Featured Research

from universities, journals, and other organizations

Combination Therapy Stops Loss Of Kidney Function In Rare Genetic Disease

Date:
July 27, 2007
Source:
American Society of Nephrology
Summary:
A combination of two types of blood pressure-lowering drugs -- an angiotensin-converting enzyme inhibitor (ACEI) plus an angiotensin-receptor blocker (ARB), added to enzyme replacement therapy (ERT) with agalsidase-beta -- is the first treatment shown to stop progressive loss of kidney function in patients with severe kidney involvement due to the rare genetic disorder Fabry disease, according to a new article.

A combination of two types of blood pressure-lowering drugs--an angiotensin-converting enzyme inhibitor (ACEI) plus an angiotensin-receptor blocker (ARB), added to enzyme replacement therapy (ERT) with agalsidase-beta (Fabrazymeฎ, Genzyme Corporation, Cambridge, MA)--is the first treatment shown to stop progressive loss of kidney function in patients with severe kidney involvement due to the rare genetic disorder Fabry disease, reports a study in the September Journal of the American Society of Nephrology.

Related Articles


"While Fabry disease is very rare, our study shows that controlling urine protein excretion will slow progression of kidney disease, even though the achieved blood pressures were lower than are usually targeted in treating chronic kidney disease," says Dr. David Warnock of University of Alabama, Birmingham, one of the study authors.

Dr. Warnock and colleagues report encouraging results with ACEI/ARB therapy in eleven patients with progressive loss of kidney function related to Fabry disease (Fabry nephropathy). Fabry disease is a rare genetic disorder caused by problems with an enzyme called alpha-galactosidase-A, which the body needs to metabolize lipids (fatty substances). Even with treatment to replace the missing enzyme, buildup of lipids can cause progressive kidney, brain, and heart disease. In the current study, four patients had relatively mild kidney disease (stage 1 to 2), while seven had more severe loss of kidney function (stage 3 to 4).

All patients received ERT and combined ACEI/ARB treatment. The ACEIs and ARBs are widely used in patients with chronic kidney disease and proteinuria--"leakage" of protein into the urine associated with progressive loss of kidney function. Treatment is usually based on the patient's blood pressure. However, the situation is different in patients with Fabry disease, who often have low to normal blood pressure despite kidney disease.

The ACEI/ARB combination effectively controlled the patients' level of proteinuria. Among six patients with severe kidney disease, treatment greatly slowed the rate of deterioration in kidney function and reduced urine protein loss--in contrast, previous studies have reported no beneficial effect of ERT alone on proteinuria. After an average of two and a half years' treatment, the UAB patients had only a minimal decline in kidney function, which was not significantly from zero.

The combination of ERT and ACEI/ARB therapy also stabilized kidney function in patients with less severe kidney disease and milder degrees of proteinuria. Aside from some minor problems related to episodes of low blood pressure, there were no serious side effects of ACEI/ARB treatment.

Studies in other groups of patients with chronic kidney disease have highlighted the importance of treatment with an ACEI and/or ARB to control proteinuria. Although most of the improvement probably results from reductions in blood pressure, other effects may contribute as well. In patients with Fabry disease, ERT is of some help in treating mild kidney disease, but is less effective in those with more severe kidney disease and proteinuria.

"This work shows for the first time that it is possible to stop progressive decline of kidney function in patients who have Fabry disease with advanced kidney involvement and proteinuria," Dr. Warnock concludes. "The results were everything we hoped for, and are now being confirmed in a sixteen-site international multicenter trial."

Dr Warnock is a consultant for Genzyme Corporation and has received research support from Genzyme Corporation. The study referenced above was not supported by the Genzyme Corporation.

The study entitled, "Anti-Proteinuric Therapy and Fabry Nephropathy: Sustained Reduction of Proteinuria in Patients Receiving Enzyme Replacement Therapy with Agalsidase-Beta" will be available online beginning on Wednesday, July 25 and in print in the September issue of the Journal of the American Society of Nephrology.


Story Source:

The above story is based on materials provided by American Society of Nephrology. Note: Materials may be edited for content and length.


Cite This Page:

American Society of Nephrology. "Combination Therapy Stops Loss Of Kidney Function In Rare Genetic Disease." ScienceDaily. ScienceDaily, 27 July 2007. <www.sciencedaily.com/releases/2007/07/070726085928.htm>.
American Society of Nephrology. (2007, July 27). Combination Therapy Stops Loss Of Kidney Function In Rare Genetic Disease. ScienceDaily. Retrieved December 21, 2014 from www.sciencedaily.com/releases/2007/07/070726085928.htm
American Society of Nephrology. "Combination Therapy Stops Loss Of Kidney Function In Rare Genetic Disease." ScienceDaily. www.sciencedaily.com/releases/2007/07/070726085928.htm (accessed December 21, 2014).

Share This


More From ScienceDaily



More Health & Medicine News

Sunday, December 21, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

The Best Tips to Curb Holiday Carbs

The Best Tips to Curb Holiday Carbs

Buzz60 (Dec. 19, 2014) — It's hard to resist those delicious but fattening carbs we all crave during the winter months, but there are some ways to stay satisfied without consuming the extra calories. Vanessa Freeman (@VanessaFreeTV) has the details. Video provided by Buzz60
Powered by NewsLook.com
Sierra Leone Bikers Spread the Message to Fight Ebola

Sierra Leone Bikers Spread the Message to Fight Ebola

AFP (Dec. 19, 2014) — More than 100 motorcyclists hit the road to spread awareness messages about Ebola. Nearly 7,000 people have now died from the virus, almost all of them in west Africa, according to the World Health Organization. Video provided by AFP
Powered by NewsLook.com
Researchers Test Colombian Village With High Alzheimer's Rates

Researchers Test Colombian Village With High Alzheimer's Rates

AFP (Dec. 19, 2014) — In Yarumal, a village in N. Colombia, Alzheimer's has ravaged a disproportionately large number of families. A genetic "curse" that may pave the way for research on how to treat the disease that claims a new victim every four seconds. Duration: 02:42 Video provided by AFP
Powered by NewsLook.com
The Best Protein-Filled Foods to Energize You for the New Year

The Best Protein-Filled Foods to Energize You for the New Year

Buzz60 (Dec. 19, 2014) — The new year is coming and nothing will energize you more for 2015 than protein-filled foods. Fitness and nutrition expert John Basedow (@JohnBasedow) gives his favorite high protein foods that will help you build muscle, lose fat and have endless energy. Video provided by Buzz60
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
 
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:  

Breaking News:

Strange & Offbeat Stories

 

Health & Medicine

Mind & Brain

Living & Well

In Other News

... from NewsDaily.com

Science News

Health News

Environment News

Technology News



Save/Print:
Share:  

Free Subscriptions


Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

Get Social & Mobile


Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

Have Feedback?


Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
Mobile iPhone Android Web
Follow Facebook Twitter Google+
Subscribe RSS Feeds Email Newsletters
Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins