Clinical trial demonstrates that bone marrow transplant followed by vitamin A-derivative treatment improves survival from a major childhood cancer

Combining the efforts of children's cancer treatment programs nationwide, a randomized clinical trial of 539 children has shown that two innovative treatments, taken together, offer nearly a three-fold improvement in the disease-free survival of children with high-risk neuroblastoma, the third most common - and one of the most deadly -- of childhood cancers.

The National Cancer Institute-sponsored study showed that these children have the best chance of disease-free survival if their initial chemotherapy and surgery is followed by high-dose chemoradiotherapy and an autologous bone marrow transplant, where a child is re-implanted with her own bone marrow after it has been cleared of cancer cells. The results are improved even further if this treatment is followed by a high-dose course of 13-cis retinoic acid, a derivative of Vitamin A that also is prescribed for acne.

The study was led by Katherine K. Matthay, M.D., professor of pediatrics at the University of California, San Francisco and director of pediatric clinical oncology for Lucile Packard Children's Health Services at UCSF. The retinoic acid portion of the trial was led by C. Patrick Reynolds, M.D., Ph.D., of Childrens Hospital Los Angeles (CHLA), associate professor of pediatrics and pathology at the University of Southern California School of Medicine. Their results were reported in the October 14, 1999 issue of the New England Journal of Medicine.

The randomized multi-center Phase III clinical trial was the only way to prove the worth of these two treatments, which had shown promise in pilot studies, according to Matthay. "Studies had been done of bone marrow transplant, and of intensive chemotherapy. There were advocates of both treatments. But no one had done a randomized study of the two in comparison," she said.

Matthay credits the Children's Cancer Group, a national cooperative research organization, for enlisting the help of more than 100 cancer centers nationwide to join the stu

Contact: Janet Basu
University of California - San Francisco

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