<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT04413539</url>
  </required_header>
  <id_info>
    <org_study_id>2020-A00729-30</org_study_id>
    <nct_id>NCT04413539</nct_id>
  </id_info>
  <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</brief_title>
  <acronym>DREPAtient</acronym>
  <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</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Argo Sante</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Filière de santé maladies rares MCGRE</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Fédération des malades drépanocytaires et thalassémiques SOS Globi</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>bluebird bio</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
  </sponsors>
  <source>Argo Sante</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      Sickle cell disease (SCD) is the most common genetic disease in France. Its consequences on&#xD;
      patient's life-course and quality of life need to be precisely identified among French&#xD;
      patients and their family to be able to improve patients care according to their specific&#xD;
      needs.&#xD;
&#xD;
      The aim of the study is to accurately describe the impact of SCD on quality of life of&#xD;
      patients living in France, or their family (for minor patients). The consequences of the&#xD;
      disease on professional life, education and material condition of patients or their parents&#xD;
      will be described by the patients themselves.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This is a retrospective study. Patients or parents of minor patients living in France and&#xD;
      suffering from SCD will be informed about the study and enrolled during their usual&#xD;
      follow-up, or by patients' associations. They will be asked to answer an anonymous survey&#xD;
      online relating to their whole life-course with SCD and exploring different aspects of the&#xD;
      consequences of the disease on their life. It contains 4 different sections:&#xD;
&#xD;
        1. socio-demographic questions to establish a respondent profile&#xD;
&#xD;
        2. health status of the respondent to establish the severity of the disease&#xD;
&#xD;
        3. education, professional life, material and economic situation&#xD;
&#xD;
        4. quality of life through MOS SF-36 questionnaire This is a one-shot survey, which will&#xD;
           take approximatively 20 to 30 minutes to answer by the respondent.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">June 2, 2020</start_date>
  <completion_date type="Actual">April 27, 2021</completion_date>
  <primary_completion_date type="Actual">April 27, 2021</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Case-Only</observational_model>
    <time_perspective>Retrospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <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.</measure>
    <time_frame>Once at enrollment</time_frame>
    <description>Measure of mutual impact of health status on patients educational and socio-professional life course.</description>
  </primary_outcome>
  <secondary_outcome>
    <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</measure>
    <time_frame>Once at enrollment</time_frame>
    <description>The perceived impact of disease on education will be confirmed by:&#xD;
Type of education adjustment introduced&#xD;
The level and the cause of leaving full-time education&#xD;
The intensity of school absences&#xD;
The feeling of discrimination on school time&#xD;
The impact of disease on professional life will be measured by:&#xD;
The professional status&#xD;
The sick leaves&#xD;
The feeling of discrimination at work&#xD;
The compatibility of work conditions with the disease&#xD;
The impact of disease on material and economic situation will be assessed by:&#xD;
The income level&#xD;
The patient cost sharing&#xD;
The remaining balance&#xD;
The recognition of handicap&#xD;
The recognition of disability&#xD;
The quality of life score of respondents:&#xD;
- Medical outcome study short form 36 item health survey (MOS SF-36)</description>
  </secondary_outcome>
  <enrollment type="Actual">1088</enrollment>
  <condition>Sickle-cell Disease (SCD)</condition>
  <eligibility>
    <study_pop>
      <textblock>
        Adults or parents of minor children with SCD living in France.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  18 years or older&#xD;
&#xD;
          -  Diagnosed with SCD or parent of a minor child diagnosed with SCD&#xD;
&#xD;
          -  Able to speak and understand French&#xD;
&#xD;
          -  Willing to participate to the study&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Person having serious difficulties to read or speak French, unable to answer the&#xD;
             questions&#xD;
&#xD;
          -  Person suffering from other chronic disease which could bias the representation they&#xD;
             have about SCD&#xD;
&#xD;
          -  Patient cured with a bone marrow transplant&#xD;
&#xD;
          -  Patient with mental disorder preventing the patient to understand the study&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Frederic Galacteros, MD,PhD,Prof.</last_name>
    <role>Principal Investigator</role>
    <affiliation>Red Cell genetic diseases unity, Henri Mondor Hospital, Paris</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Centre de références syndromes drépanocytaires majeurs thalassémie et autres maladies rares du globule rouge et de l'érythropoïése</name>
      <address>
        <city>Créteil</city>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>France</country>
  </location_countries>
  <link>
    <url>https://www.modalisa-enquete.com/drepatient</url>
    <description>Related Info</description>
  </link>
  <reference>
    <citation>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.</citation>
    <PMID>27547816</PMID>
  </reference>
  <reference>
    <citation>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.</citation>
    <PMID>24106440</PMID>
  </reference>
  <reference>
    <citation>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.</citation>
    <PMID>27439910</PMID>
  </reference>
  <reference>
    <citation>Habibi A, Arlet JB, Stankovic K, Gellen-Dautremer J, Ribeil JA, Bartolucci P, Lionnet F; centre de reference maladies rares &lt;&lt; syndromes drepanocytaires majeurs &gt;&gt;. [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.</citation>
    <PMID>26007619</PMID>
  </reference>
  <reference>
    <citation>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.</citation>
    <PMID>19682065</PMID>
  </reference>
  <reference>
    <citation>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.</citation>
    <PMID>11402515</PMID>
  </reference>
  <reference>
    <citation>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.</citation>
    <PMID>14050985</PMID>
  </reference>
  <reference>
    <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.</citation>
    <PMID>8625722</PMID>
  </reference>
  <reference>
    <citation>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.</citation>
    <PMID>21131035</PMID>
  </reference>
  <reference>
    <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>
    <PMID>30984656</PMID>
  </reference>
  <reference>
    <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.</citation>
    <PMID>25880537</PMID>
  </reference>
  <verification_date>July 2021</verification_date>
  <study_first_submitted>May 29, 2020</study_first_submitted>
  <study_first_submitted_qc>May 29, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">June 4, 2020</study_first_posted>
  <last_update_submitted>July 20, 2021</last_update_submitted>
  <last_update_submitted_qc>July 20, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">July 21, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Sickle-cell disease</keyword>
  <keyword>quality of life</keyword>
  <keyword>education</keyword>
  <keyword>socio-professional integration</keyword>
  <keyword>france</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Anemia, Sickle Cell</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT04413539
org study id: 2020-A00729-30
nct id: NCT04413539
lead sponsor: 
collaborator: 
collaborator: 
collaborator: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
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.
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.
observational model: Case-Only
time perspective: Retrospective
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.
time frame: Once at enrollment
description: Measure of mutual impact of health status on patients educational and socio-professional life course.
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
time frame: Once at enrollment
description: 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)
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Frederic Galacteros, MD,PhD,Prof.
role: Principal Investigator
affiliation: Red Cell genetic diseases unity, Henri Mondor Hospital, Paris
facility: 
country: France
url: https://www.modalisa-enquete.com/drepatient
description: Related Info
citation: 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.
PMID: 27547816
citation: 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.
PMID: 24106440
citation: 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.
PMID: 27439910
citation: 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.
PMID: 26007619
citation: 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.
PMID: 19682065
citation: 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.
PMID: 11402515
citation: 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.
PMID: 14050985
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.
PMID: 8625722
citation: 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.
PMID: 21131035
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.
PMID: 30984656
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.
PMID: 25880537
responsible party type: Sponsor
mesh term: Anemia, Sickle Cell
sharing ipd: No