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 New study: How does Europe become a leading player for software and IT services?
03.04.2017 | Fraunhofer-Institut für System- und Innovationsforschung (ISI)

nachricht Reusable carbon nanotubes could be the water filter of the future, says RIT study
30.03.2017 | Rochester Institute of Technology

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: Strathclyde-led research develops world's highest gain high-power laser amplifier

The world's highest gain high power laser amplifier - by many orders of magnitude - has been developed in research led at the University of Strathclyde.

The researchers demonstrated the feasibility of using plasma to amplify short laser pulses of picojoule-level energy up to 100 millijoules, which is a 'gain'...

Im Focus: Can the immune system be boosted against Staphylococcus aureus by delivery of messenger RNA?

Staphylococcus aureus is a feared pathogen (MRSA, multi-resistant S. aureus) due to frequent resistances against many antibiotics, especially in hospital infections. Researchers at the Paul-Ehrlich-Institut have identified immunological processes that prevent a successful immune response directed against the pathogenic agent. The delivery of bacterial proteins with RNA adjuvant or messenger RNA (mRNA) into immune cells allows the re-direction of the immune response towards an active defense against S. aureus. This could be of significant importance for the development of an effective vaccine. PLOS Pathogens has published these research results online on 25 May 2017.

Staphylococcus aureus (S. aureus) is a bacterium that colonizes by far more than half of the skin and the mucosa of adults, usually without causing infections....

Im Focus: A quantum walk of photons

Physicists from the University of Würzburg are capable of generating identical looking single light particles at the push of a button. Two new studies now demonstrate the potential this method holds.

The quantum computer has fuelled the imagination of scientists for decades: It is based on fundamentally different phenomena than a conventional computer....

Im Focus: Turmoil in sluggish electrons’ existence

An international team of physicists has monitored the scattering behaviour of electrons in a non-conducting material in real-time. Their insights could be beneficial for radiotherapy.

We can refer to electrons in non-conducting materials as ‘sluggish’. Typically, they remain fixed in a location, deep inside an atomic composite. It is hence...

Im Focus: Wafer-thin Magnetic Materials Developed for Future Quantum Technologies

Two-dimensional magnetic structures are regarded as a promising material for new types of data storage, since the magnetic properties of individual molecular building blocks can be investigated and modified. For the first time, researchers have now produced a wafer-thin ferrimagnet, in which molecules with different magnetic centers arrange themselves on a gold surface to form a checkerboard pattern. Scientists at the Swiss Nanoscience Institute at the University of Basel and the Paul Scherrer Institute published their findings in the journal Nature Communications.

Ferrimagnets are composed of two centers which are magnetized at different strengths and point in opposing directions. Two-dimensional, quasi-flat ferrimagnets...

All Focus news of the innovation-report >>>

Anzeige

Anzeige

Event News

Marine Conservation: IASS Contributes to UN Ocean Conference in New York on 5-9 June

24.05.2017 | Event News

AWK Aachen Machine Tool Colloquium 2017: Internet of Production for Agile Enterprises

23.05.2017 | Event News

Dortmund MST Conference presents Individualized Healthcare Solutions with micro and nanotechnology

22.05.2017 | Event News

 
Latest News

New photocatalyst speeds up the conversion of carbon dioxide into chemical resources

29.05.2017 | Life Sciences

NASA's SDO sees partial eclipse in space

29.05.2017 | Physics and Astronomy

New drug reduces transplant and mortality rates significantly in patients with hepatitis C

29.05.2017 | Statistics

VideoLinks
B2B-VideoLinks
More VideoLinks >>>