Clinical trial NCT04413539: Evaluation of Impact of Disease on Quality of Life, Education and Socio-professional Integration of Adults and Parents of Children Living with Sickle- Cell Disease in France
Official title: Evaluation of Impact of Disease on Quality of Life, Education and Socio-professional Integration of Adults and Parents of Children Living with Sickle- Cell Disease in France
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Sickle-cell Disease (SCD)
Keywords: Sickle-cell disease
quality of life
education
socio-professional integration
france
Lead sponsor: Argo Sante
Sponsor class: OTHER
Collaborators: name: Filière de santé maladies rares MCGRE
name: Fédération des malades drépanocytaires et thalassémiques SOS Globi
name: bluebird bio
First posted: 2020-06-04
Enrollment: 1088
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Adults or parents of minor children with SCD living in France.

Brief summary: Sickle cell disease (SCD) is the most common genetic disease in France. Its consequences on patient's life-course and quality of life need to be precisely identified among French patients and their family to be able to improve patients care according to their specific needs.

The aim of the study is to accurately describe the impact of SCD on quality of life of patients living in France, or their family (for minor patients). The consequences of the disease on professional life, education and material condition of patients or their parents will be described by the patients themselves.

Detailed description: This is a retrospective study. Patients or parents of minor patients living in France and suffering from SCD will be informed about the study and enrolled during their usual follow-up, or by patients' associations. They will be asked to answer an anonymous survey online relating to their whole life-course with SCD and exploring different aspects of the consequences of the disease on their life. It contains 4 different sections:

1. socio-demographic questions to establish a respondent profile
2. health status of the respondent to establish the severity of the disease
3. education, professional life, material and economic situation
4. quality of life through MOS SF-36 questionnaire This is a one-shot survey, which will take approximatively 20 to 30 minutes to answer by the respondent.

Eligibility criteria: Inclusion Criteria:

* 18 years or older
* Diagnosed with SCD or parent of a minor child diagnosed with SCD
* Able to speak and understand French
* Willing to participate to the study

Exclusion Criteria:

* Person having serious difficulties to read or speak French, unable to answer the questions
* Person suffering from other chronic disease which could bias the representation they have about SCD
* Patient cured with a bone marrow transplant
* Patient with mental disorder preventing the patient to understand the study

Primary outcomes:
measure: Describe the disease impact on quality of life through description of professional life, education and material condition of patients, or their parents (for minor patients), suffering from sickle-cell disease and living in France.
timeFrame: Once at enrollment
Secondary outcomes:
measure: - Study the factors of heterogeneity of patients' profiles - Explore the causal relation between health status and employment conditions, education, material and economic situation - Assess the patients' needs in care and socio-economic support
timeFrame: Once at enrollment
Other outcomes:
Locations:
city: Créteil
country: France
facility: Centre de références syndromes drépanocytaires majeurs thalassémie et autres maladies rares du globule rouge et de l'érythropoïése
Study officials:
affiliation: Red Cell genetic diseases unity, Henri Mondor Hospital, Paris
name: Frederic Galacteros, MD,PhD,Prof.
role: PRINCIPAL_INVESTIGATOR
References:
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citation: Noll RB, Vannatta K, Koontz K, Kalinyak K, Bukowski WM, Davies WH. Peer relationships and emotional well-being of youngsters with sickle cell disease. Child Dev. 1996 Apr;67(2):423-36. doi: 10.1111/j.1467-8624.1996.tb01743.x.
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citation: Salih KMA. The impact of sickle cell anemia on the quality of life of sicklers at school age. J Family Med Prim Care. 2019 Feb;8(2):468-471. doi: 10.4103/jfmpc.jfmpc_444_18.
citation: Sehlo MG, Kamfar HZ. Depression and quality of life in children with sickle cell disease: the effect of social support. BMC Psychiatry. 2015 Apr 11;15:78. doi: 10.1186/s12888-015-0461-6.