B o n e M a r ro w Tra n s p l a n t a t i o n fo r A cquired S evere A p l asti c A ne m i a Andrea Bacigalupo,

MD

KEYWORDS  Acquired severe aplastic anemia  Bone marrow transplantation  Unrelated donors  Graft-versus-host disease KEY POINTS  Stem cell transplantation for acquired aplastic anemia has made significant progress over the past decade, and this is true especially for alternative donor transplants, including unrelated, cord blood, and haploidentical grafts.  Significant predictors of survival remain the age of the patient and the interval between diagnosis and transplants: young patients with a short interval have the best survival.  Early referral to experienced centers is thus mandatory for this rare disease.  Graft-versus-host disease prophylaxis should include either antithymocyte globulin or alemtuzumab, and the stem cell source should be bone marrow.  Exceptions to this recommendation would be recent protocols using haploidentical family donors.  Haploidentical and cord blood transplants remain an experimental procedure, whereas HLA-identical donors and a well-matched (8/8) unrelated donor can be considered standard of care.

HUMAN LEUKOCYTE ANTIGEN–IDENTICAL SIBLING TRANSPLANTS Who Is a Candidate

Patients with acquired severe aplastic anemia (SAA) should be typed for human leukocyte antigen (HLA) at diagnosis, together with their siblings: if an HLA-identical sibling is identified, and if the patient is younger than 50 years, an allogeneic bone marrow transplant (BMT) is currently the recommended first-line treatment.1 Usually in patients older than 50 years, immunosuppressive therapy (IST) is given up front, because mortality increases with increasing age of patients.2 In a study conducted by the European Group for Blood and Marrow Transplantation (EBMT), a 5-year survival advantage was seen for young patients with very low neutrophil counts (