| Autor: |
Daniel C Bowers, Veena Rajaram, Matthias A Karajannis, Sharon L Gardner, Jack Meng-Fen Su, Patricia Baxter, Sonia Partap, Laura J Klesse |
| Rok vydání: |
2023 |
| Předmět: |
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| Zdroj: |
Neuro-Oncology Advances. 5 |
| ISSN: |
2632-2498 |
| DOI: |
10.1093/noajnl/vdad011 |
| Popis: |
BackgroundPreclinical studies have suggested that mTOR pathway signaling may be a potential therapeutic target for childhood ependymoma.MethodsA phase II clinical trial (ClinicalTrials.gov identifier: NCT02155920) of single-agent everolimus was performed to test the hypothesis that mTOR pathway inhibition would result in tumor responses for children with recurrent and/or progressive ependymomas.ResultsEleven subjects [sex: 4 females (36.4%); median age: 8 years (range: 2-15 years); race: 9 white; prior therapies: median 6 (range: 3-9)] were enrolled on the study. Ten primary tumors were located in the posterior fossa and one primary tumor was located in the spinal cord. Eight of 9 tumors were PF-A subtype epenydmomas. All subjects were treated with oral everolimus 4.5 mg/m2/day (each cycle = 28 days) that was titrated to achieve serum trough levels of 5-15 ng/ml. Overall, everolimus was well tolerated; except for a single event of grade 3 pneumonia, all adverse events were grade 1-2. No objective tumor responses were observed. Participating subjects experienced tumor progression and discontinued therapy after a median of 2 cycles of therapy (1 cycle = 2; 2 cycles = 6; 3, 4, and 8 cycles = 1 each).ConclusionsEverolimus does not appear to have activity for children with recurrent or progressive PF-A ependymoma. |
| Databáze: |
OpenAIRE |
| Externí odkaz: |
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