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Aplastic Anaemia and Bone Marrow Failure Syndromes

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Aplastic anaemia (AA) is defined as pancytopenia with a hypocellular bone marrow in the absence of an abnormal infiltrate or fibrosis. Up to 80% of AA is idiopathic.

Initial Assessment

Aims of initial assessment of pancytopenia are to confirm the diagnosis, assess severity, identify a cause if present and determine whether or not there is an underlying bone marrow failure syndrome. Refractory Cytopaenia of Childhood (RCC) is an important differential diagnosis.

Where AA is considered in the differential diagnosis, a paediatric haematologist should be consulted urgently and before treatment begins.


  • History
  • Examination
  • Investigation
    • FBC and film
    • Reticulocyte count
    • HBF (only useful if pre-transfusion)
    • Blood group and antibody screen
    • Biochemistry including liver function
    • Bone marrow aspirate and trephine with cytogenetics
    • Immunoglobulins and autoantibody screen
    • Tissue typing patient and family
    • Chromosomal fragility testing
    • Flow - FISH for telomere length
    • Other genetic testing dependent on clinical presentation

Supportive Care

  • Transfusion - patients are at risk of alloimmunisation therefore regimen should be conservative.
  • Anti-microbials - antifungal prophylaxis and Pneumocystis prophylaxis post ATG
  • Fevers treated as per standard febrile neutropenia protocol
  • GCSF is not recommended for routine use

Definitive management

  • If a matched sibling is available, proceed to transplant
  • If no matched sibling, current treatment algorithms indicate therapy with immune suppression (ATG and Cyclosporine). With emerging data showing excellent results with up front matched unrelated transplant, both treatment modalities should be discussed with the family.


  1. British Journal of Haematology, Volume 172, Issue 2, January 2016 p 187-207
  2. British Journal of Haematology, Volume 157, Issue 1, April 2016 p 26-40

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Document Control

  • Date last published: 01 December 2016
  • Document type: Clinical Guideline
  • Services responsible: National Child Cancer Network
  • Owner: Nyree Cole
  • Review frequency: 2 years