@lcamino, Thank you for sharing! This is indeed exciting news about a treatment for PKD - medical history in the making.

Lynn, I have been reading about your kidney health journey here on Connect, and it has been, and continues to be an honor to be a part of your journey. I am at a loss for words about what this has to mean to your daughter, your relatives, and all patients with PKD. The research being carried on by doctors such as Dr. Torres and team, along with volunteers like you, are some of the reasons why Mayo Clinic is a special place!

Will you be able to continue on this treatment until it is time for you to need a transplant? How often are you monitored to see if there is an actual decrease in your own disease progress?
Rosemary

@rosemarya - I finished the first study (the one that has been published and I got the placebo for) and immediately enrolled in a follow up study where I have been taking Tolvaptan for 8 months. That study is 24 months so I will probably get a transplant before that study is completed. I don't know the plan if the med is available to the public before the study is done. I suspect they will finish the study but instead of me getting the med for free, via the study, I might get it via the pharmacy. Either way I should continue to get the medicine until I have my transplant as long as the FDA approves it.

The study requires me to go to the Mayo monthly for blood and urine tests. My disease will continue to progress, just more slowly than if I was not on the medicine. Dr. Torres says my creatinine has been going up slower than before. The cysts I have will not get smaller and more will still grow, just slower. My kidneys are 9 times bigger than they should be (one 4x and one 5x) and they will continue to grow. The more cysts grow the more my kidney function will decrease so the whole process is just slowed down so it is buying me time. For obvious reasons that is why the sooner you get the medicine the more of a benefit you receive. My dose has had to be decreased because my creatinine got too high. Once the dose is decreased my creatinine goes down, and if it doesn't that is not a good sign.

Hope that explains it. Thanks for your interest.

Lynn,
Thanks for telling about this. How satisfying to have contributed to advancing medical care. Can you tell us a bit more what it was like to participate in the study? Did you have additional appointments or obligations? Did you know if you were receiving the treatment under study or the placebo while you were in the study?
Do you have the link to the study?

Oops, Lynn, I see you answered some of my questions already in your response to Rosemary. I got excited by your first post about the study and just started typing.

@colleenyoung - I'm sorry I don't have a link to the study. Dr. Torres gave me a copy at my appointment just three days after it was published! It is satisfying, exciting, and a true honor.

The first study (published study) involved visits every three months. At those visits I met with the study coordinator, answered questions about how I was doing with the side effects (for part of the study everyone took Tolvaptan and then once the dose you could tolerate was determined you got either the med or a placebo - double blind at this point) and my general health. Always got weighed, temp taken, and provided a urine sample. I got new meds and then went to the lab for a blood draw. In the afternoon I met with Dr. Torres, answered more questions, sometimes an exam was given. The worst part was having to bring a urine sample from the first thing in the morning which meant I had to do that in the airport in Chicago and carry it on the plane. Thankfully I never had it leak in my carry on!

For the other months I had a nurse come to my house who was trained about how to do the study. If I had lived near Mayo I would have had monthly visits but if you were far away just every 3rd visit was at Mayo and the other months were with visiting nurse. The study was a year. The visiting nurse drew blood and then stayed while she spun the blood in a centrifuge and completed paperwork. A courier then arrived, packed the blood in dry ice and drove the blood sample to the airport, or if it was too late he drove it two hours to Indianapolis. I believe all the bloodwork had to be analyzed at the same lab for consistency and the closest to my house was 2 hours away in Indianapolis.

I did need a CT scan, and I think a chest x-ray (been awhile so memory fuzzy), to be admitted into the study. There was a period where I had to show them I could drink the required 8 glasses of water a day. They know how much you are drinking based on blood work (sodium level I believe).

It really was not very invasive, especially if I had lived within driving distance of Mayo. I got a small stipend for each visit to help with travel expenses (I got the maximum amount because I had to fly but it did not come close to covering costs for me). Most of the time I left at 6:15am and arrived home at midnight but I did get stuck over night in Chicago and Rochester a few times due to weather.

It was very organized and staff were very patient if I was late due to my flight being delayed. Mayo Clinic runs very efficiently and I would encourage others to participate in studies if they are able (time) and their doctors approve. I'm delighted that I was able to be a part of this study and am so thankful to the researchers for their years of work to find the first treatment for PKD.

@colleenyoung - I don't know how to give you a link but if you go to http://www.nejm.org and search for Tolvaptan in Later-Stage Autosomal Dominant Polycystic Kidney Disease you can read the first page of the study (need a subscription to New England Journal of Medicine to access the entire study).

I found it
Torres et al. Tolvaptan in Later-Stage Autosomal Dominant Polycystic Kidney Disease; N Engl J Med 2017; 377:1930-1942November 16, 2017DOI: 10.1056/NEJMoa1710030 http://www.nejm.org/doi/full/10.1056/NEJMoa1710030

The good news is that this article is free providing one subscribes. Often one has to pay to access such studies unless you are affiliated with a learning institution.

@colleenyoung - yes that is the one I have at home but when you click on the link it only shows page 1 of 13 pages. What floored me when I read the entire study was that 2292 patients were assessed for eligibility and by the time they got to the randomization phase (splitting between placebo and Tolvaptan) there are only 1370 participants. 687 received the placebo (that's me!). That made me feel fortunate enough but then I read that participants were from around the world with 282 who got the placebo from the United States (that is such a small number that I feel so blessed to have been able to be a part of the study) and 381 that got the placebo were from "other" geographic locations. I had no idea the numbers would be so small. It's just such an honor and privilege that I can't really put it into words.

@lamino. Your post is so uplifting. The relief you feel is almost palpable to those of us reading it. I could not be happier for you and for your family, and for all the other families effected by this.
JK

@lcamino The tacrolimus, one of the immunosuppressants, causes my creatinine to go up. They are considering 1.4 to be my baseline. I am supposed to drink 80 - 100 ounces of water a day to keep my kidneys flushed. Is that an option for you? It is a nuisance because I have incontinence problems - put those two together and I am up all night plus have to always know where there will be restrooms.
JK