A trial comparing pembrolizumab and chemotherapy for people with urothelial (urinary tract) cancer

Cancer type:

Bladder cancer
Kidney cancer
Transitional cell cancer




Phase 3

This trial compared pembrolizumab with chemotherapy for cancer of the urinary tract that had come back or continued to grow despite treatment.

The cancer may have spread elsewhere in the body. This is advanced cancer. 

These urinary tract cancers include those starting from:

  • centre of the kidney (renal pelvis)
  • tube that takes urine from the kidney to the bladder (ureter)
  • bladder
  • tube that drains urine from the bladder and out of the body (urethra)

The lining of the urinary tract is called the urothelium Open a glossary item, so cancer of the urinary tract can also be called urothelial cancer.

This trial was open for people to join between 2014 and 2015. These results were published in 2017 and 2018. 

More about this trial

Doctors often treat urothelial cancer with surgery and chemotherapy using platinum drugs Open a glossary item. But sometimes the cancer can come back after treatment or continue to grow. And it can sometimes spread elsewhere in the body. In this situation, doctors may use a different type of chemotherapy. The most common types are:

In this trial, researchers wanted to see if another type of drug called pembrolizumab would work better. 

Pembrolizumab is a type of immunotherapy. It helps the immune system to kill cancer cells. Pembrolizumab is also used to treat other cancers including lung cancer, melanoma skin cancer and kidney cancer. 

The aims of this trial were to find out:

  • if pembrolizumab worked better than chemotherapy
  • more about the side effects of pembrolizumab
  • how treatment affected day to day life

Summary of results

The trial team found that overall people having pembrolizumab lived longer and had a better quality of life. 

About this trial
This was a phase 3 trial. 542 people took part. 

It was a randomised trial. Everyone was put into 1 of 2 treatment groups. Neither they nor their doctor chose which group they were in. 

521 people had treatment. Of those:

  • 266 people had pembrolizumab 
  • 255 people had chemotherapy 

The average follow up was just over 14 months (14.1). 

The team looked at how long people lived after treatment. On average they found it was:

  • just over 10 months (10.3) for people who had pembrolizumab
  • just under 7½ months (7.4) for people who had chemotherapy 

They also looked at the length of time people lived before their cancer got worse. They found on average that it was:

  • just over 2 months (2.1) for people who had pembrolizumab
  • just under 3½ months (3.3) for people who had chemotherapy

Although this average improvement was small, for some people their individual improvement was very much longer. 

PD-L1 is a protein that cancers have on the surface of their cells. PD-1 is another protein that attaches to PD-L1. When these 2 proteins attach it makes it difficult for the body’s immune system to find and kill the cancer. 

Pembrolizumab works by blocking PD-1 so it can’t attach to PD-L1.

The researchers also looked at people whose cancer had the highest levels of the PD-L1 protein. The number of people in each group was:

  • 74 people in the pembrolizumab group 
  • 90 people in the chemotherapy group

Researchers looked at how long they lived after treatment. On average this was: 

  • 8 months for those who had pembrolizumab
  • just over 5 months (5.2) for those who had chemotherapy

The team found that for people with the PD-L1 protein, there wasn’t any significant difference in the average length of time they lived without their cancer getting worse.  

Quality of life
At certain times during the trial people were asked to fill in a set of questionnaires. This was to do with their quality of life

For these results the team only included people who had filled in at least one set of questionnaires. They had the results of 519 people.

  • 266 people had pembrolizumab
  • 253 people had chemotherapy

Researchers looked at how long it took for people’s overall quality of life to get worse. They found that having pembrolizumab lengthened this time. 
The average time it took was:

  • 3½ months for people having pembrolizumab
  • just under 2½ months (2.3) for people having chemotherapy

The team was particularly interested in the difference in quality of life between the start of treatment and week 15. They found that the drop in quality of life was greater for people having chemotherapy than those having pembrolizumab. 

Side effects
15 out of every 100 people (15%) having pembrolizumab reported having severe side effects. 

Just over 49 out of every 100 people (49.4%) having chemotherapy reported having severe side effects. 

The most common side effects of pembrolizumab were:

  • itchiness
  • tiredness
  • feeling sick

Pembrolizumab affects the immune system and this can cause certain side effects. The team were interested in these side effects which included:

  • the thyroid Open a glossary item producing too little (hypothyroidism) or too much (hyperthyroidism) hormone
  • inflammation of the thyroid
  • inflammation of the lungs (pneumonitis)
  • inflammation of the bowel (colitis)
  • inflammation of the kidneys (nephritis) 
  • severe skin reactions
  • problems with the adrenal glands Open a glossary item

The most common side effects of chemotherapy were:

  • hair loss
  • tiredness
  • a drop in red blood cells (anaemia Open a glossary item)

The team concluded that pembrolizumab: 

  • worked better than chemotherapy
  • was safe
  • gave a better quality of life than chemotherapy

These are the initial results. The trial team are continuing to follow up the people in this trial. They want to find out if pembrolizumab with chemotherapy is better than chemotherapy only to treat people with advanced urinary tract cancer. 

We will update this summary when these results are available. 

Where this information comes from    
We have based this summary on information from the research team. The information they sent us has been reviewed by independent specialists (peer reviewed Open a glossary item) and published in a medical journal. The figures we quote above were provided by the trial team who did the research. We have not analysed the data ourselves.

Recruitment start:

Recruitment end:

How to join a clinical trial

Please note: In order to join a trial you will need to discuss it with your doctor, unless otherwise specified.

Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Chief Investigator

Dr Robert Jones

Supported by

Experimental Cancer Medicine Centre (ECMC)
Merck, Sharp & Dohme

Freephone 0808 800 4040

Last review date

CRUK internal database number:


Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Cara took part in a clinical trial

A picture of Cara

"I am glad that taking part in a trial might help others on their own cancer journey.”

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