Hemophilia
Conditions
Brief summary
The overarching goal of the EPOCH project is to: * Support a broad and robust needs assessment in the haemophilia space in China * Measure the variability in the needs and care provision * Generate outcome data (health economic outcomes and supporting clinical and PRO data) to enable measuring the impact of different treatment modalities and different levels of treatment access PRIMARY OBJECTIVES * Determine the burden of haemophilia in China, and the determinants of its variability * Determine the costs of haemophilia care in China, and the determinants of its variability SECONDARY OBJECTIVES * Measure treatment patterns and their variability * Measure levels of access to care * Estimate the impact of haemophilia and its treatment of patients reported outcomes * Understand consistency of care by centres/geography/demographics
Interventions
None listed
Sponsors
Institute of Hematology & Blood Diseases Hospital, China
EPOCH Study, Ltd.
Study design
Observational model
CASE_ONLY
Time perspective
CROSS_SECTIONAL
Eligibility
Sex/Gender
MALE
Age
12 Years to No maximum
Healthy volunteers
Yes
Inclusion criteria
* Male patients, aged ≥12 years at the index date * Patients must be diagnosed with non-acquired severe haemophilia A or B prior to the previous 12 months * Patients who are able to understand and assent / consent to the study
Exclusion criteria
* Patients who cannot, for any reasons (language barriers, mental incapacity, etc.) understand and answer the questions of the study questionnaires * Patients that cannot access internet connection or download the application
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Successfully registered research centers | Baseline until week 52 | Successfully registered research centers in Trial Phase |
| Number of enrolled patients | Baseline until week 52 | Number of enrolled patients in Trial Phase |
| Proportion of completed questionnaires | Baseline until week 52 | Proportion of completed questionnaires for the Trial Phase |
| Proportion of missing data | Baseline until week 52 | Proportion of missing data for the Trial Phase |
| Qualitative information on study barriers and facilitators | Baseline until week 52 | Qualitative information on study barriers and facilitators for the Trial Phase |
| Diagnostic and treatment burden for hemophilia patients | Baseline until week 52 | Diagnostic and treatment burden for hemophilia patients for the Implementation Phase |
| Costs of hemophilia diagnosis and treatment | Baseline until week 52 | Costs of hemophilia diagnosis and treatment for the Implementation Phase |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Measure treatment patterns and their variability | Baseline until week 52 | Measures of treatment patterns will be defined by type of treatment product, dose and frequency of administration. These measures will be considered in relation to severity of disease and eventually presence of inhibitors and relevant comorbidities. This measure will be collected both cross-sectionally and prospectively and reported directly by the patients. By organizing patient-level data and applying statistical analysis methods, we calculated the total cost of hemophilia diagnosis and treatment in China and specific regions. |
| Measure levels of access to care | Baseline until week 52 | The access to care will be calculated considering the mean number of visits/consultations/follow-ups and hospitalizations for patient and stratified in relation to severity of disease, frequency of bleeding and presence of inhibitors/comorbidities and haemophilia related complications or health problems. |
| Estimate the impact of haemophilia and its treatment on patients reported outcomes | Baseline until week 52 | General health problems are defined by determinants like use of mobility aids and assistive devices, use of pain medication, presence of and how acute pain and chronic pain interfere with daily life, difficulty with daily activities, surgical history and concurrent medical problems and work/school life. Haemophilia-related health problems refer to disease severity, inhibitor status and history, bleeding frequency and history, treatment regimen and history and presence of target joint(s) |
Countries
China
Outcome results
None listed