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Blood, 1 October 2007, Vol. 110, No. 7, pp. 2749-2756. Prepublished online as a Blood First Edition Paper on July 2, 2007; DOI 10.1182/blood-2007-03-079665. This Article Full Text (PDF) All Versions of this Article: blood-2007-03-079665v1 110/7/2749    most recent Alert me when this article is cited Alert me if a correction is posted Services Email this article to a friend Similar articles in this journal Similar articles in PubMed Alert me to new issues of the journal Download to citation manager Rights and Permissions Citing Articles Citing Articles via HighWire Citing Articles via CrossRef Citing Articles via Google Scholar Google Scholar Articles by Bernaudin, F. Articles by Gluckman, E. Search for Related Content PubMed PubMed Citation Articles by Bernaudin, F. Articles by Gluckman, E. Related Collections Related Articles in Blood Online Social Bookmarking                   What's this?  Previous Article  |  Next Article  Submitted March 14, 2007 Accepted June 27, 2007 Long-term results of related, myeloablative stem cell transplantation to cure sickle cell disease Francoise Bernaudin*, Gerard Socie, Mathieu Kuentz, Sylvie Chevret, Michel Duval, Yves Bertrand, Jean-Pierre Vannier, Karima Yakouben, Isabelle Thuret, Pierre Bordigoni, Alain Fischer, Patrick Lutz, Jean-Louis Stephan, Nathalie Dhedin, Emmanuel Plouvier, Genevieve Margueritte, Dominique Bories, Suzanne Verlhac, Helene Esperou, Lena Coic, Jean-Paul Vernant, and Eliane Gluckman Reference Center for Sickle Cell Disease, Intercommunal, Creteil, France Transplant Unit, St-Louis Hospital, Paris, France Hematology, Mondor Hospital, Creteil, France Department of Statistics, St-Louis Hospital, Paris, France Hemato-Pediatrics, Debre Hospital, Paris, France Hemato-Pediatrics, Debrousse Hospital, Lyon, France Hemato-Pediatrics, Charles-Nicoll Hospital, Rouen, France Hemato-Pediatrics, la Timone Hospital, Marseille, France Hemato-Pediatrics, Vandoeuvre Hospital, Nancy, France Hemato-Pediatrics, Necker Hospital, Paris, France Hemato-Pediatrics, de Hautepierre Hospital, Strasbourg, France Hemato-Pediatrics, ICL Institut, St-Etienne, France Hematology, Pitie Hospital, Paris, France Hemato-Pediatrics, St-Jacques Hospital, Besancon Hemato-Pediatrics, de Villeneuve Hospital, Montpellier, France * Corresponding author; email: francoise.bernaudin{at}chicreteil.fr. Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative treatment for sickle cell disease (SCD); nevertheless, its use has been limited by the risk of transplant-related mortality. Between November 1988 and December 2004, 87 consecutive patients with severe SCD, ranging from 2 to 22 years of age, were transplanted in France. Cerebral vasculopathy was the principal indication for transplantation (55 patients). All the patients received grafts from a sibling donor after a myeloablative conditioning regimen (CR). The only change in the CR during the study period was the introduction of antithymocyte globulin (ATG) in March 1992. The rejection rate was 22.6% before the use of ATG, but 3% thereafter. With a median follow-up of 6 years (range 2.0-17.9 years), the overall and disease-free survival (DFS) rates were 93.1% and 86.1%, respectively. Graft-versus-host disease (GvHD) was the main cause of TRM. Importantly, cord blood transplant recipients did not develop GvHD. No new ischemic lesions were detected after engraftment and cerebral velocities were significantly reduced. The outcome improved significantly with time as the DFS rate among the 44 patients transplanted after January 2000 was 95.3%. These results indicate that HLA-identical sibling HSCT after myeloablative conditioning with ATG should be considered as a standard of care for SCD children who are at high risk for stroke. CiteULike    Connotea    Del.icio.us    Digg    Reddit    Technorati    What's this? Related Articles in Blood Online: Late effects of myeloablative bone marrow transplantation (BMT) in sickle cell disease (SCD) Courtney D. Fitzhugh, Shira Perl, and Matthew M. Hsieh Blood 2008 111: 1742-1743. [Full Text] [PDF] Response: Late effects of myeloablative stem cell transplantation or late effects of sickle cell disease itself? Françoise Bernaudin, Mathieu Kuentz, and Gérard Socié Blood 2008 111: 1744. [Full Text] [PDF] Tortoise and hare win for SCD John F. Tisdale Blood 2007 110: 2223-2224. [Full Text] [PDF] This article has been cited by other articles: A. Perumbeti, T. Higashimoto, F. Urbinati, R. Franco, H. J. Meiselman, D. Witte, and P. Malik A novel human gamma-globin gene vector for genetic correction of sickle cell anemia in a humanized sickle mouse model: critical determinants for successful correction Blood, August 6, 2009; 114(6): 1174 - 1185. [Abstract] [Full Text] [PDF] C. D. Fitzhugh, S. Perl, and M. M. Hsieh Late effects of myeloablative bone marrow transplantation (BMT) in sickle cell disease (SCD) Blood, February 1, 2008; 111(3): 1742 - 1743. [Full Text] [PDF]

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