Expert Point of View: Busulfan/Melphalan Improves Survival in High-risk Neuroblastoma Patients, Phase III Study Results Show

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Julie R. Park, MDAt the ASCO Plenary Session where the HR-NBL1/SIOPEN trial was presented, formal discussant Julie R. Park, MD, of the University of Washington, Seattle, said, “Large randomized trials have previously shown that myeloablative therapy improves outcomes in high-risk neuroblastoma, and it is now considered standard of care.”

Dr. Park explained that the COJEC regimen used in this study may have contributed to the lower-than-expected 3-year event-free survival in the CEM arm. Previous studies conducted by the Children’s Oncology Group achieved a 3-year event-free survival of 46% with CEM, but those studies used a different induction regimen with lower doses of cisplatin and etoposide and did not include carboplatin.

“It is possible that rapid COJEC had a negative interaction with CEM, but not BuMel,” she said.

“The SIOPEN trial is a great achievement and shows the unity of 20 European nations. This study confirms the importance of myeloablative therapy in a cohort of high-risk neuroblastoma with a good response,” Dr. Park stated. She said that improved induction regimens should be a focus of future research so that cure rates can be increased. ■

Financial Disclosure: Dr. Park reported no potential conflicts of interest.

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Plenary Report: Bu/Mel Improves Survival in High-risk Neuroblastoma Patients, Phase III Study Shows

Ruth Ladenstein, MDMyeloablative therapy with a busulfan (Busulfex, Myleran)/melphalan combination (BuMel) was superior to a regimen of carboplatin/etoposide/melphalan (CEM) in pediatric patients with high-risk neuroblastoma, according to results of the large, phase III HR-NBL1/SIOPEN trial presented at the 2011 ASCO ...