Progressive locally advanced or metastatic radioiodine-refractory differentiated thyroid cancer (RAI-R DTC) patients treated with donafenib: A real-world study.

Abstract

e18018 Background: Donafenib was approved for the treatment of RAI-R DTC patients by National Medical Products Administration (NMPA) in 2022, The main objectives of this study were to assess real-world clinical effectiveness and safety in RAI-R DTC patients treated with donafenib at Fudan University Shanghai Cancer Center. Methods: An observational patient chart review was conducted. The study cohort comprised RAI-R DTC patients who were treated with donafenib monotherapy (200 mg, bid, up to 24 months), between January 1, 2023 and December 31, 2025. Data were collected from patient electronic health records by the prescribing physicians and were de-identified before filling in an electronic case report form. The primary endpoint was objective response rate (ORR). The secondary endpoints included progression-free survival (PFS), overall survival (OS) and disease control rate (DCR). Time to event endpoints were assessed using Kaplan-Meier methods. Results: Of the 22 patients reviewed, 63.6% were female and the median age of the patients was 61.5 years at donafenib initiation; 90.9% of patients had an ECOG PS of 1, 81.8% of patients were in stage IVB (other 4 patients were in stageⅡbecause of age under 50) , 40.9% of patients had follicular DTC and 90.9% of patients had lung metastases. The median follow-up time was 16.9 months (95%CI:12.7-21.1), the ORR and the DCR by RECIST 1.1 were 22.7% (5/22) and 86.4% (19/22), respectively. The median PFS and median OS were not reached, 1-year PFS rate was 85.6% (95% CI: 70.5-1）and 1-year survival rate was 100%. Median serum Tg dropped from baseline rapidly in the first 3 weeks; early in cycle two, biochemical response was observed in 84.6% of patients. ≥ Grade 3 treatment-related adverse events (TRAEs) were observed in 22.7% of patients, the most frequent TRAE of all grades was hand foot skin reaction (15/22, 68.2%). No treatment-related deaths were observed . Conclusions: The current study enriched the efficacy and safety data of donafenib in the treatment of RAI-R DTC patients in real-world clinical practice in china, which enrolled more patients with ECOG PS ≥ 1 and follicular DTC compared with the phase III DIRECTION study. In addition, the efficacy and safety results are consistent with DIRECTION study.