Chordoma is a rare type of cancerous tumor occurring anywhere along the spine. Since the disease is rare, no standard treatment exists. Apatinib, a tyrosine kinase inhibitor, has shown promising safety and efficacy in previous studies. This study aims to examine the safety and antitumor activity of apatinib in patients with advanced chordoma.

This phase 2, the single-arm study included a total of 27 patients aged 18-75 years with histologically confirmed advanced chordoma. The patients were assigned to receive oral 500 mg apatinib once daily until disease progression or unacceptable toxicity. The primary outcomes of the study were progression-free survival and objective response rate (according to RECIST and Choi criteria).

During a median follow-up of 14.2 months, 1 patient (3.7%) achieved an objective response rate, according to RECIST and 7 (25.9%) achieved an objective response rate according to Choi criteria. The median progression-free survival was 18 months, according to both RECIST and Choi criteria. Commonly occurring treatment-related grade 3 adverse events were hypertension (24%) and proteinuria (7%). No treatment-related grade 4 events or deaths were observed.

The research concluded that apatinib showed promising activity with a good safety profile in patients with advanced chordoma. However, the sample size was small, and further studies on a larger scale are required.