OBJECTIVES

Paediatric growth hormone deficiency (pGHD) affects 1/4,000 children. Treatment with daily sub-cutaneous injections of recombinant human growth hormone (r-hGH) increase height velocity and quality of life (QoL). However, adherence to daily r-hGH varies; one study reported 66% of children were non-compliant (>1 injection/ week missed)¹. A recent randomised controlled clinical trial (NCT02968004) evaluated the efficacy/safety of weekly Somatrogon (hGH-CTP) and daily Genotropin in pGHD. QoL (an exploratory endpoint) was evaluated using the validated Quality of Life in Short Stature Youth (QoLISSY) questionnaire, which includes three subscales (physical, social, emotional) and total score.

METHODS

The QoLISSY core module was administered to patients (aged 3-11 years [girls], 3-12 years [boys]) and parents in eight countries, at Baseline (BL) and 12 months after treatment start. The QoLISSY-CHILD was completed by children aged ≥7 years; QoLISSY-PARENT was completed by the Caregiver for children < 7 years.

RESULTS

For Total QoLISSY-PARENT, in the Somatrogon group (N=19), mean scores are 61.82 (BL) and 69.49 (month 12) with mean change of 8.48 (95% Confidence Interval [CI]: 1.06, 15.89). In the Genotropin group (N=28), mean scores are 54.58 (BL) and 63.80 (month 12) with mean change of 8.51 (CI: 2.27, 14.74). For Total QoLISSY-CHILD (> 70% male), in the Somatrogon group (N=35), mean scores are 61.48 (BL) and 74.69 (month 12) with mean change of 13.00 (CI: 5.81, 20.19). In the Genotropin group (N=35), these scores are 60.96 (BL) and 69.03 (Month 12) with mean change of 7.84 (CI: 2.71, 12.97). All three sub-scales (both QoLISSY versions) showed similar trends in improvement over 12 months. Scores of >70 indicate a good QoL.

CONCLUSIONS

QoLISSY-CHILD and QoLISSY-PARENT demonstrated comparable QoL improvements for children with pGHD by end of Month 12 in weekly Somatrogon and daily Genotropin treated groups.

¹Cutfield et al PLoS ONE 6.1 (2011): 1-3