This is a national, multicenter, retrospective/prospective, observational study in Taiwan designed to assess effectiveness, safety, and usage of efanesoctocog alfa prophylaxis treatment in hemophilia A participants. The data related to efanesoctocog alfa effectiveness, safety and usage will be recorded prospectively during routine visits for up to 5 years following enrollment initiation and the retrospective data will be collected at least 12 months and up to 24 months prior to efanesoctocog alfa initiation. Joint imaging data will be collected in centers performing Joint U/S and/or MRI (≥6 years old). At least 12 months of retrospective data will also be collected from medical records, as available. Prospectively collected data will be recorded at routine clinical visits during a five-year follow-up period. The end of study is defined as the last participant's last visit. No intervention will be administered, and no study related visits are required.
Study Type
OBSERVATIONAL
Enrollment
100
This study will not administer any treatment, only observe the treatment as prescribed in real-world clinical practices.
Investigational Site Number : 1580008
Changhua, Taiwan
RECRUITINGInvestigational Site Number : 1580009
Kaohsiung City, Taiwan
RECRUITINGInvestigational Site Number : 1580010
Kaohsiung City, Taiwan
RECRUITINGInvestigational Site Number : 1580005
Taichung, Taiwan
RECRUITINGInvestigational Site Number : 1580006
Taichung, Taiwan
RECRUITINGInvestigational Site Number : 1580007
Taichung, Taiwan
RECRUITINGInvestigational Site Number : 1580001
Taipei, Taiwan
RECRUITINGInvestigational Site Number : 1580003
Taipei, Taiwan
RECRUITINGInvestigational Site Number : 1580002
Taipei, Taiwan
RECRUITINGInvestigational Site Number : 1580004
Taoyuan, Taiwan
RECRUITINGChange from baseline in total scores of the Hemophilia Joint Health Score (HJHS) for all joints
The HJHS summarizes joint health by providing a clinical score in the domain of body structure and function (i.e., impairment), of the joints most commonly affected by bleeding in hemophilia: (left ankle, right ankle, left elbow, right elbow, left knee, right knee). The HJHS consists of assessments of swelling, duration of swelling, muscle atrophy, crepitus on motion, flexion loss, extension loss, joint pain and strength for elbows, knees and ankles and a global gait score. A higher score indicates worse joint health.
Time frame: At 1, 2, 3, 4 and 5 years
Change from baseline in annualized joint bleeding rate (AjBR) for treated and untreated bleeds
The AjBR is defined as the number of joint bleeding episodes occurring during the treatment period divided by the duration of the treatment period in days multiplied by 365.25. All types of joint bleeding episodes (spontaneous, traumatic, and type unknown) will be included in determining the annualized number.
Time frame: At 1, 2, 3, 4 and 5 years
Number of target joint development, resolution and/or recurrence
Time frame: At 1, 2 ,3, 4 and 5 years
Annualized bleeding rate (ABR) by type and location for treated and all (treated and untreated) bleeds
Time frame: At 1, 2, 3, 4 and 5 years
ABR for all bleeding episodes, including untreated bleeding episodes
Time frame: At 1, 2, 3, 4 and 5 years
Percentage of participants with zero joint bleeds
Time frame: At 1, 2, 3, 4 and 5 years
Percentage of bleeding episodes treated with a single injection of efanesoctocog alfa
Time frame: At 1, 2, 3, 4 and 5 years
Number of injections and doses of efanesoctocog alfa to treat a bleeding episode
Time frame: At 1, 2, 3, 4 and 5 years
Annualized factor consumption per participant (IU/kg) assessed by prescription during the follow-up period
Time frame: At 1, 2, 3, 4 and 5 years
Annualized injection frequency per participant (assessed by prescription) during the followup period
Time frame: At 1, 2, 3, 4 and 5 years
Treatment adherence (%) as judged by the physician during the follow-up period
Time frame: At 1, 2, 3, 4 and 5 years
Occurrence of a change in treatment regimen at baseline and follow-up
Time frame: 5 years
Change from baseline in Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) total/domain scores in all joints
Time frame: Every 6 months for 5 years
Change from baseline in joint status on functional HJHS
Time frame: At 1, 2, 3, 4 and 5 years
Number and percentage of joints with no prior joint damage at baseline, without joint damage on joint ultrasound
Time frame: At 5 years
Changes from baseline in HEAD-US synovitis domain
Time frame: Every 6 months for 5 years
Change from baseline in MRI score
Time frame: At 1, 2, 3, 4 and 5 years
Number and percentage of participants without joint damage on MRI
Time frame: At 1, 2, 3, 4 and 5 years
Number and percentage of joints with no prior joint damage at baseline, without joint damage on MRI
Time frame: At 1, 2, 3, 4 and 5 years
Hemostatic response/physician reported during peri-operative period for surgery (major, minor) with efanesoctocog alfa
Time frame: 5 years
Number of injections and dose per injection required to maintain hemostasis during perioperative period for surgery (major, minor)
Time frame: 5 years
Total efanesoctocog alfa consumption (IU) during perioperative period for surgery (major, minor)
Time frame: 5 years
Number and type of blood component transfusions used during perioperative period for surgery
Time frame: 5 years
Estimated blood loss (mL) during perioperative period for major surgery
Time frame: 5 years
Number of transfusions required for surgery (intraoperative and post-operative period)
Time frame: 5 years
Duration of hospitalization (major, minor)
Time frame: 5 years
Occurrence of adverse events (AEs), serious adverse events (SAEs) and adverse events of special interest (AESI)
Time frame: 5 years
Development of inhibitors (neutralizing antibodies directed against factor FVIII as determined via the Nijmegen modified Bethesda assay)
Time frame: 5 years
Change from baseline in HAL (haemophilia activities list) for adult participants only
HAL contains 42 items across 7 domains: lying down/ sitting/ kneeling/ standing, functions of the legs, functions of the arms, use of transportation, self-care, household tasks and leisure activities and sports. Items are scored on a 6-point Likert scale ('impossible', 'always', 'usually', 'sometimes', 'almost never', 'never'), with a 'not applicable' option for some items. A summary score as well as component scores (upper extremity, basic lower extremity and complex lower extremity) can be calculated. The scores are converted to a normalized score from 0 to 100, where higher scores represent a better functional status.
