کد مقاله | کد نشریه | سال انتشار | مقاله انگلیسی | نسخه تمام متن |
---|---|---|---|---|
6001727 | 1182956 | 2014 | 6 صفحه PDF | دانلود رایگان |
- VWF-containing FVIII products may improve immune tolerance induction success rates.
- We analysed the use of BIOSTATE for ITI in 15 paediatric patients with inhibitors.
- BIOSTATE was used for primary ITI in 8 patients and for salvage ITI in 7 patients.
- Overall response rate was 86.6% with 11 patients achieving a complete response.
- BIOSTATE was well-tolerated and effective when used for primary or salvage ITI.
IntroductionIt has been postulated that factor VIII (FVIII) products containing von Willebrand factor (VWF) may improve immune tolerance induction (ITI) success rate in patients with haemophilia A and poor prognostic factors.Materials and methodsWe conducted a retrospective cohort analysis of a FVIII/VWF concentrate (BIOSTATE®) for ITI in paediatric patients with severe haemophilia A (SHA) and inhibitors, from January 2003 to December 2011 at 3 paediatric-only Haemophilia Treatment Centres in Australia. Response to ITI was assessed at or before 33Â months and at completion of ITI. Fifteen male patients with SHA were included in the analysis.ResultsBIOSTATE was used for primary ITI in 8 patients (2Â years, range 1.1-11.5Â years) and for salvage ITI in 7 patients (9.9Â years, range 1.1-15.4). At the end of the observation period there were 11 patients who achieved a complete response with BIOSTATE after a median duration of 21Â months (range 5-85Â months); a partial response was achieved in 2 patients in whom ITI is ongoing. Therefore, the overall response rate was 86.6%. Two patients were deemed treatment failures: one due to non-compliance after 18Â months of ITI and another in whom a partial response had not been achieved after 22Â months of ITI.ConclusionBIOSTATE was well-tolerated and effective when used for primary or salvage ITI in this cohort of paediatric patients with SHA and a high-level inhibitor.
Journal: Thrombosis Research - Volume 134, Issue 5, November 2014, Pages 1046-1051