Achondroplasia, Growth Hormone Deficiency, Small for Gestational Age
Conditions
Keywords
Health related quality of life
Brief summary
The aim of the study is the adaption, implementation and validation of the instrument for the investigation of the short stature specific quality of life (QoLISSY) for children (age 0-4 years) with achondroplasia (ACH), Small for Gestational Age (SGA) and Growth Hormone Deficiency (GHD) from a parental perspective.
Detailed description
The questionnaire is intended to be particularly applicable in clinical trials, with a focus on the impact of short stature and the physical functionality of those affected. The instrument will be further developed according to the Guidelines for PRO instrument development. It should be practical in its application and interpretation and applicable in the international context. In addition, it will be investigated whether, from the parents' point of view, other instruments are suitable for measuring the quality of life of their short statured children. Their preference regarding the instruments will be assessed so that a recommendation can be made as to which instruments are particularly suitable. A systematic literature review will be conducted to identify suitable instruments to measure the quality of life of short statured children aged 0 to 4. Approximately 10-20 parents of 0-4 year old children with ACH, GHD and SGA will be recruited for the qualitative data collection (n = 30 to 60). The patients will be recruited through the BKMF (German Association for People of Short Stature and their Families). In order to test the psychometric properties of the instruments, approximately 30 parents (15 mothers / 15 fathers) of 0-4 year old children with ACH, GHD and SGA will be recruited for the quantitative part of the study (n = 90). All participants will be asked to complete the QoLISSY or other instruments found suitable in the pilot test. For the purpose of cross-validation, the PEDSQL will be used in parallel. Two weeks after receiving the field test, the re-test will be sent to ensure the reliability of the study. The re-test will be administered to a minimum of 20% of the participants.
Interventions
OTHERQuestionnaire
The original QOLISSY questionnaire was first developed in 2011, it is available as a patient reported and a parent reported instrument. It includes: * QoLISSY questionnaire for children and adolescents (8-12, 13-18 yrs) * QoLISSY questionnaire for parents of children with short stature (4-7, 8-12 and 13-18 yrs) The QoLISSY instrument can be used to assess the health-related quality of life in short stature youth regardless of its cause; however it was specifically developed for patients with GHD and ISS and their parents.The aim of the current study is the adaption, implementation and validation of the instrument for the investigation of the short stature specific quality of life (QoLISSY) for children (age 0-4 years) with achondroplasia (ACH), Small for Gestational Age (SGA) and Growth Hormone Deficiency (GHD) from a parental perspective.
Sponsors
Universitätsklinikum Hamburg-Eppendorf
Bundesverband Kleinwüchsige Menschen und ihre Familien e.V.
Pfizer
Study design
Observational model
OTHER
Time perspective
CROSS_SECTIONAL
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Parents of children aged 0-4 with the diagnosis Achondroplaisa, Short for Gestational Age or Growth Hormone Defiency
Exclusion criteria
* Other diagnosis than Achondroplaisa, Short for Gestational Age or Growth Hormone Defiency * Children older than 4 years of age
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Health related quality of life measured by the adapted version of the QoLISSY questionnaire | June 2022 - March 2023 | The studies aim is to identify factors influencing the health related quality of life of children with Achondroplaise, Growth Hormone Deficiency and Small for Gestational Age to adapt and test the QOLISSY-Questionnaire for children from the age of 0-4. |
Countries
Germany
Contacts
Primary ContactJulia Quitmann, Dipl. Psych.
Outcome results
None listed