Abstract |
Immune tolerance induction (ITI) has been shown to successfully eliminate factor VIII (FVIII) inhibitors in haemophilia patients with inhibitors. We performed a literature search to identify reports from January 1980 to October 2012 on the use of the plasma-derived, von Willebrand factor (VWF)-containing FVIII concentrate Haemate P/ Humate-P in the setting of ITI. Six reports were identified that specifically evaluated the use of Haemate P/ Humate-P including 32 children and 9 adults. Dosing regimens ranged from 20 IU kg(-1) every 2-3 days in patients with low-responding (LR; n = 5) inhibitors to 300 IU kg(-1) day(-1) in patients with high-responding (HR; n = 36) inhibitors. Complete success was achieved in all five LR patients, in all three HR patients with good prognostic factors (age ≤7 years, pre-ITI inhibitor titre <10 BU, historical inhibitor titre <200 BU, time between inhibitor detection and ITI start <2 years), and in 24 of 33 (73%) HR patients with poor prognostic factors. The time to complete success was 0.5-4 months in good-prognosis patients and 0.5-42 months in poor-prognosis patients. Few adverse events were observed during ITI, and no cases of inhibitor relapse were reported with follow-up periods of up to 12 years. On the basis of this retrospective review of a diverse range of studies and case reports, we conclude that Haemate P/ Humate-P for ITI in patients with inhibitors is effective and produces high rates of ITI success.
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Authors | C Escuriola Ettingshausen, W Kreuz |
Journal | Haemophilia : the official journal of the World Federation of Hemophilia
(Haemophilia)
Vol. 20
Issue 3
Pg. 333-9
(May 2014)
ISSN: 1365-2516 [Electronic] England |
PMID | 24165472
(Publication Type: Journal Article, Review)
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Copyright | © 2013 John Wiley & Sons Ltd. |
Chemical References |
- Drug Combinations
- factor VIII, von Willebrand factor drug combination
- von Willebrand Factor
- Factor VIII
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Topics |
- Child
- Child, Preschool
- Drug Combinations
- Factor VIII
(therapeutic use)
- Female
- Hemophilia A
(drug therapy, immunology)
- Humans
- Immune Tolerance
- Male
- von Willebrand Factor
(therapeutic use)
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