Dr Moertel on the Importance of Managing MEK Inhibitor–Associated Toxicites in NF1-Associated PN

"It’s up to us practitioners to provide good, anticipatory guidance regarding symptom management…and to realize what those AEs are and how to treat them effectively. If we can do our best to ameliorate these toxicities and enable patients to stay on [treatment] for a long time, we’ve done a good job.

Christopher L. Moertel, MD, a pediatric neuro-oncologist, professor, and director the Pediatric Neuro-Oncology Fellowship Program in the Division of Pediatric Hematology/Oncology, medical director of the Pediatric Neuro-Oncology and Neurofibromatosis Programs, co-medical director of the Katie Hageboeck Children’s Cancer Research Fund Clinic, clinical neuro-oncology leader of the Brain Tumor Program, and The Kenneth and Betty Jayne Dahlberg Profssor at the University of Minnesota School of Medicine, discussed the importance of informed, careful, and effective management of MEK inhibitor–associated toxicities for patients with neurofibromatosis type 1 (NF1)–associated plexiform neurofibromas (PN).

The introduction of MEK inhibitors as a key therapeutic option for NF1-associated PN has necessitated a greater focus on managing associated toxicities to ensure long-term treatment adherence, Moertel began. Patients receiving MEK inhibitors are susceptible to a range of adverse effects (AEs), including ocular, cardiac, dermatologic, and gastrointestinal (GI) toxicities, which can significantly affect quality of life and treatment continuation, Moertel stated. He noted that these agents require extended administration—often for a minimum of 18 months—making effective toxicity management critical.

Therefore, oncologists should adopt anticipatory guidance strategies to educate patients on potential AEs and provide proactive toxicity management when they receive a MEK inhibitor, Moertel recommended. For example, dermatologic toxicities, such as rashes and skin irritation, can often be mitigated with antibiotics, bleach baths, and emollient lotions, he shared. GI toxicity, which may include nausea and diarrhea, can be managed through dietary adjustments and supportive care measures, Moertel added. Recognizing these toxicities early and implementing targeted interventions allows patients to tolerate treatment more effectively, reducing the likelihood of treatment discontinuation, Moertel emphasized.

Despite the efficacy of MEK inhibitors in slowing tumor growth, these agents do not eliminate NF1-associated PN, Moertel stated. There remains significant opportunity for research and innovation in this area, particularly in developing strategies to improve treatment tolerability and enhance therapeutic outcomes, he said. By refining toxicity management protocols and expanding treatment options, oncologists can better support patients in maximizing the benefits of MEK inhibitors and minimizing the effect of these agents on daily life, Moertel concluded.