HRQoL in Adolescents with Idiopathic Isolated GHD: rhGH (Dis)continuation in Mid-Puberty

Abstract

Objective: To evaluate health-related quality of life (HRQoL) in adolescents with idiopathic isolated growth hormone deficiency (IIGHD) who tested GH-sufficient, comparing those who discontinued recombinant human growth hormone (rhGH) at mid-puberty with those who continued until near-adult height (NAH).

Design: This multicentre prospective study used a patient-preference design. Previous findings showed NAH did not differ between groups. Height influences quality of life (QoL), particularly during adolescence when appearance and social comparison affect psychological development. The impact of height and treatment decisions on HRQoL during puberty remains unclear.

Methods: Adolescents with IIGHD who had received rhGH for ≥3 years and tested GH-sufficient in mid-puberty chose to continue or discontinue treatment. HRQoL was assessed at mid-puberty and NAH using QoLISSY (patient and parent reports), supplemented by KIDSCREEN-52, SDQ, and EQ-5D-Y.

Results: Of 127 participants, 44 continued rhGH and 83 discontinued. Questionnaire completion was 58% (n=74) at mid-puberty and 66% (n=84) at NAH. No significant differences in patient-reported QoL were observed between groups at either time point. Parents reported higher QoL in the discontinuation group at mid-puberty. Overall, QoL scores were within normal ranges and positively correlated with height SDS at both time points.

Conclusions: Discontinuing rhGH in adolescents with IIGHD who tested GH-sufficient at mid-puberty does not appear to negatively affect perceived QoL. Parental reports suggest greater well-being in the discontinuation group, possibly reflecting pre-existing satisfaction with height and health. These findings emphasize considering both physical and psychosocial factors in treatment decisions and incorporating patient and parent perspectives during puberty.

Keywords: GHD; Growth Hormone Deficiency; HRQoL; IIGHD; Idiopathic Isolated Growth Hormone Deficiency; Mid-puberty; Puberty; QoL; Quality of Life.

Figure 1

Violin plot showing the distribution of QoLISSY total scores for both GHcont (n = 22; 50%) and GHstop (n = 52; 63%) groups at mid-puberty, separated by respondents: patients and parents. The plots illustrate the density of the data across the score range, along with median values and overall spread for each category. The horizontal lines within the violin plot indicate the median and quartiles.

Figure 2

Scatter plot illustrating the relationship between height SDS and QoLISSY-Child (QoLISSY-C) total score at the time of inclusion. Total group: r(72) = 0.54, P < 0.001; GHcont: r(20) = 0.61, P = 0.003; and GHstop: r(50) = 0.52, P < 0.001.

Figure 3

Scatter plot showing the positive correlation between NAH-SDS and total QoLISSY-Child (QoLISSY-C) score at NAH. Total group: r(82) = 0.53, P < 0.001; GHcont: r(24) = 0.51, P = 0.008; and GHstop: r(56) = 0.54, P < 0.001.

Figure 4

Scatter plot illustrating the positive correlation between total QoLISSY scores reported by children (QoLISSY-C) and their parents (QoLISSY-P) at mid-puberty. Significant correlations were observed in the total sample (r(70) = 0.66, P < 0.001), the GHcont group (r = 0.857, P < 0.001), and the GHstop group (r = 0.631, P < 0.001).