Emerging treatments
Recombinant human growth hormone
Final height may be reduced in patients with X-linked hypophosphataemia. In one small non-randomised trial, administration of recombinant human growth hormone (rhGH) to children with X-linked hypophosphataemic rickets resulted in a sustained increase in age-standardised height during 2 years of treatment. Pre-pubertal children responded better to rhGH than pubertal children.[35] In an earlier 3 year randomised controlled open-label study, growth hormone improved linear growth, without progression of body disproportion, in short children with X-linked hypophosphataemia.[36] 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 hypophosphataemia.[36][37] One systematic review did not show enough high‐certainty evidence to recommend the use of rhGH in children with X‐linked hypophosphataemia.[38]
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 hypophosphataemic rickets.[39] A subsequent 3 month randomised trial of 21 patients with X-linked hypophosphataemia found no effect of nasally administered salmon calcitonin on circulating levels of FGF-23.[40]
Paricalcitol
Prevention of hyperparathyroidism is important in the ongoing management of X-linked hypophosphataemia. One controlled trial reported a reduction in parathyroid hormone levels in adults with X-linked hypophosphataemia who were randomised to receive paricalcitol daily for one year.[41]
Use of this content is subject to our disclaimer