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

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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, 01.2019, p. 33-44.

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

Harvard

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

APA

Oldenburg, J., Mahlangu, J. N., Bujan, W., Trask, P., Callaghan, M. U., 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, 25(1), 33-44. https://doi.org/10.1111/hae.13618

Vancouver

Oldenburg J, Mahlangu JN, Bujan W, Trask P, Callaghan MU, Young G et al. The effect of emicizumab prophylaxis on health-related outcomes in persons with haemophilia A with inhibitors: HAVEN 1 Study. HAEMOPHILIA. 2019 Jan;25(1):33-44. https://doi.org/10.1111/hae.13618

Bibtex

@article{d1309b4a75f14a47bb8aead8443958be,
title = "The effect of emicizumab prophylaxis on health-related outcomes in persons with haemophilia A with inhibitors: HAVEN 1 Study",
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.",
keywords = "Adolescent, Adult, Aged, Antibodies, Bispecific/therapeutic use, Antibodies, Monoclonal, Humanized/therapeutic use, Child, Factor VIII/antagonists & inhibitors, Health Status, Hemophilia A/drug therapy, Hospitalization, Humans, Isoantibodies/blood, Male, Middle Aged, Quality of Life, Young Adult",
author = "Johannes Oldenburg and Mahlangu, {Johnny N} and Willem Bujan and Peter Trask and Callaghan, {Michael U} and Guy Young and Elina Asikanius and Flora Peyvandi and Elena Santagostino and Rebecca Kruse-Jarres and Claude Negrier and Craig Kessler and Jin Xu and Jerzy Windyga and Midori Shima and {von Mackensen}, Sylvia",
note = "{\textcopyright} 2018 John Wiley & Sons Ltd.",
year = "2019",
month = jan,
doi = "10.1111/hae.13618",
language = "English",
volume = "25",
pages = "33--44",
journal = "HAEMOPHILIA",
issn = "1351-8216",
publisher = "Wiley-Blackwell",
number = "1",

}

RIS

TY - JOUR

T1 - The effect of emicizumab prophylaxis on health-related outcomes in persons with haemophilia A with inhibitors

T2 - HAVEN 1 Study

AU - Oldenburg, Johannes

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

N1 - © 2018 John Wiley & Sons Ltd.

PY - 2019/1

Y1 - 2019/1

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.

AB - 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.

KW - Adolescent

KW - Adult

KW - Aged

KW - Antibodies, Bispecific/therapeutic use

KW - Antibodies, Monoclonal, Humanized/therapeutic use

KW - Child

KW - Factor VIII/antagonists & inhibitors

KW - Health Status

KW - Hemophilia A/drug therapy

KW - Hospitalization

KW - Humans

KW - Isoantibodies/blood

KW - Male

KW - Middle Aged

KW - Quality of Life

KW - Young Adult

U2 - 10.1111/hae.13618

DO - 10.1111/hae.13618

M3 - SCORING: Journal article

C2 - 30427582

VL - 25

SP - 33

EP - 44

JO - HAEMOPHILIA

JF - HAEMOPHILIA

SN - 1351-8216

IS - 1

ER -