Sunday, November 25, 2018

Giving Tuesday; PKD Treatment: Lanreotide Not Effective; Gift of Life: Warm Hands, Organ Donation Law Update; PKD Research: Cilia

PKD Foundation

Join the PKD Foundation for #GivingTuesday

November 27, 2018

#GivingTuesday is right around the corner, and this year, we’re asking: what can you give?

Can you give a donation? Our goal for #GivingTuedsay is to raise $18,000 — the amount needed to fund ten weeks of PKD research. When you select the PKD Foundation as your organization of choice, you help us keep our momentum going and can help bring treatments to patients faster.

Can you give your voice this season? 
When you raise awareness for PKD, you help others better understand this disease and its impact on families worldwide. You can give your voice this #GivingTuesday by:
Using our custom profile frame
Sharing your story on Voices of PKD
Sharing infographics to educate others


Can you give your time? 
Our PKD community wouldn’t be where it is today with the time selflessly given by patients, caregivers, advocates and volunteers. You can give your time this #GivingTuesday by:
Posting your #UNselfie to share how you plan to give this year
Advocating for PKD patients
Getting involved with your local Chapter

No matter what you give this #GivingTuesday, when you give to the PKD Foundation, you are making a difference in the lives of those in our community. Help us further our mission to give hope by helping us fund research, advocate for patients and build a community for all impacted by polycystic kidney disease (PKD).



PKD Treatment

MDLinx

Effect of lanreotide on kidney function in autosomal dominant polycystic kidney disease

In this randomized clinical trial, researchers examined the efficacy of somatostatin analog lanreotide for slowing the rate of decline in kidney function in patients with autosomal dominant polycystic kidney disease (ADPKD). Outcomes revealed no slowing of kidney function decline with lanreotide administration in patients with later-stage ADPKD.



Methods
Researchers performed an open-label randomized clinical trial with blinded end point assessment at 4 nephrology outpatient clinics in the Netherlands; 309 patients with ADPKD were included from July 2012 to March 2015.

Patients aged 18 to 60 years with an estimated glomerular filtration rate (eGFR) of 30 to 60 mL/min/1.73 m2 were eligible for inclusion.

In August 2017, follow-up of the 2.5-year trial ended.

They randomly assigned patients to receive either lanreotide (120 mg subcutaneously once every 4 weeks) in addition to standard care (n = 153) or standard care only (target blood pressure <140/90 mm Hg; n = 152).

Annual change in eGFR assessed as slope through eGFR values during the 2.5-year treatment phase was assessed as the primary outcome.

Change in eGFR before vs after treatment, incidence of worsening kidney function (start of dialysis or 30% decrease in eGFR), change in total kidney volume and change in quality of life (range: 1 [not bothered] to 5 [extremely bothered]) were included as secondary outcome measures.

Results
The trial was completed by 261 (85.6%) of 309 randomized patients (mean [SD] age, 48.4 [7.3] years; 53.4% women).

The lanreotide and the control group had annual rate of eGFR decline of −3.53 vs −3.46 mL/min/1.73 m2per year, respectively (difference, −0.08 [95% CI, −0.71 to 0.56]; P=.81).

Researchers noted no marked differences for incidence of worsening kidney function (hazard ratio, 0.87 [95% CI, 0.49 to 1.52]; P=.87), change in eGFR (−3.58 vs −3.45; difference, −0.13 mL/min/1.73 m2 per year [95% CI, −1.76 to 1.50]; P=.88), and change in quality of life (0.05 vs 0.07; difference, −0.03 units per year [95% CI, −0.13 to 0.08]; P=.67).

The lanreotide group displayed lower rate of growth in total kidney volume than the control group (4.15% vs 5.56%; difference, −1.33% per year [95% CI, −2.41% to −0.24%]; P=.02).

The lanreotide vs control group had adverse events including injection site discomfort (32% vs 0.7%), injection site papule (5.9% vs 0%), loose stools (91% vs 6.6%), abdominal discomfort (79% vs 20%), and hepatic cyst infections (5.2% vs 0%).




PKD Research

From PHYS.org

Monitoring real time changes during cell division

Scientist have cast new light on the behaviour of tiny hair-like structures called cilia found on almost every cell in the body.

Cilia play important roles in human development and disease. Akin to tiny antennae, they act as cell timers keeping the brakes on cell division until the right growth cues are received.

Malfunction of cilia leads to many human diseases such as polycystic kidney disease and cancer.

In the study published in Developmental Cell, researchers from the Universities of Edinburgh and Lancaster developed a multi-component fluorescent biosensor that allows users to 'light up' both cilia and cells that are actively dividing simultaneously. This is important because the machinery that drives cilia growth and cell division are shared.

Dr. Pleasantine Mill of the University of Edinburgh said: "This powerful new tool will allow us to investigate cilia function in human disease in unprecedented detail."

