Volume 18, Issue 2 pp. E41-E43
Case Report

Successful unrelated umbilical cord blood transplantation for class 3 β-thalassemia major using a reduced-toxicity regimen

Sandeep Soni

Corresponding Author

Sandeep Soni

Division of Heme/Onc/BMT, Nationwide Children's Hospital, The Ohio State University, Columbus, OH, USA

Sandeep Soni, Director of Hematology/BMT, bluebird bio, Inc., 840 Memorial Dr. Cambridge, MA 02139, USA

Tel.: 617 588 3386

Fax: 617 576 2421

E-mail: [email protected]

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Nancy Breslin

Nancy Breslin

Division of Heme/Onc/BMT, Kosair Children's Hospital, University of Louisville, Louisville, KY, USA

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Alexandra Cheerva

Alexandra Cheerva

Division of Heme/Onc/BMT, Kosair Children's Hospital, University of Louisville, Louisville, KY, USA

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First published: 16 December 2013
Citations: 8

Abstract

Unrelated umbilical CB transplant for class 3 β-thalassemia major is associated with an increased risk of mortality and non-engraftment. We describe two patients who underwent successful unrelated umbilical CB transplant using a novel reduced-toxicity preparative regimen. This regimen may be sufficiently immunosuppressive and myeloablative to ensure engraftment with reduced risks of toxicity and mortality. Close monitoring of HHV-6 viral load is advised for patients undergoing transplant with this regimen.

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