Abstract
A disease without cure
There is currently no cure for cystic fibrosis (CF). Notwithstanding that, over the past 10 years there have been great improvements in the health status, life expectancy and therefore quality of life for people with CF. There is no single breakthrough in care that has made this difference but rather the introduction of small incremental changes and a new ethos of care. These include better surveillance of lung disease, better recognition of symptoms, tailored antibiotic therapy, a better understanding of the role of inflammation and infection on the lung and improved nutrition. 1 The importance of caring for the vital childhood years to secure a better long-term future has mobilized CF teams to adopt programmes of early intervention and vigilance. There is good reason to maintain optimism with genetic and specific mutation research gathering pace and offering opportunity to halt deteriorating lung disease.2,3 But it is hard work for all involved.
DECLARATIONS
None declared
None
Not applicable
MB
Both authors contributed equally
A community with expectations
There are now expectations in the CF community that the previous improvements in health status will continue and publications state a clear goal of a higher predicted median life expectancy in children born today. The CF team has a daunting task to ensure this goal is achieved. International agreement on parameters of health status have led to established national databases in which comparisons of health outcome can be made between national centres and between countries.4–6 In effect ‘league tables’ of success have come into being. This international publication of health outcomes in CF has challenged paediatric CF centres. Standards and pathways for treatment are being set. 7 Major centres are required to coordinate and orchestrate these standards in shared or network care arrangements in peripheral clinics. There is an unstated but not unreasonable expectation that all paediatric centres in the UK will transfer their adolescents to adult CF centres with near normal lung function, normal nutritional status and normal quality of life. With the arrival of newborn diagnostic CF screening across the UK and Ireland, there is an even greater expectation for prevention of disease and maintenance of psychosomatic health. This is an enormous challenge when the pathophysiology and factors determining structural decline are not yet fully understood.
A disorder that requires a team approach
It has long been accepted that the best methods of care provision in chronic paediatric medical conditions has been via a multidisciplinary approach. Traditionally the core CF team comprises a CF paediatrician, nurse specialist, dietitian, physiotherapist, psychologist and social worker. 8 Access to other key specialties such as gastroenterology, endocrinology, lung function laboratories, radiology and microbiology are essential. Fully staffed and committed teams have the best chance of achieving standards and monitoring outcomes. There is however a potentially problematic area in this scenario: improved health outcomes reston more frequent surveillance, quick recognition and response to symptoms, prophylactic treatment and more aggressive, complex and intrusive treatment when infection is identified. Much of the responsibility for this increasingly complex and vigilant management lies squarely on the shoulders of the parents.
Staying healthy is hard work
Daily treatments for paediatric CF are a burden. Sawicki et al., 9 from a study population of 204 CF patients, determined that the median number of daily therapies was seven with 108 min per day mean time spent on treatment. The number of prescribed medications or time to complete therapies did not differ according to age or lung function status as rated by FEV1, which appears intuitively wrong. The mean rated score on a quality of life measure (CFQ-R) for the treatment burden domain was 52.3 (100 = no problem) with no significant differences in the treatment burden based on age or FEV1. Is it no surprise then that adherence fluctuated from the set standard in this group with only 49% performing airway clearance (ACT)? Interestingly the study found that using two or more nebulized medications and performing ACT for greater than or equal to 30 min were significantly associated with increased treatment burden. This is important information for health practitioners in predicting adherence to prescribed medications. 9
Poor adherence to treatment regimens in CF and other paediatric chronic health conditions is frequently reported. 10 Reports of 27–46% adherence rates in CF depending on the treatment, with airway clearance the least adhered to therapy. 11 General paediatric research indicates that less than 50% adherence overall to prescribed treatments. 12 Understanding and measuring adherence in children is difficult but there are some cited correlates including: psychological/emotional, time constraints, complexity, family issues, treatment-related problems and no perceived benefit. It is significant that a recent randomized controlled study to assess efficacy of different types of chest physiotherapies had to be abandoned because more than half the participants who had volunteered for the study found the minimum recommended schedule for therapy was too much. This was despite the fact that the schedule was the set agreed professional standard for normal practice in CF management. 13
Parents – the hitherto unacknowledged team members
Parents are the hidden and unacknowledged members of the CF multidisciplinary team. They are often perceived by the medical team as one might view a patient, i.e. the recipients of care. But they are not the patient, they are the intermediary. The relationship between paediatrician and parent has an unsaid assumption that the parent will agree with the doctor, report symptoms accurately and perceive the management of CF from the same perspective. This may be an assumption too far. There are documented findings that doctors and patients do not always share the same view on the diagnosis or the treatment in adult general practice medicine. When medications fail to produce benefit, the patient may respond by varying the dose or selecting an alternative. 14
Repeated reports of poor adherence to treatment in paediatric CF lead the health professionals to conclude that the parents and children are to blame. Interventions to improve adherence focus on changing the behaviour of the parent or child: family systems management, 15 motivational interviewing 16 and token economy reward systems. 17 It is still the case that: ‘From a medical perspective patients who do not comply with doctors orders are usually seen as deviant and deviance needs correction. But many chronically ill people view their behaviour differently, as a matter of self-regulation’ (Box 1). 18
Case example A
Patient A
First child of consanguineous parents from Pakistan
Born in the UK and enters newborn screening programme
Heal prick shows raised IRT
Genotype confirms two different CF mutations
Parents given appointment for sweat test and stool elastase
Parents refuse sweat test or any further testing; strongly refute diagnosis; self-discharge and request no further medical contact
Their reasons are given as: We have a healthy baby, she has no symptoms, a CF diagnosis would be a stigma in our community, your testing could be wrong, we have no family history of illness. We therefore refuse to engage further.
