Materiały źródłowe

• #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 […] No drug references linked in this topic. […] The course and disease-modifying treatment of ADPKD in adults are discussed here. The majority of individuals with PKD eventually require kidney replacement therapy. […] The identification of individuals who are at high risk for progression of chronic kidney disease (CKD) is important for prognostic reasons and to identify patients who may benefit from specific therapies, such as tolvaptan. […] Our preferred method for identifying high-risk patients with ADPKD is the Mayo classification system, which categorizes patients into five prognostic classes from the lowest to the highest risk for disease progression (classes 1A, 1B, 1C, 1D, and 1E). Classes 1C, 1D, and 1E are defined as high risk for progression to end-stage kidney disease (ESKD).

• #2 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. For a long time, the treatment and management strategies of this disease have not progressed in comparison with the treatment of other kidney diseases. 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. […] 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.

• #3

  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. […] The plan will also include what you’d want to do if your kidneys stop working sufficiently (kidney failure). […] 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).

• #4

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

  If you need to take a painkiller, paracetamol is the best medication to try first. […] Antidepressants or anticonvulsants, which are normally used to treat epilepsy, may be prescribed for long-term (chronic) pain. These medications can also be used to relieve some types of pain. […] 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. […] If you’re taking tolvaptan, you’ll need to initially be monitored frequently through hospital clinics.

• #5

  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. […] The plan will also include what you’d want to do if your kidneys stop working sufficiently (kidney failure). […] 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).

• #6 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 […] No drug references linked in this topic. […] The course and disease-modifying treatment of ADPKD in adults are discussed here. The majority of individuals with PKD eventually require kidney replacement therapy. […] The identification of individuals who are at high risk for progression of chronic kidney disease (CKD) is important for prognostic reasons and to identify patients who may benefit from specific therapies, such as tolvaptan. […] Our preferred method for identifying high-risk patients with ADPKD is the Mayo classification system, which categorizes patients into five prognostic classes from the lowest to the highest risk for disease progression (classes 1A, 1B, 1C, 1D, and 1E). Classes 1C, 1D, and 1E are defined as high risk for progression to end-stage kidney disease (ESKD).

• #7 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 […] No drug references linked in this topic. […] The course and disease-modifying treatment of ADPKD in adults are discussed here. The majority of individuals with PKD eventually require kidney replacement therapy. […] The identification of individuals who are at high risk for progression of chronic kidney disease (CKD) is important for prognostic reasons and to identify patients who may benefit from specific therapies, such as tolvaptan. […] Our preferred method for identifying high-risk patients with ADPKD is the Mayo classification system, which categorizes patients into five prognostic classes from the lowest to the highest risk for disease progression (classes 1A, 1B, 1C, 1D, and 1E). Classes 1C, 1D, and 1E are defined as high risk for progression to end-stage kidney disease (ESKD).

• #8 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 […] No drug references linked in this topic. […] The course and disease-modifying treatment of ADPKD in adults are discussed here. The majority of individuals with PKD eventually require kidney replacement therapy. […] The identification of individuals who are at high risk for progression of chronic kidney disease (CKD) is important for prognostic reasons and to identify patients who may benefit from specific therapies, such as tolvaptan. […] Our preferred method for identifying high-risk patients with ADPKD is the Mayo classification system, which categorizes patients into five prognostic classes from the lowest to the highest risk for disease progression (classes 1A, 1B, 1C, 1D, and 1E). Classes 1C, 1D, and 1E are defined as high risk for progression to end-stage kidney disease (ESKD).

• #9 Autosomal dominant polycystic kidney disease | Radiology Reference Article | Radiopaedia.org

  https://radiopaedia.org/articles/autosomal-dominant-polycystic-kidney-disease-1?lang=us

  Pharmacological management options are limited but include tolvaptan and angiotensin-converting enzyme (ACE) inhibitors or angiotensin receptor blockers (ARBs). […] Ultimately, ESRF requiring transplant or dialysis eventually develops in many patients (45% by the age of 60). Patients with PKD1 mutations are more likely to progress to ESRF and often do so at an earlier age. Patients with a larger height-adjusted total kidney volume are at greater risk of ESRF and may benefit from vasopressin receptor antagonists which slow cyst expansion. […] The Mayo Clinic Imaging Classification uses size and morphology to predict disease course. Radiomics and textural analysis show promise in predicting decline.

• #10 Are you ready to accompany autosomal dominant polycystic kidney disease patients in their treatment journey? Real practice for selecting rapid progressors and treatment with tolvaptan

  https://www.kosinmedj.org/journal/view.php?number=1242

  This paper will review parameters that can help predict rapid progression to find a rapid ADPKD progressor that can serve as the target of tolvaptan therapy. […] The best predictor of rapid progression is Mayo imaging classification (MIC). […] Patients with more advanced classes of ADPKD may be at higher risk for these complications. […] Therefore, patients in classes D and E are the most active targets for treatment as rapid progressors. […] The ellipsoid method can be used to calculate TKV from manual measurements of length, width, and depth from magnetic resonance imaging or computed tomography scans. […] The TKV growth rate, or the measured rapid growth rate of TKV, can be used as evidence of rapid progression, and it refers to cases where the annual growth rate is 5% or more.

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

  https://pkdcure.org/about-the-disease/living-with-pkd/treatments/

  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. […] 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.

• #12 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.

• #13

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

  If you need to take a painkiller, paracetamol is the best medication to try first. […] Antidepressants or anticonvulsants, which are normally used to treat epilepsy, may be prescribed for long-term (chronic) pain. These medications can also be used to relieve some types of pain. […] 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. […] If you’re taking tolvaptan, you’ll need to initially be monitored frequently through hospital clinics.

