Sickle Cell Disease in Children and Adolescents: A Review of the Historical, Clinical, and Public Health Perspective of Sub-Saharan Africa and Beyond

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dc.contributor.authorEgesa, Walufu Ivan
dc.contributor.authorNakalema, Gloria
dc.contributor.authorWaibi, William M.
dc.contributor.authorTuryasiima, Munanura
dc.contributor.authorAmuje, Emmanuel
dc.contributor.authorKiconco, Gloria
dc.contributor.authorOdoch, Simon
dc.contributor.authorKumbakulu, Patrick Kumbowi
dc.contributor.authorAbdirashid, Said
dc.contributor.authorAsiimwe, Daniel
dc.date.accessioned2023-04-12T16:36:12Z
dc.date.available2023-04-12T16:36:12Z
dc.date.issued2022
dc.description.abstractSickle cell disease (SCD) is an umbrella term for a group of life-long debilitating autosomal recessive disorders that are caused by a single-point mutation (Glu→Val) that results in polymerization of hemoglobin (Hb) and reversible sickle-shape deformation of erythrocytes. This leads to increased hemolysis of erythrocytes and microvascular occlusion, ischemia-reperfusion injury, and tissue infarction, ultimately causing multisystem end-organ complications. Sickle cell anemia (HbSS) is the most common and most severe genotype of SCD, followed by HbSC, HbSβ0thalassemia, HbSβ+thalassemia, and rare and benign genotypes. Clinical manifestations of SCD occur early in life, are variable, and are modified by several genetic and environmental factors. Nearly 500 children with SCD continue to die prematurely every day, due to delayed diagnosis and/or lack of access to comprehensive care in sub-Saharan Africa (SSA), a trend that needs to be urgently reversed. Despite proven efficacy in developed countries, newborn screening programs are not universal in SSA. This calls for a consolidated effort to make this possible, through the use of rapid, accurate, and cheap point-of-care test kits which require minimal training. For almost two decades, hydroxyurea (hydroxycarbamide), a century-old drug, was the only disease-modifying therapy approved by the U.S. Food and Drug Administration. Recently, the list expanded to L-glutamine, crizanlizumab, and voxelotor, with several promising novel therapies in the pipeline. Despite its several limitations, hematopoietic stem cell transplant (HSCT) remains the only curative intervention for SCD. Meanwhile, recent advances in gene therapy trials offer a glimpse of hope for the near future, although its use maybe limited to developed countries for several decades.en_US
dc.identifier.citationEgesa, W. I., Nakalema, G., Waibi, W. M., Turyasiima, M., Amuje, E., Kiconco, G., ... & Asiimwe, D. (2022). Sickle Cell Disease in Children and Adolescents: A Review of the Historical, Clinical, and Public Health Perspective of Sub-Saharan Africa and Beyond. International Journal of Pediatrics, 2022.https://doi.org/10.1155/2022/3885979en_US
dc.identifier.urihttps://nru.uncst.go.ug/handle/123456789/8443
dc.language.isoenen_US
dc.publisherInternational Journal of Pediatricsen_US
dc.subjectPublic Health Perspectiveen_US
dc.subjectSickle Cell Diseaseen_US
dc.subjectChildren and Adolescentsen_US
dc.titleSickle Cell Disease in Children and Adolescents: A Review of the Historical, Clinical, and Public Health Perspective of Sub-Saharan Africa and Beyonden_US
dc.typeArticleen_US
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