RT Journal Article
T1 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.
A1 Kreuz, W
A1 Escuriola Ettingshausen, C
A1 Vdovin, V
A1 Zozulya, N
A1 Plyushch, O
A1 Svirin, P
A1 Andreeva, T
A1 Bubanská, E
A1 Campos, M
A1 Benedik-Dolničar, M
A1 Jiménez-Yuste, V
A1 Kitanovski, L
A1 Klukowska, A
A1 Momot, A
A1 Osmulskaya, N
A1 Prieto, M
A1 Šalek, S Z
A1 Velasco, F
A1 Pavlova, A
A1 Oldenburg, J
A1 Knaub, S
A1 Jansen, M
A1 Belyanskaya, L
A1 Walter, O
A1 ObsITI study group, 
A1 ObsITI committee, 
K1 Bonn protocol
K1 factor VIII inhibitors
K1 factor VIII/von Willebrand factor concentrate
K1 haemophilia A
K1 immune tolerance induction
K1 poor prognosis for immune tolerance induction
AB 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.
YR 2015
FD 2015-07-23
LK http://hdl.handle.net/10668/10016
UL http://hdl.handle.net/10668/10016
LA en
DS RISalud
RD Apr 12, 2025