A rare cancer type with no standard of care
Upper tract urothelial cancer is a rare type of cancer that starts in the layer of tissue that lines the urothelium and is found in the kidney and ureter, the tube draining from the kidney. Urothelial tissue also lines the bladder, and consequently upper tract urothelial cancer has been treated in the same way as bladder cancer. However, there are differences in the ways these cancers behave.
At present, patients undergo surgery to remove the kidney and ureter but would not receive chemotherapy following surgery unless the cancer returns. There is evidence in similar types of cancer that having chemotherapy after surgery can stop the cancer from returning, but there is limited data supporting this for upper tract urothelial cancer. There is currently no standard of care for clinicians to follow when treating patients diagnosed with upper tract urothelial cancer once they have had their initial operation.
Because upper tract urothelial cancer is a rare cancer previous studies had been hampered by low patient numbers. However, members of the NCRI Bladder Group identified this as an area of unmet need and strived to gather evidence to provide the best possible care for this patient group.
Identifying unanswered questions in cancer treatment
The NCRI Bladder Group* brings together clinicians, scientists, statisticians and NCRI Consumers, amongst many others, to coordinate development of a strategic portfolio of trials within their field of expertise. The group decided that no one individual or organisation would be able to answer the question on the treatment of upper tract urothelial cancer on their own.
The NCRI Bladder Group and the T2 & Below and Advanced Bladder Cancer Subgroups, led by Group Chair Dr Alison Birtle, designed the POUT trial to find out whether having chemotherapy following surgery reduced the chance of cancer returning, in comparison to the traditional approach of close observation. Several group members, including Professor Emma Hall the study’s methodology and clinical trials unit lead, were part of the trial management group. They coordinated over 60 study locations in order to recruit the required number of patients with this rare cancer into the trial, using regional recruitment road-shows as well as providing teleconferences and advice for recruiters, ensuring the trial recruited to time and target. Over 260 patients were recruited.
The importance of consumer involvement
Chris Harris, a key lay member of the POUT trial management group, was involved in the trial from the beginning taking part in focus groups before the trial was developed as well as perfecting the wording for the patient information sheets. He gave valuable input into the POUT trial implementation until the study closed.
Andrew Winterbottom joined the NCRI Bladder Group as a consumer member and contributed to the progress of the trial until it closed in 2017. Andrew supported the trials patient engagement activities and helped disseminate the trial among the members of Fight Bladder Cancer, the charity he founded after he was diagnosed with the disease.
No trial can or should ever begin without asking patients what the best design should be and POUT was a trial that had patient input from conception to completion. Thanks to Chris Harris and Andrew Winterbottom’s input we made this trial a success.
Dr Alison Birtle, Former Chair of the NCRI Bladder Group and POUT trial Chief Investigator
Transforming treatment standards
The results of the POUT trial show that chemotherapy reduces the chance of cancer returning. The researchers estimate that the risk of death or the cancer returning is reduced by about half when chemotherapy is given within three months after surgery. These results are published in The Lancet.
Researchers recommend that every patient in the future who has locally advanced upper tract urothelial cancer should be offered chemotherapy after surgery instead of close observation.
Following these results, a second study is planned to investigate the results of combining immunotherapy or other targeted treatments to chemotherapy following surgery.
*The NCRI Bladder Group and Renal Group were merged in December 2015 to become the NCRI Bladder & Renal Group.