Cystic Fibrosis Care in 2017:

Overcoming Challenges to
Capitalize on Success!

Gregory Sawicki, MD, MPH

Director, Cystic Fibrosis Center Assistant Professor of Pediatrics

Division of Respiratory Diseases Harvard Medical School
Boston Children’s Hospital
 CF: A Story of Progress

1950 1989 2015

CF: A Changing Population
CF Foundation Patient Registry, 2014
Adolescence is a high risk period in CF

CF Foundation Patient Registry, 2014

Living with CF ……
A Case Study
 Diagnosis
– Failure to thrive at age 6 months led PCP to refer for sweat test
– Chronic nasal congestion
– Positive sweat test with CFTR genotype: F508del homozygous
– Social history:
• Has an older sister who does not have CF
• Father is an attorney
• Mother is a teacher
• Has had private health insurance through his father’s employer

– Parents surprised by diagnosis, express need for support, mom

stops working to care for her son instead of utilizing daycare
 Infancy - Preschool
– Started pancreatic enzymes at time of diagnosis
– Weight increased from 10%ile to 30%ile by age 2
– Had reflux as an infant
– Described as a “picky eater”, often refusing meals
– Started chest PT intermittently at age 8 months
– Attended daycare from age 1 until preschool at age 4
– Initiated Dornase alfa therapy at age 4
– Parents decide to have a third child, request genetic
counseling, no pre-natal screening available at the time
 Elementary School
– School requires him to go to nurse to obtain enzymes. Often
forgets.
– First time Pseudomonas detected at age 5, treated with inhaled
antibiotics for eradication therapy
– No further Pseudomonas found in subsequent throat cultures
– In second grade began participating more in soccer, parents of
other children ask whether he is “contagious” due to his cough
– Reported intermittent cough, but was the fastest kid on his team
– Age 5 began daily chest PT with Vest
– First attempts at lung function at age 5 with FEV1 102%
predicted
– Never hospitalized
 Middle School
– Age 12: Has soccer practice / games 4 days per week and family
reports that they don’t have time for the Vest on those days
– BMI has dropped from 40%ile to 35%ile
– Denies any new GI symptoms
– Parents state that they pack his lunch every day with enzymes in
his bag
– FEV1 at age 12: 95% predicted
– First hospitalization due to increased symptoms, decreasing
weight, and re-growth of Pseudomonas
 High School
– Continues to play competitive soccer, also lacrosse and
basketball
– Prescribed alternate month inhaled antibiotics, hypertonic saline,
dornase alfa, and daily chest PT
– BMI continues to drop – 20%ile at age 14
– Reports “forgetting” his enzymes and doesn’t think he needs
them anymore
– Develops influenza at age 15 requiring hospitalization
– Starts a summer job as a golf caddy
– Straight A student, aspires to attend college to study engineering
– Starts dating girls at age 16, does not tell friends (except for his 2
best friends) about CF
 College
– Comes to CF clinic only while home on holiday breaks
– Requests single room accommodation in order to
complete treatments in private
– Reports feeling pretty good, and is surprised with a
decrease in FEV1 (70%) and BMI% 10. 
– Reports that living away from home for the first time while
juggling a full load of classes, and social life has made it
difficult to manage all his treatments.
– Parents ask whether he should move back home to attend
a local school instead of living away from home in a dorm
– Meets adult CF care team for the first time
 Post-College
– Baseline FEV1 60% predicted
– Chronic infection with Pseudomonas
– Care now coordinated by an adult CF Center
– Hospitalized yearly since age 23 for CF exacerbation
– Works in a research laboratory as a technician, hoping to apply to
graduate schools
– Engaged, considers fiancée his primary support person
– Considering having children
– Medication regimen: Inhaled tobramycin, Dornase alfa,
Azithromycin, Inhaled sodium chloride, Pancreatic enzymes,
Multivitamin plus minerals, Fluticasone propionate , Esomeprazole
– Consideration made for initiation of lumacaftor/ivacaftor
 Challenges to CF Care Through the Life Span

Adulthood

Adolescence

Childhood

Infancy

Diagnosis
 Adulthood

Adolescence

Childhood

Infancy
Newborn
Screening

Infant Care
Diagnosis Practices
 Nutritional Assessment
Pulmonary Function Assessment
Monitoring for Complications
Prevention of Disease Progression Adulthood
Education About CF
Assessment of Family Functioning
and Social Supports Adolescence

Childhood

Infancy
Newborn
Screening

Infant Care
Diagnosis Practices
 Nutritional Assessment
Pulmonary Function Assessment
Monitoring for Complications
Prevention of Disease Progression Adulthood
Education About CF
Assessment of Family Functioning
and Social Supports Adolescence

Childhood
Transition
Self-Management
Infancy
Newborn Shared Decision-Making
Screening

Infant Care
Diagnosis Practices
 Challenges to Achieving
Optimal CF Outcomes

• Adherence to Chronic Therapies

• Transition from Pediatric to Adult-Focused CF Care

 Complexity of CF Treatment
Dietary &
Pancreatic
Chest vitamin
enzyme
supplements
physiotherapy
replacement
Daily medical Respiratory
treatments medications
High
Treatment
Burden

Bregnballe, et al. Patient Prefer Adherence. 2011;5:507-15. Sawicki, et al. Pediatr Pulmonol.
2012;47(6):523-33.
20
21
 Daily Treatment Burden for Adults
Median Number of Therapies Median Number of Minutes
(Total = 108)

Inhaled 1

Exercise
Nebulized 2 29 Nebulized
41

Oral 4
Airway
Clearance
29 Oral
9
Total 7

Sawicki, et al. J Cyst Fibros. 2009;8(2):91-6.

