Acquired Bone Marrow Failure: Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria

Review
In: The EBMT Handbook: Hematopoietic Cell Transplantation and Cellular Therapies [Internet]. 8th edition. Cham (CH): Springer; 2024. Chapter 78.
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Excerpt

Severe aplastic anemia (SAA) is an autoimmune disorder due to the attack of autoreactive cytotoxic T lymphocytes to the hematopoietic component of the bone marrow. SAA might be associated with PNH, resulting from the clonal expansion of hematopoietic stem cells that have somatic mutations in the X-linked gene PIG-A. This review is summarizing the management of SAA from the diagnosis, where inherited disorders need to be excluded, to the overall treatment algorithm of this disease, including immunosuppression and bone marrow transplantation.

Publication types

  • Review