US Results from the Hemophilia Experiences, Results and Opportunities (HERO) Study Affirm
Hemophilia Treatment Centers (HTCs) are Central to Hemophilia Care, Yet Financial Concerns
and Distance May Limit Access to Treatment for Some Patients
Nugent D,1 Guelcher C,2 Rice M,3 Forsyth A,4 Iorio A,5 and Cooper DL6
1Children’s
Hospital of Orange County, Orange, CA; 2Children’s National Medical Center, Washington, DC; 3National Hemophilia Foundation, New York, NY; 4RUSH Hemophilia and Thrombophilia Center, Chicago, IL; 5McMaster University, Hamilton, Ontario, Canada; 6Novo Nordisk Inc., Princeton, NJ.
Table 1 HTC visits per year for PWH and Parents
Introduction



Psychosocial issues affect the ability of persons with hemophilia (PWH) and their
families to lead the lives that they desire.
The Hemophilia Experiences, Results and Opportunities (HERO) initiative aimed to
increase the understanding of psychosocial issues impacting people with
moderate-severe hemophilia and was led by an International Advisory Board that
included healthcare professionals, patients, and caregivers.
HERO began with a systematic literature review1 and 150 interviews with patients,
parents, and healthcare professionals to identify issues for this quantitative study.
PWH
Mean
Median
IQR



To describe access to care and utilizations of Hemophilia Treatment Centers
(HTCs) in PWH and parents responding to HERO in the United States (US).

Surveys were developed specifically for either adult PWH (≥18 years) or parents of
children with hemophilia (CWH) (<18 years) who were either currently receiving
factor replacement or had spontaneous bleeding into one or more joints within the
previous 12 months.
Ten countries participated (Algeria, Argentina, China, Canada, France, Germany,
Italy, Spain, United Kingdom, and US) between June 2011 and February 2012.
Following Internal Review Board/Ethics Committee approvals, US PWH and
parents were recruited through the National Hemophilia Foundation eNote
distribution and Facebook page, and informed consent was obtained.
Surveys in the US and most other countries (except Algeria) were completed online
in about 45 minutes.
Data presented reflects US respondents encompassing 189 of 675 total PWH and
190 of 561 total parents.
Mean/median perception of hemophilia control (0-10, 10 = extremely well
controlled) was 7.66/8 for PWH and 8.56/9 for parents, and was lower with
inhibitors both for PWH (6.53/7 vs 8.01/8) and parents (6.33/6 vs 8.8/9) (Figure 1).
Figure 1 Perceived disease control for US PWH and parents
Parents (n=190)
10%
1 - Not at all controlled
20%
2
30%
3
4
40%
5
6
50%
7
8
60%
9
70%
80%
90%
100%
10 - Extremely well controlled
HTC visits in the last 12 months were more common in children than adults (mean
3.77 vs 1.89), and patients with inhibitors (mean 3.46) (Table 1).
Access to factor due to availability or affordability

All
WOI
HWI
OD
PPX
1.89
1.62
3.46
3.77
3.69
4.63
3.75
3.86
1
1
2.5
2
2
3
2.5
2.0
1-2
1-2
1.5-4.5
1-3.5
1-3
1.5-7
1-5
1-3
77
74
65
Difficulty with health insurance
Could not personally afford it
38
21
2
2
High cost/expensive treatment
Lack of supply at the hospital
27
9
Lack of supply throughout the country
Unpredicability of supply
Physician reluctant to prescribe it
7
16
17
14
13
0
PWH (n=48)
Parents (n=43)
23
20
Some PWH/parents (25%/23%) reported difficulty obtaining factor or concerns about
availability or affordability in the prior 5 years.
In contrast to issues identified in other countries, the predominant reasons in the US
were financial, including insurance and affordability (Figure 2).

