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Question 12: What do you consider when discussing treatment adherence in patients with Cystic Fibrosis?
Accepted Manuscript Title: Question 12: What do you consider when discussing treatment adherence in patients with Cystic Fibrosis? Authors: Mon Ohn, Dominic A. Fitzgerald PII: DOI: Reference:
S1526-0542(17)30039-8 http://dx.doi.org/doi:10.1016/j.prrv.2017.04.002 YPRRV 1214
To appear in:
YPRRV
Please cite this article as: Ohn Mon, Fitzgerald Dominic A.Question 12: What do you consider when discussing treatment adherence in patients with Cystic Fibrosis?.Paediatric Respiratory Reviews http://dx.doi.org/10.1016/j.prrv.2017.04.002 This is a PDF file of an unedited manuscript that has been accepted for publication. As a service to our customers we are providing this early version of the manuscript. The manuscript will undergo copyediting, typesetting, and review of the resulting proof before it is published in its final form. Please note that during the production process errors may be discovered which could affect the content, and all legal disclaimers that apply to the journal pertain.
CYSTIC FIBROSIS FREQUENTLY ASKED QUESTIONS Question 12: What do you consider when discussing treatment adherence in patients with Cystic Fibrosis? Submitted April 9th 2017.
Mon Ohn1,2 MBBS FRACP MMed (Clinical Epi)
Dominic A Fitzgerald 1,2 MBBS PhD FRACP 1. Department of Respiratory Medicine, The Children’s Hospital at Westmead, Sydney, NSW, Australia. 2. Discipline of Child and Adolescent Health, Sydney Medical School, University of Sydney, Sydney, NSW, Australia.
Corresponding author Professor Dominic A. Fitzgerald Department of Respiratory Medicine The Children’s Hospital at Westmead Locked Bag 4001 Westmead, NSW, Australia, 2145
Text 2679
Figures 0
References - 34
The successful management of any chronic disease demands life-long commitment of adherence to therapy. Logically, it is assumed that poor adherence can have serious negative consequences on quality of life and life expectancy. Nonetheless, poor treatment adherence is a common problem and its aetiology is multifactorial. Cystic fibrosis (CF) is a chronic, progressive and degenerative condition which has improving therapies but no cure. The advances in prevention and treatment regimen have contributed to increased longevity, with the median life expectancy now into the fifth decade.1 From the paediatric perspective, aggressive management of cystic fibrosis lung disease and nutrition allows patients to enjoy normal activities, optimise education and vocational opportunities into adulthood. The price for an improved life span is the commitment of adherence to a time-consuming, repetitive, labour intensive and often complicated daily treatment regimen.
The complex management strategies involve multiple interventions. At a minimum, patients are required to do a high level of daily treatment activities: airway clearance, physiotherapy and exercises, inhaled antibiotics and mucolytics, a high-fat, high-calorie diet and extra nutritional supplements, pancreatic enzyme replacement therapy and scheduled frequent visits to the CF clinic. Additionally, patients with CF-related diabetes are required to check blood glucose levels daily and adjust their diet and insulin requirements accordingly. The treatment intensity becomes more demanding and socially limiting when a patient experiences a pulmonary exacerbation, whether managed in the home or in hospital. The emotional impact of CF and its treatment is often underestimated. Qualitative studies have shown that children and adolescents with CF experience a sense of physical and emotional vulnerability, resentment, loss of independence and opportunities, isolation, and disempowerment.2 Poor compliance with medical advice and interventions in CF is well-documented in the literature. Adherence rates in CF patients are estimated to be around 40% − 50%.3,4 Although there may be no conclusive association between poor compliance and disease progression, poor adherence has been associated with lower lung function and more frequent pulmonary exacerbations.3 Issues around treatment adherence are often complicated and multidimensional. Evidence indicates that various factors influence the adherence patterns.5,6 Barriers to adherence Although effective treatment is essential for the stability of a chronic illness, the demands of different treatment tasks can impose a substantial burden on children and families. Some of the common reasons suggested for poor adherence are inadequate knowledge, lack of time, lack of motivation, underestimation of disease severity, and overestimation of the level of self-care, dynamics of the patient-health provider relationship, psychosocial factors and emotional resistance.4 Time is a rare commodity Time is the fundamental resource and families of children with chronic illnesses are often under considerable time pressure. Studies have reported that families of children with chronic conditions need to spend a substantial amount of time to complete recommended therapies at home.7,8 Ziaian et al. reported on 48 CF children, documenting substantial amounts of time spent each day for various treatments; on average, children with CF reported spending 74.6 +/- 57.0 min per day completing the treatment tasks.7 The authors concluded that although this was not related to health-related quality of life, it could impact upon the ability to comply with all home-basedtherapies for some children. Additionally, it is not an easy task for carers to balance the daily CF demands with the needs of other family members. It is important that treatment demands are kept at a manageable level, so that they can be implemented regularly without imposing excessively upon the family cohesion. Understanding the disease course The level of adherence is linked with parents’ or patients’ knowledge of the disease condition.9,10 Limited understanding may be underplayed by family members, unrecognized by the team and therefore may not be addressed appropriately.6,9 Assessment of knowledge is a key part of understanding of the disease. This is a dynamic phenomenon, with ebbs and flows, requiring confirmation that the knowledge imparted by the team has been understood by the family and the patient. Patients and families who understand the importance of adherence to therapy and who have confidence in their treatments are more likely to be adherent.11 Therefore, it is important to
