Concizumab prophylaxis in persons with hemophilia A or B with inhibitors

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 - © 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.

U2 - 10.1016/j.rpth.2024.102476

DO - 10.1016/j.rpth.2024.102476

M3 - SCORING: Journal article

C2 - 39099801

VL - 8

SP - 102476

JO - RES PRACT THROMB HAE

JF - RES PRACT THROMB HAE

SN - 2475-0379

IS - 4

ER -