Clinical trial NCT05195437: Evolution of the Quality of Life of Short Statured Children Treated with Growth Hormone : Evaluation At Adult Size
Official title: Evolution of the Quality of Life of Short Stature Children Treated with Growth Hormone : Evaluation At Adult Size
Study type: OBSERVATIONAL
Overall status: COMPLETED
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
Lead sponsor: Assistance Publique - Hôpitaux de Paris
Sponsor class: OTHER
First posted: 2022-01-19
Enrollment: 36
Sex: ALL
Minimum age (years): 15
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
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.

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.

Detailed description: 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.

Eligibility criteria: Inclusion Criteria:

* 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.

Exclusion Criteria:

- Refusal to participate in the study.

Primary outcomes:
measure: Evolution of the quality of life
timeFrame: Day 0
Secondary outcomes:
measure: Adult-size quality of life
timeFrame: Day 0
measure: Correlation between quality of life changes and clinical response to growth hormone therapy
timeFrame: Day 0
Other outcomes:
Locations:
city: Paris
country: France
facility: Hôpital Necker-Enfants Malades
Study officials:
affiliation: Assistance Publique - Hôpitaux de Paris
name: Michel Polak, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: