Repurposed anti-cholesterol drug could improve treatment-resistant anemias

Diamond Blackfan anemia (DBA), a rare inherited bone marrow failure syndrome, is usually diagnosed during childhood and is typically treated with glucocorticoids that cause a host of unwanted, often dangerous side effects. Using a mouse model, a research team has now determined that combining the cholesterol-lowering drug fenofibrate with glucocorticoids could allow for dramatically lower steroid doses in the treatment of DBA and other erythropoietin-resistant anemias. These promising results are the foundation for a clinical trial that will begin soon.
Source: ScienceDaily Headlines - Category: Science Source Type: news

Related Links:

Conclusions Patients with longer duration of HD, male sex, and the presence of dyspepsia had higher odds to exhibit H. pylori infection. Helicobacter pylori infection did not seem to have an influence on iron deficiency or anemia severity in HD patients and does not have relation with serum hepcidin level. However, H. pylori may to be related to inflammation in HD patients.
Source: Infectious Diseases in Clinical Practice - Category: Infectious Diseases Tags: Original Articles Source Type: research
We report a 5-month old exclusively breastfed full-term infant with emesis, lethargy, progressive pancytopenia, hemolysis, hypofibrinogenemia, elevated lactate dehydrogenase and a hypercellular bone marrow with dyserythropoiesis. The B12 level in the serum was undetectable. The infant’s lethargy resolved within 48 hours of intramuscular B12 injection, followed by rapid improvement of pancytopenia. The asymptomatic mother had a normal hemoglobin and mean corpuscular volume, but undetectable B12 level and positive antibodies to intrinsic factor, consistent with pernicious anemia masked by folate supplementation in the ...
Source: Journal of Pediatric Hematology Oncology - Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research
Iron refractory iron deficiency anemia is an autosomal recessive disorder arising from defects in iron metabolism that cause microcytic anemia to grow resistant to treatment. The patients usually do not respond to orally administered iron treatment and partially respond to intravenous iron administration. Mutations of TMPRSS6 gene which encodes matriptase-2 are the main cause of the disorder. Here, we describe the case of a 6-month-old Syrian boy who had hypochromic-microcytic anemia and normal ferritin levels at presentation. The patient did not respond to 1 month of iron therapy and his hemoglobin levels increased only a...
Source: Journal of Pediatric Hematology Oncology - Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research
Conclusions: Elevated Ang-2 highlights the role of angiogenesis in the pathophysiology of SCD and may be considered a promising marker for screening of patients at risk of sickle retinopathy and vascular dysfunction.
Source: Journal of Pediatric Hematology Oncology - Category: Hematology Tags: Original Articles Source Type: research
CONCLUSION: Conservative management is the treatment of choice in III-V grade renal trauma, and the prognostic factors found in this study make it possible to better select patients. LEVEL OF EVIDENCE: 4. PMID: 31230856 [PubMed - as supplied by publisher]
Source: Progres en Urologie - Category: Urology & Nephrology Tags: Prog Urol Source Type: research
The U.S. Food and Drug Administration (FDA) has approved important safety label changes for the class of cholesterol-lowering drugs known as statins.
Source: FDA Center for Drug Evaluation and Research - What's New - Category: Drugs & Pharmacology Authors: Source Type: news
There are a number of things that can increase the risk of dementia: age, of course, as well as certain genetic profiles and behaviors such as smoking and drinking. Some of the same things that contribute to heart disease, such as high cholesterol levels and the build up of plaques in the blood vessels, can also boost the chances of developing dementia. And in a large study published in JAMA Internal Medicine conducted in the UK, researchers report another possible factor: a group of drugs known as anticholinergics. These include prescription medications for treating depression, pulmonary disease, and Parkinson’s, as...
Source: TIME: Health - Category: Consumer Health News Authors: Tags: Uncategorized Brain Dementia Drugs Source Type: news
Publication date: 25 June 2019Source: Cell Reports, Volume 27, Issue 13Author(s): Sidse Ehmsen, Martin H. Pedersen, Guisong Wang, Mikkel G. Terp, Amina Arslanagic, Brian L. Hood, Thomas P. Conrads, Rikke Leth-Larsen, Henrik J. DitzelSummaryTumor eradication may be greatly improved by targeting cancer stem cells (CSCs), as they exhibit resistance to conventional therapy. To gain insight into the unique biology of CSCs, we developed patient-derived xenograft tumors (PDXs) from ER− breast cancers from which we isolated mammospheres that are enriched for CSCs. Comparative global proteomic analysis was performed on patien...
Source: Cell Reports - Category: Cytology Source Type: research
CONCLUSIONS: The literature looking into the treatment of TAPS is very limited, with no randomized controlled trials and only one includable comparative study. Based on the data in the case report and case study literature, there is no mortality difference between any of the treatment modalities. Expectant management may be associated with an increase in adverse perinatal outcomes when compared to laser therapy and IUT. More comparative studies are needed to assist clinicians in adopting an evidence-based approach to the treatment of TAPS. PMID: 31230662 [PubMed - in process]
Source: Journal of Obstetrics and Gynaecology Canada : JOGC - Category: OBGYN Tags: J Obstet Gynaecol Can Source Type: research
Authors: Gebhardt A, Fichtenbaum CJ Abstract Introduction: Cardiovascular disease is an important cause of morbidity and mortality in persons with human immunodeficiency virus (PWH). The risk of atherosclerotic cardiovascular disease (ASCVD) is higher in PWH compared to uninfected persons. Dyslipidemia is a critical link in the pathogenesis of ASCVD in PWH. Chronic inflammation associated with HIV infection may drive both dyslipidemia and ASCVD. Areas Covered: The authors review the evidence for using lipid-lowering therapy in PWH and includes an overview of the utility and complexity of using statins in PWH, in pa...
Source: Expert Opinion on Pharmacotherapy - Category: Drugs & Pharmacology Tags: Expert Opin Pharmacother Source Type: research
More News: Anemia | Bone Marrow Failure Syndrome (BMFS) | Cholesterol | Clinical Trials | Fenofibrate | Science | Tricor