Exploring Novel Treatment Options for Neurofibromatosis Type 1 With Plexiform Neurofibromas

Neurofibromatosis type 1 (NF1) is a common genetic disorder marked by a range of manifestations, including plexiform neurofibromas (PNs) that cause significant morbidity and require lifelong management. In a recent The American Journal of Managed Care® (AJMC) Stakeholder Summit moderated by Ryan Haumschild, PharmD, MS, MBA, vice president of ambulatory pharmacy services at Emory Healthcare and Winship Cancer Institute, a panel of experts discussed the evolving NF1-PN treatment landscape. They highlighted the transformative impact of newly FDA-approved mirdametinib alongside selumetinib and underscored the importance of multidisciplinary teams, proactive surveillance, and whole-body MRI to track disease burden and tailor care, particularly during the transition from pediatric to adult case management.

Epidemiology, Burden, and Current Management of NF1-PN

NF1 is a common autosomal dominant genetic disorder that affects approximately 1 in 2500 to 3000 individuals worldwide and is caused by mutations in the NF1 gene, which encodes the tumor suppressor protein neurofibromin.1,2 NF1 is inherited in an autosomal dominant pattern, with about half of cases due to de novo mutations. Each child of an affected individual has a 50% chance of inheriting the disease, and penetrance is nearly 100%, though clinical severity varies widely.3

Approximately 25% to 30% of patients with NF1 develop PNs, benign nerve sheath tumors that can be congenital and grow throughout life.2,4,5 PNs are typically found in the craniofacial region, neck, pelvis, and lower extremities.4 These tumors can cause significant morbidity due to disfigurement, pain, functional impairment, and a risk of malignant transformation to malignant peripheral nerve sheath tumors (MPNSTs).4

Beyond physical symptoms, about one-third of children and adolescents with NF1-PN experience social-emotional difficulties, including anxiety, depression, and social withdrawal, with worse disease severity linked to poorer quality of life (QOL).4 Patient-reported outcomes from pediatric and adult trials show that larger tumor volumes correlate with worse QOL in adults.4 These psychosocial impacts vary by age, underscoring the need for lifelong assessment and support.4

Whole-body MRI is the preferred imaging modality to identify and characterize PNs, particularly during the transition from pediatric to adult care. It helps assess size and involvement with critical structures, informs management decisions, and highlights the link between total body PN burden and lifetime risk of malignant transformation.4 Treatment decisions should involve a multidisciplinary team to determine whether surgery or medical management is most appropriate. Although surgery has historically been the mainstay of treatment, complete resection is often impossible due to the infiltrative and complex nature of these tumors.4

Stakeholder Insights

Pathophysiology, Epidemiology, and Diagnosis

Lionel Chow, MD, PhD, pediatric hematologist oncologist at Dayton Children’s Hospital and associate professor of medicine at Wright State University Boonshoft School of Medicine, provided an overview of the genetic and molecular mechanisms driving NF1 pathophysiology, describing it as a classic tumor suppressor condition due to NF1 mutations that disrupt neurofibromin. “Neurofibromin regulates the RAS-MAP-kinase pathway, and it’s…one of the major brakes of this pathway,” Chow explained. “When neurofibromin or the NF1 gene is mutated and inactivated, it can no longer catalyze GTP to GDP and so RAS is kept in the GTP-bound form…[leading] to sustained activation of this downstream series of events...which ultimately leads to [uncontrolled] cell growth…[and] different tumors.”

Mina Lobbous, MD, MSPH, staff neuro-oncologist at the Cleveland Clinic Foundation and medical director of the Adult Neurofibromatosis Clinic, described NF1 as “the most common tumor suppressor syndrome,” usually diagnosed in early childhood, with about 80% of individuals diagnosed before they were aged 6 years and 95% before they were aged 8 years. Diagnosis is typically clinical, based on meeting 2 of the key criteria, including café au lait spots, Lisch nodules, optic pathway gliomas, neurofibromas, bony lesions, or family history.

