HEMATOLOGY (US) – VOLUME 1 – WINTER 2007

When choosing the topics to cover in a reasonable and readable space in US Hematology 2007, we had to pare down the field. Experienced hematologists were chosen to help elucidate current thinking and controversies.

Von Willebrand disease (VWD)—a disorder due to dysfunctional or deficient von Willebrand factor (VWF)—is the most common inherited disorder of hemostasis and is prevalent in approximately 1% of the total worldwide population,1,2 although the prevalence of symptomatic VWD is about 10 times lower.3

To write a dissertation on coagulopathies in the era of biotechnology, protein mapping, and recombinant technology would require more space than allotted. Therefore, we confine ourselves to several areas: hemophilia, von Willebrand’s disease (VWD), immune thrombocytopenic purpura (ITP), and heparin-induced thrombocytopenia (HIT), all bleeding disorders. We will focus on the unresolved issues facing the field. […]

Factor VIII (FVIII) and von Willebrand factor (vWF) are glycoproteins that circulate in plasma in a tightly bound complex. Structural defects or deficiencies in either glycoprotein are responsible for the development of the most common inherited bleeding disorders: hemophilia A and von Willebrand disease (vWD). These diseases manifest spontaneous bleeding in the severe form of […]

Hemophilia is the most common bleeding disorder in the US, and is estimated to affect one in 5,000 males.1 The disorder is the result of a deficiency in either factor VIII (FVIII; hemophilia A) or factor IX (FIX; hemophilia B). The main treatment for the condition is through replacement therapy with either recombinant or plasma-derived […]

Just as Sisyphus in Greek mythology was condemned to repeatedly roll a large stone to the summit of a mountain, only to have it roll down again, efforts to achieve the pinnacle of hemophilia gene transfer success— sustained transgene expression—continue despite continuing obstacles. Indeed, despite those who think gene transfer is a myth, recent advances […]

Globally, multiple myeloma (MM) accounts for 0.8% of all cancer deaths, with a survival rate of 50% of those enrolled in clinical trials.1,2 It is the most common bone marrow cancer in Europe, with over 77,000 patients undergoing treatment at any one time.3 In the US, MM is the second most common haematological malignancy, affecting […]

Immune thrombocytopenic purpura (ITP) is an autoimmune bleeding disorder characterised by antibody-induced platelet destruction.1,2 Intracranial haemorrhages are the most serious and life-threatening complications, with mortality of 0.2–1% in acute ITP and up to 3% per year in the chronic form.3,4 Almost all intracranial haemorrhages occur when platelet counts (PCs) are below 20×109/l, and most below […]

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 transmission of infectious diseases. Patients receiving blood products are potentially exposed to any number […]

Prevalence of Cytomegalovirus Infection with the human herpesvirus cytomegalovirus (CMV) is a common occurrence, with an estimated 40–85% of the population in Europe and North America infected and harbouring the virus in a dormant form.1 The incidence of infection increases with age and is more common in women than in men, but infection rates depend […]

Normal blood cell production – haematopoiesis – is crucial for the maintenance of health.1 Haematopoiesis is initiated through rare populations of haematopoietic stem (HSC) and progenitor (HPC) cells that give rise to all blood-forming elements: HSCs/HPCs are found in the bone marrow of adults, where they are produced and nurtured. HSCs/HPCs are also found in […]

Cardiologists battle our nation’s number one killer: heart disease. Bone marrow transplant and cancer have their own challenges and carry grave connotations, but cardiovascular (CV) disease causes most of the deaths and disabilities in the US, claiming nearly one million lives each year.1 The cost of cardiac care is estimated to approach $400 billion per […]

Allogeneic hematopoietic cell transplantation (alloHCT) represents a definitive therapy for a number of otherwise fatal conditions. Graft-versus-host disease (GVHD) is the major cause of morbidity after transplant and limits the extended use of this critical therapeutic modality. Dendritic cells (DCs), a population of professional antigen-presenting cells (APCs), are thought to play a critical role in […]

Pathogenesis of Venous Thromboembolism

Aptamers are oligonucleotides that bind to molecular targets in a manner conceptually similar to antibodies.1,2 Through the systematic evolution of ligands by exponential enrichment (SELEX) process, aptamers have been identified against a wide range of therapeutic targets.3–8 Synthetic ribonucleic acid (RNA) aptamers can be modified to have greatly enhanced plasma stability and longer circulating half-lives, […]

Fanconi anaemia (FA) is a genetic, life-threatening disorder1,2 featuring progressive bone marrow failure, birth defects, leukaemia, increased incidence of solid tumours, spontaneous chromosomal instability and hypersensitivity to cross-linking reagents.1,2 Because of FA’s relationship to DNA damage hypersensitivity and its association with susceptibility to neoplastic transformation, the FA research field has gained much attention in recent […]

Immune thrombocytopenic purpura (ITP) is an autoimmune disorder characterized by a decreased number of platelets and mucocutaneous bleeding. ITP may be primary (idiopathic) or secondary to an underlying disorder. Secondary disorders are increasingly being recognized as important and significant contributors to immune thrombocytopenia. Disorders associated with ITP include lymphoproliferative disorders, autoimmune and collagen vascular diseases, […]