• #14 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. […] The best treatment of this disease is RAAS inhibitors with angiotensin-converting enzyme (ACE) inhibitors or angiotensin II receptor blockers (ARBs). These agents remain the most recommended drugs to treat hypertension in patients with ADPKD. […] Therefore, early and effective treatment of hypertension is very important to decrease the morbidity and mortality of ADPKD patients. […] Tolvaptan, a V2 receptor antagonist, was demonstrated to be effective in slowing deterioration of renal function and renal volume growth.

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

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

  Tolvaptan is a vasopressin V2-receptor (V2R) antagonist with proven beneficial results in ADPKD. […] Unless contraindications are present or cost is prohibitive, we prescribe tolvaptan to adult patients with ADPKD who have an estimated glomerular filtration rate (eGFR) ≥25 mL/min1.73 m² and who are at risk of rapid progression. […] Tolvaptan dosing and monitoring is presented below. […] The decision to give tolvaptan to individual ADPKD patients should be made after careful assessment of risks (liver toxicity, polyuria, polydipsia), benefits, and affordability, on an individual basis. […] Tolvaptan should not be used in patients with liver impairment or injury (except for uncomplicated polycystic liver disease). […] 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.

• #16 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: Hypertension; rigorous blood pressure control is recommended in early ADPKD. 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. The two-year phase II TAME-PKD study 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 and lower total kidney volume 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. 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. 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.

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

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

  Tolvaptan is a vasopressin V2-receptor (V2R) antagonist with proven beneficial results in ADPKD. […] Unless contraindications are present or cost is prohibitive, we prescribe tolvaptan to adult patients with ADPKD who have an estimated glomerular filtration rate (eGFR) ≥25 mL/min1.73 m² and who are at risk of rapid progression. […] Tolvaptan dosing and monitoring is presented below. […] The decision to give tolvaptan to individual ADPKD patients should be made after careful assessment of risks (liver toxicity, polyuria, polydipsia), benefits, and affordability, on an individual basis. […] Tolvaptan should not be used in patients with liver impairment or injury (except for uncomplicated polycystic liver disease). […] 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.

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

  https://pkdcure.org/about-the-disease/living-with-pkd/treatments/

  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. […] 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. […] The PKD Foundation not only supported early studies that led to the development of tolvaptan as a treatment but also helped guide PKD patients to the clinical trials. […] Today is a historic day in providing hope to patients with polycystic kidney disease, and we are thrilled to be a part of this first milestone to treat patients with ADPKD, said Andy Betts, President and CEO of the PKD Foundation, when news of the approval was announced.

• #19 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. […] The best treatment of this disease is RAAS inhibitors with angiotensin-converting enzyme (ACE) inhibitors or angiotensin II receptor blockers (ARBs). These agents remain the most recommended drugs to treat hypertension in patients with ADPKD. […] Therefore, early and effective treatment of hypertension is very important to decrease the morbidity and mortality of ADPKD patients. […] Tolvaptan, a V2 receptor antagonist, was demonstrated to be effective in slowing deterioration of renal function and renal volume growth.

• #20 Kidney Research and Clinical Practice

  https://www.krcp-ksn.org/m/journal/view.php?number=6138

  Tolvaptan is currently recommended for ADPKD patients in the United States aged 18 to 55 years, at risk of rapid progression as determined by htTKV and risk of rapid GFR decline in the future (MIC 1C, 1D, 1E) with an eGFR greater than 25 mL/min. […] Most common adverse effects of tolvaptan resulting from vasopressin receptor blockade that patients should be advised of are polyuria, excessive thirst, polydipsia, and nocturia.

• #21

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

  If you need to take a painkiller, paracetamol is the best medication to try first. […] Antidepressants or anticonvulsants, which are normally used to treat epilepsy, may be prescribed for long-term (chronic) pain. These medications can also be used to relieve some types of pain. […] 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. […] If you’re taking tolvaptan, you’ll need to initially be monitored frequently through hospital clinics.

• #22

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

  If you need to take a painkiller, paracetamol is the best medication to try first. […] Antidepressants or anticonvulsants, which are normally used to treat epilepsy, may be prescribed for long-term (chronic) pain. These medications can also be used to relieve some types of pain. […] 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. […] If you’re taking tolvaptan, you’ll need to initially be monitored frequently through hospital clinics.

• #23 Are you ready to accompany autosomal dominant polycystic kidney disease patients in their treatment journey? Real practice for selecting rapid progressors and treatment with tolvaptan

  https://www.kosinmedj.org/journal/view.php?number=1242

  The current parameters or strategies used in rapid progressor selection are not perfect, an absolutely necessary point for patients who are not selected in the process of steps 1 and 2 is shared-decision medicine; that is, a process wherein the treatment process is determined together with the patient. […] There are plenty of reviews or guidelines on the actual prescription of tolvaptan. […] Patients who are highly sensitive to tolvaptan can start at 15 mg/15 mg or 30 mg/15 mg, then gradually increase the dose to 45 mg/15 mg, 60 mg/30 mg, or 90 mg/30 mg every 1 to 4 weeks. […] This may be meaningful for some patients and may not be meaningful for other patients. […] Most importantly, patients with significantly higher eGFR for their age should be excluded from treatment, while patients with significantly lower eGFR compared to their age should be included in the treatment category. […] For more patients to receive proper treatment, medical doctors, the rest of the medical community, and patients must join forces.

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

  https://pkdcure.org/about-the-disease/living-with-pkd/treatments/

  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. […] 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. […] The PKD Foundation not only supported early studies that led to the development of tolvaptan as a treatment but also helped guide PKD patients to the clinical trials. […] Today is a historic day in providing hope to patients with polycystic kidney disease, and we are thrilled to be a part of this first milestone to treat patients with ADPKD, said Andy Betts, President and CEO of the PKD Foundation, when news of the approval was announced.

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

  https://pkdcure.org/about-the-disease/living-with-pkd/treatments/

  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. […] 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. […] The PKD Foundation not only supported early studies that led to the development of tolvaptan as a treatment but also helped guide PKD patients to the clinical trials. […] Today is a historic day in providing hope to patients with polycystic kidney disease, and we are thrilled to be a part of this first milestone to treat patients with ADPKD, said Andy Betts, President and CEO of the PKD Foundation, when news of the approval was announced.

