Keywords haemophilia - haemophilia treatment centre - mobile home care - patient needs - adherence
- adults - children
Schlüsselwörter Hämophilie - Hämophilie-Zentrum - mobile häusliche Pflege - Patientenbedürfnisse -
Adherenz - Erwachsene - Kinder
Introduction
In the last few decades, state-of-the-art management of haemophilia patients practising
a life-long treatment with safe coagulation factor concentrates and offering specialized
care through haemophilia treatment centres (HTCs) resulted in a significant reduction
of morbidity and mortality.[1 ]
[2 ]
[3 ]
[4 ]
[5 ] Meanwhile, home treatment of haemophiliacs is the standard of care in developed
countries and gets increasingly implemented also in developing countries as it has
a positive impact on patients' health-related quality of life (HRQoL).[4 ]
[6 ] In the United States, approximately 70% of patients with haemophilia receive individualized
care by a HTC.[7 ] Across Europe, the availability of HTCs and the range of services provided by different
centres vary significantly between different countries.[8 ] Because of the limited number of HTCs, it is quite common that patients need to
travel a longer distance to a comprehensive care HTC providing the highest treatment
quality. This fact might generate relevant problems for patients, especially in rural
regions with limited access to public transportation systems, causing the necessity
of alternative travel organization, e.g. by a private car transport. Parents of young
boys with haemophilia seem to prefer home treatment to hospital treatment because
it was less time-consuming, less disruptive to family life and provides a greater
sense of control.[9 ] Families of underage patients or adult patients suffering from haemophilia-related
disabilities and age-related co-morbidities might therefore face problems in organizing
regular visits at a HTC. Thus, families of young haemophiliacs, disabled and/or older
patients, or patients with limited travel options could benefit from a mobile haemophilia
outpatient care (MHOC) concept where a HTC provides home visits to a patient if necessary.
Here, we report the results of a 3-year pilot project implementing a MHOC concept
at the European Haemophilia Comprehensive Care Centre (EHCCC) of the Saarland University
Hospital in Homburg, Germany. During this pilot phase, adult haemophilia patients
as well as child and adolescent haemophiliacs and their families were home visited
at least twice by a social education worker and/or a trained physician of the HTC.
The patients were asked about their needs and expectations regarding a MHOC concept,
and interviews were performed to measure the HRQoL and treatment satisfaction (TS).
Materials and Methods
Design and Study Population
The project was implemented in 2010 and designed as a prospective single-centre pilot
study testing the feasibility of a MHOC concept at an EHCCC. The project was approved
by the local Ethics Committee of the Saarland Medical Council (No 92/11). All of the
88 adults and 28 underage haemophiliacs registered at the HTC were asked to participate
in this pilot study, and 79 patients gave informed consent to participate and were
enrolled (participation rate 68.1%).
Home Visits
A social worker and/or a trained physician of the HTC performed scheduled patient
home visits. As a minimum, the pilot phase comprised two visits, first at the beginning
of the study (baseline), and second, at least 1-year follow-up from baseline. If requested
and considered as useful by the HTC team, additional home visits were performed to
provide an individualized patient support. At the baseline and follow-up visits, patients
and/or parents were asked to fill out validated questionnaires to evaluate their HRQoL
and TS, these data will be published elsewhere. Furthermore, participants were asked
about their requirements and expectations regarding a MHOC concept.
Mobile Haemophilia Outpatient Care Questionnaire
The project-specific questionnaire contained the following nine questions: (1) ‘How
important is for you a tight connection to the HTC?’ (2) ‘Do you consider the implementation
of the MHOC as important?’ (3) ‘What kind of wishes and expectations do you have regarding
a MHOC concept?’ (4) ‘Regarding the past: did you wish to have a MHOC service available?’
