A Comparison of Different Definitions of Growth Response in Short Prepubertal Children Treated with Growth Hormone

Abstract

Background: How to define poor growth response in the management of short growth hormone (GH)-treated children is controversial. Aim: Assess various criteria of poor response. Subjects and Methods: Short GH-treated prepubertal children [n = 456; height (Ht) SD score (SDS) ≤–2] with idiopathic GH deficiency (IGHD, n = 173), idiopathic short stature (ISS, n = 37), small for gestational age (SGA, n = 54), organic GHD (OGHD, n = 40), Turner syndrome (TS, n = 43), skeletal dysplasia (n = 15), other diseases (n = 46) or syndromes (n = 48) were evaluated in this retrospective multicenter study. Median age at GH start was 6.3 years and Ht SDS –3.2. Results: Median [25–75 percentile] first-year gain in Ht SDS was 0.65 (0.40–0.90) and height velocity (HtV) 8.67 (7.51–9.90) cm/year. Almost 50% of IGHD children fulfilled at least one criterion for poor responders. In 28% of IGHD children, Ht SDS gain was <0.5 and they had lower increases in median IGF-I SDS than those with Ht SDS >0.5. Only IGHD patients with peak stimulated growth hormone level <3 µg/l responded better than those with ISS. A higher proportion of children with TS, skeletal dysplasia or born SGA had Ht SDS gain <0.5. Conclusion: Many children respond poorly to GH therapy. Recommendations defining a criterion may help in managing short stature patients.

Growth response to growth hormone treatment in patients with SHOX deficiency can be predicted by the Cologne prediction model

“…This might be explained by the fact that SDS values are adjusted for age and gender. Furthermore, it supports the work of Bang and colleagues where criteria for growth response to GH were determined [27].…”

“…SDS change below 0.5 within the first treatment year was defined as poor response based on statements by Bang et al [27].…”

“…Moreover, treatment costs are very high. Therefore, the likely efficacy of this treatment in an individual patient should be critically assessed [27]. Cut-off points for poor and good response of HV or change in height SDS have been suggested [27].…”

“…Therefore, the likely efficacy of this treatment in an individual patient should be critically assessed [27]. Cut-off points for poor and good response of HV or change in height SDS have been suggested [27]. No guidelines authored by scientific societies are available yet to address the cut-off levels for poor and good treatment response.…”

Characterization and prevalence of severe primary IGF1 deficiency in a large cohort of French children with short stature

“…Efficacy was assessed during the first treatment year based on the following criteria: i) good height velocity response (defined as height velocity above the mean K1 S.D. on the ISS curve according to gender (20) or height velocity between 7 and 11.4 cm (10th-90th percentiles) for SGA children (21)); and ii) good height response (defined as height gain O0.5 S.D.) (22); plus iii) (if available) normalization of IGF1 levels to the normal range for chronological age and pubertal stage.…”