Short Stature and Psychological Well-being

NCT06295341 | Completed

• 1 other identifier: 01C312
• Study Type: observational
• Target: 80
• Locations: 1 country
• Sites: 1

Brief Summary

The first aim of the present study is to evaluate the psychological impact of the condition of short stature (family) in a sample of Italian children, comparing them with subjects of normal stature, measuring their levels of psychological well-being, psychological distress, quality of health-related life and any behavioral issues. The secondary objective is to study the psychological impact evaluated with the tests described below (see methods section) in children with GH deficiency and the effects of replacement therapy (6 months) with GH from recombinant DNA.

Conditions

Short Stature; Short; Stature, Psychosocial

Keywords

Short stature; Psychological well-being; Psychological distress; Life quality

Outcome Measures

Primary Outcomes (4)

• Psychological well-being through the Psychological Well-Being Scales (PWB)

  A self-administered questionnaire consisting of 18 items that investigates the degree of psychological well-being by exploring six dimensions: self-acceptance, positive relationships with others, autonomy, environmental control, personal growth, and purpose of life. The questionnaire consists of 18 items on a 4-step Likert scale.

  Baseline for the three groups; baseline and after 6 months for children with growth hormone deficiency

• Psychological distress through the Depression Anxiety Stress Scale (DASS-21)

  A 21-item self-report tool that measures various negative internal states: depression, anxiety, and stress.

  Baseline for the three groups; baseline and after 6 months for children with growth hormone deficiency

• Quality of life through the Quality of Life in Short Stature Youth (QoLISSY),

  A 50-item domain-specific questionnaire that measures health-related quality of life in children with short stature.

  Baseline for the three groups; baseline and after 6 months for children with growth hormone deficiency

• Skills and problems through the Strengths and Difficulties Questionnaire (SDQ)

  A questionnaire made up of 25 items able to assess emotional symptoms, behavioral problems, hyperactivity/inattention, problematic relationships with peers, and prosocial behavior.

  Baseline for the three groups; baseline and after 6 months for children with growth hormone deficiency

Secondary Outcomes (1)

• Behavioral problems through the Child Behaviour Checklist for Children (CBCL)

  Baseline for the three groups; baseline and after 6 months for the parents of children with growth hormone deficiency

Study Arms (3)

cases - children with familial short stature

\- 35 children, aged between 6 and 14 years, of both sexes, with familial short stature (height \< 3rd centile according to the Italian reference standards) and their caregivers of reference. Children with familial short stature will be characterized by: short stature in other members of the family group, not necessarily the parents, harmonious appearance, without particular clinical signs and normal pubertal development, parallel growth curve below the 3rd centile, bone age corresponding to chronological age. Children and teenagers with obesity (BMI \> 97th centile) will be excluded

cases - children with normal height

35 children, aged between 6 and 14 years, of both sexes, with normal height (height \> 25th centile) and weight.

cases - children with growth hormone deficiency

10 children, aged between 6 and 14 years, of both sexes, affected by isolated GH deficiency according to the criteria established by AIFA note 39 for this pathology (short stature: ≤ -3 SD or ≤ -2 SD and growth velocity/year ≤ -1.0 SD for age and sex evaluated at least 6 months apart and peak GH at two different pharmacological stimulus tests \< 8 ng/ml). The exclusion criterion from the present study (and from treatment with rhGH) will be the presence of organic pathologies at the hypothalamic-pituitary level (assessed by performing brain MRI). Children with GH deficiency are evaluated in baseline conditions and after 6 months of therapy with recombinant DNA GH (at a dose of 0.025-0.035 mg/kg of body weight per day (or 0.7-1.0 mg/m2 of body surface area per day).

Drug: Recombinant human growth hormone (only for children with growth hormone deficiency)

Interventions

Recombinant human growth hormone (only for children with growth hormone deficiency) DRUG

6 months of therapy with recombinant DNA GH (at a dose of 0.025-0.035 mg/kg of body weight per day (or 0.7-1.0 mg/m2 of body surface area per day).

cases - children with growth hormone deficiency

Eligibility Criteria

• Age: 6 Years - 14 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17)
• Sampling Method: Probability Sample

Study Population

35 children with familiar short stature, 35 with normal height (height \> 25th centile), and 10 with growth hormone deficiency and their caregivers of reference (see above the description of the diagnostic criteria).

You may qualify if:

• with familiar short stature (height \< 3rd centile according to the Italian reference standards) and their caregivers of reference. Children with familial short stature are characterized by: short stature in other members of the family group, not necessarily the parents, harmonious appearance, without particular clinical signs and normal pubertal development, parallel growth curve below the 3rd centile, bone age corresponding to chronological age.
• with normal height (height \> 25th centile), aged between 6 and 14 years, of normal weight, and their caregivers of reference
• children, aged between 6 and 14 years, of both sexes, affected by isolated GH deficiency according to the criteria established by AIFA note 39 for this pathology (short stature: ≤ -3 SD or ≤ -2 SD and growth velocity/year ≤ -1.0 SD for age and sex evaluated at least 6 months apart and peak GH at two different pharmacological stimulus tests \< 8 ng/ml) and their caregivers of reference

You may not qualify if:

• children and teenagers with obesity (BMI \> 97th centile) are excluded (for the subgroups with familiar short stature or normal height
• children and teenagers with the presence of organic pathologies at the hypothalamic-pituitary level (assessed by performing brain MRI) (for the subgroup with growth hormone deficiency)

Sponsors & Collaborators

• Istituto Auxologico Italiano: lead

Study Sites (1)

Istituto Auxologico Italiano

Milan, 20145, Italy

Location

MeSH Terms

Conditions

Dwarfism; Psychological Well-Being

Interventions

Growth Hormone; Single Person

Condition Hierarchy (Ancestors)

Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Endocrine System Diseases; Personal Satisfaction; Behavior

Intervention Hierarchy (Ancestors)

Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins; Marital Status; Family Characteristics; Demography; Population Characteristics; Socioeconomic Factors

Study Design

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

Study Record Dates

• First Submitted: February 28, 2024
• First Posted: March 6, 2024
• Study Start: May 10, 2023
• Primary Completion: January 31, 2024
• Study Completion: January 31, 2024
• Last Updated: April 30, 2025

Record last verified: 2025-04

Locations

IT (1)