Of children born small for gestational age (SGA) with birth weight and/or birth length below -2 SD, about 10% fail to catch-up after birth. These children remain short over childhood /with height standard deviation score (ht-SDS) bellow -2.5 SDS/ and end up as short adults. They are assigned as SGA-SS (small for gedtational age with short stature). European Medicine Agency approved grjowth hormone therapy in SGA-SS already in 2003.
Out of 1502 SGA-SS children that are registered in the Czech national database of growth hormone recipients REPAR, 397 children (221 boys) already completed growth and achieved near-final height. Growth hormone therapy (median dose 0.034mg/kg/day) was initiated at age 8.5 years in boys (median; P5-95: 3.2-15.0) and 73. years in girls(3.9-13.5). The mean ht-SDS at treatment initiation was -3.21 (SD:0.91). Treatment was terminated at age 16.2 years in boys (median; P5-95:14.0-18.7) and 14.3 years in girls (12.4-17.3).
By treatment termination, the patients have had gained 1.59 ht-SDS (mean) and have achieved near-final height -1.62 ht-SDS (SD: 0.97; p 0.0001 if compared to pre-treatment). Thus, majority of patients exceeded -2 SDS at treatment completion and achieved height within normal limits. After adjusting for ht-SDS at age 18 years, their height corresponded to ht-SDS -2.30 (SD: 1.09; if compared pre-treatment). Growth hormone therapy was successful even after this adjustment that midly underestimates the treatment effect, and led to mean height increase of 0.91 ht-SDS.
Analysis results of the REPAR database confirm efficacy of growth hormone therapy in SGA-SS children and its potential to significantly improve the adult height.