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GH therapy improves emotional, social quality of life in children with short stature
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Children with short stature who received 1 year of growth hormone treatment experienced improvements in emotional and social quality-of-life measures when compared with baseline assessments, according to an analysis of two pediatric quality-of-life scales.
“Studies of both general and condition-specific [quality of life] have shown lower scores in children with various chronic conditions compared to healthy peers,” Michel Polak, MD, PhD, of the pediatric endocrinology department at Hôpital Universitaire Necker-Enfants Malades in Paris, and colleagues wrote in the study background. “Growth hormone deficiency and small-for-gestational age status have been associated with impairments in psychological well-being that sometimes persisted into late adolescence and early adulthood. Compared to healthy normal-stature controls, children with short stature but no GH deficiency exhibited [quality of life] impairments, whereas those of similar short stature with GH deficiency and GH treatment did not.”
In an observational study, Polak and colleagues analyzed data from 80 children aged at least 4 years who initiated GH therapy for short stature between April 2012 and September 2015 at Necker-Enfants Malades University Hospital (47 boys; mean age, 11 years). Researchers assessed health-related quality of life via the 50-item PedsQL questionnaire (score expressed as percentage, with scores from zero to 100%), and height-specific quality of life via the 50-item QoLISSY questionnaire at baseline and again during the closest visit performed after 1 year of GH treatment. For the QoLISSY questionnaire, total score is computed as the mean score on three core subscales (physical, emotional and social).
Within the cohort, 26 children had GH deficiency, 24 were born small for gestational age, 18 had bone dysplasia and six had idiopathic short stature. Median baseline height was –2.5 standard deviations (SD). Researchers stratified the cohort according to severity of short stature at baseline, including 22 children in the most severe group ( –3 SD), 23 children in the intermediate group (> –3 to –2.5 SD) and 29 children in the less severe group (> –2.5 to –2. SD).
Researchers found that children in the most severe group demonstrated baseline quality-of-life scores comparable to children with chronic illness, as well as a significant decrements in social quality of life (P = .02), school functioning quality of life (P = .02) and total quality of life (P = .03) when compared with children in the less severe short stature group.
At 1 year, researchers observed improvements in PedsQL scores across all three short stature groups, with the biggest gains observed in the severe group in the emotional component (median increase, 25%) and social component (median increase, 17%). There were no changes in PedsQL parent-report scores during the same period, apart from a small improvement in the social domain (median increase, 5%), according to researchers.
Researchers observed a correlation between self-reported and parent-reported increase in total quality of life assessed by the QoLISSY questionnaire (R = 0.45; P < .001). The researchers noted that the QoLISSY scores for GH treatment-specific aspects of quality of life exhibited a moderate positive correlation with the improvement in total quality of life in both the children’s questionnaires (R = 0.559; P < .00001) and parents’ questionnaires (R = 0.532; P < .001). The QoLISSY parent-report questionnaire was also positively correlated with gains in height SD score (R = 0.638; P < .00001).
“[Quality of life] is strongly affected by short stature, especially when height is less than –2.5 SD, even in patients with no underlying medical conditions,” the researchers wrote. “In these patients, emotional and social [quality of life], assessed by both patients and parents, significantly improves after 1 year of GH therapy. It is therefore of the utmost importance to refer children with short stature to pediatric endocrinology care as early as possible, to limit the severity of short stature and the potential long-term consequences on quality of life.” – by Regina Schaffer
Disclosures: Pfizer supported this study with an educational grant. The authors report no relevant financial disclosures.
Perspective
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This study attempts to quantify the social and emotional stress experienced by children with different degrees of short stature. Although this has been previously studied by various authors, the current study is the first to report on the variation in quality of life of short children by using a general quality-of-life scale, as well as a height-specific scale.
While the authors report significant improvement in pediatric quality-of-life and height-specific scales, it is important to remember that the number of children studied was small and the duration of therapy (1 year) may not be adequate to make a conclusion about the long-term effect of GH therapy on quality of life in children with short stature. The study also found that the quality-of-life scores of the shortest group at baseline were comparable to a “chronically ill” population.
The take-home point from this study is that severely short children (–3 SD) likely experience increased stress and decreased height-specific quality of life, and this should be taken into account when evaluating these children for therapy.
Sumana Narashimhan, MD, FAAP
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