medwireNews: Children with short stature show marked improvements in their emotional and social quality of life (QoL) during their first year of treatment with growth hormone (GH), study findings suggest.
“Our results show that GH treatment for one year has a significant impact on social and emotional QoL, whereas a physical impact, which could be expected given the visible gain in height, seems to be less important”, say Michel Polak (Hôpital Universitaire Necker-Enfants Malades, Paris, France) and co-researchers.
“This may likely be related to the moderate physical impact of short stature at baseline”, they write in The Journal of Clinical Endocrinology & Metabolism.
At treatment initiation, the 74 children (57% boys) in the study were aged a median of 10.9 years. They had GH deficiency, had been born small for gestational age, or had bone dysplasia, dyschondreosis and idiopathic short stature. The researchers excluded children with other conditions that could potentially reduce life quality, allowing them “to independently evaluate the influence of short stature on QoL.”
Patient-reported physical QoL was impaired only in children with a height standard deviation score (SDS) below –3.0, in whom it was in line with that of an age-matched population of chronically ill children. Their emotional, social and school QoL, assessed with the PedsQL tool, was below that of the chronically ill children, as was their total QoL.
Children with height SDS between –3.0 and –2.5 had emotional QoL that was similar to that of chronically ill children, but their scores were close to general population scores for other domains and for total QoL. Likewise, on the condition-specific Quality of Life in Short Stature Youth (QoLISSY) questionnaire, children with the shortest stature had the poorest QoL.
The group as a whole had a median height SDS of –2.5 at baseline, and they gained an average of 0.7 SDS during their first year of GH treatment. QoL scores also significantly improved during this time, although the researchers note that scores improved by less than 0.5 SD, the level considered to be a clinically relevant improvement, therefore “indicating a need for circumspection.”
But they stress: “It is important to keep in mind that emotional problems that appear during childhood, linked to chronic conditions, may persist beyond childhood into adult life.”
Polak and team caution that their study was not placebo controlled, so the QoL improvement could be explained by a placebo effect.
However, both children’s and parents’ QoLISSY scores correlated significantly with gains in height SDS, which accounted for around 30–40% of the improvement in QoL.
Also, the largest QoL gains were seen in children with the most severe short stature at baseline (below –3.0 SDS), and these factors are “a good argument against placebo effect”, says the team.
By Eleanor McDermid
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J Clin Endocrinol Metab 2019; doi:10.1210/jc.2018-02523