Efficacy, safety and cost of emicizumab prophylaxis in haemophilia A patients with inhibitors: A nationwide observational study in Taiwan

Ming Ching Shen, Sheng Chieh Chou, Shyh Shin Chiou, Pei Chin Lin, Yeu Chin Chen, Hsuan Yu Lin, Yang Cheng Lee, Cih En Huang, Te Fu Weng, Ting Huan Huang, Chih Yuan Chung, Jiann Shiuh Chen, Shu Huey Chen, Shin Nan Cheng, Chih Cheng Hsiao, Yen Min Huang, Shih Hsiang Chen, Yuan Bin Yu, Shih Chiang Lin, Ching Yeh LinChing Tien Peng, Jiaan Der Wang*

*Corresponding author for this work

Research output: Contribution to journalJournal Article peer-review

Abstract

INTRODUCTION: Emicizumab mimicking the cofactor function of activated factor VIII (FVIII) restores haemostasis.

METHODS: This nationwide observational study aimed to retrospectively investigate efficacy, safety, and cost in 1 year before and up to 3 years after emicizumab prophylaxis for haemophilia A (HA) patients with FVIII inhibitors.

RESULTS AND DISCUSSION: A total of 39 severe HA patients with a median age of 23.0 years were enrolled. The median historical peak FVIII inhibitor titre was 174.2 BU/mL with an interquartile range of 56.5-578.8 BU/mL. The median annualized bleeding rate reduced from 24 to 0 events in the first year after emicizumab prophylaxis (p < .01) and sustained in the second and third years. The median annualized joint bleeding rate reduced to 0 and maintained up to 3 years (p < .01). Twenty-seven patients (69.2%) had target joints before emicizumab prophylaxis and only seven patients (17.9%) of them had target joints after prophylaxis. Medical costs, including cost of haemostatic therapy, frequency of outpatient department visits, emergency room visits and hospital admission, were significantly reduced after emicizumab prophylaxis (p < .01). FVIII inhibitor titre decreased after emicizumab prophylaxis. Overall, three (7.7%) patients experienced 202 grade 1 drug-related adverse events after emicizumab prophylaxis. No serious adverse events were reported during emicizumab prophylaxis period. The adherence to emicizumab prophylaxis was 100% up to 3 years.

CONCLUSIONS: HA patients with FVIII inhibitors treated with emicizumab prophylaxis resulted in a significant reduction in treated bleeds and associated costs. No new safety events were observed.

Original languageEnglish
Pages (from-to)1499-1508
Number of pages10
JournalHaemophilia
Volume29
Issue number6
DOIs
StatePublished - 11 2023
Externally publishedYes

Bibliographical note

© 2023 John Wiley & Sons Ltd.

Keywords

  • cost
  • efficacy
  • emicizumab
  • factor VIII inhibitors
  • haemophilia A
  • safety
  • Humans
  • Factor VIII/therapeutic use
  • Young Adult
  • Antibodies, Bispecific/adverse effects
  • Hemorrhage/prevention & control
  • Hemophilia A/complications
  • Taiwan
  • Adult
  • Retrospective Studies

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