Researchers at Johns Hopkins have successfully corrected a genetic error in stem cells from patients with sickle cell disease, and then used those cells to grow mature red blood cells, they report. The study represents an important step toward more effectively treating certain patients with sickle cell disease who need frequent blood transfusions and currently have few options.
Treatment for painful episodes of blood vessel obstruction in sickle cell anemia is currently limited to controlling pain, but an investigational therapy might be able to interfere with the underlying cause of these events, known as vaso-occlusion crises, researchers at Duke Medicine report.
A University of Colorado Cancer Center study published in the journal Pediatric Blood & Cancer shows that pediatric cancer patients who receive antibiotics within 60 minutes of reporting fever and showing neutropenia (low neutrophil count), go on to have decreased intensive care needs and lower mortality compared with patients who receive antibiotics outside the 60-minute window.
Viral therapy for childhood cancer could possibly improve if treatments such as chemotherapy do not first suppress patients’ immune systems, according to findings published today in the journal Molecular Therapy—Oncolytics.
People hospitalized with certain rare blood cell disorders frequently receive a treatment that is associated with a two- to fivefold increase in death, according to a new study that reviewed hospital records nationwide. The study authors recommend that for these rare disorders, doctors should administer the treatment, a platelet transfusion, only in exceptional circumstances.
Innovative scoring system uses “Ann Arbor GVHD score” to better predict how patients will respond, minimize side effects
Enzymes linked to diabetes and obesity appear to play key roles in arthritis and leukemia, potentially opening up new avenues for treating these diverse diseases, according to researchers at Washington University School of Medicine in St. Louis.
A new test may reveal which patients will respond to treatment for graft versus host disease (GVHD).
Two studies published in the January issue of Anesthesiology, the official medical journal of the American Society of Anesthesiologists® (ASA®), shed new light on the prevalence of transfusion-related acute lung injury (TRALI) and transfusion-associated circulatory overload (TACO), the number one and two leading causes of blood transfusion-related deaths in the United States.
If approved, Iomab-B should increase the number of patients eligible for curative bone marrow transplant (BMT, also known as HSCT) and improve clinical outcomes.
Since intensive chemotherapy is associated with a high mortality rate and limited benefits to high risk patients, low intensity treatments such as Actimab-A could potentially extend overall survival in elderly patients while significantly decreasing traditional chemotherapy related toxicities, including treatment related early mortality.
A study that investigated the potential of the compound PRM-151 (PRM) for reducing progressive bone marrow fibrosis (scarring) in patients with advanced myelofibrosis has shown initial positive results. Myelofibrosis is a life-threatening bone marrow cancer.
Study results of CD19-directed chimeric antigen receptor (CAR) therapy using the Sleeping Beauty non-viral transduction system to modify T cells has demonstrated further promise in patients with advanced hematologic malignancies.
The latest results of clinical trials of more than 125 patients testing an investigational personalized cellular therapy known as CTL019 will be presented by a University of Pennsylvania research team at the 56th American Society of Hematology Annual Meeting and Exposition. Highlights of the new trial results will include a response rate of more than 90 percent among pediatric acute lymphoblastic leukemia patients, and results from the first lymphoma trials testing the approach, including a 100 percent response rate among follicular lymphoma patients and 45 percent response rate among those with diffuse large B-cell lymphoma.
A phase two study that investigated the potential of the drugs azacitidine (AZA) and lenalidomide (LEN), demonstrated that the two therapies in combination may be an effective frontline treatment regimen for patients with higher-risk forms of myelodysplastic syndrome and acute myeloid leukemia.
Patients who relapse in their battle with acute myeloid leukemia (AML) may benefit from a phase three study of therapies that combine an existing agent, cytarabine, with a newer compound, vosaroxin.
A new type of cancer therapy that targets an oncometabolite produced dramatic results in patients with advanced leukemia in an early-phase clinical trial.
In recent years, a number of scientific breakthroughs have led to the development of drugs that unleash the power of the immune system to recognize and attack cancer. For Classical Hodgkin lymphoma patients, two phase I studies are already demonstrating dramatic results.
In the treatment of multiple myeloma, the addition of carfilzomib to a currently accepted two-drug combination produced significantly better results than using the two drugs alone, according to a worldwide research team led by investigators from Mayo Clinic.
Gene therapy developed at St. Jude Children’s Research Hospital, University College London (UCL) and the Royal Free Hospital has transformed life for men with a severe form of hemophilia B by providing a safe, reliable source of the blood clotting protein Factor IX that has allowed some to adopt a more active lifestyle, researchers reported. The results appear in the November 20 edition of The New England Journal of Medicine.
Women who take a common type of medication to control their blood pressure are not at increased risk of developing breast cancer due to the drug, according to new study by researchers at the Intermountain Medical Center Heart Institute in Murray, Utah.
New evidence that suggests patients with a history of adverse reaction to the blood thinner heparin may be ready for urgent heart surgery sooner with a combination of appropriate blood screenings and therapeutic plasma exchange.
A team of researchers from Johns Hopkins Medicine has discovered a receptor on blood vessels that causes the vessel to relax in response to light, making it potentially useful in treating vascular diseases. In addition, researchers discovered a previously unknown mechanism by which blood vessel function is regulated through light wavelength.
Researchers at the University of California, San Diego School of Medicine describe the surprising and crucial involvement of a pro-inflammatory signaling protein in the creation of hematopoietic stem cells (HScs) during embryonic development, a finding that could help scientists to finally reproduce HSCs for therapeutic use.
Interim data from an ongoing Phase I/II trial of Actimab-A, an innovative targeted payload immunotherapy, demonstrated a number of positive findings, including extension of overall survival and significant reductions in bone marrow blasts in older patients with newly diagnosed Acute Myeloid Leukemia (AML).
