Last marrow standing: bone marrow transplantation for acquired bone marrow failure conditions.
Paroxysmal nocturnal hemoglobinuria, aplastic anemia, and myelodysplastic syndrome are a spectrum of acquired marrow failure, having a common pathologic thread of both immune dysregulation and the development of abnormal hematopoiesis. Allogeneic hematopoietic cell transplantation plays a critical role in the treatment of these disorders and, for many patients, is the only treatment modality with demonstrated curative potential. In recent years, there have been many breakthroughs in the understanding of the pathogenesis of these uncommon disorders. The subsequent advances in non-transplant therapies, along with concurrent improvement in outcomes after hematopoietic cell transplantation, necessitate continual appraisal of the indications, timing, and approaches to transplantation for acquired marrow failure syndromes. We review here contemporary and critical new findings driving current treatment decisions.
Fred Hutchinson Cancer Research Center and the University of Washington School of Medicine, 1100 Fairview Avenue N, D1-100, Seattle, WA, 98109-1024, USA. atgerds@fhcrc.org
Aaron T
AT
Gerds
Bart L
BL
Scott
D000741:Anemia, Aplastic / Q000188:drug therapy / Q000601:surgery*; D061067:Antibodies, Monoclonal, Humanized / Q000627:therapeutic use; D016026:Bone Marrow Transplantation / Q000379:methods*; D003131:Combined Modality Therapy; D006457:Hemoglobinuria, Paroxysmal / Q000188:drug therapy / Q000601:surgery*; D006801:Humans; D009190:Myelodysplastic Syndromes / Q000188:drug therapy / Q000601:surgery*; D019172:Transplantation Conditioning / Q000379:methods
D061067:Antibodies, Monoclonal, Humanized; C481642:eculizumab