Panelists discuss the unmet needs in NF1-associated plexiform neurofibromas, highlighting gaps in effective treatment, pain management, psychosocial support, and prevention of malignant transformation, while emphasizing the importance of early intervention, multidisciplinary care, and ongoing research into targeted therapies and improved surveillance protocols.
Summary for Physicians
Unmet Needs in NF1-PN
Patients with neurofibromatosis type 1–associated plexiform neurofibromas (NF1-PN) face significant gaps in care, particularly related to effective treatment options for tumor management, especially for those with large or symptomatic tumors. Pain management, psychosocial support, and malignant transformation prevention remain major challenges. Limited treatment options exist for slowing tumor growth or alleviating complications, and there is a need for more personalized care.
Addressing Gaps in Care
To improve outcomes and quality of life, strategies should focus on early intervention, improving access to multidisciplinary care, advancing targeted therapies, and enhancing psychosocial support. Ongoing research into better treatments and more effective pain management and surveillance protocols is critical for addressing these unmet needs.