There is literature supporting this drug; effectiveness as a single agent. The efficacy of oral panobinostat has been assessed in multiple myeloma, both as a single agent and as part of combination therapy with other agents having known activity in myeloma (. Single-agent panobinostat activity was studied in a Phase Ia/II study, which included patients with various hematologic malignancies, such as multiple myeloma. Panobinostat was administered once a day, three times per week, every week or every other week in a 28-day cycle. Based on the dose escalation phase, 40 and 60 mg doses were recommended for the expansion phase for weekly and biweekly administration, respectively. Modest activity was noted in six patients with multiple myeloma, with one patient achieving a partial response (PR). The most significant grade 3–4 adverse events noted in the study were thrombocytopenia (41.5%), fatigue (21%), and neutropenia (21%).24 Another single-agent study was a Phase II study of patients with heavily pretreated relapsed/refractory (RR) multiple myeloma. The median number of prior lines of therapy was five. In this trial, 20 mg of panobinostat was given by mouth three times per week, every week in a 21-day cycle. Again, single-agent activity of panobinostat was modest, with one patient (2.6%) achieving PR and one other patient (2.6%) developing minimal response to therapy. However, these responses were durable and lasted 19 and 28 months, respectively. Similar to the previous study, hematologic toxicities were the most common grade 3–4 adverse events (neutropenia 21.1%, thrombocytopenia 18.4%).25
E.Q.Lee et al, Phase II Study of Panobinostat in Combination With Bevacizumab for Recurrent Glioblastoma and Anaplastic Glioma Neuro Oncol . 2015 Jun;17(6):862-7
Lee at al conducted a multicenter phase II trial of panobinostat combined with bevacizumab in patients with recurrent high-grade glioma. Although it was reasonably well tolerated, the addition of panobinostat to bevacizumab did not significantly improve PFS6 compared with historical controls of bevacizumab monotherapy in either cohort.
DeAngelo DJ, Spencer A, Bhalla KN, et al. Phase Ia/II, two-arm, open-label, dose-escalation study of oral panobinostat administered via two dosing schedules in patients with advanced hematologic malignancies. Leukemia. 2013;27(8):1628–1636. [PubMed]
Wolf JL, Siegel D, Goldschmidt H, et al. Phase II trial of the pan-deacetylase inhibitor panobinostat as a single agent in advanced relapsed/refractory multiple myeloma. Leuk Lymphoma. 2012;53(9):1820–1823.
Farydak, Prscribing Information 2018.
Bailey H, Stenehjem DD, Sharma S. Panobinostat for the treatment of multiple myeloma: the evidence to date. Journal of Blood Medicine. 2015;6:269-276. doi:10.2147/JBM.S69140.