Featured Research

from universities, journals, and other organizations

Patients with rare lung disease face agonizing treatment dilemma

Date:
April 17, 2014
Source:
Loyola University Health System
Summary:
The drug sirolimus can slow the lung disease LAM, while also causing potentially fatal complications after lung transplants. But research suggests a drug similar to sirolimus may be safe for LAM patients waiting for transplants. LAM occurs almost exclusively in women. An abnormal growth of muscle cells invades the lungs, eventually causing airways to become obstructed. Symptoms include shortness of breath, chest pain, chronic cough and pneumothoraces (lung collapses). Many patients will need to go on oxygen, and some will require lung transplants.

Doctors who treat patients with a severe and progressive respiratory disease called lymphangioleiomyomatosis (LAM) can face an agonizing treatment decision.

The drug sirolimus can slow progression of the disease and help relieve shortness of breath. But some patients eventually may need lung transplants, and sirolimus can cause potentially fatal complications following transplantation.

"It's a terrible situation," said pulmonologist Dr. Daniel Dilling, medical director of Loyola University Medical Center's LAM Clinic and Lung Transplantation program.

The Food and Drug Administration has issued a "Black Box" warning about the use of sirolimus in lung transplant patients, at least when started at the time of transplantation. Many physicians therefore do not use the drug in LAM patients once they go on the transplant waiting list, since it is uncertain when the operation will occur.

But information about the drug's metabolism, an animal model and an informal physician survey suggest that a drug similar to sirolimus may be an option for LAM patients, according to a presentation Dilling made during the 34th Annual Meeting and Scientific Sessions of the International Society for Heart and Lung Transplantation in San Diego. Dilling earlier presented at the 2013 International Lymphangioleiomyomatosis Research and Patient Educational Conference, sponsored by the LAM Foundation.

LAM occurs almost exclusively in women. An abnormal growth of muscle cells invades the lungs, eventually causing airways to become obstructed. Symptoms include shortness of breath, chest pain, chronic cough and pneumothoraces (lung collapses). As the disease progresses, many patients will need to go on oxygen, and some will require lung transplants.

LAM is uncommon, but not as rare as once thought. As many as 30,000 to 50,000 women worldwide may have the disease, according to the LAM Foundation.

Sirolimus belongs to the rapamycin class of drugs that also includes everolimus. According to a 2011 study published in the New England Journal of Medicine, sirolimus in selected patients slowed the progression of the disease and was associated with a reduction in symptoms and improved quality of life.

Sirolimus sometimes is given to lung transplant patients to prevent rejection. But two studies found that sirolimus caused a serious and sometimes fatal complication called anastomotic dehiscence, which impairs wound healing if the drug is given around the time of the operation. Consequently the connections where the donor's lung are sutured into the recipient break apart.

Sirolimus (trade name, Rapamuneฎ) has a relatively long half-life, meaning it stays in the body even after a patient stops taking it. Consequently, many physicians do not prescribe sirolimus in patients who are waiting for lung transplants.

However, some LAM physicians have begun prescribing everolimus (Zortressฎ), which has a shorter half-life. Once a patient undergoes a lung transplant, the patient is immediately taken off the drug. In an informal survey, Dilling found that physicians who have used this strategy report no dehiscence problems. Moreover, a recent animal study at the University of Cincinnati found that if sirolimus treatment ceased immediately, wound healing was not impaired. And an earlier study of pulmonary fibrosis patients who took everolimus found that among 11 patients who had lung transplants, there was no increased incidence of wound-healing/anastomosis problems.

Historically, Loyola physicians have not prescribed rapamycin-class drugs to LAM patients who are on the waiting list for lung transplants. "However, in light of recent evidence and experience, we are reconsidering this policy," Dilling said.

Laura Lentz, chair of the board of directors of the LAM Foundation, said that in raising the topic, Dilling "is providing a service that is going to make a big difference for women who have LAM."

Lentz agrees with Dilling that more research is needed on rapamycin-class drugs in LAM patients. "One of the frustrations of having a rare disease is that there has not been a lot of work in this area," Lentz said. "There are more unknowns than if this were a common disease."


Story Source:

The above story is based on materials provided by Loyola University Health System. Note: Materials may be edited for content and length.


Cite This Page:

Loyola University Health System. "Patients with rare lung disease face agonizing treatment dilemma." ScienceDaily. ScienceDaily, 17 April 2014. <www.sciencedaily.com/releases/2014/04/140417141827.htm>.
Loyola University Health System. (2014, April 17). Patients with rare lung disease face agonizing treatment dilemma. ScienceDaily. Retrieved July 22, 2014 from www.sciencedaily.com/releases/2014/04/140417141827.htm
Loyola University Health System. "Patients with rare lung disease face agonizing treatment dilemma." ScienceDaily. www.sciencedaily.com/releases/2014/04/140417141827.htm (accessed July 22, 2014).

Share This




More Health & Medicine News

Tuesday, July 22, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

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
$23.6 Billion Awarded To Widow In Smoking Lawsuit

$23.6 Billion Awarded To Widow In Smoking Lawsuit

Newsy (July 20, 2014) — Cynthia Robinson claims R.J. Reynolds Tobacco Company hid the health and addiction risks of its products, leading to the death of her husband in 1996. Video provided by Newsy
Powered by NewsLook.com
Tooth Plaque Provides Insight Into Diets Of Ancient People

Tooth Plaque Provides Insight Into Diets Of Ancient People

Newsy (July 19, 2014) — Research on plaque from ancient teeth shows that our prehistoric ancestor's had a detailed understanding of plants long before developing agriculture. Video provided by Newsy
Powered by NewsLook.com
Contaminated Water Kills 3 Babies in South African Town

Contaminated Water Kills 3 Babies in South African Town

AFP (July 18, 2014) — Contaminated water in South Africa's northwestern town of Bloemhof kills three babies and hospitalises over 500 people. The incident highlights growing fears over water safety in South Africa. Duration: 02:22 Video provided by AFP
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