Early treatment improves growth and biochemical and radiographic outcome in X-linked hypophosphatemic rickets

Autor: Andrea S. Doria, Outi Mäkitie, William G. Cole, Alan Daneman, Etienne Sochett, Sang Whay Kooh
Rok vydání: 2003
Předmět:
Zdroj: The Journal of clinical endocrinology and metabolism. 88(8)
ISSN: 0021-972X
Popis: X-Linked hypophosphatemic rickets (XLH) is characterized by hypophosphatemia, rickets, and impaired growth. Despite oral phosphate and 1,25-dihydroxyvitamin D3 treatment, many patients have suboptimal growth and bone healing. The aim of this study was to assess whether age at treatment onset impacts the outcome. Growth data, biochemistry, and radiographs of 19 well-controlled patients with XLH were analyzed retrospectively. Patients were divided into two groups based on the age at treatment onset (group 1, 0.05) and until predicted adult height (−0.2 SDS vs. −1.2 SDS; P = 0.06). The degree of hypophosphatemia was similar in both groups, but serum alkaline phosphatase remained higher in group 2 throughout childhood. Radiographic signs of rickets were more marked in group 2, but even patients with early treatment developed significant skeletal changes of rickets. These data suggest that treatment commenced in early infancy results in improved outcome in patients with XLH, but does not completely normalize skeletal development.
Databáze: OpenAIRE