Treatment of children with Down syndrome and growth retardation with recombinant human growth hormone C a r m e n Torrado, MD, William Bastian, MD, Krystina E. Wisniewski, MD, a n d Salvador Castells, MD From the Departments of Pediatrics and Pediatric Neurology, State University of New York-Health Science Center at Brooklyn, and the New York State Institute for Basic Research in Developmental Disabilities, Staten Island, New York The effect of recombinant human growth hormone on children with Down syndrome who had growth retardation and microcephaly was examined. Thirteen children with trisomy 21 without congenital heart disease who were short for age (-4.49 to - 3 . 5 standard deviation score) and microcephalic (-4.58 to -6.60 standard deviation score) were given recombinant human growth hormone, 0.1 mg/kg subcutaneously, 3 days a week for 4 year. Before treatment, peak serum growth hormone concentrations were less than 10 pg/L after levodopa and clonidine stimulation tests in five patients, after clonidine in three patients, and after levodopa in three patients. Three patients had nocturnal integrated growth hormone concentrations of 0.5, 1.5, and 0.65 pg/L, respectively. The mean growth rate before treatment was 5.4 +__4.6 cm/yr and increased to 12.2 - 3.2 cm/yr (p 10

Mean

Nocturnal

Diurnal

0,7

0.7

0.8

0.5

>3.2

1.5 >2.3~

>2.1 ~

Data correspondto those of prepubertalchildren. *Integratedconcentrationsof GH duringa 24-hourperiodcalculatedat mean,nocturnal,and diurnalconcentrations.Patients I0 and 13 had bloodvolumestoo small to allowfor 24-hourconcentrations. tData from PlotnickLP, ThompsonRG, KowarskiAA, DeLacerda L, MigeonCJ, BlizzardRM. J Cliu EndocrinolMetab 1975;40:240-7.

with the Student t test for paired and unpaired subjects. Data are presented as mean + SD. A significant difference was indicated by a p value