• #26 Kidney Research and Clinical Practice

  https://www.krcp-ksn.org/m/journal/view.php?number=6138

  Autosomal dominant polycystic kidney disease (ADPKD) is the reported etiology in 10% of end-stage kidney disease (ESKD) patients and has an estimated prevalence of 12.5 million cases worldwide across all ethnicities. […] There have been major advancements over the last two decades in understanding the pathogenesis and development of disease-modifying treatment options for ADPKD, culminating in regulatory approval of tolvaptan for ADPKD patients at risk of rapid progression to kidney failure. […] In 2018, the U.S. Food and Drug Administration approved tolvaptan, a vasopressin V2 receptor antagonist, as a disease-modifying treatment option for ADPKD patients at risk of rapid progression to kidney failure. […] Tolvaptan treatment was associated with a slower rate of increase in TKV (2.8% vs. 5.5%, p 0.001) as well as a slower decline in kidney function in the tolvaptan group compared to placebo.

• #27 Kidney Research and Clinical Practice

  https://www.krcp-ksn.org/m/journal/view.php?number=6138

  Tolvaptan is currently recommended for ADPKD patients in the United States aged 18 to 55 years, at risk of rapid progression as determined by htTKV and risk of rapid GFR decline in the future (MIC 1C, 1D, 1E) with an eGFR greater than 25 mL/min. […] Most common adverse effects of tolvaptan resulting from vasopressin receptor blockade that patients should be advised of are polyuria, excessive thirst, polydipsia, and nocturia.

• #28 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: Hypertension; rigorous blood pressure control is recommended in early ADPKD. 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. The two-year phase II TAME-PKD study 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 and lower total kidney volume 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. 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. 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.

• #29 ADPKD: Symptoms, Treatment, Screenings, Diagnosis, and More

  https://www.healthline.com/health/adpkd/everything-to-know-symptoms-treatments

  For example, Jynarque may cause excessive thirst, frequent urination, and in some cases, serious liver damage. There have been reports of acute liver failure requiring liver transplantation in individuals taking Jynarque. […] Other treatments that target specific symptoms of ADPKD may also cause side effects. To learn more about the potential side effects of different treatments, talk with your doctor.

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

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

  Tolvaptan is a vasopressin V2-receptor (V2R) antagonist with proven beneficial results in ADPKD. […] Unless contraindications are present or cost is prohibitive, we prescribe tolvaptan to adult patients with ADPKD who have an estimated glomerular filtration rate (eGFR) ≥25 mL/min1.73 m² and who are at risk of rapid progression. […] Tolvaptan dosing and monitoring is presented below. […] The decision to give tolvaptan to individual ADPKD patients should be made after careful assessment of risks (liver toxicity, polyuria, polydipsia), benefits, and affordability, on an individual basis. […] Tolvaptan should not be used in patients with liver impairment or injury (except for uncomplicated polycystic liver disease). […] 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.

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

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

  Tolvaptan is a vasopressin V2-receptor (V2R) antagonist with proven beneficial results in ADPKD. […] Unless contraindications are present or cost is prohibitive, we prescribe tolvaptan to adult patients with ADPKD who have an estimated glomerular filtration rate (eGFR) ≥25 mL/min1.73 m² and who are at risk of rapid progression. […] Tolvaptan dosing and monitoring is presented below. […] The decision to give tolvaptan to individual ADPKD patients should be made after careful assessment of risks (liver toxicity, polyuria, polydipsia), benefits, and affordability, on an individual basis. […] Tolvaptan should not be used in patients with liver impairment or injury (except for uncomplicated polycystic liver disease). […] 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.

• #32 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. […] The best treatment of this disease is RAAS inhibitors with angiotensin-converting enzyme (ACE) inhibitors or angiotensin II receptor blockers (ARBs). These agents remain the most recommended drugs to treat hypertension in patients with ADPKD. […] Therefore, early and effective treatment of hypertension is very important to decrease the morbidity and mortality of ADPKD patients. […] Tolvaptan, a V2 receptor antagonist, was demonstrated to be effective in slowing deterioration of renal function and renal volume growth.

• #33 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.

• #34

  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. […] The plan will also include what you’d want to do if your kidneys stop working sufficiently (kidney failure). […] 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).

• #35 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. […] The best treatment of this disease is RAAS inhibitors with angiotensin-converting enzyme (ACE) inhibitors or angiotensin II receptor blockers (ARBs). These agents remain the most recommended drugs to treat hypertension in patients with ADPKD. […] Therefore, early and effective treatment of hypertension is very important to decrease the morbidity and mortality of ADPKD patients. […] Tolvaptan, a V2 receptor antagonist, was demonstrated to be effective in slowing deterioration of renal function and renal volume growth.

• #36 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

  Control of complications (eg, hypertension, infection, renal failure) […] Supportive measures […] Vasopressin antagonism or suppression […] 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. Percutaneous aspiration of cysts may help relieve severe pain due to hemorrhage or compression but has no effect on long-term outcome. Nephrectomy is an option to relieve severe symptoms due to massive kidney enlargement (eg, pain, hematuria) or recurrent UTIs. […] Hemodialysis, peritoneal dialysis, or kidney transplantation is required in patients who develop chronic renal failure. ADPKD does not recur in grafts.

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

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

  In young, healthy individuals aged 18 to 50 years with estimated glomerular filtration rate (eGFR) >60 mL/min/1.73 m², we generally use an angiotensin-converting enzyme (ACE) inhibitor or an angiotensin receptor blocker (ARB) to target a blood pressure of <110/75 mmHg using home blood pressure measurements or daytime ambulatory blood pressure monitoring. [...] We advise all PKD patients to restrict dietary sodium, with a goal of 2 grams intake per day or less (approximately 5 grams of salt). [...] We advise patients with ADPKD to drink >3 L of fluid per day, unless the eGFR is <30 mL/min/1.73 m² or the patient is at risk for hyponatremia (eg, taking a thiazide diuretic). [...] Patients at high risk for progression may be identified based upon criteria described above. The major clinical challenge is to define the patients at risk for rapid progression who are the most likely to derive benefit from tolvaptan.

