The most studied pharmacological intervention in sickle cell anemia aiming at elevating HbF expression is the use of hydroxyurea. At the present time the experience has been that after 1 year of treatment with maximum tolerated doses (MTD) all patients showed increases of percent HbF, with a mean of 15% HbF, without apparent side effects besides the reversible ones observed during the process of attaining the MTD. The question of efficacy is presently being investigated by a multicenter placebo controlled double blind clinical trial that involves more than 20 sites. The goal of the study is to determine if hydroxyurea can decrease the incidence of painful crises by 50%. Results of this study are not expected before the end of 1993.
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http://dx.doi.org/10.1007/BF01989416 | DOI Listing |
Hematol Rep
January 2025
Laboratory of Immunobiology and Immunogenetics, Post Graduation Program in Genetics and Molecular Biology (PPGBM), Universidade Federal do Rio Grande do Sul (UFRGS), Porto Alegre 91501-970, Brazil.
A quarter of a century ago, sickle cell disease (SCD) was mainly viewed as a typical genetic disease inherited as a classical Mendelian trait. Therefore, the main focus concerning SCD was on diagnosis, meaning, genotyping, and identification of homozygous and heterozygous individuals carrying the relevant HbS mutant allele. Nowadays, it is well established that sickle cell disease is indeed the result of homozygosis for the HbS variant, although this single feature is not capable of explaining the highly diverse clinical presentation of SCD.
View Article and Find Full Text PDFTransfusion
January 2025
Ottawa Hospital Research Institute, Ottawa Hospital Center for Transfusion, Ottawa, Ontario, Canada.
Background: Red blood cell (RBC) utilization in patients with sickle cell disease (SCD) in Canada is poorly defined. This study describes RBC utilization in an SCD cohort at a single Canadian center.
Study Design And Methods: All adults with SCD who received care at the Ottawa Hospital between January 2006 and May 2019 were included, and followed until December 2021.
Int J Biol Macromol
January 2025
Departmento de Biofísica e Radiobiologia, Universidade Federal de Pernambuco, Recife, Pernambuco 50670-901, Brazil. Electronic address:
Mannose-binding lectin (MBL) is an important glycoprotein of the human innate immune system. Furthermore, individuals with sickle cell anemia (SCA) and MBL deficiency seem more susceptible to vaso-occlusive crises, suggesting an MBL role on HbSS red blood cells (RBCs). This study investigated the interaction of MBL with HbA (healthy) and HbSS RBCs using optical tweezers (OT) and atomic force microscopy (AFM).
View Article and Find Full Text PDFSickle cell disease (SCD) is the most common genetic disease in the world and a societal challenge. SCD is characterized by multi-organ injury related to intravascular hemolysis. To understand tissue-specific responses to intravascular hemolysis and exposure to heme, we present a transcriptomic atlas in the primary target organs of HbSS vs HbAA transgenic SCD mice.
View Article and Find Full Text PDFBackground And Objective: Sickle cell disease (SCD) is a vascular disease that may affect the retina. This study aimed to evaluate differences in average velocity (AV, mm/s), blood flow (BF, μL/min) and vessel diameter (VD, μm) from the temporal retinal arcades in SCD compared to healthy eyes using Doppler optical coherence tomography (DOCT).
Patients And Methods: A cross-sectional study was conducted between 2021 and 2023.
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