Forum for Science, Industry and Business

Sponsored by:     3M 
Search our Site:

 

Stroke Prevention Study in Children with Sickle Cell Anemia, Iron Overload Stopped Early

07.06.2010
Experimental Approach Seems no Better Than Standard Treatment

The National Heart, Lung, and Blood Institute (NHLBI) has stopped a clinical trial evaluating a new approach to reduce the risk of recurrent stroke in children with sickle cell anemia and iron overload because of evidence that the new treatment was unlikely to prove better than the existing treatment.

The 26-site trial, Stroke With Transfusions Changing to Hydroxyurea, or SWiTCH, studied 133 participants between the ages of 5 and 18 who had already experienced a stroke. All had been receiving the standard treatment of blood transfusions for at least 18 months and high levels of iron before entering the study. Without further preventive measures, these children were at high risk of another stroke as well as life-threatening conditions due to iron overload.

The study tested whether the drug hydroxyurea, known to prevent complications of sickle cell disease in adults, was as effective as transfusions, the standard therapy, in reducing the risk of recurrent strokes. Hydroxyurea is the only FDA-approved drug for treating sickle cell anemia.

The study also compared two approaches to remove excess iron, a consequence of regular blood transfusions. Participants who continued to receive transfusion therapy were given the standard oral iron-removal drug deferasirox, and participants who were switched to hydroxyurea underwent regular phlebotomy (blood removal) to eliminate excess iron that had accumulated from their earlier transfusions.

Phlebotomy did not reduce liver iron better than deferasirox therapy. Analysis of the available data indicated that continuing the trial was unlikely to show that phlebotomy would provide a greater benefit than deferasirox to control iron accumulation. Without the ability to provide benefits for the management of liver iron, the potential risks of continuing study treatments were no longer warranted.

“Protecting our participants is an important factor in determining whether to stop a trial,” said Susan B. Shurin, MD, acting director of the NHLBI, who is a board-certified hematologist and pediatrician. The NHLBI is part of the National Institutes of Health. “When an experimental treatment fails to meet its predetermined goals, it is best to return participants to standard treatment as soon as possible.”

By the time the trial was halted, approximately one-third of participants had completed the study, during which they were treated and monitored for 30 months. At enrollment, participants were randomly assigned to either the alternative or the standard treatment group. The study’s independent Data and Safety Monitoring Board (DSMB) reviewed interim results from the trial and recommended stopping the study. The NHLBI accepted the recommendation and stopped the study on May 6.

The DSMB noted that no strokes occurred in the 66 participants who received the standard therapy of blood transfusions and deferasirox. In contrast, seven strokes occurred in the group of 67 participants who received hydroxyurea with phlebotomy. Study participants and their families have been contacted, and they will discuss future care options with their health care providers.

NHLBI-supported research has shown that hydroxyurea helps prevent pain crises, a common complication of the disease, and some lung complications in adults. Preliminary studies have suggested that hydroxyurea might also help reduce the risk of stroke recurrence in children with sickle cell disease. Patients currently taking hydroxyurea should continue taking the treatment as prescribed and should talk to their primary care provider if they have any concerns, Shurin advised.

Regular blood transfusions greatly reduce the number of strokes in at-risk children with sickle cell disease. Because repeated transfusions cause side effects such as buildup of excess iron in the body, researchers hope to find more effective and safer ways to reduce stroke risk in young patients. Iron overload can damage organs and lead to cirrhosis (a liver condition), poor growth and development, and heart rhythm disturbances.

“These kinds of studies are so important to finding new treatments and confirming that current standards of care are the best available options,” Shurin explained. “The field of medicine would not advance without the help of those willing to participate in clinical research. We are grateful to these young patients and their parents for helping advance treatments of sickle cell disease. Their contributions will help us find other approaches to preventing complications of sickle cell disease.”

About 10 percent of children with sickle cell disease suffer a stroke. Having experienced one, they are at high risk of having another unless they receive preventive treatment.

Sickle cell disease affects more than 70,000 Americans. It is seen mostly in persons of African descent, but also in individuals of Middle Eastern, Mediterranean, Central and South American, and Asian Indian heritage. New therapies have helped patients live into middle age and older. Sickle cell disease involves an altered gene that produces abnormal hemoglobin, the protein that carries oxygen in the blood. Red blood cells with sickle hemoglobin become C-shaped, stiff, and sticky when they release the oxygen they carry.

