PB2107 Mim8 (denecimig) prophylaxis in adults, adolescents and children with haemophilia A with or without inhibitors: Interim patient-reported outcomes from the long-term extension study (FRONTIER4)
Cihan Ay, Sanjay P. Ahuja, Pratima Chowdary, Meera Chitlur, Llenalia María García Fernández, Kaan Kavakli, Ilgiz Rakhmatullin, Chandrakala Shanmukhaiah 等 10 位
Medical University of Vienna Rainbow Babies & Children's Hospital The Royal Free Hospital Children's Hospital of Michigan
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摘要与影响
Denecimig (Mim8) is a next-generation, activated factor VIII mimetic intended for subcutaneous prophylaxis of haemophilia A with or without factor VIII inhibitors (HAwI/HA). In FRONTIER2 (NCT05053139), denecimig reduced joint pain (Joint Pain Rating Scale [JPRS]) and treatment burden (Haemophilia Treatment Experience Measure [Hemo-TEM]) in adults and adolescents, with >90% preferring it to previous treatment, according to the Haemophilia Patient Preference Questionnaire. In the FRONTIER3 (NCT05306418) study among children aged 1-11 years, denecimig reduced treatment burden according to child-Hemo-TEM.
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生物医学Hemophilia Treatment and Research
Platelet Disorders and Treatments · Coagulation, Bradykinin, Polyphosphates, and Angioedema