Acquired Aplastic Anemia: What Have We Learned and What Is in the Horizon? | Aplastic Anemia and MDS International Foundation

Acquired Aplastic Anemia: What Have We Learned and What Is in the Horizon?

Journal Title: 
Pediatr Clin North Am.
Primary Author: 
Savaşan S
Author(s): 
Savaşan S.
Original Publication Date: 
Friday, June 1, 2018

Acquired aplastic anemia (aAA) characterized by peripheral pancytopenia and bone marrow aplasia is a rare and serious disorder. Differential diagnosis includes constitutional bone marrow failure syndromes and myelodysplastic disorders. Autoimmune reaction to altered hematopoietic stem cells highlights the underlying mechanism. Matched related donor allogeneic hematopoietic stem cell transplantation is the ideal pediatric treatment; alternative approaches include immunosuppressive therapy and use of eltrombopag. Progression to clonal disorders can occur. Recently, alternative donor hematopoietic stem cell transplantation outcomes have significantly improved. Despite advances, aAA continues to be a challenge for hematologists.

Bone Marrow Diseases: