帕比司他治疗胶质瘤效果好吗？

Panobinostat is an oral pan-HDAC inhibitor that shows strong activity in a variety of malignant tumors. It is the most powerful HDAC inhibitor currently known and has been approved by the FDA for relapsed and refractory multiple myeloma. For glioma, studies have shown that panobinostat can produce cytotoxic effects on some glioma cells and promote the apoptosis of these cells. Research also shows that it can inhibit the proliferation of stem cell-like cells, which are some tumor cells that are highly susceptible to drug resistance. So, is panobinostat effective in treating glioma?

Research Methods: We report the use of panobinostat in adults treated with H2020 K2021M mutated DMG at Mirai Medical International (Rochester) from March 2018 to March 27, 2016, and survival rates were calculated using the Kaplan-Meier method [1].

Research results: Four patients with H3 K27M mutated gliomas were treated with panobinostat. The median age of patients was 40 years (range, 22-62 years), and 2 cases were female. Tumor locations in all patients were midline, spinal cord (n = 2), brainstem (n = 1), and thalamus (n = 1). All tumors were IDH1/IDH2 wild-type. Three patients received radiotherapy followed by adjuvant panobinostat. Except for 3 patients who were concurrently taking dexamethasone, all patients were not treated with other medications before or during panobinostat treatment. No patients experienced grade 2 or higher (per CTCAE grade) adverse reactions. Median overall survival was 42 months, median progression-free survival was 19 months, and 2 patients were alive at last follow-up (spinal cord tumor and radiotherapy). The condition of 2 patients was stable and 1 patient had partial response.

Conclusion

This is the first report on the clinical results of panobinostat in the treatment of H3 K27M mutated DMG in adults. We found that it was well tolerated at the dosage regimen we described, with no serious episodes throughout the study period.

References

[1]Neth BJ, Balakrishnan SN, Carabenciov ID, Uhm JH, Daniels DJ, Kizilbash SH, Ruff MW. Panobinostat in adults with H3 K27M-mutant diffuse midline glioma: a single-center experience. J Neurooncol. 2022 Mar;157(1):91-100. doi: 10.1007/s11060-022-03950-8. Epub 2022 Jan 25. PMID: 35076860.

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