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Aplastic anaemia and pure red cell aplasia 

Aplastic anaemia and pure red cell aplasia

Judith C.W. Marsh

, Austin Kulasekararaj

, and Ghulam J. Mufti


August 28, 2014: This chapter has been re-evaluated and remains up-to-date. No changes have been necessary.


This chapter has been completely rewritten (Nov 2012), to reflect in particular an increased awareness of inherited forms of AA/bone marrow failure (BMF) disorders, better understanding of the immunological changes that occur in acquired, immune-mediated AA showing the important role of CD4+ T-cells in the pathogenesis, improved clinical outcomes following haemopoietic stem cell transplantation (HSCT) for severe AA, updated information on the clinical investigation of pure red cell aplasia (PRCA), and recent changes in treatment approach to acquired PRCA, and recent advances in detection of molecular and genetic changes that are found in inherited BMF disorders.

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date: 19 September 2021

Aplastic anaemia (AA) is a rare bone marrow failure (BMF) disorder characterized by pancytopenia and a hypocellular bone marrow. AA is commonly acquired, immune-mediated and idiopathic in nature.

Activate auto-reactive, cytotxic CD8+ T-cells are present but recent work has shown that CD4+ T-cells appear to be more important in the pathogenesis of acquired AA. The immune nature of acquired AA provides the rationale for one of the treatment options, namely immunosuppressive therapy....

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