Materiały źródłowe

• #1 Treatment and Management of Autosomal Dominant Polycystic Kidney Disease – Polycystic Kidney Disease – NCBI Bookshelf

  https://www.ncbi.nlm.nih.gov/books/NBK373382/

  Autosomal dominant polycystic kidney disease (ADPKD) is the most common life-threatening single-gene disease. […] Recently, there have been new therapeutic hopes with identification of specific drugs based on the mechanisms of kidney progression. […] This chapter reviews the treatment and management of ADPKD progression, and the identification of ADPKD patients with rapidly progressing disease, hypertension, and extrarenal complications. […] For a long time, the treatment and management strategies of this disease have not progressed in comparison with the treatment of others kidney diseases. […] Recently, there have been new therapeutic hopes with identification of specific drugs based on the mechanisms of kidney progression. […] Ensuring that Tolvaptan is used in a safe and effective manner requires multiple considerations, including the careful selection of patients eligible for treatment, based on risk of progression.

• #1 Available Treatment – Polycystic kidney disease | PKD treatment research | PKD Foundation

  https://pkdcure.org/tolvaptan/

  Tolvaptan treatment for ADPKD […] On April 24, 2018, the U.S. Food and Drug Administration (FDA) granted approval of tolvaptan to be the first treatment in the United States for adult patients with autosomal dominant polycystic kidney disease (ADPKD), the most common form of polycystic kidney disease (PKD). […] Tolvaptan is a medication (taken twice a day as an oral pill) that affects how the kidneys control the concentration of urine. It has been shown to slow down the growth of kidney cysts (total kidney volume) when it is taken for a long time (several years) by adults at risk of rapidly progressing ADPKD. This may help protect the function of your kidneys and delay the need for a kidney transplant or dialysis. Your kidney function would continue to decline, but at a slower rate. […] Tolvaptan is indicated to slow kidney function decline in adults at risk of rapidly progressing ADPKD. Rapidly progressing is not a defined standard it takes many factors specific to each patient into account. Because of this, patients who are interested in the drug need to speak with their physician to consider all factors, as well as potential side effects, to decide if it is a good choice for them. If your risk of rapid progression is determined as high, it would be best to initiate the treatment early, even if kidney function it relatively normal. This will allow for maximum gain out of the tolvaptan therapy.

• #1 Drug management of autosomal dominant polycystic kidney disease

  https://australianprescriber.tg.org.au/articles/drug-management-of-autosomal-dominant-polycystic-kidney-disease.html

  Autosomal dominant polycystic kidney disease is the most common genetic kidney disease affecting adults. Approximately 60% of patients develop kidney failure by 60 years of age due to slowly expanding kidney cysts. […] A healthy lifestyle and rigorous control of blood pressure slow kidney cyst growth. These interventions can be effective in reducing progression to kidney failure and cardiovascular disease, especially if started in early adulthood. […] Tolvaptan, a vasopressin receptor antagonist, slows kidney cyst growth and the decline in the estimated glomerular filtration rate by 1 mL/minute/1.73 m2 per year. It is indicated in patients with chronic kidney disease who are at high risk of progression to kidney failure. […] The treatment of hypertension follows standard guidelines and should be integrated with routine screening for other cardiovascular disease risk factors (such as hyperlipidaemia and impaired glucose tolerance).

• #1

  https://www.nhs.uk/conditions/autosomal-dominant-polycystic-kidney-disease-adpkd/treatment/

  Occasionally, large cysts can be drained to help relieve the pain caused by the increased pressure. […] Urinary tract infections (UTIs) can often be treated with a 7- to 14-day course of antibiotic tablets. […] If the infection persists despite antibiotic treatment, the infected cysts may need to be drained during surgery or using a needle inserted through your skin. […] The 2 main treatment options for kidney failure are: dialysis, where a machine replicates some of your kidneys’ functions; a kidney transplant, where a healthy kidney is removed from a living or recently deceased donor and implanted into someone with kidney failure. […] Tolvaptan is a medication that’s recommended by the National Institute for Health and Care Excellence (NICE) to treat ADPKD in adults. […] It can be used to slow down the growth of cysts, reducing overall kidney growth and preserving kidney function for longer. […] Tolvaptan comes in tablet form and is taken twice a day as a split dose.

• #1 Autosomal dominant polycystic kidney disease (ADPKD): Treatment – UpToDate

  https://www.uptodate.com/contents/autosomal-dominant-polycystic-kidney-disease-adpkd-treatment

  Autosomal dominant polycystic kidney disease (ADPKD): Treatment […] Tolvaptan is a vasopressin V2-receptor (V2R) antagonist with proven beneficial results in ADPKD. […] In adult patients with ADPKD who have an eGFR ≥25 mL/min1.73m² and who are at high risk of CKD progression, we suggest treatment with tolvaptan. […] The preferred starting dose is 45 mg in the morning and 15 mg eight hours later and before 4 PM to diminish the risk of severe nocturia. […] The cost and affordability of tolvaptan for ADPKD (brand name Jynarque) depends upon individual prescription drug policies. […] In the two largest trials, common side effects of tolvaptan that were more frequent than with placebo included thirst, polyuria, nocturia, polydipsia, hypernatremia, and increased liver enzymes. […] We do not prescribe other potentially disease-modifying agents (other than tolvaptan), because they lack demonstrable benefit and because their interactions have not been evaluated. […] Nephrectomy is generally avoided in ADPKD because of potential complications and the adverse effects of removing a partially functioning kidney. However, nephrectomy may be an option in patients with disabling symptoms due to massively enlarged kidneys, development of ventral (abdominal wall) hernias, or suspected renal cell carcinoma. […] Patients with ADPKD who progress to end-stage kidney disease (ESKD) require kidney replacement therapy.

• #1 Drug management of autosomal dominant polycystic kidney disease

  https://australianprescriber.tg.org.au/articles/drug-management-of-autosomal-dominant-polycystic-kidney-disease.html

  The first-line drug classes for treating hypertension are blockers of the renin-angiotensin-aldosterone system (either ACE inhibitors or angiotensin receptor antagonists). […] In patients with early-stage disease (eGFR 60 mL/min/1.73 m2), the recommended blood pressure target is between 120/70 mmHg and 130/80 mmHg. […] Tolvaptan is a specific oral vasopressin type 2 receptor antagonist and is indicated in patients with ADPKD at high risk of developing kidney failure. […] The main adverse effect of tolvaptan is massive aquaresis (mean urine volume of 57 L/day) due to the off-target suppression of vasopressin-mediated water reabsorption in the collecting duct. […] Chronic pain is a common and overlooked clinical problem in ADPKD. Recognising pain and providing effective pharmacological management can significantly improve the well-being of people with ADPKD.

• #1 Polycystic Kidney Disease Treatment & Management: Approach Considerations, Blood Pressure Control, Infectious and Other Disorders

  https://emedicine.medscape.com/article/244907-treatment

  The aim of current treatment for ADPKD is to slow the decline in kidney function and reduce extrarenal complications. Specific disease features to address include the following: […] Patients with ADPKD who progress to advanced chronic kidney disease (CKD) may require hemodialysis, peritoneal dialysis, or kidney transplantation. […] Tolvaptan, a selective vasopressin V2-receptor antagonist, is approved in the United States, Japan, Canada, Europe, and elsewhere for slowing kidney function decline in adults at risk of rapidly progressive ADPKD. […] Treatment with tolvaptan led to improvement in eGFR in the 1-year REPRISE trial. […] The primary endpoint in the TEMPO 3:4 and TEMPO 4:4 studies was the intergroup difference for rate of change of total kidney volume (TKV). […] Tolvaptan causes elevations in liver enzymes in about 5% of recipients.

