Corporate Banner
Satellite Banner
Technology
Networks
Scientific Communities
 
Become a Member | Sign in
Home>News>This Article
  News
Return

Early Treatment Benefits Infants with Severe Combined Immunodeficiency

Published: Friday, August 01, 2014
Last Updated: Friday, August 01, 2014
Bookmark and Share
NIH-funded study identifies factors contributing to successful stem cell transplants.

Early transplantation of blood-forming stem cells is a highly effective treatment for infants with severe combined immunodeficiency (SCID), a group of rare, life-threatening inherited immune system disorders, a study funded by the National Institutes of Health suggests.

Approximately three-quarters of SCID infants who received transplants survived for at least five years. Infants who received transplants within the first 3.5 months of life had the best outcomes. Researchers from the Primary Immune Deficiency Treatment Consortium External Web Site Policy (PIDTC), funded by NIH’s National Institute of Allergy and Infectious Diseases (NIAID), report their findings in the July 31 issue of the New England Journal of Medicine.

SCID is caused by defects in genes involved in the development and function of infection-fighting T and B cells. Infants with SCID appear healthy at birth but are highly susceptible to infections. If untreated, SCID is fatal, usually within the first year of life. Development of a newborn screening test has made it possible to detect SCID before symptoms appear. The test was added to the U.S. Department of Health and Human Services’ Recommended Uniform Screening Panel External Web Site Policy for newborns in 2010, but to date, only 21 states External Web Site Policy have implemented newborn screening for SCID.

“The findings from this study highlight the positive impact of treating SCID early in life,” said NIAID Director Anthony S. Fauci, M.D. “They also suggest that widespread use of newborn screening tests for SCID is warranted to ensure that infants with this rare syndrome receive life-saving transplants.”

Stem cell transplantation can fully correct the T-cell and, less consistently, the B-cell deficiencies of SCID infants. To identify factors that contribute to successful transplant outcomes, PIDTC investigators analyzed data from 240 SCID infants who received transplants at 25 clinical centers across the United States and Canada between 2000 and 2009.

The researchers found that younger infants and those without infections had excellent survival rates. Almost all 68 babies transplanted within the first 3.5 months of life survived, with 64 still alive five years after transplant. Many of these babies had a family history of SCID and were diagnosed before the onset of infections. Survival rates for older infants who never had infection or whose infections cleared before transplant also were high - 90 percent and 82 percent, respectively. Only 50 percent of babies who had infections at the time of transplant survived for five years.

“These findings indicate that early transplantation and absence of infection are critical to achieving the best transplant outcomes for infants with this serious disorder,” said Daniel Rotrosen, M.D., director of NIAID’s Division of Allergy, Immunology and Transplantation. “The results of this study pave the way for further work to identify optimal stem cell transplant procedures for infants with SCID.”

Donor type also affected transplant success, with the best outcomes resulting from sibling donors whose human leukocyte antigens (HLA) - proteins that help regulate immune responses - matched those of the recipient. HLA matching reduces the risk of graft-versus-host disease, in which transplanted cells attack the recipient’s cells. Because HLA markers are inherited from both parents, siblings have a one-in-four chance of being a perfect match. In the PIDTC study, 97 percent of infants who received transplants from HLA-matched siblings survived at least five years.

Regardless of donor type, survival rates were high for infants transplanted within the first 3.5 months of life and those of any age without infection at time of transplant. SCID infants with active infection and lacking a matched sibling donor did not fare as well. These infants were most likely to survive if they received specially treated bone marrow from a parent, but did not receive any pre-transplant chemotherapy, which often is administered to help the transplanted cells survive. This finding indicates that treatment and prevention of infection and avoidance of chemotherapy if an infection cannot be cleared are important considerations before transplantation.

Transplants from matched siblings led to the best restoration of immune function. Among survivors of transplants from non-sibling donors, use of certain pre-transplant chemotherapy regimens was associated with higher T-cell numbers and more consistent B-cell function. However, chemotherapy carries the risk of severe early side effects, which can reduce chances of survival. Survivors may experience long-term chemotherapy side effects, such as poor growth. Future research will focus on developing transplant procedures that improve survival and immune recovery while avoiding harmful side effects.


Further Information

Join For Free

Access to this exclusive content is for Technology Networks Premium members only.

Join Technology Networks Premium for free access to:

  • Exclusive articles
  • Presentations from international conferences
  • Over 3,900+ scientific posters on ePosters
  • More Than 5,300+ scientific videos on LabTube
  • 35 community eNewsletters


Sign In



Forgotten your details? Click Here
If you are not a member you can join here

*Please note: By logging into TechnologyNetworks.com you agree to accept the use of cookies. To find out more about the cookies we use and how to delete them, see our privacy policy.

