Investigating Apatinib for Pretreated Advanced Penile Squamous Cell Carcinoma
A phase II trial evaluates the activity and safety of a VEGF receptor inhibitor in patients with limited remaining treatment options.
Key takeaways
- A phase II trial evaluated oral apatinib monotherapy in 23 patients with pretreated advanced penile squamous cell carcinoma.
- The study reported an investigator-assessed overall response rate of 22%, with five patients achieving a partial response.
- The median progression-free survival was 3.8 months, and the median overall survival was 7.2 months.
- Treatment-related adverse events of grade 3 or worse occurred in 30% of patients, with hand-foot syndrome, hypertension, and increased serum ALT being most common.
Researchers at a tertiary cancer center in Shanghai, China, have evaluated the activity and safety of the VEGF receptor inhibitor apatinib as a single-agent treatment for patients with advanced squamous cell carcinoma of the penis. The investigator-initiated, single-arm phase II trial focused on a patient population with scarce treatment options, specifically those whose disease had progressed after receiving one or more kinds of systemic treatment.
The trial, registered with the University hospital Medical Information Network Clinical Trials Registry (UMIN000021849), enrolled 23 patients between 2016 and 2023. All enrolled participants received at least one dose of the study drug, which was prescribed as an oral dose of 500 mg daily, with the option of dose reduction to manage adverse events. Eligible patients had histologically confirmed penile squamous cell carcinoma, were not candidates for surgery with curative intent, had measurable disease, and had an Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2.
What Happened
All 23 enrolled patients received at least one dose of apatinib. Within this cohort, 16 patients had an ECOG performance status of 2, and six patients had human papillomavirus-positive tumors. The primary endpoint of the trial was the investigator-assessed overall response rate (ORR) in patients who received at least one dose of the drug. Five patients achieved a partial response, resulting in an investigator-assessed overall response rate of 22% (95% CI: 10%-42%). The trial recorded a median progression-free survival of 3.8 months and a median overall survival of 7.2 months.
What The Evidence Shows
The investigator-initiated, single-arm phase II trial assessed oral apatinib (500 mg daily) in 23 patients at a single tertiary cancer center in Shanghai, China. The results showed limited efficacy, with an ORR of 22% (five partial responses) alongside a median progression-free survival of 3.8 months and a median overall survival of 7.2 months. Treatment-related adverse events of grade 3 or worse occurred in seven patients (30%), with the most common being hand-foot syndrome (13%), hypertension (9%), and an increase in serum alanine aminotransferase (ALT) (9%). The study findings were published in The Oncologist in September 2026.
What We Don't Know Yet
The study was designed as a single-arm trial with a small sample size of 23 patients enrolled over a seven-year period (2016 to 2023) at a single clinical site, which limits the ability to compare outcomes directly to a control group.
What Comes Next
The published study does not report any specific future plans, subsequent trials, or next steps scheduled by the investigators or sponsors. Unanswered questions remain regarding how these findings might influence alternative treatment combinations or the selection of patient subgroups in future research.
What This Means
The researchers concluded that apatinib monotherapy showed limited efficacy in patients with pretreated advanced penile cancer, but demonstrated a manageable safety profile in this clinical setting.
Original Source
PubMed (NCBI E-utilities): https://pubmed.ncbi.nlm.nih.gov/42704681/
Questions readers ask
- What is apatinib and how was it evaluated for penile cancer?
- Apatinib is an oral VEGF receptor inhibitor. It was evaluated in a single-arm phase II trial involving 23 patients with advanced penile squamous cell carcinoma whose disease had progressed after one or more systemic treatments.
- What were the efficacy results of apatinib in this clinical trial?
- The trial showed an overall response rate of 22%, with five patients achieving a partial response. The median progression-free survival was 3.8 months, and the median overall survival was 7.2 months.
- What side effects were observed in the apatinib study?
- Grade 3 or worse treatment-related adverse events occurred in 30% of the 23 patients. The most common high-grade side effects were hand-foot syndrome (13%), hypertension (9%), and an increase in serum alanine aminotransferase (ALT) (9%).
What this means
The researchers concluded that apatinib monotherapy showed limited efficacy in patients with pretreated advanced penile cancer, but demonstrated a manageable safety profile in this clinical setting.
Limitations and uncertainties
- The study was designed as a single-arm trial with a small sample size of 23 patients enrolled over a seven-year period (2016 to 2023) at a single clinical site, which limits the ability to compare outcomes directly to a control group.
Sources
- Apatinib monotherapy for pretreated advanced squamous cell carcinoma of the penis: a phase II trial. — PubMed (NCBI E-utilities)
