September 18, 2024 NIH-supported clinical trial is the first positive trial for treatment of hereditary hemorrhagic telangiectasia. A clinical trial supported by the National Institutes of Health (NIH) was stopped early after researchers found sufficient evidence that a drug used to treat bone marrow cancer and Kaposi sarcoma is safe and effective in treating hereditary...
Tag: <span>blood disorder</span>
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Clinical trial demonstrates success in treating rare blood disorder
September 18, 2024 by Cleveland Clinic Credit: CC0 Public DomainA clinical trial has demonstrated that the cancer drug pomalidomide is safe and effective in treating hereditary hemorrhagic telangiectasia (HHT), a rare bleeding disorder that impacts more than one in 5,000 people worldwide. The trial, led by Keith McCrae, M.D., of Cleveland Clinic, was stopped early...
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Research reveals age significantly impacts the diagnosis of a common blood disorder
by Laura Anderson, Royal College of Surgeons in Ireland Credit: Unsplash/CC0 Public DomainNew research by RCSI University of Medicine and Health Sciences published in the Blood Journal has revealed that the age at which individuals are tested for von Willebrand disease (VWD), a common bleeding disorder, significantly affects their diagnosis. This could be key in addressing...
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Rilzabrutinib for blood disorder shows promise in phase 1–2 clinical trial
by Michael Morrison, Massachusetts General Hospital Credit: CC0 Public Domain In people with immune thrombocytopenia (ITP), the body produces destructive antibodies against platelets in the blood, which increases the risk of bruising, bleeding, hospitalization, death, fatigue, and an impaired quality of life. A drug called rilzabrutinib has generated promising safety and efficacy results in a...
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Researchers discover mechanism behind most severe cases of a common blood disorder
by SLAC National Accelerator Laboratory Credit: CC0 Public Domain With a name like glucose-6-phosphate dehydrogenase deficiency, one would think it is a rare and obscure medical condition, but that’s far from the truth. Roughly 400 million people worldwide live with potential of blood disorders due to the enzyme deficiency. While some people are asymptomatic, others suffer...
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Novel Assay Reveals Optimal Treatment in Rare Blood Disorder
Liam Davenport April 04, 2019 GLASGOW — Combining prophylactic infusions and anti-platelet therapy normalises thrombus formation in patients with congenital thrombotic thrombocytopenic purpura (TTP) under shear flow conditions, reveals a novel assay developed by UK researchers that could revolutionise both the diagnosis and management of this rare condition. Congenital TTP is an inherited deficiency in ADAMTS13 (a disintegrin and...
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Science Newsfrom research organizations First patient cured of rare blood disorder
David Levy, shown here with his sister, is the first adult patient cured of CDA. Using a technique that avoids the use of high-dose chemotherapy and radiation in preparation for a stem cell transplant, physicians at the University of Illinois Hospital & Health Sciences System have documented the first cure of an adult patient with...