Maciej M. Mrugala, MD, PhD, neuro-oncologist, professor of neurology and medicine, and codirector of the Neurofibromatosis Clinic at Mayo Clinic and Mayo Clinic Cancer Center, explained that PNs are a hallmark of NF1 and part of the National Institutes of Health diagnostic criteria. “In terms of the anatomical description, plexiform neurofibromas are typically soft, spongy lesions, very diffuse. In literature, they’re frequently compared to a bag of worms on palpation. They typically transgress multiple anatomical planes and can be located anywhere where peripheral nerves are,” Mrugala noted, adding that common sites include the head and neck, as well as the limbs. Some PNs can remain undetected for years. He highlighted 3 main symptoms: neurological issues (weakness or sensory problems), pain that can be debilitating, and disfigurement, especially in the face or limbs.

Mrugala noted that PNs are typically present by age 5 years, with faster growth in children and more chronic pain in adults. The risk of malignant transformation, he said, is about 15% to 20% in adults, adding, “It’s critically important to detect these tumors…[to] guide monitoring.” Haumschild emphasized Mrugala’s point. “It’s important to get that diagnosis done correctly and soon, not only for quality of life with pain and disfigurement, but…[also for] malignant transformation…[so] that clinicians have the right awareness…and the right access to the therapies early on,” he said.

Prognosis and Disease Burden

Lobbous described the prognosis for NF1-PN as variable, depending on tumor location and associated complications. “It’s ranging from disfigurement with the impact on psychosocial status and how patients feel and self-image, mobility, weakness, sensory changes.… With head and neck, tumors can affect swallowing and breathing as well,” he said. Regarding mortality, he noted that survival in patients with NF1 is “10 to 15 years younger than [the] general population, driven by…the transformation of these plexiform neurofibromas into very aggressive malignant tumors.... Even with aggressive treatment for the MPNST, the 5-year survival is less than 50%.”

Mrugala highlighted the profound impact of NF1-PN on patients’ quality of life, noting how symptoms vary by age, location, and size, with small tumors in critical areas impairing swallowing or breathing and large limb tumors severely limiting mobility and preventing normal clothing wear, greatly impacting daily life.

Beyond physical symptoms, Mrugala underscored the emotional toll. “These patients may isolate themselves because they feel stigmatized,” he said. “I think we need to change this perception of the disease and educate communities so patients don’t feel stigmatized by having this disease.… Social isolation and having NF1-related PN can lead to psychological conditions such as anxiety and depression,” which often go unaddressed.

Pain also requires individualized care. “Pain is truly one of the main symptoms…and management of the pain needs to be optimized for each patient,” he said. “These patients might be stigmatized as pain medication seeking.… We need to change the perception of this disease and how we as the medical community see these patients, treat these patients, and help them deal with not only the tumor itself, but also all the implications that come along with having the tumor.”

Risk Factors and the Need for Multidisciplinary Management

There are factors that predispose individuals to more severe NF1-PN symptoms. “Plexiform neurofibromas grow a little bit faster in children than in adults, so that definitely is a population that we need to be monitoring closely,” Chow said. “We also know that this is a very heterogeneous condition, so not all patients will have plexiform neurofibromas. It’s incumbent upon the provider to identify the patients, early on, who have a plexiform neurofibroma for follow-up.” Chow added, “This is a condition that screams for a multidisciplinary approach…including, potentially, neurologists, neuro-oncologists, oncologists, and various surgeons [depending on the tumor’s location].” Lobbous emphasized that managing NF1-PN “takes a village…[a] big team to make sure that the patient is well taken care of,” and stressed the critical importance of a seamless transition from pediatric to adult care to avoid complications later in life.

Chow also mentioned the risk of malignant transformation to MPNST. “Overall plexiform neurofibroma tumor burden in a patient is related to the propensity to transform,” he noted. Although whole-body MRIs aren’t currently recommended to identify high-risk patients, he suggested that “as we enter upon an age where we have multiple treatments available…we want to readdress…[the] recommendation for whole-body MRI to identify patients [who] are potentially at a higher risk for transformation to MPNST.” Haumschild emphasized that “if we don’t address this earlier with multidisciplinary care, I can see a high total cost of care, if we don’t get these patients treated. And that’s burdensome for the patient, it’s burdensome for the provider, and really for the payer as well, who’s [treating] these patients.” Lobbous added that “a lot of the tertiary centers now are adopting a whole-brain, whole-body MRI with PET to catch any early malignant transformation,” underscoring that “access to care, transition of care, and also the multidisciplinary [teams]” are essential for achieving the best outcomes for patients.

