2024
ONC201 (Dordaviprone) in Recurrent H3 K27M–Mutant Diffuse Midline Glioma
Arrillaga-Romany I, Gardner S, Odia Y, Aguilera D, Allen J, Batchelor T, Butowski N, Chen C, Cloughesy T, Cluster A, de Groot J, Dixit K, Graber J, Haggiagi A, Harrison R, Kheradpour A, Kilburn L, Kurz S, Lu G, MacDonald T, Mehta M, Melemed A, Nghiemphu P, Ramage S, Shonka N, Sumrall A, Tarapore R, Taylor L, Umemura Y, Wen P. ONC201 (Dordaviprone) in Recurrent H3 K27M–Mutant Diffuse Midline Glioma. Journal Of Clinical Oncology 2024, 42: 1542-1552. PMID: 38335473, PMCID: PMC11095894, DOI: 10.1200/jco.23.01134.Peer-Reviewed Original ResearchConceptsH3 K27M-mutant diffuse midline gliomaDiffuse midline gliomaDuration of responseTime to responseHigh-grade gliomasLow-grade gliomasMidline gliomaMedian duration of responseMedian time to responseTreatment-emergent adverse eventsEnd pointsBlinded independent central reviewCorticosteroid dose reductionIndependent central reviewSecondary end pointsClinically meaningful efficacyRadiographic end pointsSpinal tumorsDose reductionDismal prognosisCentral reviewPerformance scoresCorticosteroid responseResponse assessmentAdverse events
2020
Clinical experience of ONC201 in patients with recurrent H3 K27M-mutant spinal cord glioma.
Kurz S, Tarapore R, Odia Y, Butowski N, Koschmann C, Aguilera D, MacDonald T, Lu G, Allen J, Oster W, Mehta M, Chi A, Wen P. Clinical experience of ONC201 in patients with recurrent H3 K27M-mutant spinal cord glioma. Journal Of Clinical Oncology 2020, 38: 2563-2563. DOI: 10.1200/jco.2020.38.15_suppl.2563.Peer-Reviewed Original ResearchSingle agent groupSpinal cord tumorsCombination groupCord tumorsPediatric patientsSpinal cordH3 K27M mutationMedian survival time rangesMedian follow-up timePhase I clinical trialPhase II clinical trialK27M mutationSurvival time rangesClinical experienceSpinal cord gliomasFollow-up timeHigh-grade gliomasII clinical trialsDose reductionDRD2 antagonistsMedian ageProlonged survivalAgent groupDiffuse gliomasUnfavorable prognosis