Cilia are important in embryonic development shaping the size of organs, like our brains and lungs. However in adults both loss and hyperactivity in these crucial signalling centres is implicated in cancer cell proliferation and migration.

Dr. Richard Mort of Lancaster University said: "It has long been known that the cilia and cell cycles are closely linked but until now it has been difficult to image these processes in parallel."

The new biosensor will allow researchers to carry out fundamental studies exploring how changes to cilia length and dynamics affect the speed of division and of tissue development.

Joint first author Dr. Matthew Ford said: "This new resource will shed light at an individual cell level on the interplay between the cilia and cell cycles in development, regeneration and disease."

It is hoped these studies will help researchers build a more detailed picture of cilia in human disease and help monitor novel therapeutic approaches.




Gift of Life

From The Courier News, Elgin, IL, by Janelle Walker



The first thing Jeff Speyers noticed when he woke up last Tuesday morning was that his hands felt warm.

In his bed at the University of Wisconsin-Madison Hospital's transplant unit, those warm hands were the first sign the previous night's kidney transplant was already changing his life.

“He was smiling from ear to ear,” his wife, Vicki, said.

Jeff, 60, of South Elgin, waited three years for that transplant. He was diagnosed with polycystic kidney disease — a genetic disorder — at age 45. The disease killed his grandfather, father and brother at young ages so Jeff changed his lifestyle and diet to help slow its progression.


The disorder causes cysts to grow on the kidneys and the stress that places on the body often leads to brain aneurysms, which is what killed his other male family members.

When he was first put on the transplant waiting list, Jeff’s kidneys were functioning at 15 percent each, according to his doctors at the Kovler Organ Transplantation Center at Northwestern Hospital.

At the advice of his Northwestern doctors and with little difference in travel times, he transferred to the Madison hospital. The hope there was for a smaller pool of people awaiting transplants.

Three weeks before the call came, he went to Madison for a checkup. Doctors said his kidney function was down to just 5 percent and either dialysis or a transplant would need to happen by February.

Then, on Nov. 10, the call came.

“They called Saturday at 11 a.m. and said, ‘Get up here by 4 p.m.,’” Vicki said.

“It was more of a shock and the reality of it all came rushing in, that we knew it would happen,” she said.

The Speyers own Hans and Sons Plumbing in South Elgin. Hans was Vicki’s grandfather, and she took the business over from her father. Jeff married into the business. He also serves on the South Elgin and Countryside Fire Protection District Board of Trustees.

Friends in South Elgin held a fundraiser for the couple to cover some treatment costs and missed income — and to publicize Jeff’s need for a kidney donor. The couple never had children so they couldn’t go to a child for a kidney. They also knew that any male children would be at risk of also developing the disorder.

While a few people had made overtures about donating, they were not good matches, the couple said.

They do not know who Jeff’s donor was other than his or her family decided to end life support and allow the organ to be harvested for donation. The kidneys — the other went to another person at the Madison hospital — were helicoptered in from 90 minutes away.

“It is so sad but God bless the person who is the donor. Two lives were saved within hours and that is just from the kidneys. They also had the heart, the lungs, the liver. In the back of my mind, I still think about that family,” Vicki said.

Jeff went into surgery Monday evening. From pre-op to recovery room was just five hours — the new kidney was implanted and his old kidneys remain in place. There is no chance the donated kidney will get the same cysts, and it was an 85 percent match for blood and tissue types, he said.

Still, the couple said from their living room the following Saturday, the kidney is “waking up.”

“It is a sleepy kidney,” which needs to adjust to a new body. Jeff will be on anti-rejection medications for the rest of his life.

As a plumber, Jeff has some understanding of the science.

“It is three pipes you are hooking back up” to connect the kidney to his body, he said with a laugh. A stent from his new kidney to his bladder will also remain in place for three weeks, and Jeff will need to go back to Madison for blood draws and tests twice a week for the next several weeks. Those tests will monitor the new kidney to see if it is working correctly.

There were some immediate changes, like warmer hands and feet. He's still tired but expects to live a full life without worry of further issues as long as there is no organ rejection.

He was back home in South Elgin by Friday night, sitting on the couch where he’d spend much of his weekends for the past few years. Weekends, he said, were the only time he allowed himself to just rest.

The fact that Jeff kept working every day helped his body wait out the time between when a transplant was first suggested and finally getting the surgery, Jeff said.

“My pathologist said my work — physically working, keeping my weight down, not smoking, not drinking, not sitting around” — would help, Jeff said.

“The way Jeff and I look at it, it is a waste of a good day of life moping around and being angry. His attitude, he is the most positive and upbeat person I know,” Vicki said.

Jeff has already outlived the men in his family who died young from the disease, he said. Back when his grandfather and father died from PKD, it was just “kidney disease” and little was known about the why, he said.

“This is best thing that has ever happen to me, outside of marrying Vicki. I am just so thankful,” Jeff said.




