Long-term somatropin data underline need for early GH deficiency treatment

Eleanor McDermid
Eur J Endocrinol 2017; Advance online publication
31 August 2017

medwireNews: Findings from the real-world NordiNet® International Outcome Study demonstrate clear benefits of early diagnosis and treatment initiation in patients with isolated growth hormone deficiency (GHD).

However, Michel Polak (Hôpital Universitaire Necker Enfants Malades, Paris, France) and study co-authors stress that all patients achieved a near-adult height standard deviation score (SDS) within the normal range of –2.0 to +2.0, “albeit at the lower end of the range for those who were older at treatment start”.

The team assessed 172 patients (66.3% male) with isolated GHD and a GH stimulation test peak value of 10 ng/mL or less who achieved near-adult height during the study period. They divided them into those who started somatropin treatment early (<8 years for girls and <9 years for boys), at an intermediate age (8–10 and 9–11 years, respectively) or late (>10 and >11 years, respectively).

At near-adult height, the average height SDS was –1.0, –1.5 and –1.3 in the early, intermediate and late groups, respectively. The corresponding differences with mid-parental heights were –0.2, –0.2 and –0.6, and the SDS changes since starting treatment were 2.3, 1.4 and 1.5.

Of note, 90 – more than half – of the children fell into the late starters category, the researchers report in the European Journal of Endocrinology.

“[T]hese data being representative of real-life clinical practice may suggest that a proportion of children with isolated GHD start GH treatment late, even though compelling evidence shows that they would benefit most from starting GH treatment at an early age”, they write.

Having severe GHD, defined as a GH stimulation test peak value below 3 ng/mL, was also associated with improved treatment response, particularly for the early and intermediate starters, who achieved respective SDS increases of 3.5 and 2.7 compared with 1.7 and 1.1 for those with non-severe GHD.

“Our results highlight the importance of adequately communicating to children with non-severe GHD and their carers the possibility of a more modest outcome at the end of treatment”, say Polak and team.

In line with previous research, higher baseline and target height SDS predicted a good treatment response. The researchers say the latter “is not surprising given that short children with tall parents have a greater genetic height potential than those with short parents.”

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