convey adequate treatment advice which is targeted at the level of understanding and is easy to follow. Furthermore, it is important to appreciate that there can be a divergence in perception and opinion between parents and care-givers, which includes extended family members. This needs to be explored by team members, especially when inconsistencies to treatment are apparent, as may occur in shared care parental custodial arrangements. Here the ability to find common ground through further explanation of the disease and workable, even if sub-optimal, solutions to treatment obstacles is required. Perception of severity and adherence The association between perceived severity and adherence may be complex. Adherence is low when severity is perceived as high or low while a patient’s adherence is better with moderate disease severity.12 Severity of disease can influence and be influenced by adherence.13 Patients tend to see their health issues as less severe than their true status.12,14 Such incorrect perceptions may engender poor adherence which results from a selective understanding of their disease.4 However, it is interesting to note that well informed families may display psychosocial resistance despite the CF team’s best efforts at repeated education to patients and families.15 Patients tend to overestimate their adherence to interventions while their CF team members may have different perspectives.12,14 Arias-Llorente et al. observed in their study that only 70% of selfdescribed compliant patients were considered to be so by their CF team members.8 This is in agreement with previous studies which showed that self-reported adherence consistently exceeds the objective information of adherence, for instance measured with prescription records.16,17 Health professionals need to explore the patients’ and families’ belief systems and effectively communicate the realities of the disease condition, its progression and consequences for everyday activities into adulthood. Open and honest dialogue may reveal barriers to non-adherence. The importance of adherence should be reinforced at each visit. Perception of treatment benefits The perceived personal barriers to treatment can have a negative impact on the health behaviours.18 This may be reflected in how patients and families are able to appreciate the value of treatment and the consequences of not undertaking the treatment. Patients display better adherence with some treatments if they believed those to be more important and to have more repercussions for the quality of life.4,8,12 Adherence can be worst among the most severe cases due to lack of noticeable beneficial effects of the treatment.8,17 Adherence is treatment specific It is known that there are different levels of adherence to the different components of various treatments. Arias-Llorente et al. explored 34 CF patients’ perceptions regarding the importance of the different types of treatment on a scale from 1 to 10. The authors found that patients assigned the highest score (9.4 points) to digestive medications including pancreatic enzyme replacement therapy.8 This reflects the immediate negative consequences of malabsorption on quality of life which are rapidly appreciated. Generally, adherence to nutritional supplements and physiotherapy is low (5%-40%) while better compliance with pancreatic enzymes and antibiotics (75%-90%) has been observed in previous studies. 10,17
Most of the CF patients tend to downplay the importance of physiotherapy in their daily routine.8,10 The main reasons offered are lack of time, perception of little reward or they can have alternative activities instead of physiotherapy.4 Therefore, poor adherence can be partly explained by patients’ perceptions with regard to the benefits and need for each type of treatment, which again emphasises the importance of parental and patient education. Perception of treatment burdens Another factor which may influence adherence is perceived treatment burden. With progression of disease the demands for therapies increase, and the balance of treatment burden and its intrusion on the social life of young people may shift perceptions of the benefits of therapies.8,16 Team members should not underestimate the effect of peer pressure in the school playground with regard to taking enzymes or embarrassment that may come with needing to leave classroom to go to the bathroom urgently. Providing support to the child through better understanding of the teachers and class mates can be achieved with school visits from CF nurses and the provision of written information. Similarly, if the treatments are too complicated to understand or too time consuming, they can be perceived as very stressful and consequently there will be less adherence to therapy.19 A study with 37 CF children by Modi and Quittner showed that fewer treatments was associated with a better adherence rate.6 The level of “inconvenience” to the patient and family resulting from the treatment burden cannot be under-estimated.20 As a step towards improving treatment adherence, it is important to assess both objective and perceived treatment burden with an emphasis on what is practical and achievable for that individual patient and family.18 Age Age plays an important role in maintaining good adherence, perhaps best considered as a “U” shaped curve. For younger children, parents or carers are responsible for the treatment. Parents initially both initiate and implement home treatments. As the child becomes older, there will be a transition of responsibility from the carers to the young person, reflecting the appropriate developmental need to increasingly share the responsibility for initiating and completing therapies. Lower adherence is generally observed during the parent-child responsibility transition period with less parental supervision.13 Adolescent years can be quite challenging. Teenagers have educational and social demands while progressing to adulthood. For CF teenagers, those challenges are exaggerated with the CF care demands. Consequently, the level of adherence tends to decline during adolescence, before improving as adulthood approaches.8,16 Therefore, as frustrating as it may seem for clinicians, there is often much more effort and patience required to support their transition to adult healthcare. Family functioning and structure One of the major challenges for families is to have a balance between adherence to daily CF care and the needs of other family members.13,20,21 This will be influenced by the family dynamics.22 White et al. conducted interviews on 52 CF patients between the ages of 9 and 17 and concluded that cohesive and balanced families showed significantly higher rates of adherence to CF treatment.23 Studies have shown the link between the family routines and adherence. It was reported that the more routine the family behaviours were, the more treatments were undertaken.13 Therefore, it is important to help families to establish daily and weekly routines to ensure family members could
