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<docDscr>
  <citation>
    <titlStmt>
      <titl>Therapeutic management of adult or child patients with hemophilia A : A retrospective observational study - HEMONIS study</titl>
      <IDNo>FRESH-PEF74089-en</IDNo>
    </titlStmt>
    <prodStmt>
      <producer abbr="" affiliation="F. HOFFMANN-LA ROCHE AG" role="">Camille BACHOT</producer>
    </prodStmt>
    <verStmt>
      <version></version>
    </verStmt>
  </citation>
</docDscr>
<stdyDscr>
  <citation>
    <titlStmt>
      <titl>
                Therapeutic management of adult or child patients with hemophilia A : A retrospective observational study - HEMONIS study            </titl>
      <altTitl>
                    HEMONIS                </altTitl>
      <IDNo agency="FReSH-lang">
                FRESH-PEF74089-en            </IDNo>
      <IDNo agency="PEF">
                    74089                </IDNo>
      <IDNo agency="FReSH">
                    FRESH-PEF74089                </IDNo>
    </titlStmt>
    <rspStmt>
      <othId affiliation="" type="">
                                    </othId>
    </rspStmt>
    <prodStmt>
      <producer role="sponsor">
                                            F. HOFFMANN-LA ROCHE AG                    
                                    </producer>
      <prodPlac>
                Epidemiology France Portal (PEF)            </prodPlac>
    </prodStmt>
    <distStmt>
      <distrbtr abbr="null">
                    null</distrbtr>
      <contact affiliation="F. HOFFMANN-LA ROCHE AG" email="data_sharing.france@roche.com">
                    ;Medical data center
                                            <ExtLink title="ROR" URI="https://ror.org/01mqmer16" role="organisation id"/>
                                            <ExtLink title="SIREN" URI="775752140" role="organisation id"/>
                                    </contact>
      <depDate date="2026-06-16"/>
      <distDate date="2026-06-16"/>
    </distStmt>
    <biblCit format="">
            </biblCit>
    <holdings URI="null"/>
    <notes> </notes>
  </citation>
  <stdyInfo>
    <studyBudget/>
    <subject>
      <keyword vocab="" vocabURI="">
                    emicizumab</keyword>
      <topcClas vocab="health theme">
                        Hematology                                                        <ExtLink title="ESV" URI="http://data.europa.eu/8mn/euroscivoc/9239f328-78c9-40a5-a0b9-8e66fd9b6245"/>
                                                            <ExtLink title="MeSH" URI="http://id.nlm.nih.gov/mesh/D006402"/>
                                                </topcClas>
      <topcClas vocab="cim-11">
                        Hereditary factor VIII deficiency                                            </topcClas>
      <topcClas vocab="health determinant">
                        Healthcare system determinants: Use of care                    </topcClas>
      <topcClas vocab="health determinant">
                        Healthcare system determinants                    </topcClas>
    </subject>
    <abstract contentType="purpose">Primary Objective: To describe the current therapeutic regimen for moderate and severe HA patients (ITI / on-demand / short-term prophylaxis / long-term prophylaxis) in the MS population. Secondary Objectives: - To describe the current therapeutic regimen (ITI / on-demand / short-term prophylaxis / long-term prophylaxis) in severe HA patients in the Sev population. In both Moderate or Severe (MS) population and Severe (Sev) population : - To describe the profile of patients with HA - To describe the current treatment type according to therapeutic regimen, inhibitors status and severity - To describe patient status regarding medical conditions and disease characteristics (severity, presence of inhibitors) - To describe the patient’s musculoskeletal complications In Severe (Sev) population: - To describe medical history and related conditions - To describe thrombosis events - To describe therapeutic management administered to patients with severe HA from January 1, 2000 to the last visit (therapeutic regimens in the 2 years preceding the index date , therapeutic regimen from January 1, 2000 until 2 years preceeding the last visit) - To describe ITI prescribed treatment from initial diagnosis - To describe significant events (bleeding) or prevention of physical activities and their management occurring in the 2 years preceeding the last visit. - To describe all surgeries occurring in the 2 years preceeding the last visit. Exploratory Objectives: - To identify factors associated with choice of therapeutic regimen (on-demand vs prophylaxis) in the Sev population with inhibitors status at last visit: Never inhibitor or Tolerized inhibitor. - To identify factors associated with choice of therapeutic regimen (Prophylaxis vs on-demand, ITI vs on-demand) in the Sev population with inhibitors status at last visit: Current inhibitor . - To identify factors associated with choice of ITI regimen (no ITI vs ITI) in the Sev population among patients with inhibitors.</abstract>
    <abstract contentType="abstract">null</abstract>
