Research Title：Adult and adolescent hemophilia patients treated with Marstacimab: a patient Experience Registry (AMBER) Protocol Number：94250855 Protocol Version Number：version 0.3 Date：15-November-2024 Leading site：Institution of Hematology \& Blood Diseases Hospital, Chinese Academy of Medical Sciences Active Pharmaceutical Ingredient：a human IgG1 monoclonal antibody that targets the tissue factor pathway inhibitor (TFPI) Drug Name Generic name: marstacimab-hncq Purpose of the Study Primary Objectives:To quantify patient preferences for subcutaneous versus intravenous (IV) injection using the Marstacimab-Patient Preference Questionnaire (M-PPQ) after 1 month of marstacimab for routine prophylaxis treatment.
To assess the treatment burden using the Hemophilia Treatment Experience Measure (Hemo-TEM) in hemophilia patients after 6 months of marstacimab for routine prophylaxis treatment.
Exploratory Objectives:
To evaluate the annualized bleeding rate (ABR) of different types of bleeds after 6 months of subcutaneous marstacimab injections in hemophilia patients.
To assess changes in joint status scores between baseline and the final visit after 6 months of subcutaneous marstacimab injections in hemophilia patients, using the Hemophilia Early Arthropathy Detection with Ultrasound in China (HEAD-US-C).
To evaluate patient preferences regarding treatment experience via M-PPQ after 6 months
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patients with hemophilia who will receive marstacimab for routine prophylactic treatment in a real-world setting. Each patient will receive treatment and care according to the standard clinical practice. The total recruitment period will be 18 months. Patients will be prescribed marstacimab in routine practice and initiate marstacimab treatment on the same day (or as soon as possible after prescribing). The observation period for individual patients is defined as ending on the 180th day from the index date (the date of the first dose of marstacimab).
Case-only
Prospective
Marstacimab
Drugthe first visit (V1) is scheduled on the day of the first administration of marstacimab. The recommended dosing frequency for the patient should follow the instructions provided, which are as follows: On Day 1, administer 300 mg subcutaneously (divided into two injections, each 150 mg) (loading dose); starting from Day 8, administer 150 mg subcutaneously once a week. Record the patient's prescription recommendations, actual treatment records (administration time, dosage, frequency), and adherence rates. Visit 2 (V2) and visit 3 (V3) are defined as 30 days (±7 days) and 180 days (±30 days) after the initiation of marstacimab treatment, respectively.
patient preferences for subcutaneous
To quantify patient preferences for subcutaneous versus intravenous (IV) injection using the Marstacimab-Patient Preference Questionnaire (M-PPQ) after 1 month of marstacimab for routine prophylaxis treatment.
To assess the treatment burden
To assess the treatment burden using the Hemophilia Treatment Experience Measure (Hemo-TEM) in hemophilia patients after 6 months of marstacimab for routine prophylaxis treatment.
To evaluate the annualized bleeding rate (ABR)
To evaluate the annualized bleeding rate (ABR) of different types of bleeds after 6 months of subcutaneous marstacimab injections in hemophilia patients.
To assess changes in joint status scores
To assess changes in joint status scores between baseline and the final visit after 6 months of subcutaneous marstacimab injections in hemophilia patients, using the Hemophilia Early Arthropathy Detection with Ultrasound in China(HEAD-US-C).
patient preferences for subcutaneous
To evaluate patient preferences regarding treatment experience via M-PPQ after 6 months.
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