Corporate Banner
Satellite Banner
Pharma Outsourcing
Scientific Community
 
Become a Member | Sign in
Home>News>This Article
  News
Return

Monthly Blood Transfusions Reduce Sickle Cell Anemia-related Brain Injury in Children

Published: Friday, August 22, 2014
Last Updated: Friday, August 22, 2014
Bookmark and Share
NIH-funded study provides hope for children with disease-related brain damage.

Regular blood transfusions prevent recurrent blockage of brain blood vessels, a serious neurological side effect that occurs in one third of children with sickle cell anemia, according to a study funded by the National Institutes of Health. The findings appear in the August 21 issue of the New England Journal of Medicine.

In the United States, approximately 100,000 people, primarily African-Americans, live with sickle cell anemia, a genetic disorder in which red blood cells, which should be round, can take on abnormal, crescent-like shapes. These sickle cells are stickier than regular cells and can block blood flow, resulting in pain, organ damage and strokes.

Cerebral infarcts occur when blood vessels in the brain get blocked. Silent cerebral infarcts do not cause any obvious symptoms, so people are unaware that something is wrong. The only way to detect a silent stroke is with magnetic resonance imaging (MRI) scans of the brain, which are not routinely conducted in people with sickle cell anemia. Silent infarcts in children with sickle cell disease are known to be associated with long-term cognitive problems and poor academic performance.

“If scientists detect silent strokes early, young children can benefit from a wide array of educational interventions and can get support from federally mandated educational assistance programs to keep them from falling behind in school,” said Michael R. DeBaun, M.D., from Vanderbilt University, Nashville, the lead author and principal investigator for the trial.

Previous studies have demonstrated that blood transfusions may help prevent stroke in patients with sickle cell anemia. Transfusions of healthy blood increase the number of normal red blood cells and make the occurrence of blocked blood vessels less likely. In the current study, Dr. DeBaun and his colleagues investigated whether monthly blood transfusions would help prevent recurrence of silent cerebral infarcts in children with sickle cell anemia who had evidence of a previous incident.

In this multi-center international clinical trial, 1,074 children had MRI scans and 35 percent had silent infarcts. The 196 children with sickle cell anemia who exhibited infarct-like lesions on brain scans were randomized to receive regular blood transfusions or standard medical care. The blood transfusions were administered every month for three years.

The investigators’ results show that receiving blood transfusions on a regular basis prevented the recurrence of silent strokes. In the standard medical care group, 14.4 percent of patients experienced a stroke or new silent cerebral infarcts. Among patients who received regular blood transfusions, only 6.1 percent experienced a stroke or new silent cerebral infarct.

“The study provides clear evidence that we may be able to decrease the progression of silent strokes, which occur in 30 percent of children with sickle cell anemia. These results suggest that children who have this disease should be screened early for silent strokes, at least by the time they begin elementary school, to help them manage the disease and to ensure minimal impact on school performance,” said Dr. DeBaun.

The researchers noted that this trial included a very intense regimen for the patients, who were as young as 5 years old, and for their families. “One of the reasons we conducted this study was to check if the burden of the blood transfusion therapy, such as monthly doctor’s visits, was worth the potential benefits,” said Deborah Hirtz, M.D., program director at the NIH’s National Institute of Neurological Disorders and Stroke, which provided funding for the study. Some risks linked to blood transfusions include severe allergic reactions and high levels of iron throughout the body.

“The commitment of the children and their parents, many of whom were working poor families, was quite impressive. Despite the burden of a three-year intensive trial, 86 percent of the participants completed all aspects of the trial including 36 months of transfusion therapy, three non-sedated MRI scans, multiple neurology evaluations, two two-hour cognitive testing sessions, and two quality of life assessments,” said Dr. DeBaun.

An unexpected result, according to the researchers, was that intelligence measures were not different between the two study groups. Previous studies from this trial and others have shown that silent strokes are associated with a five-point reduction in IQ. This issue will be further explored by the investigators.

“The study indicates that screening for silent strokes in children starting school can lead to early detection and prevention of recurrences as well as reduction in other complications of sickle cell anemia such as acute episodes of pain and acute chest syndrome. The results of this trial will make a difference to children with sickle cell anemia and their families,” said Dr. Hirtz.