• #38 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 reninangiotensinaldosterone 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.

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

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

  In young, healthy individuals aged 18 to 50 years with estimated glomerular filtration rate (eGFR) >60 mL/min/1.73 m², we generally use an angiotensin-converting enzyme (ACE) inhibitor or an angiotensin receptor blocker (ARB) to target a blood pressure of <110/75 mmHg using home blood pressure measurements or daytime ambulatory blood pressure monitoring. [...] We advise all PKD patients to restrict dietary sodium, with a goal of 2 grams intake per day or less (approximately 5 grams of salt). [...] We advise patients with ADPKD to drink >3 L of fluid per day, unless the eGFR is <30 mL/min/1.73 m² or the patient is at risk for hyponatremia (eg, taking a thiazide diuretic). [...] Patients at high risk for progression may be identified based upon criteria described above. The major clinical challenge is to define the patients at risk for rapid progression who are the most likely to derive benefit from tolvaptan.

• #40 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. […] painkillers such as paracetamol for loin pains – but you may be advised to avoid non-steroidal anti-inflammatory drugs (NSAIDs) such as ibuprofen, naproxen and diclofenac which can reduce the blood supply to the kidneys. […] antibiotics (to treat any UTIs). […] a range of methods to break up and remove kidney stones. […] You may also be advised to: exercise regularly but avoid contact sports (such as rugby or football) which could cause cysts to rupture and bleed. […] eat a healthy balanced low-salt diet.

• #41 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.

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

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

  In young, healthy individuals aged 18 to 50 years with estimated glomerular filtration rate (eGFR) >60 mL/min/1.73 m², we generally use an angiotensin-converting enzyme (ACE) inhibitor or an angiotensin receptor blocker (ARB) to target a blood pressure of <110/75 mmHg using home blood pressure measurements or daytime ambulatory blood pressure monitoring. [...] We advise all PKD patients to restrict dietary sodium, with a goal of 2 grams intake per day or less (approximately 5 grams of salt). [...] We advise patients with ADPKD to drink >3 L of fluid per day, unless the eGFR is <30 mL/min/1.73 m² or the patient is at risk for hyponatremia (eg, taking a thiazide diuretic). [...] Patients at high risk for progression may be identified based upon criteria described above. The major clinical challenge is to define the patients at risk for rapid progression who are the most likely to derive benefit from tolvaptan.

• #43 Treatment of Polycystic Kidney Disease

  https://www.verywellhealth.com/treatment-for-polycystic-kidney-disease-4150162

  When a patient learns about their diagnosis of PKD, the first question that comes up is whether it is treatable. […] Understanding the role of ADH helps in understanding the treatment options available and why they might work, from increased water intake to cutting-edge drugs. […] Increased water intake: As simple as this sounds, drinking water is an effective way to keep ADH levels down. […] Managing complications: In the absence of other specific treatments currently available, we are limited to managing the complications of PKD. […] Our understanding of ADH’s role in the worsening of PKD has led to promising research that could offer more concrete treatment options beyond the „Band-Aid” interventions described above. […] Tolvaptan: This medication, originally approved for treatment of low sodium levels, acts by blocking the site (called the V2 receptor) to which ADH would normally attach in the kidney. […] Octreotide: This is a long-acting synthetic version of a hormone called somatostatin. […] Although both of these agents have shown promise so far (in addition to other contenders like mTOR inhibitors and other drugs in clinical trials), cost is a major concern.

• #44

  https://journals.lww.com/kidney360/fulltext/2024/05000/feasibility_of_water_therapy_for_slowing_autosomal.11.aspx

  Water therapy in autosomal dominant polycystic kidney disease (ADPKD) reduces urine osmolality and serum copeptin level, a marker of vasopressin activity. […] Water therapy reduces the ADPKD kidney growth rate indicating it is slowing disease progression. […] Patients with ADPKD are less likely to report pain on water therapy. […] HWI in patients at risk of rapid progression of ADPKD slowed the rate of ht-TKV growth and reduced pain. This suggests that suppressing vasopressin levels by HWI provides an effective nonpharmacologic treatment of ADPKD. […] Our data suggest that HWI may be a treatment option. For patients with ADPKD at high risk of rapid progression, we found that HWI that was sufficient to decrease 24-hour Uosm and serum copeptin levels also decreased the rate of increase in ht-TKV, a biomarker of disease severity and risk of ESKD. Additional clinical trials of HWI on the progression of ADPKD are warranted.

• #45 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.

• #46 Autosomal Dominant Polycystic Kidney Disease: Symptoms, Diagnosis, and Treatment

  https://www.webmd.com/digestive-disorders/autosomal-dominant-polycystic-kidney-disease

  Eat right. Stick to a healthy, well-balanced diet that’s low in calories. Try to limit salt, because it can raise your blood pressure. […] Stay active. Exercise can help control your weight and blood pressure. Just avoid any contact sports where you might injure your kidneys. […] Don’t smoke. If you smoke, get help from your doctor to quit. Smoking damages the blood vessels in the kidneys, and it may create more cysts. […] Drink plenty of water. Dehydration may cause you to have more cysts.

• #47 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.

• #48 Autosomal Dominant Polycystic Kidney Disease: Symptoms, Diagnosis, and Treatment

  https://www.webmd.com/digestive-disorders/autosomal-dominant-polycystic-kidney-disease

  Eat right. Stick to a healthy, well-balanced diet that’s low in calories. Try to limit salt, because it can raise your blood pressure. […] Stay active. Exercise can help control your weight and blood pressure. Just avoid any contact sports where you might injure your kidneys. […] Don’t smoke. If you smoke, get help from your doctor to quit. Smoking damages the blood vessels in the kidneys, and it may create more cysts. […] Drink plenty of water. Dehydration may cause you to have more cysts.

