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Key Words: chemotherapy, etoposide, Ewing sarcoma, ifosfamide. (Definitions of many terms related to cancer can be found in the Cancer.gov Dictionary ⁴.)

Addition of the drugs ifosfamide and etoposide to standard chemotherapy for Ewing sarcoma -- a rare but highly malignant bone tumor affecting children and young adults -- significantly improved five-year survival in patients whose disease had not spread to other organs. The study results were reported in the February 20, 2003, issue of the New England Journal of Medicine (see the journal abstract of the study.)

Previously, standard therapy for Ewing sarcoma involved surgery, radiation therapy, or both, plus chemotherapy with the drugs doxorubicin, vincristine, cyclophosphamide, and dactinomycin. In the current study, 518 patients were assigned at random to one of two groups. One group received the standard chemotherapy regimen. The second group received experimental therapy in which the four standard drugs were alternated with ifosfamide and etoposide. Of the 518 patients, 120 had advanced (metastatic) disease; the remainder had localized disease (not spread to other organs).

Five-year survival

Among the patients with localized disease, 69 percent of those who received the experimental therapy were alive, with no recurrence of their disease, after five years. By contrast, 54 percent of those who received standard therapy were alive and disease-free after five years. Overall survival at five years was 72 percent in the experimental therapy group, compared with 61 percent in the group treated with standard therapy.

However, among patients whose disease had spread to other organs, those receiving the experimental therapy fared no better than those treated with the standard drug regimen. After five years, 22 percent of patients in both groups were alive and disease-free. Overall survival at five years for patients with advanced disease was 34 percent in the experimental-therapy group and 35 percent in the standard-therapy group, a difference that was statistically insignificant (that is, it could have occurred by chance).

More infections, more hospital days

Patients who received the experimental therapy suffered more infections and spent more days in the hospital than those who received standard therapy. Regardless of which treatment they received, patients aged 17 and under and those with smaller tumors had better survival rates than patients who were aged 18 and over or who had large tumors.

In the United States each year, about 200 children and adolescents are diagnosed with Ewing sarcoma or one of its close cousins. These tumors are most common in the teenage years. Most occur in the long bones of the legs or arms.

The research was conducted by the Children’s Cancer Group and the Pediatric Oncology Group, with a research team led by Holcombe E. Grier, M.D. These two groups have since merged to form the Children's Oncology Group ⁵ (COG), which is supported by the National Cancer Institute to conduct clinical trials around the world devoted to children and adolescents with cancer.

Glossary Terms

Treatment with drugs that kill cancer cells.

A drug that is used to treat many types of cancer and is being studied in the treatment of other types of cancer. It is also used to treat some types of kidney disease in children. Cyclophosphamide attaches to DNA in cells and may kill cancer cells. It is a type of alkylating agent. Also called CTX and Cytoxan.

An anticancer drug that is a type of antitumor antibiotic.

A drug that is used to treat many types of cancer and is being studied in the treatment of other types of cancer. Doxorubicin comes from the bacterium Streptomyces peucetius. It damages DNA and may kill cancer cells. It is a type of anthracycline antitumor antibiotic. Also called Adriamycin PFS, Adriamycin RDF, doxorubicin hydrochloride, hydroxydaunorubicin, and Rubex.

A drug used to treat testicular and small cell lung cancers. It is also being studied in the treatment of several other types of cancer. Etoposide blocks certain enzymes needed for cell division and DNA repair, and it may kill cancer cells. It is a type of podophyllotoxin derivative and a type of topoisomerase inhibitor. Also called Toposar and Vepesid.

A type of cancer that forms in bone or soft tissue. Also called peripheral primitive neuroectodermal tumor and pPNET.

A drug that is used together with other drugs to treat germ cell testicular cancer that did not respond to previous treatment with other drugs. It is also being studied in the treatment of other types of cancer. Ifosfamide attaches to DNA in cells and may kill cancer cells. It is a type of alkylating agent and a type of antimetabolite. Also called Ifex.

The use of high-energy radiation from x-rays, gamma rays, neutrons, protons, and other sources to kill cancer cells and shrink tumors. Radiation may come from a machine outside the body (external-beam radiation therapy), or it may come from radioactive material placed in the body near cancer cells (internal radiation therapy). Systemic radiation therapy uses a radioactive substance, such as a radiolabeled monoclonal antibody, that travels in the blood to tissues throughout the body. Also called irradiation and radiotherapy.

A study in which the participants are assigned by chance to separate groups that compare different treatments; neither the researchers nor the participants can choose which group. Using chance to assign people to groups means that the groups will be similar and that the treatments they receive can be compared objectively. At the time of the trial, it is not known which treatment is best. It is the patient's choice to be in a randomized trial.

In medicine, treatment that experts agree is appropriate, accepted, and widely used. Health care providers are obligated to provide patients with standard therapy. Also called best practice and standard of care.

The active ingredient in a drug used to treat acute leukemia. It is used in combination with other drugs to treat Hodgkin disease, non-Hodgkin lymphoma, rhabdomyosarcoma, neuroblastoma, and Wilms tumor. Vincristine is also being studied in the treatment of other types of cancer. It blocks cell growth by stopping cell division. It is a type of vinca alkaloid and a type of antimitotic agent.