Authors' objectives: To determine whether extended half-life factor VIII and IX clotting concentrates were at least as safe and effective as standard half-life clotting factors, over and above pharmacokinetic parameters, for treating clotting disorders haemophilia A and B

Authors' results and conclusions: Haemophilia A - Seven extended half-life (EHL) factor VIII products were reviewed. The primary clinical outcome of interest was annualised bleeding rates (ABR). With the exception of human cl-rFVIII (which should possibly not be classified as an EHL product), the EHL products examined, appeared as safe as standard half-life (SHL) products, and more effective than SHL products, over and above pharmacokinetic parameters, for treating haemophilia A. Overall, the quality of the evidence was considered to be very poor. Limitations include the lack of direct comparison on clinical outcomes; inconsistent use of the type of estimate (e.g. means and medians across studies); and lack of estimates of variance. The study populations across all studies was highly consistent in terms of disease severity and cut-offs for age. Haemophilia B - Three EHL factor IX products were reviewed. The primary clinical outcome of interest was ABR. All studies compared prophylaxis with SHL FIX products (historical data) with prophylaxis with EHL FIX products (trial data). Bleeding rates were reduced through the use of EHL products. Likewise, bleeding rates in those treated on-demand with EHL products were reduced compared to historical bleeding rates in those treated on-demand with SHL products. Quality of life improvements were seen in groups of patients receiving EHL product prophylactically, particularly if they had been on an on-demand regimen prior to trial entry. Real world data showed that adherence to treatment regimens was considerably higher when patients were on a treatment regimen with EHL products rather than SHL products. There were no data to suggest that EHL products are associated with a higher rate of adverse events than SHL products. Limitations of the data were that all trials were restricted to patients with moderately-severe to severe haemophilia B, previously treated patients, with no history of inhibitors. The safety and effectiveness of EHL products outside of this population is therefore unknown.

Project Status: Completed

Year Published: 2017

URL for published report: https://view.officeapps.live.com/op/view.aspx?src=https%3A%2F%2Fblood.gov.au%2Fsystem%2Ffiles%2Fdocuments%2FAppendix2-Assessment-Extended-Half-Life-Clotting-Factor-Concentrates.docx&wdOrigin=BROWSELINK

English language abstract: An English language summary is available

Publication Type: Other

Country: Australia

Organisation Name: Adelaide Health Technology Assessment

Contact Address: School of Public Health, Mail Drop 545, University of Adelaide, Adelaide SA 5005, AUSTRALIA, Tel: +61 8 8313 4617

Contact Name: ahta@adelaide.edu.au

Contact Email: ahta@adelaide.edu.au

Copyright: <p>Adelaide Health Technology Assessment (AHTA) on behalf of NICS</p>