From Times Herald, PA, By Dutch Godshalk For Digital First Media

RESTORING LIFE: Donate Life PA Act updates state organ donation law


When Rosalie Hetrick realized it could be years before she’d receive a kidney through the national waiting list, she resolved to take matters into her own hands.

A longtime carrier of polycystic kidney disease, Hetrick, who lives in Norristown, was added to the transplant list in 2015 when her kidney function dropped below 20 percent. Even so, it wasn’t long before her doctor started talking about dialysis.

“I thought the plan was to get a transplant?’” Hetrick asked, confused.

“Not if you don’t look harder for a donor,” her doctor replied. “That may not happen.”

The average wait time for a kidney from the national deceased donor waiting list is five years. Sometimes it’s less, sometimes it’s more. Suffice it to say, someone in Hetrick’s position can never be sure when their number is going to get called.

This real-talk from her doctor gave Hetrick “the boost” she needed to look outside of the list. She asked friends, family, and acquaintances for a kidney. Eventually, “I put it on Facebook,” she said. Where better to issue a widespread call for help?

As luck would have it, a Facebook friend by the name of Ann-Marie Hulstine answered that call and offered a kidney to Hetrick. The transplant operation took place in February.

“When I put it out there, Ann-Marie didn’t hesitate, and she didn’t hesitate during the entire process. It was kismet, or the grace of God,” said Hetrick. “I have not found a way to tell her how grateful I am. I don’t know how you thank somebody for giving you life again.”

Hetrick’s story is inspiring, emotional; it’s a tearjerker about human kindness and life restored. But her story also hints at a growing problem in the U.S. According to government statistics, more than 114,000 Americans are currently on the ever-growing organ transplant waiting list—and they’re dying at rates of roughly 20 per day.

With the recent passage of a new law, state legislators are aiming to tell a different story, one where more people register as organ donors so that fewer people are waiting years for a life-saving transplant (or resorting to a Hail Mary plea on Facebook to find one).

The Donate Life PA Act, which passed unanimously in the House and Senate at the beginning of October, aims to increase the organ donor pool in Pennsylvania, mostly through increased awareness and donor registration opportunities.

For one thing, the new law “provides for all high schools to have access to a model curriculum to teach teenagers about organ donation and transplants,” said Howard M. Nathan, CEO of Gift of Life Donor Program in Philadelphia.

By educating students—grades 9 through 12—the hope is to inspire them to register early on. Students can register to be organ donors when they’re 16 with parental consent.

According to numbers from Gift of Life, Pennsylvania currently has around 4.7 million registered organ donors. Of those millions, Montgomery County has roughly 360,000 donors and Chester County hovers in the area of 231,000, Nathan said.

While these numbers seem large, they can be deceiving.

“Organ donation is a somewhat rare event,” Nathan said. “Less than 2 percent of all people who die in hospital” are medically suitable to have their heart, lung, or kidney transplanted. “And it’s not because people aren’t willing to donate; it’s because the number of people who could be donors is small.”

There are vast criteria for deeming an organ viable for transplantation, including a review of the patient’s medical status, past medical history, and the manner of death. Furthermore, the majority of organs extracted for donation come from patients who have been declared brain dead and are being kept alive through mechanical ventilation.

These many necessary requirements tend to take large donor numbers—like 360,000 in Montgomery County—and whittle them down to slivers.

Last year, the Gift of Life Donor Program had 565 donors, which translated to 1,546 organs transplanted. But if you consider the total number of patients currently waiting for organs in Pennsylvania—around 7,300—“that doesn’t sound like a lot,” Nathan said.

That’s why increasing overall registrants is one of the surest ways of helping patients in need.

“What’s interesting is, when we survey people in general, about 85 percent of the public say they want to be an organ donor,” Nathan said. With about 48 percent signed up in Pennsylvania, that makes for “a gap between the people who sign up and the people who say they want to be donors. We’re trying to make people understand.”

Other provisions under the Donate Life PA Act, which was signed by Gov. Tom Wolf on Oct. 23, include increasing opportunities for adults to register as organ donors. It also ensures a full assessment of organ donation potential by county coroners and health care professionals, with added assurance that any denial of organ donations will be well-documented.

“The coroner has jurisdiction,” Nathan explained. “Even if the individual or family has said yes” to an organ donation, the coroner has authority to deny it. Occasionally, an organ may be turned down for donation due to the needs of a criminal investigation.

“Over the past four years there have been about 40-some turndowns,” said Nathan. The new law “allows procurement organizations to have huddles before the coroner turns it down.”

As lawmakers and advocates continue developing new methods of encouraging Americans to register as organ donors, many patients in need are hedging their bets and taking Rosalie Hetrick’s route. They’re looking for living donors wherever they can. They’re beseeching friends, family members, coworkers, and the nebulous hordes of social media.

When asked her feelings on the Donate Life PA Act, Hetrick said she’s especially in favor of the provisions for access to model curriculum in high schools: “I’m all for anything that brings awareness. The more you can shed light on this, the better.”

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