Evolution of the Quality of Life of Short Statured Children Treated With Growth Hormone : Evaluation at Adult Size

NCT05195437 | Completed

• 2 other identifiers: APHP2115232024-A01272-45
• Study Type: observational
• Target: 36
• Locations: 1 country
• Sites: 1

Brief Summary

Short stature can lead to emotional and social stress in children and adolescents, as well as their parents. Children and their parents want to be able to identify the cause of stunted growth and address it with treatment. Mitigating the impact of short stature on quality of life is one of the main goals of treatment. The quality of life in children can be measured using adapted self-questionnaires. The investigative team published in 2019 the results of a preliminary study which shows that after one year of treatment with growth hormone, the quality of life improves in children, in particular on the scales emotional and social. These evaluations were carried out in particular thanks to the general questionnaire of quality of life: Pediatric Quality of Life Inventory (PedsQL) 4.0, but also via a specific questionnaire of the size: Quality of Life of Short Stature Youth questionnaire (QoLiSSY). 50 of the 74 patients who participated in this study have now reached their final height. The objective of the present study is to reassess this cohort using the QoLiSSY and PedsQL 4.0 questionnaires. The patient will be his own witness.

Conditions

Growth Hormone Treatment

Keywords

Growth hormone treatment; Quality of Life of Short Stature Youth (QoLiSSY) questionnaire; Pediatric Quality of Life Inventory (PedsQL) 4.0; Quality of life improvement

Outcome Measures

Primary Outcomes (1)

• Evolution of the quality of life

  Evolution of the quality of life in short patients who have been treated with growth hormone. Comparison of the scores of the initial Quality of Life in Short Stature Youth (QoLiSSY) questionnaire (start of growth hormone treatment) compared to the scores obtained when the patients reached their final height. QoLISSY questionnaires include two different forms: parent questionnaire (children aged 4 to 18 years of age) and child questionnaire (only for children ≥8 years of age). Both questionnaires have 50 items that cover three core domains (physical, emotional, and social), and additional complementary domains. The parent questionnaire includes two additional scales (16 items) assessing parental stress and anxiety. Scores are converted in SD scores and compared to age- and gender-specific data from a reference population of children and adolescents with short stature. A total score is computed as the mean score on the three core scales.

  Day 0

Secondary Outcomes (2)

• Adult-size quality of life

  Day 0

• Correlation between quality of life changes and clinical response to growth hormone therapy

  Day 0

Study Arms (1)

Growth hormone treatment

Adolescents and young adults followed or having been followed by the endocrinology, gynecology and pediatric diabetology department of the Necker Enfants Malades hospital, who reached their final height and who have been treated with growth hormone due to short stature.

Other: QoLiSSY and PedsQL 4.0 questionnaires

Interventions

QoLiSSY and PedsQL 4.0 questionnaires OTHER

Patients have been reaching adult size complete the two questionnaires of the study : Quality of Life of Short Stature Youth questionnaire (QoLiSSY), assessing health-related quality of life in short stature youth. Pediatric Quality of Life Inventory (PedsQL) 4.0 : general health-related quality of life. The patients have previously completed the PedsQL 4.0 and QoLiSSY questionnaires when starting their growth hormone therapy and after one year of growth hormone therapy.

Growth hormone treatment

Eligibility Criteria

• Age: 15 Years+
• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Adolescents and young adults followed or having been followed by the endocrinology, gynecology and pediatric diabetology department of the Necker Enfants Malades hospital, who reached their final height, who have been treated with growth hormone due to short stature and who had participed in the initial study : PedsQL 4.0 and QoLiSSY questionnaires completed at starting growth hormone therapy and after one year of growth hormone therapy.

You may qualify if:

• Adolescents and young adults followed or having been followed by the endocrinology, gynecology and pediatric diabetology department of the Necker Enfants Malades hospital and treated with growth hormone because of their short stature.
• Patient who participated in the initial study.
• Patient having reached the final height during the last consultation (growth rate less than 1 cm / year and / or bone age ≥ 15 years for girls, ≥ 17 years for boys).
• Holders of parental authority, minor patients and adult patients informed and not opposed to their participation in the study.

You may not qualify if:

• \- Refusal to participate in the study.

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead
• URC-CIC Paris Descartes Necker Cochin: collaborator

Study Sites (1)

Hôpital Necker-Enfants Malades

Paris, 75015, France

Location

Study Officials

• Michel Polak, MD, PhD

  Assistance Publique - Hôpitaux de Paris

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 4, 2022
• First Posted: January 19, 2022
• Study Start: March 8, 2022
• Primary Completion: October 12, 2024
• Study Completion: October 12, 2024
• Last Updated: September 12, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1)