• #49

  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. […] The plan will also include what you’d want to do if your kidneys stop working sufficiently (kidney failure). […] 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).

• #50

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

  If you need to take a painkiller, paracetamol is the best medication to try first. […] Antidepressants or anticonvulsants, which are normally used to treat epilepsy, may be prescribed for long-term (chronic) pain. These medications can also be used to relieve some types of pain. […] 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. […] If you’re taking tolvaptan, you’ll need to initially be monitored frequently through hospital clinics.

• #51 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.

• #52

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

  If you need to take a painkiller, paracetamol is the best medication to try first. […] Antidepressants or anticonvulsants, which are normally used to treat epilepsy, may be prescribed for long-term (chronic) pain. These medications can also be used to relieve some types of pain. […] 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. […] If you’re taking tolvaptan, you’ll need to initially be monitored frequently through hospital clinics.

• #53 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. […] painkillers such as paracetamol for loin pains – but you may be advised to avoid non-steroidal anti-inflammatory drugs (NSAIDs) such as ibuprofen, naproxen and diclofenac which can reduce the blood supply to the kidneys. […] antibiotics (to treat any UTIs). […] a range of methods to break up and remove kidney stones. […] You may also be advised to: exercise regularly but avoid contact sports (such as rugby or football) which could cause cysts to rupture and bleed. […] eat a healthy balanced low-salt diet.

• #54 Autosomal Dominant Polycystic Kidney Disease | AAFP

  https://www.aafp.org/pubs/afp/issues/2014/0901/p303.html

  Nonsteroidal anti-inflammatory drugs should be avoided because of renal toxicity. […] Adjuvant therapy for chronic pain includes antidepressants, lifestyle modification (e.g., avoidance of inciting activities, such as contact sports or heavy lifting), or reassurance. […] If renal replacement therapy is required, peritoneal dialysis and hemodialysis are options.

• #55 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.

• #56 Autosomal dominant polycystic kidney disease – Wikipedia

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

  Chronic pain in patients with ADPKD is often refractory to conservative, noninvasive treatments, but nonopioid analgesics and conservative interventions can be first used before opioid analgesics are considered; if pain continues, then surgical interventions can target renal or hepatic cysts to directly address the cause of pain, with surgical options including renal cyst decortication, renal denervation, and nephrectomy. […] Aspiration with ethanol sclerotherapy can be performed for the treatment of symptomatic simple renal cysts, but can be impractical in advanced patients with multiple cysts. […] Laparoscopic cyst decortication (also referred to as marsupialization) consists in the removal of one or more kidney cysts through laparoscopic surgery, during which cysts are punctured, and the outer wall of the larger cysts is excised with care not to incise the renal parenchyma.

• #57 Autosomal dominant polycystic kidney disease – Wikipedia

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

  Chronic pain in patients with ADPKD is often refractory to conservative, noninvasive treatments, but nonopioid analgesics and conservative interventions can be first used before opioid analgesics are considered; if pain continues, then surgical interventions can target renal or hepatic cysts to directly address the cause of pain, with surgical options including renal cyst decortication, renal denervation, and nephrectomy. […] Aspiration with ethanol sclerotherapy can be performed for the treatment of symptomatic simple renal cysts, but can be impractical in advanced patients with multiple cysts. […] Laparoscopic cyst decortication (also referred to as marsupialization) consists in the removal of one or more kidney cysts through laparoscopic surgery, during which cysts are punctured, and the outer wall of the larger cysts is excised with care not to incise the renal parenchyma.

• #58 Autosomal dominant polycystic kidney disease – Wikipedia

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

  A novel treatment of specifically the chronic pain experienced by many with ADPKD is Celiac plexus neurolysis. […] Kidney transplantation is accepted as the preferred treatment for ADPKD patients with ESRD. […] There are several novel therapies currently underway aimed at slowing the progression of disease in APKD. […] Increased water intake downregulates vasopressin activity As a result water therapy has been explored as a potential therapeutic intervention for individuals with ADPKD, however studies have examining its role in ADPKD advancement remain unclear. […] The progression of ADPKD leads to decline in kidney function, with a marked decreased in glomerular filtration rate. […] Somatostatin are artificial peptides designed to mimic the function of endogenous hormone that has many regulatory functions within the body including restraining cell proliferation. […] Proliferation of epithelial cells lining cyst resulting in cyst expansion and contributing to disease progression.

• #59 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.

• #60 Autosomal Dominant Polycystic Kidney Disease | AAFP

  https://www.aafp.org/pubs/afp/issues/2014/0901/p303.html

  Autosomal dominant polycystic kidney disease (ADPKD) is the most common inherited cause of kidney disease. […] The progression of ADPKD cannot be reversed with current treatment modalities; therefore, therapies target the resulting clinical manifestations. Early detection and management of hypertension are important to delay the progression of renal dysfunction and development of cardiovascular complications. Pain management includes evaluation of concomitant illnesses, use of analgesics, and adjuvant therapy. […] Angiotensin-converting enzyme inhibitors and angiotensin receptor blockers are recommended for initial hypertension management in patients with ADPKD. […] Fluoroquinolones, third-generation cephalosporins, and trimethoprim/sulfamethoxazole are effective for treating urinary tract infections and infected renal cysts in patients with ADPKD. Fluoroquinolones may be preferred given their lipophilic properties and bactericidal action against gram-negative pathogens.

• #61 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.

