Featured Research

from universities, journals, and other organizations

Study shows benefits, limits of therapy for rare inflammatory syndrome

Date:
December 8, 2011
Source:
NIH/National Institute of Arthritis and Musculoskeletal and Skin Diseases
Summary:
A study shows that the medication etanercept reduces the frequency and severity of symptoms of TNF receptor-associated periodic syndrome, a rare inherited condition characterized by recurrent fevers, abdominal pain and skin rashes. The study also points out the need for the development of additional therapies to more thoroughly ease symptoms and prevent long-term complications of the disease.

A study shows that the medication etanercept reduces the frequency and severity of symptoms of TNF receptor-associated periodic syndrome (TRAPS), a rare inherited condition characterized by recurrent fevers, abdominal pain and skin rashes. The study, published in Arthritis & Rheumatism, also points out the need for the development of additional therapies to more thoroughly ease symptoms and prevent long-term complications of the disease. The study was released by researchers at the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), a part of the National Institutes of Health.

TRAPS is associated with mutations in the gene coding for tumor necrosis factor receptor 1 (TNFR1), a critical molecule in receiving inflammatory signals in the body's immune system. Etanercept, trade name Enbrel is one of a class of drugs that block tumor necrosis factor, a protein implicated in the harmful inflammation in TRAPS, as well as a number of common rheumatic diseases, including rheumatoid arthritis. While the drug has been used in the treatment of TRAPS for about 10 years, this is the first formal study to look at its effectiveness long-term, said Richard Siegel, M.D., Ph.D., NIAMS acting clinical director and one of the senior authors.

The study was conceived in 2001 by Keith Hull, M.D., Ph.D., then a rheumatology fellow in the NIAMS under the supervision of Daniel Kastner, M.D, Ph.D., one of the discoverers of the TNF receptor mutations in TRAPS, and now the scientific director of the NIH's National Human Genome Research Institute. The initial study enrolled 15 patients with TRAPS. Each patient kept a daily diary of attacks, symptom severity, and use of additional medicines, and had periodic blood tests to measure acute phase reactants, proteins that are produced by the liver to fight infection and serve as markers of inflammation in the blood.

While on treatment with etanercept, patients reported lower symptom scores and a greater number of symptom-free days each week.

"Patients generally reported that their attacks still happened, but they were less severe and don't last as long," said Dr. Siegel. "They were still having discomfort, but in between attacks, they could be relatively symptom-free."

Etanercept also reduced levels of acute phase reactants, particularly during asymptomatic periods.

To find out whether etanercept was effective as a long-term treatment, NIAMS' Ariel Bulua, Ph.D., a medical student in the NIH's Clinical Research Training Program, contacted all 15 patients treated with etanercept seven to nine years after the conclusion of the initial study period. Dr. Bulua arranged for them to revisit the NIH Clinical Center and be evaluated, if they had not continued to be followed regularly.

Despite the overall beneficial effects of etanercept, most patients discontinued the drug during the follow-up period due to a perceived lack of efficacy or painful injection site reactions, which could be related to the skin manifestations of the disease. The three patients who remained on the drug, however, continued to report benefits, suggesting that for some, the drug can be an effective long-term treatment option.

However, the study did not show whether etanercept could prevent the long-term consequences of TRAPS, chiefly a condition called amyloidosis, in which inflammatory proteins build up in the body, damaging the kidneys, heart and other organs, said Dr. Siegel.

"We are concerned that it may not prevent amyloidosis because we have not completely suppressed inflammatory markers the way we would want to," Dr. Siegel said. "Etanercept studied in this group of TRAPS patients in an organized way works, but not as well as we would like for it to. Patients are still having some residual symptoms, attacks of fevers and rashes. We are still looking for other pathways to target in this disease."


Story Source:

The above story is based on materials provided by NIH/National Institute of Arthritis and Musculoskeletal and Skin Diseases. Note: Materials may be edited for content and length.


Journal Reference:

  1. Ariel C. Bulua, Douglas B. Mogul, Ivona Aksentijevich, Harjot Singh, David He, Larry Muenz, Michael M. Ward, Cheryl Yarboro, Daniel L. Kastner, Richard M. Siegel, Keith M. Hull. Efficacy of etanercept in the tumor necrosis factor receptor-associated periodic syndrome (TRAPS). Arthritis & Rheumatism, 2011; DOI: 10.1002/art.33416

Cite This Page:

NIH/National Institute of Arthritis and Musculoskeletal and Skin Diseases. "Study shows benefits, limits of therapy for rare inflammatory syndrome." ScienceDaily. ScienceDaily, 8 December 2011. <www.sciencedaily.com/releases/2011/10/111026113837.htm>.
NIH/National Institute of Arthritis and Musculoskeletal and Skin Diseases. (2011, December 8). Study shows benefits, limits of therapy for rare inflammatory syndrome. ScienceDaily. Retrieved April 16, 2014 from www.sciencedaily.com/releases/2011/10/111026113837.htm
NIH/National Institute of Arthritis and Musculoskeletal and Skin Diseases. "Study shows benefits, limits of therapy for rare inflammatory syndrome." ScienceDaily. www.sciencedaily.com/releases/2011/10/111026113837.htm (accessed April 16, 2014).

Share This



More Health & Medicine News

Wednesday, April 16, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Could Even Casual Marijuana Use Alter Your Brain?

Could Even Casual Marijuana Use Alter Your Brain?

Newsy (Apr. 16, 2014) A new study conducted by researchers at Northwestern and Harvard suggests even casual marijuana use can alter your brain. Video provided by Newsy
Powered by NewsLook.com
Ebola Outbreak Now Linked To 121 Deaths

Ebola Outbreak Now Linked To 121 Deaths

Newsy (Apr. 15, 2014) The ebola virus outbreak in West Africa is now linked to 121 deaths. Health officials fear the virus will continue to spread in urban areas. Video provided by Newsy
Powered by NewsLook.com
Cognitive Function: Is It All Downhill From Age 24?

Cognitive Function: Is It All Downhill From Age 24?

Newsy (Apr. 15, 2014) A new study out of Canada says cognitive motor performance begins deteriorating around age 24. Video provided by Newsy
Powered by NewsLook.com
How Mt. Everest Helped Scientists Research Diabetes

How Mt. Everest Helped Scientists Research Diabetes

Newsy (Apr. 15, 2014) British researchers were able to use Mount Everest's low altitudes to study insulin resistance. They hope to find ways to treat diabetes. 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