(5) ‘Do you remember a situation in the past where a MHOC would have been helpful
for you?’ (6) ‘Would a MHOC concept be helpful at present?’ (7) ‘Do you think a MHOC
could be helpful in the future?’ (8) ‘Do you assume disadvantages through a MHOC concept?’
(9) ‘Regarding the MHOC, what kind of services have you utilised so far or would you
expect?’ (from a list of 13 MHOC services, multiple answers were possible to choose
with the following answer categories: 1 = service used; 2 = service desired). The
first two questions included 5-point Likert scale answers ranging from not important
to very important; the questions 3 to 8 were open questions, first asking dichotomously
yes/no, and if yes, the patient or parent should report which aspects.
Statistical Analysis
All analyses were performed by the SPSS statistic tool version 23 (SPSS Chicago, Illinois,
United States). Categorical data are presented using counts and percentages, and continuous
variables are expressed as patient numbers, means ± standard deviations, medians and
minimums/maximums. For testing on normal data distribution, the Kolmogorov–Smirnov
test was performed. Calculations for differences in attitude towards MHOC between
different clinical sub-groups (age group, treatment, infections) were performed by
chi-squared test. A p -value of < 0.05 was considered as significant.
Results
Baseline
Socio-Demographic Characteristics
Seventy-nine patients were enrolled, 56 adults and 23 children, with a mean age of
37.4 ± 16.4 years (17–78) and 9.8 ± 4.2 years (3–16), respectively. [Table 1 ] shows socio-demographic data of all participants (adults and parents of children
with haemophilia). The median distance from patients' home to the HTC was 43.5 km
(3–200) with a median travel time of 40 minutes (10–120). Most patients used their
private car to reach the HTC (79.5%), even though 16.6% were dependent on private
transport by a family member or friends or taxi transfer. Only a small minority used
public transport systems.
Table 1
Socio-demographic data (n = 79)
Socio-demographic characteristics
Adults (n = 56)
Parents (n = 23)
N
%
N
%
Marital status[a ]
Single
32
57.1
3
13.0
Married
20
35.7
16
69.6
Divorced
2
3.6
2
8.7
Living with a partner[a ]
Yes
27
48.2
19
82.6
No
28
50.0
3
13.0
Number of children living in household[b ]
0
46
82.1
–
–
1
8
14.3
–
–
2
2
3.6
–
–
Siblings[b ]
Yes
43
76.8
–
–
No
13
23.2
–
–
Educational qualification[a ]
No formal qualification
–
–
3
13.0
Lowest formal qualification
18
32.1
3
13.0
Middle formal qualification
14
25.0
8
34.8
Highest school qualification
17
30.4
7
30.4
University degree
4
7.1
1
4.3
Working status[a ]
Full-time
24
42.9
6
26.1
Part-time (50–75%)
6
10.7
3
13.0
Part-time (< 50%)
2
3.6
4
17.4
Not always in work
4
7.1
–
–
Living situation[a ]
Big city (> 100,000)
2
3.6
–
–
Suburbs of a big city
2
3.6
–
–
Town/small city (20,000–100,000)
9
16.1
7
30.4
Country village (5,000–20,000)
12
21.4
4
17.4
Village (< 5,000)
30
53.6
9
39.1
Single house/farm
1
1.8
2
8.7
Transportation to HTC[a ]
Private car
44
78.6
17
73.9
Dependent on transport by others
8
14.3
3
13.0
Public transport
3
5.4
–
–
Taxi
1
1.8
2
8.7
Abbreviation: HTC, haemophilia treatment centre.
a Missing data.
b Parents have not been asked about children and siblings.
Clinical Data
Of all study participants, 79.7% suffered from haemophilia A, 19.0% from haemophilia
B and one patient from type 3 von Willebrand disease. Forty-nine patients (62.0%)
were severely affected. The mean body mass index at enrolment for adults was 26.7 ± 4.5,
and for children it was 17.9 ± 3.6. More clinical data are provided in [Table 2 ] as a summary of both adults and children.
