Clinical Trial Details
— Status: Completed
Administrative data
| NCT number |
NCT03299699 |
| Other study ID # |
2017-06 |
| Secondary ID |
|
| Status |
Completed |
| Phase |
|
| First received |
|
| Last updated |
|
| Start date |
March 15, 2020 |
| Est. completion date |
May 1, 2021 |
Study information
| Verified date |
June 2023 |
| Source |
Assistance Publique Hopitaux De Marseille |
| Contact |
n/a |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Observational
|
Clinical Trial 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.
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).
