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

TY - JOUR

T1 - Current view and outcome of ITI therapy - 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

AU - European Haemophilia Therapy Strategy Board (EHTSB)

N1 - Copyright © 2016 Elsevier Ltd. All rights reserved.

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 (<50IU/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 (<50IU/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 - Adolescent

KW - Adult

KW - Child

KW - Europe

KW - Female

KW - Hemophilia A

KW - Hemophilia B

KW - Humans

KW - Immune Tolerance

KW - Immunosuppression

KW - Male

KW - Treatment Outcome

KW - Young Adult

KW - Journal Article

U2 - 10.1016/j.thromres.2016.10.015

DO - 10.1016/j.thromres.2016.10.015

M3 - SCORING: Journal article

C2 - 27770665

VL - 148

SP - 38

EP - 44

JO - THROMB RES

JF - THROMB RES

SN - 0049-3848

ER -