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Sorafenib in metastatic radioactive iodine-refractory differentiated thyroid cancer: A pilot study
Yang Luo1, Yuankai Shi1, Puyuan Xing1
1Department of Medical Oncology, Cancer Institute/Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing 100021, P.R. China ; Beijing Key Laboratory of Clinical Study on Anticancer Molecular Target Drugs, Beijing 100021, P.R. China.
Abstract:
The incidence and mortality of thyroid cancer are on the increase worldwide and the treatment options for progressive, radioactive iodine (RAI)-refractory metastatic differentiated thyroid cancer (DTC) patients are currently limited. Sorafenib is a multikinase inhibitor that targets several molecular signals, which are believed to be involved in the pathogenesis of DTC. In this study, we reported our experience with the off-label use of sorafenib in Chinese cancer patients. A total of 8 patients (7 with papillary and 1 with follicular thyroid cancer) were recruited in this study. The partial response (PR) rate was 50.0% and 5 of the 8 patients (62.5%) achieved a durable response. The median progression-free survival (PFS) and overall survival (OS) were 40.1 and 55.0 weeks, respectively. Lung metastases were more sensitive to sorafenib compared to lymph nodes. The tumor marker response was not in accordance with the radiological response, although patients with tumor marker complete response (CR) exhibited a longer PFS and OS compared to those without a CR. Common adverse events (AEs) included palmar-plantar erythrodysesthesia, hypertension, diarrhea, weight loss and alopecia. Grade 4 AEs comprised hypocalcemia (1 patient) and elevated amylase levels (1 patient). A dose reduction was required in 62.5% of the patients. In conclusion, sorafenib exhibited a clinically relevant antitumor activity in patients with progressive metastatic RAI-refractory DTC, although the majority of the patients required a dose reduction due to intolerable toxicity.
Insights
Sorafenib shows promise for treating advanced radioactive iodine-refractory differentiated thyroid cancer (DTC). Despite side effects requiring dose reduction, it offers significant tumor response and survival benefits for these patients.
Area of Science:
- Oncology
- Pharmacology
Background:
- Thyroid cancer incidence and mortality are rising globally.
- Limited treatment options exist for progressive, radioactive iodine (RAI)-refractory metastatic differentiated thyroid cancer (DTC).
Purpose of the Study:
- To evaluate the efficacy and safety of off-label sorafenib use in Chinese patients with progressive metastatic RAI-refractory DTC.
Main Methods:
- Retrospective study of 8 DTC patients (7 papillary, 1 follicular) treated with sorafenib.
- Assessed partial response (PR) rate, durable response, progression-free survival (PFS), and overall survival (OS).
- Monitored radiological response, tumor marker response, and adverse events (AEs).
Main Results:
- Partial response (PR) rate was 50.0%, with 62.5% achieving durable response.
- Median PFS was 40.1 weeks and median OS was 55.0 weeks.
- Lung metastases were more responsive than lymph nodes; common AEs included palmar-plantar erythrodysesthesia and hypertension, necessitating dose reduction in 62.5% of patients.
Conclusions:
- Sorafenib demonstrates clinically relevant antitumor activity in progressive metastatic RAI-refractory DTC.
- Majority of patients required dose reduction due to toxicity, highlighting the need for careful management.
- Tumor marker response correlated with improved survival outcomes, suggesting its utility as a prognostic indicator.
