Personalizing Treatment in Relapsed/Refractory pLGG: Expert Insights

When managing progressive pLGGs, especially in cases where complete surgical resection isn’t possible, it is important to set expectations early. Patients who have residual tumor after surgery face a higher likelihood of disease progression, making it probable that multiple treatment approaches will be needed over time. Sharing this information with families up front helps prepare them for a treatment journey that may involve several lines of therapy, reducing the emotional impact if the tumor recurs or progresses.

Treatment decisions for refractory or progressive disease require a personalized approach that goes beyond molecular tumor analysis. Factors such as the child’s age, growth potential, and adverse effect profiles of available therapies must be carefully weighed. Equally important is considering the family’s situation, including treatment burden, travel distance, and access to care. The availability of targeted therapies that can be administered with fewer clinic visits or through telehealth can significantly improve quality of life for families facing long distances or logistical challenges.

One potential adverse effect of targeted therapy like tovorafenib is hemorrhage, which has been reported in clinical trials—mostly intratumoral or minor bleeding events such as epistaxis. Although serious bleeds are rare, it is critical to educate families about these risks alongside other adverse effects like growth suppression or cardiac- and eye-related toxicities seen with some targeted agents. Families value transparency and knowledge about treatment options and potential complications, which empowers them to participate actively in decision-making and feel confident in the treatment path chosen.