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Treating Malignant Peripheral Nerve Sheath Tumors

Name of the Trial

Phase II Study of Neoadjuvant Chemotherapy Comprising Doxorubicin Hydrochloride and Ifosfamide Followed By Etoposide and Ifosfamide in Patients With Sporadic or Neurofibromatosis Type 1-Associated High-Grade Unresectable Stage III or IV Malignant Peripheral Nerve Sheath Tumors (NCI-06-C-0043). See the protocol summary.

Principal Investigator

Dr. Brigitte Widemann

Dr. Brigitte Widemann Principal Investigator

Dr. Brigitte Widemann, NCI Center for Cancer Research.

Why This Trial Is Important

Malignant peripheral nerve sheath tumors (MPNSTs) are soft tissue sarcomas that form in the outer layers of peripheral nerves (nerves outside the brain and spinal cord). About half of MPNSTs are found in individuals with a hereditary condition called neurofibromatosis type 1 (NF1). Surgery is the only curative treatment for MPNSTs. The prognosis for patients with unresectable tumors (tumors that cannot be surgically removed) is poor.

In this trial, patients with unresectable MPNSTs will be treated with neoadjuvant chemotherapy followed by surgery and/or radiation therapy and more chemotherapy. Neoadjuvant chemotherapy is given to reduce the size of the MPNSTs prior to the administration of definitive local therapy (surgery and/or radiation therapy). The choice of treatment following neoadjuvant chemotherapy will be based on tumor location and tumor response to the chemotherapy.

"We hope MPNSTs shrink in response to neoadjuvant chemotherapy with doxorubicin, ifosfamide, and etoposide, which are standard agents with proven activity in other pediatric and adult sarcomas," said Dr. Widemann.

"Furthermore, because outcomes for NF1-associated MPNST have been reported to be worse compared to sporadic tumors," Dr. Widemann said, "we will evaluate outcomes in the two groups treated with identical therapy to determine if patients with NF1-associated MPNSTs face a worse prognosis."

Who Can Join This Trial

Researchers will recruit 74 patients with newly diagnosed sporadic or NF1-associated high-grade stage III or IV MPNSTs. See the list of eligibility criteria.

Study Sites and Contact Information

Study sites in the United States are recruiting patients for this trial. See the list of study contacts or call NCI's Cancer Information Service at 1-800-4-CANCER (1-800-422-6237) for more information. The toll-free call is confidential.

  • Posted: September 12, 2006