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Hematopoietic stem cell transplantation is an option for patients with severe aplastic anemia (SAA) refractory to immunosuppressive therapy and the thrombopoietin-receptor agonist eltrombopag (NEJM JW Oncol Hematol Feb 2014 and Blood 2014; 123:1818). If matched related or unrelated donors are not available, is cord blood transplantation a safe and effective alternative?
To answer this question, investigators in France conducted a prospective phase II study involving 27 patients (mean age, 16 years) with refractory SAA who received one or more courses of prior immunosuppressive therapy and transfusion of >20 units of red cells, platelets, or both. The conditioning regimen included fludarabine, cyclophosphamide, antithymocyte globulin, total b…