POCUS: Hemostatic Potential and Joint Health in Patients with Severe Hemophilia A on Novel Replacement Therapies
Study ID: STU-2020-1378
Summary
Study will be prospective, randomized control trial to avoid selection bias in which each patient will be randomly assigned to receive either extended half-life factor Viii based replacement therapy or non-FViii based replacement therapy, which are both standard of care treatment for persons with Hemophilia a. We considered but rejected two alternate study designs: single arm parallel study and a cross-over study. Single arm parallel study was mainly rejected in order to eliminate bias, and cross-over study was rejected since we expect to follow these subjects longitudinally evaluating for early joint damage, and we would not be able to precisely determine which intervention lead to this outcome. Study participants who wish to switch therapies while on trial will undergo exclusion from randomization in the trial, but instead continue to be followed in the study as a non-interventional, observational group or [Quote]parallel cohort[Quote] and will still undergo research assessments
Specific aim 1: To evaluate differences in hemostatic potential and bleeding rates on prophylactic FViii-based versus non-FViii-based replacement therapies in PwHa without evidence of hemophilic target joints Specific aim 2: To prospectively identify early joint-health markers associated with bleeding on prophylactic FViii-based versus non-FViii-based replacement therapies in PwHa without evidence of hemophilic target joints