• #62 Autosomal Dominant Polycystic Kidney Disease Therapies on the Horizon – PubMed

  https://pubmed.ncbi.nlm.nih.gov/37088527/

  Autosomal dominant polycystic kidney disease (ADPKD) is characterized by the formation of numerous kidney cysts which leads to kidney failure. […] Tolvaptan is the only drug approved by Food and Drug Administration to treat ADPKD patients at the risk of rapid disease progression. […] This review discusses the potential strategies to improve the tolerability of tolvaptan, the progress on the use of an alternative vasopressin 2 receptor antagonist lixivaptan, and somatostatin analogs. […] We review the new pharmacotherapies and dietary interventions of ADPKD that are promising in the preclinical studies and investigated in clinical trials.

• #63 Autosomal Dominant Polycystic Kidney Disease Therapies on the Horizon – PubMed

  https://pubmed.ncbi.nlm.nih.gov/37088527/

  Autosomal dominant polycystic kidney disease (ADPKD) is characterized by the formation of numerous kidney cysts which leads to kidney failure. […] Tolvaptan is the only drug approved by Food and Drug Administration to treat ADPKD patients at the risk of rapid disease progression. […] This review discusses the potential strategies to improve the tolerability of tolvaptan, the progress on the use of an alternative vasopressin 2 receptor antagonist lixivaptan, and somatostatin analogs. […] We review the new pharmacotherapies and dietary interventions of ADPKD that are promising in the preclinical studies and investigated in clinical trials.

• #64 Autosomal Dominant Polycystic Kidney Disease: From Pathophysiology of Cystogenesis to Advances in the Treatment

  https://www.mdpi.com/1422-0067/23/6/3317

  Nowadays, there is no evidence that somatostatin analogues should be used in patients with polycystic renal disease. […] Lixivaptan is a selective V2 receptor antagonist. […] Metformin is a biguanide drug, which is used in type 2 diabetes, Metformin activates 5′AMP-activated protein kinase (AMPK) that inhibits CFTR by phosphorylation which leads to the suppression of epithelial fluid and electrolyte secretion. […] Statins are recommended in all patients with reduced glomerular filtration rate.

• #65 Evaluating Treatments for Autosomal Dominant PKD

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

  The TAME PKD study is a phase II, double-blind, placebo-controlled, randomized, controlled trial investigating the primary outcome of safety and tolerability of metformin in patients in the early stages of ADPKD with an eGFR of 50 mL/min per 1.73 m2. The secondary outcome investigated the eGFR decline and total kidney volume growth. 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). […] Available therapies to slow the loss of kidney function in this patient population are limited. Continued research is needed to ensure that treatment options for ADPKD confer longer-term benefits and are not only safe and effective but also widely available and affordable.

• #66 Autosomal Dominant Polycystic Kidney Disease: From Pathophysiology of Cystogenesis to Advances in the Treatment

  https://www.mdpi.com/1422-0067/23/6/3317

  Nowadays, there is no evidence that somatostatin analogues should be used in patients with polycystic renal disease. […] Lixivaptan is a selective V2 receptor antagonist. […] Metformin is a biguanide drug, which is used in type 2 diabetes, Metformin activates 5′AMP-activated protein kinase (AMPK) that inhibits CFTR by phosphorylation which leads to the suppression of epithelial fluid and electrolyte secretion. […] Statins are recommended in all patients with reduced glomerular filtration rate.

• #67 Evaluating Treatments for Autosomal Dominant PKD

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

  The TAME PKD study is a phase II, double-blind, placebo-controlled, randomized, controlled trial investigating the primary outcome of safety and tolerability of metformin in patients in the early stages of ADPKD with an eGFR of 50 mL/min per 1.73 m2. The secondary outcome investigated the eGFR decline and total kidney volume growth. 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). […] Available therapies to slow the loss of kidney function in this patient population are limited. Continued research is needed to ensure that treatment options for ADPKD confer longer-term benefits and are not only safe and effective but also widely available and affordable.

• #68 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/

  Unfortunately, clinical trials failed to show any clinical evidence for their use. […] 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. Therefore, these agents arent recommended for use in the treatment of ADPKD. […] 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. […] 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. mTOR inhibitors (Sirolimus, Everolimus), somatostatin analogue (Octreotide), and glucoceramide synthase inhibitor (Venglustat) have all been studied but proven to be non-effective. We anxiously await new therapies that can improve the outcomes of our patients with ADPKD.

• #69 Autosomal dominant polycystic kidney disease and pioglitazone for its therapy: a comprehensive review with an emphasis on the molecular pathogenesis and pharmacological aspects | Molecular Medicine | Full Text

  https://molmed.biomedcentral.com/articles/10.1186/s10020-020-00246-3

  Pioglitazone; a well-known anti-diabetic drug that is a peroxisome proliferator-activated receptor gamma (PPAR-) agonist, has been found to suppress the development of renal cyst in the preclinical studies via different mechanisms which inarguably suggests its pleiotropy. […] The characteristic property to improve the molecular and phenotypic defects with a lower risk of adverse effects in preclinical animal models has proved pioglitazone an excellent and emerging candidate to treat ADPKD. […] The review of the cAMP role in ADPKD will be incomplete without discussing the tolvaptan, and vasopressin [also called arginine vasopressin (AVP)] in the context of ADPKD. […] The role of vasopressin in ADPKD is substantial and has been proved in different studies. […] The activation of PPAR-gamma leads to various actions that consequently leads to the actions that include transactivation of the genes that regulate the adipocyte differentiation that leads to an increased number of small insulin-sensitive adipocytes, increased uptake of glucose by skeletal muscle, and decreased production of glucose by the liver which consequently are responsible for its glucose-lowering effect in type-2 diabetes mellitus patients; thereby decreasing the insulin resistance.

