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

• Published in: Pediatrics (Evanston) 1999-06, Vol.103 (6), p.1145-1149
• Main Authors: Kaban, Leonard B, Mulliken, John B, Ezekowitz, R. Alan, Phil, D, Ebb, David, Smith, Peter S, Folkman, Judah
• Format: Article
• Language: English
• Subjects: Antineoplastic Agents - therapeutic use; Biological and medical sciences; Bones; Case studies; Child, Preschool; Clinical outcomes; Diseases of the osteoarticular system; Drug therapy; Female; Fibroblast Growth Factor 2 - genetics; Giant cell tumors; Giant Cell Tumors - diagnostic imaging; Giant Cell Tumors - drug therapy; Giant Cell Tumors - surgery; Head and neck tumors; Health aspects; Humans; Interferon; Interferon alpha; Interferon alpha-2; Interferon-alpha - therapeutic use; Mandible - blood supply; Mandibular Neoplasms - diagnostic imaging; Mandibular Neoplasms - drug therapy; Mandibular Neoplasms - surgery; Medical sciences; Neoplasm Recurrence, Local; Neovascularization, Pathologic - genetics; Pediatric tumors; Pediatrics; Recombinant Proteins; Tomography, X-Ray Computed; Tumors; Tumors in children; Tumors of striated muscle and skeleton
• ISSN: 0031-4005; 1098-4275
• DOI: 10.1542/peds.103.6.1145

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.