Treatment for plexiform neurofibromas depends on the size, location and symptoms of the tumor. Some tumors don’t need treatment right away. Others may require surgery or medicine to manage pain, improve function or reduce tumor size.
- Surgery. This is the most common treatment for plexiform neurofibroma. Surgeons may try to remove a tumor that causes problems. But many plexiform neurofibromas are hard to remove completely because they grow into nearby nerves and tissues. This can make surgery risky and may lead to complications such as nerve damage, bleeding or slow healing.
- Medicine. Selumetinib (Koselugo), a pill taken by mouth, is an MEK inhibitor approved by the U.S. Food and Drug Administration (FDA) for children age 2 and older with plexiform neurofibroma tumors. This medicine helps shrink tumors by blocking a signal in cells that causes unhealthy growth. Mirdametinib (GOMEKLI), another MEK inhibitor, also is an FDA-approved medicine taken by mouth for use in adults and children age 2 and older with NF1 who have plexiform neurofibromas that cause symptoms and cannot be completely removed.
- Monitoring. Because these tumors often grow slowly, healthcare professionals usually monitor them with regular checkups. Sometimes this monitoring is all that’s needed. Imaging may be repeated if the tumor grows or new symptoms appear, such as pain, numbness or weakness. A sudden change in symptoms may be a warning sign that the tumor is growing or becoming cancerous.
- Supportive care. Some children with plexiform neurofibromas benefit from therapies that help them stay active and independent. Physical therapy can improve strength, balance and coordination. Occupational therapy may help with fine motor skills such as writing, getting dressed or using utensils. At times, pain or discomfort related to plexiform neurofibromas may be treated with medicines targeting nerve pain.
Accommodations for living with plexiform neurofibroma
People living with plexiform neurofibromas (PNFs) may need certain accommodations depending on tumor size, location and symptoms such as pain, fatigue or physical limitations.
Depending on your symptoms and how they impact your daily life, accommodations may include:
- Mobility support, including wheelchair access, accessible seating or limits on physical activity.
- Flexible scheduling or rest breaks to manage fatigue or pain.
- Excused absences for medical appointments or therapy.
- Learning supports, such as extra time or assistive technology, if thinking skills are affected.
- Adaptive furniture or layout changes to reduce pressure or discomfort caused by tumors.
- Therapy or counseling, such as counseling for body image, stigma or adjustment issues.
Prognosis
There is no cure for plexiform neurofibromas, but many people live well with them for years. Some tumors stay the same size and don’t cause symptoms, while others may grow and lead to pain, changes in appearance, or pressure on nearby nerves or tissues.
Regular checkups and imaging tests are important to watch for any changes in plexiform neurofibromas. In some cases, a plexiform neurofibroma can turn into an aggressive type of cancer called malignant peripheral nerve sheath tumor (MPNST).
Most plexiform neurofibromas do not become cancerous. With routine monitoring, and treatment when symptoms appear, many people are able to manage the tumors and maintain a good quality of life.
Potential future treatments
Researchers continue to study MEK inhibitors beyond selumetinib as treatments for plexiform neurofibromas. These medicines work by blocking signals that tell tumors to grow. This may help shrink the tumors or slow their growth.
Researchers are now studying whether other MEK inhibitors — such as trametinib (Mekinist) and binimetinib (Mektovi) — can provide similar or better tumor reduction with fewer or more manageable side effects compared with selumetinib. Cabozantinib (CABOMETYX) — a multityrosine kinase inhibitor — is under investigation.