also participate in other activities. Families should be provided with strategies for organization, for example, mobile phone reminders for treatments such as inhaled medications or day planners.11 Treatment Complexity Medications and devices for CF are complex and time-consuming to administer.24 An adult study showed that patients spent an average of 41 min/day on nebulized therapies.19 As the complexity of treatment impacts adherence, there is a need for simplification of the treatment plan and improved delivery systems [eg ultra-fast nebulizers] to help achieve good adherence.19 Ways of coping with the disease Patients and parents display different styles of coping with their disease conditions. Coping behaviours have an impact on adherence.25 A higher level of positivity can be related to a greater level of adherence.12,25,26 On the other hand, high parental anxiety and depression can be barriers to adherence.25 It is also important to note that denial and avoidance can reflect a families’ coping mechanism for dealing with CF. Positive coping strategies, for instance, positive reframing, acceptance and humour are associated with a positive outlook and adherence.12,27,28 Therefore, emotional support to families is important and should be reflected in the clinicians’ approaches to interactions with the patients and families.29 Monitoring adherence The measurement of treatment adherence can be difficult. Patients’ self-reports and clinicians’ impressions are not accurate enough to determine the actual degree of adherence to treatments.4,5 Therefore, different methods have been employed to collect objective information, often in the research setting. These have included quality of life questionnaires, symptom diaries, objective therapeutic response measures [e.g. FEV1; weight gain], urinary or serum drug levels, and electronic recording devices to monitor medication dispensing.5 However, each method of monitoring may have its own drawbacks.5 For instance, a blood sample for a serum drug level is an invasive method, recall biases may be associated with self-reporting and electronic monitoring devices are expensive. Therefore, it is not easy to accurately measure treatment adherence in the clinical setting. Nonetheless, it is important to maximise adherence rates and this is practically achieved through building a rapport with families over time. How to enhance adherence? It is important for CF team members to understand the coping model of families to be able to deliver the interventions to promote their adjustment. Understanding of what therapies patients and families actually complete and factors influencing their choice of therapies is important. Such knowledge may help CF team members to develop strategies to enhance adherence for individual patients. CF children display multiple problems at different stages of their life. Although these issues are not necessarily specific to CF, they may have a more significant impact with a chronic illness like CF. This includes mealtime issues in young children, issues with oppositional behaviours in school aged children30 and treatment refusal in adolescents who just want to be “normal” like their peers.2,31 Consequently, parenting guidance should be part of the advice offered to help tackle some of the normal developmental “adherence” issues.30 Previous studies have shown that children and adolescents with CF and their parents have higher depression rates.32 As there is an association between poor adherence and depressive symptoms,
psychotherapeutic approaches may help to enhance adherence. Behavioural modification programmes focusing on positive reinforcement result in better adherence.30 It is important to take the mental wellbeing of carers into consideration and referral for psychological support through counselling services should be undertaken if required. As poor or variable adherence, more typically than non-adherence, stems from multiple considerations, it is important to understand the individual’s adherence behaviours. It is helpful to reinforce the positive perceptions, health beliefs and coping mechanisms in order to foster adherence behaviours. Open dialogue may reveal previously unknown barriers to non-adherence. Supportive and collaborative relationships between the CF team and families are necessary so that patients and families can report their adherence honestly without feeling judged. Consequently, interventions should be tailored to the patients’ and families’ specific adherence patterns rather than presuming that a blanket approach should be successful.33 Due to the busy nature of outpatient clinics, CF team members may not routinely ask about patients’ understanding of their conditions.6 However, team members should aim to assess the educational needs at each visit. Providing information in a layered fashion [building upon acquired knowledge] is one of the ways to enhance adherence. Skills training, for example in how to use a nebulizer or a positive expiratory pressure [PEP] mask for airway clearance, should be incorporated into education sessions and the technique reviewed periodically to ensure optimum results with the treatment modalities. Time constraints are a major barrier to adherence. Families should be equipped with practical suggestions as to how to accommodate CF care with other social-educational needs like organised sport or school activities. Such practical guides are available through resources from most large CF clinics, websites [www.cff.org, www.cysticfibrosis.org.au, www.cftrust.org.uk, www.nhlbi.nih.gov/health/dci/Diseases/cf/cf_what.html] and on-line and print books34. Strategies to encourage adherence need to be age appropriate and minimally burdensome. Reminders using mobile phone apps for therapies may be provided now with the hope that novel delivery devices and technological advances may be more useful for fostering adherence in future.11 Conclusion Although excellent adherence may not necessarily guarantee good health, it is appreciated that the lack of adherence can lead to more rapid disease progression.4 Improving compliance is challenging and concerted efforts within the multi-disciplinary CF team are necessary to overcome barriers to poor adherence. Poor adherence is usually multifactorial in nature. Strategies to tackle such a common problem include having non-judgmental negotiation between CF team and families, exploring the barriers to poor adherence, improving patient education, providing adequate socio-familial support, developing simplified, individualized interventions and keeping the treatment plan as least burdensome as possible.
