Sunday, February 24, 2013

8 week Tolvaptan Study Part One

Dear Lucy,

     Mommy has completed an 8 week study for her pkd through Emory Hospital. I started the study in November of 2012. It was a double blind study, meaning I, nor the doctors, knew what dose of Tolvaptan I was on, or if I was on the placebo.

A little history, November of 2011, mommy became extremely sick and had a lot of blood in her urine. I was throwing up until I was dry heaving, daddy was at work, and it was just you and me kiddo. You were almost a year old. You were still crawling and just starting to walk alone, you were the sweetest little girl I had ever seen. You were beautiful and chubby. Perfect in every way. You're still the most beautiful girl I've ever seen, still the sweetest, but you aren't so chubby anymore. Anyways, as I threw up, you crawled from the bedroom to the bathroom, and sat behind me with your little baby hands on my back. As if you were trying to comfort me. I had been sick all day and dealt with it as long as possible, but it got to a point that I felt faint from dehydration. So we called daddy who rushed home, and took mommy to the ER. A few days before this, mommy felt a lump below her ribs, above her belly button, and right at her left side. Mommy thought it could be diverticulitis. Mommy has never had diverticulitis, but it seemed to meet the symptoms. So, we rush to the ER, they pumped mommy full of fluids, perform an X-ray, blood work, etc...

The X-ray showed that I had ruptured a cyst, it also showed that the lump I was feeling was in fact a massive cyst. This was the first time mommy had ever experienced a cyst that she could feel on the outside of her body. One that would bulge like your little elbows would, or like your little feet when mommy was pregnant with you. Before releasing me, my nephrologist informed us that it was time to have some cysts drained. Mommy knew we had all the holidays coming up. So, I sucked it up, dealt with it, and waited until things slowed down.

In April of 2012, we went to Emory for a consultation. It was at that time, that I was introduced to Tolvaptan, and what it was doing for Pkd patients. My doctor at Emory wanted me to join the study but she felt the drainage needed to be done very soon. So a week and a half later, we headed back to Emory for a bilateral percutaneous cyst drainage. Boy oh boy was that fun! We spent around 12 hours in the hospital, one reason was that the department that was performing the procedure was running behind. Once in the room, I remember being asked to lay on my stomach, and I remember the nurse telling me that she was giving me something to help me relax. After that, the only other thing I remember was feeling the lidocaine shots in my sides. When I woke up, they informed me they had drained a few cysts on each kidney and before we left the hospital, I had to get up and go to the bathroom. As I got up for the first time, I turned and looked at the hospital bed. That's when I saw a lot of blood on the bed where I had been laying. I was sent home and that was when more fun began. The cyst drainage was an extremely painful procedure for me. Well the afterwards was painful. They didn't send me home with any pain medicine, but I didn't feel I would need it. When I gave birth to you, I refused to be sent home with pain meds, I took Ibuprofen, and was for sure I could handle this. It was a deep ache in each kidney, if I coughed, moved quickly, moved too much to one side, I would feel this horrific, sharp pain. At that time, you were about 16 months old. I wasn't suppose to pick you up for 6 weeks, but little did they know, you are a mommy's girl, and that was just not possible. So, about 3 days after the procedure, mommy was holding you again. I couldn't standby watching you stretch your chubby arms out at me to hold you, and not carry you, or pick you up. After the procedure, I had to wait 6 months before I could go through screening for the Tolvaptan Study.

This is a photo of the day after the procedure. They used a 10 gauge needle and well, Lucy, mommy does NOT like needles. You seem to share my love of needles.

I read everything I could on the drug and I read all the information about the clinical trial. Seeing that I have a background in pharmacy, it wasn't hard for mommy to understand the chemical makeup of the drug, and the benefits of taking the drug. We headed back to Emory for the screening visit, in hopes that mommy would be accepted. At that appointment, they performed an MRI, blood work, an EKG, recorded weight, blood pressure measurements, height, and well, pretty much you name it, they checked it. Knowing that I was in Stage 3 kidney disease, a GFR under 60, and creatinine at 1.17, my coordinator felt pretty confident I would qualify. The main qualifying factor would be my total kidney volume. The total volume to qualify for the study had to be 340ml. If mommy is not mistaken, the average total kidney volume for someone my age, weight, etc. is 154ml. My kidneys combined were a total kidney volume of over 3200ml. The head doctors over the pkd research department informed me that my numbers may look good, but by looking at my kidneys, they would classify my progression of the disease as severe. Well, my sweet darling girl, they don't know our Jesus! After what felt like a year, I finally got the email, I was accepted!!!! The biggest blessing second to you! The one hope that they have found for pkd patients, and I was in line to receive it! Within a couple of days, I was mailed a huge box with materials pertaining to the study. Inside the box was a cooler, a toilet cap(which you immediately snatched up and turned into a hat), containers for a 24-hr void, a bp monitor, an electronic diary, and instructions for everything. My coordinator mailed me three containers for the 24-hr void. I was to do it the day before my next appointment. The blood pressure monitor was programmed to take my bp every 30 minutes for 24 hrs. Then there was the diary. The diary was supposed to be used to record every time I went pee for 5 days So, once again, we head back to Emory, I turn in my urine, which I barely hit half of one container. We turn in the bp monitor, they reprogrammed the diary, and brought of the medicine. There it was...RIGHT IN FRONT OF ME. Medicine that is so incredibly expensive, the only medicine that has shown to stop the progression of this disease. So we leave with the medicine and immediately after getting into the car, I tear it all open and start examining the pills. Of course, because this is a study, the pills were completely white, no defining markings, no letters, no numbers, nothing! The medicine came in two boxes, each with three small bottles. two bottles marked am, and one marked pm.

Now, we fast forward slightly to the first day taking the medicine. Due to this being a double blind study, there was no indication I would be on the actual drug. My chances of getting the actual drug and not a placebo were 75%, though. Even though the odds were in my favor, I didn't want to get too excited, just in the event I was on the placebo. Mommy made sure to stay skeptical and made sure not to allow her mind to play games with her. Sometimes, our mind can make us believe things are one way, when that is not the actual matter. Day one of the medicine starts, at 8am I take my morning dose which was 2 small, white, round tablets and one white capsule. That afternoon at 4pm, I took one small, white, round tablet. Around 6pm, I determined I was on the placebo. I had taken all of this medicine and felt nothing. Going to bed that night, I kept in mind that after completing the 8 week study, I would qualify for the 3 year open label study. I didn't sleep long that night. I woke up 8 times in the night to pee. 8!!!! That was insane, but that was when I knew! Now, my sweet love, it is time for mommy to put you to bed. I will continue to write you about the journey tomorrow. I love you just about better than anything.




  1. Hi Jamie, I have PKD and am in stage 4. My right kidney takes up 80% of my abdomen now. As you can imagine, it is causing me to have issues as well as pain. I was wondering if having the cysts drained has helped you. I know I will have to have it removed at some point, but I would like to prolong it as long as I can (I have 3 beautiful children to take care of!)

    1. I had the same exact concerns. I needed to be able to take care of my daughter, Lucy. It was very well worth it and after the first week, the pain was gone. The first week, especially the first few days, are pretty painful. Just make sure you are sent home with a pain med to get you through those days. AND SLEEP! Sleep as much as possible for the first 2 days.