RECRUITINGOBSERVATIONAL
Effects of Emicizumab vs. Factor VIII Prophylaxis on Joint and Bone Health in Severe Hemophilia A
About This Trial
The investigators propose to study longitudinal joint and bone density changes in patients with severe Hemophilia A. Per current standard of care, most patients are on prophylactic FVIII replacement therapy intravenously several times weekly with a goal of keeping the trough \>1% FVIII. Recent phase 3 data suggest superior bleed protection with emicizumab prophylaxis every 1-2 weeks. It is the purpose of this study to longitudinally assess joint health and bone density over 3 years and to compare the effect of routine factor VIII prophylaxis with emicizumab prophylaxis.
Who May Be Eligible (Plain English)
Who May Qualify:
- Male gender
- Severe hemophilia A (factor VIII \< 1%)
- Age ≥ 16 year
- Either on prophylaxis with factor VIII or emicizumab with the intention to stay on the current regimen for the next 3 years
- Willing and able to give written willing to sign a consent form/assent
- Willing to undergo MSKUS, DEXA scan +/- collection of blood sampling for repository biomarkers
- Willing to come in for baseline and 3 yearly visits
- Willing to answer phone survey for bleeding and safety every 3 months
Who Should NOT Join This Trial:
- Current FVIII inhibitor of \> 0.6 BU
- Unable to take FVIII replacement
- Other known bleeding disorder
- Other rheumatologic disorder affecting joints
- Other known neuromotor defect (making physical exam difficult)
Always talk to your doctor about whether this trial is right for you.
Original Eligibility Criteria
View original clinical language
Inclusion Criteria:
* Male gender
* Severe hemophilia A (factor VIII \< 1%)
* Age ≥ 16 year
* Either on prophylaxis with factor VIII or emicizumab with the intention to stay on the current regimen for the next 3 years
* Willing and able to give written informed consent/assent
* Willing to undergo MSKUS, DEXA scan +/- collection of blood sampling for repository biomarkers
* Willing to come in for baseline and 3 yearly visits
* Willing to answer phone survey for bleeding and safety every 3 months
Exclusion Criteria:
* Current FVIII inhibitor of \> 0.6 BU
* Unable to take FVIII replacement
* Other known bleeding disorder
* Other rheumatologic disorder affecting joints
* Other known neuromotor defect (making physical exam difficult)
Treatments Being Tested
OTHER
assessment of joint health and bone density
We propose to study longitudinal joint and bone density changes in patients with severe Hemophilia A.
Locations (4)
Orthopedic Hemophilia Treatment Center
Los Angeles, California, United States
Hemophilia and Thrombosis Treatment Center, University of California, San Diego
San Diego, California, United States
The University of Texas Health Science Center at San Antonio
San Antonio, Texas, United States
Washington Center for Bleeding Disorders at Washington Institute for Coagulation
Seattle, Washington, United States