This is a Phase 4, multi-center, observational study conducted in patients aged 12 to 50 years with moderate or severe hemophilia A who are newly starting prophylaxis with efa in the US and Japan. This study aims to enroll 35 patients.
Study Type
OBSERVATIONAL
Enrollment
3
All treatment decisions must be made independent of study participation and study-related visits/assessments are not intended to inform treatment decisions. No study medication is provided as part of study participation.
Bleeding and Clotting Disorders Institute
Peoria, Illinois, United States
Proportion of total goals achieved
Reported as number of individual goals with a raw GAS score of 0, +1, or +2 divided by the total number of goals set
Time frame: Month 3
Proportion of total goals achieved
Time frame: Month 6
Proportion of total goals achieved
Time frame: Month 9
Proportion of total goals achieved
Time frame: Month 12
Proportion of patients who reached a +2 or +1 on at least one goal
Each goal will be ranked in a 5-point Likert scale ranging from +2 to -2 with +2 indicating a better goal achievement.
Time frame: Month 3
Proportion of patients who reached a +2 or +1 on at least one goal
Time frame: Month 6
Proportion of patients who reached a +2 or +1 on at least one goal
Time frame: Month 9
Proportion of patients who reached a +2 or +1 on at least one goal
Time frame: Month 12
Overall GAS score
Goal Attainment Scaling (GAS) is a patient-centric clinician-reported outcome measure utilized to select and scale goals, with higher scores indicating greater goal attainment. An overall GAS score utilizing all goals set by a patient at each time point of interest will be calculated.
Time frame: Month 3
Overall GAS score
Goal Attainment Scaling (GAS) is a patient-centric clinician-reported outcome measure utilized to select and scale goals, with higher scores indicating greater goal attainment. An overall GAS score utilizing all goals set by a patient at each time point of interest will be calculated.
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Time frame: Month 6
Overall GAS score
Goal Attainment Scaling (GAS) is a patient-centric clinician-reported outcome measure utilized to select and scale goals, with higher scores indicating greater goal attainment. An overall GAS score utilizing all goals set by a patient at each time point of interest will be calculated.
Time frame: Month 9
Overall GAS score
Goal Attainment Scaling (GAS) is a patient-centric clinician-reported outcome measure utilized to select and scale goals, with higher scores indicating greater goal attainment. An overall GAS score utilizing all goals set by a patient at each time point of interest will be calculated.
Time frame: Month 12
Frequency of selection of category of physical activity
Categories of the goals selected from the goal inventory
Time frame: Baseline
Monthly average of daily step count
The monthly average of daily step count (recorded by physical tracker) will be calculated using the total steps recorded in a 30-day period, accounting for the number of compliant days in that period.
Time frame: From Baseline to Month 12
Monthly average of daily activity count
Activity count includes: time spent in x, y, and z axes, vector magnitude counts, estimated calories, wear time, time spent in locomotion and non-locomotion. Counts will be averaged across the month.
Time frame: From Baseline to Month 12
Monthly average of daily Metabolic equivalent of tasks (MET)
Estimated by the physical activity tracker
Time frame: From Baseline to Month 12
Monthly average of daily Moderate and Vigorous Physical Activity (MVPA)
Summarized from MET data with time spent in moderate intensity, and vigorous intensity physical activity
Time frame: From Baseline to Month 12
Monthly average of daily intensity gradient
The intensity gradient is a measure of activity intensity distribution that reflects the distribution of intensity of physical activity by modeling the relationship between intensity and time.
Time frame: From Baseline to Month 12
Monthly average of weekly intensity gradient
Time frame: From Baseline to Month 12
Change in PROMIS Pain intensity score
Patient-Reported Outcome Measurement Information System (PROMIS): assessment of pain over the prior 7-day period (2 of the 3 questions) and right now (1 of the 3 questions), with patients rating their pain on a scale of 0 (had no pain) to 5 (very severe). Total score ranges from 0 to 15 and a higher score indicates worst pain intensity.
Time frame: From Baseline to Month 12
Change in HAL Score
Hemophilia Activities List (HAL) is a 42-item questionnaire to access patient ability across 7 domains: lying down/ sitting/ kneeling/ standing (8 items), functions of the legs (9 items), functions of the arms (4 items), use of transportation (3 items), self-care (5 items), household tasks (6 items), and leisure activities (7 items). Patients rate each question on a Likert scale ranging from 1 (impossible) to 6 (never). Several items have a "not applicable" option as well. There is also a visual analog scale allowing patients to self-report an overall score. A normalized total score (sum of all normalized domains) ranges from 0 (the worst possible functional status) to 100 (the best possible functional status)
Time frame: From Baseline to Month 12
Change in PROMIS pediatric Pain intensity score
Assessment of the average pain over the prior 7-day period, with patients rating their pain on a scale of 0 (no pain) to 10 (worst pain they can think of). A higher score indicates worst pain intensity.
