Regional variation and cost implications of prescribed extended half-life factor concentrates among U.S. Haemophilia Treatment Centres for patients with moderate and severe haemophilia.

Authors:
Stacy E Croteau
Stacy E Croteau
Boston Children's Hospital
United States
Dunlei Cheng
Dunlei Cheng
Baylor Jack and Jane Hamilton Heart and Vascular Hospital
United States
Alice J Cohen
Alice J Cohen
St. Luke's and Roosevelt Hospitals
United States
Chris E Holmes
Chris E Holmes
University of Vermont
United States
Lynn M Malec
Lynn M Malec
*Children's Hospital of Pittsburgh
Michael Silvey
Michael Silvey
Kansas City University of Medicine and Biosciences
United States
Courtney D Thornburg
Courtney D Thornburg
Duke University Medical Center
United States
Allison P Wheeler
Allison P Wheeler
Vanderbilt University

Haemophilia 2019 Apr 17. Epub 2019 Apr 17.

St. Jude Children's Research Hospital, Memphis, Tennessee.

Background: Extended half-life (EHL) factor VIII (FVIII) and IX (FIX) products are intended to decrease the burden of prophylaxis for patients with haemophilia A or B. Whether these newer concentrates have led to meaningful clinical practice change remains vague.

Aim: To characterize the longitudinal use of standard (SHL) and EHL factor concentrates at haemophilia treatment centres (HTCs), using the ATHNdataset, a US database of 138 ATHN-affiliated HTCs.

Methods: Factor concentrate use among moderate and severe haemophilia A and B patients without inhibitors was analysed at three time points over 18 months.

Results: Use of EHL concentrates rose from 10% of patients to 22% during this study. EHL FVIII prophylaxis is prescribed to the minority of patients, 28%; EHL FIX now predominates for prophylaxis, 52%. Rates of prescribed EHL products varied significantly by age group and HTC region. Median prescribed prophylaxis for SHL compared to EHL products was FVIII 6240 and 5200 and FIX 6968 and FIX 3900 IU/kg/y, respectively. On-demand EHL use has grown but has minimal contribution to overall usage (2%).

Conclusion: Haemophilia treatment centre region and patient age impact the rate of adoption of EHL products; however, EHL prescribing continues to rise nationally, particularly for EHL FIX. Careful attention to annual cost of prophylaxis is imperative as the decrease in median EHL prophylaxis consumption is not offset by the higher unit cost of these products. It is unclear how further growth in use of EHLs will be impacted by emerging non-factor replacement and gene therapies.

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Source
http://dx.doi.org/10.1111/hae.13758DOI Listing
April 2019
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References

(Supplied by CrossRef)
Real‐world analysis of dispensed international units of coagulation factor VIII and resultant expenditures for hemophilia A patients: a comparison between standard half‐life and extended half‐life products
Chhabra A et al.
Manag Care 2018

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