Nonmyeloablative Stem Cell Transplantation with Alemtuzumab/Low-Dose Irradiation to Cure and Improve the Quality of Life of Adults with Sickle Cell Disease.

TitleNonmyeloablative Stem Cell Transplantation with Alemtuzumab/Low-Dose Irradiation to Cure and Improve the Quality of Life of Adults with Sickle Cell Disease.
Publication TypeJournal Article
Year of Publication2016
AuthorsSaraf SL, Oh AL, Patel PR, Jalundhwala Y, Sweiss K, Koshy M, Campbell-Lee S, Gowhari M, Hassan J, Peace D, Quigley JG, Khan I, Molokie RE, Hsu LL, Mahmud N, Levinson DJ, A Pickard S, Garcia JGN, Gordeuk VR, Rondelli D
JournalBiol Blood Marrow Transplant
Volume22
Issue3
Pagination441-8
Date Published2016 Mar
ISSN Number1523-6536
KeywordsAdolescent, Adult, Allografts, Anemia, Sickle Cell, Antibodies, Monoclonal, Humanized, Female, Follow-Up Studies, Hematopoietic Stem Cell Transplantation, Humans, Male, Middle Aged, Prospective Studies, Quality of Life, Transplantation Conditioning, Whole-Body Irradiation
Abstract

<p>Allogeneic hematopoietic stem cell transplantation (HSCT) is rarely performed in adult patients with sickle cell disease (SCD). We utilized the chemotherapy-free, alemtuzumab/total body irradiation 300 cGy regimen with sirolimus as post-transplantation immunosuppression in 13 high-risk SCD adult patients between November 2011 and June 2014. Patients received matched related donor (MRD) granulocyte colony-stimulating factor-mobilized peripheral blood stem cells, including 2 cases that were ABO incompatible. Quality-of-life (QoL) measurements were performed at different time points after HSCT. All 13 patients initially engrafted. A stable mixed donor/recipient chimerism was maintained in 12 patients (92%), whereas 1 patient not compliant with sirolimus experienced secondary graft failure. With a median follow-up of 22 months (range, 12 to 44 months) there was no mortality, no acute or chronic graft-versus-host disease (GVHD), and no grades 3 or 4 extramedullary toxicities. At 1 year after transplantation, patients with stable donor chimerism have normalized hemoglobin concentrations and improved cardiopulmonary and QoL parameters including bodily pain, general health, and vitality. In 4 patients, sirolimus was stopped without rejection or SCD-related complications. These results underscore the successful use of a chemotherapy-free regimen in MRD HSCT for high-risk adult SCD patients and demonstrates a high cure rate, absence of GVHD or mortality, and improvement in QoL including the applicability of this regimen in ABO mismatched cases (NCT number 01499888).</p>

DOI10.1016/j.bbmt.2015.08.036
Alternate JournalBiol. Blood Marrow Transplant.
PubMed ID26348889