Health-related quality of life and health status in persons with haemophilia A with inhibitors
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Health-related quality of life and health status in persons with haemophilia A with inhibitors : A prospective, multicentre, non-interventional study (NIS). / Mahlangu, Johnny; Oldenburg, Johannes; Callaghan, Michael U; Shima, Midori; Mancuso, Maria Elisa; Trask, Peter; Recht, Michael; Garcia, Claudia; Yang, Renchi; Lehle, Michaela; Macharia, Harrison; Asikanius, Elina; Levy, Gallia G; Kruse-Jarres, Rebecca; von Mackensen, Sylvia.
in: HAEMOPHILIA, Jahrgang 25, Nr. 3, 05.2019, S. 382-391.Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung
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TY - JOUR
T1 - Health-related quality of life and health status in persons with haemophilia A with inhibitors
T2 - A prospective, multicentre, non-interventional study (NIS)
AU - Mahlangu, Johnny
AU - Oldenburg, Johannes
AU - Callaghan, Michael U
AU - Shima, Midori
AU - Mancuso, Maria Elisa
AU - Trask, Peter
AU - Recht, Michael
AU - Garcia, Claudia
AU - Yang, Renchi
AU - Lehle, Michaela
AU - Macharia, Harrison
AU - Asikanius, Elina
AU - Levy, Gallia G
AU - Kruse-Jarres, Rebecca
AU - von Mackensen, Sylvia
N1 - © 2019 The Authors. Haemophilia Published by John Wiley & Sons Ltd.
PY - 2019/5
Y1 - 2019/5
N2 - INTRODUCTION: Real-world data (RWD) on health-related outcomes in persons with haemophilia A (PwHA) provide insights into patient needs and can guide clinical study design. A global, prospective, non-interventional study (NIS; NCT02476942) collected detailed RWD on bleeding outcomes, health-related quality of life (HRQoL) and health status in PwHA treated per local routine clinical practice.AIM: To report HRQoL and health status in the adult/adolescent PwHA with inhibitors cohort in the NIS.METHODS: This cohort enrolled PwHA aged ≥12 years with high-titre factor VIII inhibitor history. Participants remained on their usual treatment (no protocol-specified interventions). Health-related outcomes: 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).RESULTS: One hundred three participants were enrolled on episodic (n = 75) or prophylactic treatment (n = 28); median (range) age, 31 (12-75) years; median (range) observation time, 26 (4-70) weeks. Haem-A-QoL scores indicated impairments in HRQoL aspects; comparable between episodic/prophylactic regimens and relatively consistent over time. Haemo-QoL SF scores with both regimens varied over time, and appeared poorer with episodic than prophylactic treatment. IUS and EQ-VAS were comparable between regimens, stable over time and lower on bleeding days. Mean proportions of missed work and school days were 16% and 23%, respectively; mean (standard deviation) number of days hospitalized was 3.2 (8.8) (comparable between groups).CONCLUSIONS: These RWD demonstrate that PwHA with inhibitors have impaired HRQoL, despite standard treatment, and that more effective treatment options are needed.
AB - INTRODUCTION: Real-world data (RWD) on health-related outcomes in persons with haemophilia A (PwHA) provide insights into patient needs and can guide clinical study design. A global, prospective, non-interventional study (NIS; NCT02476942) collected detailed RWD on bleeding outcomes, health-related quality of life (HRQoL) and health status in PwHA treated per local routine clinical practice.AIM: To report HRQoL and health status in the adult/adolescent PwHA with inhibitors cohort in the NIS.METHODS: This cohort enrolled PwHA aged ≥12 years with high-titre factor VIII inhibitor history. Participants remained on their usual treatment (no protocol-specified interventions). Health-related outcomes: 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).RESULTS: One hundred three participants were enrolled on episodic (n = 75) or prophylactic treatment (n = 28); median (range) age, 31 (12-75) years; median (range) observation time, 26 (4-70) weeks. Haem-A-QoL scores indicated impairments in HRQoL aspects; comparable between episodic/prophylactic regimens and relatively consistent over time. Haemo-QoL SF scores with both regimens varied over time, and appeared poorer with episodic than prophylactic treatment. IUS and EQ-VAS were comparable between regimens, stable over time and lower on bleeding days. Mean proportions of missed work and school days were 16% and 23%, respectively; mean (standard deviation) number of days hospitalized was 3.2 (8.8) (comparable between groups).CONCLUSIONS: These RWD demonstrate that PwHA with inhibitors have impaired HRQoL, despite standard treatment, and that more effective treatment options are needed.
KW - Absenteeism
KW - Adolescent
KW - Adult
KW - Aged
KW - Child
KW - Female
KW - Health Status
KW - Hemophilia A/complications
KW - Hemorrhage/complications
KW - Hospitalization/statistics & numerical data
KW - Humans
KW - Male
KW - Middle Aged
KW - Prospective Studies
KW - Quality of Life
KW - Schools/statistics & numerical data
KW - Surveys and Questionnaires
KW - Work/statistics & numerical data
KW - Young Adult
U2 - 10.1111/hae.13731
DO - 10.1111/hae.13731
M3 - SCORING: Journal article
C2 - 31016855
VL - 25
SP - 382
EP - 391
JO - HAEMOPHILIA
JF - HAEMOPHILIA
SN - 1351-8216
IS - 3
ER -