• #1 Polycystic kidney disease – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/polycystic-kidney-disease/diagnosis-treatment/drc-20352825

  How bad polycystic kidney disease is varies from person to person. That’s true even among people in the same family. Often, people with PKD reach end-stage kidney disease between ages 55 and 65. But some people with PKD have mild disease. They might never get to end-stage kidney disease. […] Treating polycystic kidney disease involves dealing with the following symptoms and complications in their early stages: […] The medicine tolvaptan (Jynarque, Samsca) may be used for adults at risk of ADPKD that’s getting worse fast. Tolvaptan is a pill that you swallow that slows how fast kidney cysts grow. It also slows the decline in how well your kidneys work. […] Keeping high blood pressure under control can slow the disease and kidney damage. […] To help your kidneys stay as healthy as possible for as long as possible, experts suggest being at a healthy weight and body mass index. Drinking water and fluids throughout the day may help slow the growth of kidney cysts. This could slow the loss of kidney function.

• #1

  https://www.nhs.uk/conditions/autosomal-dominant-polycystic-kidney-disease-adpkd/treatment/

  There’s currently no cure for autosomal dominant polycystic kidney disease (ADPKD), and it’s not possible to stop cysts forming in the kidneys. […] But there are some potentially useful medications, such as tolvaptan, that can sometimes be used to reduce the growth rate of cysts. […] If you’re diagnosed with ADPKD, you’ll usually see a kidney specialist, who can help draw up a suitable treatment plan. […] Medication is usually used to treat high blood pressure in people with ADPKD. […] Angiotensin-converting enzyme (ACE) inhibitors and angiotensin-2 receptor blockers (ARBs) are the 2 medications most widely used. […] In many cases of ADPKD, any pain you experience can be relieved by treating the underlying cause, such as kidney stones or a urinary tract infection (UTI). […] If you need to take a painkiller, paracetamol is the best medication to try first.

• #1 Autosomal Dominant Polycystic Kidney Disease (ADPKD) – Genitourinary Disorders – Merck Manual Professional Edition

  https://www.merckmanuals.com/professional/genitourinary-disorders/cystic-kidney-disease/autosomal-dominant-polycystic-kidney-disease-adpkd

  Treatment of ADPKD includes control of complications (eg, hypertension, infection, renal failure) […] Supportive measures include increased fluid intake (particularly water) to suppress vasopressin release, even if only partially, in patients who are able to safely excrete the load. […] Strict control of hypertension is essential. Typically an angiotensin-converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) is used. In addition to controlling blood pressure, these medications help block angiotensin and aldosterone, growth factors that contribute to renal scarring and loss of renal function. […] Urinary tract infections (UTIs) should be treated promptly. […] Nephrectomy is an option to relieve severe symptoms due to massive kidney enlargement (eg, pain, hematuria) or recurrent UTIs.

• #1

  https://www.painscale.com/article/at-home-treatment-for-polycystic-kidney-disease

  There is currently no cure for PKD; however, treatment options can slow the progression of PKD and reduce the rate that cysts are formed. […] At-home treatment options can help manage symptoms and control other issues. At-home treatment includes medications, nutrition, physical activity, reduced stress, and lifestyle changes. […] Acetaminophen and other over-the-counter pain-relievers may alleviate or reduce the pain associated with PKD. […] Increased fluid intake is important in the process of slowing the growth of kidney cysts and diluting bloody urine, which helps to prevent blood clots in the urinary tract. […] Increased physical activity is recommended to help control high blood pressure that is associated with PKD. […] Since stress can elevate blood pressure, reducing stress has the potential to lower high blood pressure. […] Individuals with PKD should not smoke. Quitting or decreasing tobacco is beneficial for lowering blood pressure and preventing aneurysms.

• #1 Managing Pain with ADPKD

  https://www.webmd.com/a-to-z-guides/manage-pain-adpkd

  Chronic pain is a common challenge when you have autosomal dominant polycystic kidney disease (ADPKD). But there are many treatment options to help you manage your pain and other symptoms so you can feel your best. […] Pain and treatments for pain affect people differently, and many things can cause pain for people with ADPKD. So its important to talk to your doctor about your specific problems to see what options are best for you. […] To best treat your pain, your doctor has to first diagnose whats causing it. […] If your pain is coming from a urinary tract infection, cyst infection, kidney stone, bleeding cyst, or other problem, your doctor may treat it with antibiotics or other medicine or treatment. […] If you have a cyst hemorrhage or a lot of blood in your urine, your doctor may suggest medication and rest.

• #1 Polycystic Kidney Disease | Ausmed

  https://www.ausmed.com/learn/articles/polycystic-kidney-disease

  There is no cure for PKD, but early detection and management can help to reduce the risk of complications (Better Health Channel 2017). […] Typically, first-line treatment involves slowing the growth of cysts via lifestyle management and modification (e.g. regular exercise, smoking cessation, dietary changes), along with controlling blood pressure. In some cases, these are the only interventions needed (Kidney Health Australia 2019). […] Other interventions may include: antihypertensive medicine to manage hypertension, draining cysts to relieve pain, fluids, analgesia, antibiotics and rest to treat haematuria, antibiotic treatment for any urinary tract infections, medicine (tolvaptan) to slow the progression of cysts (for adults with rapidly progressing ADPKD), dialysis or a kidney transplant to treat kidney failure, psychological support to help manage feelings of sadness and anxiety associated with a PKD diagnosis, avoiding contact sports if the kidneys, liver, spleen or abdomen are enlarged, as these organs could potentially be injured if hit (Kidney Health Australia 2019; Better Health Channel 2017). […] Patients should also be advised to avoid taking non-steroidal anti-inflammatory drugs (NSAIDs) as they can potentially worsen kidney function (Better Health Channel 2017).

• #1 Polycystic Kidney Disease (PKD) Symptoms, Treatments & Causes – American Kidney Fund (AKF)

  https://www.kidneyfund.org/all-about-kidneys/types-kidney-diseases/polycystic-kidney-disease

  Polycystic kidney disease (PKD) is a genetic disorder that causes many fluid-filled cysts to grow in your kidneys, leading to kidney damage. […] There is no cure for ADPKD. Your doctor can recommend strategies to manage some of the symptoms and health problems caused by ADPKD. […] For those with mild pain, acetaminophen is typically used to manage it. […] If you have very large cysts that are causing pain, your doctor may decide to treat them by aspirating (draining) and sclerosing (shrinking) them. […] In ADPKD, hypertension occurs because the growing cysts can damage the kidneys and cause them to retain sodium and water, which raises blood pressure. […] It’s important to treat hypertension because controlling high blood pressure has shown not slow the growth of kidney cysts in ADPKD.

