History and exam

Key diagnostic factors

common

presence of risk factors

Key risk factors include paroxysmal nocturnal haemoglobinuria, hepatitis, and drug exposure (such as non-steroidal anti-inflammatory drug therapy).

Other diagnostic factors

common

history of recurrent infection

May indicate underlying cytopenia.

fatigue

May indicate underlying cytopenia.

pallor

May indicate underlying cytopenia.

history of bleeding or easy bruising

May indicate underlying cytopenia.

tachycardia

May indicate underlying cytopenia.

dyspnoea

May indicate underlying cytopenia.

Dyspnoea due to pulmonary fibrosis may suggest dyskeratosis congenita.[5]

persistent warts

May suggest inherited GATA2-related disorder.

uncommon

hearing loss or deafness

May suggest Fanconi anaemia or inherited GATA2-related disorder.

short stature, pigmentation abnormalities, or urogenital abnormalities

May suggest Fanconi anaemia.[4]

nail malformations, reticular rash, oral leukoplakia, or epiphora

May suggest dyskeratosis congenita.[5]

osteoporosis

May suggest dyskeratosis congenita.[5]

premature hair loss/premature greying

May suggest dyskeratosis congenita.[5]

hyperhidrosis

May suggest dyskeratosis congenita.[5]

dysphagia

Dysphagia due to oesophageal stricture may suggest dyskeratosis congenita.[5]

extensive dental caries or tooth loss

May suggest dyskeratosis congenita or Shwachman-Diamond syndrome.[5][6]

steatorrhoea

Features of exocrine pancreatic insufficiency such as fat malabsorption may suggest Shwachman-Diamond syndrome.[6]

skeletal dysplasia

May suggest Shwachman-Diamond syndrome.[6]

monocytopenia

May suggest inherited GATA2-related disorder.

non-tuberculous mycobacterial infections

May suggest inherited GATA2-related disorder.

pulmonary alveolar proteinosis

May suggest inherited GATA2-related disorder.

congenital lymphoedema, Emberger syndrome

May suggest inherited GATA2-related disorder.

immunodeficiency (DCML [dendritic cell, monocyte, B cell, and NK cell deficiency])

May suggest inherited GATA2-related disorder.

Risk factors

strong

drug or toxin exposure

Drugs (e.g., chloramphenicol and non-steroidal anti-inflammatory drugs) and toxins (e.g., chemicals or pesticides) have been implicated in the aetiology of AA. However, in only very few instances is there reasonable evidence for an association (from case control studies), and even then it is nearly impossible to prove causality.

The lag time between exposure and disease presentation is usually several weeks to months.[2]

Other drugs with a weak association to AA, which may be coincidental, include penicillamine, gold therapy, and exposures to benzene and dipyrone.[2][3]

paroxysmal nocturnal haemoglobinuria (PNH)

PNH is a rare acquired disorder of the blood characterised by intravascular haemolysis and thrombophilia due to the absence of glycosylphosphatidylinositol-anchored proteins on the membrane surface of blood cells.

PNH and AA are closely related. Patients with PNH can develop AA, and patients with AA often harbour PNH clones even if they do not have the clinical manifestations of PNH. One hypothesis for this relationship is that PNH cells somehow escape the autoimmune attack seen in AA and thus can grow out of proportion to non-PNH cells.[19] See Paroxysmal nocturnal haemoglobinuria.

recent hepatitis

In 5% to 10% of cases, AA can occur in patients with a recent clinical episode of hepatitis.[10] The agent causing hepatitis in these cases is believed to be viral, although it is not any of the known hepatitis virus A-E subtypes.

weak

pregnancy

This association, although described, is poorly understood.[28]

autoimmune disease

Anecdotal reports exist for an association between AA and systemic lupus erythematosus, thymoma, eosinophilic fasciitis, and coeliac disease.[29] The mechanism underlying this association is unclear.

family history

Family history of AA or AA-related abnormalities may suggest congenital AA. Increased incidence of cancer among family members may also suggest congenital AA.

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