{"doc_desc":{"title":"Therapeutic management of adult or child patients with hemophilia A : A retrospective observational study - HEMONIS study","idno":"FRESH-PEF74089-en","producers":[{"name":"Camille BACHOT","affiliation":"F. HOFFMANN-LA ROCHE AG"}]},"study_desc":{"title_statement":{"idno":"FRESH-PEF74089-en","IDno":{"metadata_no":[{"agency":"PEF","code":"74089"},{"agency":"FReSH","code":"FRESH-PEF74089"}]},"title":"Therapeutic management of adult or child patients with hemophilia A : A retrospective observational study - HEMONIS study","alternate_title":"HEMONIS"},"study_authorization":{"agency":[]},"authoring_entity":[],"oth_id":[{"name":""}],"production_statement":{"prod_place":"Epidemiology France Portal (PEF)","producers":[{"name":"F. HOFFMANN-LA ROCHE AG","extlink":[{"title":"SIREN","uri":"775752140","role":"sponsor id"}],"role":"sponsor"}],"funding_agencies":[{"name":""}]},"distribution_statement":{"contact":[{"name":";Medical data center","email":"data_sharing.france@roche.com","type":"contact","affiliationName":"F. HOFFMANN-LA ROCHE AG","contactPointLabo":"","extlink":[{"uri":"https:\/\/ror.org\/01mqmer16","role":"organisation id","title":"ROR"},{"uri":"775752140","role":"organisation id","title":"SIREN"}]}]},"study_info":{"keywords":[{"keyword":"emicizumab"}],"topics":[{"topic":"Hematology","vocab":"health theme","extLink":[{"title":"ESV","uri":"http:\/\/data.europa.eu\/8mn\/euroscivoc\/9239f328-78c9-40a5-a0b9-8e66fd9b6245"},{"title":"MeSH","uri":"http:\/\/id.nlm.nih.gov\/mesh\/D006402"}]},{"topic":"Hereditary factor VIII deficiency","vocab":"cim-11","extLink":[{"uri":"http:\/\/id.who.int\/icd\/entity\/1573164739","title":"CIM-11"}]},{"topic":"Healthcare system determinants: Use of care","vocab":"health determinant"},{"topic":"Healthcare system determinants","vocab":"health determinant"}],"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\u2019s 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: \u0093Never inhibitor\u0094 or \u0093Tolerized inhibitor\u0094. - 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: \u0093Current inhibitor\u0094 . - To identify factors associated with choice of ITI regimen (no ITI vs ITI) in the Sev population among patients with inhibitors.","abstract":"","coll_dates":[{"start":"2016-01-01","end":"2018-01-01"}],"nation":[{"name":"France","abbreviation":"fr","extLink":{"vocab":"ISO","vocabURI":"fr"}}],"geog_coverage":"","analysis_unit":"Individuals","universe":"{\n    \"level_sex_clusion_I\": [\n        {\n            \"value\": \"Male\",\n            \"concept\": {\n                \"vocab\": \"MeSH\",\n                \"vocabURI\": \"http:\\\/\\\/id.nlm.nih.gov\\\/mesh\\\/D008297\"\n            }\n        },\n        {\n            \"value\": \"Female\",\n            \"concept\": {\n                \"vocab\": \"MeSH\",\n                \"vocabURI\": \"http:\\\/\\\/id.nlm.nih.gov\\\/mesh\\\/D005260\"\n            }\n        }\n    ],\n    \"level_age_clusion_I\": [\n        {\n            \"value\": \"Child (6 to 12 years)\",\n            \"concept\": {\n                \"vocab\": \"MeSH\",\n                \"vocabURI\": \"http:\\\/\\\/id.nlm.nih.gov\\\/mesh\\\/D002648\"\n            }\n        },\n        {\n            \"value\": \"Young Adult (19 to 24 years)\",\n            \"concept\": {\n                \"vocab\": \"MeSH\",\n                \"vocabURI\": \"http:\\\/\\\/id.nlm.nih.gov\\\/mesh\\\/D055815\"\n            }\n        },\n        {\n            \"value\": \"Adult (25 to 44 years)\",\n            \"concept\": {\n                \"vocab\": \"MeSH\",\n                \"vocabURI\": \"http:\\\/\\\/id.nlm.nih.gov\\\/mesh\\\/D000328\"\n            }\n        },\n        {\n            \"value\": \"Middle Aged (45 to 64 years)\",\n            \"concept\": {\n                \"vocab\": \"MeSH\",\n                \"vocabURI\": \"http:\\\/\\\/id.nlm.nih.gov\\\/mesh\\\/D008875\"\n            }\n        },\n        {\n            \"value\": \"Aged (65 to 79 years)\",\n            \"concept\": {\n                \"vocab\": \"MeSH\",\n                \"vocabURI\": \"http:\\\/\\\/id.nlm.nih.gov\\\/mesh\\\/D000368\"\n            }\n        },\n        {\n            \"value\": \"Aged, 80 and over (80 years and more)\",\n            \"concept\": {\n                \"vocab\": \"MeSH\",\n                \"vocabURI\": \"http:\\\/\\\/id.nlm.nih.gov\\\/mesh\\\/D000369\"\n            }\n        }\n    ],\n    \"level_type_clusion_I\": \"Patients population\",\n    \"level_type_clusion_other\": \"\",\n    \"clusion_I\": \"Inclusion criteria: o Patient aged \u2265 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  \",\n    \"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\"\n}","data_kind":"['Clinical data','Participant-reported health data']","quality_statement":{"standards":[{"name":"['CDISC like']","committee":"","governance":""}],"other_quality_statement":"['GCP\/GVP']"}},"study_development":{"development_activity":[]},"method":{"data_collection":{"time_method":"","frequency":"","sampling_procedure":"['{\"concept\":{\"vocabURI\":\"Other\",\"vocab\":\"CESSDA\"},\"value\":\"Other\"}']","sample_frame":{"frame_unit":{"unit_type":"['Through independent healthcare practitioners']"}},"coll_mode":["{\"concept\":{\"vocabURI\":\"Transcription\",\"vocab\":\"CESSDA\"},\"value\":\"Converting or copying information into a structured record\"}"],"research_instrument":"Access on specific project only","sources":[],"target_sample_size":"< 500 individuals","response_rate":"430"},"method_notes":"Observational Study","study_class":"Unknown","notes":[{"subject":"research type","values":["Observational Study"]},{"subject":"observational study method","values":["Cohort study"]}]},"data_access":{"dataset_availability":{"access_place":"","complete":"","status":"{\"value\":\"Restricted access\",\"extLink\":[{\"title\":\"COAR\",\"uri\":\"http:\/\/purl.org\/coar\/access_right\/c_16ec\"}]}"},"dataset_use":{"restrictions":"","conditions":"","conf_dec":[{"txt":""}],"spec_perm":[{"txt":"","required":""}],"contact":[{"name":""}],"deposit_req":"","cit_req":""},"notes":""}},"additional":{"IsImport":true,"versionLang":"en","originLang":"en","autoTranslation":false,"status":"imported","creationDate":"02-11-2021","lastUpdatedAuto":null,"lastUpdatedManual":"07-07-2025","isContributorPI":"No","contributorName":"Camille BACHOT","contributorAffiliation":"F. HOFFMANN-LA ROCHE AG","addTeamMember":"","obtainedAuthorization":{"otherAuthorizingAgency":[]},"relatedDocument":[],"regulatoryRequirements":{"conformityDeclaration":""},"fundingAgent":{"fundingAgentType":[],"otherFundingAgentType":[]},"sponsor":{"sponsorType":["Industry"],"otherSponsorType":[""]},"governance":{"committee":"Yes"},"collaborations":{"networkConsortium":""},"theme":{"complementaryInformation":"","RareDiseases":""},"activeFollowUp":{"isActiveFollowUp":"Yes","followUpModeOther":""},"interventionalStudy":{"researchPurpose":[],"trialPhase":[],"interventionalStudyModel":"","isClinicalTrial":"","otherResearchPurpose":""},"isInclusionGroups":"","allocation":{"allocationMode":"","allocationUnit":""},"masking":{"maskingType":"","blindedMaskingDetails":[]},"arms":[],"intervention":[],"inclusionGroups":[],"collectionProcess":{"collectionModeDetails":"eCRF","collectionModeOther":""},"dataCollection":{"inclusionStrategyOther":"","samplingModeOther":"","recruitmentSourceOther":"","otherDocumentation":""},"dataCollectionIntegration":{"isDataIntegration":"No"},"geographicalCoverage":{"geoDetail":""},"dataTypes":{"clinicalDataDetails":"Type of data collected : verification of the selection criteria before inclusion, hemophilia A characteristics, demography, history of hemophilia A, medical conditions, therapeutic regimen of moderate and severe hemophilia A at last visit date, musculoskeletal complications, medical history and related conditions at the last visit, therapeutic regimens for hemophilia A in the 2 years preceding the last visit, therapeutic regimen of hemophilia A from January 1, 2000 until 2 years preceding the last visit, ITI from initial diagnosis, events (bleeding) or prevention of physical activities occurring in the 2 years preceding the last visit, all surgeries occurring in the 2 years preceding the last visit.","biologicalDataDetails":"","isDataInBiobank":"No","biobankContent":[],"biobankContentOther":"","dataTypeOther":""},"variableDictionnary":{"variableDictionnaryAvailable":"","variableDictionnaryLink":""},"dataQuality":{"otherDocumentation":""},"mockSample":{"mockSampleAvailable":"","mockSampleLocation":""},"thirdPartySource":{"otherSourceType":[]},"isHealthTheme":"Yes","fileDscr":{"fileTxt":{"fileCitation":{"titlStmt":{"IDno":[]}}}}},"schematype":"survey"}