Genetic modifiers of HbF and response to hydroxyurea in sickle cell disease

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Genetic modifiers of sickle cell disease.

Sickle cell disease is one of the best characterized human monogenic disorders. Complex genotype/phenotype correlations clearly demonstrate the interaction of multiple genetic and environmental factors. In the last 20 years, scientific research has applied genetic approaches to dissect some of these modifiers. This review highlights the more recent genetic association studies that have been app...

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Sickle Cell Disease: Is Hydroxyurea

We read with interest the recent article on efficacy of hydroxyurea (HU) in sickle cell disease (SCD) [1]. We share our experience in managing nine such patients (4 males) aged between 3 and 19 years. Seven patients were on regular HU therapy (15-20 mg/kg/d) and penicillin prophylaxis for median period of 2.8 years. All had history of repeated fever, chest infections and vasoocclusive episodes ...

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Side Effects of Hydroxyurea in Patients with Sickle Cell Anemia

Background: Hemoglobin S arises is the result of a point mutation (A-T) in the sixth codon on the -globin gene on chromosome 11 causing sickle cell anemia. The presence of fetal hemoglobin in infancy plays a relatively protective role for vaso-occlusive symptoms that are the major contributor for the morbidity and mortality among patients with sickle cell anemia. hydroxyurea, an s-phase-specif...

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Genetic modulation of HbF in Brazilians with HbSC disease and sickle cell anemia.

may even be asymptomatic and not diagnosed until much later in life [3,4]. In contrast, heterozygous patients with N370S=other present earlier and show predominantly visceral disease and more severe skeletal involvement [4]. Our patient has heterozygous mutation N370S=G377S. Unlike N370S, the G377S mutation is rare and usually seen in patients of Portuguese and Spanish descent [1] and is also s...

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Hydroxyurea treatment for sickle cell disease.

High fetal hemoglobin (HbF) levels inhibit the polymerization of sickle hemoglobin (HbS) and reduce the complications of sickle cell disease. Pharmacologic agents that can reverse the switch from gamma- to beta-chain synthesis--gama-globin chains characterize HbF, and sickle beta-globin chains are present in HbS--or selectively increase the proportion of adult erythroid precursors that maintain...

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ژورنال

عنوان ژورنال: Pediatric Blood & Cancer

سال: 2010

ISSN: 1545-5009

DOI: 10.1002/pbc.22754