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PEDIATRICS Vol. 103 No. 6 June 1999, pp. 1145-1149

Antiangiogenic Therapy of a Recurrent Giant Cell Tumor of the Mandible With Interferon Alfa-2a

Received Feb 22, 1999; accepted Mar 25, 1999.

Leonard B. Kaban*, John B. Mulliken§, R. Alan Ezekowitz, D PhilDagger , David Ebb, Peter S. Smith, and Judah Folkman§

From the Departments of * Oral and Maxillofacial Surgery and Dagger  Pediatrics, Massachusetts General Hospital, Boston, Massachusetts; the § Department of Surgery, Children's Hospital, Boston, Massachusetts; and the  Division of Pediatric Hematology/Oncology, Rhode Island Hospital, Providence Rhode Island.

We report a 5-year-old girl with a large rapidly growing giant cell tumor of the mandible that recurred 2 months after the first surgical excision and 3 months after a second resection. An angiogenic protein, (bFGF), was abnormally elevated in her urine. The patient was treated with interferon alfa-2a for 1 year because this agent inhibits angiogenesis by suppressing bFGF overexpression in infantile hemangiomas and in other human tumors. During this time the bone tumor regressed and disappeared, the urinary bFGF fell to normal levels, and the mandible regenerated. She has remained tumor-free and has been off therapy for 3 years at this writing.

This first successful use of interferon alfa-2a to treat a mandibular tumor in a child demonstrates: 1) low grade tumors that overexpress bFGF may respond to interferon alfa-2a, in a manner similar to life-threatening infantile hemangiomas; 2) antiangiogenic therapy, given without interruption for 1 year, was safe and effective in this patient; and 3) treatment may be continued for 1 year without the development of drug resistance.

 Key words:  interferon alfa-2a, basic fibroblast growth factor, angiogenesis, giant cell tumor of mandible.




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