Real-world analysis of extended half-life product posology in hemophilia a: results from a retrospective analysis of medical chart and claims data

Cedric Hermans; John Waller; Steven R Lentz

doi:10.1080/17474086.2025.2520312

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Real-world analysis of extended half-life product posology in hemophilia a: results from a retrospective analysis of medical chart and claims data

Journal article

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Real-world analysis of extended half-life product posology in hemophilia a: results from a retrospective analysis of medical chart and claims data

Cedric Hermans, John Waller and Steven R Lentz

Expert review of hematology, Vol.18(7), pp.537-545

07/03/2025

DOI: 10.1080/17474086.2025.2520312

PMID: 40528339

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Abstract

Extended half-life (EHL) products have changed the treatment landscape of patients with hemophilia as patients maintain protective FVIII levels with minimal occurrence of spontaneous bleeding. Two independent datasets from the multi-country patient record form (PRF)-based study and PicnicHealth/Komodo Health study were analyzed to understand the real-world use of EHL products for patients with hemophilia A (HA). Most patients receiving EHL prophylaxis were on per-label dosing. Those receiving individualized dosing had moderate to severe disease. EHL dose adjustment occurred in 20% of patients of which 14.3% experienced a second dose adjustment. Treatment efficacy was mainly monitored via annualized bleeding rate (ABR) with quality of life, tolerability, and treatment efficacy being the primary considerations for Healthcare Personnel when selecting treatment. Switching to a different EHL or standard half-life (SHL) product did not significantly reduce median ABR while switching to non-factor therapy significantly reduced median ABR from 5.2 to 0.94, < 0.0001. Patients on individualized dosing had higher ABRs than those on per-label dosing both before index EHL treatment and while on EHL treatment, whereas individualization via dose adjustment was associated with significant median ABR reduction ( < 0.0001). Individualization may support improved outcomes in patients unable to achieve satisfactory outcomes on a per-label dosing regimen.

Hemophilia

Extended half-life

medical records

surveys and questionnaires

factor VIII

Details

Title: Subtitle: Real-world analysis of extended half-life product posology in hemophilia a: results from a retrospective analysis of medical chart and claims data
Creators: Cedric Hermans - Cliniques Universitaires Saint-Luc
John Waller - Novo Nordisk (Switzerland)
Steven R Lentz - University of Iowa
Resource Type: Journal article
Publication Details: Expert review of hematology, Vol.18(7), pp.537-545
DOI: 10.1080/17474086.2025.2520312
PMID: 40528339
NLM abbreviation: Expert Rev Hematol
ISSN: 1747-4094
eISSN: 1747-4094
Publisher: TAYLOR & FRANCIS LTD
Grant note: Novo Nordisk Health Care AG, Zurich, SwitzerlandNovo Nordisk Health Care AG, Zurich, Switzerland
The authors thank the hemophilia specialists and patients for their participation and support in the multi-country PRF-based study and the PicnicHealth/Komodo Health study, respectively. Medical writing support was provided by Ashfield MedComms GmbH (Mannheim, Germany), an Inizio company, and funded by Novo Nordisk Health Care AG, Zurich, Switzerland.
Language: English
Electronic publication date: 06/17/2025
Date published: 07/03/2025
Academic Unit: Hematology, Oncology, and Blood & Marrow Transplantation; Internal Medicine
Record Identifier: 9984832188902771

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