Abstract

Congenital adrenal hyperplasia (CAH) is caused by an enzyme defect in the adrenal steroid biosynthetic pathway. This disease is managed primarily by glucocorticoid replacement therapy, but the appropriate therapeutic range is narrow and changes over time. Under-treatment leads to excess adrenal androgen production with consequent adverse effects, including hirsutism, virilization, early puberty, and short stature. Over-treatment causes direct growth suppression and appetite stimulation with consequent adverse effects of short stature and obesity. The hypothesis for this study is that fingerstick bloodspot 17-OH-P can be a useful assessment of adequacy of therapy for CAH. This study will involve hourly measurement of the venous blood 17-OH-P of patients with CAH on their usual dose of replacement adrenal steroids. The patients will be assessed objectively by urinary pregnanetriol measurements with substantiation by history, physical exam, and other adrenal hormone assays to determine whether each patient is being optimally treated or over-treated or under-treated.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Center for Research Resources (NCRR)
Type
General Clinical Research Centers Program (M01)
Project #
3M01RR008084-06S1
Application #
6265909
Study Section
Project Start
1998-12-01
Project End
1999-11-30
Budget Start
1998-10-01
Budget End
1999-09-30
Support Year
6
Fiscal Year
1999
Total Cost
Indirect Cost

  Institution

Name
Cincinnati Children's Hospital Medical Center
Department
Type
DUNS #
071284913
City
Cincinnati
State
OH
Country
United States
Zip Code
45229

  Publications

Natarajan, Girija; Shankaran, Seetha; Laptook, Abbot R et al. (2018) Association between sedation-analgesia and neurodevelopment outcomes in neonatal hypoxic-ischemic encephalopathy. J Perinatol 38:1060-1067
Askie, Lisa M; Darlow, Brian A; Finer, Neil et al. (2018) Association Between Oxygen Saturation Targeting and Death or Disability in Extremely Preterm Infants in the Neonatal Oxygenation Prospective Meta-analysis Collaboration. JAMA 319:2190-2201
DiFrancesco, Mark W; Shamsuzzaman, Abu; McConnell, Keith B et al. (2018) Age-related changes in baroreflex sensitivity and cardiac autonomic tone in children mirrored by regional brain gray matter volume trajectories. Pediatr Res 83:498-505
Autmizguine, Julie; Tan, Sylvia; Cohen-Wolkowiez, Michael et al. (2018) Antifungal Susceptibility and Clinical Outcome in Neonatal Candidiasis. Pediatr Infect Dis J 37:923-929
Jilling, Tamas; Ambalavanan, Namasivayam; Cotten, C Michael et al. (2018) Surgical necrotizing enterocolitis in extremely premature neonates is associated with genetic variations in an intergenic region of chromosome 8. Pediatr Res 83:943-953
Hogan, Jonathan J; Palmer, Matthew D; Loren, Alison W et al. (2017) Quiz Page May 2017: CKD and Nephrotic Syndrome After Allogeneic Hematopoietic Cell Transplantation. Am J Kidney Dis 69:A10-A13
Di Fiore, Juliann M; Martin, Richard J; Li, Hong et al. (2017) Patterns of Oxygenation, Mortality, and Growth Status in the Surfactant Positive Pressure and Oxygen Trial Cohort. J Pediatr 186:49-56.e1
Hahn, Andrew D; Higano, Nara S; Walkup, Laura L et al. (2017) Pulmonary MRI of neonates in the intensive care unit using 3D ultrashort echo time and a small footprint MRI system. J Magn Reson Imaging 45:463-471
Kingery, Kathleen M; Narad, Megan E; Taylor, H Gerry et al. (2017) Do Children Who Sustain Traumatic Brain Injury in Early Childhood Need and Receive Academic Services 7 Years After Injury? J Dev Behav Pediatr 38:728-735
Glauser, Tracy A; Holland, Katherine; O'Brien, Valerie P et al. (2017) Pharmacogenetics of antiepileptic drug efficacy in childhood absence epilepsy. Ann Neurol 81:444-453

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