Hemoglobin-H Disease and Hemoglobin Bart's Disease
diseases May 9th. 2021, 1:53pmα-Thalassemia mutations affect up to 5% of the world’s population. The clinical spectrum ranges from an asymptomatic condition to a fatal in utero disease. Hemoglobin H disease results from mutations of three α-globin genes.
Deletional forms result in a relatively mild anemia, whereas nondeletional mutations result in a moderate to severe disease characterized by ineffective erythropoiesis, recurrent transfusions, and growth delay.
Hemosiderosis develops secondary to increased iron absorption, as well as transfusion burden. Hemoglobin Bart’s hydrops fetalis is usually a fatal in utero disease caused by the absence of α genes. Population screening to identify at-risk couples is essential. Affected pregnancies result in severe fetal and maternal complications.
Doppler ultrasonography with intrauterine transfusion therapy may improve the fetal prognosis but creates ethical challenges for the family and health providers. (See citation below)
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Topic References
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🌎https://www.stjude.org/disease/alpha-thalassemia.html
🌎https://www.stanfordchildrens.org/en/topic/default?id=alpha-thalassemia-in-children-90-P02329
🌎https://ghr.nlm.nih.gov/condition/alpha-thalassemia
Description’s Citation
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Vichinsky, Elliott P. “Clinical manifestations of α-thalassemia.” Cold Spring Harbor perspectives in medicine vol. 3,5 a011742. 1 May. 2013, doi:10.1101/cshperspect.a011742
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