TY - JOUR
T1 - Concizumab prophylaxis in persons with hemophilia A or B with inhibitors
T2 - patient-reported outcome results from the phase 3 explorer7 study
AU - Tran, Huyen
AU - von Mackensen, Sylvia
AU - Abraham, Aby
AU - Castaman, Giancarlo
AU - Hampton, Kingsley
AU - Knoebl, Paul
AU - Linari, Silvia
AU - Odgaard-Jensen, Jan
AU - Neergaard, Jesper Skov
AU - Stasyshyn, Oleksandra
AU - Thaung Zaw, Jay Jay
AU - Zulfikar, Bulent
AU - Shapiro, Amy
N1 - Funding Information:
The authors would like to thank the patients, their families, the Data Monitoring Committee, and the study site investigators, nurses, coordinators, and staff. The authors are grateful to Julien Bovet for their critical review of the manuscript. Medical writing support for the development of this manuscript, under the direction of the authors, was provided by Ashfield MedComms GmbH (Mannheim, Germany), an Inizio company, and funded by Novo Nordisk A/S (Denmark).
Funding Information:
A.A.: Research and travel grant from Roche and Novo Nordisk . Honoraria from Novo Nordisk.
Funding Information:
H.T.: Research project grants from Sanofi and AstraZeneca . Honoraria from Roche, CSL-Behring, Novo Nordisk, and Pfizer.
Funding Information:
B.Z.: Research funding from Pfizer and Sobi. Honoraria for advisory boards from Genveon, Novo Nordisk, Pfizer, Roche, and Takeda.
Publisher Copyright:
© 2024 The Authors
PY - 2024/5
Y1 - 2024/5
N2 - Background: Patient-reported outcomes (PROs) reflect patient perceptions of disease and treatment and are important for evaluating new therapies. Objectives: Evaluate the effects of once-daily concizumab prophylaxis on health-related quality of life (HRQoL), treatment burden, and treatment preference in males aged ≥12 years with hemophilia A/B with inhibitors. Methods: Patients enrolled in the multicenter, open-label explorer7 phase 3 study (ClinicalTrials.gov identifier: NCT04083781) were randomized to receive no prophylaxis (arm 1) or concizumab prophylaxis (arm 2) or were nonrandomly allocated to concizumab prophylaxis (arms 3 and 4). The study included questionnaires to assess patients’ perception of HRQoL (Haemophilia Quality of Life Questionnaire for Adults), treatment burden (Hemophilia Treatment Experience Measure), and treatment preference (Haemophilia Patient Preference Questionnaire). Results: The estimated treatment difference between patients receiving concizumab prophylaxis vs no prophylaxis at week 24 for Haemophilia Quality of Life Questionnaire for Adults “total score” was −22.6 points (95% CI, −42.5; −2.7), directionally favoring patients receiving concizumab prophylaxis. For Hemophilia Treatment Experience Measure “total score,” the estimated treatment difference was −19.9 points (95% CI, −34.3, −5.6) in favor of concizumab vs no prophylaxis. The majority of patients receiving concizumab expressed a preference for concizumab over their previous treatment, the main reasons being “fewer bleeds,” “require less time,” and “less painful to inject.” Across all PROs, there were less responses collected than anticipated, limiting interpretations. Conclusion: PROs collected during the explorer7 study showed improvements in some domains of HRQoL, treatment burden, and patient treatment preference in persons with hemophilia A or B with inhibitors receiving concizumab prophylaxis compared with no prophylaxis.
AB - Background: Patient-reported outcomes (PROs) reflect patient perceptions of disease and treatment and are important for evaluating new therapies. Objectives: Evaluate the effects of once-daily concizumab prophylaxis on health-related quality of life (HRQoL), treatment burden, and treatment preference in males aged ≥12 years with hemophilia A/B with inhibitors. Methods: Patients enrolled in the multicenter, open-label explorer7 phase 3 study (ClinicalTrials.gov identifier: NCT04083781) were randomized to receive no prophylaxis (arm 1) or concizumab prophylaxis (arm 2) or were nonrandomly allocated to concizumab prophylaxis (arms 3 and 4). The study included questionnaires to assess patients’ perception of HRQoL (Haemophilia Quality of Life Questionnaire for Adults), treatment burden (Hemophilia Treatment Experience Measure), and treatment preference (Haemophilia Patient Preference Questionnaire). Results: The estimated treatment difference between patients receiving concizumab prophylaxis vs no prophylaxis at week 24 for Haemophilia Quality of Life Questionnaire for Adults “total score” was −22.6 points (95% CI, −42.5; −2.7), directionally favoring patients receiving concizumab prophylaxis. For Hemophilia Treatment Experience Measure “total score,” the estimated treatment difference was −19.9 points (95% CI, −34.3, −5.6) in favor of concizumab vs no prophylaxis. The majority of patients receiving concizumab expressed a preference for concizumab over their previous treatment, the main reasons being “fewer bleeds,” “require less time,” and “less painful to inject.” Across all PROs, there were less responses collected than anticipated, limiting interpretations. Conclusion: PROs collected during the explorer7 study showed improvements in some domains of HRQoL, treatment burden, and patient treatment preference in persons with hemophilia A or B with inhibitors receiving concizumab prophylaxis compared with no prophylaxis.
KW - concizumab
KW - health-related quality of life
KW - hemophilia A
KW - hemophilia B
KW - patient preference
KW - patient-reported outcomes
UR - https://www.scopus.com/pages/publications/85198040538
U2 - 10.1016/j.rpth.2024.102476
DO - 10.1016/j.rpth.2024.102476
M3 - Article
C2 - 39099801
AN - SCOPUS:85198040538
SN - 2475-0379
VL - 8
JO - Research and Practice in Thrombosis and Haemostasis
JF - Research and Practice in Thrombosis and Haemostasis
IS - 4
M1 - e102476
ER -