TREATMENT AND DISEASE EFFECTS ON SHORT-TERM GROWTH AND ADULT HEIGHT IN CHILDREN AND ADOLESCENTS WITH 21-HYDROXYLASE DEFICIENCY

Background In clinical practice, height velocity and rate of bone matu ration during each follow-up interval are among the parameters used to modify glucocorticoid treatment in children with 21-hydroxylase defic iency. There is controversy on the relative importance of treatment an d disease variables for short-term growth and final height. Patients a nd methods Short- and long-term growth data of 87 patients (n = 35 sal t-wasting form; n = 52 simple virilizing form) were analysed retrospec tively by stepwise multiple regression. Results Height SDS for chronol ogical age (HtSDS(CA)) was elevated prior to treatment in boys (m = 3. 22, p < 0.0001) and decreased when compared to HtSDS(CA) greater than or equal to 4 years after the start of treatment in both boys (m = -0. 64, p < 0.0001) and girls (m = -1.24, p < 0.0023). When the simultaneo us effects of hydrocortisone dose, salt-wasting status, degree of horm onal control, and patient sex on short-term growth between follow-up v isits were analysed, only a minor effect of hydrocortisone dose (parti al coefficient of determination [Pr-2] = 0.04, p < 0.011) and salt-was ting status (Pr-2 = 0.03, p < 0.024) in the prepubertal group, and a s mall effect of the treatment quality rating in the pubertal group (Pr- 2 = 0.07, p < 0.0042) on height velocity SDS for chronological age (HV SDSCA) were noted. Height velocity SDS for bone age (HVSDSBA) was infl uenced to a similar degree by treatment quality (Pr-2 = 0.098, p < 0.0 017) in the prepubertal, and by hydrocortisone dose (Pr-2 = 0.063, p < 0.021) in the pubertal group. Mean daily hydrocortisone doses used in normally growing patients ranged between 17.9 and 21.8 mg/m(2)/d if a nalysed separately for sex and salt-wasting status. 34 patients had re ached final height which with the exception of one boy was below the p opulation mean and ranged from 82.3% to 100.1% of target height in the total group. 41.2% of the patients had adult short stature (HtSDS < - 2). Treatment with synthetic glucocorticoids for more than 1 year sta rted before the age of 1.5 years was associated with the most severely compromised final height SDS (m = -3.73, p < 0.029). Conclusions The proportion of the short-term height velocity SDS that can be explained by the tested treatment and disease variables is low (less than or eq ual to 9.8%). Conclusions drawn from observed changes in height veloci ty during single short follow-up intervals on treatment modalities mus t therefore be viewed with caution. In the long run, however, use of d airy hydrocortisone doses > 25 mg/m(2)/day and of synthetic glucocorti coids started early in the course of the disease does not only lead to a transient deceleration of height velocity in growing children with 21-hydroxylase deficiency, but carries a definite risk for decreased f inal height.