A deficiency of blood cells of all lineages (but generally excludes lymphocytes) - not a diagnosis, and does not always mean bone marrow failure or malignancy
Aetiology
Reduced production - bone marrow failure
- Inherited syndromes - very rare
- Characterised by impaired haeopoiesis, congenital abnormalities and cancer predisposition
- Arise due to defects in DNA repair/ribosomes/telomeres
- Example: patients with Fanconi’s anaemia are unable to correct inter-strand cross-links (DNA damage)
- Acquired
- Primary - no obvious cause, usually stem cell defect
- Idiopathic aplasic anaemia - autoreactive T-cells target haemapoeisis
- Myelodysplastic syndromes - increased apoptosis of progenitor and mature cells, propensity for evolution into AML
- Acute leukaemia (WCC can be variable) - proliferation of abnormal cells from LSC, failure to differentiate into mature or normal cells, prevent normal HSC development
- Secondary
- Drug induced e.g. chemotherapy, alcohol, azathioprine, metotrexate, chlorampenicol - causes aplasia
- B12/folate deficiency - nuclear maturation can affect all lineages
- Infiltrative - non-haemopoietic malignant infiltration, lymphoma
- Misc. - viral (e.g. HIV), storage diseases
Increased destruction - hypersplenism
- Pancytopenia can be caused by increased splenic pool or increased destruction that exceeds bone marrow capacity, usually associated with a significantly enlarged spleen
- Causes of hypersplenism:
- Splenic congestion e.g. portal hypertension
- Systemic diseases e.g. rheumatoid arthritis
- Haematological diseases e.g. splenic lymphoma
Pathophysology
Marrow cellularity in pancytopenia
- Variable depending on the cause
- Hypocellular in aplasic anaemia
- Hypercellular in myelodysplastic syndromes, B12/folate deficiency, hypersplenism
Clinical presentation
- Anaemia - fatigue, shortness of breath, cardiovascular compromise
- Neutropenia - increased severity and duration of infections, opportunistic infections
- Thrombocytopenia - bleeding (purpura, petechiae, ‘wet’ bleeds)
- Presentation can also be influenced by underlying cause
Investigations
- Bloods - FBC, blood film
- Additional routine tests guided by history (inc. family history), clinical findings, and initial blood results
- Bone marrow examination
- Specliased tests guided by above e.g. cytogenetics (e.g. chromosome fragility testing in Fanconi’s syndrome, NGS, WES)
Management
Supportive
- Transfusions - red cell transfusions, platelet transfusions, (neutrophil transfusions not routine)
- Antibiotic prophylaxis/treatment where appropriate
- Treat neutropenic fever promptly based on local unit antibiotic policy without waiting for microbiology results
Specific
- Dependent on cause
Primary bone marrow disorder
- Malignancy - consider chemotherapy
- Congenital - consider bone marrow transplantation
- Idiopathic aplastic anaemia - immunisuppression
Secondary bone marrow disorder
- Drug reaction - STOP
- Viral - e.g. treat HIV
- Replace B12/folate
Hypersplenism
- Treat cause if possible
- Consider splenectomy (not appropriate in all cases)