Health-related quality of life in pre-pubertal children with pediatric growth hormone deficiency: 12-month results from a phase 3 clinical trial of once-weekly somatrogon versus once-daily somatropin

Abstract

Objective

Treatment of pediatric growth hormone deficiency (pGHD) with daily injection of recombinant human growth hormone (somatropin) aims to increase height velocity and improve health-related quality of life (HRQoL). The Quality of Life in Short Stature Youth (QoLISSY) questionnaire was administered in a phase 3 clinical trial that evaluated efficacy and safety of once-weekly somatrogon versus once-daily somatropin in children with pGHD (ClinicalTrials.gov no NCT02968004).

Methods

Treatment-naïve prepubertal children with pGHD received once-weekly somatrogon or once-daily somatropin for 12 months. The QoLISSY core module (physical/social/emotional subscales) was administered at baseline and 12 months after treatment initiation. QoLISSY-Parent was completed by parents/caregivers of children <7 years old and some parents/caregivers of children ≥7 years old; children ≥7 years old self-completed QoLISSY-Child.

Results

Baseline characteristics were similar between treatment groups (N = 117). Among children <7 years old, QoLISSY-Parent total and subscale scores showed similarly improved HRQoL at 12 months relative to baseline in both treatment groups. Self-reported QoLISSY-Child total and subscale scores in children ≥7 years old indicated HRQoL improvements at 12 months that were numerically better with somatrogon than somatropin (similar results with QoLISSY-Parent in this age group). At both time points, children reported better HRQoL than perceived by their parents/caregivers.

Conclusion

Treatment for 12 months with once-weekly somatrogon or once-daily somatropin resulted in comparable improvements in HRQoL among children with pGHD. Lower HRQoL perceived by parents/caregivers possibly reflect children’s tendency to emphasize adaptation. These results suggest that evaluation of HRQoL could help support treatment decisions in children with pGHD treated with growth hormone.

PLAIN LANGUAGE SUMMARY

Pediatric growth hormone deficiency is a condition that causes slow growth. Children with this condition have height that is lower than normal unless the condition is treated. The slow growth and short height may have bad effects on the emotional and social well-being of these children. Treatment usually consists of a growth hormone that is administered by daily injection under the skin over a period of years. However, children and their parents may not like these daily injections and often stop treatment. A newer treatment is available that can be injected once weekly. This newer treatment increases growth the same as daily injections. We looked at whether 12 months of treatment given once a week has the same positive effects on the physical, social, and emotional health of children as the daily treatment. Children and their parents answered questions that asked how being short affects the physical, social, and emotional parts of their life. These questions were asked before starting treatment and 12 months after starting treatment. In children younger than 7 years old, improvements at 12 months in their physical, social, and emotional health were similar between the treatments. In children 7 years old or older, those who received the once-weekly injections had slightly better improvements than those who received the daily injections. These results can help parents and doctors make decisions about treating children with pediatric growth hormone deficiency.

Keywords:

• Health-related quality of life
• long-acting growth hormone
• pediatric growth hormone deficiency
• QoLISSY
• somatrogon
• somatropin

Transparency

Declaration of funding

This study was sponsored by OPKO Health and Pfizer Inc.

Declaration of financial/other relationships

Jane Loftus and Srinivas Rao Valluri are employees and stockholders of Pfizer Inc. Julia Quitmann is involved in an ongoing QoLISSY-related study that is funded by Pfizer.

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Author contributions

All authors participated in the interpretation and analysis of the study data, and in the drafting, critical revision, and approval of the final version of the manuscript.

Acknowledgements

This study was sponsored by OPKO Health which is a co-development partner with Pfizer. The authors thank the subjects and their families/caregivers, investigators, research nurses, study coordinators, and operations staff who contributed to this study. Medical writing support was provided by E. Jay Bienen, PhD, and was funded by Pfizer.

Data availability statement

Upon request, and subject to review, Pfizer will provide the data that support the findings of this study. Subject to certain criteria, conditions, and exceptions, Pfizer may also provide access to the related individual de-identified participant data. See https://www.pfizer.com/science/clinical-trials/trial-data-and-results for more information.