Aplastic anaemia (AA) is a rare, life-threatening bone marrow failure syndrome resulting from immune-mediated damage to haematopoietic stem cells, which causes low blood counts. It can affect both children and adults.

Accurate diagnosis is essential, and has traditionally been based on history, examination, blood tests and bone marrow biopsy findings, exclusion of mimicking disorders such as inherited bone marrow failure syndromes and certain medications and viral infections. Genomic testing is now playing an increasingly important role, and can help distinguish acquired AA from other bone marrow failure syndromes, but diagnosis often remains challenging.

Currently available treatments for AA include immunosuppressive therapy and stem cell transplantation, depending on the severity of the disease, patient age and the availability of a suitable stem cell donor. Clinical outcomes remain poor for many patients and better therapeutic options are needed.

Many patients receive supportive therapy, such as red cell transfusions for anaemia, platelet transfusions to prevent or manage bleeding due to low platelet counts and antibiotics to prevent or treat infections.


The aims of the Aplastic Anaemia Registry are to:

  • Better define the incidence of AA in Australia
  • Provide information on the range of therapeutic strategies being employed in the treatment of AA patients, including immunosuppressive therapy, haematopoietic stem cell transplant, and supportive care
  • Explore factors influencing clinical outcomes
  • Investigate the relationship of PNH clones to progress of disease and response to therapy
  • Better define optimal management of AA patients
  • Inform and inspire future hypothesis-driven research in this area

Data Collection

Data will be collected through routine clinical visits and will not require the collection of any extra non-clinical information.

The following categories of data items will be collected:

  • Demographic details
  • Clinical context, including possible precipitants
  • Family history, including IBMFS, IPF and liver disease
  • Clinical presentation
  • Laboratory test results at initial presentation and follow-up reviews
  • Therapy, including immunosuppressive therapy, bone marrow or haematopoietic stem cell transplant, and supportive therapy
  • Clinical outcomes, including details of any relapse, complications, performance status indicators and disease progression

Participating Sites

Coming soon