Table 2
Clinical data (n = 79)
Clinical characteristics
Total
(n = 79)
N
%
Type of haemophilia
A
63
79.7
B
15
19.0
von Willebrand syndrome type 3
1
1.3
Severity
Severe (< 1%)
49
62.0
Moderate (1–5%)
6
7.6
Mild (6–15%)
15
19.0
Sub-haemophilia (> 15–50%)
9
11.4
Treatment with factor concentrates
Prophylactic
38
48.1
On-demand
41
51.9
Target joints
Yes
10
12.7
No
69
87.3
History of inhibitor
Yes
5
6.3
No
74
93.7
Infections
HBV
17
21.5
HCV
28
35.4
HIV
9
11.4
Home treatment
Yes
63
79.7
No
16
20.3
Factor concentrate administration
Self-administered
40
50.6
By family member
13
16.5
By member of HTC
6
7.6
By family doctor
4
5.1
No substitution last year
16
20.3
Abbreviations: HBV, hepatitis B virus; HCV, hepatitis C virus; HIV, human immunodeficiency
virus; HTC, haemophilia treatment centre.
Thirty-nine adult patients (69.6%) and 11 children (47.6%) experienced at least one
bleeding episode in the last 12 months prior to enrolment, and 23 (41.2%) and 6 (25.9%)
experienced two or more bleeding events. The mean bleeding rate in adults was 3.2 ± 5.6
per year with a median of 1 (range, 0–26) and in children it was 1.3 ± 2.1 with a
median of 0 (range, 0–8). The majority of episodes were joint bleeds with a mean rate
in adults of 2.5 ± 5.4 per year (median 0, range, 0–26) and in children of 0.4 ± 1.7
per year (median 0, range, 0–8).
Mobile Haemophilia Outpatient Care
Overall, 149 home visits were conducted to generate the baseline and follow-up data.
Most of the home visits were performed by a social worker, and only four visits were
done by a trained physician. Seventy visits were performed in addition to the baseline/follow-up
data generation visits to provide a more individualized patient support in terms of
(1) information of teachers and physicians outside the HTC to haemophilia-related
issues (47.1%), (2) consultancy in social–legal affairs and support in contacting
authorities (42.9%) and (3) factor concentrate delivery and support in factor injection
(10%).
Regarding questions 1 and 2 of the MHOC questionnaire, all 79 participants responded.
Seventy-three participants (92.4%) reported an intense binding to the HTC as ‘rather
or very important’ and 70 (88.6%) considered the implementation of the MHOC service
as ‘rather or very important’ ([Fig. 1 ]). Out of the 79 participants, 45 formulated their wishes towards a MHOC (57%); some
patients mentioned more than one aspect. They expected and requested from a MHOC consulting
or educational activities, support in elderhood, care/treatment, factor application/factor
delivery, accessibility/transport connection and support in emergency. From all patients,
22 have wished the existence of a MHOC in the past (28.2%), and even 43% stated that
they would have needed a MHOC in the past , 35.4% reported about conditions in which a MHOC would currently provide support—mainly due to information transfer, support in emergency or disease-related
immobility and support in factor application or factor delivery—and 74.7% could imagine
future situations for using a MHOC service ([Table 3 ]). Only one patient assessed the implementation of a MHOC disadvantageous due to
missing direct contact with the HTC. [Table 4 ] shows which services participants reported to have used, would be in general desired
or were not desired. At baseline, only 31.6% of participants had used at least one
of the MHOC services, 1 to 3 services were used by 27.9% and one participant used
7 MHOC services. MHOC services concerning consultancy in social–legal affairs (12.7%),
support in contacting authorities regarding haemophilia-related issues (11.4%), factor
delivery (11.4%) and permanent availability of HTC specialists (11.4%) were used mainly.
Note that 89.9% of participants in general desired to have MHOC services available,
in median 3 out of the 13 listed MHOC services, only one participant desired 10 services.