Time frame: At 1, 2, 3, 4 and 5 years
Change from baseline in PROMIS pain intensity 3a questionnaire for adult participants only
The PROMIS Pain Intensity instrument assesses how much a person hurts. The Pain Intensity short forms are universal rather than disease specific. The 1a and the first two items within the PROMIS Scale- Pain Intensity 3a short form assesses pain intensity over the past seven days. The version 2.0 of Pain Intensity 3a instruments are available for adults (ages 18+), and the version v1.0 of Pediatric Pain Intensity 1a self-report (ages 8-17) will be used for this study.
Time frame: At 1, 2, 3, 4 and 5 years
Change from baseline in PROBE full questionnaire for adult participants only
The PROBE questionnaire assesses participant reported outcomes in people with hemophilia and people without a bleeding disorder. The PROBE questionnaire is comprised of four major sections, including demographic data, general health problems, hemophilia-related health problems and health-related quality of life. It combines generic and disease-specific outcomes in 29 questions.
Time frame: At 1, 2, 3, 4 and 5 years
Change from baseline in Ped-HAL (haemophilia activities list) for adolescent participants only (≥ 12 to <18 years)
PedHAL contains 53 items across 7 domains: sitting/ kneeling/ standing, functions of the legs, functions of the arms, use of transportation, self-care, household tasks and leisure activities and sports. It consists of a participant version (8-18 years) and parent version (4-17 years). Items are scored on a 6-point Likert scale ('impossible', 'always', 'usually', 'sometimes', 'almost never', 'never'), with a 'not applicable (N/A)' scoring option for all items. A summary score as well as domain scores can be calculated. The scores are converted to a normalized score from 0 to 100, where higher scores represent a better functional status.
Time frame: At 1, 2, 3, 4 and 5 years
Change from baseline in PROMIS pain intensity 1a questionnaire for adolescent participants only (≥ 12 to <18 years)
The PROMIS Pain Intensity instrument assesses how much a person hurts. The Pain Intensity short forms are universal rather than disease specific. The 1a and the first two items within the PROMIS Scale- Pain Intensity 3a short form assesses pain intensity over the past seven days. The version 2.0 of Pain Intensity 3a instruments are available for adults (ages 18+), and the version v1.0 of Pediatric Pain Intensity 1a self-report (ages 8-17) will be used for this study.
Time frame: At 1, 2, 3, 4 and 5 years
Change from baseline in PROBE full questionnaire for adolescent participants only (≥ 12 to <18 years)
The PROBE questionnaire assesses participant reported outcomes in people with hemophilia and people without a bleeding disorder. The PROBE questionnaire is comprised of four major sections, including demographic data, general health problems, hemophilia-related health problems and health-related quality of life. It combines generic and disease-specific outcomes in 29 questions.
Time frame: At 1, 2, 3, 4 and 5 years
Change from baseline in Ped-HAL (pediatric haemophilia activities list) for pediatric participants only (12 years and younger)
PedHAL contains 53 items across 7 domains: sitting/ kneeling/ standing, functions of the legs, functions of the arms, use of transportation, self-care, household tasks and leisure activities and sports. It consists of a participant version (8-18 years) and parent version (4-17 years). Items are scored on a 6-point Likert scale ('impossible', 'always', 'usually', 'sometimes', 'almost never', 'never'), with a 'not applicable (N/A)' scoring option for all items. A summary score as well as domain scores can be calculated. The scores are converted to a normalized score from 0 to 100, where higher scores represent a better functional status.
Time frame: At 1, 2, 3, 4 and 5 years
Number of hospitalizations excluding surgery and admission/discharge diagnoses
Time frame: Change from baseline at 1, 2, 3, 4 and 5 years
Number of hospitalization days
Time frame: Change from baseline at 1, 2, 3, 4 and 5 years
Number of intensive care unit (ICU) stays
Time frame: Change from baseline at 1, 2, 3, 4 and 5 years
Number of emergency room visits
Time frame: Change from baseline at 1, 2, 3, 4 and 5 years
Number of outpatient visits
Time frame: Change from baseline at 1, 2, 3, 4 and 5 years
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