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Phu Quoc Le

University Children’s Hospital Queen Fabiola (HUDERF), Belgium

Presentation Title:

Improvements in the treatment of haemophilia

Abstract

Hemophilia A and B are X-linked inherited bleeding disorders caused by a deficiency of factor VIII (FVIII) or factor IX (FIX), respectively. The mainstay of hemophilia management has been regular prophylactic infusions of the missing coagulation factor (FVIII/FIX) to reduce mortality and morbidity associated with chronic, crippling arthropathy. This approach is burdensome and costly for patients due to the need for frequent intravenous infusions, high costs, limited availability, and the development of inhibitory antibodies against FVIII/FIX. Advances in the engineering and manufacturing of clotting concentrates have led to the creation of novel molecules that address many of these limitations. These include the development of extended half-life factors, which require less frequent infusions, and new non-factor replacement treatments that can be administered subcutaneously and infrequently, such as FVIII-mimetic antibodies (e.g., emicizumab) and downregulators of natural anticoagulants (e.g., antithrombin, tissue factor pathway inhibitor, or activated protein C). Finally, gene therapy is set to offer patients the possibility of a cure by transferring a functional copy of the gene required to express the missing or dysfunctional clotting factor. This therapy was approved for the treatment of hemophilia B in 2023 in the United States and Europe and is currently in late-phase clinical investigation for hemophilia A.

Biography

Dr. Phu Quoc Le graduated as a pediatrician in 1999 from the Université Libre de Bruxelles, Belgium, and obtained his PhD in Hematology in 2018 with a thesis titled “Improving the Multidisciplinary Care of Patients with Sickle Cell Disease.” He is a consultant in Pediatric Immuno-Hemato-Rheumato-Oncology at the University Children’s Hospital Queen Fabiola (HUDERF) in Brussels. He also serves as the pediatric lead of the HemoWab group, one of the seven hemophilia reference centers in Belgium. Additionally, he was the head of the General Pediatrics department at the Etterbeek-Ixelles Hospital Centre from 2008 to 2024. Dr. Phu Quoc Le is a member of various national and international associations in the fields of hematology, rheumatology, and oncology. He publishes in renowned scientific journals and regularly presents data at international congresses.