Chemotherapy for Unresectable Endocrine Neoplasms

Anne C. Larkin, MD ■ Kathryn L. Edmiston, MD ■ Nilima A. Patwardhan, MD

Neoplasms arising from endocrine organs are rare and have unique clinical features. In contrast with most solid tumors, symptoms from endocrine neoplasms may arise as a result of the production of biologically active substances by the tumor or invasion of surrounding structures and their ability to metastasize. Tumors that are biologically active may become clinically apparent as a result of endocrine hyper-function, whereas those that do not produce active hormones become apparent through local invasion and distant spread.

More sophisticated diagnostic tests have improved the ability to recognize and characterize endocrine neoplasms. The development of radioimmunoassays for circulating peptides produced by endocrine neoplasms has revolutionized the recognition and diagnosis of hormone-secreting tumors. Improved radiologic methods such as ultrasonography, endoscopic ultrasonography, computed tomography (CT), magnetic resonance imaging, radionuclide scans, and selective arteriography with selective venous sampling for measurement of peptide concentration have improved diagnostic localization of endocrine neoplasms.

Most endocrine neoplasms have an indolent clinical course. Survival after diagnosis is often measured in years and decades. Because the morbidity associated with endocrine tumors is often related to the metabolic and endocrine abnormalities caused by hormonal hypersecretion, the suppression of tumor-related peptide secretion can produce relatively long-lasting symptomatic and biochemical remission, even in the absence of treatment directed at halting tumor growth.

Because most endocrine neoplasms are slowly progressive, surgery has been the mainstay of treatment, even in cases of metastatic disease.12 Hepatic resection for metastatic neuroendocrine tumors is safe, provides effective palliation, and prolongs survival.3 Cytotoxic therapy is reserved for patients with unresectable local or metastatic disease.4"6

The efficacy of therapy for endocrine neoplasms is judged not only by the effect of treatment on tumor size but also by its effect on circulating levels of biologically active substances produced by the tumor. The results of clinical trials of chemotherapy for endocrine neoplasms are confounded by the rarity of these tumors and the variable criteria for assessing response. A decrease in the production of hormone may or may not be accompanied by a decrease in symptoms, shrinkage of the tumor, or prolongation of survival.

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