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 - Publisher Copyright:
© 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 - alloantibodies
KW - haemophilia
KW - health-related quality of life
KW - inhibitors
KW - non-interventional
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U2 - 10.1111/hae.13731
DO - 10.1111/hae.13731
M3 - Article
C2 - 31016855
AN - SCOPUS:85065048659
SN - 1351-8216
VL - 25
SP - 382
EP - 391
JO - Haemophilia
JF - Haemophilia
IS - 3
ER -