Study Finds That Ornithine Decarboxylase Inhibitor DFMO Increases Survival for Children with High-Risk Neuroblastoma

A paper published online on September 27, 2018 in Scientific Reports shows the positive results of a phase II clinical trial using the oral medication DFMO (difluoromethylornithine) to prevent relapse in children with high-risk neuroblastoma (HRNB). The open-access article is titled “Maintenance DFMO Increases Survival in High Risk Neuroblastoma.” Neuroblastoma is a form of cancer that develops from immature nerve cells found in several areas of the body. It occurs most often in infants and young children, usually under the age of five. The disease remains a challenge in pediatric oncology and current treatments include therapies that have significant long-term side effects for patients. HRNB accounts for 15 percent of all childhood cancer deaths, in part, due to the fact that nearly half of all patients who reach remission will relapse. "These results are promising and have changed the outlook for our patients with high risk neuroblastoma," said Giselle Sholler, MD, Director of Pediatric Oncology Research at Spectrum Health Helen DeVos Children's Hospital in Grand Rapids, Michigan, and principal investigator of the study. "By using DFMO for two years after finishing conventional therapy, we've seen an overall two-year survival rate for these children of 97 percent. This is a large increase in survival," Dr. Sholler added. "Previously it was believed that children with refractory and relapsed neuroblastoma were considered incurable. This study shows more than 50 percent of patients remaining in remission up to four years." The trial, organized by the Beat Childhood Cancer research consortium, studied the use of DFMO as a single agent for enrolled patients at 20 children's hospitals from June 2012 to February 2016.
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