Pheochromocytomas and paragangliomas are rare neural crest–derived tumors of sympathetic (generally catecholamine producing) or parasympathetic (rarely catecholamine producing) origin. Patients affected by these tumors present with a variable clinical picture, often making diagnosis troublesome. Surgery is the treatment of choice, but requires appropriate medical management before, during, and after tumor resection. Appropriate follow-up of patients is particularly important to identify recurrences, remaining disease, or developing malignancy. Currently, however, no firm guidelines exist about what form follow-up should take. There is also a general lack of prospective studies establishing the best approaches for management and treatment of the tumor. Choice of the many available different therapeutic options instead usually depends on institutional experience and clinical setting, which may vary for different groups of patients.
Although several therapeutic options exist for patients with metastatic pheochromocytoma, all options are limited and there is no cure. Reduction of tumor size palliates symptoms, but a survival advantage of debulking is unproven. A reduced tumor burden can facilitate subsequent radiotherapy or chemotherapy. External-beam irradiation of bone metastases and radiofrequency ablation of lesions are treatment alternatives. Chemotherapy with a combination of cyclophosphamide, vincristin, and dacarbazine can provide tumor regression and symptom relief in up to 50% of patients, but the responses are usually short-lived. To date, 131I-labeled MIBG therapy is the single most valuable adjunct to surgical treatment of malignant pheochromocytomas. As a single agent, 131I-labeled MIBG has a limited efficacy of cure, and there is no consensus on what doses to use for treating either bone or organ metastases. Multicenter studies are required to reach a consensus on the efficacy of high-dose versus fractionated medium doses of 131I-labeled MIBG and monotherapy versus combination therapy with other radionuclides or modes of chemotherapy.
A combination of temozolomide and thalidomide achieved a 40% biochemical and a 33% radiological response in patients with malignant chromaffin-cell tumours. However, as best as I can tell, there are no single agent thalidomide studies and no other studies.
MASSIMO MANNELLI Management and Treatment of Pheochromocytomas and Paragangliomas Pheochromocytoma: First International Symposium Volume 1073 published August 2006
Ann. N.Y. Acad. Sci. 1073: 405–416 (2006).
Matthew H. Kulke, Keith Stuart, Peter C. Enzinger, David P. Ryan, Jeffrey W. Clark, Alona Muzikansky, Michele Vincitore, Ann Michelini, Charles S. Fuchs Phase II Study of Temozolomide and Thalidomide in Patients With Metastatic Neuroendocrine Tumors Journal of Clinical Oncology, Vol 24, No 3 (January 20), 2006: pp. 401-406
A. Chrisoulidou, G. Kaltsas, I. Ilias, and A. B Grossman
The diagnosis and management of malignant phaeochromocytoma and paraganglioma
Endocr. Relat. Cancer, September 1, 2007; 14(3): 569 – 585.