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In the latest edition of touchREVIEWS in Oncology & Haematology, we are pleased to present a collection of articles that delve into the latest research and advancements in the field. From innovative therapies and genetic treatments to analyses of digital ...
Severe haemophilia A (HA), defined as baseline factor VIII (FVIII) levels of <1%, results in traumatic and spontaneous bleeding episodes, which occur primarily in the joints, in addition to the muscles, soft tissue and the central nervous system. Prophylactic treatment ...
We had the pleasure to talk to Steven Pipe (University of Michigan, Ann Arbor, MI, USA) to discuss the use of gene therapy for the treatment of haemophilia B. The abstract 'First Data from the Phase 3 HOPE-B Gene Therapy Trial: ...
In haematologic malignancies with an unfavourable prognosis, high-dose chemotherapy followed by haematopoietic stem cell transplantation (HSCT) is the only realistic curative therapeutic option.1 Allogeneic and autologous haematopoietic stem cells are routinely used for transplantation. Allogeneic HSCT is associated with a ...
Advances have been made in the use of genetically enhanced T cell therapy, in particular, chimeric antigen receptor (CAR) T cells. Such CAR T cells have been shown to be efficacious in erradicating a number of haematologic malignancies.1–3 CARs are ...
Programmed death 1 (PD-1) and cytotoxic T-lymphocyte-associated protein 4 (CTLA-4) are immune checkpoints that are expressed on the surface of antigen-presenting cells in the initiator and effector phase of T-cell activation respectively. They are responsible for “switching off” the T-cell. Inhibition of ...
Haemophilia B is an X-linked recessive coagulation disorder with an estimated incidence of 1 in 60,000 people.1,2 It is characterised by repeated bleeding, particularly into the joints and muscles, which can initiate a cascade of events leading to destruction of the synovium ...
Von Willebrand disease is the most common inherited bleeding disorder and is caused by quantitative (VWD1 and VWD3) or qualitative (VWD2) defects of von Willebrand factor (VWF). Inherited by autosomal dominant or recessive patterns, women with mild forms of VWF ...
Von Willebrand disease (VWD) is a common hereditary bleeding disorder that may affect as many as 1% of the general population.1 VWD is classified into three major categories based on the type of defect and degree of deficiency of von Willebrand ...
The X-chromosome-linked condition of haemophilia is a bleeding disorder that impairs the body’s natural control over coagulation. While there is currently no cure for haemophilia, the disorder can be effectively controlled with infusions of the deficient clotting factor, i.
Inherited bleeding disorders are rare in the general population. Nevertheless, the most common bleeding disorders, haemophilia and von Willebrand disease (VWD), occur in one in 5,000 males and 1% of the general population.1 Since fibrin formation or platelet aggregation in these patients ...
This issue of European Haematology covers several topics that are of great interest to haematologists because they highlight recent advances in the field or discuss current controversies in the managament of patients. This issue of European Haematology covers several topics ...
The transfusion of allogeneic blood products is a complex medical therapy relying on materials that are often available only in short supply and carry a number of substantial risks for the recipient. Perhaps the most feared complication of transfusion is ...
The transfusion of allogeneic blood products is a complex medical therapy relying on materials that are often available only in short supply and carry a number of substantial risks for the recipient. Perhaps the most feared complication of transfusion is ...
von Willebrand disease (vWD) and haemophilia A are the most frequent bleeding disorders in the general population, occurring in 66–100 people per million worldwide.1 vWD is caused by both qualitative and quantitative defects in von Willebrand factor (vWF) resulting in a ...
Haemophilia A (classic haemophilia) is an X-chromosome-linked bleeding disorder occurring in approximately one in 5,000–10,000 males worldwide.1,2 Haemophilia A is caused by a partial or total deficiency of functionally active coagulation factor VIII (FVIII). Haemophilia produces abnormal bleeding that may be ...
The development of inhibitors in children with haemophilia A presents a major problem in terms of both the success and the cost of treatment.1 Inhibitors develop in response to treatment with factor VIII (FVIII), which is administered to haemophilia A ...
Approximately 30% of severe haemophilia A patients suffer an immune response to therapeutically administered factor VIII (FVIII). The formation of inhibitor antibodies is a serious complication in the treatment of haemophilia, and neutralisation of FVIII coagulation activity results in an inadequate ...
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