Abstract
Duchenne muscular dystrophy is a progressive muscle-wasting condition that only affects boys, most use a wheelchair by the age of 12 and increasing levels of support and medical intervention are needed as they mature into young adulthood. Medical research is working to find treatments and an eventual cure for Duchenne muscular dystrophy, and this research can require the participation of those with the condition. Therefore, boys and young men who have Duchenne muscular dystrophy were invited to discuss their thoughts on how they might make a decision to take part in medical research, and to explore issues impacting their daily lives. Their accounts highlight experiences of agency and contextualised competence, challenging assumptions of vulnerability and immaturity in those who are young and severely disabled. The findings, based on fieldwork in England, enrich understandings of how such lives have meaning, wherein agency, independence and decision-making capabilities are present and exercised, albeit in ways that may be different to ‘mainstream’ notions of the terms.
Introduction
The focus in this article is on some key findings from qualitative semi-structured interviews with boys and young men aged 10–21 who have Duchenne muscular dystrophy (DMD), a degenerative, life-limiting condition. The interviews were part of a funded doctoral study that focused on understanding what boys with DMD think about medical research, how they thought they might approach making a decision to take part and who or what influences their decisions. DMD is a muscle-wasting disease that only affects boys, it is currently untreatable and muscle strength deteriorates as they mature. A wheelchair is required by age 10–13 and health problems develop as muscle strength declines. Better management of DMD has improved life-expectancy, and the use of steroids slows muscle weakness (Bianchi et al., 2011); however, an absence of treatments to halt or minimise disease progression means that respiratory and cardiac complications develop, leading to a fatal outcome in early to mid adult life (Landfeldt et al., 2014).
Medical research is developing treatments to modify the condition, and this research includes clinical trials necessitating the involvement of boys with DMD who might undergo procedures such as a series of injections, or the regular consumption of test drugs to measure safety and efficacy (Condin, 2014). It was out of this necessity that the study emerged, as the process of involvement in medical research raises questions about how much ‘say’ children and young people have when making a decision, how competent they are to be effectively involved and, importantly, how they want to be involved. Those with DMD are under-researched due, in part, to sensitivities in researching disabled childhoods (Cocks, 2006); this study contributes to our knowledge of diverse childhoods by foregrounding the experiences of children and young people with a severe disability.
DMD and decision-making
As introduced, research requiring the participation of those with DMD sometimes involves onerous commitments such as taking an experimental drug, having blood and cardiac tests and undergoing muscle biopsies (Condin, 2014). With regard to participation in medical research, an older child or young person’s competence to give consent to participate is not always sufficient, and adult consent may also be needed (British Medical Association, 2010). In clinical trials, which are used to test experimental drugs in strictly controlled settings, those under 16 cannot consent to participation whatever their level of competence (Medical Research Council (MRC), 2004). Maintaining a balance between children and young people’s involvement in decision-making while ensuring their protection can be challenging to regulate. There are risks both in protecting children to the extent that they are excluded from research ‘versus the risks of coercing or exploiting their involvement’ (Alderson, 2007: 2281). Ideally, protection is balanced with children and young people’s active involvement (Nuffield Council on Bioethics, 2011), and this can be nurtured through ongoing, effective communication with them.
Little is known about how children experience participation in medical research (Dixon-Woods et al., 2006) or, more specifically, how those with DMD view their potential involvement, and how they live and cope with DMD. These two matters intersect, as research decisions are likely to be made in ways influenced by their sociocultural setting, healthcare and family attitudes to medical research. As Condin (2014) observes, the connection between children and families’ hope in experimental medicine for DMD, and the ‘qualitative dimensions of their lives … have received comparatively little scholarly attention and are at present poorly understood’ (p. 24). In this article, the participants’ accounts will be drawn on to explore some of the ‘qualitative dimensions’ of living with a degenerative condition and decision-making. As suggested, these issues are interrelated; daily life and experiences of agency are likely to shape how decisions are approached and, likewise, the way decisions are formulated can reflect the values and influences circulating in their lifeworlds. It is observed that ‘children and disabled people have been treated as “lesser” because they are positioned as dependent on adults or carers’ (Tisdall, 2012: 6). However, this study draws on the sociology of childhood by acknowledging the ways children are active in their lives (Tisdall, 2012) and are capable of interpreting information on their health and then making it meaningful to their lives (Brady et al., 2015).
