Prediction of response to growth hormone treatment in short children born smallfor gestational age: analysis of data from KIGS (Pharmacia International Growth Database).
Journal Title: Journal of Clinical Endocrinology and Metabolism - Year 2003, Vol 88, Issue 1
Abstract
A model was developed that allows physicians to individualize GH treatmentin children born short for gestational age (SGA) who fail to show spontaneous catch-up growth. Data fromchildren (n = 613) in a large pharmacoepidemiological survey, the KIGS (Pharmacia International GrowthDatabase), or who had participated in clinical trials were used to develop the model. Another group ofsimilar children (n = 68) from KIGS was used for validation. In the first year of GH treatment, the growthresponse correlated positively with GH dose, weight at the start of GH treatment, and midparental heightSD score and negatively with age at treatment start. Using this model, 52% of the variability of thegrowth response could be explained, with a mean error SD of 1.3 cm. GH dose was the most important responsepredictor (35% of variability), followed by age at treatment start. The second year growth response wasbest predicted by a three-parameter model (height velocity in yr 1 of treatment, age at start of treatment,and GH dose), which accounted for 34% of the variability, with an error SD of 1.1 cm. The first yearresponse to GH treatment was the most important predictor of the second year response, accounting for29% of the variability. No statistically significant differences between the predicted and observed growthresponses were found when the models were applied to the validation groups. In conclusion, using simplevariables, we have developed a model that can be used in clinical practice to adjust the GH dose to achievethe desired growth response in patients born SGA. Furthermore, this model can be used to provide patientswith a realistic expectation of treatment and may help to identify compliance problems or other underlyingcauses of treatment failure.
Authors and Affiliations
Anders Lindberg, Christopher T Cowell, Kerstin Albertsson Wikland, Michael B Ranke, David A Price, Patrick Wilton, Edward O Reiter
Keywords
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