PWH and parents consistently identified hematologists and hemophilia nurses as key
individuals involved in managing hemophilia (Figures 4 and 5), and were generally
very satisfied with the care provided.
PWH and parents less commonly reported social workers and physical therapists as
being involved in managing hemophilia, but were satisfied with care provided.
Figure 4 Access to and satisfaction with healthcare
professionals involved in managing hemophilia for PWH
Type of HCP
% seeing
Hematologist
84
86
Hemophilia nurse
75
87
Social worker
43
Physiotherapist
40
89
Counselor/Psychologist/
Psychotherapist
32
90
General practitioner/family doctor
20
Complementary therapist
(e.g. acupuncture, massage)
5
40
60
Respondents (%)
80
100
Access to Hemophilia Treatment Centers

Only 22%/22% of PWH/parents reported it very/quite difficult to access the HTC.

Of those reporting difficulty, the most consistent themes were around travel, although
limited HTC hours was cited by many PWH and parents (Figure 3).
71
45
  HERO’s US adult PWH and parents of CWH generally perceive
good control of hemophilia.
  Of concern, 23% to 25% report difficulty with access or affordability
primarily due to financial factors. Follow-up will be important as
healthcare reforms are implemented.
79
67
PWH satisfied (%)
100%
45
40
29
21
26
Sometimes there is no one available to take me
PWH (n=42)
Parents (n=42)
7
31
I am unable to take time off
26
0
20
40
60
Respondents (%)
80
100
  Even though there are ~130 HTCs in the US, 22% of PWH and
parents still have difficulty accessing HTCs primarily due to
distance/travel.
Figure 5 Access to and satisfaction with healthcare
professionals involved in managing hemophilia for parents
  The HTC hematologist/nurse remain at the center of
comprehensive care, with high PWH/parent satisfaction.
Type of HCP
% seeing
  Reports of social work and physical therapy contact as part of
management of hemophilia were lower than expected.
Hematologist
95
Hemophilia Nurse
83
Social Worker
49
95
‒  This may relate to variability or part-time social worker/physical
therapist staffing or narrow PWH/parent definitions of
“management” that do not account for routine PT assessment,
psychosocial issues management, and insurance support.
91
86
50
The center/clinic has limited hours


Most PWH/parents reported being very/somewhat knowledgeable about
hemophilia (88%/97%), less so for PWH with inhibitors (56% vs 98%),
and parents of CWH with inhibitors (72% vs 99%).
The majority of PWH/parents belonged to a patient organization (67%/77%).
PWH/parents reported their main sources of information included:
–  Hematologist (71%/86%)
–  Hemophilia nurse (67%/77%)
–  Patient associations (46%/71%)
–  Non-pharmaceutical websites (48%/64%)
–  Physical therapist (32%/9%)
The single source considered most useful by PWH/parents was the hematologist
(31%/23%) or nurse (11%/23%).
Conclusions
81
The travel is expensive


72
50%
It takes a long time to get to the center
Sources and usefulness of information
70
It is a long way to travel
0%

HWI
Figure 3 Difficulty in access to the HTC for
US PWH and parents
PWH (n=189)

WOI
Any financial (insurance or cost)
Results

All
Figure 2 Difficulty in obtaining factor in the past 5 years for
US PWH and parents
Methods


Parents
WOI, hemophilia without inhibitors; HWI, hemophilia with inhibitors; OD, on demand; PPX, prophylaxis
Objective

Relationship with healthcare professionals managing hemophilia
Physiotherapist
24
General practitioner/family doctor
24
85
Counselor/Psychologist/
Psychotherapist
18
85
Complementary therapist
(e.g. acupuncture, massage)
6
Presented at NHF’s 64th Annual Meeting; Orlando, Florida; November 8-10, 2012.
  Expanded roles for allied health professionals addressing
psychosocial issues at the HTC deserve exploration.
89
  Hematologists, hemophilia nurses, and associations are reported as
PWH and parents’ main source of hemophilia information.
100
50%
Parents satisfied (%)
100%
Reference
1. Cassis FR et al. Haemophilia. 2012;18(3):e101-114.