Current Unmet Needs and Gaps in Care

Haumschild acknowledged the ongoing challenges of managing NF1-PN. “With all those transitions of care, with all those specialists and needing centers of excellence, there are still unmet needs for these patients as they progress along. And that’s something that we’ve got to continue to work toward addressing,” he said.

Mrugala outlined 3 major gaps in care, as follows:

1. Delayed Diagnosis

“Many of these patients are diagnosed late…because general practitioners are typically unaware of NF1 or unaware of its complications or signs and symptoms.”

2. Lack of Access to NF Centers of Excellence

“We’ve done a good job here in the United States with the Children’s Tumor Foundation network…but many of these clinics would see mostly children. And we are lacking centers where adults could be seen.”

3. Lack of Effective Treatments

“Thankfully, this gap is changing.… In the past, the primary treatment…was observation or…surgery, [which] can frequently be challenging, associated with…morbidity.… Frequently the tumors cannot be removed completely, so there’s residual disease and…recurrence. Surgery is not a perfect solution.… We now have options—systemic options that might change the trajectory and hopefully help these patients.”

To address these gaps, Mrugala called for “educating the general providers in recognition of NF1 and its symptoms, and…expanding the network and building more centers of excellence for NF patient care.” He also stressed the need to address secondary symptoms like anxiety, depression, and chronic pain. “We can solve this by No. 1, education; No. 2, creating centers of excellence for NF care that would include psychological management and pain control,” he said.

Mrugala emphasized the importance of a seamless transition from pediatric to adult care: “Many of these patients are diagnosed in childhood…and then they age out and they become adults. And the transition is very clunky. Many of these patients are lost to follow-up.… [We need to] build the bridge between the pediatric world and adult world and [identify] these patients [who] might have greater needs.”

Regarding imaging, Mrugala reiterated that whole-body MRI is a very highly debatable modality but it can be cost-effective and efficient in assessing the overall tumor burden. “This becomes even more important now when the systemic therapies are available and can work…on a variety of tumors.” He added, “It does not need to be expensive.… Scans get done without contrast.… I would like to advocate for this methodology to be available to our patients.”

Chow shared his hope that “as we move forward, we’re going to enter more of a preventive type of a disease care model...where we can hopefully prevent, not sit and wait for symptoms to occur before deciding on treating patients. Preventive medicine is probably ultimately going to prove to be the most cost-effective way of [treating] patients…but in my experience, we’re still in the reactive mode.”

Current and Emerging Treatment Strategies in NF1-PN

Significant progress has been made in NF1-PN management, with recent MEK inhibitor approvals transforming treatment.4,6 Clinical trials demonstrated tumor shrinkage and symptomatic improvement with both selumetinib and mirdametinib, though optimal treatment duration, response durability, and long-term safety remain unclear.4,6,7 Some PNs do not respond to monotherapy, highlighting the need for combination therapies and preventive strategies to further reduce morbidity.4

Because MEK inhibitors can take approximately 8 months to show benefit, surgery may still be needed up front for symptomatic relief or to protect critical structures, as part of a tailored, multidisciplinary approach that aligns with the patient’s QOL goals.6-9 These agents are also associated with disfiguring acneiform rashes and gastrointestinal toxicities, including diarrhea, nausea, and stomatitis, which, though generally mild to moderate, can impact adherence and QOL.8,10 Proactive management, including dietary adjustments, oral care, antibiotics, dose modifications, and psychological support for acneiform rashes, can reduce treatment disruptions and improve outcomes.8,10 A multidisciplinary team is essential to provide comprehensive care and maintain adherence throughout treatment.9

Stakeholder Insights

Evolving Strategies for NF1-PN Treatment

Treatment plans for NF1-PN are highly individualized, depending on tumor growth rate, patient age, and tumor location. “As a part of a multidisciplinary discussion, the first thing we explore is surgery,” Lobbous explained. “Can we do surgery to fix whatever problems the tumor, the plexiform neurofibroma, has created, safely?” However, he noted: “Surgery can be quite challenging. These tumors are diffused. They wrap around major structures, blood vessels, nerves. Surgery rarely is curative, unfortunately. And even [when] the surgeon goes in and…[performs] debulking surgery, what’s left usually grows back.”

Lobbous added, “If surgery is not safe or not feasible, we discuss with the patient systemic treatment with MEK inhibitors. It depends on the rate of the growth, what symptoms this is causing—disfigurement, pain, dysphagia, breathing problems. And we discuss risks and benefit, duration of treatment, expectations as well.” He emphasized that “it takes a village. So we work closely with our dermatologists, with our ophthalmology colleagues, monitoring the patient at treatment initiation, and also as they’re being followed.

“Thanks to the robust studies that have led to the FDA approval, we have guidance of what we counsel patients on, what kind of [adverse] effects we expect, how to monitor in a multidisciplinary approach,” Lobbous said. “We still [are] at the reactive mode, like symptom management. We’re hoping as we learn more about the impact of the therapies on the natural history of the disease, that in the future we will have a more proactive disease-modifying approach to these tumors.”

Haumschild stressed the need to move beyond symptom management, noting, “I see how MEK inhibitors play an important role in that, and even systemic therapy as opposed to constantly going through surgical procedures, having that impact your quality of life, but really taking it more holistically is an important key there.”

MEK Inhibition and Emerging Combination Strategies

Haumschild noted that NF1-associated tumors rely on RAS, MEK, and ALK pathway activation to drive abnormal cell growth. Mrugala expanded, explaining that “NF mutation is a loss of function mutation.… RAS pathway gets hyperactivated, and it happens that MEK is one of the components of the pathway that’s downstream. Blocking MEK results in the inhibition of the Schwann cells in terms of their growth and proliferation.”

He described the first FDA-approved MEK inhibitor for children with inoperable NF1-related plexiform neurofibromas. “Selumetinib truly was a game changer for all of us in the NF field because previously, we did not have a systemic therapy that could influence the tumor growth, especially of the plexiform neurofibromas.” Mrugala shared that “in many of the selumetinib studies…the observation was that 20% or more tumor shrinkage can be observed in up to 70% of patients, so quite robust results.” He also noted improvements in pain and functional outcomes. “These drugs obviously work on the biological level. They lead to more shrinkage, but they also have multiple downstream effects in terms of improvement of patients’ quality of life and other factors.”

Mrugala emphasized that treatment decisions for selumetinib are made through a multidisciplinary process. “It’s typically a case-by-case, multidisciplinary discussion.... It’s not a one-time visit.… It’s typically a process during which we discuss this with the patient, with the family, with the medical team. And as a group, we make a decision when to intervene.”

Chow noted that a practical approach to NF1-PN involves combining surgery with MEK inhibitor therapy. He explained that although MEK inhibitor responses can take 8 to 9 months, immediate intervention through debulking surgery can address tumors threatening critical structures. “In certain situations, you want to do some debulking of the tumor up front, especially if it’s affecting critical structures like your eye or your airway, before embarking on MEK inhibitor [therapy] to prevent the regrowth of these tumors and recurrent symptoms,” he said.

Turning to pharmacological combinations, Chow noted that “to date, I’m not aware of any clinical trials that have been done in people…looking at combination therapies,” although he acknowledged some promising preclinical studies. In animal models, researchers have combined MEK inhibitors with PI3 kinase pathway inhibition and investigated using CDK4/6 inhibitors alongside MEK inhibitors. Chow added that “it’s important to note that inhibition of these pathways is commonly used in other types of pediatric and adult cancer, so we do have pharmacological agents that are FDA approved to target both the PI3 kinase pathway and the CDK4/6. It’s not a big stretch to imagine that if these preclinical studies are replicated and are robust, that might lead to a clinical trial to look at this.”

Chow also discussed the potential of combining BRAF and MEK inhibition, noting that “this combination is contraindicated in other types of tumors with activation of elsewhere along this MAP kinase pathway because…BRAF inhibitors actually cause paradoxical…accelerated growth of tumors when other parts of the pathway are mutated.… So we don’t do that combination in NF1 [and] that has not been tested.” He added that newer-generation BRAF inhibitors, like tovorafenib, have also shown paradoxical tumor growth in preclinical models of NF1-associated low-grade gliomas.

Psychosocial Impacts of NF1-PN

Addressing the psychosocial impacts of NF1-PN, Lobbous emphasized that FDA approval of MEK inhibitors requires considering these broader outcomes. “We treat patients, not pictures,” he noted, explaining that patient-reported outcomes—especially pain and psychosocial impacts—are critical for evaluating treatment effectiveness. Although imaging responses matter, he said, “it’s not the whole goal.… The pain, the psychosocial impact are key parts of how we actually evaluate if the treatment is working.”

