AUB ScholarWorks

Differences in response to fetal hemoglobin induction therapy in β-thalassemia and sickle cell disease

Show simple item record Fathallah H. Taher A. Bazarbachi A. Atweh G.F.
dc.contributor.editor Apr-2009 2017-10-05T15:39:22Z 2017-10-05T15:39:22Z 2009
dc.identifier 10.1016/j.bcmd.2009.02.006
dc.identifier.issn 10799796
dc.description.abstract Inducers of fetal hemoglobin (HbF) have shown considerable promise in the treatment of sickle cell disease (SCD). However, the same agents have shown less clinical activity in β-thalassemia (β-Thal). To understand the basis of these differences in clinical effectiveness, we compared the effects of butyrate and hemin on the expression of the different globin genes in progenitors-derived erythroid cells from patients with β-Thal intermedia and SCD. Exposure to butyrate resulted in an augmentation of γ-globin mRNA levels in both SCD and β-Thal. Interestingly, butyrate exposure increased α-globin expression in β-Thal, while α-globin mRNA levels decreased in SCD in response to butyrate. As a result, the favorable effects of the butyrate-induced increase in γ-globin expression on α:β-like globin mRNA imbalance in β-Thal were reduced as a result of the associated increase in α-globin expression. Hemin had similar but less profound effects on all three globin genes in both categories of patients. Although the majority of patients with β-Thal did not correct their globin imbalance in response to butyrate or hemin induction of HbF in a minority of patients resulted in marked reduction in globin imbalance. Thus, we believe that the poor clinical response in a majority of patients with β-Thal to inducers of γ-globin expression may be a reflection of unfavorable effects of these agents on the other globin genes. © 2009 Elsevier Inc. All rights reserved.
dc.format.extent Pages: (58-62)
dc.language English
dc.publisher SAN DIEGO
dc.relation.ispartof Publication Name: Blood Cells, Molecules, and Diseases; Publication Year: 2009; Volume: 43; no. 1; Pages: (58-62);
dc.source Scopus
dc.title Differences in response to fetal hemoglobin induction therapy in β-thalassemia and sickle cell disease
dc.type Article
dc.contributor.affiliation Fathallah, H., Mount Sinai School of Medicine, Division of Hematology-Medical Oncology, Box 1079 One, Gustave L. Levy Place, New York, NY 10029, United States
dc.contributor.affiliation Taher, A., Department of Internal Medicine, American University of Beirut, Beirut, Lebanon
dc.contributor.affiliation Bazarbachi, A., Department of Internal Medicine, American University of Beirut, Beirut, Lebanon
dc.contributor.affiliation Atweh, G.F., Mount Sinai School of Medicine, Division of Hematology-Medical Oncology, Box 1079 One, Gustave L. Levy Place, New York, NY 10029, United States
dc.contributor.authorAddress Fathallah, H.; Mount Sinai School of Medicine, Division of Hematology-Medical Oncology, Box 1079 One, Gustave L. Levy Place, New York, NY 10029, United States; email:
dc.contributor.authorCorporate University: American University of Beirut Medical Center; Faculty: Faculty of Medicine; Department: Internal Medicine;
dc.contributor.authorDepartment Internal Medicine
dc.contributor.authorFaculty Faculty of Medicine
dc.contributor.authorInitials Fathallah, H
dc.contributor.authorInitials Taher, A
dc.contributor.authorInitials Bazarbachi, A
dc.contributor.authorInitials Atweh, GF
dc.contributor.authorReprintAddress Fathallah, H (reprint author), Mt Sinai Sch Med, Div Hematol Med Oncol, Box 1079,1 Gustave L Levy Pl, New York, NY 10029 USA.
dc.contributor.authorResearcherID Fathallah, Habib-F-3429-2010
dc.contributor.authorUniversity American University of Beirut Medical Center
