Recombinant growth hormone therapy for X-linked hypophosphatemia in children

Cochrane Database Syst Rev. 2005 Jan 25:(1):CD004447. doi: 10.1002/14651858.CD004447.pub2.


Background: Conventional treatment of X-linked hypophosphatemia with oral phosphate and calcitriol can heal rickets, but it does not always raise serum phosphate concentrations significantly, nor does it always normalize linear growth. Some clinical trials suggest that combining recombinant human growth hormone therapy with conventional treatment improves growth velocity, phosphate retention, and bone mineral density, but some clinical trials suggest that it appears to aggravate the pre-existent disproportionate stature of such children.

Objectives: To determine whether recombinant human growth hormone therapy for children with X-linked hypophosphatemia is associated with changes in longitudinal growth, mineral metabolism, endocrine function, renal function, bone mineral density, body proportions, and also with any adverse effects.

Search strategy: Relevant trials were identified from searching the Cochrane Central Register of Controlled Trials Issue 3, 2003 and Ovid MEDLINE 1966 to September 2003. Additional trials were identified from the reference lists of identified trials and other reviews. We also searched the Journal of Bone and Mineral Research (1986 to 2003) and proceedings of the American Society for Bone and Mineral Research Annual Meeting (1st to 24th). Date of most recent search: November 2003.

Selection criteria: All randomized controlled trials or quasi-randomized controlled trials comparing growth hormone (alone or combined with conventional treatment) with either placebo or conventional treatment alone in children with X-linked hypophosphatemia.

Data collection and analysis: Two reviewers independently assessed trials for methodological quality and extracted data from eligible trials.

Main results: The searches identified five trials, of which one met the inclusion criteria, including a total of five participants. In this trial, rhGH therapy improved the height standard deviation score (z score), and transiently increased serum phosphate and tubular maximum for phosphate reabsorption.

Authors' conclusions: We have found no conclusive evidence to indicate that the use of recombinant human growth hormone therapy in children with XLH is associated with changes in longitudinal growth, mineral metabolism, endocrine, renal function, bone mineral density, body proportions, but it does not appear to have any adverse effects.

Publication types

  • Review
  • Systematic Review

MeSH terms

  • Child
  • Genetic Diseases, X-Linked / drug therapy*
  • Growth Hormone / therapeutic use*
  • Humans
  • Hypophosphatemia, Familial / drug therapy*
  • Hypophosphatemia, Familial / genetics
  • Randomized Controlled Trials as Topic
  • Recombinant Proteins / therapeutic use


  • Recombinant Proteins
  • Growth Hormone