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Double stem cell transplant improves outcome for children with deadly form of cancer

Results of a recent clinical trial show that adding a second autologous stem cell transplant (which uses the patient’s own stem cells) to standard therapy improves outcomes for children with neuroblastoma. Neuroblastoma is a rare cancer that most commonly affects children age 5 or younger. Historically, less than half of children with high-risk neuroblastoma live five or more years after diagnosis.

The clinical trial involved children newly diagnosed with high-risk neuroblastoma, who were a median age of 3.1 years. The majority (88 percent) had Stage 4 disease and 38.2 percent had a tumor high-risk genetic abnormality called MYCN amplification. Three years after treatment, 61.4 percent of the patients who received a double transplant were alive and cancer-free, compared to 48.4 percent of those receiving a single transplant. Side effects were similar between the single and double transplants.

“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 researchers, who presented their findings at the American Society of Clinical Oncology (ASCO) Annual Meeting earlier this month, will continue to follow the patients on this study for 10 years.