Management of autosomal dominant polycystic kidney disease in the era of disease-modifying treatment options

Radhakrishnan, Yeshwanter; Duriseti, Parikshit; Chebib, Fouad T.

doi:10.23876/j.krcp.21.309

Cited by 16 publications

(13 citation statements)

References 38 publications

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“…In the United States, ADPKD has an estimated incidence of about 0.6 to 0.7 million cases [ 6 ]. In majority of the cases, ADPKD is associated with genetic pathogenic variants involving PKD1 and PKD2 genes, which encode polycystic-1 and 2 proteins, respectively [ 7 ].…”

Section: Discussionmentioning

confidence: 99%

“…In majority of the cases, ADPKD is associated with genetic pathogenic variants involving PKD1 and PKD2 genes, which encode polycystic-1 and 2 proteins, respectively [ 7 ]. Compared to PKD2 , PKD1 pathogenic variants are more common, and these patients often present with relatively more severe disease, higher total kidney volume (TKV), and earlier onset kidney failure (usually by the 5 th or 6th decade of life) [ 4 , 6 , 7 ]. Our patient had imaging evidence of bilaterally enlarged kidneys with numerous renal cysts in each kidney, total kidney volume (TKV) of 5744 mL and positive genetic test with pathogenic variant in PKD1 gene, consistent with the diagnosis of ADPKD [ 6 – 9 ].…”

Section: Discussionmentioning

confidence: 99%

“…Cyst hemorrhage is a frequent complication seen in ADPKD patients [ 2 – 5 ]. Clinical presentation of cyst bleeding is usually relatively benign, with flank pain and tenderness, which subsides with conservative management involving bed rest, pain control, and hydration [ 6 , 10 , 11 ]. Rarely, patients present with severe persistent bleeding requiring hospitalization.…”

Section: Discussionmentioning

confidence: 99%

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Life-Threatening Retroperitoneal Hemorrhage Following Cyst Rupture in Autosomal Dominant Polycystic Kidney Disease (ADPKD): A Case Report

et al. 2023

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Patient: Male, 60-year-old Final Diagnosis: Retroperitoneal hemorrhage Symptoms: Altered mental status • lethargy Clinical Procedure: — Specialty: Nephrology Objective: Congenital defects/diseases Background: Autosomal dominant polycystic kidney disease (ADPKD) is the leading genetic cause of kidney failure worldwide. It is characterized by cyst formation and growth, kidney parenchymal destruction, and complications including cyst infection, nephrolithiasis, cyst rupture, and cyst hemorrhage. Cyst bleeding is typically a self-limited event. This case report describes a 60-year-old man with ADPKD admitted with retroperitoneal hemorrhage following renal cyst rupture requiring embolization of a bleeding left lumbar artery and use of tranexamic acid. Case Report: A 60-year-old man with ADPKD presented with altered mental status. Labs noted hemoglobin of 4.7 g/dL. Abdominal imaging revealed polycystic kidneys and large left retroperitoneal hematoma. Angiogram demonstrated active bleeding from left L3 lumbar artery which was embolized. He was admitted to intensive care unit for hemorrhagic shock requiring multiple blood transfusions. Hemoglobin continued to downtrend despite blood products with repeat imaging demonstrating expanding retroperitoneal bleed. He underwent repeat angiogram and though there was no active bleeding, prophylactic embolization of left L1, L3, L4 lumbar and left renal capsular arteries were performed. Hemoglobin stabilized for next 3 days but continued to downtrend subsequently. Oral tranexamic acid was trialed with stabilization of the hemoglobin. Conclusions: Life-threatening retroperitoneal hemorrhage following cyst rupture in the absence of major trauma or use of anti-coagulants, is a rare complication in ADPKD. Treatment involves resuscitation with blood products, management of shock, and interventional radiology-guided embolization. Tranexamic acid may be considered when the above measures fail. Nephrectomy may be indicated for refractory bleeding. This report highlights the diagnosis and management of massive cyst bleeding in ADPKD.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

