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

American Journal of Hematology

Volumn 88, Issue 11, 2013, Pages

  Kutlar, Abdullah ^a   Reid, Marvin E ^b   Inati, Adlette ^c   Taher, Ali T ^d   Abboud, Miguel R ^d   El Beshlawy, Amal ^e   Buchanan, George R ^f   Smith, Hedy ^g   Ataga, Kenneth I ^h   Perrine, Susan P ⁱ   Ghalie, Richard G ^j  

^a MEDICAL COLLEGE OF GEORGIA   (United States)

^b UNIVERSITY OF THE WEST INDIES   (Jamaica)

^c RAFIC HARIRI UNIVERSITY HOSPITAL   (Lebanon)

^d AMERICAN UNIVERSITY OF BEIRUT   (Lebanon)

^e CAIRO UNIVERSITY   (Egypt)

^f UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER   (United States)

^g TUFTS MEDICAL CENTER   (United States)

^h UNIVERSITY OF NORTH CAROLINA   (United States)

ⁱ BOSTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

^j HemaQuest Pharmaceuticals   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

2,2 DIMETHYLBUTYRATE; ANTIANEMIC AGENT; BUTYRIC ACID DERIVATIVE; HEMOGLOBIN; HEMOGLOBIN F; HQK 1001; HYDROXYUREA; IRON; UNCLASSIFIED DRUG;

ABDOMINAL PAIN; ADOLESCENT; ADULT; AREA UNDER THE CURVE; ARTICLE; BLOOD TRANSFUSION; CHILD; CONTROLLED STUDY; DIARRHEA; DISEASE SEVERITY; DOSE RESPONSE; DRUG BLOOD LEVEL; DRUG CLEARANCE; DRUG DOSE ESCALATION; DRUG EFFICACY; DRUG HALF LIFE; DRUG SAFETY; DRUG WITHDRAWAL; EPIGASTRIC PAIN; ERYTHROPOIESIS; FATIGUE; FEMALE; GASTRITIS; GENE INDUCTION; HEADACHE; HUMAN; IRON THERAPY; MAJOR CLINICAL STUDY; MALE; MAXIMUM PLASMA CONCENTRATION; MAXIMUM TOLERATED DOSE; MEDICATION COMPLIANCE; MONOTHERAPY; MULTICENTER STUDY; NAUSEA; OPEN STUDY; PANCREATITIS; PATIENT COMPLIANCE; PHASE 2 CLINICAL TRIAL; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; RETICULOCYTE; SCHOOL CHILD; SICKLE CELL ANEMIA; SICKLE CELL BETA THALASSEMIA; SOMNOLENCE; TIME TO MAXIMUM PLASMA CONCENTRATION; TREATMENT DURATION; UNSPECIFIED SIDE EFFECT; UPPER ABDOMINAL PAIN; VOMITING;

ADMINISTRATION, ORAL; ADOLESCENT; ADULT; ANEMIA, SICKLE CELL; ANTISICKLING AGENTS; BETA-THALASSEMIA; BUTYRATES; CHILD; COHORT STUDIES; DOSE-RESPONSE RELATIONSHIP, DRUG; FEMALE; FETAL HEMOGLOBIN; GASTRITIS; HEMATINICS; HETEROZYGOTE; HUMANS; HYDROXYUREA; INCIDENCE; MALE; MIDDLE AGED; PATIENT DROPOUTS; PROMOTER REGIONS, GENETIC; SICKLE CELL TRAIT; UP-REGULATION; YOUNG ADULT;

EID: 84886287023     PISSN: 03618609     EISSN: 10968652     Source Type: Journal    
DOI: 10.1002/ajh.23533     Document Type: Article

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