- Review Article
- Hematology
- Promising role of voxelotor in managing sickle cell disease in children: a narrative review
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Amit Agrawal, Gaurav Jadon, Japna Singh, Dalwinder Janjua
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Clin Exp Pediatr. 2025;68(2):106-114. Published online November 13, 2024
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Voxelotor has promising ability to increase hemoglobin levels and reduce hemolysis markers in patients with sickle cell disease (SCD). Several preclinical and phase II/III trials have demonstrated its efficacy, dose-dependent responses, and tolerability in children. Ongoing trials are assessing its safety and effectiveness in various populations, including children younger than 12 years. These findings suggest its potential as a disease-modifying drug, warranting further exploration of its role in SCD management. |
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- Cardiology
- Heart failure in children and adolescents: an update on diagnostic approaches and management
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Amit Agrawal, Dalwinder Janjua, Abdulrahman Ahmed Alsayed Ali Zeyada, Ahmed Taher Elsheikh
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Clin Exp Pediatr. 2024;67(4):178-190. Published online June 19, 2023
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· Pediatric heart failure (PHF) is a clinical syndrome featuring various symptoms (shortness of breath, ankle swelling, fatigue) and signs (pulmonary crackles, peripheral edema).
· Congenital heart diseases are the most common underlying etiology of PHF, whereas myocarditis and primary cardio-myopathies are common in children without structural ab-normalities.
· PHF pathophysiology is complex and multifactorial and varies by etiology and age.
· PHF management includes decongestive therapy, treatment of underlying causes, preventing progression, and managing pulmonary or systemic obstructions.
· Drugs should be chosen based on pharmacodynamics, clinical manifestations, hemodynamic state, and renal function. |
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