Multiple Endocrine Neoplasia Type 1 (MEN 1)

Treatment

Treatment options for multiple endocrine neoplasia type 1 (MEN 1) depend on each patient's situation. In the disease's early stages, specialists recommend surgery to remove some tumors and therapy to regulate hormones. If the cancer has spread (metastasized), then chemotherapy, radiation or other specialty care may be necessary.

Treatment for Pituitary Tumors

Physicians recommend treatment based on the type of pituitary tumor. After completing surgical or drug therapy for a pituitary tumor, MEN 1 patients should be screened regularly to rule out another pituitary tumor arising in the remaining tissue.

Treatment for Primary Hyperparathyroidism

Most MEN 1 patients will develop hypercalcemia (too much calcium in the blood) and hyperparathyroidism (too much parathyroid hormone) by the fourth decade of life. The first signs of hyperparathyroidism can be detected much earlier when at-risk patients undergo regular screening.

Surgical management of hyperparathyroidism is complex. MEN 1 hyperparathyroidism involves all parathyroid tissue. Surgery is indicated for high levels of calcium and management of complications including kidney stones and bone loss. Removing all parathyroid tissue can result in symptoms that are worse than the disease, including permanent hypoparathyroidism. Mayo specialists recommend delaying surgery until the patient's calcium level is unacceptably high or the patient develops kidney stones, bone fractures or Zollinger-Ellison syndrome.

Surgeons take two basic approaches to parathyroid surgeries in MEN 1 patients with hyperparathyroidism: subtotal parathyroidectomy or total parathyroidectomy with immediate autotransplantation. Mayo specialists usually recommend a subtotal parathyroidectomy. This provides acceptably low recurrence rates and minimal risk of permanent hypoparathyroidism. Patients with remnant or recurrent MEN 1 parathyroid tumors may be candidates for ultrasound-guided alcohol ablation at Mayo Clinic.

Treatment for Pancreatic and Duodenal Neuroendocrine Tumors

Pancreatic and duodenal neuroendocrine tumors represent the second most frequent manifestation of MEN 1. Tumors in the duodenum (small intestine) are typically small and located below the inner lining. Often, multiple tumors are present. All pancreatic and duodenal endocrine tumors can transform into malignant tissue. Because pancreatic and duodenal endocrine tumors are the number one cause of tumor-related death in MEN 1 patients, early detection and intervention is important.

Specialists at Mayo recommend surgery for patients with two rising serum markers that occur six months apart. Surgery is recommended when multiple tumors are seen on endoscopic ultrasound. The type of surgery for removing the tumors depends on the type of tumor or syndrome that develops in the pancreas or duodenum.

Treatment for Hypoglycemic Syndrome

Tumors associated with an excess of insulin (insulinomas) can occur in MEN 1 patients. Although these patients often have multiple pancreatic tumors, typically only one or two create excess insulin. Although these tumors are most often benign, no effective medical therapy exists. Without surgery, insulin imbalance can remain debilitating and life threatening.

Surgical intervention involves a distal pancreatectomy and shelling out (enucleation) of any remaining panreatic tumors that exist in the head of the pancreas. These tumors can often be felt by the surgeon or seen by intraoperative ultrasound. The spleen may be preserved in some patients. Leaving 20 percent of the pancreas generally will limit recurrences and help avoid endocrine insufficiency, such as diabetes, in most cases.

Treatment for Other Pancreatic Endocrine Tumors

MEN 1 patients with malignant islet cell tumors (glucagonomas, VIPomas, PTHrPomas, and some insulinomas) require an aggressive approach, including eliminating 80 percent of the pancreas (distal pancreatectomy) and removing the spleen, nearby lymph nodes and enucleating any remaining tumors. Isolated liver metastases or some other metastases can be successfully managed using a combination of hepatic resection, radiofrequency ablation, and hepatic artery embolization.

Treatment for Zollinger-Ellison Syndrome (ZES)

Gastrinomas (tumors that result in overproduction of gastric acid leading to ulcers and diarrhea) represent the most common functioning pancreatic or duodenal endocrine tumors in MEN 1 patients. One-half of MEN 1 patients develop ZES. Medical therapy with proton pump inhibitors manages the gastrin excess but does nothing to prevent cancer or progression of the disease. The procedure most often performed for the MEN 1 ZES patient involves a distal pancreatectomy, lymphadenectomy, enucleation of residual tumors in the head of the pancreas, and an exploratory duodenotomy to remove all visible duodenal carcinoid tumors. Other operative approaches include total pancreatectomy, pancreatoduodenectomy, and pancreas-sparing duodenectomy.

Treatment for Advanced Pancreatic Disease

The management of advanced disease (typically hepatic metastases) may involve hepatic resection and radiofrequency ablation. When surgery is no longer an option, physicians use chemotherapy, interferon and hormonal agents to stabilize the patient and gain control of excess hormones.

Treatment for Adrenal Tumors

Thirty-five to 40 percent of MEN 1 patients develop tumors in the adrenal glands. Most tumors do not produce hormones and are benign. Surgical removal of the tumor is common. In advanced cases, radiation and chemotherapy may be used. Benign, nonfunctioning tumors can be observed for changes.

Treatment for Carcinoid Tumors

Carcinoid tumors in MEN 1 patients arise in the lungs, thymus gland and gastrointestinal tract. They represent the second most common cause of tumor-related deaths in MEN 1 patients. Surgeons remove these tumors when they haven't spread to other areas of the body. Chemotherapy and radiation may be used for advanced cases.