What should the CF team response be to this scenario?
This is a child at risk
The parents have been told the diagnosis of a life-limiting illness
They are refusing to accept therapy or monitoring
When should parents take the blame?
Are health professionals correct in blaming the parents? Is the evidence as good as we think? How do we maintain a balance between delivering evidence-based practice within a context of patient-centred care by parents? The standards and expectations on health professional performance are high. Paediatric centres provide evidence-based care and strive for best health outcome. But the best health outcome is not one based on pharmacological and physiological success alone but a combination of prescribed treatment and what a parent/patient can bear for a decent quality of life. The best outcome at any one time for a child and family may not be the same as that desired by the doctor. It is difficult for health professionals to acknowledge that there is a patient agenda which is different from their own and dictates whether treatments are done or not. The medical team have a duty of care to diagnose, treat and advise. Listening to the parent perspective however, can be enlightening (Box 2).
Case example B
Patient B
First child of healthy professional parents
Heel prick test shows raised IRT
Genotype confirmed as homozygous DF508
Sweat test positive, stool elastase not detected Parental response to medical therapy
Father: I don't want my child to have the burden of this diagnosis; it makes him a lesser person growing up with illness. How can I be a positive role model unless I ignore his diagnosis?
Mother: Everyone tells me he is a happy baby, I'm his mother and I wipe the smile off his face several times a day when I give him creon, fluclox and vitamins. I am told that I will be doing this increasingly and every day of his childhood. This cannot continue.
It must also be acknowledged that while improvements in the health status of people with CF cannot be denied, the evidence that backs the high standards of daily treatment for these children is not as robust as we might think. Evidence for effective treatment in paediatrics ranges from around 15–40% of available treatment techniques and these are mostly in oncology. Research evidence in CF is frequently based on lung disease surrogate measures such as FEV1, a surrogate marker with significant limitations. 19 Although the randomized controlled trial is accepted as the gold standard method for defining treatment efficacy, they are limited in scope because they take approximately eight years to complete from conception to publication and have a duration of reported efficacy of only five years before the treatment is superseded. 20 Finally, and fundamentally, as already mentioned we do not yet know what causes lung disease or when it starts: is it inflammation, infection, or alterations in the air surface layer? In routine clinical practice when health professionals are asked to report the reasoning behind their decision-making for prescription of various therapies, the findings are intriguing. In a study of cardiologists in Boston the top reason for medical action was ‘Experience/anecdote: based on a memory of one or more cases’. Though parental preference for treatment was a cited reason, research evidence, be it limited, general or specific, came very low down the list below reasons such as ‘trained to do it that way’, and ‘arbitrary/instinct’. 21
Can one be both parent and healthcare provider?
It is helpful to consider the interpersonal dynamics of parent–child relationships when considering why treatment may not conform to prescribed standards. We might now accept the shift away from the term compliance, with the implication that doctor has prescribed and patient should do as they are told; we may now accept adherence and even concordance – terms which imply the collaboration and involvement of the patient in the treatment decision – a fine goal. However, in paediatrics, the problem remains one potentially of compliance. The doctor–parent interaction may have been collaborative but the parent must go home and get their child to comply! This therefore is an issue of parenting and behaviour management which has nothing to do with deliberate refusal to follow doctor's orders. In all families parents and children train each other to not do what they want. This is a process of interaction called ‘coercive family process’ (Box 3). 22
Coercive family process
Step 1: Application of aversive event
Parent: Turn off the TV and do your physiotherapy/nebulizer…
Child feels punished, and attacks with:
Step 2: Child coercive response
Child: Stop going on, you are always getting at me, I will do it LATER!!
