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One-year clinical evaluation of single morning dose prednisolone therapy for 21-hydroxylase deficiency

2004; Editora da Universidade de São Paulo; Volume: 48; Issue: 5 Linguagem: Inglês

10.1590/s0004-27302004000500017

1677-9487

Milena Coelho Fernandes Caldato, Vânia T. Fernandes, Cláudio E. Kater,

Metabolism and Genetic Disorders

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 (2.4-3.5mg/m² BSA) and the other (n=21) TID HC (10-15mg/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 to1.13 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 (1.5-3mg/m² BSA/day) suggests a higher HC:PD bioequivalence ratio of 6-8:1.