A trial of growth hormone therapy in well-controlled hypophosphataemic rickets.
OBJECTIVE
Conventional therapy of hypophosphataemic rickets (HR) with oral phosphate and calcitriol does not always result in normal linear growth. Recombinant human growth hormone (rhGH) offers theoretical advantages as an adjunctive therapy. We aimed to determine the effects of adjunctive rhGH therapy in children with well-controlled HR.


METHODS
In this report, 5 prepubertal children (aged 3.5-10.9 years) with well-controlled HR on conventional therapy were given adjunctive standard dose rhGH therapy for one year.


METHODS
Height, growth velocity, metabolic markers of calcium and phosphate metabolism, body composition, bone mineral density, wrist and knee X-rays, and renal sonography were assessed at regular intervals. Height and growth velocities were also calculated 12 months after ceasing rhGH therapy.


RESULTS
After 12 months therapy with rhGH, no significant biochemical or radiological benefits were observed. A significant increase in height SD score was observed (P = 0.023), but this was not associated with any increase in the growth velocity SD score and appears to have been due to catch-up growth caused by conventional therapy alone. When rhGH therapy was ceased, no significant decreases in mean height SD or growth velocity SD scores were observed.


CONCLUSIONS
In well-controlled hypophosphataemic rickets patients receiving conventional therapy, adjunctive therapy with standard dose rhGH offers no benefits in linear growth or rachitic disease markers.
Cameron
F J
FJ
Division of Endocrinology, University of Toronto, Hospital for Sick Children, Ontario, Canada.
Sochett
E B
EB
Daneman
A
A
Kooh
S W
SW
D001823:Body Composition / Q000187:drug effects; D001827:Body Height / Q000187:drug effects; D015519:Bone Density / Q000187:drug effects; D002117:Calcitriol / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D004359:Drug Therapy, Combination; D005260:Female; D005500:Follow-Up Studies; D013006:Growth Hormone / Q000627:therapeutic use*; D006801:Humans; D007015:Hypophosphatemia, Familial / Q000188:drug therapy* / Q000378:metabolism / Q000503:physiopathology; D008297:Male; D010710:Phosphates / Q000378:metabolism / Q000627:therapeutic use
D010710:Phosphates; D013006:Growth Hormone; D002117:Calcitriol