    <sumDscr>
      <collDate event="start" date="2016-01-01"/>
      <collDate event="end" date="2018-01-01"/>
      <nation abbr="fr">
                    France
                                            <concept vocab="ISO" vocabURI="fr"/>
                                    </nation>
      <geogUnit/>
      <anlyUnit>
                Individuals            </anlyUnit>
      <universe level="type" clusion="I">Patients population                    </universe>
      <universe level="sex" clusion="I">Male                                                            <concept vocab="MeSH" vocabURI="http://id.nlm.nih.gov/mesh/D008297"/>
                                                    </universe>
      <universe level="sex" clusion="I">Female                                                            <concept vocab="MeSH" vocabURI="http://id.nlm.nih.gov/mesh/D005260"/>
                                                    </universe>
      <universe level="age" clusion="I">Child (6 to 12 years)                                                            <concept vocab="MeSH" vocabURI="http://id.nlm.nih.gov/mesh/D002648"/>
                                                    </universe>
      <universe level="age" clusion="I">Young Adult (19 to 24 years)                                                            <concept vocab="MeSH" vocabURI="http://id.nlm.nih.gov/mesh/D055815"/>
                                                    </universe>
      <universe level="age" clusion="I">Adult (25 to 44 years)                                                            <concept vocab="MeSH" vocabURI="http://id.nlm.nih.gov/mesh/D000328"/>
                                                    </universe>
      <universe level="age" clusion="I">Middle Aged (45 to 64 years)                                                            <concept vocab="MeSH" vocabURI="http://id.nlm.nih.gov/mesh/D008875"/>
                                                    </universe>
      <universe level="age" clusion="I">Aged (65 to 79 years)                                                            <concept vocab="MeSH" vocabURI="http://id.nlm.nih.gov/mesh/D000368"/>
                                                    </universe>
      <universe level="age" clusion="I">Aged, 80 and over (80 years and more)                                                            <concept vocab="MeSH" vocabURI="http://id.nlm.nih.gov/mesh/D000369"/>
                                                    </universe>
      <universe clusion="I">Inclusion criteria: o Patient aged ≥ 5 years old  o Patient with moderate or severe constitutional  o Patient with a last visit within the last 2 years  o Patient followed in the same center for 2 years previous to the last visit, or patient followed in the same center from initial diagnosis, whichever is the shortest as collected in eCRF                      </universe>
      <universe clusion="E">o Patient who has received written information and expressed his/her refusal to participate in the study, or minor patient for whom at least one parent or guardian has expressed a refusal to participate in the study as checked by programming (patients without information date) o Patient included in a clinical trial at the date of last visit as collected in eCRF                    </universe>
      <dataKind>Clinical data</dataKind>
      <dataKind>Participant-reported health data</dataKind>
    </sumDscr>
    <qualityStatement>
      <standardsCompliance>
        <standard>
          <standardName>CDISC like</standardName>
        </standard>
      </standardsCompliance>
      <otherQualityStatement>GCP/GVP</otherQualityStatement>
    </qualityStatement>
  </stdyInfo>
  <method>
    <dataColl>
      <sampProc>Other                                                            <concept vocab="CESSDA" vocabURI="Other"/>
                                                    </sampProc>
      <sampleFrame>
        <frameUnit>
          <unitType>Through independent healthcare practitioners</unitType>
        </frameUnit>
      </sampleFrame>
      <targetSampleSize>
        <sampleSizeFormula>&lt; 500 individuals                        </sampleSizeFormula>
      </targetSampleSize>
      <collMode>Converting or copying information into a structured record                                                            <concept vocab="CESSDA" vocabURI="Transcription"/>
                                                    </collMode>
    </dataColl>
    <notes>Observational Study</notes>
    <notes subject="research type">
                        Observational Study                    </notes>
    <notes subject="observational study method">
                        Cohort study                    </notes>
    <anlyInfo>
      <respRate>430</respRate>
    </anlyInfo>
    <stdyClas>Unknown</stdyClas>
  </method>
  <dataAccs>
    <setAvail>
      <avlStatus>
                            Restricted access                                                    </avlStatus>
    </setAvail>
  </dataAccs>
  <othrStdyMat>
    <relMat>Access on specific project only</relMat>
  </othrStdyMat>
</stdyDscr>
<dataDscr>
</dataDscr></codeBook>