Conceptual framework
Work from the sociology of childhood informs the conceptual framework for this study, by understanding children’s lives as having meaning now, rather than considering childhood as subordinate to adulthood (Alderson, 1993; Mayall, 2000). Constructions of childhood shaped by psychology, education, welfare and social policy (Alanen, 1994) have been challenged, and children re-conceptualised as social subjects with valid insights (Boyden, 2003). Hence, they are capable of active engagement in their lifeworlds and they can and should participate in making decisions affecting them (James and Prout, 1997; Mayall, 2000; Tisdall, 2012). This is important for all children, but particularly for disabled children who can be regarded as inherently vulnerable, with their competence overlooked by their adult carers (Davis and Watson, 2000). In my research, the participants’ contributions are brought to the fore by placing their accounts at the centre of the study, and by considering them to be socially engaged agents (James and Prout, 1997; Mayall, 2000) and involved healthcare actors (Brady, et al., 2015).
Concepts from disability studies are also drawn on; disability studies has challenged the dominance of psychological and medical discourses of disability, and the development of the social model regards disability as being socially created (Barton and Oliver, 1997; Shakespeare, 2013). Disability studies emphasises the marginalising nature of the social world and built environment and makes a distinction between impairment, which is individual, and disability, which is structural and public (Shakespeare, 2013). There is a link between studies of childhood and disability studies as both offer theoretical and practical reconsiderations of competence, independence and dependence (Tisdall, 2012).
It is recognised that childhood experiences of health and illness take place in ‘relation to others and in the complexity of local contexts’ (Brady et al., 2015: 179). In these contexts, disabled children can be ‘disempowered … on the grounds of being not only a child, but also of being disabled’ (Viper, 2013: 43). This ambivalence regarding disabled children’s abilities is evident in healthcare guidelines which state that ill and disabled children should be ‘active partners in decisions about their health and care’ (Department of Health (DH), 2003: 9). Nevertheless, there is also concern as, ‘children’s vulnerability and the inability when young or disabled to articulate what they feel pose a challenge for all those involved in delivering health and social care services’ (DH, 2004: 4). Thus, while there is a move to improve children’s involvement in their healthcare, paternalistic attitudes co-exist in policy focusing on risks, body size and development (Brady et al., 2015). Unhelpful assumptions about disabled children’s competencies mean they can miss out on being involved and making decisions (Viper, 2013), and the move to inclusive practice is concealed under the guise of protection (Moore and Kirk, 2010).
As a counter to reductive approaches, there is a call for disabled children to be treated as ‘flexible social beings’ (Davis and Watson, 2000: 213) who can be meaningfully involved in their lives. The experiences of disabled children and young people (Alderson, 1993; Connors and Stalker, 2007; Davis and Watson, 2000) and research exploring the lives of young and adult men with DMD (Abbott and Carpenter, 2009; Dreyer et al., 2010; Gibson et al., 2009) inform my approach. This work privileges the voices and views of ill and disabled children and young people, including those with DMD, and it is within this context that my article is situated. It portrays the participants’ resourcefulness (Boyden, 2003) and their ability to express themselves and describe how they manage life with DMD and how they make decisions.
Study design
This study is based on doctoral research that posed the primary question, what frames the approach taken by boys and young men with DMD to participating in medical research? Secondary objectives were to identify sociocultural, familial and professional influences that inform their decisions. The role of children in medical research has been explored in bioethics, but not with a sociological appreciation; hence, qualitative semi-structured interviews were conducted with the participants. The participants were interviewed once, with four participants invited to follow-up interviews to elaborate on interesting points. The interview data were transcribed and systematically read through several times to identify codes, categories and themes that addressed the primary and secondary research questions; fieldnotes were used to support the analysis.
Recruitment and initial fieldwork
Recruitment material was posted out via a National Health Service clinic, a DMD charity and a wheelchair football club and adverts were placed on a charity’s website. Boys and young men with DMD aged 10–21 were invited to make contact if they were interested; nine boys and young men were recruited. Consent was obtained and the digitally recorded interviews were conducted in the participants’ homes, generally lasting 1 hour. Parents were present for three of the interviews and pseudonyms have been used to protect confidentiality.
