medwireNews: The first 2 years of recombinant growth hormone (rGH) therapy offers significant benefits for prepubertal children with Silver–Russell syndrome (SRS), French researchers have found.
As well as improving height, their study shows that early treatment with “rGH also plays a part in the nutritional intervention”, alongside early oral feeding, nutritional enrichment and cyproheptadine, the authors report in the Journal of Clinical Endocrinology & Metabolism.
And they note: “These improvements were achieved using the usual, stable doses of rGH recommended by the [European Medical Authority] for children born [small for gestational age] with no catch-up growth.”
Eloïse Giabicani (Hôpital Armand Trousseau, Paris) and co-workers reviewed the impact of rGH therapy on the growth and nutritional status of 77 children (53% boys) with a molecular diagnosis of SRS based on either loss of methylation on 11p15 (11p15 LOM; 78%) or maternal uniparental disomy for chromosome 7 (upd(7)mat; 22%).
At rGH therapy initiation, children with 11p15 LOM SRS were of a similar age to those with upd(7)mat SRS (median 3.63 vs 3.16 years) but were smaller (height standard deviation score [SDS] of –2.7 vs –3.3), although not significantly so. All children met Netchine-Harbison Clinical Scoring System criteria of 4 items for SRS diagnosis, although those with 11p15 LOM had significantly less body asymmetry than those with upd(7)mat. Children with 11p15 LOM were alsoborn at a significantly later gestational age (37.7 vs 36.0 weeks).
Overall, 71.4% of children achieved height within the normal range (SDS > –2) after 2 years of therapy and there was no relationship between rGH dosing and growth velocity, or between birth weight or length and height gain.
Children with upd(7)mat SRS had significantly greater median height gains than those with 11p15 LOM SRS, at 1.70 and 1.07 SDS, respectively, but the researchers say that after adjusting for ideal weight for height (WfH), height and age at rGH therapy initiation, as well as birth weight and length, there was no significant association between height gain after 2 years of treatment and molecular subgroup.
Further analysis indicated that height gain was significantly greater for children who began treatment before 4 years of age than those who started treatment later, at an SDS gain of 1.45 versus 1.10, and this relationship persisted after adjusting for baseline height and target height. In addition, girls had a better height improvement than boys after 2 years of therapy (SDS 1.50 vs 1.20), as well as a greater increase in BMI (SDS 0.7 vs 0.2).
Turning to nutritional status, Giabicani et al report that the children experienced a significant improvement in ideal WfH after 1 and 2 years of rGH therapy, with significantly fewer showing a WfH below 75% and significantly more having a WfH above the target of 85%, including 5.2% with a WfH above 100%.
The researchers note that across the study population there were similar improvements in WfH and BMI over 2 years of treatment, so that the proportion of children with a BMI of 12–14 kg/m2 was consistent after 1 and 2 years (66.2 and 62.3%, respectively).
After 2 years of rGH therapy, both molecular subgroups were less likely to require cyproheptadine or enteral nutrition than at baseline. Children who required cyproheptadine had lower BMI and WfH than those who did not; in the year before rGH initiation they had gains in BMI and WfH but this was no longer true after rGH therapy.
Children who required enteral nutrition had lower height and growth velocity than those who did not in the year before rGH therapy; their BMI increased significantly only in the first year of treatment, and there was no significant change in their WfH with treatment, which the researchers suggests confirms the “the preponderance of optimized nutritional management in these children with very severe feeding difficulties.”
They add that the evolution in WfH and BMI did not differ between children who did and did not begin rGH therapy before age 4 years, or by molecular subgroup.
Emphasizing that “nutritional status is crucial for these children, especially during the early years of life, as there is a high risk of hypoglycaemia”, the researchers write that “[e]ven for children with mild feeding difficulties, monitoring both BMI and WfH is essential to accurately track weight gain and prevent rapid catch-up growth, which can predispose children to metabolic disturbances.”
They continue that, “[i]n addition, precise longitudinal assessments of body composition will enhance our understanding of the nutritional trajectories of these children and allow us to better target an individualized BMI or WfH for each child.”
By Lynda Williams
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