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And the call finally came

Fri Mar 29 2019 - It finally happened. I received a call from the specialty pharmacy. They received my prescription. They received all necessary paperwork. They received insurance approval. They were ready to send. Was I ready to receive?

YES!!!

A little background. I have Polycystic Kidney Disease - Autosomal Dominant ('ADPKD') to be precise. 'What the heck is that?!?' you may ask. A most excellent question...
ADPKD is the most common hereditary kidney disease affecting over 12 million people worldwide. It's estimated that between 1 in every 400 to 2,500 people has the disease. It can affect women and men, across all ethnic groups. (pkdinternational.org)
An equal opportunity, inherited disease. Gee. Aren't we lucky?
Healthy kidneys are about 10–12 cm long and are found either side of the mid back. They’re filled with about one million special tubes for filtering the blood. These are called nephrons; they filter out waste products and excess water (making urine).

In people with ADPKD, the lining of individual nephrons can balloon out, causing fluid filled cysts to form. Over time, more and more cysts appear and increase in size, leaving fewer healthy nephrons and space to filter the blood. People with severe ADPKD can have kidneys as large as a football. (
pkdinternational.org)
Everyone with the ADPKD gene has a 50% chance of passing this mutation along to each of their children. My family hit the lottery. My grandmother died from PKD (at the time, doctors didn't really know what was wrong with her). The ADPKD gene was passed along to both my mother and uncle (two of two children). Both have had transplants (very blessed). And of my mother's three children, we're three for three. We're still waiting to see how our cousins fared in the lottery.

There is no cure for PKD. None. Nada. As for prevention, all you can do is eat right, exercise, control your blood pressure and wait for things to progress to End Stage Renal Disease ('ESRD'). At that point your choices are dialysis, possible transplant and/or letting nature run its course. Pretty dismal-sounding but don't give up hope!

A year ago, the FDA approved a drug that has been proven to slow the growth of the cysts which will delay the progression to ESRD. Now we finally have hope! And THIS is what my Friday call was all about.

On Monday, a Fed Ex package will arrive at my home with a drug called Tolvaptan aka Jynarque aka Samsca (I swear the people who name these drugs must be altered at the time).

Let the games begin!

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Round 2 deja vu

My doctor decided to keep me at the initial 45/15 mg dose for another months so I'm anticipating a similar water consumption. Here are the first four of seven (?) or eight (?) five gallon bottles of water. My local grocery store has a water bottle fill station for an extremely reasonable 39 cents a gallon. The frugal side of me thinks I should invest in reusable water bottles and take advantage of the savings. The lazy side of me thinks that it'd be a total hassle to wash the bottles, keep bugs and dust from falling into them before I'm able to refill them, remember to take them with me to the store (I can't even remember those reusable shopping bags) and then stockpile a source of bottle caps. It's just so much easier to buy these recyclable five gallon bottles. Lazy beats frugal.

Artificial kidney

I have a somewhat fanciful thought of what an artificial kidney would look like. I see a device that is shaped like a kidney (of course) but is completely clear so you could see all the blood circulating through it. Granted, once it's implanted no one's going to see it but - hey - it's my vision. The closest picture I can find of what's in my mind's eye is this image. Granted, that's a picture of a kidney-shaped paper weight and it's a little cloudy, but I know you can picture it too. My, oh my. My imagination is not even close to reality. According to a post from the American Journal of Kidney Diseases , there are three new approaches being explored right now: the automated wearable artificial kidney ('AWAK'), the wearable artificial kidney ('WAK') and the implantable artificial kidney ('IAK'). My money's on the IAK and you'll understand why once I show you the concept pictures of the other two. The AWAK is said to ...

A happy liver

I'm two weeks into my Tolvaptan adventure and I'm happy to report that liver tested with no change to the pre-medication levels. A shout out to my liver. Way to go! One of the potential side effects of the medication is liver damage (which has been proven to be reversible as long as it's caught early). Discontinuation of the drug causes the reversal. Fortunately, the reported incidence of liver damage is relatively low at only 4-5%. More recently, in large registration trials of long term therapy in patients with ADPKD, serum aminotransferase elevations occurred in 4% to 5% of patients on tolvaptan, compared to only 1% of controls.  Furthermore, clinically apparent liver injury occurred in approximately 0.1% of treated patients.  The time to onset of illness ranged from 3 to 7 months (Case 1), but occasionally arose during long term therapy (Case 2: after 3 years of intermittent use). National Institute of Health Crossing my fingers that we'll be able to incr...

Truly blessed

For all the medical issues I have, for all the uncertainty that exists in my future, for all the modifications I need to make to my lifestyle... I feel honestly and truly blessed. Yes. I have an incurable disease that can possibly lead me down a path towards  dialysis, organ transplant, lifelong immunosuppressive drugs and early death. But... I don't have diabetes. I don't have a history of aneurysms in my family. My blood pressure is well-managed. My risk for cardiovascular events is low. I work for a company with good healthcare. My job allows me the flexibility to go to doctor appointments and labs.  I have friends and family who love and support me. Could be much, much worse.

Test tube kidneys?

While I'm sure that Miromatrix would shudder at my description, it looks like they're developing the equivalent of a test tube kidney which could be used for transplant. While the science and bioengineering behind it is extremely complex, this is how I understand it. Miromatrix uses decellularization and recellularization technology to create a transplantable kidney. Huh? They take an dead organ - it doesn't even need to be human - and using detergents (I'm guessing they're stronger than Dawn ;-) ) they strip away all the cells. What remains is the kidney's extracellular matrix (collagen, enzymes and glycoproteins) and all its chemical cues. Then the "generic" organ is repopulated with the transplant candidate's cells, eliminating the risk it will be rejected upon implantation. Early studies have shown that it takes from four to eight days to culture the transplantable kidney and, even better, the new organ produces urine when tested. B...