He highlighted that as tumors shrink with MEK inhibitor treatment, downstream symptoms can also improve. “If [a neurofibroma is] causing disfigurement and affecting self-image…that also gets better. If it’s causing pain, that gets better. If it’s causing problems with swallowing or breathing, that also gets better.”

Lobbous explained that although children’s hospitals often provide comprehensive support services, adult care settings typically lack social workers, therapists, and educators familiar with NF1. “In the adult world, I think one of the key gaps is access to mental health care and mental health providers who are aware of neurofibromatosis,” Lobbous said.He expressed hope that educational programs and efforts to raise awareness will help address these gaps.

Barriers to Treatment

A major barrier to treatment access, Chow emphasized, is the lack of awareness among both patients and general health care providers: “By far the biggest barrier is access to a provider who knows about these inhibitors. Most patients do not know about these inhibitors…that treatment is available. Most primary care physicians do not know that these treatments are available.” Despite NF1’s prevalence, he noted, “the majority of patients are not followed in clinical centers that have the expertise to recommend these medications or even to prescribe them.”

Chow elaborated: “I get referrals from various different surgeons or geneticists or other specialists in my hospital who are uncomfortable prescribing these medications…because they’re not familiar. They’re not used in any other subspecialty to date, only within the oncology field. And so they’re not familiar with the [adverse] effects of these drugs and what to monitor for.” He stressed that “by far the greatest hurdle to making this class of treatments accessible to patients is that patients are not seen by providers who can actually prescribe them.”

Encouragingly, he added: “The FDA approval is very clear. These medications are approved for children and now adults who have an inoperable plexiform neurofibroma.… This is the only FDA-approved treatment for these patients. So the drug is available. I have never come across a situation in which I’ve had a patient…refused by insurance because of that FDA approval.… It’s a matter of trying to get the word out there to our patient population to seek the right care.”

Mrugala explained that “for decades we didn’t really have a lot of options for these patients. Surgery was the main option with all the limitations…in terms of resectability of the disease, the rate of recurrence, the morbidity associated with surgery. So we learned that surgery is not going to solve the problem of NF1-related plexiform neurofibroma.” Conventional chemotherapy also proved ineffective and caused “a lot of secondary toxicity…specifically myelotoxicity and gastrointestinal toxicity,” whereas radiotherapy “has a very limited option if at all.”

Mrugala emphasized the need for a more deliberate approach. “We really need to be much more intentional in how we approach this disease,” he said. Fortunately, “there are beautiful discoveries in the molecular biology of NF.… Understanding the mutation, what it does, understanding the RAS pathway activation, understanding what to target led us to where we are now with the availability of currently 2 FDA-approved drugs for use.” He expressed hope for future therapies, noting, “We will be learning over time that maybe suppressing or inhibiting the different pathways at the same time would be much more beneficial than 1 pathway at a time.”

Mrugala also highlighted a broader vision: “Can we actually cure NF? And these are the gene therapy approaches…not fixing the tumor, but maybe fixing the alteration that led to the disease to begin with. And seeing if this could have implications not just for the plexiform neurofibromas, but for all the other tumors and stigmata associated with NF1.”

Clinical Practice and Monitoring Guidelines and Future Directions in NF1-PN Care

Clinical practice and monitoring guidelines for NF1-PN, based on expert consensus, emphasize individualized care and multidisciplinary management across the patient’s lifespan, with a focus on proactive surveillance, symptom management, and risk-based monitoring to address NF1’s variable presentation and morbidity.11-14 Recommendations include comprehensive baseline assessments, regular imaging to monitor tumor growth and transformation risk, and multidisciplinary clinics integrating expertise in neurology, oncology, genetics, radiology, and more. Expert consensus also offers guidance on supportive care, dose modifications, and managing adverse events from new treatments, underscoring the critical role of a multidisciplinary team.8 Importantly, a structured health care transition program is essential to bridge the shift from pediatric to often fragmented adult care, ensuring lifelong disease management and optimal quality of life for these patients.15,16

Stakeholder Insights

Considerations for Dosing and Administration of Emerging NF1-PN Therapies

Chow described mirdametinib as “another game changer in our field… [adding] another arrow to our quiver of armamentarium for treating plexiform neurofibroma,” noting that “the overwhelming majority of patients—about 80%—[had] a response, either partial or stable disease…[and] more than three-quarters of those patients [saw] an improvement in their pain.”

