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Increasing sickle cell disease cures. (Add ATG to Stem Cells).

PHILADELPHIA -- Adding antithymocyte globulin to a myeloablative regimen led to dramatic success in transplanting stem cells in a series of 69 children with severe sickle cell disease who were treated in France.

After French physicians began adding antithymocyte globulin (ATG) to the standard busulfan and cyclophosphamide regimen in 1992, none of the 57 children who received the treatment rejected their stem cell transplant. In contrast, four of 12 children who received their transplants during 1988-1992 without ATG pretreatment had rejections, Dr. Francoise Bernaudin reported at the annual meeting of the American Society of Hematology.

A total of 59 of the transplant recipients have been cured. The last 30 children who received transplants are all alive, said Dr. Bernaudin, a pediatric hematologist formerly at the Hopital Intercommunal de Creteil (France).

"This will change how we treat these patients. It's a paradigm shift," commented Dr. Ronald Hoffman, director of the cancer center at the University of Illinois in Chicago and president of the society. "It's been known that an allogeneic stem cell transplant can cure patients with sickle cell disease, but no one had achieved this level of survival and cure. Many physicians and families were not willing to risk an allogeneic transplant."

Similar transplants have been done at a few sites in the United States, but this is the largest experience reported, Dr. Hoffman said at the meeting. The series included patients at 14 centers in France. Patients ranged in age from 2.2 to 22 years, with a median age of 8.9 years. Ten of the patients were older than 15 years. All patients had a history of severe disease; 27 had a stoke, 2 had a transient ischemic attack, and 25 other patients had more than three vasoocclusive episodes or more than two instances of acute chest syndrome. Sixty patients received a bone marrow transplant, seven got umbilical cord blood, one received cord blood and marrow, and one patient received peripheral blood stem cells.

With a median follow-up of 36 months, 63 of the 69 (9 1%) patients survived, with an event-free survival rate of 84%. The six deaths included four patients with graft-vs.-host disease, one with an intracranial hemorrhage, and one with sepsis. Disease-free survival was 90% among the children younger than 15 years who received pretreatment with ATG. Among the 29 patients with a history of an intracerebral event, 27 did not have a repeat event during a median follow-up of 5 years.

About half of children with sickle cell disease have disease that is severe enough to justify stem cell transplantation, said Dr. Bernaudin, who is now at Sainte Justine Hospital in Montreal.

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Title Annotation:antithymocyte globulin
Author:Zoler, Mitchel L.
Publication:Pediatric News
Geographic Code:4EUFR
Date:Jun 1, 2003
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