This is a prospective, observational, multi-center longitudinal cohort study to describe the real-world effectiveness, safety and treatment usage of efanesoctocog alfa in patients with hemophilia A treated per standard of care in the US and Japan. Patients will be enrolled in the study after the introduction of efanesoctocog alfa in the hemophilia treatment landscape in each study country. Decision to initiate treatment with commercially available efanesoctocog alfa will be made by the treating physician independently from the decision to include patients in the study. No study medication is provided. The data related to efanesoctocog alfa effectiveness, safety and usage will be collected prospectively during routine visits (expected annual/semi-annual visits) for up to 5 years following enrollment /treatment initiation.
Study Type
OBSERVATIONAL
Enrollment
200
Given per investigator's discretion
Banner MD Anderson Cancer Center- Site Number : 8400008
Gilbert, Arizona, United States
Loma Linda University- Site Number : 8400015
Loma Linda, California, United States
Orthopedic Institute for Children- Site Number : 8400004
Los Angeles, California, United States
The Center for Inherited Blood Disorders- Site Number : 8400001
Orange, California, United States
University of Colorado Hemophilia and Thrombosis Center- Site Number : 8400019
Aurora, Colorado, United States
Change from baseline in Annualized joint bleeding rate (AjBR) for treated bleeds
Data will be reported for the effectiveness of efanesoctocog alfa prophylaxis on clinical joint status over 5-years for prophylactic cohort
Time frame: Up to 5 years
Change from baseline in Annualized joint bleeding rate (AjBR) for all (treated and untreated) bleeds
Data will be reported for the effectiveness of efanesoctocog alfa prophylaxis on clinical joint status over 5-years for prophylactic cohort
Time frame: Up to 5 years
Number of Target joint development, resolution and/or recurrence
Data will be reported for the effectiveness of efanesoctocog alfa prophylaxis on clinical joint status over 5-years for prophylactic coho
Time frame: Up to 5 years
Change from baseline in the Hemophilia Joint Health Score (HJHS v2.1) total/domains scores
HJHS total/domain scores will be reported for prophylactic cohorts (A1 ad A2)
Time frame: At 1, 2, 3, 4, 5 years
Annual Bleeding Rate (ABR) by type for treated bleeds
Data will be reported for prophylactic cohorts (A1 and A2)
Time frame: At 1, 2, 3, 4, 5 years
ABR by type all (treated and untreated) bleeds
Data will be reported for prophylactic cohorts (A1 and A2)
Time frame: At 1, 2, 3, 4, 5 years
ABR by location for treated bleeds
Data will be reported for prophylactic cohorts (A1 and A2)
Time frame: At 1, 2, 3, 4, 5 years
ABR by location for all (treated and untreated) bleeds
Data will be reported for prophylactic cohorts (A1 and A2)
Time frame: At 1, 2, 3, 4, 5 years
ABR for all bleeding episodes (including untreated bleeding episodes)
Data will be reported for prophylactic cohorts (A1 and A2)
Time frame: At 1, 2, 3, 4, 5 years
Percentage of patients with zero joint bleeds
Data will be reported for prophylactic cohorts (A1 and A2)
Time frame: At 1, 2, 3, 4, 5 years
Annualized factor consumption per participant (IU/kg) assessed by prescription during the follow-up period.
Data will be reported for prophylactic cohorts (A1 and A2)
Time frame: At 1, 2, 3, 4, 5 years
Annualized injection frequency per participant (assessed by prescription) during the follow- up period
Data will be reported for prophylactic cohorts (A1 and A2)
Time frame: At 1, 2, 3, 4, 5 years
Treatment adherence (%) as judged by the physician during the follow-up period
Data will be reported for prophylactic cohorts (A1 and A2)
Time frame: At 1, 2, 3, 4, 5 years
Number of injections of efanesoctocog alfa to treat a bleeding episode.
Data will be reported for prophylactic cohorts (Sub Cohorts A1 and A2)
Time frame: At 1, 2, 3, 4, 5 years
Total dose of efanesoctocog alfa to treat a bleeding episode.
