Growth Hormone Deficiency (GHD)
Conditions
Keywords
Growth Hormone Disorder, Digital health, pediatric endocrinology, Personalized Growth Hormone Treatment Profile
Brief summary
The goal of this observational study is to learn how to make growth hormone treatment more personalized for children. The main questions it aims to answer are: * How can information about treatment adherence and patient experiences help improve care? * What insights can help doctors create treatment plans that are more tailored to each child? The study will include 40 families of children who are using a connected injector device for their growth hormone treatment. These devices track how often the treatment is taken, and families will also share their experiences through surveys. The information will be used to develop a new tool, similar to tools used in diabetes care, to help doctors create better, personalized treatment plans for children with growth hormone needs.
Interventions
COMBINATION_PRODUCTACDP® and growth hormone treatment
A cohort of 40 families of children undergoing growth hormone treatment using a connected injector devices that quantifies treatment adherence will be recruited via the ACDP®, to capture additional data that combine Patient Reported Outcomes and Adherence Data.
Sponsors
Hospital Miguel Servet
Luis Fernandez Luque
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
Yes
Inclusion criteria
* Pediatric patients (up to 18 years old) diagnosed with growth hormone deficiency or disorders requiring growth hormone treatment. * Patients are currently undergoing growth hormone therapy. Includes both naive patients (those newly starting weekly treatment) and those already on treatment. * Families willing to use connected injector devices and participate in mobile-based psychometrics for the study duration. * Families are able and willing to regularly provide patient-reported outcomes (PROs) and adherence data through the Adhera®️ Caring Digital Program. * Participants willing to sign the informed consent form, confirming their understanding of the essential aspects of the study, including the potential risks, benefits and rights as participants.
Exclusion criteria
* Families/patients already enrolled in other study protocols, including the use of connected injector devices and participation in mobile-based psychometrics. * Patients with other significant medical conditions that could interfere with growth hormone treatment or the study's data collection processes. * Inability to understand or communicate in the language used for data collection and study participation, unless appropriate translation services are available and can be consistently utilized.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Develop a Personalized Growth Hormone Treatment Profile (PGTP) | From enrollment to the end of the study at Month 12 | To develop a Personalized Growth Hormone Treatment Profile (PGTP) standard - analogous to the Ambulatory Glucose Profile (AGP) for diabetes care-, integrating diverse data sources to support personalized treatment strategies in pediatric endocrinology. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Physiological Monitoring via Wearable Devices - Sleep Quality | From enrollment to the end of the study at Month 12 | Unit of Measure: \- Total sleep duration (hours per night). Description: Sleep parameters will be tracked using wearable devices. |
| Physiological Monitoring via Wearable Devices - Heart Rate | From enrollment to the end of the study at Month 12 | Unit of Measure: Average daily heart rate (beats per minute, bpm). Description: Daily average heart rate recorded through wearable devices. |
| Physiological Monitoring via Wearable Devices - Physical Activity Metrics | From enrollment to the end of the study at Month 12 | Unit of Measure: * Total calories burned per day (kilocalories, kcal). * Total steps per day (steps). * Total activity time per day (minutes). Description: Data will be collected from wearable devices to assess daily physical activity. |
| Physiological Monitoring via Wearable Devices - Blood Oxygen Levels | From enrollment to the end of the study at Month 12 | Unit of Measure: Daily oxygen saturation percentage (%). Description: Measured through wearable device sensors. |
| Evaluating Usability of Connected Devices | From enrollment to the end of the study at Month 12 | Outcome Measure: System Usability Scale (SUS) score. Unit of Measure: SUS score (0-100, higher scores indicate better usability). Description: The System Usability Scale (Bangor et al., 2008) is a 10-item scale based on a 5-point Likert scale assessing perceived usability. |
| Adherence to Treatment Using Connected Injector Devices | From enrollment to the end of the study at Month 12 | Automated tracking of injection times and adherence. Adherence will be quantified through connected injector device data, capturing timestamps of each injection and calculating adherence rates. |
| Distress Assessment 2 | From enrollment to the end of the study at Month 12 | Distress Thermometer Unit of Measure: Score on a 0-10 scale (higher scores indicate higher distress). Description: The Distress Thermometer is a single-item, self-report tool measuring psychological distress on a 0-10 scale. |
| Self-Efficacy Assessment | From enrollment to the end of the study at Month 12 | General Self-Efficacy Scale (GSE) Unit of Measure: Score range 10-40 (higher scores indicate greater self-efficacy). Description: The GSE scale measures an individual's belief in their ability to cope with adversity. |
| Mood Assessment | From enrollment to the end of the study at Month 12 | Positive and Negative Affect Schedule (PANAS) Unit of Measure: * Positive Affect (PA) score range: 10-50 (higher scores indicate greater positive affect). * Negative Affect (NA) score range: 10-50 (higher scores indicate greater negative affect). Description: PANAS is a self-report questionnaire measuring two dimensions of mood: positive and negative affect. |
| Mental Health and Well-Being Assessment | From enrollment to the end of the study at Month 12 | Mental Health Continuum-Short Form (MHC-SF) Unit of Measure: Score range 0-70 (higher scores indicate better mental health). Description: The MHC-SF is a 14-item scale assessing emotional, social, and psychological well-being as indicators of positive mental health. |
| Health-Related Quality of Life (HRQoL) | From enrollment to the end of the study at Month 12 | KIDSCREEN-10 Unit of Measure: Score range 10-50 (higher scores indicate better health-related quality of life). Description: The KIDSCREEN-10 is a health-related quality of life measure for children and adolescents, assessing physical, psychological, and social well-being, autonomy, school environment, and peer and family support. |
| Mobile Application Engagement | From enrollment to the end of the study at Month 12 | Outcome Measure: User engagement with the mobile application. Unit of Measure: Frequency of interactions (e.g., number of logins per day/week). Description: Engagement will be assessed using mobile user logs, including user profiling metrics based on Hors-Fraile et al. (2018). |
Countries
Spain
Outcome results
None listed