PDCT-08. Trametinib and Dabrafenib for Refractory/Inoperable Pediatric Low Grade Gliomas

Kondyli, Maria; Ellezam, Benjamin; Saint‐Martin, Christine; Farmer, Jean‐Pierre; Crevier, Louis; Perreault, Sébastien; Jabado, Nada

doi:10.1093/neuonc/nox168.752

Cited by 3 publications

(1 citation statement)

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“…MAPK-targeting combination therapies are also being explored. Kondyli et al reported that among 6 patients with refractory V600E-mutated pLGG treated with a combination of trametinib and dabrafenib, 4 patients experienced PR, 1 SD and 1 progressive disease (PD)(48). A phase 2 industry-sponsored trial evaluating this combination is currently underway in children with both high and low-grade glioma with BRAF V600 mutations (NCT02684058).…”

Section: Targeted Treatments For Pediatric Lggmentioning

confidence: 99%

Management of pediatric low-grade glioma

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Bandopadhayay

Haas‐Kogan

et al. 2019

Current Opinion in Pediatrics

108

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Purpose of review: Pediatric low-grade gliomas have been treated with similar therapies for the last 30 years. Recent biological insights have allowed a new generation of targeted therapies to be developed for these diverse tumors. At the same time, technological advances may redefine the late toxicities associated with radiation therapy. Understanding recent developments in pediatric low-grade glioma therapy is essential to the management of these common pediatric tumors. Recent findings: It is now well understood that aberrations of the MAPK pathway are key to oncogenesis in low-grade gliomas. This understanding, along with the development of available targeted agents, have heralded a new era of understanding and treatment for these patients. Promising, sustained responses are now being seen in early phase trials among patients with multiply recurrent/progressive disease. Also, newer and highly conformal radiation approaches such as proton beam radiotherapy maintain efficacy of radiation but limit radiation-associated toxicities. Summary: Novel therapies offer the potential for tumor control with greatly reduced toxicities. However, late effects of these therapies are just now being explored. Improved radiation approaches and targeted agents have the potential to redefine traditional therapy for pediatric low-grade glioma.

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Section: Targeted Treatments For Pediatric Lggmentioning

confidence: 99%

Management of pediatric low-grade glioma

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Bandopadhayay

Haas‐Kogan

et al. 2019

Current Opinion in Pediatrics

108

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Integrated response analysis of pediatric low-grade gliomas during and after targeted therapy treatment

Tsai

Choi

Ouaalam

et al. 2022

Neuro-Oncology Advances

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Background Pediatric low grade gliomas (pLGGs) are the most common central nervous system tumor in children, characterized by RAS/MAPK pathway driver alterations. Genomic advances have facilitated use of molecular targeted therapies, however their long-term impact on tumor behavior remains critically unanswered. Methods We performed an IRB-approved, retrospective chart and imaging review of pLGGs treated with off-label targeted therapy at Dana-Farber/Boston Children’s from 2010 to 2020. Response analysis was performed for BRAFV600E and BRAF fusion/duplication driven pLGG subsets. Results Fifty-five patients were identified (dabrafenib n = 15, everolimus n = 26, trametinib n = 11, vemurafenib n = 3). Median duration of targeted therapy was 9.48 months (0.12 – 58.44). The 1-year, 3-year, and 5-year EFS from targeted therapy initiation were 62.1%, 38.2%, and 31.8%, respectively. Mean volumetric change for BRAFV600E mutated pLGG on BRAF inhibitors was -54.11%; median time to best volumetric response was 8.28 months with 9 of 12 (75%) objective RAPNO response . Median time to largest volume post-treatment was 2.86 months (+13.49%); mean volume by last follow-up was -14.02%. Mean volumetric change for BRAF fusion/duplication pLGG on trametinib was +7.34%; median time to best volumetric response was 6.71 months with 3 of 7 (43%) objective RAPNO response . Median time to largest volume post-treatment was 2.38 months (+71.86%); mean volume by last follow up was +39.41%. Conclusions Our integrated analysis suggests variability in response by pLGG molecular subgroup and targeted therapy, as well as the transience of some tumor growth following targeted therapy cessation.

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Nystagmus in the Diagnosis of Russell Diencephalic Syndrome

Tuohy¹,

Robertson²,

Rivas-Rodriguez³

et al. 2019

J Pediatr Ophthalmol Strabismus

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Russell diencephalic syndrome is a condition in which infants become emaciated in the setting of a decreased or normal caloric intake as the result of a hypothalamic astrocytoma. The diagnosis may be delayed if providers initially attribute the symptoms to a behavioral disorder. The detection of nystagmus, which is present in many patients, may be a critical diagnostic clue. The authors describe two patients in whom the discovery of nystagmus months after the onset of emaciation led to the diagnosis of Russell diencephalic syndrome. [ J Pediatr Ophthalmol Strabismus . 2019;56:e79–e83.]

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PDCT-08. Trametinib and Dabrafenib for Refractory/Inoperable Pediatric Low Grade Gliomas

Cited by 3 publications

References 0 publications

Management of pediatric low-grade glioma

Management of pediatric low-grade glioma

Integrated response analysis of pediatric low-grade gliomas during and after targeted therapy treatment

Nystagmus in the Diagnosis of Russell Diencephalic Syndrome

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