Data will be reported for prophylactic cohorts (Sub Cohorts A1 and A2)
Time frame: At 1, 2, 3, 4, 5 years
Number of injections of efanesoctocog alfa to treat a bleeding episode
Data will be reported for On-demand cohort
Time frame: At 1, 2, 3, 4, 5 years
Total dose of efanesoctocog alfa to treat a bleeding episode
Data will be reported for On-demand cohort
Time frame: At 1, 2, 3, 4, 5 years
Percentage of bleeding episodes treated with a single injection of efanesoctocog alfa.
Data will be reported for On-demand cohort
Time frame: At 1, 2, 3, 4, 5 years
Occurrence of a change in treatment regimen (on-demand to prophylactic or prophylactic to on-demand) at baseline and follow-up.
Data will be reported for On-demand cohort
Time frame: At 1, 2, 3, 4, 5 years
Change from baseline in Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) total/domain scores
HEAD-US total/domain scores will be reported for joint imaging sub cohort
Time frame: At 1, 2, 3, 4 and 5 years
Change from baseline in Hemophilia Early Arthropathy Detection with Joint Tissue Activity and Damage exam (JADE) musculoskeletal ultrasound (MSKUS) (JADE MSKUS)
Data will be reported for consenting participants from the joint imaging sub-cohort
Time frame: At 1, 2, 3, 4 and 5 years
Change from baseline in synovial hypertrophy by change in mm thickness AND/OR HEAD-US synovitis domain, AND/OR by JADE MSKUS synovial hypertrophy +/- power doppler signal
Data will be reported from the consenting participants from the joint imaging sub-cohort
Time frame: At 6 months, 1, 2, 3, 4 and 5 years
Occurrence of adverse events (AEs) and serious adverse events (SAEs)
The safety and tolerability of efanesoctocog alfa over 5 years evaluated in all participants
Time frame: Over 5 year period
Development of inhibitors (neutralizing antibodies directed against factor FVIII as determined via the Nijmegen modified Bethesda assay.
The safety and tolerability of efanesoctocog alfa over 5 years assessed in all participants
Time frame: Over 5 year period
Change from baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) pain intensity 3a questionnaire (adults)
Effectiveness of efanesoctocog alfa assessed per participants' PROs in all participants
Time frame: At 3 months, 6 months, 1, 2, 3, 4 and 5 years
Change from baseline in Canadian Hemophilia Outcomes-Kids' Life Assessment Tool (CHO-KLAT) 3.0 (boys ≤18 years old) questionnaire (including Parent Proxy)
Effectiveness of efanesoctocog alfa assessed per participants' PROs in all participants
Time frame: At 1, 2, 3, 4 and 5 years
Change from baseline in the occurrence of inpatient and outpatient visits, and length of hospital stay related to hemophilia A
Healthcare resource use (HCRU) in efanesoctocog alfa related to hemophilia A over a 5-year period
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.
Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Time frame: Over a 5 year period
Number of injections required to maintain hemostasis during perioperative period for surgery (major, minor)
Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Time frame: Over 5 year period
Dose per injection required to maintain hemostasis during perioperative period for surgery (major, minor)
Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Time frame: Over 5 year period
Total efanesoctocog alfa consumption (IU) during perioperative period for surgery (major, minor)
Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Time frame: Over 5 year period
Number of blood component transfusions used during perioperative period for surgery
Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Time frame: Over 5 year Period
Type of blood component transfusions used during perioperative period for surgery
Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Time frame: Over 5 year period
Estimated blood loss (ml) (intraoperative and post-operative period) for major surgery
Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Time frame: Over 5 year period
Number of transfusions required for surgery (intraoperative and post-operative period)
Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Time frame: Over 5 year period
Duration of hospitalization (major, minor)
Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period.
Time frame: Over 5 years period
Dose per injection required to maintain hemostasis during perioperative period for surgery (major, minor)
Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period for On-demand Cohort
Time frame: Over a 5 year period
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William A. Shands Hospital at the University of Florida- Site Number : 8400032
Gainesville, Florida, United States
Johns Hopkins All Childrens Hospital - Outpatient Care Center - PIN- Site Number : 8400025
St. Petersburg, Florida, United States
Rush University Medical Center -1725 W Harrison St- Site Number : 8400014
Chicago, Illinois, United States
The Bleeding and Clotting Disorders Institute- Site Number : 8400005
Peoria, Illinois, United States
Indiana Hemophilia and Thrombosis Center- Site Number : 8400013
Indianapolis, Indiana, United States
...and 18 more locations