NEW YORK (Reuters Health) - A new approach to bone marrow transplantation in pediatric patients with class 3 thalassemia can reduce mortality and decrease the incidence of recurrent disease by almost fourfold, according to a report the August 15th issue of Blood.
Patients with class 3 thalassemia who undergo marrow transplantation are at particularly high risk, principal investigator Dr. Guido Lucarelli, of the Mediterranean Institute of Hematology in Rome, and colleagues note.
In an earlier study of 122 such patients younger than 17 years of age, who were prepared for transplant with busulfan and cyclophosphamide, the researchers found that 30% of the children developed rejection.
Starting in 1997, however, the investigators have treated these high-risk patients with a new protocol in which hydroxyurea, azathioprine, and fludarabine were added to busulfan and cyclophosphamide. These extra agents, according to the authors, were used in an attempt to enhance immune suppression and eradicate the thalassemic clones.
In 33 consecutive pediatric patients with class 3 thalassemia, the new protocol “was well tolerated with 93% survival,” the investigators report. In their earlier study, before the new protocol was used, the Kaplan-Meier probability of survival was only 79%.
Furthermore, “the incidence of recurrent thalassemia after the transplantation decreased from 30% to 8%.”
“The protocol presented in this report...appears to be a well-tolerated and effective association of drugs for the eradication of the hematopoietic system of thalassemic patients,” the authors conclude.
Source: Blood 2004;104:1201-1203 [ Google search on this article ]
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