Chemotherapy for Neurofibromatosis

Neurofibromatosis (NF) is a group of genetic conditions that cause tumors to form along nerves in the brain, spinal cord and other parts of the body. These rare disorders result from harmful changes in the genes responsible for regulating normal cell growth. When these genes do not function properly, cells can grow uncontrollably, leading to tumor formation over time. Most NF-related tumors are noncancerous, but some may continue to grow and eventually become malignant.

The main types of neurofibromatosis are NF1, NF2 and schwannomatosis. NF1 is the most common form, affecting approximately 1 in 3,000 people. It results from a mutation in the NF1 gene on chromosome 17, which provides instructions for neurofibromin, a tumor suppressor protein that helps regulate normal cell growth and division. Symptoms may include café-au-lait skin spots, small growths on or under the skin and, in some individuals, learning or cognitive challenges.

NF2 is a less common type of neurofibromatosis characterized by acoustic neuromas (vestibular schwannomas) that form on the nerve connecting the inner ear to the brain. If left untreated, these tumors can cause hearing loss, tinnitus and balance difficulties.

Schwannomatosis is the rarest form of NF. It involves the development of tumors in Schwann cells, which wrap around and protect nerve fibers. Although these schwannomas can cause significant chronic nerve pain, they are benign and do not become cancerous.

While neurofibromatosis is not considered curable, many of its symptoms and complications can be managed effectively. Treatment can vary based on the tumor’s type and behavior and the patient’s overall health. Common approaches include chemotherapy, radiation therapy, surgery, dietary counseling and clinical trials. Chemotherapy may be appropriate if an NF-related tumor becomes cancerous or its growth needs to be controlled.

What is chemotherapy and how does it work?

Chemotherapy is a medication-based treatment that enters the bloodstream and circulates throughout the body to target rapidly dividing cells. It works by disrupting the processes these cells use to grow and multiply. Because cancer cells reproduce faster than most healthy cells, chemotherapy can be an effective tool for slowing or stopping tumor progression.

In NF-related cancers or situations where tumor growth threatens organ function, chemotherapy may be recommended as part of a broader treatment plan. The specific drug regimen will depend on the tumor’s type and growth rate and the patient’s overall health.

What are the goals of chemotherapy for neurofibromatosis?

The purpose of chemotherapy can vary depending on the specific diagnosis and the behavior of the tumor. This powerful systemic treatment may be used to shrink a tumor, slow its growth or ease symptoms caused by pressure on nearby nerves or organs. Additionally, if an NF-related tumor has transformed into a malignant peripheral nerve sheath tumor (MPNST) or another type of cancer, chemotherapy can help destroy cancerous cells that have spread beyond the original site.

While chemotherapy cannot cure neurofibromatosis or eliminate all NF-related tumors, it can play an important role in controlling cancerous changes and reducing tumor-related complications. When used appropriately, it may help improve systems, prolong survival and enhance the patient’s overall quality of life.

How is chemotherapy for neurofibromatosis administered?

Chemotherapy is usually delivered intravenously, either through a needle inserted into a vein in the arm or hand or through a port placed under the skin. While the treatment schedule can vary depending on the specific medications prescribed, chemotherapy is often administered in cycles. Each cycle includes a period of active treatment followed by a period of rest to allow the body time to recover and rebuild healthy cells.

The healthcare team will tailor the chemotherapy regimen to the specific type of NF-related tumor, its location and how the patient’s body responds over time. Regular monitoring can help ensure the treatment remains effective and that any side effects are managed promptly.

What to expect before, during and after chemotherapy for neurofibromatosis

Chemotherapy is a structured, step-by-step process designed to support the patient’s health and comfort from the beginning of treatment through recovery. Understanding what to expect at each stage can reduce uncertainty and help the patient feel better prepared. To help ensure the best possible experience, the healthcare team will offer personalized guidance, answer questions and provide access to important resources before, during and after each treatment cycle.

Preparing for chemotherapy for neurofibromatosis

Before starting chemotherapy, the patient will meet with the healthcare team to review their medical history, imaging results and diagnostic tests. Blood work will be performed to ensure the patient is healthy enough for treatment, and some individuals may have a port placed to make ongoing infusions easier and more comfortable.

Chemotherapy for neurofibromatosis is typically managed by a multispecialty team, which may include medical oncologists, neuro-oncologists, nurse practitioners, infusion nurses and pharmacists. Working together, these specialists will design and deliver the treatment plan while closely monitoring the patient’s health, side effects and overall response throughout the process.

During chemotherapy for neurofibromatosis

On each day of active treatment, the patient will check in at the hospital or outpatient clinic, have their vital signs assessed and speak with a nurse about how they are feeling. The infusion will be administered in a comfortable treatment area where pillows, blankets, beverages, snacks and other amenities will be available to help the patient relax. During the appointment, the patient may choose to read, browse the internet, watch television, listen to music or simply rest. Depending on the specific chemotherapy medications used, the infusion session may last anywhere from less than an hour to several hours.

After chemotherapy for neurofibromatosis

Between active treatment sessions, the patient can continue their usual activities within the limits recommended by the healthcare team. Because fatigue is common during chemotherapy, pacing daily activities and planning for additional rest can be helpful. The patient should promptly report any new symptoms or concerns, as early communication can allow the healthcare team to address side effects right away and adjust the treatment plan as needed.

What are the possible side effects of chemotherapy for neurofibromatosis?

The side effects of chemotherapy can vary based on the specific drugs used, the treatment schedule and the patient’s overall health. Most side effects are temporary and can be managed with supportive care and close monitoring.

Common side effects of chemotherapy for neurofibromatosis

Some of the more frequently reported side effects of chemotherapy include:

• Fatigue
• Nausea or vomiting
• Hair thinning or loss
• Reduced appetite
• Changes in bowel habits
• Increased risk of infection
• Mouth sores
• Skin changes

Possible long-term and late-onset side effects of chemotherapy for neurofibromatosis

Some side effects may appear later in treatment or persist after chemotherapy ends. These may include:

• Neuropathy
• Fertility changes
• Heart or lung effects, depending on the medications used
• Cognitive changes
• Increased risk of secondary cancers (rare)

How to manage daily life during chemotherapy for neurofibromatosis

During treatment, maintaining a balanced diet, staying well hydrated and engaging in light physical activity can help support energy levels and overall well-being. The healthcare team may also refer the patient to a dietitian for personalized nutrition guidance.

Because chemotherapy can weaken the immune system, it is important to practice good hygiene, such as frequent handwashing, avoiding contact with people who are ill and following any additional precautions recommended by the healthcare team.

Regular follow-up appointments will remain essential, allowing the healthcare team to monitor the patient’s progress, manage any side effects and adjust the treatment plan as needed.

Benefit from world-class care at Moffitt Cancer Center

Moffitt offers comprehensive expertise in treating neurofibromatosis, including rare MPNSTs and other complex tumors. Our multispecialty Neuro-Oncology Program brings together experts from multiple fields who work collaboratively to deliver advanced, individualized care. Moffitt consistently achieves outcomes that surpass national benchmarks, and we are recognized internationally for our leadership in cancer research, treatment innovation and patient-centered excellence.

Chemotherapy for neurofibromatosis is available at all Moffitt locations, and we do not require referrals. If you would like more information, you can request an appointment with a specialist in our Neuro-Oncology Program by calling 1-888-663-3488 or submitting a new patient registration form online.