Education & Care in RARE: Efficacy of Targeted Psychoeducational Intervention Among Pediatric Rare Disease Patients

Condition(s) studied

Investigational drug(s) / intervention(s)

Education & Care in RARE: Education \& 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.

Study summary

"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.

Eligibility

Primary outcome measure(s)

• Rare Disease Specific Self-Rating Scale — IG: T0 (at inclusion), T1 (after intervention), T2 (3 to 6 months after T1); WLG: T0 (at inclusion), T1 (2-3 months after T0), T2 (after intervention), T3 (3 to 6 months after T2)
  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;

Trial sites (7)

More Medical University of Vienna trials in Austria