• #70 Autosomal dominant polycystic kidney disease and pioglitazone for its therapy: a comprehensive review with an emphasis on the molecular pathogenesis and pharmacological aspects | Molecular Medicine | Full Text

  https://molmed.biomedcentral.com/articles/10.1186/s10020-020-00246-3

  Through PPAR- activation, pioglitazone exerts beneficial actions in ADPKD, which is discussed in the next section. […] Blazer-Yost et al. conducted a study to find the efficacy of pioglitazone in ADPKD by using the PCK rat model, which progressively develops cystic enlargement of the kidneys. […] The effect of pioglitazone on the development of cyst was also tested at the dose of 4 mg/kg bodyweight for 7 weeks, and they found that this dose was as effective as the higher dose (20 mg/kg) in lowering the cystic burden in both the male and female rats. […] The authors found that there was CFTR-positive staining in the apical membrane lining the liver cysts in the rats with ADPKD while it was much diminished in the rats treated with pioglitazone. […] There is only one clinical study (NCT02697617) which is an ongoing phase II study that is targeted to evaluate the effect of the low dose of pioglitazone (15 mg) on the treatment of ADPKD.

• #71

  https://link.springer.com/article/10.1007/s40265-022-01745-9

  A more kidney-specific target for therapeutic intervention is the vasopressin 2 receptor (V2R), located on the basolateral membrane of collecting duct cells. […] In 2012, the landmark clinical trial with a V2RA (TEMPO 3:4 trial) was published. […] These results led to a marketing authorization for tolvaptan by, among others, the FDA and the EMA for the indication of ADPKD with a high likelihood of rapid disease progression. […] Despite that disease-modifying treatment is now available for clinical use, which is an important breakthrough in ADPKD, the moderate efficacy, limited tolerability (due to aquaretic events), and risk of hepatic toxicity warrant the development of additional treatment options. […] As stated in the Introduction, treatment with tolvaptan preserves kidney function in ADPKD, but aquaretic side effects such as polyuria, nycturia, and thirst limit its use.

• #72

  https://link.springer.com/article/10.1007/s40265-022-01745-9

  Autosomal dominant polycystic kidney disease (ADPKD) is characterized by progressive cyst formation that ultimately leads to kidney failure in most patients. […] To date, a vasopressin V2 receptor antagonist (V2RA) is the only drug that has been proven to attenuate disease progression. […] This review discusses new drugs that are investigated in clinical trials to treat ADPKD, such as cystic fibrosis transmembrane conductance regulator (CFTR) modulators and micro RNA inhibitors, and drugs already marketed for other indications that are being investigated for off-label use in ADPKD, such as metformin. […] Since ADPKD is a complex disease with a high degree of interindividual heterogeneity, and the mechanisms involved in cyst growth also have important functions in various physiological processes, it may prove difficult to develop drugs that target cyst growth without causing major adverse events.

• #73 Research Suggests Avenues Toward Gene Therapies for Polycystic Kidney Disease < Yale School of Medicine

  https://medicine.yale.edu/news-article/research-suggests-avenues-toward-gene-therapies-for-polycystic-kidney-disease/

  The team plans to continue pursuing the use of gene therapy, initially in mouse models, for just the 200 amino acid piece, with hopes that their work will one day benefit humans. From a therapeutic perspective, its really exciting that well hopefully be able to at least slow down disease progression, says Onuchic.

• #74 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

  We really believe this has potential to impact the field and provide a different treatment paradigm for this important disease, says Bogdan Fedeles. […] The only drug that is FDA-approved to treat the disease, tolvaptan, slows growth of the cysts but has side effects that include frequent urination and possible liver damage. […] 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. […] 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.

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

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

  Medicine can manage pain caused by infections, kidney stones or burst cysts. Your healthcare provider should approve any pain medicines you take. Some medicines can make kidney damage worse. […] Polycystic kidney disease isnt 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. About half of people with ADKPD will need dialysis or a kidney transplant due to kidney failure by age 70. […] Treatment reduces symptoms and helps your kidneys work better. Its important to work with your provider and follow their instructions to manage the condition.

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

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

  Patients with ADPKD who progress to end-stage kidney disease (ESKD) require kidney replacement therapy. […] Patients with ADPKD and ESKD are therefore most commonly treated with hemodialysis or undergo kidney transplantation. […] 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.

• #77 Polycystic Kidney Disease, Autosomal Dominant | UCSF Health

  https://www.ucsfhealth.org/conditions/polycystic-kidney-disease-autosomal-dominant

  When PKD progresses to kidney failure, waste products and fluid begin to build up in the blood. Initial symptoms may include nausea, loss of appetite, fatigue, and vomiting. At this point, either dialysis or a transplant is necessary. […] A transplant often offers a better prognosis, but a shortage of organs means that even patients who desire a transplant are likely to rely on dialysis first. […] There are two types of dialysis: hemodialysis and peritoneal dialysis. […] For transplants, kidneys from living donors usually work best. A donor need not be a relative. Younger people with few other health problems are the best candidates for transplant. Your doctor can help you choose the best course of action for you.

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

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

  If you receive a PKD diagnosis, its important to work with your healthcare provider on a treatment plan to manage complications of the disease. […] Theres no cure for PKD. 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. Keeping your blood pressure within a safe range reduces your risk of heart disease and stroke. […] If you have kidney failure, you may need dialysis (a procedure to clean your blood). Hemodialysis uses a machine to filter blood outside the body. Peritoneal dialysis uses the lining of your belly and a special fluid to filter blood. […] You may need a kidney transplant if ADPKD progresses to kidney failure. A transplant is surgery to replace a failing kidney with a donor kidney.

• #79 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.

• #80 Autosomal dominant polycystic kidney disease – Wikipedia

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

  A novel treatment of specifically the chronic pain experienced by many with ADPKD is Celiac plexus neurolysis. […] Kidney transplantation is accepted as the preferred treatment for ADPKD patients with ESRD. […] There are several novel therapies currently underway aimed at slowing the progression of disease in APKD. […] Increased water intake downregulates vasopressin activity As a result water therapy has been explored as a potential therapeutic intervention for individuals with ADPKD, however studies have examining its role in ADPKD advancement remain unclear. […] The progression of ADPKD leads to decline in kidney function, with a marked decreased in glomerular filtration rate. […] Somatostatin are artificial peptides designed to mimic the function of endogenous hormone that has many regulatory functions within the body including restraining cell proliferation. […] Proliferation of epithelial cells lining cyst resulting in cyst expansion and contributing to disease progression.