• #1 Polycystic Kidney Disease Clinic | Ohio State Medical Center

  https://wexnermedical.osu.edu/kidney-care/nephrology-clinics/polycystic-kidney-disease

  Ohio State has revolutionized care for polycystic kidney disease patients, providing the latest medications and treatment strategies to prevent cyst formation and slow disease progression. […] Theres currently no cure for PKD, but treatment is available to help manage symptoms and slow disease progression. […] Treatment is determined by the stage of kidney disease and size of kidneys. Possible treatments include: […] Your doctor may prescribe a drug called tolvaptan the leading treatment for polycystic kidney disease. Tolvaptan suppresses a hormone associated with cyst growth, preventing cysts from growing. […] Your doctor may advise increasing your water intake. Drinking water throughout the day and avoiding caffeine can slow the growth of cysts. […] Your doctor may recommend lifestyle changes like limiting sodium in your diet, or they might prescribe medication to help manage your blood pressure.

• #1 Polycystic kidney disease (PKD) – Symptoms, causes, treatment | National Kidney Foundation

  https://www.kidney.org/kidney-topics/polycystic-kidney-disease

  At present, there is no cure for PKD. However, a lot of research is being done. Recent studies suggest that drinking plain water throughout the day and avoiding caffeine in beverages can slow the growth of cysts. Research is also helping us understand the genetic basis of PKD. […] Studies also suggest that some treatments may slow the rate of kidney disease in PKD, but further research is needed before these treatments can be used in patients. In the meantime, many supportive treatments can be done to control symptoms, help slow the growth of cysts, and prevent or slow down the loss of kidney function in people with PKD. These include: […] In April 2018, the FDA approved a new drug called tolvaptan for the treatment of autosomal dominant polycystic kidney disease (ADPKD). The drug can be used to help slow kidney function decline in adults at risk for this type of PKD. You can speak with a healthcare professional for more information about this treatment and to see if its right for you.

• #1 Polycystic kidney disease (Causes, Symptoms and Treatment)

  https://patient.info/doctor/autosomal-dominant-polycystic-kidney-disease

  Polycystic kidney disease treatment and management […] Diagnosis of ADPKD has implications both for the patient and for their family. Consider the information needs of the patient and their family members. Provide written information and sources of support relating to the disease and be prepared to discuss the nature of the disease, possible complications and likely prognosis as well as the genetic implications for other family members. Screening is usually delayed beyond childhood. However, blood pressure can rise before that age and so should be checked. The option for screening should be given in advance of an individual starting their own family. Advise against participating in contact sports which risk abdominal trauma, such as rugby or boxing, as this can provoke haematuria or even cause the rupture of an enlarged kidney. Much of the morbidity and mortality of ADPKD is due to CVD. Thus, lifestyle advice regarding avoiding smoking, maintenance of a healthy body weight, diet (eg, limiting salt intake) and regular exercise (ideally of a low-impact nature, such as swimming or walking) would seem sensible, even if not studied within the ADPKD population specifically. Care and monitoring would usually be undertaken jointly by primary care and renal teams.

• #1 Polycystic Kidney Disease (PKD): Symptoms & Treatment

  https://my.clevelandclinic.org/health/diseases/5791-polycystic-kidney-disease

  Most people with PKD will eventually need dialysis or a kidney transplant. […] If you receive a PKD diagnosis, it’s important to work with your healthcare provider on a treatment plan to manage complications of the disease. […] The goal of treatment is to slow the progression of the disease and control the symptoms it causes. The most common treatments for PKD include: […] Your provider helps you manage your blood pressure with medicine, an eating plan and exercise. […] If you have kidney failure, you may need dialysis (a procedure to clean your blood). […] You may need a kidney transplant if ADPKD progresses to kidney failure. […] Medicine can manage pain caused by infections, kidney stones or burst cysts. […] Polycystic kidney disease isn’t preventable. But you may be able to slow the disease or prevent kidney failure by practicing a healthy lifestyle. […] People with ADPKD can live long and full lives if they receive the proper treatment and manage the condition. […] Treatment reduces symptoms and helps your kidneys work better. It’s important to work with your provider and follow their instructions to manage the condition.

• #1 Polycystic kidney disease (PKD) – edren.org

  https://edren.org/ren/edren-info/polycystic-kidney-disease-pkd/

  Tolvaptan (Jinarc) is the first drug licensed to slow PKD. While taking it, cysts grow less quickly. In trials in patients with quite enlarged kidneys, it slowed down loss of kidney function by about 30%. So if you were going to get kidney failure in 15 years, this could stretch it out to 20. […] There isn’t a cure for PKD, but treatment is now becoming available to slow down the growth of cysts. All treatments are likely to have some risks and possible side effects. […] Most patients with PKD develop high blood pressure which continues for the rest of their lives unless treated effectively. High blood pressure can have serious effects on the heart and blood vessels. It should be treated for this reason. […] If kidney failure occurs, dialysis and transplantation are very successful in people with PKD. Kidney transplantation is the best way of regaining full health. Occasionally the kidneys by then are so big that one or both must be removed to make space for dialysis or for a kidney transplant. Removing a kidney this size is a big operation, and it removes any remaining kidney function, so it is only done if it is essential.

• #1 Polycystic Kidney Disease Treatment | Baptist Health | Arkansas

  https://www.baptist-health.com/services/kidney-care/polycystic-kidney-disease

  Polycystic kidney disease (PKD) is a genetic disorder characterized by the growth of numerous cysts filled with fluid in the kidneys. PKD cysts can reduce kidney function, leading to kidney failure. […] Treatment for polycystic kidney disease may include pain medication, surgery to shrink cysts and relieve pain, treatment for high blood pressure, treatment for urinary tract infections and in some cases, dialysis and kidney transplantation. […] A kidney transplant is a surgical procedure performed to replace a diseased kidney with a healthy kidney from another person. The kidney may come from a deceased donor or from a living donor. Individuals who donate a kidney can live healthy lives with the kidney that remains.

• #1 Scientists discover potential treatment approaches for polycystic kidney disease | National Institutes of Health (NIH)

  https://www.nih.gov/news-events/news-releases/scientists-discover-potential-treatment-approaches-polycystic-kidney-disease

  Researchers have shown that dangerous cysts, which form over time in polycystic kidney disease (PKD), can be prevented by a single normal copy of a defective gene. This means the potential exists that scientists could one day tailor a gene therapy to treat the disease. […] The organoid models also provided the first opportunity to study the effectiveness of a class of drugs known as eukaryotic ribosomal selective glycosides on PKD cyst formation. […] Freedman’s team found that the drugs could restore the ability of genes to make polycystin, increasing the levels of polycystin-1 to 50% and preventing cysts from forming. Even after cysts had formed, adding the drugs slowed their growth. […] Freedman suggested that a next step would be to test existing glycoside drugs in patients. Researchers also could explore the use of gene therapy as a treatment for PKD.

• #1 A promising treatment for an incurable, deadly kidney disease: Newsroom – UT Southwestern, Dallas, TX

  https://www.utsouthwestern.edu/newsroom/articles/year-2019/potential-treatment-polycystic-kidney-disease.html

  A potential treatment for polycystic kidney disease a genetic disorder that causes the kidneys to swell with multiple cysts and can eventually lead to organ failure has shown promising results in animal testing. […] The drug is now in early clinical trials in human subjects, said Dr. Vishal Patel, Associate Professor of Internal Medicine at UT Southwestern and senior author of the study. […] The only drug currently approved to treat ADPKD, called Jynarque (generic name tolvaptan), carries the FDAs highest warning in its prescribing information, a box notifying prescribers and users of the possibility of serious and potentially fatal liver injury. […] The new treatment cooperatively developed at UT Southwestern and Regulus Therapeutics Inc., a biopharmaceutical company based in California, showed no evidence of toxicity in animals or in human cell tests, according to the study. […] RGLS4326, as the new drug is called in development, works by blocking the harmful microRNA-17. […] Early phase one clinical trials began last year, conducted by Regulus Therapeutics.

