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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

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

In: HAEMOPHILIA, Vol. 27, No. 5, 09.2021, p. 854-865.

Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review

Harvard

Skinner, MW, Négrier, C, Paz-Priel, I, Chebon, S, Jiménez-Yuste, V, Callaghan, MU, Lehle, M, Niggli, M, Mahlangu, J, Shapiro, A, Shima, M, Campinha-Bacote, A, Levy, GG, Oldenburg, J, von Mackensen, S & Pipe, SW 2021, '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', HAEMOPHILIA, vol. 27, no. 5, pp. 854-865. https://doi.org/10.1111/hae.14363

APA

Skinner, M. W., Négrier, C., Paz-Priel, I., Chebon, S., Jiménez-Yuste, V., Callaghan, M. U., Lehle, M., Niggli, M., Mahlangu, J., Shapiro, A., Shima, M., Campinha-Bacote, A., Levy, G. G., Oldenburg, J., von Mackensen, S., & Pipe, S. W. (2021). 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. HAEMOPHILIA, 27(5), 854-865. https://doi.org/10.1111/hae.14363

Vancouver

Skinner MW, Négrier C, Paz-Priel I, Chebon S, Jiménez-Yuste V, Callaghan MU et al. 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. HAEMOPHILIA. 2021 Sep;27(5):854-865. https://doi.org/10.1111/hae.14363

Bibtex

@article{2ad9717628414070af70c178548515f0,
title = "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",
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.",
author = "Skinner, {Mark W} and Claude N{\'e}grier and Ido Paz-Priel and Sammy Chebon and Victor Jim{\'e}nez-Yuste and Callaghan, {Michael U} and Michaela Lehle and Markus Niggli and Johnny Mahlangu and Amy Shapiro and Midori Shima and Avrita Campinha-Bacote and Levy, {Gallia G} and Johannes Oldenburg and {von Mackensen}, Sylvia and Pipe, {Steven W}",
note = "{\textcopyright} 2021 The Authors. Haemophilia published by John Wiley & Sons Ltd.",
year = "2021",
month = sep,
doi = "10.1111/hae.14363",
language = "English",
volume = "27",
pages = "854--865",
journal = "HAEMOPHILIA",
issn = "1351-8216",
publisher = "Wiley-Blackwell",
number = "5",

}

RIS

TY - JOUR

T1 - 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

AU - Skinner, Mark W

AU - Négrier, Claude

AU - Paz-Priel, Ido

AU - Chebon, Sammy

AU - Jiménez-Yuste, Victor

AU - Callaghan, Michael U

AU - Lehle, Michaela

AU - Niggli, Markus

AU - Mahlangu, Johnny

AU - Shapiro, Amy

AU - Shima, Midori

AU - Campinha-Bacote, Avrita

AU - Levy, Gallia G

AU - Oldenburg, Johannes

AU - von Mackensen, Sylvia

AU - Pipe, Steven W

N1 - © 2021 The Authors. Haemophilia published by John Wiley & Sons Ltd.

PY - 2021/9

Y1 - 2021/9

N2 - 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.

AB - 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.

U2 - 10.1111/hae.14363

DO - 10.1111/hae.14363

M3 - SCORING: Journal article

C2 - 34171159

VL - 27

SP - 854

EP - 865

JO - HAEMOPHILIA

JF - HAEMOPHILIA

SN - 1351-8216

IS - 5

ER -