Featured Research

from universities, journals, and other organizations

Transplantation shown to be highly effective in treating immune deficiency in children

Date:
July 30, 2014
Source:
Memorial Sloan Kettering Cancer Center
Summary:
Babies who are born with severe combined immunodeficiency (SCID) can be successfully treated with a transplant of blood-forming stem cells, according to experts. SCID is a group of inherited disorders that cause the immune system to severely malfunction. When this breakdown occurs, babies no longer have the ability to fight off routine infections because their natural, built-in defense system has been damaged. If undiagnosed or left untreated, SCID is almost always fatal within the first year of life.

Richard O’Reilly, Chair of the Department of Pediatrics and Chief of the Pediatric Bone Marrow Transplant Service.
Credit: Image courtesy of Memorial Sloan Kettering Cancer Center

Babies who are born with severe combined immunodeficiency (SCID) can be successfully treated with a transplant of blood-forming stem cells, according to experts led by Memorial Sloan Kettering's Richard J. O'Reilly, MD, a world-renowned pioneer in the development of transplant protocols. Their review will be published in the July 30 issue of the New England Journal of Medicine.

Related Articles


SCID is a group of inherited disorders that cause the immune system to severely malfunction. When this breakdown occurs, babies no longer have the ability to fight off routine infections because their natural, built-in defense system has been damaged. If undiagnosed or left untreated, SCID is almost always fatal within the first year of life.

A review of more than 240 patient cases found transplants to be quite effective, especially when performed early in life. Of those children receiving transplants within three and a half months after birth, 94 percent were alive five years later. The best results, not surprisingly, were seen after transplant from "matched sibling" donors. But among patients who didn't have a matched sibling, overall five-year survival rates were quite high -- 77 to 93 percent -- if they were transplanted in the first three and a half months of life.

"This confirms that transplants for SCID work well in very young children, but it also shows that any child with this disease can be treated with a high likelihood of a cure with a transplant from a parent or unrelated donor, not just a matched brother or sister," explained Dr. O'Reilly, Chair of MSK's Pediatrics Department and Chief of the Pediatric Bone Marrow Transplant Service. "Irrespective of the transplant approach used, if the child is transplanted early -- without infection -- you will have an extraordinarily good result."

But a critical factor continues to be ensuring that the child has no infection at the time of transplant. "This really illustrates the importance of more widespread screening for SCID so that doctors can intervene immediately," said Dr. O'Reilly. Currently, only 21 states have implemented testing as part of routine newborn screening, including New York, California, and Florida. "By the time symptoms of infection appear, the circumstances for transplant are often less favorable."

MSK has played a pioneering role in advancing stem cell transplantation. The first successful transplant of stem cells from an unrelated donor was performed at MSK in 1973 specifically to treat a child with SCID. In 1981, MSK introduced T cell depletion, a groundbreaking procedure for improving the success of unmatched transplants. Most of the important benchmarks in the development of transplantation and cell therapies have come from the studies of these relatively rare cases involving children who have a lethal genetic immune system disorder, said Dr. O'Reilly.

"Great strides have been made in a relatively short time, allowing children with SCID not only to survive but also to lead normal lives," Dr. O'Reilly added. "Paul Simon had that song [The Boy in the Bubble] where he says, 'These are the days of miracles and wonder.'…. Our research provided a way to cure babies in the bubble. We don't need the bubble anymore."


Story Source:

The above story is based on materials provided by Memorial Sloan Kettering Cancer Center. The original article was written by Jim Stallard. Note: Materials may be edited for content and length.


Journal Reference:

  1. Sung-Yun Pai, Brent R. Logan, Linda M. Griffith, Rebecca H. Buckley, Roberta E. Parrott, Christopher C. Dvorak, Neena Kapoor, Imelda C. Hanson, Alexandra H. Filipovich, Soma Jyonouchi, Kathleen E. Sullivan, Trudy N. Small, Lauri Burroughs, Suzanne Skoda-Smith, Ann E. Haight, Audrey Grizzle, Michael A. Pulsipher, Ka Wah Chan, Ramsay L. Fuleihan, Elie Haddad, Brett Loechelt, Victor M. Aquino, Alfred Gillio, Jeffrey Davis, Alan Knutsen, Angela R. Smith, Theodore B. Moore, Marlis L. Schroeder, Frederick D. Goldman, James A. Connelly, Matthew H. Porteus, Qun Xiang, William T. Shearer, Thomas A. Fleisher, Donald B. Kohn, Jennifer M. Puck, Luigi D. Notarangelo, Morton J. Cowan, Richard J. O'Reilly. Transplantation Outcomes for Severe Combined Immunodeficiency, 2000–2009. New England Journal of Medicine, 2014; 371 (5): 434 DOI: 10.1056/NEJMoa1401177

Cite This Page:

Memorial Sloan Kettering Cancer Center. "Transplantation shown to be highly effective in treating immune deficiency in children." ScienceDaily. ScienceDaily, 30 July 2014. <www.sciencedaily.com/releases/2014/07/140730173127.htm>.
Memorial Sloan Kettering Cancer Center. (2014, July 30). Transplantation shown to be highly effective in treating immune deficiency in children. ScienceDaily. Retrieved February 1, 2015 from www.sciencedaily.com/releases/2014/07/140730173127.htm
Memorial Sloan Kettering Cancer Center. "Transplantation shown to be highly effective in treating immune deficiency in children." ScienceDaily. www.sciencedaily.com/releases/2014/07/140730173127.htm (accessed February 1, 2015).

Share This


More From ScienceDaily



More Health & Medicine News

Sunday, February 1, 2015

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

CDC: Get Vaccinated for Measles

CDC: Get Vaccinated for Measles

Reuters - US Online Video (Jan. 30, 2015) The CDC is urging people to get vaccinated for measles amid an outbreak that began at Disneyland and has now infected more than 90 people. Linda So reports. Video provided by Reuters
Powered by NewsLook.com
Obama To Outline New Plan For Personalized Medicine

Obama To Outline New Plan For Personalized Medicine

Newsy (Jan. 30, 2015) President Obama is expected to speak with drugmakers Friday about his Precision Medicine Initiative first introduced last week. Video provided by Newsy
Powered by NewsLook.com
NFL Concussions Down; Still on Parents' Minds

NFL Concussions Down; Still on Parents' Minds

AP (Jan. 30, 2015) The NFL announced this week that the number of game concussions dropped by a quarter over last season. Still, the dangers of the sport still weigh on players, and parents&apos; minds. (Jan. 30) Video provided by AP
Powered by NewsLook.com
U.S. Wants to Analyze DNA from 1 Million People

U.S. Wants to Analyze DNA from 1 Million People

Reuters - US Online Video (Jan. 30, 2015) The U.S. has proposed analyzing genetic information from more than 1 million American volunteers to learn how genetic variants affect health and disease. Rough Cut (no reporter narration). Video provided by Reuters
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:

Strange & Offbeat Stories


Health & Medicine

Mind & Brain

Living & Well

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