Background: In the UK, Growth Hormone (GH) is indicated for treatment of children with short stature secondary to growth hormone deficiency (GHD), Prader Willi syndrome (PWS), Turner syndrome (TS), SHOX gene mutation, chronic renal insufficiency and born small for gestational age (SGA).
Objectives: The aim was to assess the compliance of our local practice with the NICE guidance for GH therapy in children in addition to local guidance that TFTs and IGF-I concentration should be checked annually in children with GHD whilst on treatment.
Methodology: All patients commenced on GH therapy by the Paediatric Endocrine Team at Addenbrookes Hospital between November 2014 and February 2017 were identified. Patients seen in outreach clinics were excluded as we did not have access to their records. Data were collected from clinical records.
Results: 91 patients were identified. However only 47 met the inclusion criteria. 36/47(77%) of patients were treated for GHD [18/36(50%) idiopathic and 18/36(50%) acquired], 5/47(10%) TS, 2/47(4%) PWS and 4/47(9%) SGA. All patients fulfilled local standards for commencement of GH for the documented indication. An appropriate GH dose for the indication was offered and documented in all patients. All patients were followed up within a specialist clinic with the appropriate MDT and all were offered their choice of GH device following discussion with our nurse specialists. 86% of patients with GHD had (TFTs) documented at diagnosis and annually. 11% patients with idiopathic GHD were treated for hypothyroidism in comparison to 55% patients with acquired GHD. 89% of patients with GH deficiency had IGF-1 evaluated at diagnosis and 86% annually. In patients with idiopathic GHD IGF-1 concentration prior to therapy was 17.6±12.9 nmol/l and 45.3±17.8 nmol/l a year after starting therapy (P<0.0001). In patients with acquired GHD IGF-1 concentration prior to therapy was 42.0±11.6 nmol/l and 47.4±13.3 nmol/l 12 months after starting treatment (P<0.0005). Differences between baseline and on GH were compared using a paired t-test.
Conclusions: There was good compliance with NICE guidance at diagnosis and implementation of GH treatment. Compliance with local guidance for annual surveillance of TFTs and IGF-I did not meet our target. We have raised awareness within the team and planned a re-audit for 2019.
22 - 24 Nov 2017
British Society for Paediatric Endocrinology and Diabetes