The overall treatment-related mortality rates were 2% during induction and 5% overall, and did not differ by study arm. The incidence of veno-occlusive disease was 3% overall and was severe in 0.6% of patients. There were no differences in incidence of this complication between the study arms.
Dr. Joseph Mikhael, who moderated the briefing, commented that the findings are "exciting" and noted that "this agent not only has efficacy, but is particularly well tolerated in this pediatric population." He is an associate professor of medicine at the Mayo Clinic in Scottsdale, Ariz.
The study was supported by the National Cancer Institute. Dr. Gamis and Dr. Mikhael reported no relevant conflicts of interest.