Children Affected by Rare Disease and Their Families Network

Study Overview
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Brief Summary
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Families of children with rare diseases (i.e., not more than 5 out of 10.000 people are affected) are often highly burdened with fears, insecurities and concerns regarding the affected child and his/her siblings. The project at hand will test two innovative forms of care (CARE-FAM and WEP-CARE) at 17 sites in 12 federal states of Germany. The goal is to improve the mental health and quality of life of children affected by rare diseases and their relatives in a sustainable manner. If successful, these interventions will be introduced into regular care.

Detailed Description
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The central objective of the study at hand is to close the supply gap for families with children and adolescents affected by rare diseases. Two innovative forms of care (CARE-FAM and WEP-CARE) will be implemented and evaluated at the 18 participating study sites. Both interventions include psychological diagnostics, early detection and treatment of concomitant mental diseases. The study is a prospective, randomized controlled multicenter study (RCT) with a factorial design with four groups: CAREFAM (face to face), WEP-CARE (online), both interventions, control group (TAU = treatment as usual). Central psychosocial outcomes will be assessed at four time points (i.e., Baseline and after six, 12 and 18 months) from the perspectives of the parents, the affected child and the siblings (0 - 9 years only external assessment; from 10 years of age additional self-assessment) and the professionals.

Official Title
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Children Affected by Rare Disease and Their Families Network

Conditions
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Rare Diseases

Intervention / Treatment
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* Behavioral: CARE-FAM
* Behavioral: WEP-CARE


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Family with at least one child between 0 and 21 years with a rare disease or a suspected rare disease. Consent to participate in the study. Sufficient knowledge of the German language of parents and children. Insured at the participating insurance companies. Exclusion Criteria: Severe psychiatric disorders and impairments with acute symptoms such as suicidal tendencies, severe depression, addictions, acute psychotic symptoms etc., which will not be sufficiently supplied by this new low-frequency intervention. Children and parents with acute treatment demand in the control group will be placed at psychotherapists. Nevertheless, they stay in the control group.

Ages Eligible for Study
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Minimum Age: 1 Day
Maximum Age: 21 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Supportive Care
Allocation: Randomized
Intervention Model: Factorial Assignment
Interventional Model Description: The study is a prospective, randomized controlled multicenter study (RCT) with a factorial design with four groups: CAREFAM (face to face), WEP-CARE (online), both interventions, control group (TAU = treatment as usual).
Masking: Single


