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Plasma inflammatory and angiogenic protein profiling of patients with sickle cell disease.

Br J Haematol

January 2025

Department of Molecular Hematology, Sanquin Research and Landsteiner Laboratory, Amsterdam, the Netherlands.

Article Synopsis
  • The study investigated inflammation and blood vessel growth in sickle cell disease (SCD) by analyzing plasma proteins from 57 SCD patients and 13 healthy controls using advanced technology.
  • Fifty proteins showed significant differences between SCD patients and controls, particularly those related to blood vessel formation and immune responses.
  • The results suggest that certain proteins increase during painful episodes, but can return to normal levels with effective treatments, which could help develop new therapies for SCD.
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Sickle cell disease is one of the most common autosomal recessive genetic disorders with 23% and over 70% of men with this condition, experiencing episodes of ischaemic priapism and stuttering priapism, respectively, with potentially severe consequences. The effective prevention of sickle cell disease induced ischaemic priapism and stuttering priapism requires a multidisciplinary and multimodal approach. A search of the English literature was performed utilising Pubmed® and Google Scholar to identify publications on contemporary and novel treatment options, with their associated treatment outcomes if available, that are utilised to prevent stuttering priapism episodes and hence a fulminant ischaemic priapism.

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Sickle cell disease (SCD) is a monogenic disease, resulting from a single-point mutation, that presents a complex pathophysiology and high clinical heterogeneity. Inflammation stands as a prominent characteristic of SCD. Over the past few decades, the role of different cells and molecules in the regulation of the inflammatory process has been elucidated.

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Hydroxyurea has historically been the sole disease-modifying medication (DMM) for sickle cell disease (SCD). However, 3 newer DMMs, L-glutamine, voxelotor, and crizanlizumab, were approved for children and adolescents with SCD since 2017. Despite their emergence, treatment barriers, including access, affordability, and nonadherence, limit the optimization of DMMs in the clinical setting.

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Red blood cells with sickle cell anemia (SCA) have an irregular shape, and it is a genetic blood condition that can cause several problems and shorten life expectancy. Traditional treatments have focused on symptom management, but recent advancements in drug delivery systems offer promising pathways for targeted therapies. This abstract explores novel approaches to combat SCA through innovative drug delivery systems, gene therapy, and new pharmaceutical interventions.

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