Time frame: From Baseline to Month 12
Change in PedHAL Score
Pediatric Hemophilia Activities List (PedHAL) is a 53-item questionnaire to access patient functional ability across 7 domains: lying down/ sitting/ kneeling/ standing (10 items), functions of the legs (11 items), functions of the arms (6 items), use of transportation (3 items), self-care (9 items), household tasks (3 items), and leisure activities and sports (11 items). Patients rate each question on a Likert scale ranging from 1 (impossible) to 6 (never). Several items have a "not applicable" option as well. There is also a visual analog scale allowing patients to self-report an overall score. A normalized total score (sum of all normalized domains) ranges from 0 (the worst possible functional status) to 100 (the best possible functional status).
Time frame: From Baseline to Month 12
Change in school/work hours missed as assessed by WPAI+CIQ:HS
The Work Productivity and Activity Impairment plus Classroom Impairment Questions: Hemophilia Specific (WPAI + CIQ: HS) is a 9-item questionnaire assessing the effect of hemophilia on patients' ability to work and/or attend classes.
Time frame: From Baseline to Month 12
Change in productivity as assessed by WPAI+CIQ:HS
The Work Productivity and Activity Impairment plus Classroom Impairment Questions: Hemophilia Specific (WPAI + CIQ: HS) is a 9-item questionnaire assessing the effect of hemophilia on patients' ability to perform regular daily activities.
Time frame: From Baseline to Month 12
Change in impact on other activities as assessed by WPAI+CIQ:HS
The Work Productivity and Activity Impairment plus Classroom Impairment Questions: Hemophilia Specific (WPAI + CIQ: HS) is a 9-item questionnaire assessing the effect of hemophilia on patients' ability to perform other activities.
Time frame: From Baseline to 12 months
Mean change in total and joint domain scores of HJHS
The Hemophilia Joint Health Score (HJHS) assesses 6 index joints (left and right elbows, knees and ankles) and a global gait. For each of the 6 joints, the following items are scored: swelling (scored 0-3), duration of swelling (0-1), muscle atrophy (0-2), crepitus on motion (0-2), flexion loss (0-3), extension loss (0-3), joint pain (0-2), and strength (0-4). The HJHS provides a total score (0-124 points with higher score indicating worse joint health), joint specific scores (0-20 points), and a global gait score (0-4 points).
Time frame: From Baseline to 12 months
Monthly total and average of daily time spent in different intensity levels
Physical activity intensity defined as sedentary behavior, light intensity, moderate intensity, and vigorous intensity,
Time frame: From Baseline to Month 12
Monthly average of daily median activity count overall among moderate activities and among vigorous activities
Daily median time spent in moderate and vigorous physical activity will be averaged across the month
Time frame: From Baseline to Month 12
Monthly average of daily median MET overall among moderate activities and among vigorous activities
Time frame: From Baseline to Month 12
Monthly average of weekly power law exponent alpha
Time frame: From Baseline to Month 12
Monthly average of median bout length and percent of time of bout length > median bout length for each intensity level
Time frame: From Baseline to Month 12
Monthly average of weekly 80th, 90th, 95th, and 100th percentiles of activity count
Time frame: From Baseline to Month 12
Monthly average of weekly 80th, 90th, 95th, and 100th percentiles of MET
Time frame: From Baseline to Month 12
Monthly total step count
Time frame: From Baseline to Month 12
Monthly total activity count
Activity count includes: time spent in x, y, and z axes, vector magnitude counts, estimated calories, wear time, time spent in locomotion and non-locomotion. Counts will be totaled across the month.
Time frame: From Baseline to Month 12
Monthly total MET
Time frame: From Baseline to Month 12
Monthly total moderate or vigorous physical activity (MVPA)
Time spent in MVPA will be totaled across the month.
Time frame: From Baseline to Month 12
Weekly average of daily step count
Time frame: From Baseline to Month 12
Weekly average of daily activity count
Activity count includes: time spent in x, y, and z axes, vector magnitude counts, estimated calories, wear time, time spent in locomotion and non-locomotion. Counts will be averaged across the week.
Time frame: From Baseline to Month 12
Weekly average of daily MET
Time frame: From Baseline to Month 12
Weekly average of daily MVPA
Time spent in MVPA will be averaged across the week.
Time frame: From Baseline to Month 12
Weekly average of daily intensity gradient
Time frame: From Baseline to Month 12
Weekly intensity gradient
Time frame: From Baseline to Month 12
Number of participants with AE, SAEs, AESIs
Time frame: Baseline to end of study (12 months)
Number of participants with AEs, SAEs, AESIs related to efanesoctocog alfa
Time frame: Baseline to end of study (12 months)
Proportion of total planned doses that were missed
Time frame: From baseline to end of study (12 months)
Average time between treatment dose and physical activity
Time frame: From baseline to end of study (12 months)
Number of patients with no reported bleeds requiring treatment
Time frame: From baseline to end of study (12 months)
Average number of per patient treatment doses administered for treatment of bleeds
Time frame: From baseline to end of study (12 months)