Participants mainly desired permanent availability of HTC specialists (68.4%), visits
after bleeding episodes or post-surgery (59.5%) and factory delivery (58.2%). MHOC
services that were not mainly desired were regarding support in conflicts in the family
(83.5%), support in the documentation of factor use (82.3%), and support in the reduction
of anxiety (78.5%).
Table 3
Survey about needs and expectations towards a mobile home care program
Responses by the participants
Responding participants
N
%
Expectations and requests towards a MHOC (n = 45)
Consulting and educating activities
11
22
Support in elderhood (disease-related immobilization)
11
22
Care/treatment
9
18
Support in factor application or factor delivery
6
12
Accessibility and transport connection
6
12
Support in emergency
4
8
Others
3
6
MHOC service wished in the past?
Yes
22
28.2
Situation where a MHOC service was needed
in the past (n = 21)
Support in immobilization, disease, emergency or following surgery
9
42.9
Support of families with children
4
19.0
Injection training
2
9.5
Support regarding factor application or factor delivery
2
9.5
Support for social–legal affairs, or contact with authorities
2
9.5
In general
2
9.5
MHOC service required in the past?
Yes
34
43.0
Situation where a MHOC service was required
in the past (n = 28)
Support in immobilization, disease, emergency or following surgery
9
32.1
Support of families with children, information of school, kindergarten or physicians
7
25.0
Injection training, support regarding factor application or factor delivery
3
10.7
Support for social–legal affairs, or contact with authorities
3
10.7
Others
6
21.5
MHOC service required at present?
Yes
28
35.4
Situation where a MHOC service would be required at present
(n = 22)
Information of school, kindergarten or physicians
6
27.3
Support in emergency, elderhood with disease-related immobilization or following surgery
4
18.2
Injection training, support regarding factor application or factor delivery
4
18.2
Support for social–legal affairs, or contact with authorities
3
13.6
Others
5
22.7
MHOC service required in the future?
Yes
59
74.7
Situation where a MHOC service could be required in the future
(n = 48)
Support in emergency, elderhood with disease-related immobilization or following surgery
21
43.8
Support of families with children; information of school, kindergarten or physicians
8
16.6
Support for social–legal affairs, or contact with authorities
7
14.6
Injection training, support regarding factor application or factor delivery
6
12.5
Others
6
12.5
Abbreviation: MHOC, mobile haemophilia outpatient care.
Table 4
Used MHOC services during the pilot phase
MHOC services
Service
Used
In general desired
Not desired
N (%)
N (%)
N (%)
Consultancy in social–legal affairs
10 (12.7)
41 (51.9)
28 (35.4)
Support in contacting authorities regarding haemophilia-related issues
9 (11.4)
37 (46.8)
33 (41.8)
Factor delivery
9 (11.4)
46 (58.2)
24 (30.4)
Permanent availability of HTC specialists
9 (11.4)
54 (68.4)
16 (20.2)
Information of doctors outside the HTC
7 (8.9)
36 (45.6)
36 (45.6)
Consultancy in leisure-related questions
7 (8.9)
30 (37.9)
42 (53.2)
Support how to correctly store factor concentrates
6 (7.6)
14 (17.7)
59 (74.7)
Support contact school/employer
5 (6.3)
25 (31.7)
49 (62)
Visits after bleeding episodes or post-surgery
4 (5.1)
47 (59.5)
28 (35.4)
Support conflicts in the family
3 (3.8)
10 (12.7)
66 (83.5)
Training on how to inject at home
3 (3.8)
26 (32.9)
50 (63.3)
Support in the documentation of factor use
2 (12.5)
12 (15.2)
65 (82.3)
Reduction of anxiety
2 (2.5)
15 (19)
62 (78.5)
Abbreviations: HTC, haemophilia treatment centre; MHOC, mobile haemophilia outpatient
care.