Acknowledgements The authors would like to thank Professor Larry Lands for his review of the manuscript.
References 1.
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8. 9.
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Stephenson AL, Sykes J, Stanojevic S, Quon BS, Marshall BC, Petren K, et al. Survival Comparison of Patients With Cystic Fibrosis in Canada and the United States: A PopulationBased Cohort Study. Ann. Intern. Med. 2017. Jamieson N, Fitzgerald D, Singh-Grewal D, Hanson CS, Craig JC, Tong A. Children’s Experiences of Cystic Fibrosis: A Systematic Review of Qualitative Studies. Pediatrics. 2014;133(6):e1683-e1697. Townsend M, Kidwell K, Nasr S. Adherence To Medications In Cystic Fibrosis Patients: A FiveYear Retrospective Analysis. PEDIATRIC CYSTIC FIBROSIS.2014:A5530-A5530. Arias-Llorente RP, García CB, Martín JJD. The Importance of Adherence and Compliance with Treatment in Cystic Fibrosis, Cystic Fibrosis - Renewed Hopes Through Research. 2012. Modi AC, Lim CS, Yu N, Geller D, Wagner MH, Quittner AL. A multi-method assessment of treatment adherence for children with cystic fibrosis. J Cyst Fibros. 2006;5(3):177-185. Modi AC, Quittner AL. Barriers to treatment adherence for children with cystic fibrosis and asthma: what gets in the way? J. Pediatr. Psychol. 2006;31(8):846-858. Ziaian T, Sawyer MG, Reynolds KE, Carbone JA, Clark JJ, Baghurst PA, et al. Treatment burden and health‐related quality of life of children with diabetes, cystic fibrosis and asthma. J. Paediatr. Child Health. 2006;42(10):596-600. Arias Llorente RP, Bousono Garcia C, Diaz Martin JJ. Treatment compliance in children and adults with cystic fibrosis. J Cyst Fibros. 2008;7(5):359-367. Ievers CE, Brown RT, Drotar D, Caplan D, Pishevar BS, Lambert RG. Knowledge of physician prescriptions and adherence to treatment among children with cystic fibrosis and their mothers. J. Dev. Behav. Pediatr. 1999;20(5):335-343. Bucks RS, Hawkins K, Skinner TC, Horn S, Seddon P, Horne R. Adherence to Treatment in Adolescents with Cystic Fibrosis: The Role of Illness Perceptions and Treatment Beliefs. J. Pediatr. Psychol. 2009;34(8):893-902. Lomas P. Enhancing adherence to inhaled therapies in cystic fibrosis. Ther. Adv. Respir. Dis. 2014;8(2):39-47. Abbott J, Gee L. Contemporary psychosocial issues in cystic fibrosis: treatment adherence and quality of life. Disabil. Rehabil. 1998;20(6-7):262-271. Leeman J, Sandelowski M, Havill NL, Knafl K. Parent-to-Child Transition in Managing Cystic Fibrosis: A Research Synthesis. Journal of Family Theory & Review. 2015;7(2):167-183. Abbott J, Dodd M, Webb AK. Different perceptions of disease severity and self care between patients with cystic fibrosis, their close companions, and physician. Thorax. 1995;50(7):794796. Koocher GP, McGrath ML, Gudas LJ. Typologies of nonadherence in cystic fibrosis. J. Dev. Behav. Pediatr. 1990;11(6):353-358. Conway SP, Pond MN, Hamnett T, Watson A. Compliance with treatment in adult patients with cystic fibrosis. Thorax. 1996;51(1):29-33. Kettler LJ, Sawyer SM, Winefield HR, Greville HW. Determinants of adherence in adults with cystic fibrosis. Thorax. 2002;57(5):459-464. Dziuban EJ, Saab-Abazeed L, Chaudhry SR, Streetman DS, Nasr SZ. Identifying barriers to treatment adherence and related attitudinal patterns in adolescents with cystic fibrosis. Pediatr. Pulmonol. 2010;45(5):450-458. Sawicki GS, Tiddens H. Managing treatment complexity in cystic fibrosis: challenges and opportunities. Pediatr. Pulmonol. 2012;47(6):523-533. Foster C, Eiser C, Oades P, Sheldon C, Tripp J, Goldman P, et al. Treatment demands and differential treatment of patients with cystic fibrosis and their siblings: patient, parent and sibling accounts. Child Care Health Dev. 2001;27(4):349-364. Lavie M, Shemer O, Sarouk I, Bar aluma Be, Dagan A, Efrati O, et al. Several siblings with Cystic Fibrosis as a risk factor for poor outcome. Respir. Med. 2014;109(1):74-78.
