The National Platelet Research and Referral Centre

Logo of The National Platelet Research and Referral Centre (NPRC)

Contact

Please direct all research and clinical enquiries info.nprc@anu.edu.au

The National Platelet Research and Referral Centre (NPRC) seeks to improve our understanding of platelet disorders and identify the most effective treatment options. The range of platelet disorders includes those resulting in low platelet numbers and bleeding related to platelet dysfunction. In doing so we aim to help people living with these diseases by providing a more comprehensive diagnosis and a 'personalised' treatment program specific for their condition.

Our vision

is to improve the understanding of, treatment for, and quality of life for individuals with platelet disorders.

Our mission

To undertake a systematic and comprehensive approach to better classify platelet disorders through research that combines novel and established scientific methods with extensive clinical data collection. Using this approach, we aim to unravel the distinct mechanisms of disease in each patient and provide improved treatment choices that will increase the likelihood of a cure.

Our values

Determination to improve treatment outcomes for affected individuals

Inclusion of experts in the field across Australia to increase the likelihood of successful outcomes

Vision across scientific and clinical arenas with novel lines of enquiry

Funding and in-kind support

Target community

  • Patients with disorders of platelet number and function including autoimmune diseases, immuno- deficiency and unexplained bleeding
  • Clinicians and GPs
  • Medical students
  • Pharmaceutical industry
  • Government departments involved in the development of health policy

Our approach

Panel A: The current clinical approach involves an interview with a patient after a low platelet count was identified. If ITP (primary or secondary) is suspected, a standard therapy is prescribed and responses are evaluated only by post-treatment changes to platelet count. So far there are no pathways to stratify patients or reliable indicators to predict which line of treatment will work best.

Panel B: Our goal is to establish a pre-treatment set of evaluations that will ultimately help steer clinical decisions around treatments to result in better outcomes for the patient. To achieve this, blood samples 1 and 2 are taken before and after treatment and using methods listed under New Research Tools, we will evaluate new platelet and immune system parameters and develop a patient molecular profile. Clinical outcomes will be correlated with the molecular profile of the patient’s blood before/after treatment, and changes which correlate significantly with positive or negative outcomes will be identified. This new approach aims to better stratify patients and aid clinical management but also to revamp primary and secondary ITP categories based on disease pathology.

Research

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 an 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 is 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, anaemia, 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 a second line of management. Whilst newer therapies are emerging, a lack of clinical diagnostic tools together with 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.

Services

The collaborative links between researchers at The John Curtin School of Medical Research and clinicians at The Canberra Hospital provide the foundation for the NPRC. The Centre will aim to address the clinical aspects of issues surrounding diagnosis and disease management for patients with platelet-based autoimmune disease. To do this, we will establish State-of-the-Art platforms in clinical diagnostics together with a Research hub focused on platelet autoimmune dysfunction. Laboratories will be centrally based at The Australian National University within the John Curtin School of Medical Research, and The Canberra Hospital and services will be available to patients throughout Australia.

The Centre, once NATA-accredited would acquire and comment on new data, and provide recommendations relating to diagnosis and treatment. The care of individual patients would generally remain with the referring health care professional, but our geographical location would permit the clinical arm of the NPRC to be a future hub for quaternary referral, providing leadership and advocacy for this patient cohort, while maintaining a gateway to clinical trials with novel therapeutic agents.

Research

Table: Analyses listed below are not included in standard pathology services but are available at the NPRC. Some of these evaluations provide definitive Yes/No answers whilst results of others are subject to interpretation by the medical professional in collaboration with NPRC researchers.

Platelet-based Syndrome

Approaches

NPRC Capacity for further  development of listed tests

ITP

Flow cytometry

Platelet receptor levels

T and B cell subpopulations

Gene sequencing

Antiplatelet autoantibodies

Cytokine and sGPVI ELISAs

Cutting edge

HIT

HIT IgG ELISA

Antibody pathogenesis assessment (HIPA/SRA)

Gene sequencing

sGPVI ELISAs

Cutting edge

TTP

ADAMTS13 antigen

ADAMTS13 activity assays

Cytokine and sGPVI ELISAs

Developing

Unexplained bleeding

Flow cytometry

Platelet receptor levels

Functional assays

Gene sequencing (Sydney)

