Aplastic anemia natural history, complications and prognosis

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Editor-In-Chief: Aric Hall, M.D., Beth Israel Deaconess Medical Center, Boston, MA [1]

Natural History

Untreated aplastic anemia is an illness that leads to rapid death, typically within six months.

Complications

• 10-33% of all patients develop the rare disease paroxysmal nocturnal hemoglobinuria (PNH, anemia with thrombopenia and/or thrombosis), which has been explained as an escape mechanism by the bone marrow against destruction by the immune system.
• Flow cytometry testing is probably warranted in all PNH patients with recurrent aplasia.

Prognosis

• Correct and prompt diagnosis with early administration of therapy improves the 5 year survival rate.
• Occasionally, milder cases of the disease resolve on their own.
• Relapses of previously controlled disease are, however, much more common.
• Well-matched bone marrow transplants from siblings have been successful in young, otherwise healthy people, with a long-term survival rate of 80%-90%.
• Most successful BMT recipients eventually reach a point where they consider themselves cured for all practical purposes, although they need to be compliant with follow-up care permanently.
• Older people (who are generally too frail to undergo bone marrow transplants) and people who are unable to find a good bone marrow match have five year survival rate of up to 75%.