Surgery, medication, radiation therapy, endocrine treatment, or clinical trials ¹⁰

These are some of the common treatment options for neurofibromatosis, a group of genetic disorders that cause tumors on nerve tissue:

• Surgery: Surgery can remove tumors that cause pain, disfigurement, functional impairment or cancer risk. Surgery can also correct bone deformities that result from neurofibromatosis. However, surgery may not be possible or effective for some tumors, and may have risks, such as nerve damage, bleeding, infection or recurrence of tumors. ¹⁰
• Medication: Medication can help manage symptoms and complications of neurofibromatosis, such as pain, seizures, high blood pressure, headaches and behavioral problems. Medication can also slow down or shrink some types of tumors, such as plexiform neurofibromas in NF1 or vestibular schwannomas in NF2. Some examples of medication used for neurofibromatosis are analgesics, anticonvulsants, antihypertensives, antidepressants and antitumor drugs, such as MEK inhibitors, mTOR inhibitors and immunotherapy. ¹⁰
• Radiation therapy: Radiation therapy uses high-energy beams to kill tumor cells or stop their growth. Radiation therapy can treat tumors that are cancerous, large, symptomatic or difficult to remove by surgery. Radiation therapy can also help preserve hearing and balance in people with NF2 who have vestibular schwannomas. However, radiation therapy may have side effects, such as skin irritation, fatigue, nausea, hair loss or increased risk of secondary cancers. ¹⁰
• Endocrine treatment: Endocrine treatment can regulate the hormones that affect growth and development in people with neurofibromatosis. Endocrine treatment can also prevent or treat some of the complications of neurofibromatosis, such as early puberty, short stature, low bone density or thyroid problems. Endocrine treatment may involve hormone replacement therapy, growth hormone therapy, bisphosphonates or other medications that affect the endocrine system. ^{10 1}
• Clinical trials: Clinical trials are research studies that test new treatments or procedures for neurofibromatosis. Clinical trials can offer access to experimental therapies that are not yet available or approved for general use. Clinical trials can also help advance the scientific knowledge and understanding of neurofibromatosis. However, clinical trials may have unknown risks, benefits or outcomes, and may not be suitable or effective for everyone. Participation in clinical trials is voluntary and requires informed consent. ¹⁰