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What is Acquired Aplastic Anaemia?

Acquired Aplastic Anaemia is a rare haematological disorder that predominantly affects patients in young adulthood. The major causative mechanism is an immunological attack on one’s blood forming stem cells, known as haematopoietic stem cells, that leads to bone marrow destruction and bone marrow failure. A number of chemicals, environmental toxins and viral infections have been implicated in precipitating Aplastic Anaemia, but in the majority of circumstances, an agent responsible for the immune system malfunction is not identified.

There is no single definitive test to diagnose AA, rather it is a diagnosis of exclusion, with the inherited BMFS syndromes among the main differential diagnoses.

Severity of Aplastic Anaemia is determined by the number of one of the types of white blood cells (neutrophils) present and is classified as very severe, severe and non-severe.

How common is Aplastic Anaemia?

Aplastic Anaemia is rare, affecting approximately 2-3 per million people per year in North America and Europe, and approximately 5 per million per year in South East Asia.

What are the current treatments?

Current treatments include

  • Immunosuppressants – first line immunosuppressive treatments include anti-thymocyte globulin (ATG) and cyclosporin (CsA)
  • Granulocyte colony stimulating factor (G-CSF) – a medication that stimulates the bone marrow to produce white blood cells (granulocytes) and blood forming stem cells (haematopoietic stem cells) and release them from the bone marrow into the blood stream
  • Thrombopoietin receptor agonists (TPO mimetics) – Eltrombopag and Avatrombopag are medications that stimulate the production of platelets
  • Androgens – sex hormone therapies are more likely to be used in older patients
  • Bone marrow transplantation – the only current curative option available is bone marrow transplantation. In younger patients with a matched sibling donor, bone marrow transplantation may be considered as first line treatment in the setting of very severe or severe Aplastic Anaemia.