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, Jahrgang 25, Nr. 1, 01.2019, S. 33-44.Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung
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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 -