PMID- 23038998
OWN - NLM
STAT- MEDLINE
DCOM- 20130715
LR  - 20130219
IS  - 1365-2516 (Electronic)
IS  - 1351-8216 (Linking)
VI  - 19
IP  - 2
DP  - 2013 Mar
TI  - Use of Haemate((R)) P as immune tolerance induction in patients with severe 
      haemophilia A who failed previous induction attempts: a multicentre observational 
      study.
PG  - 281-6
LID - 10.1111/hae.12018 [doi]
AB  - Immune tolerance induction (ITI) can eliminate factor VIII (FVIII) inhibitory 
      antibodies that appear during FVIII replacement therapy. If first-line ITI fails, 
      switching to a different FVIII concentrate, especially one containing von 
      Willebrand factor (VWF), has been advocated. The objective of the study was to 
      assess the efficacy and safety of Haemate((R)) P, a plasma-derived FVIII 
      concentrate containing high levels of VWF, as ITI in severe haemophilia A 
      patients who had failed at least one prior ITI attempt with a different FVIII 
      concentrate. In this multicentre, observational study, Haemate((R)) P was 
      administered at a starting dose of 83-308 IU kg(-1) day(-1) (1500-6000 IU 
      day(-1)). Efficacy was assessed by standard criteria (e.g. Bethesda titre, FVIII 
      recovery and half-life), and bleeding characteristics. Nine patients from six 
      haemophilia centres were treated with Haemate((R)) P after failing one (n = 2), two 
      (n = 5) or three (n = 2) prior ITI courses. The median time from inhibitor 
      detection to Haemate((R)) P treatment was 5.4 years. The median Haemate((R)) P dose 
      was 134 IU kg(-1), and the median treatment duration 32 months. During median of 
      47 months of follow-up, complete response, partial response and treatment failure 
      were observed in one, three and five patients respectively. Five patients 
      experienced seven adverse events (AEs), including two serious AEs (sepsis). 
      Haemate((R)) P was discontinued due to an AE in one patient with a partial 
      response. Haemate((R)) P salvage ITI resulted in complete or partial tolerization 
      in four of nine patients (44%) who had failed previous ITI attempts using 
      different FVIII concentrates.
CI  - (c) 2012 Blackwell Publishing Ltd.
FAU - Rothschild, C
AU  - Rothschild C
AD  - Centre de Reference de l'Hemophilie, Hopital Necker-Enfants Malades AP-HP, Paris, 
      France.
FAU - D'Oiron, R
AU  - D'Oiron R
FAU - Borel-Derlon, A
AU  - Borel-Derlon A
FAU - Gruel, Y
AU  - Gruel Y
FAU - Navarro, R
AU  - Navarro R
FAU - Negrier, C
AU  - Negrier C
LA  - eng
PT  - Journal Article
PT  - Multicenter Study
DEP - 20121008
PL  - England
TA  - Haemophilia
JT  - Haemophilia : the official journal of the World Federation of Hemophilia
JID - 9442916
RN  - 0 (Coagulants)
RN  - 0 (Drug Combinations)
RN  - 0 (factor VIII, von Willebrand factor drug combination)
RN  - 0 (von Willebrand Factor)
RN  - 9001-27-8 (Factor VIII)
SB  - IM
MH  - Adolescent
MH  - Adult
MH  - Child
MH  - Child, Preschool
MH  - Coagulants/adverse effects/immunology/*therapeutic use
MH  - Drug Combinations
MH  - Factor VIII/adverse effects/immunology/*therapeutic use
MH  - France
MH  - Hemophilia A/*drug therapy/immunology
MH  - Humans
MH  - Immune Tolerance/*drug effects
MH  - Male
MH  - Middle Aged
MH  - Retrospective Studies
MH  - Young Adult
MH  - von Willebrand Factor/adverse effects/immunology/*therapeutic use
EDAT- 2012/10/09 06:00
MHDA- 2013/07/17 06:00
CRDT- 2012/10/09 06:00
PHST- 2012/08/12 00:00 [accepted]
PHST- 2012/10/09 06:00 [entrez]
PHST- 2012/10/09 06:00 [pubmed]
PHST- 2013/07/17 06:00 [medline]
AID - 10.1111/hae.12018 [doi]
PST - ppublish
SO  - Haemophilia. 2013 Mar;19(2):281-6. doi: 10.1111/hae.12018. Epub 2012 Oct 8.