Diagnosis of Aplastic Anaemia, Management, and Outcomes utilising a National Dataset

Clinical Trial Investigators:

Professor Erica Wood
Head, Transfusion Research Unit

A/Prof Zoe McQuilten
Deputy Head, Transfusion Research Unit

A/Prof Stephen Ting, Eastern Health / Monash University, VIC
Dr Piers Blombery, Peter MacCallum Cancer Centre, VIC
Dr Devendra Hiwase, Royal Adelaide Hospital, SA
Dr Paul Lacaze, Monash University, VIC
Dr Anthony Mills, Princess Alexandra Hospital, QLD
Dr Jeff Szer, Royal Melbourne Hospital, VIC

Clinical Trial Associate Investigators: Prof Frank Firkin, St Vincent's Hospital Melbourne, and Chair, Aplastic Anaemia Registry
Dr Lucy Fox, Peter MacCallum Cancer Centre, VIC
Prof Stephane Heritier, Monash University, VIC
Dr Alissa Higgins, Monash University, VIC
Clinical Trial Coordinator: Ms Vanessa Fox
Email: sphpm.diaamond@monash.edu
Phone: 1800 811 326
Purpose / aims:

The current standard of care for aplastic anaemia (AA) depends on the severity of the disease, patient age, and the availability of a suitable bone marrow donor. Therapies for AA are immunosuppressive therapy (IST) and bone marrow transplantation (BMT).

In young patients with severe or very severe AA, a BMT from a matched donor is the treatment of choice. In all other severe and very severe AA patients, IST is the current treatment standard, where therapies are administered to combat the dysregulated immune cells attaching bone marrow stem cells. Usually this type of treatment takes several months to produce and maintain improvement.

However, many patients will not respond to the IST treatment or will have only a moderate response, or relapse, and remain at risk of life-threatening complications of AA, such as infections and haemorrhage. These patients will require ongoing supportive treatments such as antibiotics, white cell growth factors, red blood cell and platelet transfusions, to combat these complications.

The DIAAMOND trial is being conducted to find out if giving avatrombopag, (an oral, second-generation thromobopoietin-stimulating agent) improves a range of outcomes for patients with AA.

As part of the trial, avatrombopag will be administered to both newly diagnosed patients as upfront therapy (in combination with IST) and to refractory or relapsed patients.

The trial will assess if avatrombopag increases production of blood cells, improves blood counts, and reduces the need for blood transfusions and supportive care, including hospital admissions.

The trial will be conducted using the platform of the Aplastic Anaemia Registry, a national registry capturing demographics, treatments and outcomes for patients with AA and other bone marrow failure syndromes.

Population captured:

The DIAAMOND clinical trial will be available to two patient groups:

  • Newly diagnosed AA patients who will be treated with avatrombopag in addition to the standard care of IST (equine antithymocyte immunoglobulin [ATG] and cyclosporine)
  • AA patients with no or moderate response to previous IST, or who have relapsed, will be treated with avatrombopag
Outcomes collected:

Primary efficacy outcomes will be assessed by the rate of complete haematological response at 6 months for newly diagnosed patients, and the rate of overall haematological response at 6 months for refractory/relapsed patients.

The rate of acquired clonal evolution at 6 months will be assessed as a primary safety outcome.

Funding source: Medical Research Future Fund (MRFF) #1152524