The effect of emicizumab prophylaxis on long-term, self-reported physical health in persons with haemophilia A without factor VIII inhibitors in the HAVEN 3 and HAVEN 4 studies

Mark W. Skinner, Claude Négrier, Ido Paz-Priel, Sammy Chebon, Victor Jiménez-Yuste, Michael U. Callaghan, Michaela Lehle, Markus Niggli, Johnny Mahlangu, Amy Shapiro, Midori Shima, Avrita Campinha-Bacote, Gallia G. Levy, Johannes Oldenburg, Sylvia von Mackensen, Steven W. Pipe

Research output: Contribution to journalArticlepeer-review

7 Scopus citations

Abstract

Introduction: Severe haemophilia A (HA) has a major impact on health-related quality of life (HRQoL). Aim: Assess the impact of emicizumab on HRQoL in persons with severe HA (PwHA) without factor VIII (FVIII) inhibitors in the phase 3 HAVEN 3 and 4 studies. Methods: This pooled analysis examines the HRQoL of PwHA aged ≥ 18 years treated with emicizumab prophylaxis via Haemophilia-Specific Quality of Life Questionnaire for Adults (Haem-A-QoL) and EuroQoL 5-Dimensions 5-levels (EQ-5D-5L). In particular, changes from baseline in Haem-A-QoL ‘Physical Health’ (PH) domain and ‘Total Score’ (TS) are evaluated. Results: Among 176 evaluable participants, 96 (55%) had received prior episodic treatment and 80 (45%) prophylaxis; 70% had ≥ 1 target joint and 51% had experienced ≥ 9 bleeds in the previous 24 weeks. Mean Haem-A-QoL PH and TS improved after emicizumab initiation. Mean (standard deviation) –12.0 (21.26)- and –8.6 (12.57)-point improvements were observed in PH and TS from baseline to Week 73; Week 73 scores were 27.9 (24.54) and 22.0 (14.38), respectively. Fifty-four percent of participants reported a clinically meaningful improvement in PH scores (≥ 10 points) by Week 73. Subgroups with poorer HRQoL prior to starting emicizumab (i.e. receiving episodic treatment, ≥ 9 bleeds, target joints) had the greatest improvements in PH scores, and corresponding reductions in missed workdays; change was not detected among those previously taking prophylaxis. No change over time was detected by the EQ-5D-5L questionnaire. Conclusions: Emicizumab prophylaxis in PwHA without FVIII inhibitors resulted in persistent and meaningful improvements in Haem-A-QoL PH and less work disruption than previous treatment.

Original languageEnglish
Pages (from-to)854-865
Number of pages12
JournalHaemophilia
Volume27
Issue number5
DOIs
StatePublished - Sep 2021

Keywords

  • emicizumab
  • haemophilia A
  • health-related quality of life
  • prophylaxis
  • therapeutic
  • work

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