Fig. 1 Importance of tight connection to haemophilia treatment centre (HTC) and implementation
of mobile haemophilia outpatient care (MHOC).
Significant differences were shown between parents and adults for ‘support in social
law issues’ (30.4 vs. 5.4%; p < 0.009) and ‘used consultancy concerning leisure activities’ (26.1 vs. 1.8%; p < 0.002).
Differences in MHCO were also found for clinical sub-groups concerning treatment regimen
(prophylaxis vs. on-demand) and hepatitis B virus (HBV)/hepatitis C virus (HCV) infections
(yes vs. no). More patients on prophylaxis reported that they would have needed a
MHCO in the past (p < 0.001) compared with on-demand treated patients (63.9 vs. 26.8%) or ‘used support
with contact with authorities’ (21.1 vs. 2.4%; p < 0.005). Differences were found for patients with HCV infection who considered an
intense binding to the HTC as more important (4.7 ± 0.8) than patients without HCV
infection (4.3 ± 0.7; p < 0.022). More HCV-infected patients required services for ‘questions concerning
correct storage of factor concentrate’ (28.6 vs. 11.8%; p < 0.044) or ‘consultancy regarding leisure’ (53.6 vs. 29.4%, p < 0.022), whereas more HBV-infected patients reported that they had expectations
towards a MHCO (58.8 vs. 32.3%, p < 0.046) and that there are situations in which a MHOC could currently provide additional
support for them (58.8 vs. 29.5%, p < 0.026).
No differences were observed between patients with varying severities (severe vs.
moderate vs. mild/sub-haemophilia) and presence or absence of human immunodeficiency
virus (HIV) infection.
Follow-up
At follow-up, 54 out of 56 adult patients and 16 out of 23 paediatric patients participated.
None of the patients had more than one follow-up visit. In average, patients were
visited 1.94 times during the study period; the mean interval between baseline and
follow-up visits for adults was 1.13 ± 0.4 years (median: 1.07, range: 0.44–2.69),
and for children it was 1.53 ± 0.5 years (median: 1.75, range: 0.63–2.33). Twenty
out of 54 adult patients reported a change in their life compared with baseline assessment.
Most of them mentioned positive aspects due to professional changes (such as start
of professional life, increase of salary, school or university degree) and to private
changes (such as marriage or acquired driving license allowing better mobility/autonomy).
Only few patients reported negative changes in their profession or private life (job
loss, separation). Five out of 15 parents mentioned a change in their life. Four mothers
started to work again, only one had a negative event due to her divorce. Regarding
the clinical data of the adult patients, no significant changes between the baseline
and follow-up interviews were observed.
Mobile Haemophilia Outpatient Care
At follow-up, 63 participants (90%) reported an intense binding to the HTC as ‘rather
or very important’ and 65 (92.9%) considered the implementation of the MHOC service
as ‘rather or very important’. Out of the 70 participants, 29 formulated their wishes
towards a MHOC (41.4%); some patients mentioned more than one aspect. Their expectations
towards a MHOC did not change in general from baseline; additional aspects mentioned
were support in contact with authorities, care in rural region, support searching
for a HTC at study location and continuation of the MHOC service project. Note that
78.6% could imagine future situations for demanding a MHOC service.
At follow-up, 41.4% of participants had used at least one of the MHOC services, and
1 to 3 services were used by 32.9%; three participants had used 5 MHOC services and
one had used 7 MHOC services. Only one participant did not desire to have MHOC services
available; 98.6% desired services, in median 6 out of the 13 listed MHOC services,
19 participants desired 10 to 13 of the listed MHOC services. From baseline to follow-up,
there was a trend to an increased use of MHOC services, but this was not significant.
For those patients participating both at baseline and follow-up (n = 70), the use of the following MHOC services increased: factor delivery (8.6 vs.
