AHEAD. Advate in HaEmophilia A outcome Database

Area of Science:

• Hematology
• Clinical Research
• Observational Studies

Background:

• Hemophilia A patients often experience recurrent bleeding, particularly in joints.
• Limited long-term outcome data exists regarding target joints, joint replacement, and quality of life.
• Previous studies primarily focused on safety and efficacy of treatments.

Purpose of the Study:

• To collect comprehensive, long-term outcome data for hemophilia A patients treated with ADVATE.
• To investigate the development of hemophilia-related arthropathies, joint replacement surgeries, and quality of life.
• To identify risk factors and assess the efficacy and safety of ADVATE in a large patient cohort.

Main Methods:

• Prospective, multi-center, non-interventional observational study (AHEAD study).
• Inclusion of 500 hemophilia A patients with residual FVIII activity ≤5% treated with ADVATE.
• Data collection over four years using electronic CRF, including imaging, questionnaires, and clinical assessments.

Main Results:

• Data collection ongoing, aiming for a valid database of 500 patients.
• Focus on recording hemophilia-related arthropathies via imaging and physician judgment.
• Comprehensive data on joint replacement, pseudotumors, pain, quality of life, and risk factors.

Conclusions:

• The AHEAD study will provide crucial long-term data on hemophilia A patient outcomes.
• Findings will enhance understanding of ADVATE's real-world effectiveness and safety.
• Results are expected to inform clinical practice and improve patient management strategies.