ESPE Abstracts (2015) 84 P-3-951

Predictors of Response to rhGH Treatment in 125 Children with Short Stature of Various Aetiologies

Irene Ioimoa, Alessandro Mussaa, Silvia Vannellia, Francesca Vernaa, Alberto Borraccinob & Patrizia Matarazzoa

aRegina Margherita Children Hospital, Città della Salute, Torino, Italy; bDepartment of Public Health and Paediatrics, University of Torino, Torino, Italy

Background: Response to rhGH treatment is extremely variable in pediatric growth disorders; predictors of the response are not yet clearly determined, due to disomogeneity of studied cohorts.

Aims and objectives: To investigate the correlation between clinical parameters and height gain after the 1st year of rhGH treatment and at the last visit in eight different aetiologies of short stature, with the aim of identifying predictors of response to rhGH treatment.

Methods: We selected 125 prepubertal children showing hormonal and clinic alterations (0< IGF1 <−2 S.D.s and/or GH stimulated peak secretion < 8 μg/l AND H<−3 S.D.s or HV <−2 S.D.s or H <−2 S.D.s+HV <−1.5 S.D.s) and treated with rhGH for a mean period of 5.298 years (range 2–15.66 years). Patients have eight diagnosis: 19 Organic GH-Deficiency (GHD), 19 Isolated idiopathic-GHD, 16 neurosecretory disfunction, 14 radio-treated GHD, 15 ISS, 11 SGA, 11 SHOX-Deficiency, 20 Turner Syndrome. We evaluated the correlation between clinical and hormonal parameters and height gain S.D.s after the 1st year and at the last visit.

Results: There was strong correlation between height gain sds at the 1st year and pre-treatment IGF1 levels (P: 0.045 r: 0.19) and between height gain sds at the last visit and three parameters: height gain sds at the 1st year (P<0.0001 r: 0.47), pre-treatment IGF1 values (P: 0.0033 r: −0.28), HV S.D.s at 1 year of therapy (P<0.0001 r: 0.37).

Conclusions: Predictors of response to rhGH treatment could guide rational use of this therapy, in particular to decide which patient could benefit of treatment on the long period. Our data show that children having a very low value of IGF1 are those that will benefit most from the treatment. Furthermore, the effectiveness of rhGH therapy can be evaluated by estimating the standardized height gain and the height velocity after 1st year.

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