Featured Research

from universities, journals, and other organizations

Recommendations for treatment of inherited lung disease are unjustified, review finds

Date:
July 6, 2010
Source:
Wiley-Blackwell
Summary:
An expensive treatment recommended for a genetic disorder called alpha-1 antitrypsin deficiency has no proven clinical benefit, according to a systematic review. The disorder causes chronic lung disease and the review concludes that considering the lack of evidence for its benefits, and possible adverse effects, the treatment should not be recommended.

An expensive treatment recommended for a genetic disorder called alpha-1 antitrypsin deficiency has no proven clinical benefit, according to a systematic review by Cochrane Researchers. The disorder causes chronic lung disease and the review concludes that considering the lack of evidence for its benefits, and possible adverse effects, the treatment should not be recommended.

Alpha-1 antitrypsin deficiency affects less than one in 1,600 people. Those who inherit the disorder have low levels of the protein alpha-1 antitrypsin, also called alpha-1 proteinase inhibitor, which protects the tissue of the lungs from destruction by the body's own white blood cells. At a relatively young age, this can result in symptoms of emphysema, including shortness of breath and wheezing. The aim of alpha-1 antitrypsin replacement therapy is to give the patient back the protective protein they are missing. This should limit damage to lungs and, ultimately, prevent early death. The protein is extracted from blood donated by healthy volunteers.

The researchers reviewed data from two trials involving a total of 140 people with the disorder, all of whom were at a high genetic risk of developing chronic lung disease. In one trial, patients were given intravenous alpha-1 antitrypsin or a placebo every four weeks for three years and in the other, the protein or a placebo was given weekly for a minimum of two years. There was no difference between treatment and control groups in terms of exacerbations of lung disease, or quality of life. Combining the results from the trials, the review authors found no evidence of a clinically important effect on lung function; indeed the results suggested modest harm, or at best no effect. In contrast, the treatment might cause a reduction in the deterioration of lung appearance on CT scan, but it is not clear whether this is a clinically meaningful difference.

Based on this evidence, the researchers say the treatment, which costs up to $150,000 a year in the US, cannot be recommended. "The drug has not shown any clinical benefit, is extremely costly and has important adverse effects," said lead researcher Peter Gψtzsche of the Nordic Cochrane Center at Rigshospitalet in Copenhagen, Denmark. "In view of the lack of evidence and high cost of treatment, treating alpha-1 antitrypsin deficiency by replacement therapy cannot be recommended."

Neither of the trials included in the review reported mortality data and the researchers point out that adverse events were not well reported. In previous studies, a small proportion of patients suffered allergic reactions and breathing difficulties following treatment.

The researchers say recommendations by the American Thoracic Society and European Respiratory Society that promote alpha-1 antitrypsin replacement are misguided. "Both societies recommend augmentation therapy for patients with breathing problems related to alfa-1 antitrypsin deficiency. In our opinion, these recommendations are not reasonable," said Gψtzsche.


Story Source:

The above story is based on materials provided by Wiley-Blackwell. Note: Materials may be edited for content and length.


Cite This Page:

Wiley-Blackwell. "Recommendations for treatment of inherited lung disease are unjustified, review finds." ScienceDaily. ScienceDaily, 6 July 2010. <www.sciencedaily.com/releases/2010/07/100706204701.htm>.
Wiley-Blackwell. (2010, July 6). Recommendations for treatment of inherited lung disease are unjustified, review finds. ScienceDaily. Retrieved September 14, 2014 from www.sciencedaily.com/releases/2010/07/100706204701.htm
Wiley-Blackwell. "Recommendations for treatment of inherited lung disease are unjustified, review finds." ScienceDaily. www.sciencedaily.com/releases/2010/07/100706204701.htm (accessed September 14, 2014).

Share This



More Health & Medicine News

Sunday, September 14, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Shocker: Journalists Are Utterly Addicted To Coffee

Shocker: Journalists Are Utterly Addicted To Coffee

Newsy (Sep. 13, 2014) — A U.K. survey found that journalists consumed the most amount of coffee, but that's only the tip of the coffee-related statistics iceberg. Video provided by Newsy
Powered by NewsLook.com
Contagious Respiratory Illness Continues to Spread Across U.S.

Contagious Respiratory Illness Continues to Spread Across U.S.

Reuters - US Online Video (Sep. 12, 2014) — Hundreds of children in several states have been stricken by a serious respiratory illness that is spreading across the U.S. Linda So reports. Video provided by Reuters
Powered by NewsLook.com
Ebola Batters Sierra Leone Economy Too

Ebola Batters Sierra Leone Economy Too

Reuters - Business Video Online (Sep. 12, 2014) — The World Health Organisation warns that local health workers in West Africa can't keep up with Ebola - and among those countries hardest hit by the outbreak, the economic damage is coming into focus, too. As David Pollard reports, Sierra Leone admits that growth in one of the poorest economies in the region is taking a beating. Video provided by Reuters
Powered by NewsLook.com
Health Care Workers 'Chasing' Ebola Outbreak

Health Care Workers 'Chasing' Ebola Outbreak

Newsy (Sep. 12, 2014) — The worst known Ebola outbreak is proving extremely difficult to contain. Hospitals are full, and victims of the virus are suffering in the streets. Video provided by Newsy
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:
from the past week

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