Cystic Fibrosis Care in 2017: - PowerPoint Presentation

Cystic Fibrosis Care in 2017:Overcoming Challenges to Capitalize on Success! Gregory Sawicki, MD, MPH Director, Cystic Fibrosis CenterDivision of Respiratory DiseasesBoston 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

DiagnosisFailure to thrive at age 6 months led PCP to refer for sweat testChronic nasal congestionPositive sweat test with CFTR genotype: F508del homozygousSocial history: Has an older sister who does not have CFFather is an attorneyMother is a teacherHas 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 - PreschoolStarted pancreatic enzymes at time of diagnosisWeight increased from 10%ile to 30%ile by age 2Had reflux as an infantDescribed as a “picky eater”, often refusing meals Started chest PT intermittently at age 8 monthsAttended daycare from age 1 until preschool at age 4Initiated Dornase alfa therapy at age 4Parents decide to have a third child, request genetic counseling, no pre-natal screening available at the time

Elementary SchoolSchool requires him to go to nurse to obtain enzymes. Often forgets.First time Pseudomonas detected at age 5, treated with inhaled antibiotics for eradication therapyNo 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 coughReported intermittent cough, but was the fastest kid on his teamAge 5 began daily chest PT with Vest First attempts at lung function at age 5 with FEV1 102% predictedNever hospitalized

Middle SchoolAge 12: Has soccer practice / games 4 days per week and family reports that they don’t have time for the Vest on those daysBMI has dropped from 40%ile to 35%ileDenies any new GI symptomsParents state that they pack his lunch every day with enzymes in his bag FEV1 at age 12: 95% predictedFirst hospitalization due to increased symptoms, decreasing weight, and re-growth of Pseudomonas

High SchoolContinues to play competitive soccer, also lacrosse and basketballPrescribed alternate month inhaled antibiotics, hypertonic saline, dornase alfa, and daily chest PT BMI continues to drop – 20%ile at age 14Reports “forgetting” his enzymes and doesn’t think he needs them anymoreDevelops influenza at age 15 requiring hospitalizationStarts a summer job as a golf caddy Straight A student, aspires to attend college to study engineeringStarts dating girls at age 16, does not tell friends (except for his 2 best friends) about CF

CollegeComes to CF clinic only while home on holiday breaks Requests single room accommodation in order to complete treatments in privateReports 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 dormMeets adult CF care team for the first time

Post-CollegeBaseline FEV1 60% predictedChronic infection with PseudomonasCare now coordinated by an adult CF CenterHospitalized yearly since age 23 for CF exacerbation Works in a research laboratory as a technician, hoping to apply to graduate schoolsEngaged, considers fiancée his primary support personConsidering having childrenMedication regimen: Inhaled tobramycin, Dornase alfa, Azithromycin, Inhaled sodium chloride, Pancreatic enzymes, Multivitamin plus minerals, Fluticasone propionate , EsomeprazoleConsideration made for initiation of lumacaftor/ivacaftor

Diagnosis Adulthood Adolescence Childhood Infancy Challenges to CF Care Through the Life Span

Diagnosis Adulthood Adolescence Childhood Infancy Newborn Screening Infant Care Practices

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

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

Challenges to Achieving Optimal CF OutcomesAdherence to Chronic TherapiesTransition from Pediatric to Adult-Focused CF Care

Complexity of CF Treatment 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 Sawicki , et al. J Cyst Fibros . 2009;8(2):91-6. Median Number of Therapies Median Number of Minutes (Total = 108)

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 condition24 http://apps.who.int/iris/bitstream/10665/42682/1/9241545992.pdf Modi AC et al. Pediatrics. 2012;129(2)e473–485 .

Quittner AL et al. Chest 2014;146(1): 142–151. Adherence rates to CF respiratory medications are low MPR=0.5

Adherence rates vary by age Quittner AL et al. Chest 2014;146(1 ): 142–151.

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.Adherence in adolescents is best on weekdays during school term time

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 q uality Involvement in care Adapted from Modi AC et al. Pediatrics. 2012;129(2)e473–485 . Community Neighborhood Work (hours & policies ) School Peer support Illness stigma

Adherence TypologiesUnwittingPatient 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-reportDaily diariesQuestionnaires InterviewsClinician-reportQuestionnairesPharmacy recordsMedication Possession Ratio (MPR)Proportion of days covered (PDC)Number of refillsElectronic monitorsMEMS Caps “Chipped” devices MDI monitors 30

0 Patient report Provider report Daniels T et al. Chest 2011;140(2):425–432. Identifying non-adherence is challenging

Adherence in CF: Adolescent PerspectivesBarriersImmediate time pressures Lack of timeUncertain schedulesCompeting prioritiesBalancing time trade-offsPrivacy concerns Wanting to be “normal”; not wanting to seem different or disabledLack of perceived consequencesNot seeing an impact on one's health right away from skipping treatments or medications Sawicki GS et al. Pediatr Pulmonol ,  2015 Feb;50(2):127-36 . 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 ”

What Can Be Done?33

Adherence  Sustaining Daily Care Shift from adherence in a linear view To a cyclical view of Sustaining Daily Care Reframe Adherence to Sustain Daily Care 34

CFF Partnerships for Sustaining Daily Care 35

CFF Success with Therapies Research ConsortiumTo 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 13 Pediatric programs 8 Adult programs 31 Principal Investigators 20 Physicans 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. 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 self-determinationAllow 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

Identify a health provider to coordinate transitionTrain health care providers in transition servicesMaintain a portable medical summaryCreate a written health transition planApply standard primary care guidelines to CSHCN Ensure affordable, continuous health insurance Consensus Statements on Health Care Transition Pediatrics 2011;128:182-200

Time for a Reality Check!

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

What is the Problem?Obstacles to Health Care TransitionThe youth The parent(s) / familyThe pediatric team The adult teamThe health system

Patient-Family Level Barriers Accessing care: primary and specialty care Knowledge and SkillsInsuranceFamily / 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 specialistsLack of pediatric staff skills in transition planningDifficulty 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 Traininglack of training in congenital and childhood-onset conditionslack of adolescent medicine trainingWorking 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

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 ConsiderWhat 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 Preparation for Transfer Pediatrics Adult care Empathic Intake Active Transfer Establish YA in Adult System Therapeutic Discharge Planned Hand-off

A non-CF focused resource:Got Transition! www.gottransition.org

CF R.I.S.E. was developed in collaboration with a multidisciplinary team of CF experts and is sponsored by Gilead. 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 .

Summary Health outcomes in CF have been improving for decadesMany more adolescents with CF continue to reach adulthoodNovel therapies continue to be developedNon-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