Growth hormone therapy in pre‐pubertal children with Noonan syndrome: first year growth response and comparison with Turner syndrome

Abstract

We studied the growth-promoting effect of treatment with recombinant human growth hormone in 23 pre-pubertal children with Noonan syndrome, aged between 5.4 and 14.3 y, and all with a height < 1.4 SD for Tanner standards. The growth response and skeletal maturation after 1 y of recombinant human growth hormone treatment (0.15 U/kg/day given by daily injection) in the Noonan syndrome patients was compared with the auxological changes observed in a group of 17 girls with Turner syndrome with a comparable age and height deficit who were treated with recombinant human growth hormone in a similar way. During 1 y of treatment, the mean +/- SD height velocity increased by 4.0 +/- 1.6 cm/y in the Noonan syndrome group and by 3.6 +/- 1.3 cm/y in the Turner syndrome group. Height SDS for chronological age in the Noonan syndrome group increased by 0.53 +/- 0.46 (p < 0.001). In the Noonan syndrome patients the changes in height velocity were positively related to birthweight (r = 0.48, p < 0.05). The changes in height velocity or height SDS were not related to the age, height deficit or a delay in bone age maturation at start of treatment. In neither the patients with Noonan syndrome nor Turner syndrome was an acceleration of bone maturation found. We conclude that treatment with recombinant human growth hormone in pre-pubertal NS patients induces an increase in height velocity and height SDS comparable to that observed in Turner syndrome girls.

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“…The ethical requirements are that there be a reasonable balance between the potential risks and the potential benefits to the subject or to society 1 and that the subject make a fully informed choice about participating in the research. 2 Within the context of a reasonable overall risk-benefit ratio, risks should be minimized by using medically and scientifically appropriate procedures. 1 Second, preventing subjects from deciding what risks they are willing to take is a direct violation of the principle of autonomy.…”

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“…As with all trials, patients must be informed of the potential risks, and risks must be reasonable in relation to the possible benefits to the individual patient as well as to society. [1][2][3] We believe it is a mistake to hold surgical trials to a standard more stringent than that required for the study of nonsurgical interventions.…”

“…Data from trials involving older patients are relevant only to the minority of patients who are considered suitable for entry into the trials, and the results derived are not *Data were obtained from the Surveillance, Epidemiology, and End Results program. 2 All patients were followed through 1996. The number of patients represents the total number of patients at the beginning of the five-year period in question, and the percentage represents the percentage surviving after five years.…”