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DeLambo KE, Ievers-Landis CE, Drotar D, Quittner AL. Association of observed family relationship quality and problem-solving skills with treatment adherence in older children and adolescents with cystic fibrosis. J. Pediatr. Psychol. 2004;29(5):343-353. White T, Miller J, Smith GL, McMahon WM. Adherence and psychopathology in children and adolescents with cystic fibrosis. Eur. Child Adolesc. Psychiatry. 2009;18(2):96-104. Sawicki GS, Goss CH. Tackling the increasing complexity of CF care. Pediatr. Pulmonol. 2015;50 Suppl 40:S74-79. Wong MG, Heriot SA. Parents of children with cystic fibrosis: how they hope, cope and despair. Child Care Health Dev. 2008;34(3):344-354. Abbott J, Hart A, Morton A, Gee L, Conway S. Health-related quality of life in adults with cystic fibrosis: the role of coping. J. Psychosom. Res. 2008;64(2):149-157. Abbott J. Coping with cystic fibrosis. J. R. Soc. Med. 2003;96(Suppl 43):42-50. Gudas LJ, Koocher GP, Wypij D. Perceptions of medical compliance in children and adolescents with cystic fibrosis. J. Dev. Behav. Pediatr. 1991;12(4):236-242. Jessup M, Parkinson C. “All at Sea”: The Experience of Living With Cystic Fibrosis. Qual. Health Res. 2010;20(3):352-364. Ward C, Massie J, Glazner J, Sheehan J, Canterford L, Armstrong D, et al. Problem behaviours and parenting in preschool children with cystic fibrosis. Arch. Dis. Child. 2009;94(5):341-347. Patterson JM, Wall M, Berge J, Milla C. Gender differences in treatment adherence among youth with cystic fibrosis: development of a new questionnaire. J Cyst Fibros. 2008;7(2):154164. Smith BA, Modi AC, Quittner AL, Wood BL. Depressive symptoms in children with cystic fibrosis and parents and its effects on adherence to airway clearance. Pediatr. Pulmonol. 2010;45(8):756-763. Grossoehme DH, Szczesniak RD, Britton LL, Siracusa CM, Quittner AL, Chini BA, et al. Adherence determinants in cystic fibrosis: Cluster analysis of parental psychosocial, religious, and/or spiritual factors. Ann Am Thorac Soc. 2015;12(6):838-846. Fitzgerald DA. The Cystic Fibrosis Passport: A Guide to the Needs of Young Children with Cystic Fibrosis. Clareville Press 2013.
S1526-0542(17)30039-8 http://dx.doi.org/doi:10.1016/j.prrv.2017.04.002 YPRRV 1214
To appear in:
YPRRV
Please cite this article as: Ohn Mon, Fitzgerald Dominic A.Question 12: What do you consider when discussing treatment adherence in patients with Cystic Fibrosis?.Paediatric Respiratory Reviews http://dx.doi.org/10.1016/j.prrv.2017.04.002 This is a PDF file of an unedited manuscript that has been accepted for publication. As a service to our customers we are providing this early version of the manuscript. The manuscript will undergo copyediting, typesetting, and review of the resulting proof before it is published in its final form. Please note that during the production process errors may be discovered which could affect the content, and all legal disclaimers that apply to the journal pertain.
CYSTIC FIBROSIS FREQUENTLY ASKED QUESTIONS Question 12: What do you consider when discussing treatment adherence in patients with Cystic Fibrosis? Submitted April 9th 2017.
Mon Ohn1,2 MBBS FRACP MMed (Clinical Epi)
Dominic A Fitzgerald 1,2 MBBS PhD FRACP 1. Department of Respiratory Medicine, The Children’s Hospital at Westmead, Sydney, NSW, Australia. 2. Discipline of Child and Adolescent Health, Sydney Medical School, University of Sydney, Sydney, NSW, Australia.
Corresponding author Professor Dominic A. Fitzgerald Department of Respiratory Medicine The Children’s Hospital at Westmead Locked Bag 4001 Westmead, NSW, Australia, 2145
Text 2679
Figures 0
References - 34
The successful management of any chronic disease demands life-long commitment of adherence to therapy. Logically, it is assumed that poor adherence can have serious negative consequences on quality of life and life expectancy. Nonetheless, poor treatment adherence is a common problem and its aetiology is multifactorial. Cystic fibrosis (CF) is a chronic, progressive and degenerative condition which has improving therapies but no cure. The advances in prevention and treatment regimen have contributed to increased longevity, with the median life expectancy now into the fifth decade.1 From the paediatric perspective, aggressive management of cystic fibrosis lung disease and nutrition allows patients to enjoy normal activities, optimise education and vocational opportunities into adulthood. The price for an improved life span is the commitment of adherence to a time-consuming, repetitive, labour intensive and often complicated daily treatment regimen.
The complex management strategies involve multiple interventions. At a minimum, patients are required to do a high level of daily treatment activities: airway clearance, physiotherapy and exercises, inhaled antibiotics and mucolytics, a high-fat, high-calorie diet and extra nutritional supplements, pancreatic enzyme replacement therapy and scheduled frequent visits to the CF clinic. Additionally, patients with CF-related diabetes are required to check blood glucose levels daily and adjust their diet and insulin requirements accordingly. The treatment intensity becomes more demanding and socially limiting when a patient experiences a pulmonary exacerbation, whether managed in the home or in hospital. The emotional impact of CF and its treatment is often underestimated. Qualitative studies have shown that children and adolescents with CF experience a sense of physical and emotional vulnerability, resentment, loss of independence and opportunities, isolation, and disempowerment.2 Poor compliance with medical advice and interventions in CF is well-documented in the literature. Adherence rates in CF patients are estimated to be around 40% − 50%.3,4 Although there may be no conclusive association between poor compliance and disease progression, poor adherence has been associated with lower lung function and more frequent pulmonary exacerbations.3 Issues around treatment adherence are often complicated and multidimensional. Evidence indicates that various factors influence the adherence patterns.5,6 Barriers to adherence Although effective treatment is essential for the stability of a chronic illness, the demands of different treatment tasks can impose a substantial burden on children and families. Some of the common reasons suggested for poor adherence are inadequate knowledge, lack of time, lack of motivation, underestimation of disease severity, and overestimation of the level of self-care, dynamics of the patient-health provider relationship, psychosocial factors and emotional resistance.4 Time is a rare commodity Time is the fundamental resource and families of children with chronic illnesses are often under considerable time pressure. Studies have reported that families of children with chronic conditions need to spend a substantial amount of time to complete recommended therapies at home.7,8 Ziaian et al. reported on 48 CF children, documenting substantial amounts of time spent each day for various treatments; on average, children with CF reported spending 74.6 +/- 57.0 min per day completing the treatment tasks.7 The authors concluded that although this was not related to health-related quality of life, it could impact upon the ability to comply with all home-basedtherapies for some children. Additionally, it is not an easy task for carers to balance the daily CF demands with the needs of other family members. It is important that treatment demands are kept at a manageable level, so that they can be implemented regularly without imposing excessively upon the family cohesion. Understanding the disease course The level of adherence is linked with parents’ or patients’ knowledge of the disease condition.9,10 Limited understanding may be underplayed by family members, unrecognized by the team and therefore may not be addressed appropriately.6,9 Assessment of knowledge is a key part of understanding of the disease. This is a dynamic phenomenon, with ebbs and flows, requiring confirmation that the knowledge imparted by the team has been understood by the family and the patient. Patients and families who understand the importance of adherence to therapy and who have confidence in their treatments are more likely to be adherent.11 Therefore, it is important to
convey adequate treatment advice which is targeted at the level of understanding and is easy to follow. Furthermore, it is important to appreciate that there can be a divergence in perception and opinion between parents and care-givers, which includes extended family members. This needs to be explored by team members, especially when inconsistencies to treatment are apparent, as may occur in shared care parental custodial arrangements. Here the ability to find common ground through further explanation of the disease and workable, even if sub-optimal, solutions to treatment obstacles is required. Perception of severity and adherence The association between perceived severity and adherence may be complex. Adherence is low when severity is perceived as high or low while a patient’s adherence is better with moderate disease severity.12 Severity of disease can influence and be influenced by adherence.13 Patients tend to see their health issues as less severe than their true status.12,14 Such incorrect perceptions may engender poor adherence which results from a selective understanding of their disease.4 However, it is interesting to note that well informed