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EMERGING THERAPIES FOR
HEMOPHILIA: A NEW ERA OF
CARE AND THE ROLE OF THE
INTERDISCIPLINARY TEAM
Susan Cutter, LCSW, MPA
Hospital of the University of Pennsylvania
EMERGING THERAPIES FOR HEMOPHILIA:
A NEW ERA OF CARE AND THE ROLE OF
THE INTERDISCIPLINARY TEAM
Semin Thromb Hemost 2014;
40(07): 790-802
Authors:
Michelle Witkop, Susan Cutter,
Joann Deutsche, Maria
Santaella, Robin Chapman,
Jennifer Lafranco, Angela
Lambing
EMERGING THERAPIES FOR
HEMOPHILIA

Era of potential promise and increasing
complexity, more so for those with FIX than
FVIII deficiency

HTC staff are well positioned to assess,
educate, and support patients and families
in navigating this rapidly changing
landscape

To support these challenging efforts, this
article offers a perspective on issues
affecting therapeutic transitions and
provides tools to foster ongoing adherence.
SYSTEMATIC LITERATURE
SEARCH

What is the current journal literature regarding
the following?
Therapy transition
 Adherence
 Psychosocial aspects of change
 Other medical considerations (e.g. co-morbidities,
cultural influences, health care beliefs, barriers to
care)


Medline, Embase, PsycINFO, and British
Nursing Index
TRANSITIONING TO NEW TREATMENT
TECHNOLOGIES: CONSIDERATIONS
 Safety
 Efficacy
 Necessity
 Benefits
 Adherence
 Cost/impact
on finances
 Lifestyle adjustments
 Cultural issues
 Patients’ perceptions
NON ADHERENCE IN CHRONIC
ILLNESS

Studies reveal individuals with chronic illness
adhere to their medical treatment 50% of the time¹

Adherence with treatment is higher among young
children with hemophilia than other age groups

Even nominal non adherence with hemophilia
treatment increases risk of bleeding
events/negative outcomes and still results in
exorbitant health care costs²
¹World Health Organization, at:
http://www.who.int.chp/knowledge/publications/adherence_report/en/ Accessed 3/23/2013
²DuTreil, S. , Haemophilia 2007 ;13(5):493-501.
PRIMARY MOTIVATORS FOR
HEMOPHILIA ADHERENCE
Extensive literature review by Shrijvers¹identified
primary motivators for prophylaxis adherence:
 Frequency of symptoms
 Belief in necessity of treatment
 Age
 Time spent with HTC staff
 Relationship with HTC staff
 Reduction/disappearance of the
symptom
¹Schrijvers, LH, et al, Haemophilia 2013;19(3):355-361
MOTIVATIONS & BARRIERS TO
ADHERENCE


HTC staff awareness of primary motivators is
only a start
Essential to recognize individual adherence
challenges and understand issues important to
patients and families considering transitioning
to novel factor replacement products (nFRPs)¹˒²
¹Ljung, R, et al, Haemophilia 2013;19(4):481-486
²Khair, K, et al, Haemophilia 2012;18(3):2286-9
POTENTIAL ADHERENCE
CHALLENGES: CHANGING

Lack of symptoms¹





TO NFRPS
If it’s not broken, don’t fix it!
Strong belief in their current regimen
Lack of receptivity among patients with significant
joint disease
Contrary to previous education received from health
care providers
Resistance to change
¹DeMoerloose, P. , et al Haemophilia 2008;14(5):931-938.
POTENTIAL ADHERENCE
CHALLENGES: CHANGING TO NFRPS






Recent transition to HTC
new relationship
Cultural/language barriers
Poor communication between HTC staff &
patient/family
Low self-efficacy
Mood issues (e.g. depression)
Ineffective coping strategies
POTENTIAL ADHERENCE
CHALLENGES: CHANGING TO NFRPS