Once fieldwork commenced, it became apparent the participants had a limited understanding of medical research; most have poor mobility and live with the embodied knowledge that there are no approved treatments to modify disease progression (Condin, 2014). The lack of good news concerning medical research means it is rarely discussed at home and as a result my questions required additional material to encourage discussion. Therefore, scenarios were developed to contextualise the subject, for example; the participants were asked to imagine a friend with DMD is thinking of taking part in a clinical trial but is unsure of taking a test drug; in another scenario, the imaginary friend feels coerced by his parents into taking part in research. They were asked what advice they would give this ‘friend’ and what they might decide if they were in that situation. These scenarios were posted out prior to the interviews, giving participants an opportunity to think through their responses. Adults may assume that disabled children are poorly equipped to recount aspects of their lifeworlds; however, asking questions in appropriate ways enables them to contribute to sociological and healthcare research (Davis and Watson, 2000). This adaptive approach offered participants a context relevant to them for exploring the subject of medical research and other issues impacting their lives, providing vital, situated insight.
Findings
The interview data encompassed the following themes: experiences of isolation and exclusion, agency and independence, and decision-making on healthcare and medical research.
Isolation and exclusion
Disabled children and young people can encounter marginalising attitudes, as Jay, aged 14, experienced when he used his wheelchair full-time: When I broke my leg and had to be pushed around all the time people would come out and go ‘ahh, how is he?’ to my mam and dad and I was sitting there and like ‘ask me!’; I used to get quite annoyed by it … They talk to you like you’re stupid, quite condescending … there’s nothing wrong with me, it’s just my muscles. (Interview, 22 June 2011)
He felt patronised and excluded from social interactions, an occurrence common to disabled children, and to those with DMD (Connors and Stalker, 2007; Gibson et al., 2009). Tim, the youngest participant at 10 years old, has been using a wheelchair for some time and he explained his situation at school:
I’m the only one that’s sort of disabled … there’s like, about one other, but there’s only me in a wheelchair.
Oh right, how do you feel then about being the only boy in a wheelchair?
Erm fine … people think it’s good because I don’t have to walk, but I don’t find it very good, because it’s boring … but I’m alright with it. (Interview, 2 August 2011)
Tim has adjusted to his limited mobility and is ‘fine’ with this, although feeling ‘bored’ may be his way of expressing an awareness of difference from his peers who are mobile and active. Cinema visits are part of Jay’s social life and he described how he accesses the venue with his mother’s help; she drives him and his friends there, ensures he is settled and then goes shopping in the nearby mall. However, Jay observed that ‘you’re texting us [me] constantly [says this to his mum], you’re waiting for me to text when the film’s over!’ (Interview, 22 June 2011). Boys with DMD have support needs that are usually met by parents, and this can situate them outside generally understood norms of maturing towards independence from their parents.
Ed is 18 and he has an older brother who also has DMD. Ed struggles with anxiety and prefers to stay home, I asked him what might make his life easier; ‘if I didn’t have it [DMD]’, he went on to explain that he does not attend college:
No, I never went … don’t really like being around people or anything.
So what’ve you been doing since you left school then?
Nothing really … my disability stops us [me] doing things I want to do. (Interview, 16 September 2011)
Ed is aware of the negative connotations society attributes to disability and he has witnessed his older brother’s loss of mobility and the isolation that can accompany this. Ed’s experiences and perspective on life with DMD have contributed to how he regards his place in the world, structuring his expectations and disposition (Gibson et al., 2009); he has currently withdrawn from a society he feels offers limited space for him. He was open in expressing a sense of isolation and pessimism, and it is possible that others with a similar outlook chose not to participate in this study. It was also apparent that Ed appreciated the opportunity to talk about his problems and that this was a rare chance to share his thoughts on DMD outside of a clinical setting (Abbott and Carpenter, 2015). Boys with DMD are now living into adulthood, albeit with complex, increasing support needs; however, social care is yet to meet their needs and their isolation can be understood as stemming from health issues and social barriers (Abbott and Carpenter, 2015).
Experiences of exclusion and feeling different were common among the participants and they all expressed an awareness of injustice; however, most were able to negotiate a path through these challenges as active agents (Connors and Stalker, 2007). This skill and insight in identifying unfair practices assists them in living as engaged individuals who experience agency and independence despite disablist attitudes.
Agency and independence
The participants discussed life-affirming experiences that have meaning for them, recounting how they invest time and effort in various activities. Their accounts suggest a motivation to be involved in their lives, challenging reductive notions of vulnerability and immaturity. Ollie, aged 14, values his role at school: I’m a mentor at school so it means I help out … kids with their problems at home and stuff and I’m hoping to become a prefect in year 11.
And, There’s meant to be a [disabled] girl or guy coming [to school] so hopefully I can give them advice as well because I’ve been there. (Interview, 10 November 2011)
Tim speculates that the skill and ability to beat his friends when playing on his games console is attributable to his condition:
So, why do you think you’re so good at games?
