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 3,500+ scientific posters on ePosters
  • More Than 5,000+ 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

Drug to Treat Alcohol Use Disorder Shows Promise Among Drinkers With High Stress
The findings suggest that potential future studies with drugs targeting vasopressin blockade should focus on populations of people with AUD who also report high levels of stress.
Friday, September 30, 2016
Monkeys Protected by Zika DNA Vaccine
Experimental Zika virus DNA vaccines successfully protected monkeys against Zika infection.
Thursday, September 29, 2016
Probe Identifies Schizophrenia Genes That Stunt Brain Development
Scientists have isolated schizophrenia-related gene variants that change gene expression in the brain.
Thursday, September 29, 2016
Developing Novel Ear Infection Treatments
Research team engineers antibiotic gel for treating middle ear infections.
Wednesday, September 28, 2016
“Sixth Sense” More Than a Feeling
NIH study of rare genetic disorder reveals importance of touch and body awareness.
Monday, September 26, 2016
“Sixth Sense” May Be More Than Just A Feeling
The NIH Study shows that two young patients with a mutation in the PIEZ02 have problems with touch and proprioception, or body awareness.
Friday, September 23, 2016
The Genetics of Blood Pressure
Researchers have identifed areas of the genome associated with blood-pressure including 17 previously unknown loci.
Wednesday, September 21, 2016
NIH Study Finds Link Between Depression, Gestational Diabetes
Researchers at NIH have discovered that the depression in early pregnancy doubles risk for gestational diabetes, and gestational diabetes increases risk for postpartum depression.
Tuesday, September 20, 2016
Detecting Bacterial Infections in Newborns
Researchers tested an alternative way to diagnose bacterial infections in infants—by analyzing RNA biosignatures from a small blood sample.
Wednesday, September 14, 2016
Finding Compounds That Inhibit Zika
Researchers identified compounds that inhibit the Zika virus and reduce its ability to kill brain cells.
Wednesday, September 14, 2016
Seeking Innovation to Combat Antimicrobial Resistance
Federal prize competition, with $20 million in prizes, seeks to develop new laboratory diagnostic tools to detect and distinguish antibiotic resistant bacteria.
Friday, September 09, 2016
Genetic Misdiagnoses of Heart Condition
Analysis found several genetic variations previously linked with a heart condition were harmless, leading to condition misdiagnosis.
Wednesday, September 07, 2016
Catalogue of Human Genetic Diversity Expands
The largest data set of human exomes to date has been assembled to better study seqence variants and their consequences.
Wednesday, September 07, 2016
Extreme Temperatures Could Increase Preterm Birth Risk
Researchers at NIH have found more preterm births among women exposed to extremes of hot and cold.
Friday, September 02, 2016
$12.4M Awarded to Neural Regeneration Projects
The National Institutes of Health will fund six projects to identify biological factors that influence neural regeneration.
Friday, September 02, 2016
Scientific News
Point of Care Diagnostics - A Cautious Revolution
Advances in molecular biology, coupled with the miniaturization and improved sensitivity of assays and devices in general, have enabled a new wave of point-of-care (POC) or “bedside” diagnostics.
Mass Spec Technology Drives Innovation Across the Biopharma Workflow
With greater resolving power, analytical speed, and accuracy, new mass spectrometry technology and techniques are infiltrating the biopharmaceuticals workflow.
One Step Closer to Precision Medicine for Chronic Lung Disease Sufferers
A study led by University of North Carolina at Chapel Hill, and National Jewish Health, has provided evidence of links between SNPs and known COPD blood protein biomarkers.
Charles River Acquires Agilux
Enhances Charles River’s early-stage capabilities in bioanalytical services.
Scientists Find Lethal Vulnerability in Treatment-Resistant Lung Cancer
The study describes how the drug Selinexor killed lung cancer cells and shrank tumors in mice when used against cancers driven by the aggressive and difficult-to-treat KRAS cancer gene.
How Baby’s Genes Influence Birth Weight And Later Life Disease
The large-scale study could help to target new ways of preventing and treating these diseases.
Genes Underlying Dogs’ Social Ability Revealed
The social ability of dogs is affected by genes that also seem to influence human behaviour, according to a new study from Linköping University in Sweden.
‘Cellbots’ Chase Down Cancer, Deliver Drugs Directly to Tumors
Programmable T cells shown to be versatile, precise, and powerful in lab studies.
Drug to Treat Alcohol Use Disorder Shows Promise Among Drinkers With High Stress
The findings suggest that potential future studies with drugs targeting vasopressin blockade should focus on populations of people with AUD who also report high levels of stress.
C Dots Show Powerful Tumor Killing Effect
Nanoparticles known as Cornell dots, or C dots, have shown great promise as a therapeutic tool in the detection and treatment of cancer.
Scroll Up
Scroll Down
Skyscraper Banner

Skyscraper Banner
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
3,500+ scientific and medical posters
A library of 2,500+ scientific videos on LabTube
5,000+ scientific videos
Close
Premium CrownJOIN TECHNOLOGY NETWORKS PREMIUM FOR FREE!