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 - Funding Information:
Genentech, Inc and owns Roche stock. R. Kruse‐Jarres has acted as a paid consultant to Grifols, Novo Nordisk, Pfizer, Roche and Shire; and has received research funding from CSL Behring, Pfizer and Roche. S. von Mackensen is a consultant for Roche.
Funding Information:
of Gardiner‐Caldwell Communications, Daniella Babu, PhD and Amy Lindsay, PhD of Envision Pharma Group, and was funded by F. Hoffmann‐La Roche Ltd. H. Macharia, E. Asikanius, M. Lehle and P. Trask contributed to the study concept and design. Data analysis was conducted by the trial statisticians (H. Macharia and E. Asikanius), who vouch for the completeness and accuracy of the data and analy‐ ses. H. Macharia, E. Asikanius, M. Lehle and P. Trask contributed to data interpretation. Specific direction from the authors (J. Mahlangu, J. Oldenburg, M.U. Callaghan, M. Shima, M.E. Mancuso, P. Trask, M. Recht, C. Garcia, R. Yang, M. Lehle, H. Macharia, E. Asikanius, G.G. Levy, R. Kruse‐Jarres and S. von Mackensen) informed the develop‐ ment of the first draft of the manuscript by Envision Pharma Group (funded by F. Hoffmann‐La Roche Ltd). That draft was subsequently critically reviewed by all authors and revised according to their di‐ rection. All the authors had access to the data and confirm adher‐ ence to the protocol and statistical analysis plan during the conduct of the study.
Funding Information:
This NIS was conducted at 33 centres in 12 countries (Australia, China, Costa Rica, Germany, Italy, Japan, Poland, Republic of Korea, South Africa, Spain, Taiwan and the United States). The data cut‐off was 31 March 2017 for the final analysis. The study was conducted in accordance with the International Conference on Harmonisation Guidelines for Good Clinical Practice, informed consent guidelines and the Declaration of Helsinki,25 and was approved by local eth‐ ics review groups. The protocol was developed by the sponsor, F. Hoffmann‐La Roche, Ltd.
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
UR - http://www.scopus.com/inward/record.url?scp=85065048659&partnerID=8YFLogxK
U2 - 10.1111/hae.13731
DO - 10.1111/hae.13731
M3 - Article
C2 - 31016855
AN - SCOPUS:85065048659
VL - 25
SP - 382
EP - 391
JO - Haemophilia
JF - Haemophilia
SN - 1351-8216
IS - 3
ER -