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: CARE-FAM<br>The face-to-face intervention CARE-FAM is a family-based intervention for the diagnostic, early detection and early treatment of mental health issues of children affected by rare diseases, their siblings and their parents. CARE-FAM is a brief low-frequency intervention comprising six to eight sessions per family over a period of six months. Following a preliminary talk, 2 sessions with the parents, 1 session with each affected child and each sibling and 3 sessions with the whole family will take place. This low-frequency approach (sessions every 2 to 3 weeks) allows families to integrate the intervention into their daily life. Upon request, the sessions will take place at the family's home (home-treatment). | Behavioral: CARE-FAM<br>* CARE-FAM is a family-based intervention for the diagnostic, early detection and early treatment of mental health issues of children affected by rare diseases, their siblings and their parents. CARE-FAM is a brief low-frequency intervention comprising six to eight sessions per family over a period of six months.<br>|
| Experimental: WEP-CARE<br>The online intervention WEP-CARE addresses parents of children and adolescents affected by rare diseases. The program is based on principles of cognitive-behavioral writing therapy. Supported by trained professionals, the participants perform 12 standardized writing tasks on a secured internet platform. The 12 writing tasks will be conducted with a weekly frequency and participants will receive personalized feedback. WEP-CARE aims at enhancing mental health problems and the coping strategies of the family. | Behavioral: WEP-CARE<br>* WEP-CARE is an online-intervention that addresses parents of children and adolescents affected by rare diseases. The program is based on principles of cognitive-behavioral writing therapy. Supported by trained professionals, the participants perform 12 standardized writing tasks on a secured internet platform<br>|
| Experimental: CARE-FAM + WEP-CARE<br>The families will receive both the face-to-face intervention CARE-FAM and the online intervention WEP-CARE. | Behavioral: CARE-FAM<br>* CARE-FAM is a family-based intervention for the diagnostic, early detection and early treatment of mental health issues of children affected by rare diseases, their siblings and their parents. CARE-FAM is a brief low-frequency intervention comprising six to eight sessions per family over a period of six months.<br>Behavioral: WEP-CARE<br>* WEP-CARE is an online-intervention that addresses parents of children and adolescents affected by rare diseases. The program is based on principles of cognitive-behavioral writing therapy. Supported by trained professionals, the participants perform 12 standardized writing tasks on a secured internet platform<br>|
| No Intervention: Treatment as usual<br>The treatment as usual implies that families of the control group receive the treatment that is customary in regular care. Thus, these families normally don't receive any post-treatment. If, however, a member of a control group family appears to have an urgent need for treatment (every family receives a comprehensive diagnostic investigation at the beginning of the study), the respective family will be placed in the ambulatory care system. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Mental health of parents (SCID) | Proportion of parents without mental abnormities among the parents with initial mental abnormities, assessed by the external, independent Structured clinical interview for DSM-IV (SCID; Wittchen, Zaudig & Fydrich,1997) 18 months after. | Change from baseline of the study at 6,12 and 18 months after the randomization | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Sociodemographic information of the parents | Sociodemographic information of the parents, assessed from the perspective of the parents by ad-hoc items at the beginning of the study. | At baseline of the study | 
| Health-related quality of life of the parents (EQ-5D) | Health-related quality of life of the parents, assessed from the perspective of the parents by the EQ-5D (Brooks, Rabin & Charro, 2003; Hinz, Klaiberg, Brahler & Konig, 2006) at the beginning of the study as well as six, 12 and 18 months after the randomization. | Change from baseline of the study at 6,12 and 18 months after the randomization | 
| Health-related quality of life of the parents (ULQIE) | Health-related quality of life of the parents, assessed from the perspective of the parents by the Ulmer Lebensqualitäts-inventar für Eltern chronisch kranker Kinder (ULQIE; Goldbeck & Storck, 2002) at the beginning of the study as well as six, 12 and 18 months after the randomization. | Change from baseline of the study at 6,12 and 18 months after the randomization | 
| Health-related quality of life of the parents (SF-12) | Health-related quality of life of the parents, assessed from the perspective of the parents by the Short Form 12 (SF-12; Bullinger & Kirchberger, 1998) at the beginning of the study as well as six, 12 and 18 months after the randomization. | Change from baseline of the study at 6,12 and 18 months after the randomization | 
| Health-related quality of life of the chronically-ill children/adolescents (Kidscreen-27) | Health-related quality of life of the chronically-ill children/adolescents and of the siblings, assessed from the perspective of the child/adolescent (from 10 years of age) and from the perspective of the parents by the Kidscreen-27 (The KIDSCREEN Group Europe, 2006). | Change from baseline of the study at 6,12 and 18 months after the randomization | 
| Health-related quality of life of the chronically-ill children/adolescents (DCGM-37) | Health-related quality of life of the chronically-ill children/adolescents, assessed from the perspective of the child/adolescent (from 10 years of age) and from the perspective of the parents by the Disabkids Chronic Generic Measure (DCGM-37; Bullinger, Schmidt, Petersen & The DISABKIDS Group, 2002) at the beginning of the study as well as six, 12 and 18 months after the randomization. | Change from baseline of the study at 6,12 and 18 months after the randomization | 
| Mental health of the parents (PHQ) | Mental health of the parents, assessed from the perspective of the parents by the Patient Health Questionnaire (PHQ; Löwe, Spitzer, Zipfel & Herzog, 2002). | Change from baseline of the study at 6,12 and 18 months after the randomization | 
| Mental health of the parents (BSI) | Mental health of the parents, assessed from the perspective of the parents by the Brief Symptom Inventory (BSI; Franke, 2000). | Change from baseline of the study at 6,12 and 18 months after the randomization | 
