title: One-year clinical evaluation of single morning dose prednisolone therapy for 21-hydroxylase deficiency.
abstract: Replacement schedules with hydrocortisone (HC) to treat 21OHD are generally unsatisfactory and partially successful regarding growth. Noncompliance is common since its short half-life requires TID administration. Even multiple daily HC doses do not reproduce cortisol chronobiology and may disturb hypothalamic-mediated rhythms. Because synthetic glucocorticoids could improve clinical control, we evaluated the possible benefits of a one-year treatment period with a single morning oral dose of prednisolone (PD) phosphate in 44 patients with 21OHD randomized to two sex and age-matched groups: one (n=23) receiving PD (2.4-3.5 mg/m2 BSA) and the other (n=21) TID HC (10-15 mg/m2 BSA). After one year, bone maturation ratio was kept stable in the PD group (from 1.20 to 1.14), whereas a slight increase was seen in the HC group (from 1.21 to 1.29). Growth velocity (SDS) was preserved in the PD group (from 1.2 to 1.2 in all; 0.79 to 1.13 in pre-pubertals), whereas a slight increase occurred in the pre-pubertal HC-treated patients (from 1.1 to 1.9); height SDS for BA increased significantly in the PD group. Thus, patients with 21OHD treated for one year with a single morning dose of PD appear to achieve a better clinical and hormonal control than those on TID HC, permitting a reduction of the replacement dose. The current PD schedule used by our group (1.5-3 mg/m2 BSA/day) suggests a higher HC:PD bioequivalence ratio of 6-8:1.
authors.affiliation: Adrenal and Hypertension Unit, Division of Endocrinology, Department of Medicine, Federal University of São Paulo, São Paulo, SP, Brazil.
authors.forename: Milena C F
authors.initials: MC
authors.lastname: Caldato
authors.forename: Vânia T
authors.initials: VT
authors.lastname: Fernandes
authors.forename: Claudio E
authors.initials: CE
authors.lastname: Kater
mesh_terms: D000293:Adolescent; D000312:Adrenal Hyperplasia, Congenital / Q000188:drug therapy*; D000328:Adult; D002648:Child; D002675:Child, Preschool; D004334:Drug Administration Schedule; D005260:Female; D005938:Glucocorticoids / Q000008:administration & dosage*; D006801:Humans; D007223:Infant; D008297:Male; D011239:Prednisolone / Q000008:administration & dosage*; D013997:Time Factors
chemical_list: D005938:Glucocorticoids; D011239:Prednisolone