Recombinant human growth hormone (rhGH) is approved for short stature associated with growth hormone deficiency (GHD), idiopathic short stature (ISS), Turner syndrome (TS), multiple pituitary hormone deficiencies (MPHD), Silver Russell syndrome (SRS) and being born small for gestational age non syndromic (SGA). Objectives: To assess the clinical effectiveness and cost-effectiveness of rhGH in children with GHD, TS and those born SGA. Methods: Five hundred children 331(66.2%) females & 169 (33.8%) males; 98 with GHD, 149 with ISS, 190 with TS, 18 with MPHD, 33 with SRS and 12 with SGA were referred from different schools all over Egypt to the GH National Committee of the school health insurance, where they were diagnosed, provided by growth hormone therapy in the period from (March 2015 to March 2016). Demographic, auxiological and laboratory variables were tested as being predictors for height gain (cm/year) using multiple regression analysis. Markov cost-effectiveness simulation model was used for estimation of the cost-effectiveness of growth hormone therapy for each diagnosis. Results: The cost in GHD, ISS, TS, MPHD, SRS and SGA groups were 168.88±131.57 dollars per 5.66±1.52 cm, 182.83±78.31 dollars per 6.19±1.46 cm, 339.93±202.61 dollars per 5.04±1.62 cm, 383.15±183.42 dollars per 3.5±0.47 cm, 71±13.98 dollars per 5.49±0.93 cm and 139.43±62.11 dollars per 5.13±0.21 cm height gain/year respectively. There was a significant correlation between cost & height gain (P-value =0.0001, 0.001 & 0.01) in ISS, GHD and SGA respectively. Conclusion: The study identified the predictor variables for height gain/year for the different diagnoses receiving GH therapy. Given that the rising cost of prescription drug therapies is a prominent issue for our health care system, rhGH is a cost-effective treatment strategy in Egypt for children with GHD, ISS, TS and SGA patients.
27 - 29 Sep 2018
European Society for Paediatric Endocrinology