From McCleans, Canada, by PKD Foundation of Canada
For Canadians living with a surprisingly common genetic kidney disease, new research is bringing hope and, for the first time, the possibility of treatment, but serious access challenges remain.
In communities around Canada, people are living with polycystic kidney disease (PKD). The disease is common enough that you likely know someone suffering from it invisibly, and yet awareness of PKD remains low. With options now becoming possible for managing this incurable and potentially deadly disease, advocates are redoubling their efforts to raise awareness of not only the disease but the barriers preventing those who need treatment from equal access.
Jeff Robertson is the Executive Director of the PKD Foundation of Canada, the only national organization dedicated specifically to PKD awareness, research, and advocacy. And he wants you to know just how common and serious PKD really is. “PKD is a group of genetic diseases that cause fluid-filled cysts to grow on affected organs,” he explains. “It predominantly affects the kidneys, but it can impact other organs as well. Over time, in some cases these cysts can lead to organ failure. It’s a non-discriminatory disease, affecting men and women of all ages, races, and ethnicities. The prevalence is approximately 1 in 500 people globally. That amounts to roughly 66,000 Canadians.”
Access barriers
Many of those 66,000 Canadians have been waiting decades for progress in the fight against PKD. “Before, we had no options for slowing the progression of the disease,” says Dr. Andrew Steele, Nephrologist at Lakeridge Health. “Now, with new advances in medicine, we’re finally able to slow down that progression and possibly delay the need for dialysis or transplant. With newer agents coming to the market, hopefully in the future we can continue to push renal failure back even further.”
And yet, many Canadians with PKD are still unable to access these options because of a lack of private or public coverage. “Here we are at a day that many people didn’t think would come within their lifetime,” says Robertson. “Up until a few years ago, options for PKD patients were very limited. With that no longer being the case, the biggest challenge we now face is access. More therapies are currently in development, but without access, these will likely not be available for everyone with the disease.”
This is a disease where every day without intervention potentially worsens the prognosis for patients living with PKD. “The disease is progressive,” says Dr. Steele. “The cysts continue to grow and, with them, the total kidney volume. And we know that people with bigger kidneys are at more risk of progression through the stages of kidney disease.”
That’s why it’s so important that the playing field be levelled. And, because not every patient with PKD is currently a candidate for treatment, it’s equally important that we continue to promote research into new therapies that might help the underserved or improve things even further.
For a disease that affects so many, too few know about the struggle—not only the struggle of living with PKD, but the struggle for fair and equal access. As part of the effort to raise visibility, the PKD Foundation of Canada has been organizing Walk to END PKD events all over the country, providing those with the disease and those who support them with a chance to come out, be seen, and ask this country as a whole to stand with them in the fight for innovation, care, and access.
In the end, the message about PKD should be one of hope. Options are set to expand in the coming years. People are mobilizing. But, as Canadians, it’s essential that we ensure not one person living with this disease is left behind.
If you’re looking for additional resources, the PKD Foundation of Canada is solely dedicated to fighting PKD through research, education, advocacy, support and awareness. Please visit www.endpkd.ca to learn more.
Effect of lanreotide on kidney function in patients with autosomal dominant polycystic kidney disease: The DIPAK 1 randomized clinical trial
Methods
In this open-label randomized clinical trial with blinded end point assessment, researchers examined 309 patients with ADPKD from July 2012 to March 2015 at four nephrology outpatient clinics in the Netherlands.
Patients with 18 to 60 years of age and having an estimated glomerular filtration rate (eGFR) of 30 to 60 mL/min/1.73 m2 were considered eligible.
Participants were followed-up till August 2017, meaning this trial ran for 2.5-year.
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) was randomly administered to patients.
Annual change in eGFR, evaluated as slope through eGFR values during the 2.5-year treatment phase was 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 all assessed as secondary outcomes.
Results
Randomization involved 309 patients (mean [SD] age, 48.4 [7.3] years; 53.4% women), of whom, 261 (85.6%) completed the trial.
According to findings, −3.53 vs −3.46 mL/min/1.73 m2per year (difference, −0.08; [95% CI, −0.71 to 0.56]; P=.81) was the estimated annual rate of eGFR decline for the lanreotide vs the control group.
In terms of 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), no significant differences were observed.
The lanreotide group vs the control group demonstrated lower rate of growth in total kidney volume (4.15% vs 5.56%; difference, −1.33% per year [95% CI, −2.41% to −0.24%]; P=.02).
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%) were documented as adverse events seen in the lanreotide vs control group.
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