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Pediatric Plexiform Neurofibromas
Key points about plexiform neurofibroma:
- Plexiform neurofibromas are a common complication of neurofibromatosis.
- Diagnosed through physical examinations. However, the full extent of the lesion is best seen by MRI scans.
- Approximately 75 percent of patients who undergo a complete removal of the tumor — without causing significant neurologic impairment or dysfunction — are cured.
Children's National Hospital is a member of the Department of Defense Neurofibromatosis Clinical Trials Consortium — of which Roger Packer, M.D. acts as group chair. Children's National completed a trial evaluating a new biologic agent, Pirfenidone, and is currently evaluating the efficacy of another biologic agent, Rapamycin, (through the Department of Defense Consortium), which is designed to inhibit the proliferation of plexiform neurofibromas.
What are plexiform neurofibromas?
Plexiform neurofibromas are a common complication of neurofibromatosis.
What causes plexiform neurofibromas?
Plexiform neurofibromas are predominantly inherited tumors that can occur anywhere in the body. This includes the head and neck, extremities, areas around the spine and deep in the body where they may affect organs.
What are the risk factors of plexiform neurofibromas?
Although these tumors tend to grow slowly, they may grow to an enormous size and can cause serious disfigurement, brain dysfunction or impingement on other organs. They may also cause pain.
How are plexiform neurofibromas treated?
Until recently, the only known effective treatment for plexiform neurofibromas has been surgery. Approximately 75% of patients who undergo a complete removal of the tumor without causing significant neurologic impairment or dysfunction are cured.
Can all plexiform neurofibromas be surgically removed?
Children's National Hospital and other sites around the country are developing new therapies for patients whose tumors can only be partly removed. Most of these treatments are biologically based and designed to treat the genetic foundations of the tumor.
How common are plexiform neurofibromas?
These types of congenital lesions occur in up to 60% of patients with neurofibromatosis. However, only a minority of these patients is symptomatic and requires treatment.
How are plexiform neurofibromas diagnosed?
Although diagnosis of these lesions is usually possible by examination of the child, the full extent of the lesion is best seen by MRI scan. MRI scans may show these lesions to be much more extensive than previously thought. For those patients with tumors around the spine, the lesions may be extremely extensive.