regimens

UR - http://www.scopus.com/inward/record.url?scp=84992153352&partnerID=8YFLogxK

U2 - 10.1016/j.thromres.2016.10.015

DO - 10.1016/j.thromres.2016.10.015

M3 - Article

C2 - 27770665

AN - SCOPUS:84992153352

VL - 148

SP - 38

EP - 44

JO - Thrombosis Research

JF - Thrombosis Research

SN - 1879-2472

ER -