ITMO public health
Last update : 11/08/2021 | Version : 1 | ID : 74089
| General | |
| Identification | |
| Detailed name | Therapeutic management of adult or child patients with hemophilia A : A retrospective observational study - HEMONIS study |
| Sign or acronym | HEMONIS |
| CNIL registration number, number and date of CPP agreement, AFSSAPS (French Health Products Safety Agency) authorisation | ML39418 |
| General Aspects | |
| Medical area |
Hematology |
| Study in connection with Covid-19 |
No |
| Pathology (details) | Hemophilia A |
| Health determinants |
Medicine |
| Keywords | emicizumab |
| Scientific investigator(s) (Contact) | |
| Name of the director | Roche Medical data center |
| Address | 4 cours de l'Ile Seguin - 92100 Boulogne-Billancourt |
| data_sharing.france@roche.com | |
| Organization | Roche SAS |
| Collaborations | |
| Participation in projects, networks and consortia |
No |
| Funding | |
| Funding status |
Private |
| Governance of the database | |
| Sponsor(s) or organisation(s) responsible | Roche SAS |
| Organisation status |
Private |
| Presence of scientific or steering committees |
Yes |
| Additional contact | |
| Main features | |
| Type of database | |
| Type of database |
Study databases |
| Study databases (details) |
Cohort study |
| Database recruitment is carried out by an intermediary |
A selection of health care professionals |
| Database recruitment is is made on the basis of: |
Medication(s) taken |
| Database recruitment is carried out as part of an interventional study |
No |
| Database objective | |
| Main objective |
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. |
| Inclusion criteria |
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 Non inclusion criteria: 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 |
| Population type | |
| Age |
Childhood (6 to 13 years) Adolescence (13 to 18 years) Adulthood (19 to 24 years) Adulthood (25 to 44 years) Adulthood (45 to 64 years) Elderly (65 to 79 years) Great age (80 years and more) |
| Population covered |
Sick population |
| Pathology | D66 - Hereditary factor VIII deficiency |
| Gender |
Male Woman |
| Geography area |
National |
| Data collection | |
| Dates | |
| Date of first collection (YYYY or MM/YYYY) | 2016 |
| Date of last collection (YYYY or MM/YYYY) | 2018 |
| Size of the database | |
| Size of the database (number of individuals) |
< 500 individuals |
| Details of the number of individuals | 430 |
| Data | |
| Database activity |
Data collection completed |
| Type of data collected |
Clinical data Declarative data |
| Clinical data (detail) |
Medical registration |
| Details of collected clinical data | 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. |
| Declarative data (detail) |
Internet self-questionnaire |
| Presence of a biobank |
No |
| Procedures | |
| Data collection method | eCRF |
| Classifications used | CDISC like |
| Quality procedure(s) used | GCP/GVP |
| Participant monitoring |
Yes |
| Monitoring procedures |
Monitoring by contact with the referring doctor |
| Followed pathology | D66 - Hereditary factor VIII deficiency |
| Links to administrative sources |
No |
| Promotion and access | |
| Promotion | |
| Access | |
| Dedicated website | https://www.roche.fr/fr/innovation-recherche-medicale/data-sharing-portail-d-information-partage-des-donnees.html |
| Presence of document that lists variables and coding procedures |
Yes |
| Access to aggregated data |
Access on specific project only |
| Access to individual data |
Access on specific project only |
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