• #1 A new drug candidate can shrink kidney cysts | MIT News | Massachusetts Institute of Technology

  https://news.mit.edu/2024/new-drug-candidate-can-shrink-kidney-cysts-0122

  A compound originally developed to treat cancer could be repurposed to treat polycystic kidney disease, an inherited condition that can lead to kidney failure. […] Researchers at MIT and Yale University School of Medicine have now found that a compound originally developed as a potential cancer treatment holds promise for treating ADPKD. The drug works by exploiting kidney cyst cells vulnerability to oxidative stress a state of imbalance between damaging free radicals and beneficial antioxidants. […] Using two different mouse models of ADPKD, the researchers showed that 11beta-dichloro could significantly reduce the size of kidney cysts and improve kidney function. […] The researchers also synthesized a defanged version of the compound called 11beta-dipropyl, which does not include any direct DNA-damaging ability and could potentially be safer for use in humans.

• #1 Polycystic Kidney Disease Treatment: Gene Target Identified at Yale < Yale School of Medicine

  https://medicine.yale.edu/news-article/polycystic-kidney-disease-treatment-gene-target-identified/

  An unsuspected gene, Glis2, has been found to be a target of polycystic kidney disease (PKD) signaling in preclinical models. […] New research from Yale School of Medicine, published in Nature Communications, has discovered that the gene Glis2 is a component of PKD signaling and may also be a new target for the treatment of the disease. […] Glis2 has not been suspected to be a component of the signaling pathways involved, but our research shows its quite centrally positioned in polycystin signaling. […] As part of the study, the investigators also treated models with antisense oligonucleotide (ASO) directed against Glis2, which they found suppressed the growth of cysts. […] When I found that the disease progression was significantly prevented in in vivo studies, I was very excited and realized that the Glis2 can play a crucial role in further dissecting PKD signaling and enriching our understanding of mechanisms of the human ADPKD, and also could lead to developing treatments of the disease.

• #1 Kidney disease: Research may pave the way for gene therapy for ADPKD

  https://www.medicalnewstoday.com/articles/research-suggests-avenues-toward-gene-therapies-for-polycystic-kidney-disease

  Gene therapy may be an effective future treatment for polycystic kidney disease […] A recent study using a mouse ADPKD model showed that the PC1-CTT fragment could ameliorate the development of cysts in the kidney and help preserve kidney function. […] A recent study published in the journal Nature Communications reports that expressing a small fragment of the PC1 protein, known as the C-terminal tail (CTT) fragment, helped reduce disease severity in an ADKPD mouse model. […] Our work will provide new insights into the underlying disease mechanisms for polycystic kidney disease and reveal new avenues for developing therapies. […] Studies in animal models have shown that therapeutics or interventions that target these metabolic pathways can help alleviate the symptoms of ADPKD. […] The researchers said these experiments suggest that the ability of PC1-CTT to attenuate the severity of ADPKD is mediated by its interaction with NNT.

• #1 Autosomal Dominant Polycystic Kidney Disease – PYC Therapeutics

  https://pyctx.com/autosomal-dominant-polycystic-kidney-disease/

  PYC has developed PYC-003, a treatment that could arrest the disease course of ADPKD and enable regeneration and restoration of kidney function. […] PYC-003 increases PKD1 gene expression, which could arrest disease progression, enable damaged kidneys to regenerate, and restore function. […] PYC-003 is a transformative RNA therapeutic that addresses the underlying PKD1 gene mutation to effectively treat disease at its root cause. By treating ADPKD at the genetic level, PYC-003 has the potential to halt disease progression and allow ADPKD-affected kidneys to restore function. […] PYC’s therapeutic, PYC-003, seeks to increase protein expression in the remaining healthy copy of the PKD1 gene to restore PC1 protein production to levels that are sufficient for the cells to function normally.

• #1 Polycystic kidney disease – Wikipedia

  https://en.wikipedia.org/wiki/Polycystic_kidney_disease

  In 2018, Jynarque (Tolvaptan) was introduced as the first FDA-approved treatment for PKD. In a recent long-term study, patients using Tolvaptan had a 6.4% higher kidney function after 5 years compared to standard of care. […] In 2019, a team of researchers at UCSB found that a ketogenic diet might be able to halt, or even reverse progression in mice, and the results of a first human case series study are showing potential benefit. […] The results of a 3-month randomized, prospective dietary intervention clinical trial are pending. […] In addition, recent research indicates that mild to moderate calorie restriction or time-restricted feeding slow the progression of autosomal dominant polycystic kidney disease (ADPKD) in mice. […] Patient communities have been combining both ketogenic diets and time-restricted feeding with a low-oxalate diet to prevent the formation of stones and early reports show an average of 17% increase in kidney function after approximately one year on a ketogenic, time-restricted dietary regimen.

• #1

  https://umiamihealth.org/en/treatments-and-services/nephrology/polycystic-kidney-disease

  Polycystic kidney disease is an inherited condition in which clusters of cysts, or fluid-filled sacs, develop in the kidneys. As cysts continue to multiply over time, they can cause the kidneys to enlarge and lose their function. […] University of Miami Health System nephrologists (kidney specialists) provide advanced treatments for polycystic kidney disease, including treatments to help slow the rate of kidney cyst growth, prevent declining kidney function, and manage the signs, symptoms, and complications of polycystic kidney disease. […] The ADPKD Center of the University of Miami provides comprehensive, multidisciplinary and individualized care for patients and families affected by ADPKD. Our integrated care team includes key specialists with expertise in ADPKD, including […] Your physician may prescribe tolvaptan (Jynarque), the only FDA-approved treatment for adults at risk of rapidly progressive ADPKD. Tolvaptan is a pill that works to slow down the rate of kidney cyst growth and delay the progression of kidney disease.

• #2 Polycystic kidney disease: symptoms, treatments, causes and diagnosis

  https://www.kidneyresearchuk.org/conditions-symptoms/polycystic-kidney-disease/

  ADPKD is now commonly treated by a drug called Tolvaptan, which has been available across the UK since early 2016. Tolvaptan slows the progression of cyst development and helps protect kidney function. In most cases this is likely to significantly delay the need for dialysis or a kidney transplant for ADPKD patients. […] Other treatments may also include: medications such as ACE Inhibitors and ARBs to control blood pressure. […] ARPKD is a rare condition caused by a genetic fault that disrupts normal development of the kidneys and liver. It may present in very young children or later in adulthood. Over time one or more of these organs may fail. Whilst there is no cure for ARPKD, there are various treatments that can help manage the disease, including dialysis and kidney transplantation.