• #81 Polycystic Kidney Disease, Autosomal Dominant | UCSF Health

  https://www.ucsfhealth.org/conditions/polycystic-kidney-disease-autosomal-dominant

  When PKD progresses to kidney failure, waste products and fluid begin to build up in the blood. Initial symptoms may include nausea, loss of appetite, fatigue, and vomiting. At this point, either dialysis or a transplant is necessary. […] A transplant often offers a better prognosis, but a shortage of organs means that even patients who desire a transplant are likely to rely on dialysis first. […] There are two types of dialysis: hemodialysis and peritoneal dialysis. […] For transplants, kidneys from living donors usually work best. A donor need not be a relative. Younger people with few other health problems are the best candidates for transplant. Your doctor can help you choose the best course of action for you.

• #82 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.

• #83 ADPKD basics

  https://pkdcharity.org.uk/adpkd/what-is-adpkd/adpkd-basics

  Haemodialysis uses a machine to filter your blood, whereas peritoneal dialysis uses a special fluid placed in your abdomen to absorb waste. […] A kidney transplant will do about half (50%) of the work of a healthy kidney, while dialysis will do about one tenth (10%). Even a 10% increase in function will be enough to improve some of your symptoms. […] You can learn more about treatments for ADPKD, including tolvaptan, dialysis and kidney transplants on our website.

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

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

  Patients with ADPKD who progress to end-stage kidney disease (ESKD) require kidney replacement therapy. […] Patients with ADPKD and ESKD are therefore most commonly treated with hemodialysis or undergo kidney transplantation. […] 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.

• #85 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.

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

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

  Most do not require treatment, as the majority are asymptomatic. Hepatic cysts do not cause liver failure. Rarely, symptomatic disease requires interventions to reduce cyst size or hepatic volume. The procedure will depend on the distribution and size of cysts (eg, percutaneous cyst aspiration, laparoscopic cyst fenestration, liver resection or liver transplant). […] Magnetic resonance angiography (MRA) is used to detect aneurysms in patients with ADPKD where there is a family history of aneurysm or stroke, previous aneurysm rupture, new-onset headache or other relevant CNS sign or symptom, preparation for major surgery, or high-risk occupation (eg, airline pilot). Routine screening for berry aneurysms in ADPKD patients without a family history is not recommended. Where an asymptomatic aneurysm is discovered, the decision to go ahead with intervention is a difficult balance of whether coiling/clipping is technically possible (dependent on site, size, morphology) and the risks of the procedure, versus the risk of spontaneous rupture. In many cases, aneurysms can be safely left alone.

• #87 Autosomal Dominant Polycystic Kidney Disease: Early intervention and lifestyle crucial – Mayo Clinic News Network

  https://newsnetwork.mayoclinic.org/discussion/autosomal-dominant-polycystic-kidney-disease-early-intervention-and-lifestyle-crucial/

  A comprehensive review published by Mayo Clinic researchers advances the understanding of Autosomal Dominant Polycystic Kidney Disease (ADPKD), the most common inherited form of kidney disease and the fourth leading cause of kidney failure worldwide. […] „This is a vital resource for healthcare providers to stay informed about the latest diagnostic tools and treatment strategies to enhance patient care,” says Fouad Chebib, M.D., a Mayo Clinic nephrologist and first author of the review. […] The review provides practical guidance on managing blood pressure, sodium intake, hydration and lifestyle changes to delay complications such as kidney failure, liver cysts and hypertension to slow disease progression. […] The review also explores the use of novel therapies, such as the drug tolvaptan, recently approved by the Food and Drug Administration to slow kidney function decline for high-risk adults at risk of rapidly progressing ADPKD.

• #88 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/

  Unfortunately, clinical trials failed to show any clinical evidence for their use. […] 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. Therefore, these agents arent recommended for use in the treatment of ADPKD. […] 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. […] 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. mTOR inhibitors (Sirolimus, Everolimus), somatostatin analogue (Octreotide), and glucoceramide synthase inhibitor (Venglustat) have all been studied but proven to be non-effective. We anxiously await new therapies that can improve the outcomes of our patients with ADPKD.

• #89 Autosomal Dominant Polycystic Kidney Disease: Early intervention and lifestyle crucial – Mayo Clinic News Network

  https://newsnetwork.mayoclinic.org/discussion/autosomal-dominant-polycystic-kidney-disease-early-intervention-and-lifestyle-crucial/

  A comprehensive review published by Mayo Clinic researchers advances the understanding of Autosomal Dominant Polycystic Kidney Disease (ADPKD), the most common inherited form of kidney disease and the fourth leading cause of kidney failure worldwide. […] „This is a vital resource for healthcare providers to stay informed about the latest diagnostic tools and treatment strategies to enhance patient care,” says Fouad Chebib, M.D., a Mayo Clinic nephrologist and first author of the review. […] The review provides practical guidance on managing blood pressure, sodium intake, hydration and lifestyle changes to delay complications such as kidney failure, liver cysts and hypertension to slow disease progression. […] The review also explores the use of novel therapies, such as the drug tolvaptan, recently approved by the Food and Drug Administration to slow kidney function decline for high-risk adults at risk of rapidly progressing ADPKD.

• #90 Autosomal Dominant Polycystic Kidney Disease: Early intervention and lifestyle crucial – Mayo Clinic News Network

  https://newsnetwork.mayoclinic.org/discussion/autosomal-dominant-polycystic-kidney-disease-early-intervention-and-lifestyle-crucial/

  „This drug improves care and paves the way for future therapies that enhance the quality of life and delay kidney failure,” explains Neera Dahl, M.D., Ph.D., Mayo Clinic nephrologist and senior author of the review. […] The review recommends that a kidney specialist who manages patients with ADPKD should share decision-making with the patient regarding genetic testing, treatments, monitoring and aneurysm screening.

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