ESPE Abstracts (2016) 86 P-P1-804

Near-Adult Height in a Large Cohort of Patients with Turner Syndrome and Noonan Syndrome Treated with rhGH: Results from Pfizer International Growth Database

Michael Rankea, Anders Lindbergb, Cecilia Camacho-Hübnerc, Thomas Reinehrd & Raoul Roomane

aDivision of Paediatric Endocrinology, University Children’s Hospital, Tübingen, Tübingen, Germany; bPfizer Health AB, Sollentuna, Sweden; cPfizer Inc., Endocrine Care, New York, USA; dPediatric Endocrinology, Diabetes and Nutrition Medicine, Vestische Children’s Hospital, University of Witten/Herdecke, Datteln, Germany; eUniversity of Antwerp, Antwerpen, Belgium

Background: Pfizer International Growth Database (KIGS) contains data of Turner syndrome (TS; N=7378) or Noonan syndrome (NS; N=613, female=224; male=389) patients who were treated with rhGH.

Objective: To compare the effect of rhGH on near adult height (NAH) in TS and NS patients. We hypothesized a similar outcome in both diagnoses. Determinants of the treatment outcome in NS patients were also assessed.

Patients and Methods: Patients with TS (n=2766) or NS (F=66; M=74) who reached NAH were analyzed. For demographic data Prader and disease specific references (DSR) were used (1–3).

Results: Clinical characteristics of the cohorts at GH start and at NAH are tabulated. All groups received lower GH doses than those approved. RhGH treatment was started during late childhood. At rhGH start TS girls had an average height on the TS curve but the NS patients had an average height in the lowest quartile. TS girls gained about 1 SD whereas NS (F) gained 1.3 SD and NS (M) 1.2 SD in height (Prader reference curve). As in TS, the height gain in NS is mostly explained by: height at start first year growth and GH dose (positive) and age (negative) Table 1.

Table 1.
StartAge (yrs)9.6±3.09.7±3.111.0±3.1
Height SDS*−3.3±1.0−3.8±1.0#−3.2±0.9##
Height SDS**0.1±1.0−1.1±0.9#−1.0±0.9
NAHAge (yrs)16.9±1.616.7±1.818.4±1.6##
Height SDS*−2.3±1.1−2.5±1.2−2.1±1.2
Height SDS**1.6±1.10.1±1.1#0.5±1.2
Delta Ht SDS*1.0±0.81.3±0.7#1.1±0.9
Mean GH (mg/kg/wk) 0.29±0.10.30±0.10.27±0.1
*Prader reference; **DSR; #=P<0.01, NS (F) vs TS; ##=P<0.01; NS (F) vs NS (M).

Conclusions: TS patients responded as expected (1.0 SD gain) taking into account the late onset of treatment and the suboptimal GH dose. NS patients responded slightly better which may be explained by their larger growth deficit at the start of treatment.

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