families may display psychosocial resistance despite the CF team’s best efforts at repeated education to patients and families.15 Patients tend to overestimate their adherence to interventions while their CF team members may have different perspectives.12,14 Arias-Llorente et al. observed in their study that only 70% of selfdescribed compliant patients were considered to be so by their CF team members.8 This is in agreement with previous studies which showed that self-reported adherence consistently exceeds the objective information of adherence, for instance measured with prescription records.16,17 Health professionals need to explore the patients’ and families’ belief systems and effectively communicate the realities of the disease condition, its progression and consequences for everyday activities into adulthood. Open and honest dialogue may reveal barriers to non-adherence. The importance of adherence should be reinforced at each visit. Perception of treatment benefits The perceived personal barriers to treatment can have a negative impact on the health behaviours.18 This may be reflected in how patients and families are able to appreciate the value of treatment and the consequences of not undertaking the treatment. Patients display better adherence with some treatments if they believed those to be more important and to have more repercussions for the quality of life.4,8,12 Adherence can be worst among the most severe cases due to lack of noticeable beneficial effects of the treatment.8,17 Adherence is treatment specific It is known that there are different levels of adherence to the different components of various treatments. Arias-Llorente et al. explored 34 CF patients’ perceptions regarding the importance of the different types of treatment on a scale from 1 to 10. The authors found that patients assigned the highest score (9.4 points) to digestive medications including pancreatic enzyme replacement therapy.8 This reflects the immediate negative consequences of malabsorption on quality of life which are rapidly appreciated. Generally, adherence to nutritional supplements and physiotherapy is low (5%-40%) while better compliance with pancreatic enzymes and antibiotics (75%-90%) has been observed in previous studies. 10,17
Most of the CF patients tend to downplay the importance of physiotherapy in their daily routine.8,10 The main reasons offered are lack of time, perception of little reward or they can have alternative activities instead of physiotherapy.4 Therefore, poor adherence can be partly explained by patients’ perceptions with regard to the benefits and need for each type of treatment, which again emphasises the importance of parental and patient education. Perception of treatment burdens Another factor which may influence adherence is perceived treatment burden. With progression of disease the demands for therapies increase, and the balance of treatment burden and its intrusion on the social life of young people may shift perceptions of the benefits of therapies.8,16 Team members should not underestimate the effect of peer pressure in the school playground with regard to taking enzymes or embarrassment that may come with needing to leave classroom to go to the bathroom urgently. Providing support to the child through better understanding of the teachers and class mates can be achieved with school visits from CF nurses and the provision of written information. Similarly, if the treatments are too complicated to understand or too time consuming, they can be perceived as very stressful and consequently there will be less adherence to therapy.19 A study with 37 CF children by Modi and Quittner showed that fewer treatments was associated with a better adherence rate.6 The level of “inconvenience” to the patient and family resulting from the treatment burden cannot be under-estimated.20 As a step towards improving treatment adherence, it is important to assess both objective and perceived treatment burden with an emphasis on what is practical and achievable for that individual patient and family.18 Age Age plays an important role in maintaining good adherence, perhaps best considered as a “U” shaped curve. For younger children, parents or carers are responsible for the treatment. Parents initially both initiate and implement home treatments. As the child becomes older, there will be a transition of responsibility from the carers to the young person, reflecting the appropriate developmental need to increasingly share the responsibility for initiating and completing therapies. Lower adherence is generally observed during the parent-child responsibility transition period with less parental supervision.13 Adolescent years can be quite challenging. Teenagers have educational and social demands while progressing to adulthood. For CF teenagers, those challenges are exaggerated with the CF care demands. Consequently, the level of adherence tends to decline during adolescence, before improving as adulthood approaches.8,16 Therefore, as frustrating as it may seem for clinicians, there is often much more effort and patience required to support their transition to adult healthcare. Family functioning and structure One of the major challenges for families is to have a balance between adherence to daily CF care and the needs of other family members.13,20,21 This will be influenced by the family dynamics.22 White et al. conducted interviews on 52 CF patients between the ages of 9 and 17 and concluded that cohesive and balanced families showed significantly higher rates of adherence to CF treatment.23 Studies have shown the link between the family routines and adherence. It was reported that the more routine the family behaviours were, the more treatments were undertaken.13 Therefore, it is important to help families to establish daily and weekly routines to ensure family members could
