Abstract

The primary goal of this project is to design and implement a multi- institutional, open-label phase I/II study of hydroxurea (HU) therapy in children with Sickle Cell Disease. To achieve the main goal, the project has four specific aims: a) To determine if HU will elevate the concentration of fetal hemoglobin, hematocrit and red cell mean corpsucular volume in children with Sickle Cell Anemia between the ages of 5 and 18 years old. b) To determine if the therapeutic dose of HU in pediatric patients is similar to that in adults. c) To determine if the hematologic and renal toxicities in chidlren are similar to those in adults. d) To determine if there are adverse effects on growth in children taking HU. To date, a total of 13 patients have now completed the study. Two patients were discontinued from the study over the past year; one moved from the area and the other was discontinued for non-compliance. Phase I/II will be completed March 31, 1998. Phase III, in which we will follow-up patients for five years, is planned to begin in April, 1998.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Center for Research Resources (NCRR)
Type
General Clinical Research Centers Program (M01)
Project #
5M01RR000240-35
Application #
6116854
Study Section
Project Start
1998-12-01
Project End
1999-11-30
Budget Start
1998-10-01
Budget End
1999-09-30
Support Year
35
Fiscal Year
1999
Total Cost
Indirect Cost

  Institution

Name
Children's Hospital of Philadelphia
Department
Type
DUNS #
073757627
City
Philadelphia
State
PA
Country
United States
Zip Code
19104

  Publications

Medina-Gomez, Carolina; Kemp, John P; Dimou, Niki L et al. (2017) Bivariate genome-wide association meta-analysis of pediatric musculoskeletal traits reveals pleiotropic effects at the SREBF1/TOM1L2 locus. Nat Commun 8:121
Agopian, A J; Goldmuntz, Elizabeth; Hakonarson, Hakon et al. (2017) Genome-Wide Association Studies and Meta-Analyses for Congenital Heart Defects. Circ Cardiovasc Genet 10:e001449
Ruan, Alexandra; Tobin, Nicole H; Mulligan, Kathleen et al. (2016) Brief Report: Macrophage Activation in HIV-Infected Adolescent Males Contributes to Differential Bone Loss by Sex: Adolescent Trials Network Study 021. J Acquir Immune Defic Syndr 72:372-5
Medoff-Cooper, Barbara; Irving, Sharon Y; Hanlon, Alexandra L et al. (2016) The Association among Feeding Mode, Growth, and Developmental Outcomes in Infants with Complex Congenital Heart Disease at 6 and 12 Months of Age. J Pediatr 169:154-9.e1
Rutstein, Richard M; Samson, Pearl; Fenton, Terry et al. (2015) Long-term safety and efficacy of atazanavir-based therapy in HIV-infected infants, children and adolescents: the Pediatric AIDS Clinical Trials Group Protocol 1020A. Pediatr Infect Dis J 34:162-7
Trabulsi, Jillian C; Irving, S Y; Papas, M A et al. (2015) Total Energy Expenditure of Infants with Congenital Heart Disease Who Have Undergone Surgical Intervention. Pediatr Cardiol 36:1670-9
Lappe, Joan M; Watson, Patrice; Gilsanz, Vicente et al. (2015) The longitudinal effects of physical activity and dietary calcium on bone mass accrual across stages of pubertal development. J Bone Miner Res 30:156-64
Ollberding, Nicholas J; Gilsanz, Vicente; Lappe, Joan M et al. (2015) Reproducibility and intermethod reliability of a calcium food frequency questionnaire for use in Hispanic, non-Hispanic Black, and non-Hispanic White youth. J Acad Nutr Diet 115:519-27.e2
Medina-Gómez, Carolina; Chesi, Alessandra; Heppe, Denise H M et al. (2015) BMD Loci Contribute to Ethnic and Developmental Differences in Skeletal Fragility across Populations: Assessment of Evolutionary Selection Pressures. Mol Biol Evol 32:2961-72
Avitabile, Catherine M; Goldberg, David J; Zemel, Babette S et al. (2015) Deficits in bone density and structure in children and young adults following Fontan palliation. Bone 77:12-6

Showing the most recent 10 out of 455 publications