Long-term opioids may not be best pain management option for all sickle cell patients

Small study shows some on opioids reported more pain, fatigue. In a small study looking at pain assessments in adults with sickle cell disease, researchers at Johns Hopkins says overall, those treated long-term with opioids often fared worse in measures of pain, fatigue and curtailed daily activities than those not on long-term opioids. In a report on the new research, published online on June 15 in a special sickle cell disease supplement of the American Journal of Preventive Medicine, C. Patrick Carroll, M.D., and colleagues evaluated pain experiences of participants with sickle cell disease who were prescribed long-term opioids.
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Sickle cell trait may confound blood sugar readings among African-Americans

A new study in JAMA provides evidence that hemoglobin A1c (HbA1c), a common blood biomarker used to measure blood sugar over time, may not perform as accurately among African-Americans with sickle cell trait and could be leading to a systemic underestimation of blood sugar control among that population. Sickle cell trait (SCT) is a genetic hemoglobin variant found in 8 to 10 percent of African-Americans. It occurs in people with one copy of the mutation that, if they had two copies, would result in sickle cell disease. The analysis of data from more than 4,600 people participating in two major studies found that HbA1c readings were significantly lower in individuals with SCT than in those without SCT, even after accounting for several possible confounding factors.
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A multiple drug approach to preventing sickle cell crisis

Sickle cell disease is characterized by recurrent episodes of "sickle crisis," also known as vaso-occlusive crisis, in which a patient's red blood cells change shape, clump together and block the flow of blood in small vessels resulting in pain and organ damage. In an editorial in the current issue of the New England Journal of Medicine, a Boston University School of Medicine (BUSM) researcher stresses the need for a multi-pronged approach to treating sickle-cell disease in order to prevent these harmful episodes.
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BCL11A-based gene therapy for sickle cell disease passes key preclinical test

A precision-engineered gene therapy virus, inserted into blood stem cells that are then transplanted, markedly reduced sickle-induced red-cell damage in mice with sickle cell disease, researchers from Dana-Farber/Boston Children's Cancer and Blood Disorders Center report today in the Journal of Clinical Investigation. The work sets the stage for bringing a decades-old discovery about sickle-cell disease to the bedside. A clinical gene therapy trial, using a virus rendered harmless in the laboratory, is expected to launch in the coming year.
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Methadone provides pain relief for kids with sickle cell

Many children with sickle cell disease experience frequent and severe pain episodes, requiring emergency room visits or hospitalization. In search of more effective ways to treat such pain, researchers at Washington University School of Medicine in St. Louis have found that adding a low dose of the drug methadone to standard treatment can limit pain experienced by children with the condition. "More than half of sickle cell patients have at least one episode of significant pain every year, and about 20 percent experience multiple episodes each year that require hospital stays," said first author Jennifer Horst, MD, an instructor of pediatrics at the School of Medicine and an emergency room physician at St. Louis Children's Hospital. "In this study, the pediatric patients who received a one-time dose of methadone rated their pain levels much lower than those who took standard pain-killing drugs. In many cases, their pain went away, so we believe methadone has the potential to make life better for these pediatric patients."
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Pediatric sickle cell study stopped early due to positive results

A national sickle cell disease study involving Medical University of South Carolina researchers found that for some children with sickle cell disease, the drug hydroxyurea is as effective as blood transfusions to reduce blood flow speeds in the brain. Increased blood flows are a major risk factor for stroke in these children. Study findings were published Dec. 6 in The Lancet and were presented at the American Society of Hematology meeting.

"It was a privilege to be a part of this well-designed and executed study. Russell Ware presented the results at the ASH meeting, and 18 years ago, almost to the day, I presented the STOP study results to the same meeting," said Robert J. Adams, M.D., study principal investigator, MUSC professor of neurosciences and director of the South Carolina Stroke Center of Economic Excellence. "

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Preclinical Data on Proprietary AKT Inhibitor, ARQ 092, Demonstrating Effectiveness in the Treatment of Sickle Cell Disease Presented at the American Society of Hematology Annual Meeting

ArQule, Inc. (Nasdaq: ARQL) today announced that preclinical data was presented on its proprietary AKT inhibitor, ARQ 092, in an oral presentation by the University of Illinois College of Medicine at the American Society of Hematology (ASH) Annual Meeting. The presentation highlighted preclinical studies of ARQ 092 in sickle cell disease (SCD). ARQ 092 is an orally available, selective pan-AKT inhibitor.
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Sickle Cell in Focus Conference 2018

After an exciting event in Kingston, Jamaica in October 2017, we are thrilled to announce that the 12th annual Sickle Cell in Focus (SCiF) conference will return to the National Institutes of Health (NIH) on October 22-23, 2018.

SCiF is a two-day, intensive, educational update on sickle cell disease. This year’s conference will focus on management of the older adult patients, curative therapies, systems medicine, and will also include 2 debates on issues for which there are no clear answers.

This comprehensive and global exploration of the current medical trends and research results in sickle cell disease and thalassemia is geared towards consultants, trainees, doctors, healthcare professionals, and academics interested in hemoglobin disorders.

 

Date:

October 22-23, 2018

 

Location:

Natcher Conference Center, Ruth L. Kirschstein Auditorium

45 Center Dr.

National Institute of Health, Bethesda, Maryland, United States

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2016 Annual Sickle Cell Disease Clinical Research Meetings

The NHLBI Annual Sickle Cell Disease Clinical Research Meetings provide a yearly forum for investigators, practitioners, and interested health care providers to discuss the progress of ongoing clinical trials, hear presentations about new developments in scientific and clinical aspects of SCD, and interact with other investigators and NHLBI Program Staff. This year, the meeting will begin on August 15, 2016, and end on August 17, 2016. There will be three symposiums, one on each day: Monday, August 15, 2016: Symposium: Stem Cell Transplantation in Sickle Cell Disease (Lakshmanan Krishnamurti, MD, Emory, organizer). Tuesday, August 16, 2016: Symposium for Data Science for Sickle Cell Disease and Health Care (Ellen Werner, PhD, NHLBI). This symposium will serve as a practical introduction to the data science for clinicians and health care providers. Wednesday, August 17, 2016: Symposium on Transfusion Medicine in Sickle Cell Disease (Ify Osunkwo, MD (University of North Carolina, Charlotte) and Julie Kanter, MD (Medical University of South Carolina, organizers). This meeting is an opportunity to meet and exchange ideas with a diverse group of SCD investigators and providers, as well as NIH Program Officers in an informal setting.
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Acetylon Presents Preclinical Data Demonstrating the Utility of Selective HDAC1,2 Inhibition by ACY-957 to Induce Gamma Globin (HBG) Protein Expression for the Treatment of Sickle Cell Disease and Beta-Thalassemia

Acetylon Pharmaceuticals, Inc., the leader in the development of selective histone deacetylase (HDAC) inhibitors for enhanced therapeutic outcomes, today announced that it will present preclinical data comparing the effects of alternative dosing schedules for the selective HDAC1,2 inhibitor, ACY-957, on gamma globin (HBG) protein expression. In an oral presentation at the American Society of Hematology Annual Meeting (ASH) in San Diego, California, Jeffrey R. Shearstone, Ph.D., Associate Director of Biology at Acetylon, will present data demonstrating that treatment with ACY-957 was well tolerated in a 3-week dosing regime and led to greater than 50-fold increases of HBG mRNA and protein in non-anemic primates.
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