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Watch and Wait in Neurofibromatosis Type 1 Cases


 

Basic follow-up tests should include checking blood pressure and monitoring for scoliosis as well as an ophthalmology evaluation and an assessment of developmental skills. “Learning disabilities are common,” she said. “Expressive language delay is the area of development most commonly affected.”

Many parents ask Dr. Bird if an MRI of the brain and optic nerves is needed in children who present with multiple café au lait macules. “There is no evidence that detecting optic gliomas before they're symptomatic translates into better outcome. So you could argue that doing an MRI, which requires anesthesia, is not worth the money or the risk. I am not in the habit of getting routine MRIs. I oblige for the families who really want them.”

NF1 patients with neurofibromas have a 10% lifetime risk of developing a malignant peripheral nerve sheath tumor within one of the lesions. Signs of malignant degeneration include persistent pain, a change in texture, a rapid increase in size, or development of a neurologic deficit associated with the neurofibroma.

Dr. Bird had no relevant disclosures.

'Families should be told that symptoms which are not self-limited need to be brought to your attention.' DR. BIRD

Cafe au lait macules can appear at birth or in the first months of life.

Axillary freckling is usually evident in school-age children.

The Riccardi sign, a tuft of hair near the spine, is often present at birth and may precede other signs of NF1. Photos courtesy Dr. Lynne M. Bird and Dr. Marilyn C. Jones

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