"Rare Diseases" is an umbrella term including more than 8.000 different diseases which individually affect only a small percentage of people. Rare diseases predominantly affect children and adolescents and are associated with high medical and psychosocial burden of disease.
The investigators invented Education \& Care in RARE - a short-term, structured, resource-oriented and child-friendly psychoeducation program for children and adolescents with rare diseases.
This study is a prospective, multicenter, randomized and controlled study with a waiting list. Aim of the study is to investigate the efficacy of Education \& Care in RARE on knowledge about rare diseases and on mental health well-being in pediatric rare disease patients, compared to a control group.
In this study participants are randomized in an intervention group and a waiting list control group. Both study groups thus receive the psychoeducation with Education \& Care in RARE and complete the identical questionnaires. Compared to the Intervention group, the waiting list control group receives the intervention with a time delay (8-12 weeks later) and has one additional appointment for questionnaire evaluation before start of the psychoeducation.
Children and adolescents with a confirmed diagnosis of a rare disease with
Age 5-20 years, corresponding to a developmental age of 5-18 years
Existing medical care at a participating study center because of the rare disease
Voluntary participation and informed consent
Moderate or severe cognitive impairment
Simultaneous admission of the child / adolescent to a setting with high-frequency psychotherapeutic intervention (e.g. admission to psychosomatic medicine, child and adolescent psychiatry)
No informed consent
Language barrier of the child / adolescent
Parallel
Education & Care in RARE
Other interventionEducation \& Care in RARE (https://www.youtube.com/watch?v=R3fr-q-6JIw) is a short-term, structured, resource-oriented and child-friendly psychoeducation program for children and adolescents with rare diseases. It promotes knowledge and competence on rare diseases in children in order to reduce the psychosocial rare disease burden and to improve individual self-competence in managing the rare disease and to improve their quality of life. Education \& Care in RARE can be used for all pediatric rare diseases. This has the great advantage that users only need to be trained in the use of one program.
Rare Disease Specific Self-Rating Scale
Description: The Rare Disease Specific Self-Rating Scale is defined as primary outcome measurement of this study. The Rare Disease Specific Self-Rating Scale provides 10 items to assess rare disease specific knowledge and well-being in children and adolescents and uses a Six-Point Smiley Faces Likert Scale as a global subjective outcome scale for children assessment. For the primary hypothesis, a mean value of the 10 items is computed for each participant. The Rare Disease Specific Self-Rating Scale covers specific topics on Rare Diseases which are not covered by the generic research instruments of this study. Outcome Measurements: Knowledge about rare diseases; Well-being Items; Scale level: 10 items; 6-Point Smiley Faces Likert Scale; sum total score 10-60;
Rare Diseases Specific Expert-Rating Scale
Description: The Rare Disease Specific Expert-Rating Scale provides 12 items on participants' knowledge about rare diseases and on competences to cope with rare disease specific challenges and uses a Six-Point Smiley Faces Likert Scale as a global subjective outcome scale for expert assessment. For the analyses, a mean value of the 12 items is computed for each participant Outcome measurements: Knowledge about rare diseases, Competences to cope with rare disease specific challenges Items; Scales: 12 items; 6-Point Smiley Faces Likert Scale; sum total score 12-72
SDQ Strengths and Difficulties Questionnaire self-report
Description: The SDQ Strengths and Difficulties Questionnaire (https://www.sdqinfo.org/a0.html) is a brief behavioral screening questionnaire in children and adolescents and provide 25 items on psychological attributes and includes a self-report version and a third-party survey version. There are two versions of the SDQ for two different age and development stages. A self-report version and a third-party survey version are offered for each age version of the SDQ. Norm values for different nationalities including German is available. Outcome Measurements: Mental health well-being - self-report Items; Scale level: 25 items; 3-Point Likert Scale; sum total score 25-75
SDQ Strengths and Difficulties Questionnaire, third- party-report
Description: The SDQ Strengths and Difficulties Questionnaire (https://www.sdqinfo.org/a0.html) is a brief behavioral screening questionnaire in children and adolescents and provide 25 items on psychological attributes and includes a self-report version and a third-party survey version. There are two versions of the SDQ for two different age and development stages. A self-report version and a third-party survey version are offered for each age version of the SDQ. Norm values for different nationalities including German is available. Outcome Measurements: Mental health well-being - third-party-report Items; Scale level: 25 items; 3-Point Likert Scale; sum total score 25-75
KINDL self-report
Description: The KINDL (https://www.kindl.org/english/) is a standardized questionnaire for the assessment of quality of life in children and adolescents and provides 24 items. The KINDL thus is a short, methodologically suitable, psychometrically sound and flexible measure of Health-Related Quality of Life in children and adolescents. There are three versions of the KINDL for different age and development stages. A self-report version and a third-party survey version are offered for each age version of the KINDL. Norm values are given based on representative German data from the German National Health Interview and Examination Survey for Children and Adolescents (KiGGS). The KiGGS study is a broad survey realized by the German-Robert-Koch-Institute. Outcome Measurements: Quality of life - self-report Items; Scale level: 24 items; 3-Point Likert Scale; sum total score 24-72
Documentation of the program
Documentation of the program provides number of sessions and levels of the Education \& Care in RARE program in each participant. Items; Scales: 2 items, descriptive
Medical Data
Medical Data asses medical data of study participants including Rare disease diagnosis Secondary conditions Developmental age Visibility of a rare disease Type of visibility of a rare disease Type of diagnostic confirmation Life-threatening rare disease Age at rare disease diagnosis Time of diagnostic odyssey Existence of an emergency card or a rare disease card Items; Scales: 10 items, descriptive
Demographic Data
Participants sociodemographic data including Patient's age, sex, school form, language skills, mother language, multilingualism; Parents' education and language skills; Items; Scales: 8 items, descriptive
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