A dose-escalation phase IIa study of 2,2-dimethylbutyrate (HQK-1001), an oral fetal globin inducer, in sickle cell disease (Q39390596)

11 September 2017

title

A dose-escalation phase IIa study of 2,2-dimethylbutyrate (HQK-1001), an oral fetal globin inducer, in sickle cell disease (English)

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11 September 2017

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Richard G Ghalie

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author name string

Abdullah Kutlar

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Adlette Inati

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Ali T Taher

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Miguel R Abboud

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Amal El-Beshlawy

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George R Buchanan

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Hedy Smith

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Kenneth I Ataga

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Susan P Perrine

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publication date

3 October 2013

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American Journal of Hematology

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published in

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volume

88

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issue

11

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page(s)

E255-60

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Effect of hydroxyurea on the frequency of painful crises in sickle cell anemia. Investigators of the Multicenter Study of Hydroxyurea in Sickle Cell Anemia

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cites work

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Treatment of sickle cell anemia with 5-azacytidine results in increased fetal hemoglobin production and is associated with nonrandom hypomethylation of DNA around the gamma-delta-beta-globin gene complex

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Effects of 5-aza-2'-deoxycytidine on fetal hemoglobin levels, red cell adhesion, and hematopoietic differentiation in patients with sickle cell disease

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The HDAC inhibitors trichostatin A and suberoylanilide hydroxamic acid exhibit multiple modalities of benefit for the vascular pathobiology of sickle transgenic mice

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A randomized phase II trial of Arginine Butyrate with standard local therapy in refractory sickle cell leg ulcers

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Pulsed-dosing with oral sodium phenylbutyrate increases hemoglobin F in a patient with sickle cell anemia.

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Augmentation by erythropoietin of the fetal-hemoglobin response to hydroxyurea in sickle cell disease

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Stroke With Transfusions Changing to Hydroxyurea (SWiTCH).

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Short-chain fatty acid derivatives induce fetal globin expression and erythropoiesis in vivo

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Short-chain fatty acids induce gamma-globin gene expression by displacement of a HDAC3-NCoR repressor complex

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Short-chain fatty acid derivatives stimulate cell proliferation and induce STAT-5 activation

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Enhancement of growth and survival and alterations in Bcl-family proteins in beta-thalassemic erythroid progenitors by novel short-chain fatty acid derivatives

21 January 2018

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Erythroid Kruppel-like factor (EKLF) is recruited to the gamma-globin gene promoter as a co-activator and is required for gamma-globin gene induction by short-chain fatty acid derivatives

21 January 2018

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Evaluation of safety and pharmacokinetics of sodium 2,2 dimethylbutyrate, a novel short chain fatty acid derivative, in a phase 1, double-blind, placebo-controlled, single-dose, and repeat-dose studies in healthy volunteers.

21 January 2018

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A phase 1/2 trial of HQK-1001, an oral fetal globin inducer, in sickle cell disease

21 January 2018

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A randomized phase I/II trial of HQK-1001, an oral fetal globin gene inducer, in β-thalassaemia intermedia and HbE/β-thalassaemia

21 January 2018

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A phase 1/2 trial of arginine butyrate and ganciclovir in patients with Epstein-Barr virus-associated lymphoid malignancies

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Mortality in sickle cell disease. Life expectancy and risk factors for early death.

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Neuropsychological dysfunction and neuroimaging abnormalities in neurologically intact adults with sickle cell anemia

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Pulmonary hypertension as a risk factor for death in patients with sickle cell disease

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Association between hemolysis and albuminuria in adults with sickle cell anemia

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Identifiers

DOI

10.1002/AJH.23533

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11 September 2017

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