New Treatment Options for Hemophilia Come with Discussions on Important Issues
The organizing committee of this year's virtual congress of the European Association for Haemophilia and Allied Disorders (EAHAD 2021) promised in the opening speech a "showcase of the latest innovations and advances in the field of hemophilia and bleeding disorders." The program naturally included discussions on current research findings and practical experiences. Topics included the potential role of coagulation factor VIII even with non-factor therapies and the importance of monitoring the success of new therapeutic approaches.
Hot Topic: Questions About the Future Role of FVIII
The recent introduction of new, non-factor treatment options for hemophilia, such as non-replacement and gene therapy, is revolutionizing patient care. Dr. Roberta Gualtierotti from the University of Milan emphasizes the importance of monitoring to evaluate their success. She noted that there is currently intense discussion about whether coagulation factor VIII (FVIII) might have a specific non-coagulative biological role related to joint and bone health. Until this is clearer, she believes that careful monitoring when using non-factor treatment methods is essential.
Professor David Lillicrap from Queen's University in Kingston, Canada, also highlighted the need to verify the potential importance of FVIII for future use. According to him, the introduction of new therapeutic approaches into clinical practice should be accompanied by answers to three main questions: whether the new treatments are safe, effective "in a range of clinical contexts," and whether FVIII has any biological function that new treatment strategies lack. In his presentation, he discussed various types of non-factor therapies currently being evaluated for use in hemophilia management and outlined the latest findings regarding their safety and efficacy. He confirmed that the biological function of FVIII will be reviewed with particular attention to physiological roles that might be missing in non-factor treatment strategies.
Why and How to Monitor?
Dr. Gualtierotti further elaborated on today's options for monitoring the overall and joint health of hemophilia patients. She reminded that until recently, this was mainly based on pharmacokinetics, the annual bleeding rate into joints, the Hemophilia Joint Health Score (HJHS), and imaging methods, specifically radiography and MRI.
Recently, ultrasound has become part of routine comprehensive joint health care for hemophilia patients. It is beneficial due to its high sensitivity for early detection of hemophilic arthropathy, easy accessibility when needing to confirm obvious or detect subclinical bleeding, and, last but not least, low cost.
The COVID-19 pandemic also increased the importance of telemedicine and the need for its implementation. Dr. Gualtierotti mentioned that several applications are currently available for monitoring breakthrough bleeding and treatments administered outside of prophylaxis (replacement or non-replacement therapy) and for providing information on the quality of life for patients reporting their outcomes. She confirmed that the introduction of these applications helps improve communication between the patient and the doctor and shortens the time between diagnosis and the initiation of timely treatment. Currently, the feasibility of remote home ultrasound examinations for early detection is being investigated in several studies.
A number of serum and synovial biomarkers have also been proposed, and some of them are reportedly promising, although they have not yet been validated for routine use in the diagnosis and monitoring of hemophiliacs.
According to the author, monitoring of hemophilia patients should involve a complex of all the aforementioned aspects, namely clinical data, results of imaging methods, and patient-reported outcomes. This allows for an accurate and personalized definition of overall and joint health, aiming to prevent joint bleeding, support treatment adherence, and potentially improve the quality of life.
Eva Srbová
MeDitorial editorial staff
Sources:
1. Gualtierotti R. Role of novel therapies in joint health in haemophilia: how to monitor. EAHAD Congress 2021; abstract SPK014.
2. Lillicrap D. Will there be a role for factor VIII concentrate in haemophilia in the future? EAHAD Congress 2021; abstract SPK022.
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