• #2 Polycystic kidney disease (PKD) – Symptoms, causes, treatment | National Kidney Foundation

  https://www.kidney.org/kidney-topics/polycystic-kidney-disease

  At present, there is no cure for PKD. However, a lot of research is being done. Recent studies suggest that drinking plain water throughout the day and avoiding caffeine in beverages can slow the growth of cysts. Research is also helping us understand the genetic basis of PKD. […] Studies also suggest that some treatments may slow the rate of kidney disease in PKD, but further research is needed before these treatments can be used in patients. In the meantime, many supportive treatments can be done to control symptoms, help slow the growth of cysts, and prevent or slow down the loss of kidney function in people with PKD. These include: […] In April 2018, the FDA approved a new drug called tolvaptan for the treatment of autosomal dominant polycystic kidney disease (ADPKD). The drug can be used to help slow kidney function decline in adults at risk for this type of PKD. You can speak with a healthcare professional for more information about this treatment and to see if its right for you.

• #2 Available Treatment – Polycystic kidney disease | PKD treatment research | PKD Foundation

  https://pkdcure.org/tolvaptan/

  It is not possible to know exactly how long tolvaptan may preserve kidney function. Clinical studies showed that the drug slowed the growth of cysts and preserved kidney function as compared to placebo. In patients with risk of rapid progression, it is estimated that each four years of tolvaptan treatment could delay the need for renal replacement therapy (dialysis or transplant) by one year. […] Tolvaptan is approved for use in patient with rapid progressing ADPKD. Future studies are still needed to determine if tolvaptan could help reducing the liver cysts. At this time, tolvaptan is not indicated to slow progression of polycystic liver disease.

• #2 Treatment and Management of Autosomal Dominant Polycystic Kidney Disease – Polycystic Kidney Disease – NCBI Bookshelf

  https://www.ncbi.nlm.nih.gov/books/NBK373382/

  Japan and the European Medical Agency have approved Tolvaptan for treatment of ADPKD in adults with stage 1 to 3 chronic kidney disease at the outset and evidence of rapidly progressing disease. […] Identifying ADPKD patients with evidence of rapidly progressing disease requires a consensus to define which scores to use. […] The best treatment of this disease is RAAS inhibitors with angiotensin-converting enzyme (ACE) inhibitors or angiotensin II receptor blockers (ARBs). […] Therefore, early and effective treatment of hypertension is very important to decrease the morbidity and mortality of ADPKD patients. […] Improvements in screening and diagnosis of ADPKD have allowed earlier diagnosis of disease, later onset of ESKD and better survival. […] However, the main and most effective therapy remains control of hypertension. […] Tolvaptan, a V2 receptor antagonist, was demonstrated to be effective in slowing deterioration of renal function and renal volume growth.

• #2 Get Polycystic Kidney Disease Treatment | Cleveland Clinic

  https://my.clevelandclinic.org/services/polycystic-kidney-disease-treatment

  Cleveland Clinics kidney disease providers will find out why you have polycystic kidney disease. And theyll work with you to develop a personalized treatment plan to help you manage this condition. […] As we build a treatment plan for your polycystic kidney disease, our expert team dives deep into testing to better understand your diagnosis. […] Your personalized treatment for polycystic kidney disease depends on your test results, health, symptoms and needs. There are different things your care team may recommend to treat and manage this condition, like: […] High blood pressure is a common symptom and complication of PKD. Its important to manage it and keep your blood pressure within a safe range. […] Complications that come with polycystic kidney disease can be painful. You may get kidney stones, infections or ruptured cysts.

• #2 Polycystic Kidney Disease Treatment & Management: Approach Considerations, Blood Pressure Control, Infectious and Other Disorders

  https://emedicine.medscape.com/article/244907-treatment

  The two-year phase II TAME-PKD study, conducted in 97 patients with early-stage ADPKD, found that long-term use of metformin is safe and tolerable. […] A meta-analysis of 10 randomized controlled trials evaluating somatostatin analogs concluded that the use of somatostatin analogs slows increases in total liver volume (TLV) and lower total kidney volume (TKV) but does not affect eGFR. […] Many trials have been completed or are under way to investigate different treatments, including the use of statins, vitamin B3 or niacinamide, antiproliferative drugs, and somatostatin analogues. […] The target blood pressure is 110/75 mm Hg in patients aged 18-50 years who have an eGFR 60 mL/min. […] Achieving good blood pressure control helps slow the progression of kidney disease, and the importance of early detection and treatment of hypertension in ADPKD has been demonstrated in several studies.

• #2 Drug management of autosomal dominant polycystic kidney disease

  https://australianprescriber.tg.org.au/articles/drug-management-of-autosomal-dominant-polycystic-kidney-disease.html

  The first-line drug classes for treating hypertension are blockers of the renin-angiotensin-aldosterone system (either ACE inhibitors or angiotensin receptor antagonists). […] In patients with early-stage disease (eGFR 60 mL/min/1.73 m2), the recommended blood pressure target is between 120/70 mmHg and 130/80 mmHg. […] Tolvaptan is a specific oral vasopressin type 2 receptor antagonist and is indicated in patients with ADPKD at high risk of developing kidney failure. […] The main adverse effect of tolvaptan is massive aquaresis (mean urine volume of 57 L/day) due to the off-target suppression of vasopressin-mediated water reabsorption in the collecting duct. […] Chronic pain is a common and overlooked clinical problem in ADPKD. Recognising pain and providing effective pharmacological management can significantly improve the well-being of people with ADPKD.

• #2 Patient education: Polycystic kidney disease (Beyond the Basics) – UpToDate

  https://www.uptodate.com/contents/polycystic-kidney-disease-beyond-the-basics/print

  Angiotensin-converting enzyme (ACE) inhibitors and angiotensin receptor blockers (ARBs) can effectively lower blood pressure in most people with ADPKD. […] Low-salt diet — The amount of salt (sodium) ingested on a daily basis has been linked to more rapid progression of kidney disease in patients with ADPKD. It can also raise blood pressure levels. For those reasons, a sodium intake of <2 g (2000 mg) per day is recommended. [...] Weight control — Patients who are overweight (a body mass index [BMI] of 25 to 30) and patients with obesity (BMI greater than 30) have faster progression of kidney disease in ADPKD. Thus, we recommend keeping a healthy BMI (<25). [...] Dietary protein restriction — There are conflicting findings on the benefit of a low-protein diet in people with ADPKD. Given the limited evidence of benefit, we do not recommend restricting protein intake below 0.8 g/kg of body weight per day. In this example, a 180 pound (82 kg) man would need approximately 66 grams of protein per day.

• #2 Polycystic kidney disease – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/polycystic-kidney-disease/diagnosis-treatment/drc-20352825

  How bad polycystic kidney disease is varies from person to person. That’s true even among people in the same family. Often, people with PKD reach end-stage kidney disease between ages 55 and 65. But some people with PKD have mild disease. They might never get to end-stage kidney disease. […] Treating polycystic kidney disease involves dealing with the following symptoms and complications in their early stages: […] The medicine tolvaptan (Jynarque, Samsca) may be used for adults at risk of ADPKD that’s getting worse fast. Tolvaptan is a pill that you swallow that slows how fast kidney cysts grow. It also slows the decline in how well your kidneys work. […] Keeping high blood pressure under control can slow the disease and kidney damage. […] To help your kidneys stay as healthy as possible for as long as possible, experts suggest being at a healthy weight and body mass index. Drinking water and fluids throughout the day may help slow the growth of kidney cysts. This could slow the loss of kidney function.