Researchers at UC Davis have found that the drug bortezomib effectively treats chronic graft-versus-host disease (GVHD), a common and debilitating side effect from allogeneic hematopoietic stem cell transplants. The trial showed that bortezomib provides better outcomes than existing treatments and does not impair the immune response against residual cancer cells, or the graft-versus-tumor effect (GVT).
A solitary cell containing a unique abnormality can result in certain types of blood cancers known as myeloproliferative neoplasms (MPN), according to researchers in Switzerland. The results open new opportunities to examine single mutant cells and follow tumor initiation and progression of human MPN cancers.
UT Southwestern Medical Center researchers seeking novel treatments for anemia found that giving acetate, the major component of household vinegar, to anemic mice stimulated the formation of new red blood cells.
An international team of scientists has identified a gene mutation that causes aplastic anemia, a serious blood disorder in which the bone marrow fails to produce normal amounts of blood cells. The gene regulates telomeres on the ends of chromosomes.
A new molecule, the first of its kind, allows for the multiplication of stem cells in a unit of cord blood. Umbilical cord stem cells are used for transplants aimed at curing a number of blood-related diseases, including leukemia, myeloma and lymphoma. For many patients this therapy comprises a treatment of last resort.
In an analysis of the results of nearly 50 randomized trials that examined treatments of venous thromboembolisms (blood clot in a vein), there were no significant differences in clinical and safety outcomes associated with most treatment strategies when compared with the low-molecular-weight heparin-vitamin K antagonist combination, according to a study in the September 17 issue of JAMA.
Sickle cell disease had been considered a pediatric ailment since people with it generally didn’t live to adulthood. As pediatricians, we’ve done a good job caring for our patients – 95% now live to their 20th birthday. Unfortunately, when our patients prepare to leave the pediatric system, a smooth transition to adult healthcare is lacking.
A simple point-of-care testing device for anemia could provide more rapid diagnosis of the common blood disorder and allow inexpensive at-home self-monitoring of persons with chronic forms of the disease.
More than one-quarter of young adults with the most common form of acute lymphoblastic leukemia (ALL) have a high-risk subtype with a poor prognosis and may benefit from drugs widely used to treat other types of leukemia that are more common in adults, according to multi-institutional research led by St. Jude Children’s Research Hospital investigators.
A Johns Hopkins Sickle Cell Disease researcher and patient led a study of other Sickle Cell patients and the connection between their adherence to medical advice and their perceived discrimination by the healthcare system.
The National Heart, Lung, and Blood Institute (NHLBI) has released the first comprehensive, evidence-based guidelines for management of sickle cell disease from birth to end of life.
Results of a Phase I clinical trial showed that a new drug targeting mitochondrial function in human cancer cells was safe and showed some efficacy. The findings, reported by doctors at Wake Forest Baptist Medical Center, are published in the current online edition of the journal Clinical Cancer Research.
Accidents as minor as a slip of the knife while chopping onions can turn dangerous for patients with hemophilia, who lack the necessary proteins in their blood to stem the flow from a wound.
John Wood, MD, PhD, and colleagues are looking into how the body regulates blood flow to the muscles and brain in patients with sickle cell trait (SCT). They hope to determine specific factors that put certain SCT athletes at risk for life-threatening complications during vigorous exercise.
A Penn Medicine-developed drug has received orphan status in Europe this week for the treatment of paroxysmal nocturnal hemoglobinuria, a rare, life-threatening disease that causes anemia due to destruction of red blood cells and thrombosis.
Monthly blood transfusions can substantially reduce the risk of recurrent strokes in children with sickle cell disease (SCD) who have already suffered a silent stroke, according to the results of an international study by investigators at the Johns Hopkins Children’s Center, Vanderbilt University and 27 other medical institutions.
Vanderbilt-led research, as part of an international, multicenter trial, found regular blood transfusion therapy significantly reduces the recurrence of silent strokes and strokes in children with sickle cell anemia who have had pre-existing silent strokes, according to study results released today in the New England Journal of Medicine (NEJM).
Monthly blood transfusions reduce the risk of stroke in young patients with sickle cell anemia, scientists report Aug. 20 in The New England Journal of Medicine.
Scientists have altered key biological events in red blood cells, causing the cells to produce a form of hemoglobin normally absent after the newborn period. This approach may lead to a novel treatment for sickle cell disease.
In what is believed to be the largest genetic analysis of what triggers and propels progression of tumor growth in a common childhood blood cancer, researchers at NYU Langone Medical Center report that they have identified a possible new drug target for treating the disease.
Millions of people in the United States have a circulatory problem of the legs called peripheral vascular disease. It can be painful and may even require surgery in serious cases. This disease can lead to severe skeletal muscle wasting and, in turn, limb amputation.
Research derived from early national experience of liver transplantation has shown that deceased donor liver transplants offered recipients better survival rates than living donor liver transplants, making them the preferred method of transplantation for most physicians. Now, the first data-driven study in over a decade disputes this notion. Penn Medicine researchers found that living donor transplant outcomes are superior to those found with deceased donors with appropriate donor selection and when surgeries are performed at an experienced center. The research is published this week in the journal Hepatology.
Researchers used x-ray crystallography to publish the first image of prothrombin. The protein’s flexible structure is key to the development of blood-clotting.
The ability to reliably and safely make in the laboratory all of the different types of cells in human blood is one key step closer to reality.
Like a line of falling dominos, a cascade of molecular events in the bone marrow produces high levels of inflammation that disrupt normal blood formation and lead to potentially deadly disorders including leukemia, an Indiana University-led research team has reported.
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