Chapter 7 148 Real-world efficacy and safety of lenvatinib: data from a compassionate use in the treatment of radioactive iodine-refractory differentiated thyroid cancer patients in Italy Setup From November 2014 to September 2016, the lenvatinib expanded access program was open for patients in Italy to provide access to lenvatinib prior to the commercialization of the drug in Italy. 16 Italian sites participated in retrospectively reviewing the data of these patients. The study was approved by local ethics committees and was funded by an unrestricted grant from the pharmaceutical company and sponsor Eisai. Patients The expanded access program was open to patients with radioactive iodine resistant differentiated thyroid cancer. 94 patients were enrolled and included in the analysis population. Patients had a median age of 60 years (range, 23-82). Sixty-four percent of the patients received a previous systemic treatment and fifteen percent had an ECOG performance status (PS) of 2. Intervention Patients received lenvatinib, a tyrosine kinase inhibitor. Lenvatinib was given at an initial dose of 24 mg /day for 64 patients, 20 mg/day for ten patients and 14 mg/ day for 11 patients. Five patients started at 10 mg/day or less. Dosing could be reduced upon physician discretion. Treatment with lenvatinib continued until disease progression, a lack of therapeutic effect, or manifestation of unacceptable side-effects). Outcome The response rate (RR), progression free survival (PFS), overall survival (OS) and toxicity data during a period of 36 months were retrospectively collected. Overall, median PFS was 10.8 months (95% confidence interval (CI) 7.7-12). The OS was 23.8 months (95% CI, 19.7-25.0). All 82 patients that were evaluable for toxicity presented at least one adverse event (AE), of which 21 patients experienced at least one AE of grade 3 or higher (22.3%). The most common AEs included fatigue (13.6%) and hypertension (11.6%). Comparison with other data When comparing the expanded access program with the pivotal trial named SELECT, the authors conclude that: ‘This retrospective observational study confirms the efficacy of lenvatinib in RAIrefractory, progressive, unselected DTC patients in a real-world practice in Italy. Global results, however, are inferior to those reported in the SELECT trial, being ORR 36% versus 64% and median PFS 10.8 months versus 18 months.’ The authors explain the inferior expanded access program results by the
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