The Impact of Pharmacokinetic-Guided Prophylaxis on Clinical Outcomes and Healthcare Resource Utilization in Hemophilia A Patients: Real-World Evidence from the CHESS II Study

Enrico Ferri Grazzi; Shawn X Sun; Tom Burke; Jamie O'Hara

doi:10.2147/JBM.S363028

The Impact of Pharmacokinetic-Guided Prophylaxis on Clinical Outcomes and Healthcare Resource Utilization in Hemophilia A Patients: Real-World Evidence from the CHESS II Study

J Blood Med. 2022 Sep 19:13:505-516. doi: 10.2147/JBM.S363028. eCollection 2022.

Authors

Enrico Ferri Grazzi¹, Shawn X Sun², Tom Burke^{1

3}, Jamie O'Hara^{1

3}

Affiliations

¹ Health Economics and Outcomes Research, HCD Economics Ltd, Daresbury, Warrington, UK.
² Global Evidence and Outcomes, Takeda Development Center Americas, Inc, Cambridge, MA, USA.
³ Department of Health and Social Care, University of Chester, Chester, UK.

Abstract

Background: Using a pharmacokinetic (PK)-guided approach to personalize the dose and frequency of prophylactic treatment can help achieve and maintain targeted factor VIII (FVIII) trough levels in patients with hemophilia A.

Objective: Investigate clinical and healthcare resource use outcomes in patients with hemophilia A treated with or without PK-guided prophylaxis using data from the Cost of Haemophilia in Europe: A Socioeconomic Survey (CHESS) II database.

Methods: CHESS II was a cross-sectional, retrospective, burden-of-illness study incorporating data from eight European countries. Patients were eligible for this analysis if they were male, ≥18 years of age, and diagnosed with congenital hemophilia A of any severity. The clinical endpoints included annualized bleeding rate (ABR), presence and number of problem/target joints, and occurrence of joint surgeries. Healthcare resource utilization endpoints included the number of hematologist consultations and bleed-related hospitalizations or emergency department admissions. Data from November 2018 to October 2020 were included and were stratified according to treatment regimen and use of PK-guided dosing.

Results: Altogether, 281 patients on prophylaxis had available FVIII trough level data. Mean (SD) age was 35.7 (13.8) years. A specific FVIII trough level was targeted in 120 (42.7%) patients and 47 (39.2%) received PK-guided dosing. Patients receiving PK-guided dosing had a mean (SD) ABR of 2.8 (2.1) and target joint number of 0.5 (0.7), compared with 3.9 (2.7) and 0.9 (1.4), respectively, for patients receiving non-PK-guided treatment. The mean (SD) number of hematologist consultations was 7.1 (5.3) for patients receiving PK-guided dosing versus 10.7 (5.7) for those who were not. A higher proportion of patients in the non-PK-guided group required hospitalization during their lifetime compared with the PK-guided group.

Conclusion: This analysis of real-world data suggests that PK-guided dosing for prophylaxis has a beneficial impact on clinical and healthcare resource utilization outcomes in patients with hemophilia A.

Keywords: CHESS II; PK-guided dosing; hemophilia A; personalized treatment; prophylaxis; recombinant factor VIII.

Grants and funding

This study was funded by Takeda Development Center Americas, Inc., Lexington, MA, USA. Takeda were involved in study conception, design, and interpretation of results, as well as drafting and submission of the manuscript.