hemophilia therapy / 3B Pharma, Takeda - LARVOL DELTA

Practical Advances in the Diagnosis of Haemophilia and von Willebrand Disease Including Monitoring of Non-Factor Replacement Therapies. (PubMed, Haemophilia) - "Traditional haemophilia therapies act to replace the relevant missing clotting factor, are not interchangeable between haemophilia A and B and cannot be used in patients with high titre inhibitors...Comprehensive haemophilia care, including access to and laboratory monitoring of replacement and NFTs, is well established in high income countries, but there are profound global inequities in the diagnosis and treatment of haemophilia which still need to be remedied. The authors examine the challenges of haemophilia and von Willebrand diagnosis and monitoring of NFTs using conventional and global assays of haemostasis."

Journal • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

Riding the wave of change: Providing solid ground to support nursing with patient transitions to novel haemophilia therapies. (PubMed, Haemophilia) - "Examples of these models in practice could give guidance and examples to reflect on for haemophilia nurses needing to make changes in their practice settings. These models of change, alongside existing haemophilia nurse competencies and tools such as the shared decision-making tool from the World Federation of Hemophilia, can assist the nurse to be a capable change agent to usher in these new innovations."

Journal • Hematological Disorders • Hemophilia • Rare Diseases

Immune tolerance induction in severe haemophilia A: A UKHCDO inhibitor and paediatric working party consensus update. (PubMed, Haemophilia) - "This consensus protocol will facilitate future evaluation of ITI outcomes in the evolving landscape of haemophilia therapeutics and ITI strategies."

Journal • Hematological Disorders • Hemophilia • Pediatrics • Rare Diseases

Real-World Age-Stratified FVIII Consumption and Bleed Outcomes Before and After Switching to Rurioctocog Alfa Pegol in a Retrospective, Observational Study Using US Specialty Pharmacy Data (ASH 2019) - "Assessments included prior hemophilia therapy, FVIII administration frequency and consumption, and annualized bleeding rate (ABR) before and after switching to TAK-660...The SHL antihemophilic factor (recombinant) (Advate®; Baxalta US Inc., a Takeda company, Lexington, MA, USA) was used by 67% (55/82) of patients overall, of whom 47% (26/55) were <18 years old and 53% (29/55) were ≥18 years old... In patients with hemophilia A previously treated with SHL- or EHL-FVIII products, switching to TAK-660 prophylaxis resulted in a significant decrease in ABR of 40–50% in both age groups analyzed. The adult population (ie, ≥18 years old) showed a tendency for reduced weekly FVIII consumption. These findings from real-world data are in agreement with TAK-660 clinical trial results."

Observational data • Real-World Evidence • Retrospective data