Background Information on Polycystic Kidney Disease Treatments

Polycystic kidney disease (PKD) is the most common inherited kidney disease in the United States occurring in approximately 1 in every 1,000 live births. It affects more than 20,000 people in the New York area, 600,000 people in the United States and 12 million worldwide. It is the third most common single cause of kidney failure.

Polycystic kidney disease is characterized by the growth of numerous kidney cysts, which cause abnormalities in both the kidney structure and function. Cysts also develop in other organs, particularly in the liver. Other manifestations of polycystic kidney disease include high blood pressure, urinary tract infections, kidney stones, blood in the urine, hernia and weakening of blood vessels, referred to as aneurysm. Pain in the back, abdomen and pelvis affects many PKD patients.

There are two types of polycystic kidney disease, autosomal dominant (ADPKD) and autosomal recessive (ARPKD). In the more common form, autosomal dominant PKD, the abnormal PKD gene (PKD1 or PKD2) is inherited from one parent who also has PKD. Affected individuals often become aware in adulthood, when cysts are detected during the evaluation of symptoms such as pain or blood in the urine. There are however, a small percentage of people with autosomal dominant PKD who do not have a family member with the disease. ARPKD is associated with abnormal function of the kidneys and liver and is usually diagnosed by ultrasound during fetal development or shortly after birth. Unlike ADPKD, an abnormal PKHD1 gene must be contributed by each parent for ARPKD to occur in the offspring. Parents are not usually aware that they are carrying an abnormal gene because they have no signs of ARPKD.

While there is currently no cure for PKD, regular medical care can help to manage symptoms and identify problems that may need additional evaluation and treatment such as high blood pressure.

The Rogosin Institute, Susan R. Knafel Polycystic Kidney
Disease Center

The Rogosin Institute, Susan R. Knafel Polycystic Kidney Disease Center provides a comprehensive approach to the care of patients with PKD at all stages. Patients receive care from a team of medical professions who are dedicated to polycystic kidney disease treatment, including dialysis and transplantation. Physicians and researchers of the Susan R. Knafel Polycystic Kidney Disease Center are working to advance understanding of how PKD develops and, ultimately, to design strategies to prevent its progression and new polycystic kidney disease treatments.
For more information contact:

Stephanie L. Donahue, N.P.
Nurse Practitioner/Manager
The Rogosin Institute,
Susan R. Knafel Polycystic Kidney Disease Center
212-746-1591

The Polycystic Kidney Disease Patient Repository

All patients with autosomal dominant polycystic kidney disease can participate in The Polycystic Kidney Disease Patient Repository. The purpose of this program is to evaluate PKD genotype, clinic history, physical examination, laboratory data and kidney imaging data from PKD patients to improve our understanding of the relationship between the genetic abnormalities and other factors that contribute to complications of the disease.

participate in this registry

Potential Treatment

There is no known cure or specific treatment for PKD. Clinical Trials to evaluate new polycystic kidney disease treatments are currently underway at The Rogosin Institute Susan R. Knafel Polycystic Kidney Disease Center.

Clinical Trials
Polycystic Kidney Disease: Phase 2
Polycystic Kidney Disease Phase 3

The Rogosin Institute
Polycystic Kidney Disease Center
Stephanie Donahue, NP
212-746-1591

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