Abstract
Context
Radioiodine refractory differentiated thyroid cancer (RAIR-DTC) has been a global challenge due to its poor prognosis and limited treatment options.
Objective
To report the long-term results of the phase II clinical trial of apatinib, an anti-angiogenic tyrosine kinase inhibitor, for RAIR-DTC.
Design, Setting, Participants
Open-label, exploratory phase II clinical trial among progressive RAIR-DTC patients.
Intervention
Apatinib treatment once daily until disease progression, unmanageable toxicity, withdrawal, or death.
Main Outcome Measures
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 (BRAF V600E and TERT promotor mutation) and their PFS were also assessed.
Results
The ORR was 80%, and the DCR was 95%. The overall median PFS was 18.4 months (95% confidence interval [CI], 9.2-36.8 months) and median OS was 51.6 months (95%CI, 29.2-not reached [NR]). Patients with BRAF V600E 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 erythrodysaesthesia syndrome (19/20), proteinuria (18/20) and hypertension (16/20).
Conclusions
In this long-term evaluation, apatinib displayed sustainable efficacy and tolerable safety profile, warranting it as a promising treatment option for progressive RAIR-DTC.
Response to [90Yttrium-DOTA]-TOC Treatment is Associated with Long-term Survival Benefit in Metastasized Medullary Thyroid Cancer: A Phase II Clinical Trial