Recombinant growth hormone therapy in children with short stature in Kuwait: a cross-sectional study of use and treatment outcomes

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.

Differences in Response to Recombinant Growth Hormone Therapy on Height Gain in Patients with Idiopathic Short Stature Vs. Patients with Growth Hormone Deficiency

“…Many studies have shown that the use of rhGH can improve height gain and growth velocity in patients with ISS treated for a short period of time. However, the use of this medication in ISS might result in a rapid increase in bone age and early closure of the epiphyseal plate, which could eventually affect these patients' ability to attain expected adult height [9][10][11][12][13]. In contrast, many published research papers documented the efficacy of using rhGH in increasing the expected final adult height and growth velocity in patients with ISS [14][15][16][17][18][19][20][21][22][23][24].…”

Assessment and referral of patients with short stature by primary care physicians in the Arabian gulf region: Current perspectives from a regional survey

“…Evidence supporting the safety profile of GH therapy includes large registries comprising more than 600,000 patients-years of GH exposure, and long-term safety cohorts of adults who received GH therapy as children (22). Several studies have reported favorable outcomes of GH treatment in the Arabian Gulf region (23)(24)(25)(26). Despite this, only 60% of the respondents considered GH safe.…”

Diagnosis and management of growth disorders in Gulf Cooperation Council (GCC) countries

“…In contrast, approval has not been given by the EMA despite numerous applications by pharmaceutical companies [21]. Although a consensus approach to the diagnosis or treatment of ISS in GCC countries has not been established [6], it appears that FDA rather than EMA guidelines are generally followed, with approximately 20% of hGH-treated children having ISS as reported from Kuwait [51] and the UAE [52].…”