Download Supplemental Methods: Access to diagnosis and clinical care. Age

Supplemental Methods:
Access to diagnosis and clinical care. Age at diagnosis was categorized as either at birth
or sometime after birth, and age at first HTC visit as ≤2 years old or >2 years old. HTC
staff categorized participants’ utilization of the HTC as frequent if visits were made at
least once annually. Clinic records were used to categorize health insurance status at the
most recent visit as commercial health insurance (e.g. employer based), Medicaid
(government program for the financially needy) Medicare (government program for the
elderly or disabled of any age), Uninsured, or other insurance.
The treatment regimen was categorized as either prophylaxis if, during the year preceding
the visit, factor infusions were used on a regular schedule with the intention to prevent
any and all bleeding and to continue indefinitely; or episodic if infusions were performed
in response to a bleeding episode. Data were collected on whether or not factor was
infused at home and, if so, the participant’s age at initiation of home infusion, categorized
for this analysis as either ≤ 6 years or > 6 years old.
Social and physical functioning was assessed by measures of employment and activity
level. Participants were designated as a student or employed if they reported current
attendance at school and/or either part-time or full-time employment. If not employed,
their status was designated as retired, permanently disabled, or other. Participants were
also asked about the number of days absent from work or school during the last year
because of either upper or lower extremity joint problems. Days missed were designated
as ≤11 days versus >11 days corresponding to the excessive school days lost category
used by the National Survey of Children with Special Healthcare Needs.1-3
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Activity Level and Mobility: Participants were asked to rate their current overall activity
level as: 1) unrestricted school/work and recreation activities; 2) full school/work but
limited recreation activities; 3) limited school/work and recreation activities; 4) limited
school/work, recreational and self-care activities; or 5) requires assistance for
school/work/self-care and unable to participate in recreational activities. Limitations
could be the result of either pain, loss of motion or weakness. For this analysis, a
participant with a self-reported activity level of categories 3 – 5 was considered to have
activity limitation. Participants were asked to categorize as “never”, “intermittently”, or
“always” their use in the preceding year of a cane, crutches, or walker and separately
their use of a wheelchair for mobility.
Joint bleeding: The number of bleeding episodes in the joints during the last six months
was assessed, based on either infusion logs or participant self-report. Joint bleeding
episodes were categorized for analytic purposes as either <5 or ≥ 5 episodes and also as
either ≤2 episodes or >2 episodes. Target joint was defined as a joint with recurrent
bleeding on four or more occasions in the past 6 months.
Treatment-related complications: Liver disease, Hepatitis B and C Viruses, HIV, and
inhibitors were assessed from clinical records and blood test results. Signs or symptoms
of liver disease since the last clinic visit included jaundice, ascites, varices or other or the
presence of any elevated liver enzyme (alanine or aspartate transaminase). Information
about whether antiviral pharmacologic therapy had ever been administered for
participants with HCV infection was collected from all UDC visits. CDC laboratory
provided test results for infection with or previous exposure to HBV, HCV and HIV.
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Inhibitor development was recorded if an inhibitory antibody >1 Bethesda Unit was
detected by the HTC in the interim since the preceding annual HTC visit.
Mortality: HTCs reported mortality by date and single attributable cause of death.
1.
National Survey of Children with Special Health Care Needs 2009/10. Child and
Adolescent Health Measurement Initiative, Data Resource Center on Child and
Adolescent Health website; childhealthdata.org; accessed September 2015.
2.
Centers for Disease Control and Prevention. Workplace Health Promotion:
Increase Productivity. Vol. 2014; 2013.
3.
Blackwell D, Lucas J, Clarke T. Summary health statistics for U.S. adults:
National Health Interview Survey, 2012. In: National Center for Health Statistics. Vol.
Vital Health Stat 10. Washington, DC: Government Printing Office; 2014.
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