Allogeneic HSCT in Aplastic Anemia: Current Evidence, Controversies, and Practical Decision-Making

Morbidity and mortality in bone marrow failure bone marrow failure: A condition that occurs when the bone marrow stops making enough healthy blood cells. The most common of these rare diseases are aplastic anemia, myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH). Bone marrow failure can be acquired (begin any time in life) or can be… syndromes such as acquired aplastic anemia aplastic anemia: (ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most… (AA) are driven by severe and prolonged cytopenia cytopenia: (sie-tuh-PEE-nee-uh) A shortage of one or more blood cell types. Also called a low blood count. . Allogeneic hematopoietic stem cell transplantation (alloHSCT) is potentially curative and offers a rapid route to hematopoietic reconstitution. In 2026, reduced-toxicity conditioning, improved supportive care supportive care: Care given to improve the quality of life, or comfort, of a person with a chronic illness. Supportive care treats the symptoms rather than the underlying cause of a disease. The goal is to help the patient feel better. Patients with low blood counts may be given blood transfusions as supportive… , and novel graft versus host disease prevention through post-transplant cyclophosphamide cyclophosphamide: Cyclophosphamide is in a class of medications called alkylating agents. When used to treat cancer, it works by slowing or stopping the growth of cancer cells in your body. When cyclophosphamide is used to treat bone marrow failure, it works by suppressing your body's immune system. (PTCy)-based platforms have brought alternative donor transplantation into the mainstream. This review examines contemporary evidence, particularly looking at the expanded role of transplant outside the widely accepted setting of younger patients with matched sibling donors and offers a pragmatic framework for context-adapted decision-making.