Abstract
BackgroundRecombinant Growth hormone (rGH) therapy is approved in many countries for treatment of short stature in a number of childhood diagnoses. Despite the increasing body of international literature on rGH use, there is paucity of data on rGH use in Kuwait and the broader Middle-East which share unique ethnic and socio-cultural backgrounds. This study aimed to describe the pattern of use and treatment outcomes of rGH therapy in Kuwait.MethodsThis is a cross-sectional retrospective review of children treated with rGH in the Department of Pediatrics, in a major hospital in Kuwait between December 2013 and December 2014. Data were extracted using standard data extraction form and the response to rGH therapy was defined as a gain of ≥ 0.3 standard deviation score (SDS) of height per year.ResultsA total of 60 children were treated with rGH in the center. Their Median (Interquartile) age at rGH initiation was 9.0 (6.2, 10.7) years. The most common indications for rGH therapy were Growth Hormone Deficiency (GHD) 23 (38.3 %), Idiopathic Short Stature (ISS) 12 (20.0 %) and Small for Gestational Age (SGA) 9 (15.0 %). After excluding patients with TS, no significant differences were found in gender of those who received rGH therapy in all indications combined or in each group (p ≥ 0.40). At 1-year follow-up, children in all groups had median height SDS change of ≥ 0.3 SDS except for children with ISS. Age at rGH initiation was negatively associated with 1-year treatment response, Adjusted odds ratio (AOR) 0.56 (95 % CI: 0.04–1.49); p = 0.011).ConclusionsGHD is the most common indication of rGH therapy. All indications except for ISS showed significant 1-year treatment response to therapy. Treatment outcomes in patients with ISS should be further investigated in Kuwait. Younger age at initiation of rGH therapy was independently associated with significant response to therapy suggesting the importance of identifying children with short stature and prompt initiation of rGH therapy.
Highlights
Recombinant Growth hormone therapy is approved in many countries for treatment of short stature in a number of childhood diagnoses
Small for Gestational Age (SGA) was diagnosed when birth weight is less than the 10thcentile for gestational age [12]. Recombinant Growth hormone (rGH) therapy was initiated at a dose of 25–30 μg/kg/day for children with Growth Hormone Deficiency (GHD) [2], 50 μg/kg/day for children with Idiopathic Short Stature (ISS) [11], 35 μg/kg/day for children with SGA [13], and 50 μg/kg/day for children with Turner Syndrome (TS) and chronic renal disease [2]
After excluding patients with TS from the analysis, no significant difference was found in gender of those who received rGH therapy in all indications combined or in each group (p ≥ 0.40) only 9 out of 23 patients with GHD were females (p = 0.40)
Summary
Recombinant Growth hormone (rGH) therapy is approved in many countries for treatment of short stature in a number of childhood diagnoses. This study aimed to describe the pattern of use and treatment outcomes of rGH therapy in Kuwait. Recombinant Growth Hormone (rGH) has been used to promote linear growth since 1985. RGH acts mainly through increasing the secretion of insulin-like growth factor-1 (IGF-1) [1]. The indications for rGH have gradually been extended from replacement therapy in Growth Hormone Deficiency (GHD) to an increasing number of conditions in which short stature is not due to GHD. In most of the indications listed above, rGH therapy has been shown to improve final adult height [5, 7,8,9]. Despite the increasing body of international literature on rGH use, there is paucity of data on rGH use in Kuwait and the broader Middle-East which share unique ethnic and socio-cultural backgrounds
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