Your browser doesn't support javascript.
loading
Concizumab prophylaxis in persons with hemophilia A or B with inhibitors: patient-reported outcome results from the phase 3 explorer7 study.
Tran, Huyen; von Mackensen, Sylvia; Abraham, Aby; Castaman, Giancarlo; Hampton, Kingsley; Knoebl, Paul; Linari, Silvia; Odgaard-Jensen, Jan; Neergaard, Jesper Skov; Stasyshyn, Oleksandra; Thaung Zaw, Jay Jay; Zulfikar, Bulent; Shapiro, Amy.
Afiliação
  • Tran H; Ronald Sawers Haemophilia Centre, The Alfred Hospital, Melbourne, Victoria, Australia.
  • von Mackensen S; Australian Centre for Blood Diseases, Monash University, Melbourne, Victoria, Australia.
  • Abraham A; Department of Medical Psychology, University Medical Centre Hamburg-Eppendorf, Hamburg, Germany.
  • Castaman G; Department of Hematology, Christian Medical College, Vellore, India.
  • Hampton K; Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy.
  • Knoebl P; Department of Haematology, Royal Hallamshire Hospital, Sheffield, UK.
  • Linari S; Division of Hematology and Hemostasis, Department of Medicine 1, Medical University of Vienna, Vienna, Austria.
  • Odgaard-Jensen J; Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy.
  • Neergaard JS; Biostatistics HTA, Novo Nordisk A/S, Søborg, Denmark.
  • Stasyshyn O; Safety Surveillance CVD I, Novo Nordisk A/S, Søborg, Denmark.
  • Thaung Zaw JJ; Institute of Blood Pathology and Transfusion Medicine, Lviv, Ukraine.
  • Zulfikar B; Medical & Science Rare Bleeding Disorders, Novo Nordisk A/S, Søborg, Denmark.
  • Shapiro A; Division of Pediatric Hematology-Oncology, Istanbul University Oncology Institute, Istanbul, Turkey.
Res Pract Thromb Haemost ; 8(4): 102476, 2024 May.
Article em En | MEDLINE | ID: mdl-39099801
ABSTRACT

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.
Palavras-chave

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Revista: Res Pract Thromb Haemost Ano de publicação: 2024 Tipo de documento: Article País de afiliação: Austrália País de publicação: Estados Unidos

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Revista: Res Pract Thromb Haemost Ano de publicação: 2024 Tipo de documento: Article País de afiliação: Austrália País de publicação: Estados Unidos