@article{968038a1b85f4b54921aad0525266967,
title = "The impact of extended half-life factor concentrates on prophylaxis for severe hemophilia in the United States",
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 = "Malec, {Lynn M.} and Dunlei Cheng and Witmer, {Char M.} and Julie Jaffray and Kouides, {Peter A.} and Haley, {Kristina M.} and Sidonio, {Robert F.} and Kelsey Johnson and Michael Recht and Gilbert White and Croteau, {Stacy E.} and Ragni, {Margaret V.}",
note = "Funding Information: This project was supported by the DREAM Award, a mentored career development award, through the Hemostasis and Thrombosis Research Society (HTRS) and the American Thrombosis and Hemostasis Network (ATHN) which granted access to the ATHN-dataset. The DREAM Award was financially supported by Hemophilia of Georgia. The study was conducted at the Children's Hospital of Wisconsin. We aimed to quantify the number of patients treated at U.S. HTCs on prophylaxis utilizing the ATHN-dataset. The objective was to determine the impact of EHL products on the proportion of patients with severe hemophilia receiving prophylaxis, and to characterize use of prophylaxis according to age, race and ethnicity, geographic region, and payer. Additionally, we aimed to examine the impact of EHL factor products in prophylaxis in a select cohort of patients. This was done by comparing bleeding, joint health, and quality of life data among groups receiving prophylaxis vs on demand therapy, and those using EHLs vs standard half-life (SHLs) products for prophylaxis. We hypothesized that individuals receiving EHLs would have superior outcomes in these domains, as compared to those utilizing SHLs in both hemophilia A and hemophilia B. Utilizing a longitudinal observational design, the study utilized data captured from 136 HTCs participating in the United States. The ATHN-dataset was queried in June 2018 and again in March 2019 to obtain data regarding the demographics (age, race, ethnicity, hemophilia diagnosis), factor regimen (prophylaxis or on demand therapy), factor product class (EHL or SHL), factor dosing regimen (interval between doses), HTC region and payer (insurance) type. The proportion of subjects with severe hemophilia (defined as factor VIII or factor IX activity of <1%) on prophylaxis were compared to those on demand by age cohort in June 2018 and March 2019. Individuals with an active inhibitor were excluded from analysis. Funding Information: This project was supported by the DREAM Award, a mentored career development award, through the Hemostasis and Thrombosis Research Society (HTRS) and the American Thrombosis and Hemostasis Network (ATHN) which granted access to the ATHN‐dataset. The DREAM Award was financially supported by Hemophilia of Georgia. The study was conducted at the Children's Hospital of Wisconsin. Publisher Copyright: {\textcopyright} 2020 Wiley Periodicals, Inc.{\textcopyright} 2020 Wiley Periodicals, Inc. Copyright: Copyright 2020 Elsevier B.V., All rights reserved.",
year = "2020",
month = aug,
day = "1",
doi = "10.1002/ajh.25844",
language = "English (US)",
volume = "95",
pages = "960--965",
journal = "American Journal of Hematology",
issn = "0361-8609",
publisher = "Wiley-Liss Inc.",
number = "8",
}