Background: Rathkes cleft cyst (RCC) has been reported rarely in children and adolescents. The occurrence of RCC with hypoplastic anterior pituitary can be explained by their common embryologic origin. However, the effects and sfety of recombinant human GH (rhGH) in GH deficient (GHD) children with RCC has not been previously reported.
Objective and hypotheses: To assess the effects and safety of recombinant human GH (rhGH) in GH deficient (GHD) children with RCC.
Method: The clinical data of 15 GHD children aged from 512 years old, whose radiologic diagnosis showed RCC during Jan 2013 to Dec 2015 in Sun Yat-sen Memorial Hospital, were analyzed retrospectively. rhGH was given subcutaneously to each enrolled child with a night dose of 0.1 IU/kg 67 times a week for 1230 months. The serum biochemical indices as well as endocrine hormone level were detected regularly. The clinical data before and after treatment were compared, including height, weight, growth velocity, height standard deviation scores (height SDS), insulin-like growth factor 1 (IGF-1), bone age and the magnetic resonance imaging result.
Results: With rhGH treatment, a significant increase (P<0.01) of growth velocity in all 12 children, from 4.06±0.61 to 9.86±4.01 cm, was observed in the first 12 months. Meanwhile the height SDS increased obviously from −3.31±1.47 to −2.63±1.36 (P<0.01). In addition, IGF-1 rose from (186.73±73.32) μg/l to (436.78±208.60) μg/l (P<0.01), with IGFBP-3 from (4.32±0.96) mg/l to (5.63±1.45) mg/l. The peak values of both IGF-1 and IGFBP-3 were within normal limits. During the treatment and the follow-up period, the biochemical indices were normal and the volumes of the Rathke cysts did not increase.
Conclusion: The treatment of low level rhGH in GHD children with RCC was demonstrated effective in this study. Moreover, GH treatment is safe when fully evaluated and closely monitored.
10 - 12 Sep 2016
European Society for Paediatric Endocrinology