medwireNews: The results of a meta-analysis support a modest positive effect of recombinant human growth hormone (rhGH) on the height of children with achondroplasia.

The 498 children evaluated, from 12 studies, had a baseline height standard deviation score (SDS) of –5.069. They received rhGH at a median weekly dose of 0.21 mg/kg, which, over the course of 5 years, brought their height SDS up to –3.941.

However, most of the height gains occurred over the first 12 months, with average height SDS improving to –4.325. It improved slightly more, to –4.073, by 24 months, after which it stabilised, although only 21 children had data for 5 years of treatment.

And just one study reported height data for more than 5 years of treatment, preventing Francesco Massart (St Chiara University Hospital of Pisa, Italy) and study co-authors from determining the effects of rhGH treatment on the patients’ final adult height.

Data on sitting height were available for 70 patients from two studies. Among these patients, sitting height SDS was –1.516 at baseline, improving to –0.890 at 12 months and –0.469 at 24 months.

A few individual studies reported that rhGH treatment improved, or at least did not worsen, patients’ body proportions, but the researchers were unable to analyse the data across the studies because of “limited and nonstandardized” body proportion measurements.

“Therefore, the effect on body disproportion in [achondroplasia] during rhGH treatment still needs to be established”, they write in Hormone Research in Paediatrics.

The team also looked in the individual studies for reports of spinal cord compression, narrowing of the foramen magnum or other signs of worsening stenosis – the presumed cause of hydrocephalus in patients with achondroplasia – but found none, suggesting that rhGH treatment did not worsen stenosis.

“Although the present meta-analysis suggests that rhGH treatment may improve growth pattern in children with [achondroplasia], more data are needed before a final conclusion can be reached”, conclude the researchers.

By Eleanor McDermid

Horm Res Paediatr 2016; Advance online publication

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