He emphasized that, unlike selumetinib, mirdametinib is FDA approved for adult patients. “We were using selumetinib off-label.… Now, we have something that is on label, which definitely makes the approval process… much easier.” Lobbous echoed this. “As a physician who takes care of adults with [NF1], we had no options for a while.… To have an FDA-approved treatment option for adults with [NF1]–associated plexiform neurofibromas, it’s more than just extension.”

Chow also highlighted mirdametinib’s formulation advantage, a dispersible oral tablet, for younger patients who are unable to swallow capsules and patients who have swallowing difficulties due to head and neck PNs, benefiting “a much broader swath of patients who now can benefit from MEK inhibition.” Haumschild added that “having something that fills an unmet need is an important evolution of treatment.”

Lobbous noted that mirdametinib is approved for adults and has a 3-weeks-on, 1-week-off dosing schedule, in contrast to selumetinib’s continuous dosing. Mrugala also said this schedule “may be attractive…to be off the medication for a week to perform, or perhaps go on a trip…and then not think about taking a medication.”

Discussing adverse effects, Lobbous noted that there hadn’t been a study that compared the 2 different medications that were available head-to-head. However, he said that the 2 have similar adverse effect profile with the dermatologic and laboratory abnormalities, including cardiac and ocular abnormalities that are closely monitored.

“First thing is to highlight…now we have another drug. We only had selumetinib; now we have another agent. It’s great for our patients and for our community to have multiple options,” Mrugala said, explaining that mirdametinib “will probably become the first choice” for adult patients “at least until there [are] additional data with selumetinib or there are other agents.” He also noted that decisions around prescribing mirdametinib depend on “provider confidence and comfort with managing [adverse] effects.… We all learn the new drug when it becomes available, and we need to become comfortable with prescribing it.”

Finally, Mrugala stressed that cost and insurance coverage will be critical and expressed hope that “insurance companies will favorably look at these agents given the unmet need and a significant disability and suffering these patients go through. And these drugs can alter the course of the disease.”

Clinical Practice and Monitoring Guidelines for NF1-PN Therapies

Lobbous highlighted the importance of new treatment options beyond repeated surgeries. “The fact that there is a medical treatment option right now—that’s very exciting.” He stressed selecting the right patients and assembling the appropriate team, with monitoring that includes “laboratory imaging and…toxicity monitoring, particularly skin exams, eye exams, and [echocardiogram] for heart function.”

Chow emphasized vigilant monitoring of rare but significant adverse effects, particularly ophthalmologic and cardiac issues. “Those patients…need to be monitored closely, perhaps go on a drug holiday,” he said, noting that the effects usually reverse themselves. He also highlighted dermatologic adverse effects, especially acne in adolescents, urging proactive prevention to maintain adherence.

Lobbous raised the question of treatment duration: “Is it 2 years, which is the duration of the trial? We have yet to see how patients fare long term.”

The Future of NF1-PN Care

Reflecting on the progress in NF1-PN care, Lobbous said there have been leaps in the field, including the recent FDA approval for MEK inhibitor, mirdametinib. “There is a great momentum already.… The future is more promising.” He envisioned using combination therapies and stressed the need for “biomarker-driven care” to identify high-risk patients and tailor treatment.

Chow echoed the optimism, highlighting the availability of 2 FDA- approved drugs with more to come. He urged insurers to cover both selumetinib and mirdametinib, not just as preferred drugs. “We don’t know actually how long patients will respond.… They may eventually become resistant, and this is a lifelong condition.… So we need to have the availability of all the arrows in our quiver to do sequential therapy if needed.”

Mrugala again emphasized priorities for the NF1-PN community: “No. 1, educate.… No. 2, improve access [to] multidisciplinary teams, NF Centers of Excellence, [and] access to the treatments.”

Emphasizing the importance of care continuity, Lobbous said: “When you move to adults, things can get somewhat fragmented.… Transition of care from pediatric to adult…is fundamental…[for] patients [to] continue to do well.”

Mrugala stressed the need to address “secondary symptoms of this condition, including pain control, but also psychological aspects.… We need to make sure that these patients have access to appropriate care.” He concluded, “We need to continue work on understanding the disease and also developing new treatments.” •

References

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