Kaban, Leonard B., Mulliken, John B., Ezekowitz, R. Alan, Phil, D, Ebb, David, Smith, Peter S., and Folkman, Judah
Interferon alfa-2a was used successfully to treat a recurrent giant cell tumor of the mandible in a 5-year-old girl. The child developed a rapidly growing tumor of the jaw that was surgically removed twice, but continued to grow. Interferon alfa-2a inhibits the development of new blood vessels, and has been used in vascular tumors. Doctors began treating the child with interferon, desiring to avoid disfiguring surgery and radiation therapy. Progressively higher doses of interferon effectively eradicated the tumor, and the child remained free of the tumor three years after therapy was terminated., 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 he mangiomas; 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. Pediatrics 1979;103:1145-1149; interferon alfa-2a, basic fibroblast growth factor, angiogenesis, giant cell tumor of mandible., ABBREVIATIONS. bFGF, basic fibroblast growth factor; VEGF, vascular endothelial growth factor. Interferon alfa-2a was found to inhibit angiogenesis through a series of laboratory experiments that began in 1980.[1-3] In 1989, [...]