Faculty Collaboration Database

The impact of extended half-life factor concentrates on prophylaxis for severe hemophilia in the United States. Am J Hematol 2020 Aug;95(8):960-965

Date

05/02/2020

Pubmed ID

32356313

DOI

10.1002/ajh.25844

Scopus ID

2-s2.0-85087914846 (requires institutional sign-in at Scopus site)   18 Citations

Abstract

With licensure of extended half-life (EHL) factor products and the changing landscape of available hemophilia products, patients and providers have options for less treatment-intense prophylaxis. The impact of these products in clinical practice to date remains understudied. We aimed to quantify the use of EHL products in prophylaxis in the US using the ATHN-dataset, a database of 145 ATHN-affiliated hemophilia treatment centers (HTCs). Further, we aimed to quantify the impact of EHL on key hemophilia indicators including annualized bleed rates (ABRs), hemophilia joint health scores (HJHS) and quality of life (QOL) metrics. The use of EHL vs standard half-life (SHL) products in severe hemophilia was compared between June 2018 and March 2019 using the ATHN-dataset. A cohort of patients was also recruited from seven participating HTCs in order to compare ABR, HJHS and QOL between product classes. By March 2019 the number of individuals with severe Hemophilia A (SHA) receiving EHLs remained relatively stable (28.4%), whereas the number of prescribed non-factor products increased to 7.1%, with a diminishing majority of patients (64.0%) continuing to receive SHLs. The majority of patients with severe hemophilia B (SHB) received treatment with EHLs including 57.5% by March 2019. There was a trend toward lower ABR with use of EHLs in SHA and SHB, although this did not result in improved HJHS nor QOL. EHL use in the United States in severe hemophilia continues to increase, although at a slower rate in SHA with the availability of non-factor therapy. The impact of the EHL therapies in clinical practice should continue to be examined prospectively.

Author List

Malec LM, Cheng D, Witmer CM, Jaffray J, Kouides PA, Haley KM, Sidonio RF Jr, Johnson K, Recht M, White G, Croteau SE, Ragni MV

Author

Lynn M. Malec MD Associate Professor in the Medicine department at Medical College of Wisconsin

MESH terms used to index this publication - Major topics in bold

Adolescent
Adult
Aged
Cohort Studies
Female
Half-Life
Hemophilia A
Humans
Male
Middle Aged
United States
Young Adult