• #2 Polycystic Kidney Disease: Symptoms, Diagnosis, and Treatment

  https://www.healthline.com/health/polycystic-kidney-disease

  The goal of PKD treatment is to manage symptoms and avoid complications. Controlling high blood pressure is the most important part of treatment. […] Some treatment options may include: pain medication, except ibuprofen (Advil), which isn’t recommended since it may worsen kidney disease; blood pressure medication; antibiotics to treat UTIs; a low-sodium diet; diuretics to help remove excess fluid from the body; surgery to drain cysts and help relieve discomfort. […] In 2018, the Food and Drug Administration approved a drug called tolvaptan (brand name Jynarque) as a treatment for ADPKD. It’s used to slow the progression of kidney decline. […] With advanced PKD that causes renal failure, dialysis and kidney transplant may be necessary. One or both of the kidneys may need to be removed.

• #2 Polycystic Kidney Disease (PKD) Symptoms, Treatments & Causes – American Kidney Fund (AKF)

  https://www.kidneyfund.org/all-about-kidneys/types-kidney-diseases/polycystic-kidney-disease

  Your doctor might suggest simple things like using heat or ice, getting massages, or taking medicine like acetaminophen, and if those don’t work, there are other treatments they can try to help you manage your pain. […] For long-lasting pain, your doctor might recommend stronger medicines like opioids or gabapentin, which can help with nerve pain. […] In some cases, if the pain is really bad and won’t go away, your doctor might talk to you about special procedures like nerve blocks or even surgery to remove cysts. […] Doctors usually use two classes of medicines, ACE inhibitors and ARBs, to help lower your blood pressure. […] A new medicine, Tolvaptan, was approved by the FDA that slows the growth of cysts for people with ADPKD and helps the kidneys continue to function, delaying the need for dialysis or transplant. […] You can also ask your doctor if there are any clinical trials studying treatments for ADPKD that you could take part in.

• #2

  https://www.nhs.uk/conditions/autosomal-dominant-polycystic-kidney-disease-adpkd/treatment/

  Occasionally, large cysts can be drained to help relieve the pain caused by the increased pressure. […] Urinary tract infections (UTIs) can often be treated with a 7- to 14-day course of antibiotic tablets. […] If the infection persists despite antibiotic treatment, the infected cysts may need to be drained during surgery or using a needle inserted through your skin. […] The 2 main treatment options for kidney failure are: dialysis, where a machine replicates some of your kidneys’ functions; a kidney transplant, where a healthy kidney is removed from a living or recently deceased donor and implanted into someone with kidney failure. […] Tolvaptan is a medication that’s recommended by the National Institute for Health and Care Excellence (NICE) to treat ADPKD in adults. […] It can be used to slow down the growth of cysts, reducing overall kidney growth and preserving kidney function for longer. […] Tolvaptan comes in tablet form and is taken twice a day as a split dose.

• #2 Polycystic kidney disease – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/polycystic-kidney-disease/diagnosis-treatment/drc-20352825

  You might be able to control the pain of polycystic kidney disease with medicines available without a prescription, such as acetaminophen (Tylenol, others). […] Treating infections quickly with antibiotics can help prevent kidney damage. […] Your kidneys can stop removing waste and extra fluids from your blood. Then you need either dialysis or a kidney transplant. […] Early treatment offers the best chance of slowing the progress of polycystic kidney disease.

• #2 Autosomal Dominant Polycystic Kidney Disease (ADPKD) – Genitourinary Disorders – Merck Manual Professional Edition

  https://www.merckmanuals.com/professional/genitourinary-disorders/cystic-kidney-disease/autosomal-dominant-polycystic-kidney-disease-adpkd

  Hemodialysis, peritoneal dialysis, or kidney transplantation is required in patients who develop chronic renal failure. ADPKD does not recur in grafts. […] Tolvaptan, a vasopressin receptor 2 antagonist, is a medication that may benefit patients with ADPKD. […] Tolvaptan appears to slow increase in renal volume and decline in renal function, but it can cause adverse effects due to free water diuresis (eg, thirst, polydipsia, polyuria) that can make adherence difficult. […] In children with ADPKD, early use of pravastatin may slow the progression of structural kidney disease.

• #2

  https://healthmatch.io/kidney-disease/polycystic-kidney-disease-treatment

  Medications known as angiotensin II receptor blockers (ARBs) or angiotensin-converting enzymes (ACE) inhibitors help control high blood pressure, which delays the progression of the disease. In addition to the medications, having a healthy balanced diet and more active hours may help control your blood pressure. […] Over-the-counter medications containing acetaminophen may help you control the pain caused by polycystic kidney disease. However, if the pain is unbearable, the doctor may recommend shrinking your kidneys using a needle to draw out cyst fluid and inject the medicine. […] If your kidneys stop working, the next step is likely to be dialysis or a kidney transplant. This will help return your kidney function to near normal levels but will require intense immunosuppression. […] Polycystic kidney disease is not preventable but is manageable through treatment options and lifestyle changes. This will slow down the progression of further kidney damage, preventing kidney failure.

• #2 Polycystic Kidney Disease Treatments | Advances in Treating Kidney Disease

  https://resources.healthgrades.com/right-care/kidney-disease/new-treatments-for-rapidly-progressing-polycystic-kidney-disease

  Lixivaptan: Like tolvaptan, this medication blocks vasopressin, but it’s being studied to see if it’s safer on your liver. […] Water therapy: Large amounts of water appear to suppress a pathway in your body that causes fluids to build up and cysts to grow. Researchers are trying to determine if drinking increased amounts of water can slow polycystic kidney disease progression. […] Metformin: This drug has long been used to treat type 2 diabetes, so it has an established safety profile. Early studies in mice indicate that it may also slow the growth of cysts in your kidneys. […] Tesevatinib: This new medication is being researched as a treatment for cancer as well as PKD. It stops a protein called epidermal growth factor (EGFR) that causes cysts to become bigger. […] RGLS4326: This treatment can specifically target your kidneys. Studies on animals show decreased development of new kidney cysts, slower growth of existing cysts, and preserved kidney function. […] These are just a few of the PKD treatments being explored. It can be anticipated that more drugs and therapies will emerge for investigation.

• #2 Evaluating Treatments for Autosomal Dominant PKD

  https://www.uspharmacist.com/article/evaluating-treatments-for-autosomal-dominant-pkd

  Metformin has been shown to impact the production of cysts and decrease the decline in glomerular filtration rates in patients with PKD. […] The primary endpoint showed that metformin at clinically relevant doses for type 2 diabetes (2,000 mg/d) is safe in patients in the early stages of ADPKD (eGFR 60 mL/min per 1.73 m2). […] If metformin is approved by the FDA in the treatment of ADPKD, it will be the most affordable option on the market helping to reduce social determinants of health (SDOH) related barriers, contributing to better health equity, access, and outcomes. […] The drugs discussed can have an impact on reducing the decline in kidney function in both CKD and ADPKD, therefore delaying ESRD. […] Available therapies to slow the loss of kidney function in this patient population are limited.

