Use of Trametinib in Children and Young Adults With Progressive Low-grade Glioma and Glioneuronal Tumors. Issue 4 (24th May 2023)

• Record Type: Journal Article
• Authors: Hanzlik, Emily; Archambault, Bridget; El-Dairi, Mays; Schroeder, Kristin; Patel, Mallika P.; Lipp, Eric S.; Peters, Katherine B.; Ashley, David M.; Landi, Daniel
• Is Part Of: Journal of pediatric hematology/oncology. Volume 45:Issue 4(2023)
• Page Start: e464
• Page End: e470
• Publication Date: 2023-05-24
• Subjects: low-grade glioma -- pediatrics -- therapy -- trametinib -- outcomes; Pediatric hematology -- Periodicals; Tumors in children -- Periodicals; 618.9215
• DOI: 10.1097/MPH.0000000000002598
• Languages: English
• ISSNs: 1077-4114

Abstract

Abstract : Low-grade gliomas/glioneuronal tumors comprise one-third of all pediatric-type CNS tumors. These tumors are generally caused by activating mutations in the mitogen-activated protein kinase (MAPK) pathway. Targeted drugs, such as trametinib, have shown promise in other cancers and are being utilized in low-grade gliomas. A retrospective chart review was conducted to evaluate radiographic response, visual outcomes, tolerability, and durability of response in progressive circumscribed low-grade gliomas treated with trametinib. Eleven patients were treated with trametinib. The best radiographic response was 2/11 partial response, 3/11 minor response, 3/11 stable disease, and 3/13 progressive disease. In the patients with partial or minor response, the best response was seen after longer durations of therapy; 4 of 5 best responses occurred after at least 9 months of therapy with a median of 21 months. Patients with optic pathway tumors showed at least stable vision throughout treatment, with 3 having improved vision on treatment. Trametinib is effective and well-tolerated in patients with progressive low-grade glioma. Best responses were seen after a longer duration of therapy in those with a positive response. Patients with optic pathway lesions showed stable to improved vision while on treatment.