Are Short Boys with Constitutional Delay of Growth and Puberty Candidates for rGH Therapy according to FDA Recommendations?

Abstract

Background/Aims: According to FDA-approved guidelines, boys whose height predictions fall to 160 cm or less are considered for treatment with recombinant growth hormone (rGH). The aim of this study was to analyze the value of different height prediction methods by accurately identifying those boys with constitutional delay of growth and puberty (CDGP) in whom final height (FH) prognosis was poor (≤160 cm) and who might therefore be candidates for this treatment modality. Methods: In 69 boys with CDGP diagnosed at a mean age of 14.9 ±1.2 years, FH prediction was calculated by means of Bayley-Pinneau (BP), Roche-Wainer-Tissen (RWT), Tanner-Whitehouse II (TWII) and target height (TH) methods. At the age of 22.6 ± 3.5 years their height was remeasured and the accuracy of height prediction was analyzed. Results: In 6 men (8.7%) measured FH was ≤160 cm. Depending on the prediction method, different individual patients within the 14- to 16-year age range would have been candidates for rGH treatment. The BP method would have recruited 8 subjects of whom only 3 had FH ≤160 cm (sensitivity 50%, specificity 92%). The RWT and TW II methods identified only one recruit, and this patient did achieve FH ≤160 cm (sensitivity 17%, specificity 100%). None of the 8 subjects with FH ≤160 cm would have qualified for GH treatment using the TH method. In three boys none of the four methods predicted their final height ≤160 cm. Conclusion: Although some boys with CDGP may be considered the candidates for rGH treatment according to FDA recommendations, none of the available methods of prediction are sufficiently sensitive to reliably recruit 14- to 16-year-old boys whose final height will fall at or below 160 cm.

Causes of short stature identified in children presenting at a tertiary care hospital in Multan Pakistan

“…13 Those boys whose height predictions fall below160 centimeters, are candidates for treatment with recombinant growth hormone (rGH) according to FDA-approved guidelines is under investigation. 14 …”

An approach to constitutional delay of growth and puberty

“…However, none of the available methods of prediction are sufficiently sensitive to reliably recruit 14 to 16-year- old boys with CDGP whose final Ht will fall at or below 160 cm. [68–70] Before the onset of puberty, many boys with CDGP are short (HtSDS < -2) and have delayed bone age versus their chronological age. GH treatment for boys ( n = 36) with non-familial short children with delayed bone age, started at an average age of 9.3 years (using 0.6 U/ kg/ week divided on daily doses) resulted in a Ht gain of 1.4 SD at the final Ht.…”

The Effect of Clonidine Therapy on Height and Growth Velocity in Constitutional Growth and Puberty Delay

“…The use of clonidine in children with CGDP has been the subject of several studies [8,9] but, very divergent results have been reported and therefore the effectiveness of clonidine in increasing growth rate of CGDP is not clear. Many subjects affected by constitutional short stature show a good response to human growth hormone (HGH) therapy, whereas others do not benefit by this treatment [10][11][12][13]. The use of aromatase inhibitors and oxandrolone, a weak anabolic steroid has been shown to be effective in children with CGDP.…”