Treatment of the rickets of cystinosis with 1-alpha-hydroxycholecalciferol


Infantile nephropathic cystinosis is a disease in which affected children present with the consequences of renal tubular dysfunction. Usually these children develop progressive renal impairment and die before the age of 10. Rickets is usually an early manifestation of the disease and normally requires considerable doses of vitamin D to control it. The patients described in this report all had radiological signs of rickets despite treatment with large doses of vitamin D. An attempt was made to control the rickets with la-hydroxycholecalciferol (la-OHD3), an available analogue of the active metabolite of vitamin D. By using this compound we aimed to achieve safe and effective control of the rickets while deriving information which might lead to a better understanding of the pathophysiology of the rickets of cystinosis.

DOI: 10.1007/BF02064042

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@article{Gertner1977TreatmentOT, title={Treatment of the rickets of cystinosis with 1-alpha-hydroxycholecalciferol}, author={Joseph M. Gertner and David P. Brenton and C. E. Dent and Montserrat Dom{\'e}nech}, journal={Calcified Tissue Research}, year={1977}, volume={22}, pages={63-67} }