Related Content

Study to Assess Shorter-Duration Antibiotics in Children
Physicians plan a clinical trial to evaluate whether short course anti-biotics are effective at treating CAP in children.
Wednesday, November 30, 2016
First New HIV Vaccine Study for Seven Years Begins
South Africa hosts historic clinical trial of experimental HIV vaccine aiming to safely prevent HIV infection.
Wednesday, November 30, 2016
Antibody Protects Mice from Zika Infection
Researchers develop human-derived antibody protected pregnant mice and their developing fetuses from Zika infection.
Wednesday, November 23, 2016
Food Additives Promote Inflammation, Colon Cancer
Dietary emulsifiers promoted colon cancer in a mouse model by altering gut microbes and increasing gut inflammation.
Wednesday, November 23, 2016
Protein-Folding Gene Helps Heal Wounds
Researchers identified a protein that dramatically accelerates wound healing in animal models.
Wednesday, November 23, 2016
More Immunotherapy Options Approved for Lung Cancer
The FDA has approved a new immunotherapy drug for certain patients with non-small cell lung cancer.
Monday, November 21, 2016
Big Data for Infectious Disease Surveillance
NIH-led effort examines use of big data from health records and other digital sources for uses in infectious disease surveillance.
Tuesday, November 15, 2016
Potential Therapies Against Drug-Resistant Bacteria Identified
Researchers create new identification method for drug and drug combinations that may combat resistant infections.
Thursday, November 10, 2016
Testing Zika Vaccine in Humans Begins
The first of five planned clinical trials to test ZPIV vaccine in humans has begun.
Tuesday, November 08, 2016
Genetic Markers Predict Malaria Treatment Failure
By comparing 297 parasite genomes to a reference malaria parasite genome, researchers have identified two genetic markers that are strongly associated with the parasites’ ability to resist piperaquine.
Monday, November 07, 2016
Cannabinoid Receptor Structure Revealed
Scientists provided a detailed view of the primary molecule through which cannabinoids exert their effects on the brain. The findings might help guide the design of more targeted medicines with fewer side effects.
Wednesday, November 02, 2016
NIH Researchers Unveil New Wound-Healing Role for Protein-Folding Gene in Mice
The study found that topical treatment of an Hsp60-containing gel dramatically accelerates wound closure in a diabetic mouse model.
Friday, October 28, 2016
Ebola-Affected Countries Receive NIH Support
The National Institutes of Health has established a new program to further research capacity to study Ebola and other epidemics.
Thursday, October 27, 2016
Skin Patch to Treat Peanut Allergy
NIH-funded study suggests peanut protein patch is a safe and convenient method of treatment.
Thursday, October 27, 2016
Gene Editing Corrects Sickle Cell Mutation
Researchers demonstrate a potential pathway to developing gene-editing treatments for sickle cell disease.
Wednesday, October 26, 2016
Scientific News
Big Genetics in BC: The American Society for Human Genetics 2016 Meeting
Themes at this year's meeting ranged from the verification, validation, and sharing of data, to the translation of laboratory findings into actionable clinical results.
Stem Cells in Drug Discovery
Potential Source of Unlimited Human Test Cells, but Roadblocks Remain.
Cancer Genetics: Key to Diagnosis, Therapy
When applied judiciously, cancer genetics directs caregivers to the right drug at the right time, while sparing patients of unnecessary or harmful treatments.
BGI Sequences Gingko Tree, Revealing Large, Highly Repetitive Genome
Researchers at BGI have sequenced the more than 10-gigabase ginkgo genome to find a high number of repetitive sequences as well as a number of gene clusters that appear to be involved in defense mechanisms.
Survey of New York City Soil Uncovers Medicine-Making Microbes
Microbes have long been an invaluable source of new drugs. And to find more, we may have to look no further than the ground beneath our feet.
Accelerating the Detection of Foodborne Bacterial Outbreaks
The speed of diagnosis of foodborne bacterial outbreaks could be improved by a new technique developed by researchers at the Georgia Institute of Technology.
Making Personalized Medicine a Reality
Groundbreaking technique developed at McMaster University is helping to pave the way for advances in personalized medicine.
Scientists Identify Unique Genomic Features in Testicular Cancer
The findings may shed light on factors in other cancers that influence their sensitivity to chemotherapy.
Top 10 Life Science Innovations of 2016
2016 has seen the release of some truly innovative products. To help you digest these developments, The Scientist have listed their top picks for the year.
BioCision Forms MedCision
The new company will focus on technologies for the management and automation of vital clinical processes.
Scroll Up
Scroll Down
Skyscraper Banner

SELECTBIO Market Reports
Go to LabTube
Go to eposters
 
Access to the latest scientific news
Exclusive articles
Upload and share your posters on ePosters
Latest presentations and webinars
View a library of 1,800+ scientific and medical posters
3,900+ scientific and medical posters
A library of 2,500+ scientific videos on LabTube
5,300+ scientific videos
Close
Premium CrownJOIN TECHNOLOGY NETWORKS PREMIUM FOR FREE!