The deformed cells impede blood flow, causing severe pain and organ damage.
The study was scheduled to run until 2012. Rho Inc. of Chapel Hill, N.C., served as the SWiTCH statistics and data management center. Researchers will analyze and publish the final data in the coming months. SWiTCH was conducted at the following locations:
University of Alabama at Birmingham Children’s National Medical Center, Washington, D.C.Nemours Children’s Clinic, Jacksonville, Fla. University of Miami, Fla. Nemours Children’s Clinic, Orlando, Fla. Children’s Healthcare of Atlanta at Scottish Rite Children’s Healthcare of Atlanta at Egleston Children’s Healthcare of Atlanta at GradyChildren’s Memorial Hospital, Chicago Boston Children’s Hospital Wayne State University, Detroit University of Mississippi Medical Center, Jackson The Children’s Mercy Hospital, Kansas City St. Joseph’s Children’s Hospital, Paterson, N.J. Montefiore Medical Center, New York CityState University of New York/Downstate Medical Center, New York CitySchneider Children’s Hospital, New Hyde Park, N.Y.Columbia University Medical Center, New York CityEast Carolina University, Greenville.Cincinnati Children’s Hospital The Children’s Hospital of Philadelphia Children’s Hospital of Pittsburg Medical University of South Carolina, CharlestonSt. Jude Children’s Research Hospital, Memphis, Tenn. University of Texas Southwestern Medical Center at Dallas Baylor College of Medicine, HoustonEast Virginia Medical School, Norfolk Medical College of Wisconsin, Milwaukee
Novartis US donated the deferasirox (EXJADE). Bristol-Myers Squibb and UPM Pharmaceuticals Inc. provided the hydroxyurea for the trial.
To speak with an NHLBI spokesperson about the SWiTCH trial (NCT00122980), please contact the NHLBI Communications Office at (301) 496-4236.
Part of the National Institutes of Health, the National Heart, Lung, and Blood Institute (NHLBI) plans, conducts, and supports research related to the causes, prevention, diagnosis, and treatment of heart, blood vessel, lung, and blood diseases; and sleep disorders. The Institute also administers national health education campaigns on women and heart disease, healthy weight for children, and other topics. NHLBI press releases and other materials are available online at www.nhlbi.nih.gov.
The National Institutes of Health (NIH) — The Nation's Medical Research Agency — includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. It is the primary federal agency for conducting and supporting basic, clinical and translational medical research, and it investigates the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit www.nih.gov.
Resources:
Diseases and Conditions Index: Sickle Cell Disease: http://www.nhlbi.nih.gov/health/dci/Diseases/Sca/SCA_Treatments.html
NIH Consensus Development Conference Statement: Hydroxyurea Treatment for Sickle Cell Disease. http://www.guideline.gov/summary/summary.aspx?ss=15&doc_id=12656&nbr=6551

Press Release on Consensus Document http://public.nhlbi.nih.gov/newsroom/home/GetPressRelease.aspx?id=2552

NHLBI Communications Office | EurekAlert!
Further information:
http://www.nhlbi.nih.gov

More articles from Studies and Analyses:

nachricht WAKE-UP provides new treatment option for stroke patients | International study led by UKE
17.05.2018 | Universitätsklinikum Hamburg-Eppendorf

nachricht First form of therapy for childhood dementia CLN2 developed
25.04.2018 | Universitätsklinikum Hamburg-Eppendorf

All articles from Studies and Analyses >>>

The most recent press releases about innovation >>>

Die letzten 5 Focus-News des innovations-reports im Überblick:

Im Focus: Powerful IT security for the car of the future – research alliance develops new approaches

The more electronics steer, accelerate and brake cars, the more important it is to protect them against cyber-attacks. That is why 15 partners from industry and academia will work together over the next three years on new approaches to IT security in self-driving cars. The joint project goes by the name Security For Connected, Autonomous Cars (SecForCARs) and has funding of €7.2 million from the German Federal Ministry of Education and Research. Infineon is leading the project.

Vehicles already offer diverse communication interfaces and more and more automated functions, such as distance and lane-keeping assist systems. At the same...

Im Focus: Molecular switch will facilitate the development of pioneering electro-optical devices

A research team led by physicists at the Technical University of Munich (TUM) has developed molecular nanoswitches that can be toggled between two structurally different states using an applied voltage. They can serve as the basis for a pioneering class of devices that could replace silicon-based components with organic molecules.

The development of new electronic technologies drives the incessant reduction of functional component sizes. In the context of an international collaborative...

Im Focus: LZH showcases laser material processing of tomorrow at the LASYS 2018

At the LASYS 2018, from June 5th to 7th, the Laser Zentrum Hannover e.V. (LZH) will be showcasing processes for the laser material processing of tomorrow in hall 4 at stand 4E75. With blown bomb shells the LZH will present first results of a research project on civil security.

At this year's LASYS, the LZH will exhibit light-based processes such as cutting, welding, ablation and structuring as well as additive manufacturing for...

Im Focus: Self-illuminating pixels for a new display generation

There are videos on the internet that can make one marvel at technology. For example, a smartphone is casually bent around the arm or a thin-film display is rolled in all directions and with almost every diameter. From the user's point of view, this looks fantastic. From a professional point of view, however, the question arises: Is that already possible?

At Display Week 2018, scientists from the Fraunhofer Institute for Applied Polymer Research IAP will be demonstrating today’s technological possibilities and...

Im Focus: Explanation for puzzling quantum oscillations has been found

So-called quantum many-body scars allow quantum systems to stay out of equilibrium much longer, explaining experiment | Study published in Nature Physics

Recently, researchers from Harvard and MIT succeeded in trapping a record 53 atoms and individually controlling their quantum state, realizing what is called a...

All Focus news of the innovation-report >>>

Anzeige

Anzeige

VideoLinks
Industry & Economy
Event News

In focus: Climate adapted plants

25.05.2018 | Event News

Save the date: Forum European Neuroscience – 07-11 July 2018 in Berlin, Germany

02.05.2018 | Event News

Invitation to the upcoming "Current Topics in Bioinformatics: Big Data in Genomics and Medicine"

13.04.2018 | Event News

 
Latest News

In focus: Climate adapted plants

25.05.2018 | Event News

Flow probes from the 3D printer

25.05.2018 | Machine Engineering

Less is more? Gene switch for healthy aging found

25.05.2018 | Life Sciences

VideoLinks
Science & Research
Overview of more VideoLinks >>>