title: The effects of hydroxycarbamide and magnesium on haemoglobin SC disease: results of the multi-centre CHAMPS trial.
abstract: In a phase-II multi-centre double-blinded trial, we evaluated haematological effects of oral hydroxycarbamide (HC) and magnesium (Mg) in patients with HbSC, aged 5-53 years old. Subjects were randomized to HC + placebo, Mg + placebo, HC + Mg, or placebo + placebo. The primary endpoint was the proportion of hyperdense red blood cells after 8 weeks. Thirty-six subjects were evaluable, but the study was terminated early because of slow enrollment. In the combined HC groups, mean cell volume and HbF were increased, but differences were not seen in hyperdense red cells or vaso-occlusive events. Mg had no effects. Further investigation of hydroxycarbamide as monotherapy in HbSC disease is warranted.
authors.affiliation: Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA. winfred.wang@stjude.org
authors.forename: Winfred
authors.initials: W
authors.lastname: Wang
authors.forename: Carlo
authors.initials: C
authors.lastname: Brugnara
authors.forename: Cathie
authors.initials: C
authors.lastname: Snyder
authors.forename: Lynn
authors.initials: L
authors.lastname: Wynn
authors.forename: Zora
authors.initials: Z
authors.lastname: Rogers
authors.forename: Karen
authors.initials: K
authors.lastname: Kalinyak
authors.forename: Clark
authors.initials: C
authors.lastname: Brown
authors.forename: Asif
authors.initials: A
authors.lastname: Qureshi
authors.forename: Carolyn
authors.initials: C
authors.lastname: Bigelow
authors.forename: Lynne
authors.initials: L
authors.lastname: Neumayr
authors.forename: Kim
authors.initials: K
authors.lastname: Smith-Whitley
authors.forename: David H K
authors.initials: DH
authors.lastname: Chui
authors.forename: Mardee
authors.initials: M
authors.lastname: Delahunty
authors.forename: Rob
authors.initials: R
authors.lastname: Woolson
authors.forename: Martin
authors.initials: M
authors.lastname: Steinberg
authors.forename: Marilyn
authors.initials: M
authors.lastname: Telen
authors.forename: Karen
authors.initials: K
authors.lastname: Kesler
mesh_terms: D000293:Adolescent; D000328:Adult; D000986:Antisickling Agents / Q000627:therapeutic use*; D015415:Biomarkers / Q000097:blood; D002448:Cell Adhesion / Q000187:drug effects; D002648:Child; D002675:Child, Preschool; D004311:Double-Blind Method; D004909:Erythrocyte Indices / Q000187:drug effects; D004912:Erythrocytes / Q000187:drug effects / Q000502:physiology; D005260:Female; D005319:Fetal Hemoglobin / Q000378:metabolism; D006450:Hemoglobin SC Disease / Q000097:blood / Q000188:drug therapy*; D006801:Humans; D006918:Hydroxyurea / Q000009:adverse effects / Q000627:therapeutic use*; D008274:Magnesium / Q000009:adverse effects / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged
chemical_list: D000986:Antisickling Agents; D015415:Biomarkers; D005319:Fetal Hemoglobin; D008274:Magnesium; D006918:Hydroxyurea