Disagreement among family
Impaired family functioning
Inconvenient/lifestyle disruption
Forgetfulness: intentional & non-intentional¹
Insurance/financial barriers
HTC Staff: Canadian study revealed 12.5 %
physicians & 17.6% nurses did not assess adherence²
¹Farmer, A., et al, BMC Family Practice, 2012;13:30.
²Chan, A.K, et al, Haemophilia, 2011; 17:816-7.
STRATEGIES FOR BUY IN: WIIFM

Extended half-life products
 Venous access difficulties
fewer
infusions/needle sticks¹˒²˒³˒⁴
 Maintain adequate factor levels¹˒²˒³˒⁴
 Less lifestyle disruption
 Personalized prophylaxis⁵
¹Pipe, S et al, Am J Hematol 2012;87(Suppl 1):S33-S39
²Fogarty, P, Hematology (Am Soc Hematol Educ Program) 2011:397-404
³Branchford B, et al, Curr Opin Pediatr 2013;25(1):23-30
⁴Pipe, S et al, Hematology (Am Soc Hematol Educ Program) 2012:203-209
⁵ Skinner, M., Haemophilia. 2012;18 suppl 4:1-12
STRATEGIES FOR SUSTAINED
ADHERENCE




Work together with the patient/family to identify
& solve issues related to non-adherence
Utilize the resources & expertise of all HTC team
members to identify & address adherence issues.
Provide positive reinforcement for all progress
towards adherence
Utilize an individualized approach based on
specific adherence issues (tool 2, pg. 793)