| Mental health of the chronically-ill children/adolescents and the siblings (Kiddie-SADS-PL) | Mental health of the chronically-ill children/adolescents and the siblings, assessed from the perspective of the parents and from the perspective of the children/adolescents (from 10 years of age) by an external independent interview Diagnostic Interview Kiddie-Sads-Present and Lifetime Version (Kiddie-SADS-PL; Delmo, Weiffenbach, Gabriel, Stadler & Poustka, 2001). | Change from baseline of the study at 6,12 and 18 months after the randomization | 
| Psychiatric disorders of the chronically-ill children/adolescents and the siblings (CBCL) | Psychiatric disorders of the chronically-ill children/adolescents and the siblings, assessed from the perspective of the parents by the Child Behaviour Checklist (CBCL; Döpfner, Pflück, Kinnen & Arbeitsgruppe Deutsche Child Behavior Checklist, 2014). | Change from baseline of the study at 6,12 and 18 months after the randomization | 
| Psychiatric disorders of the chronically-ill children/adolescents and the siblings (YSR) | Psychiatric disorders of the chronically-ill children/adolescents and the siblings, assessed from the perspective of the children/adolescents (from 10 years of age) by the Youth Self Report (YSR; Döpfner, Pflück, Kinnen & Arbeitsgruppe Deutsche Child Behavior Checklist, 2014). | Change from baseline of the study at 6,12 and 18 months after the randomization | 
| Coping of the parents (CHIP-D) | Coping of the parents, assessed from the perspective of the parents by the German version of the Coping Health Inventory for Parents (CHIP-D; McCubbin, McCubbin, Cauble & Goldbeck, 2001). | Change from baseline of the study at 6,12 and 18 months after the randomization | 
| Coping of the chronically-ill children/adolescents and the siblings (Kidcope) | Coping of the chronically-ill children/adolescents and the siblings, assessed from the perspective of the children/adolescents (from 10 years of age) by the Kidcope Checklist (Kidcope; Spirito, Stark & Williams, 1988). | Change from baseline of the study at 6,12 and 18 months after the randomization | 
| Social support of the parents, of the chronically-ill children/adolescents and of the siblings (OSSQ) | Social support of the parents, of the chronically-ill children/adolescents and of the siblings, assessed from the perspective of the parents, of the chronically-ill children/adolescents and from the sibling, respectively, by the Oslo Social Support Questionnaire (OSSQ; Dalgard, 2006). | Change from baseline of the study at 6,12 and 18 months after the randomization | 
| Family functioning (GARF) | Family functioning, assessed from the perspective of the therapist by the Global Assessment of Relational Functioning (GARF; Saß, Wittchen, Zaudig & Houben, 2003). | Change from baseline of the study at 6,12 and 18 months after the randomization | 
| Relationships between siblings (SRQ) | Sibling relationship, assessed from the perspective of the siblings (from 10 years of age) by the Sibling Relationship Questionnaire (SRQ; Fuhrmann & Burmester, 1985). | Change from baseline of the study at 6,12 and 18 months after the randomization | 
| Satisfaction with the relationship and parenting relationship of the parents (PFB) | Satisfaction with the relationship and parenting relationship of the parents, assessed from the perspective of the parents by the Partnerschaftsfragebogen (PFB; Hahlweg, 2016). | Change from baseline of the study at 6,12 and 18 months after the randomization | 
| Eating behaviour of the chronically-ill children/adolescents (EDY-Q) | Eating behaviour of the chronically-ill children/adolescents, assessed from the perspective of the parents and from the perspective of the chronically-ill children/adolescents (from 10 years of age) by the Eating Disorders in Youth - Questionnaire (EDY-Q; van Dyck & Hilbert, 2016). | Change from baseline of the study at 6,12 and 18 months after the randomization | 
| Body-related eating behaviour of the chronically-ill children/adolescents (ChEDE-Q8) | Body-related eating behaviour of the chronically-ill children/adolescents, assesse from the persepective of the chronically-ill children/adolescents (from 10 years of age) by the Eating Disorder Examination - Questionnaire (Short Form) (ChEDE-Q8; Kliem, Schmidt, Vogel, Hiemisch, Kiess & Hilbert, 2017). | Change from baseline of the study at 6,12 and 18 months after the randomization | 
| Elimination disorders of the chronically-ill children/adolescents (Anamnesebogen Enuresis/Funktionelle Harninkontinenz) | Elimination disorders of the chronically-ill children/adolescents, assessed from the perspective of the parents by the Anamnesebogen Enuresis/Funktionelle Harninkontinenz (von Gontard, 2010). | Change from baseline of the study at 6,12 and 18 months after the randomization | 
| Treatment costs of the parents (CSSRI-DE) | Treatment costs of the parents, assessed from the perspective of an external rater by the German version of the Client Socioeconomic and Services Receipt Inventory (CSSRI-DE; Roick, Kilian, Matschinger, Bernert, Mory & Angermeyer, 2001). | Change from baseline of the study at 6 months after the randomization | 
| Treatment costs of the chronically-ill children/adolescents and the siblings (CAMHSRI-DE) | Treatment costs of the chronically-ill children/adolescents and the siblings, assessed from the perspective of an external rater by the German version of the Children and adolescent mental health services receipt inventory (CAMHSRI-DE; Kilian, Losert, McDaid, Park, Knapp, Beecham, Kusakovskaja, Murauskiene & the CAMHEE Project, 2009). | Change from baseline of the study at 6 months after the randomization | 
| Treatment assessment (FBB-T) | Treatment assessment of the parents and the chronically ill children/adolescents and their siblings, in self-assessment from the age of 10 years and by the therapist, assessed on the basis of the treatment assessment questionnaire (FBB-T; Mattejat & Remschmid, 1998). | Change from 6 months after randomization at 12 and 18 months. | 
| Patient satisfaction (ZUF-8) Patient satisfaction | Patient satisfaction of the parents and the chronically ill children/adolescents and their siblings, assessed on the basis of the questionnaire on patient satisfaction (ZUF-8; Schmid & Nübling, 2002). | Change from 6 months after randomization at 12 and 18 months. | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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rare disease, children and adolescents, e-mental health, early detection and early treatment of mental disorders