The effect of emicizumab prophylaxis on health-related outcomes in persons with haemophilia A with inhibitors

HAVEN 1 Study

Johannes Oldenburg, Johnny N. Mahlangu, Willem Bujan, Peter Trask, Michael U. Callaghan, Guy Young, Elina Asikanius, Flora Peyvandi, Elena Santagostino, Rebecca Kruse-Jarres, Claude Negrier, Craig Kessler, Jin Xu, Jerzy Windyga, Midori Shima, Sylvia von Mackensen

Research output: Contribution to journalArticle

6 Citations (Scopus)

Abstract

Introduction: Persons with haemophilia A (PwHA) with inhibitors to factor VIII often experience decreased health-related outcomes. In HAVEN 1 (NCT02622321), there was a statistically significant reduction in bleeding with emicizumab prophylaxis versus no prophylaxis. Aim: Describe health-related outcomes in PwHA with inhibitors in HAVEN 1. Methods: PwHA with inhibitors aged ≥12 years previously on episodic bypassing agents (BPAs) were randomized to emicizumab prophylaxis (Arm A; n = 35) or no prophylaxis (Arm B; n = 18); participants previously on BPA prophylaxis received emicizumab prophylaxis (Arm C; n = 49). Health-related outcomes assessed at baseline and monthly thereafter: Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL), Haemophilia-specific Quality of Life Questionnaire for Children Short Form (Haemo-QoL SF), EuroQol 5-Dimensions 5-Levels (EQ-5D-5L) index utility score (IUS) and visual analogue scale (EQ-VAS) and work/school days. Days hospitalized also recorded. Results: At week 25, differences (ANCOVA) in adjusted mean scores (95% confidence interval) favoured Arm A versus B for Haem-A-QoL “Total” score (14.0 [5.6, 22.5]; P = 0.002) and “Physical Health” (21.6 [7.9, 35.2]; P = 0.003); EQ-VAS (−9.7 [−17.6, −1.82]; P = 0.017); and IUS (−0.16 [−0.25, −0.07]; P = 0.001); mean scores are comparable in Arms A and C. Throughout the study, a greater proportion of participants on emicizumab prophylaxis than no prophylaxis exceeded questionnaire-specific responder thresholds. Mean proportion of missed work days and number of days hospitalized were lower with emicizumab prophylaxis than no prophylaxis. Conclusions: In PwHA with inhibitors, emicizumab prophylaxis was associated with substantial and meaningful improvements in health-related outcomes.

Original languageEnglish
Pages (from-to)33-44
JournalHaemophilia
Volume25
Issue number1
DOIs
Publication statusPublished - 2019

Fingerprint

Hemophilia A
Health
Heme
Quality of Life
Factor VIII
Visual Analog Scale
emicizumab
Confidence Intervals
Hemorrhage
Surveys and Questionnaires

Keywords

  • emicizumab
  • factor VIII inhibitors
  • haemophilia A
  • health-related quality of life
  • prophylaxis

ASJC Scopus subject areas

  • Hematology
  • Genetics(clinical)

Cite this

Oldenburg, J., Mahlangu, J. N., Bujan, W., Trask, P., Callaghan, M. U., Young, G., ... von Mackensen, S. (2019). The effect of emicizumab prophylaxis on health-related outcomes in persons with haemophilia A with inhibitors: HAVEN 1 Study. Haemophilia, 25(1), 33-44. https://doi.org/10.1111/hae.13618

The effect of emicizumab prophylaxis on health-related outcomes in persons with haemophilia A with inhibitors : HAVEN 1 Study. / Oldenburg, Johannes; Mahlangu, Johnny N.; Bujan, Willem; Trask, Peter; Callaghan, Michael U.; Young, Guy; Asikanius, Elina; Peyvandi, Flora; Santagostino, Elena; Kruse-Jarres, Rebecca; Negrier, Claude; Kessler, Craig; Xu, Jin; Windyga, Jerzy; Shima, Midori; von Mackensen, Sylvia.

In: Haemophilia, Vol. 25, No. 1, 2019, p. 33-44.

