Are there any new medicines to safely treat my neurofibroma?

Question

Hello doctor,

I have NF1 and am now noticing some pain and tingling in my arm where I have a big plexiform neurofibroma. I have a few concerns:

Can these tumors cause nerve damage or paralysis?
Is surgery safe, or do they grow back after?
I am also scared it might turn into cancer. How will I know early?
Are there any new medicines to shrink these safely without surgery?

Kindly help.

Answer 1

Hello,

Welcome to icliniq.com

I read your query and understand your concern.

Living with neurofibromatosis type 1 (NF1) and a plexiform neurofibroma (PN) can be physically and emotionally difficult, especially when new symptoms like pain or tingling develop.

Especially when located near major nerves like the brachial plexus or spinal nerve roots. Pain, tingling, numbness, or weakness often means the tumor is compressing or infiltrating nerves. Over time, this pressure can cause permanent nerve damage or even paralysis if not addressed. Rapid changes in symptoms need urgent medical evaluation.

Surgery can be helpful, but it is complicated. Plexiform neurofibromas are often entwined with nerves and blood vessels, making complete removal difficult. Surgery can reduce pain and pressure, especially if the tumor is impairing function. Recurrence is common if the tumor is not completely removed, and even full resection does not guarantee that it will not grow back. There is a risk of nerve damage during surgery, especially with large, complex tumors. Surgical decisions should be made in specialty NF1 centers with experience in PNs and neuro-oncology.

A small percentage (eight to 13 percent) of plexiform neurofibromas can become a malignant peripheral nerve sheath tumor (MPNST), the most serious complication.

Warning signs of malignant transformation:

New or worsening pain, especially deep, constant pain.
Rapid growth of the tumor.
Firmness or change in texture.
Sudden changes in sensation or motor function (weakness, loss of mobility).
Unexplained fevers or weight loss.

If any of these are happening, you should get an urgent MRI (magnetic resonance imaging) with contrast and possibly a PET (positron emission tomography) scan or biopsy, depending on what imaging shows. Selumetinib is a game-changing oral medication. Other MEK inhibitors like Trametinib and Mirdametinib are being studied in clinical trials for adults with NF1.

I hope this helps.

Thank you.