Research underscores the value of a
separate evaluation tool used during
clinic visits to assess and foster regular
discussion of adherence¹˒²
To facilitate HTC staff identification of
primary adherence motivators, barriers, and
opportunities for impact, the authors
developed a tool that provides individualized
recommended interventions for each
identified barrier.
¹Duncan, N, et al, Haemophilia 2010;16(1):47-53
²Duncan, N, et al, Haemophilia 2010;16(2):247-255
Table 2. Prominent Factors Motivating Treatment Adherence
Motivators to Adherence with
hemophilia treatment, including
longer-acting nFRPs
1)Experience of symptoms
(frequency/reduction)
Challenges to longer-acting FRPs
Opportunities for HTC staff impact & intervention strategies
a) Infrequent or absence of symptoms, especially those with i) Assess if minimize or ignore symptoms & address accordingly
mild or moderate hemophilia,
ii)Provide prevention focused education based on individual's preferred style of learning (listening, reading, &/or hands on).
Additional education described in 1, b.
iii)Assess for mood issues (past or present) as it may affect attitude toward treatment; address accordingly
iv) Discuss past history of adherence with hemophilia treatment, including barriers/solutions
v) Actively engage PWH by employing highly participatory counseling techniques (e.g. motivational interviewing, behavior
modification therapy, stages of change therapy, &/or ongoing psychosocial counseling)
vi) Encourage patient/family to re-state agreed upon solutions to ensure everyone is on the same page
vii) Utilize Patient -Provider Agreement (figure 1)
b) A strong belief in their current regimen; it is very effective i)Facilitate open discussion of patient/family perceptions regarding current & proposed treatments.
& is well incorporated into their daily routine & lifestyle
ii) Provide education regarding risks & benefis: including safety, efficacy, necessity, irregularly spaced treatment, dosing,
financial impact, PK studies, tailored treatment regimen, etc.
iii) Assess barriers to lifestyle incorporation, including ability/commitment to adhere to changes in treatment regimen.
iv) Develop treatment plan with patient/family input to facilitate adherence, include challenges related to access, geography,
scheduling, etc.
v)Encourage patient/family to re-state agreed upon solutions to ensure everyone is on the same page
c) Lack of receptivity among adult patients with significant i) Assess & discuss possible contributing issues such as: perception of low treatment effect or lack of improvement in
joint disease
symptoms; low self-efficacy/esteem; difficulty discerning between a joint bleed & arthritis; mood/memory/cognitive impairment;
additional education needs; psychosocial issues/needs; and possible barriers to adherence such as reduced ability to selfinfuse related to decreased dexterity from joint disease.
ii) Same as opportunities for impact & interventions in 1., a & b.
2) Positive belief in necessity of treatment a) Information is contrary to previous education received Same as opportunities for impact & interventions in 1., a & b.
about dosing, frequency, and optimal level of factor activity.
b)Resistance to change
Same as opportunities for impact & interventions in 1.,a - c.
(Bombardie, 2008; Coppola, 2012; Shrijvers, 2013; Saunders, 2010; Khair, 2012; Remor, 2011; Luing 2013; DeMooreloose, 2008; Farmer, 2012; Geraghty, 2006)
copyright 2014 by Thieme Medical Publishers, Inc
Motivators to Adherence with
hemophilia treatment, including
longer-acting nFRPs
Challenges to longer-acting FRPs
3)Positive relationship with HTC staff
a) Patient/family recently transitioned to the HTC
4)Strong family functioning and support
Opportunities for HTC staff impact & intervention strategies
i) Acknowledge & address issues & challenges related to transitioning care providers.
ii) Promote partnership in care & begin building a relationship
iii) Assess preferences for learning, communicating, etc
iv) Identify specific barriers, concerns, fears, etc related to treatment transition & provide education, counseling, & resource
referrals accordingly
v) Same as opportunities for impact & interventions in 1., a -c.
b) Poor communication and/or disagreements between HTC i) Collaborate as a team to adapt communication styles to better fit the needs of the patient/family
staff and patients/families.
ii) Promote partnership in care & begin building a more positive relationship.
iii) Facilitate open discussion with patient/family to gain increased insight into specific barriers to communication, their
communication needs, & if necessary, attempt to resolve past grievances/issues,.
iv) Consider pairing staff with patient/family based on their needs, abilities, and relationships.
v) Provide consistent messages & information to patient/family through strong team collaboration.
vi) Same as opportunities for impact & interventions in 1., a -c.
B. Mistrust among patients/families due to prior viral
contamination from factor products
i) Same as opportunities for impact & interventions in 1., a - c & 3., b.
b) Cultural and/or language barriers
i). Provide staff cultural sensitivty training
ii) Learn basic greetings in patient/family primary language
iii) Utilize translation services for non-English speaking staff
iv) Assess perceptions of health, illness, and recommended treatments
v) Assess & address other potential barriers to care
vi) Same as opportunities for impact & interventions in 1., a - c.
a) Disagreement among family members and other key
influencers regarding transitioning to nFRPs
I) Assess & address communication issues within family
ii) Identify key decision makers & build/strengthen relationships with them
iii) Identify specific barriers, concerns, fears, etc related to treatment transition & provide education & resource referrals
accordingly.
iv) Same as opportunities for impact & interventions in 1., a - c.
b) Impaired family function (e.g. mental health issues,
physical health issues in other family members, substance
