Cystic Fibrosis Care in 2017 Overcoming Challenges to

Cystic Fibrosis Care in 2017: Overcoming Challenges to Capitalize on Success! Gregory Sawicki, MD, MPH Director, Cystic Fibrosis Center Division of Respiratory Diseases Boston Children’s Hospital Assistant Professor of Pediatrics Harvard Medical School

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: F 508 del 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 FEV 1 102% 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 – FEV 1 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 FEV 1 (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 FEV 1 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 Newborn Screening Diagnosis Infancy Infant Care Practices

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

Nutritional Assessment Pulmonary Function Assessment Monitoring for Complications Adulthood Prevention of Disease Progression Education About CF Assessment of Family Functioning and Social Supports Adolescence Childhood Transition Newborn Screening Diagnosis Infancy Infant Care Practices Self-Management Shared Decision-Making

Challenges to Achieving Optimal CF Outcomes • Adherence to Chronic Therapies • Transition from Pediatric to Adult-Focused CF Care

Complexity of CF Treatment Dietary & vitamin supplements Pancreatic enzyme replacement Chest physiotherapy Daily medical treatments High Treatment Burden Respiratory medications Bregnballe, et al. Patient Prefer Adherence. 2011; 5: 507 -15. Sawicki, et al. Pediatr Pulmonol. 2012; 47(6): 523 -33.

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Daily Treatment Burden for Adults Median Number of Therapies Median Number of Minutes (Total = 108) Inhaled Nebulized Oral Total 1 Exercise 29 2 Nebulized 41 4 7 Sawicki, et al. J Cyst Fibros. 2009; 8(2): 91 -6. Airway Clearance 29 Oral 9

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)e 473– 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 Family • Age • Gender • Health literacy • Disease & treatment knowledge • Mental health / behavioral problems • Coping style • Health beliefs & perceptions • Family structure • Income / health insurance • Disease knowledge • Mental health / behavioral problems • Coping style • Health beliefs & perceptions • Relationship quality • Involvement in care Health Care System Community • Access to care • Continuity of care • Patient-provider communication • Shared decision making • Frequency of clinic visits • Provider biases • Neighborhood • Work (hours & policies) • School • Peer support • Illness stigma Adapted from Modi AC et al. Pediatrics. 2012; 129(2)e 473– 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 – Daily diaries – Questionnaires – Interviews • Clinician-report – Questionnaires • Pharmacy records – Medication Possession Ratio (MPR) – Proportion of days covered (PDC) – Number of refills • Electronic monitors – MEMS Caps – “Chipped” devices – MDI monitors 30

Identifying non-adherence is challenging PATIENT REPORT PROVIDER REPORT 0 Daniels T et al. Chest 2011; 140(2): 425– 432.

Adherence in CF: Adolescent Perspectives • Barriers Immediate time pressures – Lack of time – Uncertain schedules • Competing priorities – Balancing time trade-offs • Privacy concerns – Wanting to be “normal”; not wanting to seem different or disabled • Lack of perceived consequences – Not seeing an impact on one's health right away from skipping treatments or medications Facilitators • Improving understanding of the importance of therapies • Fostering relationships with the CF care team – CF team should be creative in problem-solving with the adolescent and parent • Empowering adolescents – Enabling parents to cede control and entrust responsibility to adolescents • Establish a structure – 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 a linear view Adherence Sustaining Daily Care team – Patient Family Interaction Treatment plan Expectation of adherence to treatment plan To a cyclical view of Sustaining Daily Care Ongoing collaborative conversation between care team and patient/family Adapting plan into daily life Co-create the right treatment 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) 2) 3) 4) 5) Include measures of adherence and self-management that are validated for use in CF. Be patient-centered and empower individuals with CF. Be tailored to people’s unique strengths and needs. Be feasible, practical and adaptable. Leverage collaborations with family, CF Care team, and community.

STRC Sites and Members 17 Institutions Ø 13 Pediatric programs Ø 8 Adult programs 31 Principal Investigators Ø 20 Physicans Ø 10 Ph. Ds – Psychology, Health Economics, Health Education Ø 1 Pharm. D

Health Care Transition: A Health System Dilemma

The Current State of Transition in the U. S. Pediatric Care Faith, Trust, and Pixie Dust Adult Care

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 selfdetermination • 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 adultoriented services with uninterrupted services (when appropriate)

≠ TRANSITION TRANSFER

Should we be transferring everyone? Adults, including those with childhoodacquired 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 2/3 rds discuss current sexual activity or fertility intention • Half of programs never or rarely prepare medical summaries Mc. Laughlin, S. E. et al. Pediatrics 2008; 121: e 1160 -e 1166

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 Reimbursement Insurance/SSI Employment Independent Living Access Time Knowledge

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 Preparation for Transfer Adult care Planned Hand-off Establish YA 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 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