Research output: Contribution to journalArticle

Oldenburg, J, Mahlangu, JN, Bujan, W, Trask, P, Callaghan, MU, Young, G, Asikanius, E, Peyvandi, F, Santagostino, E, Kruse-Jarres, R, Negrier, C, Kessler, C, Xu, J, Windyga, J, Shima, M & von Mackensen, S 2019, 'The effect of emicizumab prophylaxis on health-related outcomes in persons with haemophilia A with inhibitors: HAVEN 1 Study', Haemophilia, vol. 25, no. 1, pp. 33-44. https://doi.org/10.1111/hae.13618
Oldenburg, Johannes ; Mahlangu, Johnny N. ; Bujan, Willem ; Trask, Peter ; Callaghan, Michael U. ; Young, Guy ; Asikanius, Elina ; Peyvandi, Flora ; Santagostino, Elena ; Kruse-Jarres, Rebecca ; Negrier, Claude ; Kessler, Craig ; Xu, Jin ; Windyga, Jerzy ; Shima, Midori ; von Mackensen, Sylvia. / The effect of emicizumab prophylaxis on health-related outcomes in persons with haemophilia A with inhibitors : HAVEN 1 Study. In: Haemophilia. 2019 ; Vol. 25, No. 1. pp. 33-44.
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abstract = "Introduction: Persons with haemophilia A (PwHA) with inhibitors to factor VIII often experience decreased health-related outcomes. In HAVEN 1 (NCT02622321), there was a statistically significant reduction in bleeding with emicizumab prophylaxis versus no prophylaxis. Aim: Describe health-related outcomes in PwHA with inhibitors in HAVEN 1. Methods: PwHA with inhibitors aged ≥12 years previously on episodic bypassing agents (BPAs) were randomized to emicizumab prophylaxis (Arm A; n = 35) or no prophylaxis (Arm B; n = 18); participants previously on BPA prophylaxis received emicizumab prophylaxis (Arm C; n = 49). Health-related outcomes assessed at baseline and monthly thereafter: Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL), Haemophilia-specific Quality of Life Questionnaire for Children Short Form (Haemo-QoL SF), EuroQol 5-Dimensions 5-Levels (EQ-5D-5L) index utility score (IUS) and visual analogue scale (EQ-VAS) and work/school days. Days hospitalized also recorded. Results: At week 25, differences (ANCOVA) in adjusted mean scores (95{\%} confidence interval) favoured Arm A versus B for Haem-A-QoL “Total” score (14.0 [5.6, 22.5]; P = 0.002) and “Physical Health” (21.6 [7.9, 35.2]; P = 0.003); EQ-VAS (−9.7 [−17.6, −1.82]; P = 0.017); and IUS (−0.16 [−0.25, −0.07]; P = 0.001); mean scores are comparable in Arms A and C. Throughout the study, a greater proportion of participants on emicizumab prophylaxis than no prophylaxis exceeded questionnaire-specific responder thresholds. Mean proportion of missed work days and number of days hospitalized were lower with emicizumab prophylaxis than no prophylaxis. Conclusions: In PwHA with inhibitors, emicizumab prophylaxis was associated with substantial and meaningful improvements in health-related outcomes.",
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AU - Mahlangu, Johnny N.

AU - Bujan, Willem

AU - Trask, Peter

AU - Callaghan, Michael U.

AU - Young, Guy

AU - Asikanius, Elina

AU - Peyvandi, Flora

AU - Santagostino, Elena

AU - Kruse-Jarres, Rebecca

AU - Negrier, Claude

AU - Kessler, Craig

AU - Xu, Jin

AU - Windyga, Jerzy

AU - Shima, Midori

AU - von Mackensen, Sylvia

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N2 - Introduction: Persons with haemophilia A (PwHA) with inhibitors to factor VIII often experience decreased health-related outcomes. In HAVEN 1 (NCT02622321), there was a statistically significant reduction in bleeding with emicizumab prophylaxis versus no prophylaxis. Aim: Describe health-related outcomes in PwHA with inhibitors in HAVEN 1. Methods: PwHA with inhibitors aged ≥12 years previously on episodic bypassing agents (BPAs) were randomized to emicizumab prophylaxis (Arm A; n = 35) or no prophylaxis (Arm B; n = 18); participants previously on BPA prophylaxis received emicizumab prophylaxis (Arm C; n = 49). Health-related outcomes assessed at baseline and monthly thereafter: Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL), Haemophilia-specific Quality of Life Questionnaire for Children Short Form (Haemo-QoL SF), EuroQol 5-Dimensions 5-Levels (EQ-5D-5L) index utility score (IUS) and visual analogue scale (EQ-VAS) and work/school days. Days hospitalized also recorded. Results: At week 25, differences (ANCOVA) in adjusted mean scores (95% confidence interval) favoured Arm A versus B for Haem-A-QoL “Total” score (14.0 [5.6, 22.5]; P = 0.002) and “Physical Health” (21.6 [7.9, 35.2]; P = 0.003); EQ-VAS (−9.7 [−17.6, −1.82]; P = 0.017); and IUS (−0.16 [−0.25, −0.07]; P = 0.001); mean scores are comparable in Arms A and C. Throughout the study, a greater proportion of participants on emicizumab prophylaxis than no prophylaxis exceeded questionnaire-specific responder thresholds. Mean proportion of missed work days and number of days hospitalized were lower with emicizumab prophylaxis than no prophylaxis. Conclusions: In PwHA with inhibitors, emicizumab prophylaxis was associated with substantial and meaningful improvements in health-related outcomes.

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