Emerging treatments
Recombinant human growth hormone
Final height may be reduced in patients with X-linked hypophosphatemia. In one small nonrandomized trial, administration of recombinant human growth hormone (rhGH) to children with X-linked hypophosphatemic rickets resulted in a sustained increase in age-standardized height during 2 years of treatment. Prepubertal children responded better to rhGH than pubertal children.[33] In an earlier 3 year randomized controlled open-label study, growth hormone improved linear growth, without progression of body disproportion, in short children with X-linked hypophosphatemia.[34] Follow-up analysis of the open label study found that growth hormone treatment did not significantly increase adult height in this group of short children with X-linked hypophosphatemia.[34][35] One systematic review did not show enough high‐certainty evidence to recommend the use of rhGH in children with X‐linked hypophosphatemia.[36]
Calcitonin
One report noted that a single subcutaneous injection of salmon calcitonin could reduce FGF-23 levels (a hormone that decreases phosphate reuptake in the kidney) in adults (n=7) with X-linked hypophosphatemic rickets.[37] A subsequent 3 month randomized trial of 21 patients with X-linked hypophosphatemia found no effect of nasally administered salmon calcitonin on circulating levels of FGF-23.[38]
Paricalcitol
Prevention of hyperparathyroidism is important in the ongoing management of X-linked hypophosphatemia. One controlled trial reported a reduction in parathyroid hormone levels in adults with X-linked hypophosphatemia who were randomized to receive paricalcitol daily for one year.[39]
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