Sickle cell disease: current treatment and emerging therapies.

Sickle cell disease: current treatment and emerging therapies. Am J Manag Care. 2019 Nov;25(18 Suppl):S335-S343 Authors: Neumayr LD, Hoppe CC, Brown C Abstract Sickle cell disease (SCD) is among the most common genetic diseases in the United States, affecting approximately 100,000 people. In the United States, SCD is characterized by a shortened life expectancy of only about 50 years in severe subtypes, significant quality-of-life impairments, and increased healthcare utilization and spending. SCD is characterized by chronic hemolytic anemia, vaso-occlusion, and progressive vascular injury affecting multiple organ systems. The pathophysiology is directly related to polymerization of deoxygenated hemoglobin, leading to a cascade of pathologic events including erythrocyte sickling, vaso-occlusion, tissue ischemia, and reperfusion injury as well as hemolysis, abnormal activation of inflammatory and oxidative pathways, endothelial dysfunction, increased oxidative stress, and activation of coagulation pathways. These multifactorial abnormalities have both acute and chronic clinical consequences across multiple organ systems, including acute pain episodes, chronic pain syndromes, acute chest syndrome, anemia, stroke and silent cerebral infarcts, cognitive dysfunction, pulmonary hypertension, and a wide range of other clinical consequences. Hydroxyurea was the only approved treatment for SCD for nearly 2 decades; in 2017, L-glutamine oral powder was approved fo...
Source: The American Journal of Managed Care - Category: Health Management Authors: Tags: Am J Manag Care Source Type: research

Related Links:

Publication date: 1 June 2020Source: Chemical Engineering Journal, Volume 389Author(s): Xiaojing Wang, Peng Wang, Xiaomeng Liu, Limin Hu, Qiao Wang, Peng Xu, Guangshan ZhangAbstractPerfluorooctanoic acid (PFOA) has been used as a dispersant in the fabrication of fluoropolymers and is classified as a “likely carcinogen” by the United States Environmental Protection Agency. In this work, the enhanced degradation of PFOA in water by dielectric barrier discharge (DBD) plasma in a coaxial cylindrical structure with the assistance of peroxymonosulfate (PMS) was explored. The effects of PMS dosage, input voltage and s...
Source: Chemical Engineering Journal - Category: Chemistry Source Type: research
AbstractAim of testosterone replacement therapy (TRT) is to improve symptoms and signs of testosterone deficiency including decreased libido, erectile dysfunction, depressed mood, anaemia, loss of muscle and bone mass, by increasing serum testosterone levels to physiologic range. TRT has been used in the last 70 years and overtime, numerous preparations and formulations have been developed to improve pharmacokinetics (PKs) and patient compliance. The routes of delivery approved for use in western world include buccal, nasal, sub dermal, transdermal, and intramuscular (IM). The aim of this narrative review is to describe an...
Source: Andrology - Category: Urology & Nephrology Authors: Tags: REVIEW ARTICLE Source Type: research
Approximately 28% of roadway crash fatalities in the United States occur at or near horizontal curves, and three times as many crashes occur on horizontal curves than on tangential sections. Roadway conditions, and particularly pavement surface friction, p...
Source: SafetyLit - Category: International Medicine & Public Health Tags: Engineering, Physics, Structural Soundness and Failure Source Type: news
If you want to be a doctor, hold off on medical school until age 28 - STAT There are advantages to spending your formative years working at a first career and meeting your life partner, and then starting medical school at age 28. www.statnews.com "Every generation of physicians must be free to act for itself. Claims to a physician’s 20s have withered away. Corporate health care has deemed physicians replaceable, either by those who accept lower pay or by... STAT: Why Medical School Should Start at 28...
Source: Student Doctor Network - Category: Universities & Medical Training Authors: Tags: Pain Medicine Source Type: forums
Publication date: Available online 17 February 2020Source: Surgery (Oxford)Author(s): Tristan E. McMillan, Timothy Gardner, Alan J. JohnstoneAbstractTourniquets have been around for many years, providing surgeons with a bloodless operative field and saving lives in the pre-hospital care of major limb trauma. However, their use does come with risk. It is therefore extremely important that we, as surgeons, fully understand the physiological influence they impose on patients, the complications associated with their use and, moreover, how to minimize the incidence of these complications. Most tourniquets are now electronically...
Source: Surgery (Oxford) - Category: Surgery Source Type: research
AbstractBakker et al found a change in the magnetic resonance imaging (MRI) findings in 29 of 188 patients, when the examination was repeated at three months and at one year (1). The patients were all being investigated on account of back pain, with presumably varying degrees of concern about the possibility of inflammatory spondyloarthritis.Twelve (6.8%) patients were negative at the initial examination but became positive on subsequent testing. A percentage that was lower, but not dissimilar to Van Onna et al's report of 15% at two years (2).
Source: Arthritis and Rheumatology - Category: Rheumatology Authors: Tags: LETTER TO THE EDITOR Source Type: research
For decades, NASA has been the kid politely asking for a raise in its allowance. Time was NASA’s pocket money was huge. Go back 54 years, to 1966, and the space agency was getting $5.9 billion a year—which would be a tidy $47 billion in 2020 dollars. That represented 4.1% of America’s annual household budget. But in 1966 there was also a bully on the block, whose mailbox at the time read “the Union of Soviet Socialist Republics.” They were racing the U.S. to be the first to leave the block altogether and fly to the moon, so the States were willing to spend whatever amount of money it took to b...
Source: TIME: Science - Category: Science Authors: Tags: Uncategorized newsletter Space Source Type: news
UCLA researchers have found that it is possible to assess a person ’s ability to feel empathy by studying their brain activity while they are resting rather than while they are engaged in specific tasks.  Traditionally, empathy is assessed through the use of questionnaires and psychological assessments. The findings of this study offer an alternative to people who may have difficulty filling out questionnaires, such as people with severe mental illness or autism, said senior author Dr. Marco Iacoboni, professor of psychiatry and biobehavioral sciences at the David Geffen School of Medicine at UCLA.“Assessi...
Source: UCLA Newsroom: Health Sciences - Category: Universities & Medical Training Source Type: news
In this study, we evaluated the safety, tolerability, and immunogenicity of shorter, simpler regimens of trivalent Ad26.Mos.HIV expressing mosaic HIV-1 Env/Gag/Pol antigens combined with aluminium phosphate-adjuvanted clade C gp140 protein.MethodsWe did this randomised, double-blind, placebo-controlled phase 1 trial (IPCAVD010/HPX1002) at Beth Israel Deaconess Medical Center in Boston, MA, USA. We included healthy, HIV-uninfected participants (aged 18–50 years) who were considered at low risk for HIV infection and had not received any vaccines in the 14 days before study commencement. We randomly assigned participant...
Source: The Lancet HIV - Category: Infectious Diseases Source Type: research
Conclusions: Data from Part A of the HOPE study demonstrate that treatment with voxelotor resulted in a dose-dependent increase in Hb with a large proportion of patients achieving Hb>1 g/dL improvement from baseline compared with placebo at 12 weeks. In addition, there was a dose-dependent decrease in hemolysis markers. Voxelotor was generally well tolerated at both doses. Hemolytic anemia of SCD has severe and life-threatening consequences and presents an unmet medical need. Voxelotor has potential to ameliorate complications of anemia associated with SCD.DisclosuresVichinsky: bluebird bio: Membership on an entity's Bo...
Source: Blood - Category: Hematology Authors: Tags: 114. Hemoglobinopathies, Excluding Thalassemia-Clinical: Novel or Improved Approaches To Treating Sickle Cell Disease Source Type: research
More News: Anemia | Chronic Pain | Genetics | Health Management | Hypertension | Managed Care | Pain | Pulmonary Hypertension | Sickle Cell Anemia | Stroke | USA Health