Parent feels punished, either attacks back with increased anger or resorts to:
Step 3: Removal of aversive response
Parent withdraws initial demand and walks away, mumbling and grumbling
Outcome 1: Child's refusal and outburst are rewarded as effective techniques to avoid treatment
Outcome 2: Parent walking away removes the child's outburst so parent learns not to insist on treatment
In all families, parental control of child behaviour is a potentially central issue. Problems with sleeping behaviour, eating and physiotherapy are often seen together and are more likely to occur when the major caregiver has mental health needs themselves. In these cases it is recommended that services target child problem behaviours generally not just adherence to treatment issues. 23 Unhappy and conflicted family relationships have been found to be at greater risk for poor adherence and so recommended intervention would be for family therapy generally. 24
CF can occur in any family, irrespective of socioeconomic, supportive resources or mental health circumstances. Some families have psycho-social functioning difficulties such that CF is not the only challenge for that family, nor may it be perceived as a priority. In such families, no matter how effective the treatment, the health outcome for the child may be poor.
Should the team intervene when a parent lets them down?
Let's return to Patient A (Box 1). Is parent failure to comply with health professional prescribed treatment or medical management a case of neglect? The National Society for the Prevention of Cruelty to Children in the UK defines neglect as ‘The persistent failure to meet a child's basic physical and/or psychological needs likely to result in the serious impairment of the child's health or development’ (see
The legal issues in proving neglect as a result of poor adherence to medical recommendations is fraught with difficulty: 25
The standard of care for CF appears to rest purely upon medical judgement. The mere opinions of doctors, however learned, are never sufficient alone to constitute factual truth. The learning of medical professionals may make it more likely that the truth will lie in the direction of their opinions, but this is not guaranteed;
Medical knowledge must be factually correct in order to accuse parents of non-compliance resulting in deteriorated condition. The evidence in CF is still in the early stages of scientific proof;
The requirement to comply is grounded in an assumption that medical advice is good and this assumption can be flawed. The medical practitioner may not have explained clearly, may not have listened to the parental concerns, the treatment may have been too complicated, and so on.
Families may not fall into a clear case of neglect, but may be performing treatment at a suboptimal level. This in itself needs clarification because lifestyles that paediatricians find objectionable do not themselves constitute neglect, or suboptimal parenting. Judicial intervention even if not resulting in child care proceedings, can he helpful when there is an issue over non-compliance for a single treatment event, e.g. blood transfusion. But it can be worse than unhelpful in chronic illness where long-term contact with the medical multidisciplinary team is seen as best practice. Therefore, the best response is to mobilize resources and support. The notion of a continuum of care standards may be helpful in this respect:
Legal definitions of neglect can be agreed but the difficulty is the point at which these behaviours become neglect. A decision must be reached about the threshold at which sub-optimal patenting becomes neglect. Australian Institute of Family Studies (see
Some clarification of the position is required:
It is clear when parents fall into child protection categories;
This is rare in CF;
Parents who do not administer treatment in CF exactly as prescribed by health professionals are so common as to be the norm and do not constitute a case for neglect of parental duties;
Some families require specific help and support;
Bio-ethicists would argue the importance, ethically, of autonomy: respect for the individual and their ability to make decisions with
regard to their own health and future. Actions that enhance autonomy are thought of as desirable. Actions that ‘dwarf’ an individual and their autonomy are undesirable. 26
Parents as equal team members against CF
The health professional's duty of care may be best expressed in CF as part of a delivery-of-care model whereby the parent is explicitly included as a member of the treatment team. Parents have a role of inclusion with respect to their child, with the rights to full explanation and expression of preference. Parents and professionals should not be construed as ‘us’ and ‘them’ but as partners on the same side with CF disease and complications as the shared target. This suggests that inclusion and perhaps improvement of the doctor–parent relationship is the desired goal. For such a relationship to be successful there are rules of practice: Both doctor and parent have responsibilities in making the partnership work. If the doctor's side has an adversarial grounding, i.e. ‘if you don't do as I say I can assert sanctions’, then it may function but if the relationship is grounded supportively ie in a framework of mutual trust then it can function and flourish. For longitudinal care such as paediatric CF, this is the framework required. 27 The standard system for CF paediatric teams should be to consider the parent as inclusive and aim for partnership with parents, collaboration for treatment decisions, shared treatment goals and health outcomes, recognition of family pressures, and consider family and child quality of life parameters as valid. Returning again to Patient A (Box 1), perhaps the best outcome would be:
The parental perspective is accepted but challenging for health practitioners as it risks the onset of disease and potentially reversible or preventative changes;
Consider allowing the parents to drop the label of CF but agree regular low-key medical monitoring at a CF centre;
Encourage monitoring and maintenance of health in their child;
Work on maintaining a relationship with parents – understanding their perspective, develop mutual respect – recognizing that this may be a slow process that takes time.
In this way a relationship is established where the child may have access to medical monitoring but remain in a family who are protective of the child's overall quality of life. To threaten child care proceedings in this or similar cases would destroy any chance of a doctor – patient relationship, and that would be the worst health outcome for the child.
Footnotes
Acknowledgements
None