Abstract
BackgroundThere is no consensus on the definition of poor growth response after the first year of growth hormone (GH) treatment. We determined the proportion of poor responders identified by different criteria in children with GH deficiency (GHD) and born small for gestational age (SGA). The second aim was to analyze the IGF-1 response in poor growth responders.MethodsFirst-year height data of 171 SGA and 122 GHD children who remained prepubertal during the first GH treatment year were retrieved from the BESPEED database and analyzed. Criteria for poor first-year response/responsiveness were: change in height (∆Ht) SDS<0.3 or<0.5, height velocity (HV) SDS<0.5 or <1 based on the population reference, HV SDS<− 1 based on the KIGS expected HV curve (HV Ranke SDS), studentized residual (SR) <− 1 in the KIGS first-year prediction model.Results∆Ht SDS<0.5 gave the highest percentage poor responders (37% SGA, 26% GHD). Although % poor responders were comparable for ∆Ht SDS<0.3, HV SDS<+ 0.5, HV SDS<+ 1, SR<− 1, and HV Ranke SDS<− 1, these criteria did not always identify the same patients as poor responders. Among the poor growth responders 24% SGA and 14% GHD patients had an IGF-1 increase < 40%.ConclusionsThe different response criteria yield high but comparable percentages poor responders, but identify different patients. This study does not provide evidence that one criterion is better than another. A limited IGF-1 generation is not the major reason for a poor growth response in the first year of GH treatment in SGA and GHD children.Trial registrationRetrospectively registered.
Highlights
There is no consensus on the definition of poor growth response after the first year of growth hormone (GH) treatment
Subjects The auxological data and first year treatment characteristics of prepubertal children diagnosed with small for gestational age (SGA) and non-acquired GH deficiency (GHD), who had been treated exclusively with recombinant human GH on a daily basis, were retrieved from the Belgian Registry of children treated with GH (BELGROW), which is administrated by BElgian Society for PEdiatric Endocrinology and Diabetology (BESPEED) since 1985
Boys started GH treatment at a significantly older age than girls (7.5 vs. 6.6 years, respectively; p = 0.01); 26 (=15%) SGA and 19 (=16%) GHD boys were older than 10 years at the start of treatment
Summary
There is no consensus on the definition of poor growth response after the first year of growth hormone (GH) treatment. A number of definitions of poor first-year growth response have been proposed in clinical trials and consensus statements, such as a gain in height
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