abuse, estrangements, poor communication, emotionfocused coping strategies, inability to adapt to change)
i) Assess for mood issues as they may impact attitude toward treatment; address accordingly
ii) Provide counseling to strengthen baseline skills (e.g. coping, decision making, communication).
iii) Refer to specialists for more complex mental health needs
iv) Strategize with patient/family ways to incorporate nFRP into their lifestyle to best facilitate adherence.
v) Educate (described in opportunities for impact & interventions in 1, a & b.)
vi) Employ highly participatory counseling techniques, as described in 1. a.
(Bombardie, 2008; Coppola, 2012; Shrijvers, 2013; Saunders, 2010; Khair, 2012; Remor, 2011; Luing 2013; DeMooreloose, 2008; Farmer, 2012; Geraghty, 2006)
copyright 2014 by Thieme Medical Publishers, Inc
Motivators to Adherence with
hemophilia treatment, including
longer-acting nFRPs
5) Treatment easily incorporated into
patient/family lifestyle
Challenges to longer-acting FRPs
Opportunities for HTC staff impact & intervention strategies
a)Treatment and/or timing of treatment is inconvenient (e.g. i) Assess & address current barriers to treatment & strategize solutions (e.g. distraction techniques, further home infusion
lost time from school or work; disruptive to patient/family training, transportation needs/costs, scheduling flexibility, etc)
schedule; lack of proficiency with self-infusion, and/or
ii) Same as opportunities for impact & interventions in 1., a -c.
child's resistence prolongs treatment time; reliance on home
care nursing or HTC nurses for infusions; geographic
barriers to care, etc)
b) Venous access issues
i) Educate patient/family; include techniques to decrease discomfort during access and increase pain tolerance
ii) Provide self-infusion training for patient/parents/caregivers, including training in CVAD, if necessary.
iii) Explore other access options (e.g. AV fistula)
iv) Same as opportunities for impact & interventions in 1., a -c.
c) Forgetfulness
6) Insurance and stable financial
resources to cover treatment
i) Facilitate open discussion re: forgetfulness and discern if it is accidental or deliberate/avoidance
ii) Tailor intervention strategies based on type of forgetfulness
iii) Consider reminder prompters as incidences of forgetfulness may increase with irregularly spaced/difficult-to-remember
nFRP regimens.
iv) Provide education, training, and highly participatory counseling techniques, accordingly as described in opportunities for
impact & interventions in 1, a-c.
v) Encourage patient/family to re-state agreed upon solutions to ensure everyone is on the same page
vi) Utilize Patient -Provider Agreement (figure 1)
Patient/family desire to change to nFRP, but cost
i) Assess insurance & cost concerns as possible barrier to adherence and/or ability to change to nFRPs
implications are too great. (e.g. insurer limits coverage,
ii) Strategize re: possibilities to mitigate financial consequences (e.g. patient/family/provider advocacy with insurer, change
product/brand is not on their drug formulary, speciality drug insurance provider during re-enrollment period, pharmaceutical assistance programs, other insurance assistance programs,etc)
tiers result in increase in patient/family out-of-pocket
iii) If able to resolve insurance and cost issues, proceed with opportunities for impact & interventions in 1., a & b.
expenses, etc)
iv) If no viable alternatives are feasible, assist the patient/family in understanding the outcome & need for ongoing advocacy
within the hemophilia community re: access to treatment.
7) Exhibits positive health behaviors, has a) Lack of knowledge re: nFRPs
i) As described in opportunities for impact & interventions in 1, a-c.
strong sense of self-efficacy & self b) Time/inconvenience
i) As described in opportunities for impact & interventions in 5, a.
esteem, is motivativated, and utilizes
c) Cost
i) As described in opportunities for impact & interventions in item 6.
effective coping skills
(Bombardie, 2008; Coppola, 2012; Shrijvers, 2013; Saunders, 2010; Khair, 2012; Remor, 2011; Luing 2013; DeMooreloose, 2008; Farmer, 2012; Geraghty, 2006)
copyright 2014 by Thieme Medical Publishers, Inc
STRATEGIES FOR SUSTAINED
ADHERENCE TO NFRPS
Fig. 1 Sample patient-provider agreement when initiating clotting factor
replacement therapy
1. The new clotting factor replacement therapy _____________________ may help your hemophilia
in ways we don’t fully understand. In an effort to meet your needs, personalize your clotting factor
replacement therapy, and decrease bleeds, it is important to have your active involvement.
2. It is not clear how your body will use this new clotting factor. Additional blood testing may be
required as follows: _______________________________. These results will help us understand
how this clotting factor will work best for you and can provide a personalized dosing plan.
3. If you experience ANY bleeding while taking this clotting factor, it is necessary to contact the HTC
to make changes to your treatment plan.
4. Letting us know how and when you take your clotting factor is very important. We call this “keeping
a log.” We will work with you on the best way for you to provide this information, on a regular
schedule: every ___________weeks/months. If you currently do not have a clotting factor log
process, the HTC will assist you with paper or electronic versions.
5. It is important to take the clotting factor as prescribed. If you feel that changes are needed to your
treatment plan, contact the HTC to discuss the issues BEFORE making any changes.
6. If you cannot meet these steps, you will agree to go back to your previous clotting factor treatment
regimen (what you used before the change).
Patient Name: (print) ___________________________________
Patient Signature: _____________________________________ Date: ____________
Provider Name: (print) _________________________________
Provider Signature: ____________________________________ Date: ___________
©2014 by Thieme Medical Publishers, Inc