Tabulation and Presentation of Data
Data in this report are provisional. Only information from the “Initial Visit” form, required to be completed at enrollment, was used for this Report. Missing data prevent the totals for some tables from adding up to the total sample size; counts of ≤ 5 within a table cell are not presented in order to protect the confidentiality of participants. The data presented are for study visits prior to January 1, 2018 (submitted to CDC no later than August 31, 2018); information from 42 participants enrolled in December 2013 were included in this Report.
Age for the Registry is calculated by subtracting the participant’s date of birth from the date of visit and dividing by 365.
Age for the HTC PP is calculated by subtracting the year of birth from the year of visit.
BMI for the Registry is calculated using the height and weight variables for patients older than 2 years old; patients younger than 2 years of age were excluded from BMI calculations.
A CVAD is an indwelling medical device used to access the central venous circulation in order to draw blood samples or infuse factor (such as a peripherally inserted central catheters [PICC], non-tunneled central venous catheter, tunneled central venous catheter, or port).
The Registry reports participants’ primary health insurance. If an individual reported only one insurance, it was designated as their primary insurance, regardless of whether it was specifically indicated as such. If an individual reported multiple insurance plans, the plan indicated as the primary insurance was designated as such.
HTC Primary Location – the patient received care at the HTC’s primary physical location.
HTC Outreach Clinic – the patient received care at a physical location to which HTC staff have traveled, distant from the HTC’s primary location.
HTC Telemedicine Session or Telemedicine Clinic – the patient received care from HTC staff remotely via a tele-video connection.
An invasive joint procedure is one requiring the insertion of an instrument or device into the joint space.
Severe – less than 1% of clotting factor activity in blood.
Moderate – 1%–5% of clotting factor activity in blood.
Mild – more than 5% of clotting factor activity in blood. Patients with more than 40% of clotting factor activity in the blood were also included in the mild category.
Treatment regimen is categorized as a patient practicing 1 of 5 treatment types listed at the time of Registry enrollment.
“Prophylaxis-continuous” if a patient uses any treatment product on a regular basis to prevent any and all bleeds or to maintain tolerance to factor, or both (even if adherence to the regimen isn’t perfect), and is expected to do so indefinitely.
“Prophylaxis-continuous with bypassing agents plus ITI” if the patient uses FVIII or FIX concentrates on a regular basis for the primary purpose of eradicating an inhibitor AND uses bypassing agents on a regular basis to prevent any and all bleeds (even if adherence to the regimen isn’t perfect), and is expected to do so indefinitely.
“Prophylaxis-event-based, short-term, or intermittent prophylaxis” if a patient uses treatment product to prevent anticipated bleeds associated with an event (such as a dental procedure or surgery) or an activity (such as a sports event) on an intermittent basis, repeatedly, over a short period of time, or on a single occasion OR on a regular basis to prevent any and all bleeds, and is expected to do so for an extended period of time (weeks to months), but not indefinitely (such as a sports season).
“Immune tolerance induction” if the patient uses FVIII or FIX concentrates on a regular basis for the primary purpose of eradicating an inhibitor (even if adherence to the regimen isn’t perfect) AND does not also use bypassing agents for continuous prophylaxis.
“Episodic (on-demand)” if the patient uses treatment product ONLY for treatment of existent bleeds.
Treatment product is categorized based on products used at the time of Registry enrollment or those products used in the last 12 months or since the participant’s last UDC visit.
Non-plasma products – products not derived from human blood or plasma, including desmopressin and antifibrinolytics.
Blood products – Non-clotting factor concentrates derived from human blood, including but not limited to whole blood and cryoprecipitate.
Plasma-derived clotting factor concentrates – A clotting factor concentrate that is industrially derived from human plasma.
Recombinant clotting factor concentrates – A clotting factor concentrate that is produced in a laboratory, including both standard half-life products and extended half-life products.
No products – Used no products to treat bleeding disorder since their last UDC visit or in the last 12 months.
Other products – Products such as Hemlibra®, factor VIII clotting factor concentrates of unspecified derivation, thrombin (bovine, human, or recombinant origin), and products reported as “study drug” or “investigational drug”.
Figure- or Table-specific Comments
Table 6 & Figures 12-16
- HIV and hepatitis B and C infection status were based on the status reported on the participant’s “Initial Visit” form, and were not based on laboratory testing performed for Community Counts.
Pages in this Report
- Highlights & Acknowledgements
- Geographic Distribution of Registry Participants
- Diagnosis & Severity
- Registry Characteristics
- Weight Status
- Health Insurance Coverage
- Viral and Vaccination History
- Healthcare Utilization and Absenteeism
- Family History and Genetic Mutation
- Procedures and Comorbid Conditions
- ›Technical Notes
- Participating HTCs