MEK Inhibitors for NF1 Plexiform Neurofibromas: Success Guide

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How Doctors Measure Success

Doctors judge whether a MEK inhibitor is working in two main ways: by measuring the tumor on MRI scans and by asking how you’re feeling, including your pain and how well you can move and function, says Giglio.

Tumor Volume

Plexiform neurofibromas are measured with volumetric MRI, which calculates a tumor’s full three-dimensional size. In clinical trials, a tumor was considered responsive to treatment if it shrank by at least 20 percent and the change held up on a second scan two to six months later.

Because tumors take months to start shrinking, an MRI about every six months is reasonable, though there’s no hard-and-fast rule, says Brown. “If we scan too early and don’t see a change yet, patients can get discouraged,” she says.

Function and Quality of Life

In clinical trials, people rate their worst tumor pain on a scale of 0 to 10 and fill out questionnaires on how much pain gets in the way of daily life and on their overall quality of life.

Outside major academic centers, those questionnaires are rarely part of everyday care, says Brown. Instead, tracking quality of life usually comes down to conversations with your care team, she says.

“Those conversations are different for every patient. Quality of life may involve pain, strength, appearance, vision, swallowing, or even the way your clothes fit,” says Brown.

“It helps to have one goal in mind when you start treatment, whether that’s less pain, walking without a cane, or being able to use an affected arm or leg more easily,” she says.

For some people, success means a tumor that stops growing, or feeling less anxious, which translates to a better quality of life, says Brown.

In a pivotal mirdametinib trial, about 70 percent of adults said they felt at least a little better overall after about a year of treatment.

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