|Polycystic Kidney Disease: NIH and PKD Foundation Launch HALT-PKD
The National Institutes of Health (NIH) and the PKD Foundation have launched
two treatment trials for autosomal dominant polycystic kidney disease (ADPKD).
The common inherited disorder is characterized by cysts in the kidneys and other
organs, high blood pressure, and aneurysms (bulges in blood vessels, which may
burst) in the brain. Symptoms usually appear between the ages of 30 and 40 and
include back and side pain and headaches. About half of ADPKD patients eventually
develop kidney failure and require dialysis or a kidney transplant. The first
Halt Progression of Polycystic Kidney Disease (HALT-PKD) patient enrolled last
week at Emory University in Atlanta, one of seven recruitment sites (www.pkd.wustl.edu/pkd-tn).
“Decades of clinical and basic studies by NIH and others have delivered this
exciting opportunity for translational research,” says Catherine M. Meyers, M.D.,
a kidney specialist who directs HALT-PKD at the National Institute of Diabetes
and Digestive and Kidney Diseases (NIDDK). “Naturally, we appreciate the PKD
Foundation’s invaluable guidance and support.”
PKD Foundation President and CEO Dan Larson applauded the start of the trials. "PKD
families are eager to learn of any potential benefits," says Larson. "Their hope
and the hope of the PKD Foundation is that this will be a step toward finding
a cure for PKD and improving the care and treatment of those it affects."
Carefully controlling blood pressure and using ACE-inhibitors or ARBs significantly
delays or prevents kidney disease and failure from diabetes and other causes
by reducing protein in the urine and preventing damage to the small blood vessels
in the kidneys. Earlier trials of these treatments in PKD were not definitive,
possibly because a small number of patients were involved.
Over the next 2 years, HALT-PKD will recruit more than 1,000 people with ADPKD
and treat them for up to 4 years at centers in Atlanta, Boston, Cleveland, Denver,
Kansas City, Kansas, and Rochester, Minnesota. The two trials will compare standard
therapy using an angiotensin-converting enzyme inhibitor (ACE-inhibitor) to intensive
therapy using both an ACE-inhibitor and an angiotensin receptor blocker (ARB).
Patients will receive a device for measuring blood pressure at home, clinic visits,
lab tests, and study medications at no charge. They will also have their kidney
function estimated using a standard blood test measurement called eGFR and other
measures to track progression of kidney disease.
HALT-PKD Study A, for people 15 to 49 years of age with early disease (eGFR >60),
will also compare standard (120-130/70-80 mm Hg) and low (95-110/60-75 mm Hg)
blood pressure targets and measure changes in cyst and kidney size using a Magnetic
Resonance Imaging method developed by NIDDK’s Consortium for Radiologic Imaging
Studies of PKD. Study B, for people 18 to 64 and more advanced disease (eGFR
30-60), will track the time it takes eGFR to drop by 50 percent, the need for
kidney failure treatment, and patient deaths.
PKD affects an estimated 500,000 people, about 90 percent of whom have ADPKD.
In 2003, 23,000 people with cystic kidney disease (mostly PKD) received dialysis
or a kidney transplant, making it this country’s fourth leading cause of kidney
failure. While genetic testing for ADPKD can help determine whether a family
member can safely donate a kidney, testing can’t predict onset of symptoms or
severity of the disease, which also increases the risk of heart attacks, strokes
and early death compared to the general population.
Support for HALT-PKD is also provided by Boehringer-Ingelheim Pharmaceuticals,
Inc., and Merck & Co., Inc.
For more information:
- Learn more about PKD from NIDDK at www.niddk.nih.gov and
from the PKD Foundation at www.pkdcure.org and
- Learn about NIDDK’s National Kidney Disease Education Program (NKDEP) at www.nkdep.nih.gov.
NKDEP encourages early diagnosis and treatment; encourages system changes such
as uniform reporting by labs; and develops time-saving tools for health professionals,
including eGFR calculators and a consult letter to generate next steps based
on disease stage.
The National Institutes of Health (NIH) — The Nation's Medical Research
Agency — includes 27 Institutes and Centers and is a component of
the U. S. Department of Health and Human Services. It is the primary Federal
agency for conducting and supporting basic, clinical, and translational medical
research, and it investigates the causes, treatments, and cures for both common
and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.