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 July 23, 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 July 23, 2014).

Share This




More Health & Medicine News

Wednesday, July 23, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Courts Conflicted Over Healthcare Law

Courts Conflicted Over Healthcare Law

AP (July 22, 2014) — Two federal appeals courts issued conflicting rulings Tuesday on the legality of the federally-run healthcare exchange that operates in 36 states. (July 22) Video provided by AP
Powered by NewsLook.com
Why Do People Believe We Only Use 10 Percent Of Our Brains?

Why Do People Believe We Only Use 10 Percent Of Our Brains?

Newsy (July 22, 2014) — The new sci-fi thriller "Lucy" is making people question whether we really use all our brainpower. But, as scientists have insisted for years, we do. Video provided by Newsy
Powered by NewsLook.com
Scientists Find New Way To Make Human Platelets

Scientists Find New Way To Make Human Platelets

Newsy (July 22, 2014) — Boston scientists have discovered a new way to create fully functioning human platelets using a bioreactor and human stem cells. Video provided by Newsy
Powered by NewsLook.com
Gilead's $1000-a-Pill Drug Could Cure Hep C in HIV-Positive People

Gilead's $1000-a-Pill Drug Could Cure Hep C in HIV-Positive People

TheStreet (July 21, 2014) — New research shows Gilead Science's drug Sovaldi helps in curing hepatitis C in those who suffer from HIV. In a medical study, the combination of Gilead's Hep C drug with anti-viral drug Ribavirin cured 76% of HIV-positive patients suffering from the most common hepatitis C strain. Hepatitis C and related complications have been a top cause of death in HIV-positive patients. Typical medication used to treat the disease, including interferon proteins, tended to react badly with HIV drugs. However, Sovaldi's %1,000-a-pill price tag could limit the number of patients able to access the treatment. TheStreet's Keris Lahiff reports from New York. Video provided by TheStreet
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