This is an observational research study to find out if there is a difference in the way children with moderate or severe hemophilia A, treated on two different types of factor replacement, form a clot and also evaluate if they develop tiny bleeds within the joint and subsequently early joint changes when receiving extended half-life factor VIII.
Participants with moderate or severe HA who are on prophylaxis with either weekly, biweekly, or every 4-weeks emicizumab or weekly efanesoctocog alfa for at least 2 months.
>6 months of age
Participants with active FVIII inhibitor (>0.5 BU/mL)
Presence of an additional bleeding disorder other than hemophilia A
* Children (defined as persons who have not attained the legal age for consent to treatments or procedures involved in the research, under the applicable law of the jurisdiction in which the research will be conducted. In Texas the legal age is 18.) * Non-English Speakers * Patients (defined as individuals in a clinical setting with whom there is a treatment relationship)
Cohort
Prospective
Half-life factor VIII based replacement therapy
DrugChildren with moderate or severe hemophilia A being treated with prophylactic extended half-life factor VIII based replacement therapy (such as Altuviiio) as part of their hemophilia treatment to prevent spontaneous joint bleeds.
Non-FVIII based replacement therapy
DrugChildren with moderate or severe hemophilia A being treated with non-FVIII based replacement therapy (such as Hemlibra) as part of their hemophilia treatment to prevent spontaneous joint bleeds.
Thrombin Generation
To determine and compare in vivo thrombin generation parameters; endogenous thrombin potential (ETP) and peak thrombin (PT) for patients on efanesoctocog alfa or emicizumab
Hemophilia Joint Health Score
To obtain and compare HJHS in patients on efanesoctocog alfa or emicizumab
Annualized bleeding rate
To collect bleeding episodes in study participants and to calculate their respective total, treated, and joint annualized bleeding rates
Health related quality of life - CHO-KLAT
Canadian Hemophilia Outcomes-Kids Life Assessment Tool (CHO-KLAT) will be used to evaluate the impact of different treatment strategies. CHO-KLAT will be obtained and compared in study participants on efanesoctocog alfa or emicizumab.
Health related quality of life - PedHAL
PedHAL is a validated patient-reported outcome measure designed to assess self-perceived functional abilities in children with hemophilia. PedHAL will be obtained and compared in study participants on efanesoctocog alfa or emicizumab
Health related quality of life - CATCH
CATCH (Comprehensive Assessment Tool of Challenges in Hemophilia) is a validated, multi-version patient-reported outcome instrument designed to assess the real-world impact of hemophilia on individuals and their caregivers. CATCH will be obtained and compared in study participants on efanesoctocog alfa or emicizumab
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University of Texas Southwestern Medical Center
Lead sponsor
Sanofi
Collaborator
