Quality of Life and Adjustment Among Siblings of Children and Adolescents With Severe Hemophilia
Condition: Severe Haemophilia
MeSH terms: Hemophilia A
Study type: Observational

Summary: 
      Severe hemophilia is a rare and chronic disease characterized by spontaneous bleedings from
      early childhood, which may lead to various complications especially in joints. The diagnosis
      of this disease, but also its long term care have an impact on the relatives of the affected
      persons, including the siblings who bear daily the cognitive, emotional and social impacts of
      the disease.

      Studies conducted in the framework of pediatric chronic diseases showed that siblings of
      affected children presented a higher prevalence of physical and psychological troubles
      (emotional distress, behavioral disorders, etc.) than siblings who were not concerned by a
      disease. Few studies have been conducted in the framework of severe hemophilia, and to our
      knowledge, no study addresses this issue in France.
    

Detailed description: 
      -The assessment of the quality of life would allow assessing both positive and negative
      impacts, by exploring in a global manner the children and adolescents functioning in various
      domains (physical and psychological well-being, relations with parents and friends, school…).

      Regarding the specific context of hemophilia:

        -  Few studies have been conducted on the experience of severe hemophilia of a young
           patient by the siblings, and to our knowledge, no studies have been conducted in France
           on this topic.

        -  There are numerous studies concerning the impact of chronic diseases such as diabetes or
           cancer of a child on the siblings, contrary to those concerning rare diseases.
           Hemophilia, a frequent disease among rare diseases, might be an interesting model in
           order to understand the impact of this type of disorder on the functioning of the
           siblings.

        -  Contrary to insufficiently treated hemophilia potentially leading to disability,
           well-treated hemophilia might be considered as an at-risk disorder. In this specific
           context, the representation of the disease by the siblings might be singular.

        -  Parents are the major actors of the surveillance of the affected young patient in the
           everyday life. The transfer of parental responsibilities to healthy siblings, and its
           impact on the fraternal relationships might therefore be increased. Gender might have a
           specific modulating role on these elements (risk of transmission in female and different
           representation of the social role).

        -  Results presented in the literature are reported from studies whose representativeness
           of the study sample (or sample size) is questionable. The cohort of the French National
           Network FranceCoag represents at an international level a rare exhaustive population of
           persons with severe hemophilia followed over time.

      Objectives The main objective of this study is to understand the mechanisms involved in the
      quality of the siblings' functioning and interactions in the context of severe hemophilia, in
      order to be able to propose adapted support modes.

      The secondary (and operational) objectives are:

        -  To assess the impact of the functioning and interactions of the family on the quality of
           life (considered as a marker of the global health) reported by the siblings

        -  To compare the quality of life reported by siblings of children and adolescents with
           severe hemophilia, with the quality of life of the patients themselves, and with
           published reference values (age and gender-matched controls from the general population)

        -  To assess the emotional and social adjustment (behavioral disorders, academic success),
           which represents a mediator of the quality of life, among siblings

        -  To assess the moderating effects of gender, age, rank among siblings, time since
           diagnosis, quality of social support received, skills acquired to manage emotions, on
           quality of life as well as on the emotional and social adjustment

        -  To highlight clusters of siblings with different profiles of quality of life

        -  To propose tools, meetings and training sessions in order to support concerned families

      Material and methods This study is complementary to the multicentric, observational,
      cross-sectional, TRANSHEMO study. TRANSHEMO focuses on the issues surrounding the transition
      into adulthood among young persons with severe hemophilia in France. This study also aims to
      identify some of the socio- cognitive, emotional, and familial determinants of a good
      transition into adulthood. Perceptions of older children and adolescents (aged 14-17 years
      old) with severe hemophilia and those of young adults (aged 20-29 years old) will be
      described et compared, regarding their expectations and their feelings about growing into
      adulthood.

      The present study which is also a multicentric, observational, cross-sectional study,
      proposes to include the siblings [i.e. brothers, sisters, half-brothers and half-sisters
      (aged 8-18 years old)] of the older children and adolescents included in the TRANSHEMO study,
      living in the same household. The investigators will ask them to fill-up a booklet with
      several questionnaires, focused on quality of life. This study will allow to have preliminary
      results in order to extend the project to another age group of children with severe
      hemophilia and their siblings.

      Expected results This study will allow to comprehend what the impact of the disease on the
      siblings could be, which is of particular interest in the global approach whose goal is to
      take care of and to support the affected persons and their relatives. The identification of
      difficulties among siblings and of their determinants will allow to detect children at risk
      of adjustment problem, in order to offer them an adapted support, but also to develop
      specific tools to support the families (brochures, meetings, therapeutic education
      activities).
    

Observational model: Cohort
Time perspective: Prospective
Sampling method: Non-Probability Sample
Study population: 
        siblings of children and adolescents with severe hemophilia