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Life-Threatening Retroperitoneal Hemorrhage Following Cyst Rupture in Autosomal Dominant Polycystic Kidney Disease (ADPKD): A Case Report

et al. 2023

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“…Moreover, pharmacological inhibition of cAMP signaling using tolvaptan, a selective vasopressin 2 receptor (V2R) antagonist, has been reported to reduce fluid secretion, cell proliferation, and cyst growth rate in human ADPKD cyst epithelial cells [9,10]. Based on www.kjim.org https://doi.org/10.3904/kjim.2022.376 these positive results in preclinical research, clinical trials following a stepwise approach to evaluate the safety and efficacy of tolvaptan were introduced to patients with ADPKD [11][12][13]. Following approval in Japan in March 2014, Canada and European Union in February 2015, and South Korea in December 2015, tolvaptan was approved by the U. S. Food and Drug Administration as the first therapeutic agent for adult patients with ADPKD in April 2018 (Fig.…”

Section: Introductionmentioning

confidence: 99%

Recent updates in therapeutic approach using tolvaptan for autosomal dominant polycystic kidney disease

Kim¹,

Han²

2023

Korean J Intern Med

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As a genetic disease, there has been a long-standing effort to identify therapeutic options for autosomal dominant polycystic kidney disease (ADPKD). Following the development of tolvaptan, a vasopressin 2 receptor antagonist, the treatment strategy for ADPKD patients with rapid disease progression has been changed with a disease-targeted approach. Tolvaptan showed significant efficacy in preserving kidney function and reducing the total kidney volume (TKV) growth rate. These effects were especially pronounced in patients with more severe clinical phenotypes, such as higher TKV and rapidly declining kidney function. Despite the therapeutic effects of tolvaptan, aquaretic symptoms are unavoidable side effects related to the mechanism of the drug and are also directly related to the quality of life. A shared decision-making process could be a valuable strategy for reducing the incidence of side effects and improving medication adherence. Herein, we aimed to review overall clinical trials for applying tolvaptan and suggest important factors during the shared decision-making process.

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“…The aquaretic effect of Tolvaptan was maintained even in the patients with lower baseline eGFR showing larger fractional free-water clearance, which means higher responses to Tolvaptan per functioning nephron 56) . Therefore, recent articles suggest using Tolvaptan no matter what the baseline eGFR for rapidly progressive ADPKD 6 57) .…”

Section: Introductionmentioning

confidence: 99%

Practical Issues in the Management of Polycystic Kidney Disease: Blood Pressure and Water Balance

Park

2022

Electrolyte Blood Press

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Autosomal dominant polycystic kidney disease is the most common hereditary renal disease affecting more than 13 million people worldwide. Renal function deteriorates as the cysts in both kidneys increase in number and size, which eventually results in end-stage kidney failure. Until recently, conservative management for chronic kidney disease such as blood pressure control, low sodium diet, adequate water intake, and weight control were known for the only treatment of polycystic kidney disease. However, the introduction of disease-modifying drug has led to the new paradigm shift in the management of polycystic kidney disease. Tolvaptan, the vasopressin V2 receptor antagonist, has been introduced to the patients with large kidneys since it can inhibit cyclic adenosine monophosphate, attenuates cyst growth, and delays renal failure. This article reviews the two important practical issues in the management of polycystic kidney disease: blood pressure and water balance. Firstly, the article will review the pathogenesis of high blood pressure in polycystic kidney disease and will demonstrate the current up-to-date management plan for blood pressure control. Secondly, this article will explain the mechanism of Tolvaptan on the treatment of polycystic kidney disease and its possible adverse effect on water and sodium balance.

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Management of autosomal dominant polycystic kidney disease in the era of disease-modifying treatment options

Cited by 16 publications

References 38 publications

Life-Threatening Retroperitoneal Hemorrhage Following Cyst Rupture in Autosomal Dominant Polycystic Kidney Disease (ADPKD): A Case Report

Life-Threatening Retroperitoneal Hemorrhage Following Cyst Rupture in Autosomal Dominant Polycystic Kidney Disease (ADPKD): A Case Report

Recent updates in therapeutic approach using tolvaptan for autosomal dominant polycystic kidney disease

Practical Issues in the Management of Polycystic Kidney Disease: Blood Pressure and Water Balance

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