APMAT Study Synopsis

Aim

To establish a collaborative network of leading haematology centres in the Asia Pacific Region to study the pathophysiology, diagnosis and treatment of thrombotic thrombocytopenic purpura.

Thrombotic Thrombocytopenic Purpura (TTP) is a rare disease (5-10 cases per million population) characterised by occlusion of the microcirculation by platelet rich thrombi leading to neurological abnormalities, stroke, renal failure and death.

Diagnosis is difficult and despite early recognition and the institution of prompt standard care with plasmapheresis and plasma exchange, the mortality rate is still high and subsequent relapses after remission frequently occur. ADAMTS13 is a circulating protease that cleaves the prothrombotic ultra large multimers of von Willebrand factor (VWF) and severe deficiency has been described in a substantial proportion of patients with TTP.

There is current uncertainty about the definition, diagnosis, the role and reliability of ADAMTS13 testing in a large prospective cohort of patients with the diagnosis of TTP. There is an unmet clinical need for standardised criteria including diagnostic testing for TTP to identify patients who may benefit from new therapies, including recombinant ADAMTS13 therapy.

This is a prospective and retrospective multicentre study of consecutive patients fulfilling standardised clinical criteria for TTP. At diagnosis, venous blood (40ml) will be collected for local and central laboratory testing and storage for ADAMTS13 activity and antibody level, and other biomarkers for TTP and DNA/RNA extracted for genetic analysis. Additional samples will be collected for further analysis if the platelet count returns to normal prior to day 30: 10ml each, at day 30, day 90, day 180 and day 360.

Patients will then be followed for a period of three years for clinical and laboratory evidence of relapse.

Number of patients

300 patients with TTP over a 5-year period (2015-2020).

Number of centres

Over 25 sites from Australia, New Zealand, Singapore, Malaysia, South Korea, Thailand, Hong Kong, India, Taiwan, Japan, Indonesia, and China.

Coordinating centre and central laboratory

• PBI Research Division
• Murdoch University

Partners

• The Chinese University of Hong Kong, Hong Kong
• Manidol University, Thailand
• Nara Medical University, Nara Japan
• Suzhou University, Suzhou China
• Tianjin Institute of Hematology, Tianjin China
• CHA University, Seoul Republic of Korea
• Seoul National University Hospital, Seoul Republic of Korea
• Yonsei University Hospital, Seoul Republic of Korea
• Canterbury District Health Board, Canterburry NZ
• Northshore Hospital Waitemata District Health Board, Waitemata NZ
• National Taiwan University Hospital, Taichung, Taiwan
• Singapore General Hospital, Singapore
• Ampang Hospital, Kuala Lumpur Malaysia
• Centre for Stem Cell Research Christian Medical College, Vellore India
• Monash University, VIC
• The Australian National University, ACT
• Sydney University, NSW
• Hollywood Private Hospital, WA
• Sir Charles Gardiner Hospital, WA
• Royal Adelaide Hospital, SA
• The Alfred Hospital, VIC
• Austin Health, VIC
• The Canberra Hospital, ACT
• Royal North Shore Hospital, NSW
• Calvary Mater Newcastle, NSW
• Westmead Hospital, NSW
• Princess Alexandra Hospital, QLD
• Royal Brisbane and Women’s Hospital, QLD
• Newcastle Hospital, NSW

Supporting organisations

• Thrombosis & Haemostasis Society of Australia and New Zealand
• Asia Pacific Society on Thrombosis and Haemostasis