The National Platelet Research and Referral Centre (NPRC) for Platelet-based Autoimmune Disorders is planned as a collaboration between The Australian National University and The Canberra Hospital, which aims to bring together the latest breakthroughs in research along with excellence in clinical medicine to improve diagnosis and treatment management for patients with chronic autoimmune diseases.
Platelet disorders often remain unrecognised and under-diagnosed until individual experiences a major surgical or physical trauma. These disorders can result in chronic autoimmune diseases that are associated with a significant burden of disease in patients. However, once these patients are identified, guidelines to help clinicians manage them are generally vague and of limited use. In addition, successful disease management using primary and second-line therapies may only emerge after months of treatment and is often anecdotal.
The research arm of the NPRC will principally investigate platelet receptor parameters in people at risk of thrombosis or bleeding. This represents an entirely novel approach to evaluating patients bleeding risk. Currently, clinical and basic investigative analysis of platelet function is geographically fragmented throughout Australia and is carried out locally in an ad hoc fashion. This decentralised environment undermines haematological excellence in patient care. By centralising the resources and services available to these patients within the NPRC, we expect to improve patient morbidity and decrease the significant economic burden on the Australian health system.
An example of a platelet-based autoimmune disease: Immune Thrombocytopenia
Immune thrombocytopenia (ITP) is a chronic, immune-mediated disease characterised by a transient or long-lasting decrease in platelet counts. This is a rare disease, however, it is the most prevalent autoimmune disorder involving blood cells, afflicting 2 - 5 in 100,000 children per year and 3 in 100,000 adults per year. This is equivalent to approximately 2000 - 2500 adult cases of ITP in Australia each year. For patients between 18-65 years, the incidence of ITP increases with age and is slightly higher in women than men. While the acute form of ITP is benign and self-limited, the chronic form of the disease is accompanied by permanent thrombocytopenia. More than 7 million people worldwide have the chronic form of this disease.
Diagnosis and treatment of ITP are limited by the available resources. Primary ITP is diagnosed by exclusion, with isolated thrombocytopenia as the only truly universal feature. Patients with ITP experience numerous serious clinical consequences, including significantly higher rates of infection, bleeding, anemia, debilitating fatigue and psychological changes. Clinical investigation of patients with ITP varies widely but generally consists of a peripheral blood count and evaluation of a peripheral blood smear. Treatment generally comprises conventional first-line treatments, which affect the immune system generally (IV gamma globulins, corticosteroids), followed by splenectomy as the second line of management. Whilst newer therapies are emerging, a lack of clinical diagnostic tools together with the limited financial resources of the individual patient and of the health care system, strongly impact the choice of treatment.
The NPRC will benefit patients with ITP and other platelet-based autoimmune disorders by providing a central hub where clinical diagnostic services unite with the latest findings from platelet-based research labs to provide targeted individual disease management.