ALLOGENEIC STEM CELL TRANSPLANTATION FOR NON-MALIGNANT DISEASES: Edited by Andrea BacigalupoHematopoietic stem cell transplantation for acquired aplastic anemiaGeorges, George E.a,b; Storb, Rainera,bAuthor Information aClinical Research Division, Fred Hutchinson Cancer Research Center bDepartment of Medicine, University of Washington, Seattle, Washington, USA Correspondence to Dr George E. Georges, MD, Clinical Research Division, Fred Hutchinson Cancer Research Center, 1100 Fairview Ave N, Seattle, WA 98109, USA. Tel: +1 206 667 6886; fax: +1 206 667 6124; e-mail: [email protected] Current Opinion in Hematology: November 2016 - Volume 23 - Issue 6 - p 495-500 doi: 10.1097/MOH.0000000000000281 Buy Metrics Abstract Purpose of review There has been a steady improvement in outcomes with allogeneic bone marrow transplantation (BMT) for severe aplastic anemia (SAA), because of progress in optimization of the conditioning regimens, donor hematopoietic cell source, and supportive care. Here, we review recently published data that highlight the improvements and current issues in the treatment of SAA. Recent findings Approximately one-third of aplastic anemia patients treated with immune suppressive therapy (IST) have acquired mutations in myeloid cancer candidate genes. Because of the greater probability for eventual failure of IST, human leukocyte antigen (HLA)-matched sibling donor BMT is the first-line of treatment for SAA. HLA-matched unrelated donor (URD) BMT is generally recommended for patients who have failed IST. However, in younger patients for whom a 10/10-HLA-allele matched URD can be rapidly identified, there is a strong rationale to proceed with URD BMT as first-line therapy. HLA-haploidentical BMT using posttransplant cyclophosphamide conditioning regimens is now a reasonable second-line treatment for patients who failed IST. Summary Improved outcomes have led to an increased first-line role of BMT for treatment of SAA. The optimal cell source from an HLA-matched donor is bone marrow. Additional studies are needed to determine the optimal conditioning regimen for HLA-haploidentical donors. Copyright © 2016 Wolters Kluwer Health, Inc. All rights reserved.