Effect of Growth Hormone (GH) Treatment on the Final Height of 1258 Patients with Idiopathic GH Deficiency: Analysis of a Large International Database

• Context: Treatment with growth hormone (GH) has been used to correct the growth deficit in children with GH deficiency (GHD). Although successful in increasing height velocity, such treatment often falls short of helping patients achieve full genetic height potential. Objective: This study set out to analyze near final height data from a cohort of GH-treated children with idiopathic GHD. Design, Setting, and Participants: Of 1258 evaluable patients in the Pfizer International Growth Database (KIGS) with GHD, 980 were of Caucasian origin and 278 of Japanese origin; 747 had isolated GHD (IGHD), while 511 had multiple pituitary hormone deficiencies (MPHD). Main Outcome Measures: Near final height, relation to midparental height, and factors predictive of growth outcomes. Results: Median height SD scores (SDS) at start of treatment were -2.4 (IGHD) and -2.9 (MPHD) for Caucasian males and -2.6 (IGHD) and -3.4 (MPHD) for females, respectively; comparable starting heights were -2.9 (IGHD) and -3.6 (MPHD) for Japanese males and -3.3 (IGHD) and -4.0 (MPHD) for females, respectively. Corresponding near adult height SDS after GH treatment were -0.8 (IGHD) and -0.7 (MPHD) for Caucasian males and -1.0 (IGHD) and -1.1 (MPHD) for females, respectively; and -1.6 (IGHD) and -1.9 (MPHD) for Japanese males and -2.1 (IGHD) and -1.8 (MPHD) for females, respectively. Differences between near adult height and midparental height ranged between -0.6 and +0.2 SDS for the different groups, with the closest approximation to MPH occurring in Japanese males with MPHD. First-year increase in height SDS and prepubertal height gain were highly correlated with total height gain confirming importance of treatment before pubertal onset. Conclusions: It is possible to achieve final height within the midparental height range in patients with idiopathic GHD treated from an early age with GH, but absolute height outcomes remain in the lower part of the normal range. Patients with MPHD generally had a slightly better long term height outcome.

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