• #2 Treatment Options for Autosomal Dominant Polycystic Kidney Disease (ADPKD): What Works and What Does Not – AJKD Blog

  https://ajkdblog.org/2023/06/21/treatment-options-for-autosomal-dominant-polycystic-kidney-disease-adpkd-what-works-and-what-does-not/

  The mTOR pathway is central to cyst formation, so mTOR inhibitors were thought to be good options for treatment of ADPKD. Unfortunately, clinical trials failed to show any clinical evidence for their use. […] Metformin is another drug that can potentially slow eGFR decline. […] Somatostatin and its analogs (octreotide and lanreotide) may reduce kidney and liver cyst fluid accumulation among patients with PKD. However, these agents have not been shown to slow the progression of kidney function decline and produce a variety of undesirable adverse effects. […] Venglustat is a glucosylceramide synthase inhibitor that has been shown to inhibit cyst growth and reduce kidney failure in preclinical models of ADPKD. […] Several other drugs are currently under investigation to determine their efficacy in ADPKD patients.

• #2 A new drug candidate can shrink kidney cysts | MIT News | Massachusetts Institute of Technology

  https://news.mit.edu/2024/new-drug-candidate-can-shrink-kidney-cysts-0122

  In addition to restoring kidney function, the treatment also ameliorated other clinical features of ADPKD; biomarkers for tissue inflammation and fibrosis were decreased in the treated mice compared to the control animals. […] The results also suggest that in patients, treatment with 11beta compounds once every few months, or even once a year, could significantly delay disease progression, and thus avoid the need for continuous, burdensome antiproliferative therapies such as tolvaptan. […] Based on what we know about the cyst growth paradigm, you could in theory treat patients in a pulsatile manner once a year, or perhaps even less often and have a meaningful impact on total kidney volume and kidney function, Sorin Fedeles says. […] The researchers now hope to run further tests on 11beta-dipropyl, as well as develop ways to produce it on a larger scale. They also plan to explore related compounds that could be good drug candidates for PKD.

• #2 Targeting TRPM3 as a potential therapeutic approach for autosomal dominant polycystic kidney disease | Scientific Reports

  https://www.nature.com/articles/s41598-025-89200-z

  The central role of cAMP in the model is the basis of the clinical use of Tolvaptan, which represses vasopressin-induced rises in cAMP, as a licensed drug for treatment of ADPKD patients: the drug is not without its problems, which is why it is used only for highest risk patients. […] Our results show that the cyst-promoting effect of cAMP elevation on developing mouse kidneys is exacerbated by co-incubation with any of a range of TRPM3 inhibitors, but is attenuated by co-incubation with TRPM3 activators. […] Our aim, in this work, was to explore a possible therapy to block cyst initiation and progression. There is evidence from mouse models that reinstatement of PC1 and PC2 expression, after their suppression caused development of early ADPKD, reverses the disease at least at a gross phenotypic level. This raises the possibility that imposing control over the cystogenic phenotype by TRPM3 activators may be useful even in patients in whom the disease has begun.

• #2 Autosomal Dominant Polycystic Kidney Disease – PYC Therapeutics

  https://pyctx.com/autosomal-dominant-polycystic-kidney-disease/

  PYC-003 has disease-modifying potential to halt cellular changes and arrest disease progression – it’s a potentially life-changing therapeutic. […] In pre-clinical testing, high concentrations of PYC-003 reached target kidney cells affected by ADPKD, and PYC-003 increased PC1 protein production by approximately 50% three days after a single dose. […] Visible reductions in cyst size and frequency were observed in PYC-003-treated patient cells compared to untreated cells – demonstrating the potential efficacy of PYC-003 for ADPKD patients.

• #2 Polycystic Kidney Disease Care | UC San Diego Health

  https://health.ucsd.edu/care/kidney/polycystic/

  Medical therapies for slowing the growth of cysts in the kidney […] Medications to relieve pain from cysts, kidney stones and other related complications […] Blood pressure management with medicine, nutritional counseling, smoking cessation strategies, exercise and weight management help […] Management of cyst-related symptoms, including interventions aimed at reducing cyst size […] Preparation for dialysis and kidney transplantation […] Genetic counseling, including preimplantation genetic testing during in vitro fertilization (IVF) and high-risk obstetric care […] Urologic management of enlarged kidneys and kidney stone disease […] Neurosurgical management of vascular aneurysms and blood vessel disease […] UC San Diego Health nephrologists are actively researching ways to improve treatment options for polycystic kidney disease. By joining a clinical trial, you may receive a new kidney treatment before it is available to the public.

• #2 Polycystic Kidney Disease Clinic – Nephrology | Loma Linda University Health

  https://lluh.org/services/nephrology/polycystic-kidney-disease-clinic

  We prioritize patient safety, comfort, and well-being, ensuring every aspect of care is fully managed and supported. […] Our clinic primarily focuses on diagnosing, managing, and treating PKD and related conditions, including: Autosomal dominant polycystic kidney disease, Chronic kidney disease (CKD), High blood pressure (hypertension), Urinary tract infections (UTIs), Kidney stones, Hematuria (blood in urine), Liver cysts, Pain management, End-stage renal disease. […] Comprehensive evaluation, including imaging studies such as ultrasounds, MRIs, and CT scans to monitor kidney cyst growth and function. […] Assistance in understanding the genetic aspects of PKD, predicting risks for family members, and making informed decisions about family planning. […] Monitoring and treatment plans for controlling hypertension, a common complication in PKD patients.

• #2 Kidney disease: Research may pave the way for gene therapy for ADPKD

  https://www.medicalnewstoday.com/articles/research-suggests-avenues-toward-gene-therapies-for-polycystic-kidney-disease

  Dr. Diane Triolo, a nephrologist at Holy Name Medical Center in Teaneck, NJ, told Medical News Today that the latest therapies for treatment include slowing the progression of the cysts but unfortunately have not shown to prevent them from forming which ultimately these cysts lead to end-stage renal disease. […] [The present study provides] nephrologists and their patients hope for a potential cure rather than delay to end-stage renal disease in ADPKD. They have shown that expressing the C-terminal fragment of PC1 through interaction with Nicotinamide Nucleotide Transhydrogemase can suppress these cysts and preserve renal function in mouse tissue. This gives hope to millions of people and shows how important gene therapy is not only for ADPKD but for many other diseases as well.

• #3 Patient education: Polycystic kidney disease (Beyond the Basics) – UpToDate

  https://www.uptodate.com/contents/polycystic-kidney-disease-beyond-the-basics/print

  Tolvaptan — Tolvaptan is a type of drug called a vasopressin receptor antagonist. It has been shown to slow the decline in kidney function and reduce pain symptoms in people with ADPKD. The use of tolvaptan should be considered in patients who are at risk of rapid progression based on their kidney size for a given age and on their kidney function. Even though tolvaptan does not cure or reverse PKD, it may increase the kidney life span by seven to eight years if it is started early in life. Tolvaptan can cause side effects including liver problems (in approximately 4 percent of people), so frequent monitoring of the liver function tests (by blood draws) is needed. Most often, patients experience frequent urination, urination at night, and increased thirst. […] End-stage kidney disease — Patients with ADPKD who progress to end-stage kidney disease require either dialysis or kidney transplantation.

