Yan-Song Lin1,2, Xin Zhang1,2, Chen Wang1,2, Yan-Qing Liu1,2, Wen-Min Guan3, Jun Liang4. 1. Department of Nuclear Medicine, State Key Laboratory of Complex Severe and Rare Diseases, Peking Union Medical College (PUMC) Hospital, Beijing, Chinese Academy of Medical Sciences & PUMC, 100730, Beijing, China. 2. Beijing Key Laboratory of Molecular Targeted Diagnosis and Therapy in Nuclear Medicine, 100730, Beijing, China. 3. Department of Radiology, Peking Union Medical College (PUMC) Hospital, Beijing, Chinese Academy of Medical Sciences & PUMC, Beijing, 100730, China. 4. Department of Oncology, Peking University International Hospital, Beijing, 102206, China.
Abstract
CONTEXT: Radioiodine refractory differentiated thyroid cancer (RAIR-DTC) has been a global challenge due to its poor prognosis and limited treatment options. OBJECTIVE: We report here the long-term results of the phase II clinical trial of apatinib, an anti-angiogenic tyrosine kinase inhibitor, for RAIR-DTC. METHODS: This was an open-label, exploratory phase II clinical trial among progressive RAIR-DTC patients. Apatinib treatment was given once daily until disease progression, unmanageable toxicity, withdrawal, or death. The primary end points were objective response rate (ORR) and disease control rate (DCR). Progression-free survival (PFS), overall survival (OS), duration of response, long-term safety, and the association between patients with different tumor genotype (BRAFV600E and TERT promotor mutation) and their PFS rates were also assessed. RESULTS: The ORR was 80%, and the DCR was 95%. The overall median PFS was 18.4 months (95% CI, 9.2-36.8 months) and the median OS was 51.6 months (95% CI, 29.2-not reached [NR]). Patients with BRAFV600E mutation (10 of 18 evaluated) had a longer median PFS compared with patients with BRAF wild-type (NR vs 9.2 months; P = 0.002). The most common adverse events included palmar-plantar erythrodysesthesia syndrome (19/20), proteinuria (18/20), and hypertension (16/20). CONCLUSION: In this long-term evaluation, apatinib displayed sustainable efficacy and tolerable safety profile, warranting it as a promising treatment option for progressive RAIR-DTC.
CONTEXT: Radioiodine refractory differentiated thyroid cancer (RAIR-DTC) has been a global challenge due to its poor prognosis and limited treatment options. OBJECTIVE: We report here the long-term results of the phase II clinical trial of apatinib, an anti-angiogenic tyrosine kinase inhibitor, for RAIR-DTC. METHODS: This was an open-label, exploratory phase II clinical trial among progressive RAIR-DTC patients. Apatinib treatment was given once daily until disease progression, unmanageable toxicity, withdrawal, or death. The primary end points were objective response rate (ORR) and disease control rate (DCR). Progression-free survival (PFS), overall survival (OS), duration of response, long-term safety, and the association between patients with different tumor genotype (BRAFV600E and TERT promotor mutation) and their PFS rates were also assessed. RESULTS: The ORR was 80%, and the DCR was 95%. The overall median PFS was 18.4 months (95% CI, 9.2-36.8 months) and the median OS was 51.6 months (95% CI, 29.2-not reached [NR]). Patients with BRAFV600E mutation (10 of 18 evaluated) had a longer median PFS compared with patients with BRAF wild-type (NR vs 9.2 months; P = 0.002). The most common adverse events included palmar-plantar erythrodysesthesia syndrome (19/20), proteinuria (18/20), and hypertension (16/20). CONCLUSION: In this long-term evaluation, apatinib displayed sustainable efficacy and tolerable safety profile, warranting it as a promising treatment option for progressive RAIR-DTC.