Clinical Updates on Haemophilia
A special issue of Journal of Clinical Medicine (ISSN 2077-0383). This special issue belongs to the section "Hematology".
Deadline for manuscript submissions: 20 December 2024 | Viewed by 23
Special Issue Editor
Special Issue Information
Dear Colleagues,
Over the last decade, the number of products used to treat a person with haemophilia (PwH) has grown dramatically. Novel therapies offer both improved protection from bleeds and more comfort of administration. Extended half-life products allow for an increase in factor levels, even reducing the number of infusions, especially in haemophilia B. The requirement of less infusions licenses the start of regular prophylaxis even in moderate haemophilia patients. In severe haemophilia A patients, subcutaneous non-replacement therapy permits the start of prophylaxis very early in children’s lives, as intravenous administration is no longer a problem. Lastly, gene therapy is now available, and PwH who undergo gene therapy may see their factor levels increase, sometimes even to normal levels.
Within this scenario, we must set our sights on achieving a more ambitious goal: to restore normal haemostasis and to optimize quality of life (QoL) according to the concept of health equity, as defined by the World Health Organization. The aim of this Special Issue is to report experiences in treatments, with particular interest in new products, laboratory support, and new measurements to evaluate the joint status and QoL. Reports focused on both paediatric and elderly PwH settings are of particular interest.
Dr. Irene Ricca
Guest Editor
Manuscript Submission Information
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Keywords
- haemophilia
- early prophylaxis
- non-replacement therapy
- gene therapy
- joint status
- quality of life
- patient-reported outcomes