Adherence in CF:
What do we know?
 Some Terminology
• “Compliance”
– The accuracy with which a person follows the
regimen prescribed by a health professional
• “Adherence”
– Extent to which a person’s behavior coincides
with medical advice
• “Self-management”
– Health behaviors of individuals and families
use to care for a chronic condition
http://apps.who.int/iris/bitstream/10665/42682/1/9241545992.pdf
Modi AC et al. Pediatrics. 2012;129(2)e473–485.

24
Adherence rates to CF respiratory
medications are low

MPR=0.5

Quittner AL et al. Chest 2014;146(1):142–151.

Adherence rates vary by age

Quittner AL et al. Chest 2014;146(1):142–151.

 Adherence in adolescents is best on
weekdays during school term time

Fig. 1 Comparison of adherence to treatment for individual patients during a) weekdays and weekends and
b) holidays and term-times. The horizontal thickened bars represent mean adherence for the group
(*p<0.001).
Ball R et al. Journal of Cystic Fibrosis 2013;12(5) 440–444.
 Challenges to adherence and
self-management
Individual
• Age
• Gender
• Health literacy
• Disease & treatment knowledge
• Mental health / behavioral problems
• Coping style
• Health beliefs & perceptions
Health Care System
• Access to care
• Continuity of care
• Patient-provider communication
• Shared decision making
• Frequency of clinic visits
• Provider biases
Family
• Family structure
• Income / health insurance
• Disease knowledge
• Mental health / behavioral problems
• Coping style
• Health beliefs & perceptions
• Relationship quality
• Involvement in care
Community
• Neighborhood
• Work (hours & policies)
• School
• Peer support
• Illness stigma
Adapted from Modi AC et al. Pediatrics. 2012;129(2)e473–485.
Adherence Typologies

Unwitting
– Patient and provider mistakenly believe that the
patient is adherent

Erratic
– Patient understands and agrees with therapy but
has difficulty consistently maintaining regimen

“Rationalized”
– Patient deliberately alters or discontinues
therapy
 Adherence in CF:
Measurement matters!
• Self-report • Pharmacy records
– Daily diaries – Medication Possession
– Questionnaires Ratio (MPR)
– Interviews – Proportion of days covered
(PDC)
• Clinician-report – Number of refills
– Questionnaires • Electronic monitors
– MEMS Caps
– “Chipped” devices
– MDI monitors

30
Identifying non-adherence is challenging

PATIENT REPORT PROVIDER REPORT

Daniels T et al. Chest 2011;140(2):425–432.

 Adherence in CF:
Adolescent Perspectives
Facilitators
Barriers
• Improving understanding of
• Immediate time pressures
– Lack of time
the importance of therapies
– Uncertain schedules • Fostering relationships with
• Competing priorities the CF care team
– Balancing time trade-offs – CF team should be creative in
problem-solving with the
• Privacy concerns adolescent and parent
– Wanting to be “normal”; not wanting
to seem different or disabled • Empowering adolescents
• – Enabling parents to cede control
Lack of perceived
and entrust responsibility to
consequences adolescents
– Not seeing an impact on one's
health right away from skipping • Establish a structure
treatments or medications – Having a daily routine, “making it
a ritual”

Sawicki GS et al. Pediatr Pulmonol , 2015 Feb;50(2):127-36.

What Can Be Done?

33
 Reframe Adherence to Sustain Daily Care
Shift from adherence in
C
a linear view
Adherence  Sustaining Daily Care
a
r
T
e
t
r E

e
x
e p
a e
m
a ct
at
– io
P t n
of
a
ti
e
m a
d
h
n
t
e e
r
e
F
a
n n
c
m
il t e
to
tr
y
p e

Ongoing
I at
n
l
m
t e
nt
e
a
collabor
pl
r a
a n

To a cyclical view of Sustaining Daily Care n

c
ti
o
n ative
convers
ation
between
care
team
and
patient/f
amily

Co-
Adaptin
create
g plan
the right
into
treatmen
daily life
t plan

34
CFF Partnerships for Sustaining Daily Care

35
 CFF Success with Therapies
Research Consortium
To facilitate the clinical study of interventions to improve
adherence and CF disease self-management in order to
optimize health outcomes and quality of life.

