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Children's national co-leads nationwide study of landmark sickle cell treatment
August 19, 2008WASHINGTON, DC-Children's National Medical Center immunologist and blood and marrow transplant physician Naynesh Kamani, MD, will serve as the study co-chair for a new national clinical trial of unrelated donor marrow and umbilical cord blood transplants for severe sickle cell disease. Children's National will join more than 20 other medical institutions in the first-ever Phase II clinical trial of this treatment. If successful, the Sickle Cell Unrelated Transplant (SCURT) trial has the potential to extend a promising and possibly curative treatment option to more severely affected patients. Sickle cell disease affects approximately 70,000 people in the United States.
For many years, doctors have used blood or marrow transplants to treat severe cases of sickle cell disease. However these treatments have been available only to patients with a matched family member to donate blood or marrow stem cells. Only 14 percent of severe sickle cell cases have a matched donor within their family. Successful transplants using unrelated donors could create a viable treatment option for more sickle cell patients.
"Blood and marrow transplantation from unrelated donors has been routinely utilized to treat treatment-refractory leukemia and lymphoma for almost two decades," said Dr. Kamani, co-principal investigator of the study. "Preliminary results from the few unrelated donor transplants that have been performed for other non-malignant conditions show great promise for those who suffer a severely diminished quality of life due to sickle cell disease. Participants in this large-scale study will help us understand how to better treat, and possibly even cure, sickle cell disease for more people."
Starting in late August, the clinical trial will enroll 45 patients ages 3 to 16 with severe symptoms of sickle cell disease, including stroke, recurring acute chest syndrome, or frequent pain crises. For all marrow and cord blood transplants, patients must undergo intense chemotherapy to prevent their immune systems from rejecting donor cells. This study will use a reduced-intensity chemotherapy to minimize side effects and increase the likelihood that the donor cells will be accepted by the body. After the transplant, participants will be followed for two years to gauge the effectiveness of the transplant at minimizing the ravages of sickle cell disease.
Children's National Medical Center
Starting in late August, the clinical trial will enroll 45 patients ages 3 to 16 with severe symptoms of sickle cell disease, including stroke, recurring acute chest syndrome, or frequent pain crises. For all marrow and cord blood transplants, patients must undergo intense chemotherapy to prevent their immune systems from rejecting donor cells. This study will use a reduced-intensity chemotherapy to minimize side effects and increase the likelihood that the donor cells will be accepted by the body. After the transplant, participants will be followed for two years to gauge the effectiveness of the transplant at minimizing the ravages of sickle cell disease.
Children's National Medical Center
Sickle Cell Current Events and Sickle Cell News RSSChildren's Hospital study demonstrates how bone marrow transplant can cure sickle cell disease
A unique approach to bone marrow transplantation pioneered in part by a Children's Hospital of Pittsburgh of UPMC physician has proven to be the only safe and effective cure for sickle cell disease, according to a new study.
Most generalists reluctant to provide primary care for young adults with chronic illness
The majority of general internists and pediatricians in the United States are not comfortable serving as primary care providers for young adults with complex chronic illnesses that originate during childhood, according to findings from a new national survey.
Consortium develops new method to manipulate genetic material
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Gaining ground on sickle cell disease
Although sickle cell disease is a single-gene disorder, its symptoms are highly variable. In a study published online July 14 by the Proceedings of the National Academy of Sciences, scientists at Children's Hospital Boston and the Dana Farber Cancer Institute (DFCI), in collaboration with the Broad Institute of MIT and Harvard, report five gene variants that could potentially be helpful in predicting sickle cell disease severity, perhaps even leading to better treatment approaches in the future.
Effective treatment for sickle cell underused by doctors
Uncertainties about proper use and possible long-term effects of hydroxyurea in the treatment of sickle cell anemia may be wrongly influencing doctors to avoid prescribing it to those in serious need, according to results of a literature review by specialists at Johns Hopkins.
Statistical tool could explain gene study variations
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Major 'missed' biochemical pathway emerges as important in virtually all cells
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For children with sickle cell disease, lung disease is part of the package
Children with sickle cell disease (SCD) have a significantly sharper decline in lung function with age when compared to other children of the same race and age.
Clumps of red and white blood cells may contribute to sickle cell disease
It's long been known that patients with sickle cell disease have malformed, "sickle-shaped" red blood cells - which are normally disc-shaped - that can cause sudden painful episodes when they block small blood vessels.
Researchers at UCLA engineering discover theoretical model to predict jamming
Researchers at the UCLA Henry Samueli School of Engineering and Applied Science have come up with a theoretical model to predict when granular materials become jammed.
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