Publications by authors named "N Jeffries"
Individuals with sickle cell disease (SCD) have a unique type of dyslipidemia characterized by low total cholesterol (TC), low low-density lipoprotein cholesterol (LDL-c), low high-density lipoprotein cholesterol (HDL-c), and normal triglycerides (TG). This lipid state is theorized to be cardioprotective against atherosclerosis. In SCD, hematopoietic cell transplant (HCT) offers a potentially curative therapy.
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- Deferasirox (DFX) is an iron chelator that effectively treats iron overload and can enhance red blood cell production in myelodysplastic syndrome patients, reducing their need for blood transfusions.
- The study explores how DFX impacts myeloid cell maturation and shows that its effects vary depending on the differentiation stage, influencing mitochondrial reactive oxygen species (ROS) production and NF-kB pathway regulation.
- Findings indicate that DFX increases mitochondrial ROS in neutrophils and alters gene expression associated with NF-kB and MYC in progenitors, suggesting it impairs the final maturation of these immune cells.
In a phase 1 study (NCT04000165), we established proof of concept for activating pyruvate kinase (PK) in sickle cell disease (SCD) as a viable antisickling therapy. AG-348 (mitapivat), a PK activator, increased adenosine triphosphate (ATP) and decreased 2,3-diphosphoglycerate levels while patients were on treatment, in line with the mechanism of the drug. We noted that the increased hemoglobin (Hb) persisted for 4 weeks after stopping AG-348 until the end of study (EOS).
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- - Sickle cell disease (SCD) leads to complications, including lung issues, and while treatments like hematopoietic cell transplant (HCT) are becoming more available, understanding pulmonary function post-HCT is important for better patient care.
- - A study tracked 97 SCD patients over three years after receiving HCT to examine changes in lung function, specifically through measurements like forced expiratory volume (FEV) and six-minute walk distance (6MWD).
- - Results showed that while FEV remained relatively stable over three years, there was a significant improvement in lung diffusing capacity and a notable increase in 6MWD, indicating better overall pulmonary health after HCT.
Integrated care systems join up health and care services, so that people have the support they need, in the right place, at the right time. The aims include improving outcomes in healthcare, tackling inequalities in access and enhancing productivity and value for money. This is needed for neuroscience care as the traditional delivery of neuroscience care is inefficient, outdated and expensive, and can involve complex referral pathways and long waiting times.
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