Background

Aplastic anaemia (AA) is a rare but serious bone marrow failure syndrome (BMFS) with a high morbidity and mortality rate. AA is a diagnosis of exclusion and defined as pancytopenia with a hypocellular bone marrow, absence of an abnormal marrow infiltrate and no increase in reticulin. Patients commonly present with symptoms of anaemia, skin and mucosal bleeding or retinal haemorrhage. Infection is a less common presentation.

The term Aplastic Anaemia is usually understood to refer to the acquired disease, however there are also inherited BMFS (IBMFS) that can mimic the presentation of AA. Both AA and IBMFS are rare conditions but are increasingly recognised as distinct entities, especially now with greater access to molecular diagnostics.

Conducting clinical trials for these patient populations is hampered due to the rarity of the conditions, so registries play important roles in understanding the clinical journey and long-term outcomes of patients with AA and BMFS.

Aims

The aims of the Registry are to:

  • Better define the incidence of aplastic anaemia (AA) and inherited bone marrow failure syndromes (IBMFS) in Australia
  • Provide information on the range of therapeutic strategies being employed in the treatment of these patients, including immunosuppressive therapy, haematopoietic stem cell transplant, and supportive care
  • Explore factors influencing clinical outcomes
  • Better define optimal management of AA and IBMFS patients
  • Inform and inspire future 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

Victoria

Alfred Hospital

Austin Hospital

Box Hill Hospital

Geelong Hospital

Monash Medical Centre

Royal Children’s Hospital

Royal Melbourne Hospital

St Vincent’s Hospital, Melbourne

Western Hospital

 

New South Wales

Children’s Hospital at Westmead

Concord Hospital

Gosford Hospital

Liverpool Hospital

Nepean Hospital

Prince of Wales Hospital

Royal North Shore Hospital

Royal Prince Alfred Hospital

St George Hospital

St Vincent’s Hospital, Sydney

Sydney Children’s Hospital

Westmead Hospital

Wollongong Hospital

 

 

Queensland

Queensland Children’s Hospital

Royal Brisbane and Women’s Hospital

Princess Alexandra Hospital

 

Tasmania

Royal Hobart Hospital

 

Australian Capital Territory

Canberra Hospital

Western Australia

Fiona Stanley Hospital

Sir Charles Gardiner Hospital

Royal Perth Hospital

Perth Children’s Hospital

 

South Australia

Royal Adelaide Hospital

The Queen Elizabeth Hospital

Flinders Medical Centre

Women and Children’s Hospital