Real-world Bleeding Outcomes and Product Utilization in People with Severe-type Hemophilia A Before and After Switching to Extended Half-life RFVIIIFc Prophylaxis Therapy

Overview

Journal Int J Hematol

Specialty Hematology

Date 2022 Dec 4

PMID 36463568

Authors

Chia-Yau Chang

Shiue-Wei Lai

Mei-Mei Cheng

Jung-Tzu Ku

Shu-Hsia Hu

Yen-Lin Liu

Jia-Ruey Tsai

Chen-Hua Tsai

Chao-Neng Cheng

Yeu-Chin Chen

Affiliations

Soon will be listed here.

Abstract

Background: Recombinant factor VIII-Fc (rFVIIIFc) became available in Taiwan in 2018. Before this date, no people with hemophilia A (PwHA) were enrolled in a clinical trial of rFVIIIFc. We investigated changes in bleeding outcomes and product utilization in PwHA switching from rFVIII to rFVIIIFc.

Methods: Data were collected for Taiwanese PwHA (severe-type) who switched from rFVIII to rFVIIIFc, including annualized bleeding rate (ABR) and weekly dose consumption 12 months pre-switch and > 6 months post-switch.

Results: The 51 patients were divided into 3 groups according to their pre-switch treatment: on-demand treatment, intermittent periodic prophylaxis, and regular prophylaxis. In every group, the post-switch median ABR was significantly reduced, with no significant differences between groups. Meanwhile, the post-switch median weekly dose of each group was significantly increased. In 32 patients on pre-switch prophylaxis, switching brought a further reduction in median ABR, associated with a significant increase in median weekly dose. No adverse effects or novel inhibitor development were seen.

Conclusion: This is the first report from Asia on real-world experience of rFVIIIFc, showing that switching to rFVIIIFc prophylaxis led to further reduction in ABR and increase in weekly dose for all patient groups, even those on pre-switch rFVIII prophylaxis.

Citing Articles

Clinical Predictors and Prediction Models for rFVIII-Fc Half Life in Real-World People with Severe Hemophilia A.

Chang C, Chiou S, Weng T, Lin P, Lai S, Tsai C J Clin Med. 2023; 12(6).

PMID: 36983209 PMC: 10053229. DOI: 10.3390/jcm12062207.

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