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Title: First prospective report on immune tolerance in poor risk haemophilia A inhibitor patients with a single factor VIII/von Willebrand factor concentrate in an observational immune tolerance induction study.
Authors: Kreuz, W
Escuriola Ettingshausen, C
Vdovin, V
Zozulya, N
Plyushch, O
Svirin, P
Andreeva, T
Bubanská, E
Campos, M
Benedik-Dolničar, M
Jiménez-Yuste, V
Kitanovski, L
Klukowska, A
Momot, A
Osmulskaya, N
Prieto, M
Šalek, S Z
Velasco, F
Pavlova, A
Oldenburg, J
Knaub, S
Jansen, M
Belyanskaya, L
Walter, O
ObsITI study group
ObsITI committee
Keywords: Bonn protocol;factor VIII inhibitors;factor VIII/von Willebrand factor concentrate;haemophilia A;immune tolerance induction;poor prognosis for immune tolerance induction
metadata.dc.subject.mesh: Adolescent
Antibodies, Neutralizing
Child, Preschool
Drug Combinations
Factor VIII
Hemophilia A
Immune Tolerance
Prospective Studies
Risk Factors
Young Adult
von Willebrand Factor
Issue Date: 23-Jul-2015
Abstract: Development of neutralizing inhibitors against factor VIII (FVIII) is a major complication of haemophilia A treatment. The ongoing, international, open-label, uncontrolled, observational immune tolerance induction (ObsITI) study evaluates ITI, the standard of care in patients with inhibitors. Forty-eight prospective patients in this interim analysis received a single plasma-derived, von Willebrand factor-stabilized, FVIII concentrate (pdFVIII/VWF) for ITI. According to recommended Bonn protocol, 'low responders' at ITI start ( Forty of 48 patients (83.3%), had at least one risk factor for poor ITI-prognosis at ITI start (i.e. age ≥7 years, >2 years since inhibitor diagnosis, inhibitor titre ≥10 BU at the start of ITI, or prior ITI failure). Nonetheless, 34 patients (70.8%) achieved complete success, 3 (6.3%) partial success, 1 (2.1%) partial response; ITI failed in 10 patients (20.8%), all with poor prognosis factors. All six low responders achieved complete success. ITI outcome was significantly associated with inhibitor titre level at ITI start (P = 0.0068), number of poor prognosis factors for ITI success (P = 0.0187), monthly bleeding rate during ITI (P = 0.0005) and peak inhibitor titre during ITI (P = 0.0007). Twenty-two of 35 high responder patients (62.9%) with ≥1 poor prognosis factor achieved complete success. Treatment with a single pdFVIII/VWF concentrate, mainly according to the Bonn protocol, resulted in a high ITI success rate in haemophilia A patients with inhibitors and poor prognosis for ITI success.
metadata.dc.identifier.doi: 10.1111/hae.12774
Appears in Collections:Producción 2020

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