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 3,100+ scientific posters on ePosters
  • More than 4,500+ 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

Investigational Malaria Vaccine Protects Healthy U.S. Adults
Researchers at NIH have found that the malaria vaccine protected a small number of healthy, malaria-naïve adults in the U.S. from infection for more than one year after immunization.
Tuesday, May 10, 2016
Study Finds Factors That May Influence Influenza Vaccine Effectiveness
Researchers at NIH have suggested that the long-held approach to predicting seasonal influenza vaccine effectiveness may need to be revisited.
Wednesday, April 20, 2016
Submissions Open for the Cancer Moonshot Program
NCI opens online platform to submit ideas about research for Cancer Moonshot.
Tuesday, April 19, 2016
Promising Experimental Dengue Vaccine
A clinical trial in which volunteers were infected with dengue virus six months after receiving either an experimental dengue vaccine or a placebo injection yielded starkly contrasting results.
Thursday, March 17, 2016
Eylea Outperforms Avastin for Diabetic Macular Edema with Moderate or Worse Vision Loss
NIH-funded clinical trial shows Eylea, Avastin, and Lucentis perform similarly when vision loss is mild.
Wednesday, March 02, 2016
Vaginal Ring Provides Partial Protection From HIV In Large Multinational Trial
Study finds protective effect is strongest in women over age 25.
Friday, February 26, 2016
Experimental Ebola Antibody Protects Monkeys
Antibody isolated from Ebola survivor can advance to clinical trials.
Friday, February 26, 2016
Experimental Combination Surprises with Anti-HIV Effectiveness
A compound developed to protect the nervous system from HIV surprised researchers by augmenting the effectiveness of an investigational antiretroviral drug beyond anything expected.
Monday, January 25, 2016
Dengue Vaccine Enters Phase 3 Trial
Investigational vaccine to prevent ‘breakbone fever’ developed at NIH.
Friday, January 15, 2016
Trying to Conceive Soon After a Pregnancy Loss May Increase Chances of Live Birth
NIH study finds no reason for delaying pregnancy attempts after a loss without complications.
Wednesday, January 13, 2016
NIH-funded Memory Drug Moves into Phase 1 Clinical Study
Collaboration between NIH and Tetra Discovery Partners leads to development of treatment that may affect cognition.
Monday, January 04, 2016
NIH Unveils FY2016–2020 Strategic Plan
Detailed plan sets course for advancing scientific discoveries and human health.
Thursday, December 17, 2015
Lucentis Effective for Proliferative Diabetic Retinopathy
NIH-funded clinical trial marks first major advance in therapy in 40 years.
Tuesday, November 24, 2015
Gene Therapy Staves Off Blindness from Retinitis Pigmentosa in Canine Model
NIH-funded study suggests therapeutic window may extend to later-stage disease.
Tuesday, October 20, 2015
Scientists Test New Gene Therapy for Vision Loss from a Mitochondrial Disease
NIH-funded study shows success in targeting mitochondrial DNA in mice.
Tuesday, October 06, 2015
Scientific News
Investigational Malaria Vaccine Protects Healthy U.S. Adults
Researchers at NIH have found that the malaria vaccine protected a small number of healthy, malaria-naïve adults in the U.S. from infection for more than one year after immunization.
AACR 2016: Cancer Immunotherapy and Beyond
At this year's meeting there was a palpable buzz around subjects ranging from microbiomics to the tumor microenvironment and cancer vaccines, big data to in vitro and in vivo modeling and drug delivery (to name just a few).
New Database for Sharing MS Clinical Trial Data
A new database containing nearly 2500 patient records from the placebo arms of nine multiple sclerosis (MS) clinical trials is now available for research by qualified investigators.
Study Finds Factors That May Influence Influenza Vaccine Effectiveness
Researchers at NIH have suggested that the long-held approach to predicting seasonal influenza vaccine effectiveness may need to be revisited.
BMS’s Opdivo Clinical Trial Shows Promise
Safety profile of the combination regimen from CheckMate -069 was consistent with previously reported studies and adverse events were managed using established safety algorithms.
Treatment of Common Prostate Cancer
Researchers at UTSW have found that the prostate cancer treatments suppress immune response and may promote relapse.
Cancer Drug Could Treat Blood Vessel Deformities
A drug currently being trialled in cancer patients could also be used to treat an often incurable condition that can cause painful blood vessel overgrowths inside the skin.
Structure of Crucial Enzyme Identified
Researchers at UTSW have determined the atomic structure of an enzyme that plays an essential role in cell division and better treatment of cancer.
New Immunotherapy Trial for Type 1 Diabetes
The search for a treatment for Type 1 diabetes (T1D) - which affects over 400,000 people in the UK – will be stepped up with the start of a new phase one clinical trial at Guy’s Hospital in London.
Recruitment of First Patient in Clinical Study
Company has announced recruitment of first patient in clinical study assessing Visco-ease with Beatson Cancer Centre for the treatment of RIX.
Scroll Up
Scroll Down
SELECTBIO

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
3,100+ scientific and medical posters
A library of 2,500+ scientific videos on LabTube
4,500+ scientific videos
Close
Premium CrownJOIN TECHNOLOGY NETWORKS PREMIUM FOR FREE!