BACKGROUND: X-linked hypophosphatemic rickets (XLH) is characterized by rickets, disproportionate short stature, and impaired renal phosphate reabsorption and vitamin D metabolism. Despite oral phosphate and vitamin D treatment, most children with XLH demonstrate reduced adult height. OBJECTIVE: To determine the beneficial effects of recombinant human growth hormone (rhGH) therapy on body proportions and adult height among patients with XLH. METHODS: Three initially prepubertal short children (age, 9.4-12.9 years) with XLH were treated with rhGH for 3.1 to 6.3 years until adult height was attained. RESULTS: rhGH treatment led to sustained increases in standardized height for all children. The median adult height was 0.9 SD (range: 0.5-1.3 SD) greater than that at the initiation of rhGH treatment and exceeded the predicted adult height by 6.2 cm (range: 5.3-9.8 cm). However, longitudinal growth of the trunk was stimulated more than leg growth. During rhGH treatment, the standardized sitting height increased by 1.6 SD (range: 1.1-2.7 SD), compared with baseline values. In contrast, the median subischial leg length did not change consistently (median change: 0.3 SD; range: -0.1 to 0.6 SD). CONCLUSION: The increase in final height after rhGH treatment is of potential benefit for children with XLH. However, the exaggeration of disproportionate truncal growth observed for our prepubertal patients is a potential negative effect of treatment and should be confirmed with additional studies.
BACKGROUND:X-linked hypophosphatemic rickets (XLH) is characterized by rickets, disproportionate short stature, and impaired renal phosphate reabsorption and vitamin D metabolism. Despite oral phosphate and vitamin D treatment, most children with XLH demonstrate reduced adult height. OBJECTIVE: To determine the beneficial effects of recombinant humangrowth hormone (rhGH) therapy on body proportions and adult height among patients with XLH. METHODS: Three initially prepubertal short children (age, 9.4-12.9 years) with XLH were treated with rhGH for 3.1 to 6.3 years until adult height was attained. RESULTS: rhGH treatment led to sustained increases in standardized height for all children. The median adult height was 0.9 SD (range: 0.5-1.3 SD) greater than that at the initiation of rhGH treatment and exceeded the predicted adult height by 6.2 cm (range: 5.3-9.8 cm). However, longitudinal growth of the trunk was stimulated more than leg growth. During rhGH treatment, the standardized sitting height increased by 1.6 SD (range: 1.1-2.7 SD), compared with baseline values. In contrast, the median subischial leg length did not change consistently (median change: 0.3 SD; range: -0.1 to 0.6 SD). CONCLUSION: The increase in final height after rhGH treatment is of potential benefit for children with XLH. However, the exaggeration of disproportionate truncal growth observed for our prepubertal patients is a potential negative effect of treatment and should be confirmed with additional studies.
Authors: R Fuente; H Gil-Peña; D Claramunt-Taberner; O Hernández; A Fernández-Iglesias; L Alonso-Durán; E Rodríguez-Rubio; F Santos Journal: Rev Endocr Metab Disord Date: 2017-03 Impact factor: 6.514