Isolated childhood growth hormone deficiency: a 30-year experience on final height and a new prediction model

Abstract

Purpose We aimed to evaluate the near-final height (nFHt) in a large cohort of pediatricpatients with growth hormone deficiency (GHD) and to elaborate a new predictive method of nFHt. Methods We recruited GHD patients diagnosed between 1987 and 2014 and followed-up until nFHt. To predict the values of nFHt, each predictor was run in a univariable spline. ResultsWe enrolled 1051 patients. Pre-treatment height was −2.43 SDS, lower than parental height (THt) (−1.09 SDS, p < 0.001). The dose of recombinant human GH (rhGH) was 0.21mg/kg/week at start of treatment. nFHt was −1.08 SDS (height gain 1.27 SDS), higher than pre-treatment height (p < 0.001) and comparable to THt. 1.6% of the patients were shorter than −2 SDS from THt. The rhGH dose at nFHt was 0.19 mg/kg/week, lower than at the start (p < 0.001). The polynomial regression showed that nFHt was affected by gender, THt, age at puberty, height at puberty, age at the end of treatment (F = 325.37, p < 0.0001, R 2 87.2%). Conclusion This large national study shows that GHD children can reach their THt. The rhGH/kg/day dose significantly decreased from the start to the end of the treatment. Our model suggests the importance of a timely diagnosis, possibly before puberty, the beneficial effect of long-term treatment with rhGH, and the key-role of THt. Our prediction model has a very acceptable error compared to the majority of other published studies.

Transient Isolated Idiopathic Growth Hormone Deficiency – a Self-Limiting Pediatric Disease with Male Predominance or a Diagnosis Based on Uncertain Criteria? Lesson from 20-Years’ Real-World Experience with Retesting in One Center

“…One direction of research should be introducing prediction models of rhGH therapy effectiveness into clinical practice in order to have reasonable expectations about the effects of treatment and to identify poor responders as early and possible. Prediction models are developed for many years with use of different methods, like linear and non-linear regression, neural networks or other advanced computational techniques, however only few of them allows to predict the therapy outcome [29][30][31][32][33][34]. It seems that the availability of such models in the form of easy-to-use calculators would facilitate the decision to discontinue GH therapy and to perform retesting earlier.…”

Transient Isolated, Idiopathic Growth Hormone Deficiency—A Self-Limiting Pediatric Disease with Male Predominance or a Diagnosis Based on Uncertain Criteria? Lesson from 20 Years’ Real-World Experience with Retesting at One Center

“…One direction of research should be the introduction of prediction models of rhGH therapy effectiveness into clinical practice in order to have reasonable expectations about the effects of treatment and to identify poor responders as early as possible. Prediction models have been developed for many years with the use of different methods, like linear and non-linear regression, neural networks or other advanced computational techniques; however, only few of them allow the therapy outcome to be predicted [ 29 , 30 , 31 , 32 , 33 , 34 ]. It seems that the availability of such models in the form of easy-to-use calculators would facilitate the decision to discontinue GH therapy and to perform retesting earlier.…”