15.7%), consultancy in social–legal affairs (12.9 vs. 20%) and support in contacting
authorities regarding haemophilia-related issues (11.4 vs. 14.3%). By contrast, the
number of desired MHOC services increased significantly (p < 0.0001) from baseline to follow-up (M = 3.9 ± 2.7 vs. 6.6 ± 3.9) ([Fig. 2 ]). The frequency of all single MHOC services desired increased from baseline to follow-up,
but remained stable for wished consultancy in social–legal affairs which was 54.3%
([Fig. 3 ]).
Fig. 2 Differences concerning the mobile haemophilia outpatient care (MHOC) services between
baseline and follow-up.
Fig. 3 Mobile haemophilia outpatient care (MHOC) services desired (comparison: baseline
vs. follow-up).
Discussion
The German federal state ‘Saarland’ where the EHCCC is located is a more rural region
with limited availability of public means regarding transport issues. For patients
who do not own a private car this implies a high dependency on other family members
or friends when planning a visit to the EHCCC. The major goal of the MHOC pilot study
was to provide an extended social and medical care in the private environment of haemophilia
patients and their families. By visiting the patients at home, we expected to get
a more complete picture about the individual disease-specific problems and resulting
needs of the patients and their families. Of the participants, 74.7% were open to
use the services of a MHOC concept in the future, especially for support in emergency,
elderhood and their immobility. They were generally very satisfied with the MHOC concept
and felt quite supported by the care of the HTC.
The implementation of the MHOC concept provided patients with an extra support in
social- and work-related issues allowing them to deal better with the circumstances
and social consequences of their disease. There was a significant increase of desired
number of MHOC services from baseline to follow-up, which can be interpreted as an
indicator that participants appreciated the provided services and want to use them
in the future, if necessary, implying the feasibility of the MHOC services. This assumption
is underlined by the direct expectation concerning the continuation of the MHOC service
project.
In terms of marital status and living with a partner, our German adult haemophilia
cohort was different compared with an Austrian haemophilia cohort in which more patients
were married (35.7 vs. 57%) or were living with a partner (48.2 vs. 68%).[10 ] The prevalence of HIV and HCV infections in our cohort was comparable to a huge
multi-national study in 21 European countries.[11 ]
A limitation of this pilot project was the relatively small number of enrolled patients.
Nevertheless, 68.1% of the patient population registered at the HTC participated in
the study. Another limitation could be the short follow-up period. A real impact of
the implementation of a MHOC concept is probably only detectable after a couple of
years when patients are using such a service on a routine basis.
Irrespective of the current structures for the treatment of haemophilia patients,
all health care players have to prepare for an increasing number of patients over
65 years of age due to the increasing life expectancy of this population.[12 ] Although the treatment quality of haemophilia has significantly improved over the
last decades, older haemophiliacs often suffer from an increasing burden of their
disease, e.g. due to infections and arthropathy in combination with age-related co-morbidities,
psychological and economical aspects.[13 ] Therefore, the provided care by HTCs should be adapted or expanded to age-related
problems such as impairments and problems in daily living.[14 ] Another problem which occurs in haemophilia patients is the development of inhibitors
requiring frequent infusions.[8 ] Besides the patients with limited access to public means, families of younger patients
and disabled and elderly patients would benefit from the implementation of a MHOC
concept, e.g. to ease the procedure of factor application. Moreover, current data
show that acceptance of the disease and self-management skills are important aspects
for patient's adherence to treatment, and patients may require tailored professional
support.[14 ] Services for home treatment by qualified staff could be such a support tool; this
is also reflected by the engagement of pharmaceutical companies supporting HTCs for
the integration of MHOC concepts.[12 ]
In conclusion, the results of this prospective pilot study do support our hypothesis
that a MHOC concept is a helpful supplement to improve the treatment portfolio of
a comprehensive care HTC for patients of all age groups. It supports patients and
families with limited access to HTCs living in rural regions to receive regular health
care on request.