Interventions will be developed, implemented, and disseminated to:

1) Include measures of adherence and self-management that are

validated for use in CF.
2) Be patient-centered and empower individuals with CF.
3) Be tailored to people’s unique strengths and needs.
4) Be feasible, practical and adaptable.
5) Leverage collaborations with family, CF Care team, and community.
 STRC Sites and Members

17 Institutions 31 Principal Investigators

 13 Pediatric programs  20 Physicans
 8 Adult programs  10 PhDs – Psychology, Health Economics,
Health Education
 1 PharmD
 Health Care Transition:
A Health System Dilemma
The Current State of Transition in the U.S.

Faith, Trust,
Pediatric Adult
and Pixie
Care Care
Dust
 Operational Definition of
Health Care Transition

A purposeful planned process that supports

adolescents and young adults with chronic health
conditions and disabilities to move from child-centered
(pediatric) to adult-oriented health-care practices,
providers, programs, and facilities.

Reiss & Gibson, Pediatrics. 2002 Dec;110(6 Pt 2):1307-14.

 Goals of Health Care Transition

• Promote autonomy, self-care and self-

determination
• Allow assumption of adult roles and responsibilities
• Maximize life-long functioning and potential
• Incorporate lifespan developmental perspectives
and addresses knowledge and skills
• Transfer of care from child-oriented to adult-
oriented services with uninterrupted services (when
appropriate)
TRANSITION ≠ TRANSFER
 Should we be transferring everyone?

Adults, including those with childhood-

acquired chronic conditions, should
receive adult-oriented primary health
care from appropriately trained and
certified providers, in adult health care
settings

Journal of Adolescent Health 2003; 33:309–311

 Consensus Statements on
Health Care Transition

• Identify a health provider to coordinate transition

• Train health care providers in transition services
• Maintain a portable medical summary
• Create a written health transition plan
• Apply standard primary care guidelines to CSHCN
• Ensure affordable, continuous health insurance

Pediatrics 2011;128:182-200
Time for a Reality Check!
National Survey of US CF Centers: 2007

• Transfer of care occurs at median age of 19, but initial discussion

begins at median age of 17

• Fewer than half of CF programs perform readiness assessments

• Fewer than 10% have written list of desirable self-management

skills

• Fewer than 2/3rds discuss current sexual activity or fertility

intention

• Half of programs never or rarely prepare medical summaries

McLaughlin, S. E. et al. Pediatrics 2008;121:e1160-e1166

 What is the Problem?
Obstacles to Health Care Transition

• The youth
• The parent(s) / family
• The pediatric team
• The adult team
• The health system
 Patient-Family Level Barriers

• Accessing care: primary and specialty care

• Knowledge and Skills
• Insurance
• Family / Individual resistance
• Familiarity with the pediatrician / Fear of a new team or facility
• Need for family to maintain control
• Perception adolescent cannot handle condition
 Health System Barriers:
Are Pediatricians are From Mars,
and Internists From Venus?
 Barriers for Pediatricians

• Lack of available adult specialists

• Lack of pediatric staff skills in transition planning
• Difficulty in breaking bond between pediatricians and
adolescents and their parents
• Lack sufficient pediatric staff time to provide transition
services
• Lack of adolescent knowledge about health conditions
and/or skills to self advocate during physician visits
 Barriers for Internists

• Training
• lack of training in congenital and childhood-onset conditions
• lack of adolescent medicine training
• Working with families
• lack of family involvement
• families' high expectations
• Practice management
• difficulty meeting patients' psychosocial needs
• facing disability/end-of-life issues during youth and early in
the relationship
• financial pressures limiting visit time
There Are Many System Barriers

System Providers
 Patient Protection and Affordable Care Act:
Can it Improve Transition?

• Subsidies for 133%-400% Federal Poverty Level

• No lifetime coverage limits
• Can’t deny coverage for pre-existing conditions
• Can keep children on parents’ policies until age 26
• Can’t cancel policies because of illness
• Encouragement of Patient Centered Medical Home
 Key Questions to Consider
• What can parents do from an early age, to encourage a
smooth transition?
• What parenting traits and styles inhibit a smooth
transition?
• How can families and medical teams work with an
adolescent or young adult who resists, protests or
otherwise makes transition more difficult?
• By what ages would you expect patients to be
independent in certain treatment tasks?
• How can patients and families work with medical team
members in their transition process?

54
 The Goal: A Comprehensive
HCT Process
Active Transfer

Therapeutic Discharge Empathic Intake

Pediatrics Adult care

Preparation Planned Establish YA

for Transfer Hand-off in Adult System
A non-CF focused resource:
Got Transition!

www.gottransition.org
 CF R.I.S.E.:
A CF-Specific Resource
THIS INFORMATION MEETS THE GUIDELINES AND
STANDARDS OF THE OF THE CF FOUNDATION’S
EDUCATION COMMITTEE.

CF R.I.S.E. was developed in collaboration with

a multidisciplinary team of CF experts and is
sponsored by Gilead.
 Summary
• Health outcomes in CF have been improving for decades
• Many more adolescents with CF continue to reach adulthood
• Novel therapies continue to be developed
• Non-adherence to chronic CF therapies is common and is
linked to poor health outcomes
• “Precision medicine” for CF needs to focus beyond genetic
therapies
• Interventions to improve adherence and transitional care need
to be developed, studied, and implemented on a larger scale

58
 Thank You!

59