Clinical Perspectives

Moffitt’s Neurofibromatosis Clinic Celebrates Prestigious Designation

September 01, 2018

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Within its first year of operation, the Neurofibromatosis Clinic at Moffitt Cancer Center earned clinic network designation by the Children’s Tumor Foundation (CTF). Under the direction of Xia Wang, MD, PhD and Sepideh Mokhtari, MD, Moffitt’s Neurofibromatosis Clinic became the only adult neurofibromatosis clinic in the state with 98% of its patients over the age of 18.

Neurofibromatosis (NF) is usually diagnosed in childhood or early adulthood with health risks persisting throughout life. NF is actually a group of genetic conditions that can either be inherited or occur for the first the time in the family. Traditionally, comprehensive specialty care has been provided to children in pediatric hospitals. This leaves a great number of adults affected with NF without access to comprehensive specialty care.

NF mainly causes tumors to develop anywhere in the nervous system including the brain, spinal cord and nerves. Most tumors are not cancerous, but complications from NF can compromise multiple organs and systems and lead to hearing loss, blindness, paralysis and severe pain. Some tumors can be cancerous. Very often, each individual can have multiple health conditions as a result from NF. 

Three Types

NF1 affects 1 in 3,000 people and is the most common form of NF. Besides tumors on the nerve system, there is an increased risk for cancerous tumors in multiple organs. Individuals with NF1 may have typical features such as small, light brown patches on the skin (often referred to as café-au-lait spots) and small lumps on the skin as dermal neurofibromas. The trademark, café-au-lait spots, often appear shortly after birth, while dermal neurofibromas usually appear later in childhood or in young adulthood. Tumors on the optic nerve, responsible for vision, can lead to blindness in childhood. Large tumors next to the spine may lead to paralysis. A type of soft tissue cancer, MPNST, can occur in 10% of the individuals with NF1. Increased risk for additional cancers, such as breast cancer and gastrointestinal stromal tumor (GIST) can also be life-threatening. 

NF2 affects about 1 in 25,000 people worldwide. The disorder is characterized by the development of non-cancerous tumors on the nerves that carry sound and balance information from the inner ear to the brain (the eighth cranial nerve). These are called vestibular schwannomas (formerly called acoustic neuromas). These tumors usually affect both ears, often leading to partial or complete hearing loss. Schwannomas develop when Schwann cells, which form the insulating cover around nerve fibers, grow abnormally. Schwannomas may also develop on other nerves throughout the body. Additional non-cancerous but devastating tumors can also develop in the brain and spinal cord, such as meningiomas and ependymomas. 

Schwannomatosis is a rare third form of neurofibromatosis that has only recently been identified. It affects less than 1 in 40,000 people, and causes the development of multiple schwannomas. 

Treatments Available

Moffitt takes a comprehensive, multispecialty approach to care and treatment of individuals with neurofibromatosis.

  • Our surgical oncologists and neurosurgeons use advanced, minimally invasive techniques to remove neurofibromas with the highest level of precision – even from difficult-to-access locations near nerve pathways.
  • Our medical oncologists and radiation oncologists design individualized nonsurgical treatment plans that may include chemotherapy, radiation therapy, as well as novel options such as targeted therapy.
  • Our genetic specialists determine the genetic causes in the blood and tumor and provide care and support for the entire family.
  • Our radiologists provide comprehensive evaluation of the tumors, pathologists provide precise histological information, and personalized medicine team identify targets for cutting-edge therapies and clinical trial options.
  • Our supportive care specialists provide medications and other therapies to ensure the greatest degree of comfort for our patients. For a younger patient, our team can also help his or her parents develop action plans with teachers, psychologists and other key professionals.

“Moffitt is home to so many talents,” says Dr. Wang. ”We have great potential to become not only the center providing comprehensive screening, prevention and treatment in the entire state, but also accelerating in research to contribute to the cure.”

Moffitt’s multidisciplinary approach to treating NF extends outside the walls of the cancer center and brings medical professionals together across Tampa Bay and beyond. Currently, Moffitt’s team collaborates with Tampa Bay Hearing and Balance Center and Dr. Nicholas Avgeropoulos of Orlando Health to treat progressive vestibular schwannoma with Avastin.

“Team work is key and I am so happy we can make it happen for patients,” says Dr. Wang.