Background and Aim: Recombinant growth hormone (GH) is reimbursed for the treatment of short stature (<-2.5 Z-score) in children born small for gestational age (SGA) without postnatal growth, aged ≥ 4 years with a height Z-score >1 below mid-parental height (MPH). We wanted to determine the current GH prescribing practices by pediatric endocrinologists (PE) for SGA related short stature and document the percentages of treated children at risk for a poor adult height outcome.
Patients & Methods: Clinical and auxological data of 159 short children starting a GH therapy in 2017 and 2018 for SGA related short stature were retrieved from BELGROW, a national database held by the Belgian Society for Pediatric Endocrinology and Diabetology (BESPEED), incorporating PE from 15 hospitals. Growth references of Niklasson, 1991 and Intergrowth (when <28.5 weeks gestational age) and those of Roelants, 2004 for weight and height Z-score calculations, respectively at birth and start GH, were used. Older age, height Z-score at start <-3 and the shortest parental height (SPH) Z-score <-2 were defined as predictive parameters of poor adult height outcome.
Results: Clinical data for analysis were available in 146 (72 males) children. In total, 99 files underwent a peer-review organized by the BESPEED. 100 (68%) patients fulfilled strictly all the reimbursement criteria. Not presenting a height Z-score <-2.5 was the most frequent aberration (19%). Patients whose files were peer-reviewed had a higher reimbursement criteria agreement (83% vs 38%), were shorter at start of GH (median (P10; P90) (-3.0 (-4.0;-2.4) vs -2.7 (-3.5;-1.7)) and had more often a prepubertal status (82% vs 64%). Considering the 100 (53 males) patients respecting strictly all reimbursement criteria, median age at start of GH was 7.9 (4.5;13.3) years, median weight Z-score -3.3 (-4.8;-1.7), BMI Z-score -1.3 (-3.1;0.3), height Z-score -3.1 (-4.0;-2.6) and -2.0 (-3.3;1.3), when corrected for MPH. Respectively 52 (52%) and 27 (27%) had a height Z-score <- 3 and a SPH Z-score <-2, whereas 28 (28%) started treatment after 11 years. Median GH starting dose was 0.036 (0.029; 0.046) mg/kg/day.
Conclusions: Currently, GH is prescribed in patients with SGA related short stature in as many girls as boys, but with a quarter starting during adolescence and more than a half having a severe height deficit. Our findings highlight the utility of a peer review system and the need to raise awareness for earlier referral to PE in order to obtain a better adult height outcome.
22 Sep 2021 - 26 Sep 2021