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

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
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 2,800+ scientific posters on ePosters
  • More than 4,000+ 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 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

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
NIH Framework Points The Way Forward For Developing The President’s Precision Medicine Initiative
The NIH Advisory Committee to the Director has presented to NIH Director Francis S. Collins, M.D., Ph.D., a detailed design framework for building a national research participant group, called a cohort, of 1 million or more Americans to expand our knowledge and practice of precision medicine.
Monday, September 21, 2015
Landmark NIH Study Shows Intensive Blood Pressure Management May Save Lives
Lower blood pressure target greatly reduces cardiovascular complications and deaths in older adults.
Saturday, September 12, 2015
NIH Study Finds Calorie Restriction Lowers Some Risk Factors for Age-Related Diseases
Two-year trial did not produce expected metabolic changes, but influenced other life span markers.
Wednesday, September 02, 2015
NIH Study Shows No Benefit of Omega-3 Supplements for Cognitive Decline
Research was published in the Journal of the American Medical Association.
Thursday, August 27, 2015
NIH Launches Human RSV Study
Study aims to understand infection in healthy adults to aid development of RSV medicines, vaccines.
Thursday, August 27, 2015
Dr. Peter Kilmarx Appointed Deputy Director of Fogarty International Center
An expert in infectious disease research and HIV/AIDS prevention.
Wednesday, August 12, 2015
Benefits of Early Antiretroviral Therapy in HIV Infection
Scientists have explored the clinical importance of starting treatment early in individuals suffering with HIV.
Tuesday, August 11, 2015
Young South African Women can Adhere to Daily PrEP Regimen as HIV Prevention
NIH-funded study finds men in Bangkok, Harlem also successful in taking daily dose.
Saturday, July 25, 2015
Study Shows Promise of Precision Medicine for Most Common Type of Lymphoma
The study appeared online July 20, 2015, in Nature Medicine.
Tuesday, July 21, 2015
HIV Control Through Treatment Durably Prevents Heterosexual Transmission of Virus
NIH-funded trial proves suppressive antiretroviral therapy for HIV-infected people effective in protecting uninfected partners.
Tuesday, July 21, 2015
Early Antiretroviral Therapy Prevents Non-AIDS Outcomes in HIV-infected People
NIH-supported findings illustrate manifold benefit of therapy.
Tuesday, July 21, 2015
NIH-funded Vaccine for West Nile Virus Enters Human Clinical Trials
Enrollment is expected to be completed by December 2015.
Tuesday, July 07, 2015
Scientific News
Lucentis Effective for Proliferative Diabetic Retinopathy
NIH-funded clinical trial marks first major advance in therapy in 40 years.
Blocking the Transmission Of Malaria Parasites
Vaccine candidate administered for the first time in humans in a phase I clinical trial led by Oxford University’s Jenner Institute, with partners Imaxio and GSK.
First Therapy Appearing to Reverse Decline in Parkinson’s
An FDA-approved drug for leukemia improved cognition, motor skills and non-motor function in patients with Parkinson’s disease and Lewy body dementia in a small clinical trial, say researchers at Georgetown University Medical Center (GUMC).
Gene Therapy Staves Off Blindness from Retinitis Pigmentosa in Canine Model
NIH-funded study suggests therapeutic window may extend to later-stage disease.
Treatment for Rare Bleeding Disorder is Effective
Researchers in Manchester have demonstrated for the first time the relative safety and effectiveness of treatment, eltrombopag, in children with persistent or chronic immune thrombocytopenia (ITP), as part of an international duo of studies.
HIV Vaccine Human Trials Begin
Baltimore-based Institute has begun enrolling volunteers for initial phase 1 clinical trials.
New Therapy Reduces Symptoms of Inherited Enzyme Deficiency
A phase three clinical trial of a new enzyme replacement medication, sebelipase alfa, showed a reduction in multiple disease-related symptoms in children and adults with lysosomal acid lipase deficiency, an inherited enzyme deficiency that can result in scarring of the liver and high cholesterol.
Fixing Holes in the Heart Without Invasive Surgery
UV-light enabled catheter is a medical device which represents a major shift in how cardiac defects are repaired.
Atriva Therapeutics GmbH Develops Innovative Flu Drug
Highly effective against seasonal and pandemic influenza.
Study Removes Cancer Doubt for Multiple Sclerosis Drug
Researchers from Queen Mary University of London are calling on the medical community to reconsider developing a known drug to treat people with relapsing Multiple sclerosis after new evidence shows it does not increase the risk of cancer as previously thought.
Scroll Up
Scroll Down

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
2,800+ scientific and medical posters
A library of 2,500+ scientific videos on LabTube
4,000+ scientific videos