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.
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TRANSPLANTATION
Long-term results of related myeloablative stem-cell transplantation to cure sickle cell disease
Françoise Bernaudin1,3,
Gérard Socie2,
Mathieu Kuentz3,
Sylvie Chevret4,
Michel Duval5,
Yves Bertrand6,
Jean-Pierre Vannier7,
Karima Yakouben5,
Isabelle Thuret8,
Pierre Bordigoni9,
Alain Fischer10,
Patrick Lutz11,
Jean-Louis Stephan12,
Nathalie Dhedin13,
Emmanuel Plouvier14,
Geneviève Margueritte15,
Dominique Bories3,
Suzanne Verlhac1,
Hélène Esperou2,
Lena Coic1,
Jean-Paul Vernant13,
Eliane Gluckman, for the Société Française de Greffe de Moelle et de Thérapie Cellulaire (SFGM-TC)2
1 Reference Center for Sickle Cell Disease, Intercommunal Hospital, Créteil;
2 Transplant Unit, St-Louis Hospital, Paris;
3 Hematology Mondor Hospital, Créteil;
4 Department of Statistics, St-Louis Hospital, Paris;
5 Hemato-Pediatrics Debré Hospital, Paris;
6 Hemato-Pediatrics, Debrousse Hospital, Lyon;
7 Hemato-Pediatrics, Charles Nicoll Hospital, Rouen;
8 Hemato-Pediatrics la Timone Hospital, Marseille;
9 Hemato-Pediatrics, Vandoeuvre Hospital, Nancy;
10 Hemato-Pediatrics, Necker Hospital, Paris;
11 Hemato-Pediatrics de Hautepierre Hospital, Strasbourg;
12 Hemato-Pediatrics Institut de Cancerologie de la Loire (ICL), St-Etienne;
13 Hematology Pitié Hospital, Paris;
14 Hemato-Pediatrics St-Jacques Hospital, Besançon; and
15 Hemato-Pediatrics, de Villeneuve Hospital, Montpellier, France
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 transplantation-related mortality (TRM). Between November 1988 and December 2004, 87 consecutive patients with severe SCD ranging from 2 to 22 years of age received transplants 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 to 17.9 years), the overall and event-free survival (EFS) 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: the EFS rate among the 44 patients receiving transplants 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.
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