Synopsis
Recognise hypocellular marrow failure, exclude reversible and clonal mimics, stabilise infection and bleeding, and choose transplant or immunosuppression according to severity, age and donor status.
- Aplastic anaemia is pancytopenia caused by failure of haematopoietic stem and progenitor cells in a hypocellular marrow without malignant replacement or substantial fibrosis.
- Most acquired cases are immune mediated and idiopathic; implicated exposures include medicines, toxins, hepatitis and other infections, while inherited marrow-failure syndromes require active consideration.
- Patients present through consequences of low cell lines: fatigue and dyspnoea, infection or mouth ulceration, petechiae, bruising and mucosal bleeding.
Key red flags
Hypotension, confusion, hypoxia or fever with profound neutropenia requires empirical antibiotics and resuscitation before diagnostic completion.
Investigation priorities
Confirm cytopenias and quantify marrow output.
Management branches
New pancytopenia includes severe neutropenia, thrombocytopenia or symptomatic anaemia.
- Admit unstable or severely cytopenic patients, culture suspected infection and deliver the local neutropenic-sepsis antibiotic regimen without waiting for marrow biopsy.
- Discuss platelet and red-cell support with haematology and transfusion medicine, using appropriately selected components and minimising avoidable donor exposure before transplant.
Key medicines
Horse antithymocyte globulinA common adult severe-aplastic-anaemia regimen is 40 mg/kg intravenously once daily for 4 days, given in a specialist centre with corticosteroid reaction prophylaxis and ciclosporin when indicated.
CiclosporinA specialist commonly starts about 5 mg/kg/day orally in 2 divided doses, then adjusts to the centre's trough target, renal function, blood pressure and interactions. Taper only after a sustained haematological response.