also participate in other activities. Families should be provided with strategies for organization, for example, mobile phone reminders for treatments such as inhaled medications or day planners.11 Treatment Complexity Medications and devices for CF are complex and time-consuming to administer.24 An adult study showed that patients spent an average of 41 min/day on nebulized therapies.19 As the complexity of treatment impacts adherence, there is a need for simplification of the treatment plan and improved delivery systems [eg ultra-fast nebulizers] to help achieve good adherence.19 Ways of coping with the disease Patients and parents display different styles of coping with their disease conditions. Coping behaviours have an impact on adherence.25 A higher level of positivity can be related to a greater level of adherence.12,25,26 On the other hand, high parental anxiety and depression can be barriers to adherence.25 It is also important to note that denial and avoidance can reflect a families’ coping mechanism for dealing with CF. Positive coping strategies, for instance, positive reframing, acceptance and humour are associated with a positive outlook and adherence.12,27,28 Therefore, emotional support to families is important and should be reflected in the clinicians’ approaches to interactions with the patients and families.29 Monitoring adherence The measurement of treatment adherence can be difficult. Patients’ self-reports and clinicians’ impressions are not accurate enough to determine the actual degree of adherence to treatments.4,5 Therefore, different methods have been employed to collect objective information, often in the research setting. These have included quality of life questionnaires, symptom diaries, objective therapeutic response measures [e.g. FEV1; weight gain], urinary or serum drug levels, and electronic recording devices to monitor medication dispensing.5 However, each method of monitoring may have its own drawbacks.5 For instance, a blood sample for a serum drug level is an invasive method, recall biases may be associated with self-reporting and electronic monitoring devices are expensive. Therefore, it is not easy to accurately measure treatment adherence in the clinical setting. Nonetheless, it is important to maximise adherence rates and this is practically achieved through building a rapport with families over time. How to enhance adherence? It is important for CF team members to understand the coping model of families to be able to deliver the interventions to promote their adjustment. Understanding of what therapies patients and families actually complete and factors influencing their choice of therapies is important. Such knowledge may help CF team members to develop strategies to enhance adherence for individual patients. CF children display multiple problems at different stages of their life. Although these issues are not necessarily specific to CF, they may have a more significant impact with a chronic illness like CF. This includes mealtime issues in young children, issues with oppositional behaviours in school aged children30 and treatment refusal in adolescents who just want to be “normal” like their peers.2,31 Consequently, parenting guidance should be part of the advice offered to help tackle some of the normal developmental “adherence” issues.30 Previous studies have shown that children and adolescents with CF and their parents have higher depression rates.32 As there is an association between poor adherence and depressive symptoms,
psychotherapeutic approaches may help to enhance adherence. Behavioural modification programmes focusing on positive reinforcement result in better adherence.30 It is important to take the mental wellbeing of carers into consideration and referral for psychological support through counselling services should be undertaken if required. As poor or variable adherence, more typically than non-adherence, stems from multiple considerations, it is important to understand the individual’s adherence behaviours. It is helpful to reinforce the positive perceptions, health beliefs and coping mechanisms in order to foster adherence behaviours. Open dialogue may reveal previously unknown barriers to non-adherence. Supportive and collaborative relationships between the CF team and families are necessary so that patients and families can report their adherence honestly without feeling judged. Consequently, interventions should be tailored to the patients’ and families’ specific adherence patterns rather than presuming that a blanket approach should be successful.33 Due to the busy nature of outpatient clinics, CF team members may not routinely ask about patients’ understanding of their conditions.6 However, team members should aim to assess the educational needs at each visit. Providing information in a layered fashion [building upon acquired knowledge] is one of the ways to enhance adherence. Skills training, for example in how to use a nebulizer or a positive expiratory pressure [PEP] mask for airway clearance, should be incorporated into education sessions and the technique reviewed periodically to ensure optimum results with the treatment modalities. Time constraints are a major barrier to adherence. Families should be equipped with practical suggestions as to how to accommodate CF care with other social-educational needs like organised sport or school activities. Such practical guides are available through resources from most large CF clinics, websites [www.cff.org, www.cysticfibrosis.org.au, www.cftrust.org.uk, www.nhlbi.nih.gov/health/dci/Diseases/cf/cf_what.html] and on-line and print books34. Strategies to encourage adherence need to be age appropriate and minimally burdensome. Reminders using mobile phone apps for therapies may be provided now with the hope that novel delivery devices and technological advances may be more useful for fostering adherence in future.11 Conclusion Although excellent adherence may not necessarily guarantee good health, it is appreciated that the lack of adherence can lead to more rapid disease progression.4 Improving compliance is challenging and concerted efforts within the multi-disciplinary CF team are necessary to overcome barriers to poor adherence. Poor adherence is usually multifactorial in nature. Strategies to tackle such a common problem include having non-judgmental negotiation between CF team and families, exploring the barriers to poor adherence, improving patient education, providing adequate socio-familial support, developing simplified, individualized interventions and keeping the treatment plan as least burdensome as possible.
Acknowledgements The authors would like to thank Professor Larry Lands for his review of the manuscript.
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