Aplastic anemia natural history, complications and prognosis

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Editor(s)-in-Chief: C. Michael Gibson, M.S., M.D. [1] Phone:617-632-7753; Associate Editor(s)-In-Chief: Priyamvada Singh, M.D. [2]

Overview

Correct, timely diagnosis with prompt initiation of therapy can increase the survival rates.

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%.