Although the disease is called polycystic kidney disease, it has also an effect on the liver, especially in women. Prof. Gansevoort elaborates: “Women with APKD can have polycystic livers; these livers can be greatly enlarged, up to eleven liters.” These enlarged livers are the cause of a lot of complaints, as prof. Gansevoort explains, “These patients have abdominal fullness, so they have limited appetite, they lose weight, they have constipation, distorted body image; they look like they are nine-month pregnant, and normal sexual behaviour is not possible.”
His team recently discovered that the big livers can shrink after menopause, which gave him the idea for his awarded research project: “What we would like to do is induce premature menopause; so inhibit estrogen production, in the hope that we can stop the growth of these polycystic livers or even decrease the size of these livers.” Prof. Gansevoort wants to improve the quality of life of these women with APKD, as emphasised also in the project, and is aware of the fact that inducing premature menopause in women also causes side effects. “On one hand, we hope to improve quality of life by shrinking those huge livers. On the other hand, it can also be that women have more complaints, reduced quality of life, because they experience menopause at a young age.”
When writing the proposal prof. Gansevoort realised the dilemma that the benefit of his research approach may be counteracted by side-effects and concluded that the best thing to do was to involve patients. Prof. Gansevoort explains: “We immediately contacted the working group for polycystic kidney disease of the Dutch Kidney Patient Society (NVN), but also the Dutch Liver Patient Organization (NLV) […] they came up with their own ideas, for instance, the focus groups.” A focus group is a group of people assembled to participate in a discussion about a product before it is launched, in this case a clinical research design. Prof. Gansevoort and this team hired an independent social scientist to lead the focus groups. In addition, he as researcher did not attend these meetings, so that the women could give their unbiased opinion. “Would the complaints of premature menopause be outweighed by the benefit you might have from shrinking the liver and postponing or even preventing the need of a liver transplant?” was one of the questions the women were asked. Furthermore, these women gave valuable advice on how to improve trial design from a patient perspective. This approach has been quite successful: until now all women agreed on participating in such a trial, so in their opinion the complaints of the treatment would be outweighed by the benefits of the treatment.
The scientist who led the focus groups also wrote a report about it that resulted in clear recommendations and was added to the grant application. Prof. Gansevoort explains: “We tried to accommodate all the recommendations from that report, but were not able to accommodate one or two. We wrote in our application why we could not accommodate those specific recommendations in the protocol. This shows that it is clear that you have listened to the patients and tried to do your best to get the trial design as optimal as possible for the patients that should participate.” Prof. Gansevoort thinks these focus groups and the way they were organised have been a pivotal advantage in applying for the ZonMw grant. “The reviewers and the granting body also found it a great example of patient involvement.”
