ESPE Abstracts (2015) 84 P-2-408

ESPE2015 Poster Category 2 GH & IGF (40 abstracts)

Does Skeletal Disproportion in Children with Idiopathic Short Stature Influence Response to GH Therapy?

Wayne S Cutfield a , Anders Lindberg b , Paul Hofman a , Jose Derraik a , Mitchell E Geffner c & Cecilia Camacho-Hubner a,


aPediatric Endocrinology, Liggins Institute, University of Auckland, Auckland, New Zealand; bPfizer Health AB, Sollentuna, Sweden; cCenter for Endocrinology, Diabetes, and Metabolism, The Saban Research Institute, Children’s Hospital Los Angeles, Los Angeles, CA, USA; dEndocrine Care, Pfizer Inc., New York, NY, USA


Background: Children with ISS have an array of causes that lead to short stature and/or poor growth velocity. Genetic causes of short stature, notably SHOX mutations, can be associated with subtle skeletal disproportion with shorter limbs, manifesting as increased sitting-to-standing height ratios or SDS.

Objective and hypothesis: Children with ISS and skeletal disproportions have a diminished growth response to GH treatment compared to children with proportionate short stature after 1 year (short-term) and at near-adult height (NAH; long-term).

Method: ISS patients registered in Pfizer international growth database with a stimulated peak GH >10 μg/l and treated with GH were included. Short- and long-term growth responses were analyzed. Sitting height % SDS was grouped as: normal (SDS −1.0–<1.1), mild (SDS 1.1–<2.1), and moderate (SDS >2.1) disproportionate short stature. Wilcoxon rank sum test was used for univariate statistical comparisons. ANOVA was used for group comparisons. P-value <0.05 was considered significant.

Results: Prior to GH treatment, the ISS group displayed Gaussian distribution for skeletal proportion. For short-term analyses, the number of patients in each group was: normal (n=193), mild (n=191), and moderate (n=140). The corresponding number of patients in each group attaining NAH was: normal (57), mild (52), and moderate (28). Short-term growth responses, expressed as Studentized Residuals using the KIGS ISS 1st-year prediction model (Ranke MB et al. Horm Res 2007; 68:53–62), showed a trend toward poorer growth response with greater severity of disproportion (mean values; normal=−0.04, mild=−0.16, and moderate=−0.25, P=0.07). Long-term growth showed a larger difference, expressed as delta height SDS from GH start to NAH (median values; normal/mild=1.75 vs moderate=1.39, P<0.05)

Conclusion: Children with ISS and skeletal disproportion (shorter limbs) have reduced long-term height responsiveness to GH compared to those without disproportion suggesting subtle GH resistance in the former.

Conflict of interest: C Camacho-Hubner and A Lindberg are employees of Pfizer Inc. W Cutfield is a member of the KIGS Steering Committee.

Funding: Pfizer International Growth Database (KIGS) currently is a static database sponsor by Pfizer Inc. No authors were paid to conduct the research study submitted in this abstract.

Volume 84

54th Annual ESPE (ESPE 2015)

Barcelona, Spain
01 Oct 2015 - 03 Oct 2015

European Society for Paediatric Endocrinology 

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