The long-term safety and effectiveness of growth hormone treatment in Japanese children with short stature born small for gestational age

Abstract

. This study aimed to characterize the safety and effectiveness of GH treatments, in usual clinical practice, in children with short stature born small for gestational age (SGA). This was a multicenter, open-label, non-interventional study ( NCT01110928 ) conducted at 150 sites in Japan (2009–2018). The primary objective was to assess the type and frequency of serious adverse drug reactions (SADRs) associated with long-term GH use. Overall, 452 naïve and 46 non-naïve (previously treated) children were enrolled. GH treatment was well‑tolerated, with SADRs occurring in 1.3% (6/452) and 0% (0/46) of naïve and non-naïve children, respectively. No new safety concerns or notable changes in glucose metabolism were identified during long-term treatment. Altogether, 57 children (32 naïve and 25 non-naïve) reached near adult height (NAH). In naïve and non-naïve children, mean ± standard deviation (SD) height standard deviation score (SDS) at NAH were –2.03 ± 0.77 and –1.53 ± 0.81, respectively, representing a change of +0.85 ± 0.72 and +1.24 ± 0.66 from baseline height SDS, respectively. Mean treatment duration to NAH was 4.29 (naïve) and 7.26 (non-naïve) yr. Thus, long-term GH treatment for short stature in children born SGA was confirmed to have a good safety profile and was effective for improving adult height.

Response to Treatment with Recombinant Human Growth Hormone (rhGH) of Short Stature Children Born Too Small for Gestational Age (SGA) in Selected Centres in Poland

“…In our study, the CA-BA was high, and the vast majority of children did not equalise bone age with calendar age. In their analysis, Horikawa et al [ 28 ] found no significant progression of bone age to calendar age during long-term follow-up, which is in line with our findings.…”

Effectiveness of a smartphone application on medication adherence in children with short stature receiving GH therapy: A multicenter prospective cohort study (GTL-App)

“…The treatment of choice for children with short stature and the aforementioned conditions is GH therapy, which requires long-term daily self-injection of recombinant human GH at home. GH therapy is expected to improve the future quality of life (QoL) of children ( 3 , 4 , 5 ) by ultimately helping them achieve a normal adult height. However, suboptimal medication adherence can impair the long-term clinical effectiveness of GH therapy, thereby resulting in a shorter final adult height ( 6 ).…”

Long-term effectiveness of growth hormone therapy in children born small for gestational age: An analysis of LG growth study data

“…In our study, the mean height gain was 1.42 SDS for 3 years of GH treatment in short children born SGA. Several studies have reported that GH treatment in SGA children without catch-up growth increased height velocity and improves adult height [ 17 – 20 ]. A meta-analysis identified four randomized controlled trials on near adult height in short children with SGA who received GH treatment [ 21 ].…”

“…From the four trials, the overall mean height gain was 1.5 SDS in GH treated versus 0.25 SDS in untreated SGA children, similar to our results. Recently, Horikawa et al [ 17 ] reported that mean height gain was 1.80 SDS from the start to the end of 5 years of GH treatment. Rapaport et al [ 22 ] also reported that SGA children who received GH treatment for 3 years achieved an increase of 0.8 height SDS, and the mean height gain was not different between children with SGA and those with GHD.…”