Houston Forum Highlights Awareness Gaps in NF1 Plexiform Neurofibroma Care

On September 2, 2026, NeurologyLive and Targeted Oncology convened a clinical forum in Houston titled “Recognizing and Treating Plexiform Neurofibromas in NF1: A New Era of Systemic Management,” bringing together community oncologists, hematologists, and neurologists to discuss the recognition, referral, and treatment of neurofibromatosis type 1 (NF1) and its associated plexiform neurofibromas (PN). The program was led by Kevin J. Bielamowicz, MD, a pediatric neuro-oncologist who co-directs the multidisciplinary NF1 clinic at Arkansas Children’s Hospital in Little Rock, Arkansas.

Bielamowicz opened by noting how often NF1, affecting roughly 1 in 3,000 births, surfaces through channels that have nothing to do with a formal NF referral. Attendees described patients identified through workups for iron overload, treatment-resistant blood clots, or imaging ordered for unrelated cancers, rather than a direct referral for NF1. That disconnect, Bielamowicz said, often starts even earlier, when pediatric patients age out of dedicated NF clinics. “A lot of times in pediatrics we have these really nice subspecialty clinics, and then we graduate patients and they don’t really have a place to go,” he said. “Sometimes I think they just don’t end up getting regular follow up, and they’re just kind of living with the condition.”

Using the case of a 44-year-old man whose facial PN was first noticed at age 20 and had grown for 24 years before treatment, Bielamowicz walked attendees through why surgery, the longtime default for PN, so often falls short. Because these tumors infiltrate nerves and surrounding tissue, a maximal safe resection, rather than complete removal, is typically the goal, and incomplete resection raises the odds of regrowth. He described a teenager who underwent an 80% resection of a head and neck PN only to find it back to its original size five years later. In the case patient, the tumor’s size and its proximity to the facial nerve led the surgical team to deem it inoperable.

That reality has pushed MEK inhibitors, selumetinib and mirdametinib, to the center of the conversation. Attendees compared the two agents’ trial data at length, several noting that mirdametinib’s higher response rate in adults, without head-to-head data to confirm superiority, shaped their preference. Bielamowicz cautioned against over-reading cross-trial comparisons but acknowledged the data pointed in a consistent direction. In the case patient, a MEK inhibitor produced a 24% reduction in tumor volume at one year, a result he considered clinically important even though it fell short of a complete response. “In some patients, a 24% reduction is very meaningful,” he said. “It can help their pain, it can help their edema, it can help their physical appearance.”

Despite the expanding treatment landscape, live polling and discussion pointed to a persistent awareness gap as the leading barrier to care, ahead of access or cost. Several attendees said they had not known systemic therapy existed for NF1-PN until the forum itself. Bielamowicz traced part of that gap back to training: pediatric residents at his own institution, he said, “literally get a mention of NF in one talk in the entire three years” of residency. Attendees identified two paths forward, starting MEK inhibitor therapy earlier, before a PN becomes disfiguring or symptomatic, and expanding education for pediatricians and referring physicians so patients reach systemic treatment sooner.

Bielamowicz closed the evening by thanking the group for a conversation he called more substantive than he expected from physicians who rarely treat NF1 directly. “For folks who don’t do a ton of NF, I thought your questions were excellent, and it was a great conversation,” he said. For a condition that so often hides behind another diagnosis, that kind of cross-specialty engagement, organizers said, may do as much to close the referral gap as any single new therapy.