Double Stem-Cell Transplant Improves Outcomes for Children with High-Risk Neuroblastoma

Historically, less than 50% of children with high-risk neuroblastoma live five or more years after diagnosis. A National Cancer Institute (NCI)-funded phase III trial performed by the Children’s Oncology Group found that adding a second autologous stem-cell transplant (ASCT, a transplant that uses the patient’s own stem cells) to standard therapy improves outcomes for these patients. At 3 years, 61.4% of patients who received a double transplant were alive and cancer-free, compared to 48.4% of those who received a single transplant. Side effects were similar between single and double transplant. These data were presented at ASCO’s Sunday, June 5, 2016 Plenary Session, which featured four abstracts deemed to have the greatest potential to impact patient care, out of the more than 5,000 abstracts featured as part of the 2016 American Society of Clinical Oncology (ASCO) Annual Meeting (June 3-7) in Chicago, Illinois. “This finding will change the way we treat children with high-risk neuroblastoma in North America, which still claims many young lives and is in urgent need of better treatments,” said lead study author Julie R. Park, M.D., an attending physician at Seattle Children’s Hospital and Professor in Pediatrics at the University of Washington School of Medicine in Seattle, Washington. “However, the regimen we use for high-risk neuroblastoma is also the most aggressive and toxic regimen we give to children with cancer. For that reason, future research needs to focus on both exploring possible late effects of current therapy and developing newer less toxic therapies.” The trial enrolled children newly diagnosed with high-risk neuroblastoma, who were a median age of 3.1 years. The majority of patients (88%) had Stage 4 disease and 38.2% had a tumor high-risk genetic abnormality called MYCN amplification.
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