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 (DREPAtient)

• ClinicalTrials.gov Identifier: NCT04413539
• Recruitment Status: Completed
• First Posted: June 4, 2020
• Last Update Posted: July 21, 2021
• Sponsor: Argo Sante
• Collaborators: Filière de santé maladies rares MCGRE; Fédération des malades drépanocytaires et thalassémiques SOS Globi; bluebird bio
• Information provided by (Responsible Party): Argo Sante

Tracking Information

• First Submitted Date: May 29, 2020
• First Posted Date: June 4, 2020
• Last Update Posted Date: July 21, 2021
• Actual Study Start Date: June 2, 2020
• Actual Primary Completion Date: April 27, 2021 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: May 29, 2020)

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. [ Time Frame: Once at enrollment ]

Measure of mutual impact of health status on patients educational and socio-professional life course.

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: May 29, 2020)

- 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 [ Time Frame: Once at enrollment ]

The perceived impact of disease on education will be confirmed by:

• Type of education adjustment introduced
• The level and the cause of leaving full-time education
• The intensity of school absences
• The feeling of discrimination on school time

The impact of disease on professional life will be measured by:

• The professional status
• The sick leaves
• The feeling of discrimination at work
• The compatibility of work conditions with the disease

The impact of disease on material and economic situation will be assessed by:

• The income level
• The patient cost sharing
• The remaining balance
• The recognition of handicap
• The recognition of disability

The quality of life score of respondents: - Medical outcome study short form 36 item health survey (MOS SF-36)

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief 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
• 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

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.

• Study Type: Observational
• Study Design: Observational Model: Case-Only; Time Perspective: Retrospective
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• Sampling Method: Non-Probability Sample
• Study Population: Adults or parents of minor children with SCD living in France.
• Condition: Sickle-cell Disease (SCD)
• Intervention: Not Provided
• Study Groups/Cohorts: Not Provided

Publications *

• Crosby LE, Joffe NE, Irwin MK, Strong H, Peugh J, Shook L, Kalinyak KA, Mitchell MJ. School Performance and Disease Interference in Adolescents with Sickle Cell Disease. Phys Disabil. 2015;34(1):14-30. doi: 10.14434/pders.v34i1.13918.
• Dos Santos JP, Gomes Neto M. Sociodemographic aspects and quality of life of patients with sickle cell anemia. Rev Bras Hematol Hemoter. 2013;35(4):242-5. doi: 10.5581/1516-8484.20130093.
• Gardner K, Douiri A, Drasar E, Allman M, Mwirigi A, Awogbade M, Thein SL. Survival in adults with sickle cell disease in a high-income setting. Blood. 2016 Sep 8;128(10):1436-8. doi: 10.1182/blood-2016-05-716910. Epub 2016 Jul 20. No abstract available.
• Habibi A, Arlet JB, Stankovic K, Gellen-Dautremer J, Ribeil JA, Bartolucci P, Lionnet F; centre de reference maladies rares << syndromes drepanocytaires majeurs >>. [French guidelines for the management of adult sickle cell disease: 2015 update]. Rev Med Interne. 2015 May 11;36(5 Suppl 1):5S3-84. doi: 10.1016/S0248-8663(15)60002-9. French.
• Chapelon E, Garabedian M, Brousse V, Souberbielle JC, Bresson JL, de Montalembert M. Osteopenia and vitamin D deficiency in children with sickle cell disease. Eur J Haematol. 2009 Dec 1;83(6):572-8. doi: 10.1111/j.1600-0609.2009.01333.x. Epub 2009 Aug 13.
• Godeau B, Noel V, Habibi A, Schaeffer A, Bachir D, Galacteros F. [Sickle cell disease in adults: which emergency care by the internists?]. Rev Med Interne. 2001 May;22(5):440-51. doi: 10.1016/s0248-8663(01)00369-1. French.
• LINDENBAUM J, KLIPSTEIN FA. FOLIC ACID DEFICIENCY IN SICKLE-CELL ANEMIA. N Engl J Med. 1963 Oct 24;269:875-82. doi: 10.1056/NEJM196310242691701. No abstract available.
• 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.
• Rees DC, Williams TN, Gladwin MT. Sickle-cell disease. Lancet. 2010 Dec 11;376(9757):2018-31. doi: 10.1016/S0140-6736(10)61029-X. Epub 2010 Dec 3.
• 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.
• 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.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: July 20, 2021): 1088
• Original Estimated Enrollment (submitted: May 29, 2020): 926
• Actual Study Completion Date: April 27, 2021
• Actual Primary Completion Date: April 27, 2021 (Final data collection date for primary outcome measure)

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: France

Administrative Information

• NCT Number: NCT04413539
• Other Study ID Numbers: 2020-A00729-30
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Argo Sante
• Original Responsible Party: Same as current
• Current Study Sponsor: Argo Sante
• Original Study Sponsor: Same as current

Collaborators

• Filière de santé maladies rares MCGRE
• Fédération des malades drépanocytaires et thalassémiques SOS Globi
• bluebird bio

Investigators

• Principal Investigator: Frederic Galacteros, MD,PhD,Prof.; Red Cell genetic diseases unity, Henri Mondor Hospital, Paris

• PRS Account: Argo Sante
• Verification Date: July 2021