dc.description.cited Atweh GF, 2001, SEMIN HEMATOL, V38, P367, DOI 10.1053-shem.2001.27566; BAILEY K, 1992, ARCH DIS CHILD, V67, P517; BOLLEKENS JA, 1991, HEMATOL ONCOL CLIN N, V5, P399; Bunn HF, 1997, NEW ENGL J MED, V337, P762; CHARACHE S, 1987, BLOOD, V69, P109; Fathallah H, 2008, BLOOD CELL MOL DIS, V40, P166, DOI 10.1016-j.bcmd.2007.08.001; Fathallah H, 2007, BLOOD, V110, P3391, DOI 10.1182-blood-2007-02-076091; Fathallah H, 2005, ANN NY ACAD SCI, V1054, P228, DOI 10.1196-annals.1345.029; FIBACH E, 1993, BLOOD, V82, P2203; Frenette PS, 2007, J CLIN INVEST, V117, P850, DOI 10.1172-JCI30920; HAJJAR FM, 1994, J PEDIATR, V125, P490, DOI 10.1016-S0022-3476(05)83304-9; HUMPHRIES RK, 1985, J CLIN INVEST, V75, P547, DOI 10.1172-JCI111731; Ikuta T, 1998, ANN NY ACAD SCI, V850, P87, DOI 10.1111-j.1749-6632.1998.tb10466.x; LEY TJ, 1982, NEW ENGL J MED, V307, P1469, DOI 10.1056-NEJM198212093072401; LEY TJ, 1983, BLOOD, V62, P370; Loukopoulos D, 1998, ANN NY ACAD SCI, V850, P120, DOI 10.1111-j.1749-6632.1998.tb10469.x; NIENHUIS AW, 1985, ANN NY ACAD SCI, V445, P198, DOI 10.1111-j.1749-6632.1985.tb17189.x; Serjeant G R, 1995, Curr Opin Hematol, V2, P103; Smith RD, 2000, BLOOD, V95, P863; STEVENS MCG, 1981, J PEDIATR, V98, P37, DOI 10.1016-S0022-3476(81)80529-X; Weatherall DJ, 1998, BAILLIERE CLIN HAEM, V11, P127, DOI 10.1016-S0950-3536(98)80072-3; Weinberg RS, 2005, BLOOD, V105, P1807, DOI 10.1182-blood-2004-02-0454; ZENG YT, 1995, BRIT J HAEMATOL, V90, P557, DOI 10.1111-j.1365-2141.1995.tb05584.x
dc.description.citedCount 2
dc.description.citedTotWOSCount 4
dc.description.citedWOSCount 3
dc.format.extentCount 5
dc.identifier.coden BCMDF
dc.identifier.pubmedID 19346141
dc.identifier.scopusID 68649098489
dc.publisher.address 525 B ST, STE 1900, SAN DIEGO, CA 92101-4495 USA
dc.relation.ispartOfISOAbbr Blood Cells Mol. Dis.
dc.relation.ispartOfIssue 1
dc.relation.ispartofPubTitle Blood Cells, Molecules, and Diseases
dc.relation.ispartofPubTitleAbbr Blood Cells Mol. Dis.
dc.relation.ispartOfVolume 43
dc.source.ID WOS:000271076500011
dc.type.publication Journal
dc.subject.otherAuthKeyword Butyrate
dc.subject.otherAuthKeyword Hemin
dc.subject.otherAuthKeyword Hemoglobin expression
dc.subject.otherAuthKeyword Sickle cells
dc.subject.otherAuthKeyword Thalassemia
dc.subject.otherChemCAS butyric acid, 107-92-6, 156-54-7, 461-55-2
dc.subject.otherChemCAS hemin, 16009-13-5
dc.subject.otherChemCAS hemoglobin F, 9034-63-3
dc.subject.otherChemCAS Butyrates
dc.subject.otherChemCAS Fetal Hemoglobin, 9034-63-3
dc.subject.otherChemCAS Hemin, 16009-13-5
dc.subject.otherChemCAS Hemoglobin Subunits
dc.subject.otherChemCAS Hemoglobins
dc.subject.otherChemCAS RNA, Messenger
dc.subject.otherIndex alpha globin
dc.subject.otherIndex beta globin
dc.subject.otherIndex butyric acid
dc.subject.otherIndex gamma globin
dc.subject.otherIndex hemin
dc.subject.otherIndex hemoglobin F
dc.subject.otherIndex adult
dc.subject.otherIndex article
dc.subject.otherIndex beta thalassemia
dc.subject.otherIndex concentration response
dc.subject.otherIndex controlled study
dc.subject.otherIndex drug efficacy
dc.subject.otherIndex erythroid cell
dc.subject.otherIndex female
dc.subject.otherIndex gene expression
dc.subject.otherIndex human
dc.subject.otherIndex human cell
dc.subject.otherIndex male
dc.subject.otherIndex pharmacogenetics
dc.subject.otherIndex priority journal
dc.subject.otherIndex sickle cell anemia
dc.subject.otherIndex Adult
dc.subject.otherIndex Anemia, Sickle Cell
dc.subject.otherIndex beta-Thalassemia
dc.subject.otherIndex Butyrates
dc.subject.otherIndex Cells, Cultured
dc.subject.otherIndex Erythroid Cells
dc.subject.otherIndex Female
dc.subject.otherIndex Fetal Hemoglobin
dc.subject.otherIndex Gene Expression
dc.subject.otherIndex Hemin
dc.subject.otherIndex Hemoglobin Subunits
dc.subject.otherIndex Hemoglobins
dc.subject.otherIndex Humans
dc.subject.otherIndex Male
dc.subject.otherIndex Middle Aged
dc.subject.otherIndex RNA, Messenger
dc.subject.otherIndex Young Adult
dc.subject.otherKeywordPlus GAMMA-GLOBIN SYNTHESIS
dc.subject.otherKeywordPlus PHARMACOLOGICAL INDUCTION
dc.subject.otherKeywordPlus ERYTHROID PRECURSORS
dc.subject.otherKeywordPlus HYDROXYUREA THERAPY
dc.subject.otherKeywordPlus INCREASES
dc.subject.otherKeywordPlus ANEMIA
dc.subject.otherKeywordPlus INTERMEDIA
dc.subject.otherKeywordPlus INDUCERS
dc.subject.otherWOS Hematology

Files in this item

Files Size Format View

There are no files associated with this item.

This item appears in the following Collection(s)

Show simple item record

Search AUB ScholarWorks


My Account