Treatment response comparison for children born small or appropriate for gestational age (SGA or AGA) within the Australian indications for growth hormone (GH) therapy: An OZGROW analysis

Abstract

Objectives: SGA is not an indication for GH treatment in Australia although SGA patients are seen within each Australian indication. We are interested in if SGA patients respond differently to AGA patients within GH Deficiency (GHD), Short Stature and Slow Growth (SSSG), Turner Syndrome (TS), and Prader-Willi Syndrome (PWS). Methods: 6277 patients treated between 1977 and 2014 were identified in the OZGROW database. SGA was defined as birth weight below the 10thAustralian centile for gestation period. % SGA was calculated for each of GHD, SSSG, TS, and PWS, and each gender (F, M). %'s were compared to an expectation of 10%. Response to treatment was measured as a change in height standard deviation score (dSDS) after 1, and 3 years of treatment. Mean responses were compared (SGAvs AGA) within each indication/gender. Results: SGA was overrepresented: GHD-22% (F20%, M23%), PWS-55% (F54%, M57%), SSSG-41% (F46%, M38%), TS-48% (each p < 10-6). Sample sizes ranged: PWS 3rd year (F-AGA, SGA = 24.29; M-AGA, SGA = 21.31) to SSSG 1st year (F = 423,226; M = 740,461). Only significant result, 3rd year TS. Conclusions: SGA individuals are overrepresented in Australian GH indications. SGA thus meet criteria for treatment under existing guidelines. SGA patients were not seen to respond significantly differently from AGA patients within their specific indication/gender cohort over the first 3 years of treatment. The exception (TS 3 years) indicated a plateauing of response. The current indications adequately accommodate SGA. TS SGA may need further scrutiny. (Table Presented).L720733752015-11-20 <br />