Featured Research

from universities, journals, and other organizations

Tailored pre-transplant therapy boosts survival rate in rare immune deficiency

Date:
November 12, 2013
Source:
Universite de Montreal
Summary:
Chronic Granulomatous Disease is a rare immune deficiency that is life-threatening. Tailored doses of the pre-transplant drug therapy boosts survival rates to over 90%.

Chronic Granulomatous Disease is a rare immune deficiency that seriously compromises organ function and is life-threatening, with 20-30 per cent of patients dying within the first two decades of life. Cell transplantation, the only cure available to date, requires chemotherapy prior to transplantation in order to avoid transplant rejection, although there is a risk of complications such as central nervous system damage, organ failure, and infertility.

Researchers from 16 university hospitals and 10 countries, including Switzerland, Sweden, and Canada have demonstrated in a clinical study published in The Lancet that tailored doses of the pre-transplant drug therapy boosts survival rates to over 90 per cent. This success rate is particularly impressive since most patients were transplanted with donors that are not in the same family as the patient, a situation in which the survival was below 60% with other protocols, making many centers being reluctant even in considering the transplantation.

Very young children, infants, as well as adolescents and young adults suffering from intractable infections and inflammation benefited from this innovative approach. Because variations in the exposure to busulfan, an agent used in the conventional cytostatics-based pre-transplant therapy, and its related impact on metabolism are greater in children than in adults, the researchers considered it imperative to monitor blood levels of the drug especially in children and adolescents. "Our 14 children who were administered this treatment all survived. This outcome goes far beyond our expectations. It has so to speak "emptied" our region from children with the disease," said Montreal-based co-author Elie Haddad, clinician and scientist, head of the Immunology Division at the mother-child university hospital center CHU Sainte-Justine and a professor in the Department of Pediatrics at Université de Montréal. "Contributing our cohort of children and adolescents into this multicenter clinical trial clearly benefited our patients," said Pierre Teira, co-author, hemato-oncologist at the Blood and Marrow Transplantation Division of the CHU Sainte-Justine and associate Professor in the Department of Pediatrics at Université de Montréal.

"By tailoring doses of busulfan and carefully sampling patients, we achieved a survival rate of 93 per cent with minimal adverse reaction, independent of the age of the patient, even in those with poor prognosis or highly at risk of graft failure and mortality," said lead author and principal investigator Tayfun Güngör, Assistant Professor of Pediatric Stem Cell Transplantation at the University Children's Hospital Zürich. "Two adult patients have fathered children after successful transplantation, a fact that makes me hope that fertility may have been preserved in a lot of children and adults treated with this approach," he continued. Until now, patients would be administered cytostatic drugs to help their body accept the cell transplantation. However, excessive doses of these drugs can harm the recipient's organs, while insufficient doses can cause the patient's body to reject the transplanted stem cells.

Chronic Granulomatous Disease causes recurrent, often difficult-to-treat bacterial and fungal infections and non-bacterial inflammations of the inner organs, which may lead to organ dysfunction (such as bladder and kidney problems) and endanger life. Up to one-third of children affected die before the age of 20, and those entering adulthood are often handicapped with compromised organ functions and low quality of life.

The investigators' aim is for tailored treatments to become standard practice in other primary immunodeficiencies and non-malignant diseases. Indeed, Prs. Drs. Güngör, Haddad, and Teira are already using this regimen with success in other primary immunodeficiencies and non-malignant diseases.


Story Source:

The above story is based on materials provided by Universite de Montreal. Note: Materials may be edited for content and length.


Journal Reference:

  1. Tayfun Güngör, Pierre Teira, Mary Slatter, Georg Stussi, Polina Stepensky, Despina Moshous, Clementien Vermont, Imran Ahmad, Peter J Shaw, José Marcos Telles da Cunha, Paul G Schlegel, Rachel Hough, Anders Fasth, Karim Kentouche, Bernd Gruhn, Juliana F Fernandes, Silvy Lachance, Robbert Bredius, Igor B Resnick, Bernd H Belohradsky, Andrew Gennery, Alain Fischer, H Bobby Gaspar, Urs Schanz, Reinhard Seger, Katharina Rentsch, Paul Veys, Elie Haddad, Michael H Albert, Moustapha Hassan. Reduced-intensity conditioning and HLA-matched haemopoietic stem-cell transplantation in patients with chronic granulomatous disease: a prospective multicentre study. The Lancet, 2013; DOI: 10.1016/S0140-6736(13)62069-3

Cite This Page:

Universite de Montreal. "Tailored pre-transplant therapy boosts survival rate in rare immune deficiency." ScienceDaily. ScienceDaily, 12 November 2013. <www.sciencedaily.com/releases/2013/11/131112095345.htm>.
Universite de Montreal. (2013, November 12). Tailored pre-transplant therapy boosts survival rate in rare immune deficiency. ScienceDaily. Retrieved April 19, 2014 from www.sciencedaily.com/releases/2013/11/131112095345.htm
Universite de Montreal. "Tailored pre-transplant therapy boosts survival rate in rare immune deficiency." ScienceDaily. www.sciencedaily.com/releases/2013/11/131112095345.htm (accessed April 19, 2014).

Share This



More Health & Medicine News

Saturday, April 19, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

'Holy Grail' Of Weight Loss? New Find Could Be It

'Holy Grail' Of Weight Loss? New Find Could Be It

Newsy (Apr. 18, 2014) — In a potential breakthrough for future obesity treatments, scientists have used MRI scans to pinpoint brown fat in a living adult for the first time. Video provided by Newsy
Powered by NewsLook.com
Little Progress Made In Fighting Food Poisoning, CDC Says

Little Progress Made In Fighting Food Poisoning, CDC Says

Newsy (Apr. 18, 2014) — A new report shows rates of two foodborne infections increased in the U.S. in recent years, while salmonella actually dropped 9 percent. Video provided by Newsy
Powered by NewsLook.com
Scientists Create Stem Cells From Adult Skin Cells

Scientists Create Stem Cells From Adult Skin Cells

Newsy (Apr. 17, 2014) — The breakthrough could mean a cure for some serious diseases and even the possibility of human cloning, but it's all still a way off. Video provided by Newsy
Powered by NewsLook.com
Obama: 8 Million Healthcare Signups

Obama: 8 Million Healthcare Signups

AP (Apr. 17, 2014) — President Barack Obama gave a briefing Thursday announcing 8 million people have signed up under the Affordable Care Act. He blasted continued Republican efforts to repeal the law. (April 17) Video provided by AP
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