• #3 Polycystic Kidney Disease Treatment & Management: Approach Considerations, Blood Pressure Control, Infectious and Other Disorders

  https://emedicine.medscape.com/article/244907-treatment

  The drugs of choice for this condition are angiotensin-converting enzyme (ACE) inhibitors or angiotensin II receptor blockers (ARBs). […] Treatment of nephrolithiasis in patients with ADPKD is the same as in those without ADPKD: high fluid intake and potassium citrate in uric acid lithiasis, hypocitric calcium oxalate nephrolithiasis, and distal acidification defects. […] If infected kidney or hepatic cysts do not respond to conventional antibiotic therapy, surgical drainage may be necessary. […] Surgical treatment of a ruptured aneurysm involves clipping it at its neck.

• #3 Pediatric Polycystic Kidney Disease Treatment & Management: Approach Considerations, Long-Term Monitoring

  https://emedicine.medscape.com/article/983281-treatment

  Renal insufficiency is less common in children with ADPKD than in those with the recessive form, but hemodialysis or peritoneal dialysis or transplantation may be required, as in patients with ARPKD. […] The primary care physician and consulting nephrologist should participate in the care of children and adults with polycystic kidney disease. Once polycystic kidney disease is diagnosed, the frequency of outpatient follow-up with the nephrologist depends on the degree of renal dysfunction and on complicating features, such as a failure to thrive, nutritional and feeding difficulties, hypertension, electrolyte disturbances, urinary infections, and hepatic fibrosis (ie, portal hypertension). […] In addition to the significant medical problems, the psychosocial stress on the patient and family can be overwhelming. A team approach in which the skills of the nephrologist are used together with those of other medical specialists (eg, gastroenterologist), specialized nurses, nutritionists, social workers, psychiatrists, and other support staff provides optimal comprehensive care.

• #3 Polycystic kidney disease (Causes, Symptoms and Treatment)

  https://patient.info/doctor/autosomal-dominant-polycystic-kidney-disease

  If blood pressure and blood chemistry are normal then annual blood tests and ultrasound are sufficient to monitor the disease. Early detection of hypertension is important – ambulatory blood pressure monitoring in young adults or children can reveal raised blood pressure not detected with the one-off blood pressure reading. Remember that progressive kidney disease will occur over time but serum creatinine concentrations are insensitive measures, only rising once more than 50% of renal parenchyma has been destroyed, and eGFR typically remains within normal range for several decades. Ultrasound is a more sensitive means of monitoring progression, as it enables estimate of renal volume. […] Current advice is that blood pressure should be controlled to a target of 130/80 mm Hg. Ongoing research is investigating whether a lower target (110/75 mm Hg) is effective at slowing disease progression in those with preserved function. Where there is significant proteinuria (1 g/day), a lower target blood pressure is recommended. Angiotensin-converting enzyme (ACE) inhibitors or angiotensin-II receptor antagonists are the preferred choice (based on limited observational data) but other agents (eg, diuretics) may also be required. Monitor blood chemistry closely if kidneys are failing and these drugs are used.

• #3 Polycystic Kidney Disease Clinic – Nephrology | Loma Linda University Health

  https://lluh.org/services/nephrology/polycystic-kidney-disease-clinic

  Addressing and alleviating pain caused by growing cysts or related complications through various therapeutic methods. […] Guidance on maintaining a kidney-friendly diet to help manage and possibly slow down the progression of PKD. […] Access to research studies on the latest treatments and therapies for PKD. […] Procedures such as cyst aspiration or sclerotherapy to manage and alleviate symptoms related to large kidney cysts. […] Guidance and management for patients progressing toward end-stage renal disease, including evaluations for potential kidney transplantation. […] Emotional and psychological support through group sessions or individual counseling for patients and their families coping with PKD. […] Reviewing and prescribing medications to control symptoms, manage complications, and possibly slow down the disease progression.

• #3 Treatment Options for Autosomal Dominant Polycystic Kidney Disease (ADPKD): What Works and What Does Not – AJKD Blog

  https://ajkdblog.org/2023/06/21/treatment-options-for-autosomal-dominant-polycystic-kidney-disease-adpkd-what-works-and-what-does-not/

  Major research efforts in both the clinical and preclinical setting in the last two decades resulted in a number of pivotal clinical trials aimed to address ADPKD. Several medications have been suggested to alter the course of kidney disease progression. However, the only medication that was proven effective in slowing kidney function decline has been Tolvaptan.

• #4 Polycystic kidney disease (Causes, Symptoms and Treatment)

  https://patient.info/doctor/autosomal-dominant-polycystic-kidney-disease

  Treat UTIs as they occur, as for the general population. Women are more susceptible and more prone to parenchymal and cyst infections. Acute pyelonephritis and symptomatic cyst infection require admission for intravenous antibiotic therapy. Where fever persists beyond 1-2 weeks of appropriate antibiotic therapy, percutaneous or surgical drainage of infected cysts should be considered. Where there is end-stage disease, nephrectomy may be indicated. […] Treat renal colic with adequate analgesia. Bed rest and hydration (sufficient to increase the urinary flow rate to 2-3 litres/day) are recommended. Patients should become familiar with self-management for straightforward repeated episodes. Avoid anticoagulants (including low-dose aspirin) unless there is a strong indication. Renal stones are managed with the same strategies as in the general population.

• #4 Patient education: Polycystic kidney disease (Beyond the Basics) – UpToDate

  https://www.uptodate.com/contents/polycystic-kidney-disease-beyond-the-basics/print

  People with ADPKD who require dialysis are usually treated with hemodialysis. People with ADPKD who have hemodialysis appear to survive longer than people with other types of end-stage kidney disease. […] Peritoneal dialysis, a form of dialysis that involves infusing fluid into the abdomen and then draining the fluid, is less commonly performed due to the presence of the enlarged kidneys but can be considered on an individual basis. […] The prognosis after kidney transplantation is usually excellent.

• #5 Polycystic kidney disease (Causes, Symptoms and Treatment)

  https://patient.info/doctor/autosomal-dominant-polycystic-kidney-disease

  Treat the cause where possible – eg, renal stones, infection. Avoid long-term use of nephrotoxic drugs – eg, non-steroidal anti-inflammatory drugs (NSAIDs). Chronic, unrelenting pain may require the involvement of a pain management specialist. Different strategies have been tried, including transcutaneous stimulation, use of local injections of anaesthetic and renal denervation. Depending upon size and accessibility, large, painful cysts of kidneys or other organs may require percutaneous drainage guided by ultrasound, a laparoscopic technique or a more formal operation at laparotomy. Enormous cysts in excess of 40 cm diameter usually require nephrectomy. Renal cell carcinoma is a rare cause of pain in ADPKD. It is not more common than in the general population but tends to present at an earlier age.

• #6 Polycystic kidney disease (Causes, Symptoms and Treatment)

  https://patient.info/doctor/autosomal-dominant-polycystic-kidney-disease

  As end-stage kidney disease is reached, dialysis is required. Abdominal hernias (common in ADPKD) can complicate peritoneal dialysis. Heparin anticoagulation for haemodialysis can be problematic in those with haematuria. Renal transplantation has similar outcomes for patients with ADPKD as for those without the disease. It is very important to exclude affected relatives as living donors (usually with MRI +/- gene testing). […] Oestrogens appear to provoke the growth of hepatic cysts, so avoid their use in women with ADPKD or, if prescribed, use in the lowest effective dose or with transdermal administration. Pregnancies should be overseen by an obstetric physician. Those with pre-conceptual hypertension or renal insufficiency are at higher risk of severe hypertension or pre-eclampsia. Normotensive women generally have uncomplicated pregnancies.

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