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The modern use of hydroxyurea for children with sickle cell anemia.
Haematologica
January 2025
Division of Hematology, Cincinnati Children's Hospital Medical Center, Cincinnati OH; University of Cincinnati College of Medicine, Cincinnati OH; Global Health Center, Cincinnati Children's Hospital Medical Center, Cincinnati OH.
Over the past 40 years, the introduction and refinement of hydroxyurea therapy has led to remarkable progress for the care of individuals with sickle cell anemia (SCA). From initial small proof-of-principle studies to multi-center Phase 3 controlled clinical trials and then numerous open-label studies, the consistent benefits of once-daily oral hydroxyurea have been demonstrated across the lifespan. Elevated fetal hemoglobin (HbF) serves as the most important treatment response, as HbF delays sickle hemoglobin polymerization and reduces erythrocyte sickling.
View Article and Find Full Text PDFHydroxyurea inhibits proliferation and stimulates apoptosis through inducible nitric oxide synthase in erythroid cells.
Biomed Pharmacother
December 2024
Department of molecular oncology, Institute for Medical Research, National Institute of the Republic of Serbia, University of Belgrade, Serbia. Electronic address:
Hydroxyurea (hydroxycarbamide, HU) arrests cells in the S-phase by inhibiting ribonucleotide reductase and DNA synthesis, significantly contributing to the release of nitric oxide (NO). We investigated the involvement of inducible NO synthase (NOS2) in the cytostatic effect of HU using in vitro shRNA-induced knockdown of the NOS2 transcript (NOS2) or a specific NOS2 inhibitor (1400W) in human erythroleukemic HEL92.1.
View Article and Find Full Text PDFCardiac hypertrophy in polycythemia vera: A case report and review of literature.
World J Cardiol
November 2024
Department of Cardiology, China-Japan Union Hospital of Jilin University, Changchun 130031, Jilin Province, China.
Article Synopsis
- The study discusses a rare case of a 34-year-old man with polycythemia vera (PV) who also suffered from cardiac hypertrophy, indicating a possible link between the two conditions.
- Initially diagnosed with hypertrophic cardiomyopathy after experiencing chest pain and shortness of breath, he later presented with severe respiratory distress and high blood pressure, leading to treatment with hydroxyurea.
- The conclusion stresses the importance for doctors to monitor PV patients closely for potential cardiovascular issues, especially those dealing with high blood pressure.
CRISPR-based gene therapy for the induction of fetal hemoglobin in sickle cell disease.
Expert Rev Hematol
December 2024
Department of Medicine, Division of Hematology and Medical Oncology, Center of Excellence for Sickle Cell Disease, Boston University School of Medicine and Boston Medical Center, Boston, MA, USA.
Introduction: Sickle cell disease is ameliorated and perhaps can be 'cured' if enough fetal hemoglobin is present in most erythrocytes. Hydroxyurea, which increases fetal hemoglobin levels, is widely available and effective, especially in children. Nevertheless, only cell-based gene therapy can achieve a 'curative' fetal hemoglobin threshold.
View Article and Find Full Text PDFPromising role of Voxelotor in managing sickle cell disease in children: a narrative review.
Clin Exp Pediatr
November 2024
Al Jalila Children's Specialty Hospital, Dubai, United Arab Emirates.
Sickle cell disease (SCD) is characterized by chronic hemolytic anemia and intermittent vaso-occlusive crises. To date, four disease-modifying drugs have been approved for the treatment of SCD: hydroxyurea (an S-phase inhibitor), L-glutamine (an amino acid), crizanlizumab (a P-selectin inhibitor), and voxelotor (a hemoglobin S polymerization inhibitor). Preclinical studies suggested that voxelotor effectively treats SCD and sickle cell anemia (SCA).
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