Cytokine and sGPVI ELISAs

Strong

 

 

Bernard-Soulier Syndrome

Flow cytometry

Platelet receptor levels

Functional assays

 

Definitive

MYH-9 analysis

Flow cytometry

Platelet receptor levels

Functional assays

Immunofluorescence of neutrophils

Definitive

Marketing and visibility

In keeping with its position as a National Centre, the NPRC will aim to foster national and international collaboration. In order to this, key members of the NPRC (Gardiner, Choi, D’Rozario) will link with their existing extensive network of clinical haematologists and scientists. It is anticipated that the Canberra facility will link with centres in Sydney and Melbourne where some key services have already been established, such as the specialist sequencing of platelet receptors in patients and families with unexplained bleeding in Sydney. The proposed network is outlined below.

  • Sydney (Chris Ward, Jenny Curnow, Joanne Joseph, Vivien Chen, Scott Dunkley, Beng Chong) and scientists at Australian Red Cross Blood Services (Denese Marks, Diane van der Wal)
  • Melbourne (Robert Andrews, Harshal Nandurkar, Mandy Davis, James McFadyen, Huyen Tran, Zane Kaplan, Erica Wood Alfred Hospital, Royal Melbourne Hospital)
  • Brisbane (Robert Bird)
  • Perth (Ross Baker)
  • Victoria/Tasmania (Steve Opat, Harshal Nandurkar, Monash Medical Centre).

The Centre will also increase visibility via key links to

People

A/Prof Elizabeth Gardiner

 Associate Professor Elizabeth Gardiner is an NHMRC Senior Research Fellow in the ACRF Department of Cancer Biology and Therapeutics at The John Curtin School of Medical Research, ANU, Canberra, Australia. She has published extensively in the area of platelet biochemistry and platelet function, particularly relevant to both thrombosis and bleeding in patients. She identified a novel mechanism for shedding of vascular receptors triggered by shear stress, enabling new capabilities in diagnostic and therapeutic reagent development. She is co-chair of the International Society for Thrombosis and Haemostasis (ISTH) Scientific Subcommittee for Biorheology and a member of the Asian-Pacific Microangiopathic Thrombocytopenia (APMAT) Network steering committee and the American Society for Hematology Scientific Committee on Platelets. She is the immediate past Treasurer of the Australian Vascular Biology Society. She is a Principal Editor of the journal Platelets. 

ANU Researchers page

Dr Lucy Coupland

Lucy Coupland has a background of 15 years managing clinical trials in Cardiology and malignant and non-malignant Haematology and 10 years of laboratory-based research into platelet biology and pathology and establishing patient sample and databanks.

Lucy’s ongoing research interests include the role of self-recognition receptors in controlling Fc-receptor-mediated platelet clearance, the potential role for ROTEM analysis to aid in identifying ITP patients at a greater risk of bleeding, 
the contribution of platelets to cancer metastasis, the investigation of novel compounds for use in diseases associated with NETosis, including sepsis and diabetes. 

ANU researchers page

Dr Phil Choi

Phil Choi is a consultant haematologist at The Canberra Hospital. He completed his PhD under the guidance of Professor Beng Chong and has developed a series of research questions around platelet based autoimmune diseases with focus on primary ITP which comprises several distinct but overlapping pathologies. His goal is to develop and refine treatment strategies that are based on clinical and research data, to improve diagnosis and management of ITP by stratifying patients based on pathobiological subtypes that are more predictably sensitive to specific ITP therapies. He is a Principal Editor of the journal Platelets.

ANU researchers page

Ms Sarah Hicks

Sarah Hicks is a Research Officer in the Gardiner Group in the ACRF Department of Cancer Biology and Therapeutics at the John Curtin School of Medical Research. Her research interests include the regulation of platelet receptor shedding and its role in health and disease. She completed a Bachelor of Medical Science in 2014 and a Masters of Biological Science Advanced in 2017 both at the Australian National University.  Sarah has experience in laboratory-based research in platelet biology and in the handling of patient data through previous work at The Canberra Hospital and a medical imaging practice.

ANU researchers page

Updated:  25 April 2018/Responsible Officer:  Director, JCSMR/Page Contact:  Web Manager