Takeda Pharmaceutical Company Limited announced a scientific update from the AHEAD real-world study investigating the long-term outcomes associated with ADVATE [antihemophilic factor (recombinant), rAHF] in patients with hemophilia A, presented as an oral presentation at the World Federation of Hemophilia Virtual Summit 2020 (WFH 2020). The update is one of the three abstracts being presented at WFH 2020 from Takeda’s Hematology portfolio and pipeline. Echoing Takeda’s theme at the virtual summit – “It’s Personal” – the company also shared data from a physician survey in China, providing insights into optimizing personalized care and hemophilia patient management in centers of excellence (COE).
“Takeda’s vision for a bleed-free world is as important today as ever,” commented Dr. med. Wolfhard Erdlenbruch, M.D., vice president Head of Global Medical Affairs Hematology, Takeda. “The data we presented at WFH 2020 continue to add to the body of evidence for the management of hemophilia, a bleeding disorder that affects at least 210,000 people worldwide as of 2018 and potentially a lot more. As a leader in Hematology, Takeda is committed to improving patient outcomes, and the findings of our studies demonstrate the value personalized strategies can offer to patients as part of the ongoing management of their bleeding disorder.”
Prophylaxis for hemophilia A is the standard of care treatment for patients because it can help prevent spontaneous bleeds, as even a single bleed may cause joint damage and impact their quality of life.
Interim analysis results from the AHEAD real-world outcomes study demonstrate that the number of hemophilia A patients who were able to achieve zero bleeds increased over the years by receiving rAHF. For those receiving prophylaxis, the number of patients with zero bleeds increased from 34% in year 1 to 53% in year 6. For those receiving on-demand treatment, it increased from 28% in year 1 to 38% in year 6. The results also show that those receiving rAHF prophylaxis generally reported slightly better health-related quality of life (HRQoL) than those receiving rAHF on-demand. The results support the role of rAHF as an important option for hemophilia A, a condition which requires life-long monitoring and management.
“The results of the interim analysis from the AHEAD real-world outcomes study support the long-term effectiveness of rAHF for hemophilia A patients, which was maintained in a six-year observational period and bolstered by consistent HRQoL scores,” added Prof. Dr. med. Johannes Oldenburg, chairman and director, Institute of Experimental Haematology and Transfusion Medicine, University Clinic Bonn AöR, Germany, who is the researcher leading the study. “Every patient is unique, and these results support a tailored and personalized approach to optimize care for patients with bleeding disorders, delivered in partnership with their treating physician.”
A substantial number of patients with hemophilia A live in China, due to the large population.i Results from a physician survey explored the Center of Excellence (COE) model for the optimization of multi-department collaboration for patients with hemophilia A in China; low patient awareness of the need for treatment/prophylaxis and fewer than 40% of patients received prophylaxis, demonstrated the need for improvements in both public and physician awareness and knowledge of hemophilia. Results showed that a COE model may improve collaboration across departments, optimize patient management and thereby improve outcomes.
Data from a prospective, post-marketing surveillance study evaluating the safety and efficacy profile of Rixubis [Nonacog gamma, recombinant FIX concentrate] demonstrated the safety and efficacy profile for bleeds, perioperative/surgery, and prophylaxis in adult and pediatric patients with hemophilia B in the real-world setting in South Korea. The study showed that 86.6% (123/142) of hemostatic effectiveness assessments for nonacog gamma were reported as good or excellent, and of the 11 adverse events reported, all were mild in severity, with 10 resolved/recovered events not related to nonacog gamma, and one event (inhibitory antibody development) unconfirmed.
“The findings presented at WFH 2020 highlight our commitment to patients living with bleeding disorders, and are adding to the bank of scientific evidence supporting the importance of personalized care in Hematology,” Dr. Erdlenbruch concluded. “In addition to the AHEAD study, results from the physician survey in China supported the rationale for cross-department collaboration to optimize patient care. All the results from WFH 2020 emphasize the importance of understanding each patient and offering tailored support for those with bleeding disorders who may have differing needs.”
ADVATE is a full-length (derived from the complete FVIII gene) recombinant FVIII product that is processed without any blood-based additives. ADVATE is approved in the EU for the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor VIII deficiency) in all age groups.