Abstract
Background
Prophylactic treatment regimens lead to improvements in health-related quality-of-life (HRQoL) among individuals with hemophilia. Turoctocog alfa pegol (N8-GP) provides the benefit of extending the duration of protection from bleeding and reducing the number of injections, which is expected to impact HRQoL and treatment satisfaction (TS).
Aim
To investigate the HRQoL and TS of patients with severe hemophilia A from two phase III trials evaluating the safety and efficacy of N8-GP.
Methods
HRQoL was assessed using the Haemo-QoL (reported by children and their parents) and Haem-A-QoL (reported by adults). TS was assessed using Hemo-Sat. Domain and total scores for all questionnaires ranged from 0 to 100, with lower scores indicating a better HRQoL or TS. A negative change in score indicates an improvement in HRQoL/TS.
Results
Mean changes in HRQoL scores were reported for 14 children aged 4–7 years, 21 children aged 8–11 years, 10 adolescents aged 13–16 years, and 163 adults (17 years and above). Mean changes in children/adolescents-reported Haemo-QoL total score were -14.0 for ages 4–7 years, -3.6 for ages 8–11 years, and -0.1 for ages 13–16 years. Mean changes in parent-reported Haemo-QoL total scores were -11.5 for 4–7 years, -8.6 for ages 8–11 years, and -4.0 for 13–16 years. Adults’ mean change in Haem-A-QoL total score was -3.1 for those receiving on-demand treatment and -2.3 for those receiving prophylaxis treatment. High levels of TS with N8-GP were reported by parents of children/adolescents and the adults at the end of the trial.
Conclusion
While most patients reported a relatively good baseline HRQoL when entering the respective trials, the HRQoL of patients was either maintained or further improved when treated with N8-GP. Adults and parents of children and adolescents reported a high level of treatment satisfaction with N8-GP.
Supplementary materials
Table S1 Description of Haemo-QoL and Haem-A-QoL domains
Table S2 Pathfinder™2 and Pathfinder™5 ethics committee approval list
Acknowledgments
This study was funded by Novo Nordisk A/S Denmark. The authors thank all participating investigators, patients, and trial staff. The authors also thank Jeremy Lambert (Mapi) for medical writing assistance and editorial support in the manuscript preparation and Frank Driessler (Novo Nordisk) for their critical review during manuscript preparation.
Author contributions
LJR, SK, JO, and HT were principal investigators and enrolled and cared for patients during the trial. TPP conducted the analyses of the data, interpreted the data, and wrote and revised the manuscript. AL provided clinical input for the data analysis. XYL provided input for the analysis and interpretation of PRO data. SvM is the developer of the Haemo-QoL, Haem-A-QoL, and Hemo-Sat questionnaires and provided input for the analysis and interpretation of PRO data. All authors contributed to data analysis, drafting and revising the article, gave final approval of the version to be published, and agree to be accountable for all aspects of the work.
Disclosure
LJR was a paid consultant for Bayer, CSL Behring, Genentech, and Green Cross Inc. LJR also reports personal fees from Bayer, personal fees from Roche, outside the submitted work. SK has received grant/research support from Bayer, Bioverativ, Daiichi Sankyo, Grisfols, and Novo Nordisk, and served on speaker/advisory boards for Bayer, Bioverativ, and Novo Nordisk. SK also reports research support as the local principal investigation from Bayer, Bioverativ, Daiichi Sankyo, Grisfols, and Novo Nordisk, and work on advisory boards for Bayer, Bioverativ, and Novo Nordisk. JO has received grant/research support from Novo Nordisk and Baxter, Bayer, Biotest, CSL Behring, Grifols, Octapharma, and Pfizer, outside the submitted work, and has received personal fees and acted as a speaker for Baxter, Bayer, Biogen Idec, Biotest, CSL Behring, Grifols, Novo Nordisk, Octapharma, Swedish Orphan Biovitrum, and Pfizer. JO also reports grants and personal fees from Bayer, grants and personal fees from Biotest, personal fees from Chugai, grants, personal fees from CSL Behring, personal fees from Grifols, grants and personal fees from Novo Nordisk, grants and personal fees from Octapharma, personal fees from Pfizer, personal fees from Roche, personal fees from SOBI, and grants and personal fees from Shire, outside the submitted work. HT has received honoraria for speaking or participated in scientific advisory boards or symposia from Baxalta/Shire, Bayer, Biogen, Bioverativ, Chugai Pharmaceutical, CSL Behring, Kaketsuken, Novo Nordisk, and Pfizer, and grants from CSL Behring. AL and XYL are employees of Novo Nordisk A/S. SvM is the developer of the HRQoL and treatment satisfaction questionnaires (Haemo-QoL, Haem-A-QoL and Hemo-Sat) used in the study and a consultant of Novo Nordisk. TPP was a paid consultant for Novo Nordisk A/S as an employee of ICON plc (formerly Mapi). The authors report no other conflicts of interest in this work.