I don’t know, I think it’s because I’ve got Duchenne muscular dystrophy,
it might help me be better on this, but I don’t know. (Interview, 2 August 2011)
Computer games create opportunities for those with DMD to compete as equals with their non-disabled peers (Abbott, 2012), and most of the boys spend time playing with friends on their consoles.
As well as making time for recreation, lobbying is an important activity for Adam, aged 16:
I do go up to London to lobby Parliament to give more money; we go and stand near Downing Street with a load of banners … I like to go, ’cos I could stay here if I wanted to and mum just go, because I could get a carer but I like to go and show my support.
Do you feel it’s good to be involved in, in your case lobbying; is that something you feel positive about?
Yeah I really do, I think it’s a really good idea because me and other boys are the ones we’re trying to lobby for so we might as well show them that we’re there and sort of helping. (Interview, 24 November 2011)
As Adam observes, he could stay home, but he chooses to be involved in pressing for change as an advocate for his cause. I asked Rick, who is 12, whether being independent was important to him:
Yeah I think so … as my disability progresses I get weaker so I just try to be independent as much as possible.
Could you tell me a bit more about what you mean by independence?
Like, you don’t have someone to help you all the time and you can go off and do things by yourself. (Second interview, 1 February 2012)
Rick ventures out unaccompanied in his electric wheelchair and he has been coached by his mother: Mum walked me up there [to school] and back quite a lot and taught me how to be safe on the road and … gradually I just built it up and just managed to be able to do it by myself. (Second interview, 1 February 2012)
He can visit the shops and call on neighbourhood friends independently, and he values his wheelchair’s versatility: I find the electric wheelchair’s really important … I’m quite confident of the limitations of this wheelchair ’cos I test it on quite a few terrains so I know what it can do and can’t do. (Interview, 24 November 2011)
Rick’s ability to plan and carry out local excursions gives him a viable means to enact independence and feel he is part of the life of the community (Gibson et al., 2007). Although he will get weaker, his aspirations for the future were ‘probably the same as this [his life now], I’ll be quite a bit weaker but I’ll still manage to do everything that I probably do now’ (interview, 24 November 2011). He focuses on doing the things he can achieve and demonstrates maturity and purpose in using his wheelchair and optimising personal skills.
The participants’ accounts portray their resilience, despite challenges because of their condition and societal attitudes towards them. They all, to some degree, experience and enact agency in daily life; even Ed, who prefers to stay home, enjoys his family’s support and his brother’s company; ‘I always talk to my older brother … ’cos we basically like the same music and films’ (interview, 16 September 2011). Independence sometimes evolves differently and in subtle ways for those with DMD, needing periods of readjustment as the condition changes (Dreyer et al., 2010). The participants encounter marginality and isolation, but they are not victims (McLaughlin and Goodley, 2008), and they also experience independence and agency.
Communicating with doctors and making decisions
Understanding DMD and being able to ask questions gives the participants some sense of control, equipping them to deal with the challenges of having an untreatable condition. Rick has worked with his mum to learn how DMD will affect him and he believes that ‘the more you know, the better you know how to handle it’ (interview, 24 November 2011). He described how he talks to doctors and the importance of speaking up and being involved:
They [doctors] usually talk to me instead of my parents.
Right, is that something then that your mum has encouraged, or,
No, just as I grow older I think, probably need to be heard or something … you need to be heard. (Interview, 24 November 2011)
At hospital appointments, Adam is content for his mother to act as his spokesperson; however, he also expects to be spoken to as a present and engaged participant in consultations:
Is it important that doctors do talk to you?
Yeah it’s very important to me that doctors talk to me because when they do talk over my head it seems quite rude, and I do understand what they’re saying and so that means they can talk to me ’cos I know what they’re talking about. (Second interview, 23 March 2012)
Hospital appointments can be discouraging, they mark the passage of time and the inevitable loss of mobility and strength, so parental support is significant, but the participants recognise their own agency and the right to be involved and respected.
Tom, aged 17, has had surgery to correct scoliosis, a curvature of the spine, and he explained how he decided to have the surgery after talking to his doctor: They did an X-ray on my back … and the doctor doing the operation spoke to me and explained it … then I asked if I could look at the X-ray [laughs] and it was just really bad … I couldn’t tell it was that bad … so when he showed me how bad it was I thought … ‘right I’ve got to have this done’. (Interview, 29 June 2011)
Being informed about treatments helps children and young people understand the logic and purpose behind painful procedures (Alderson, 1993), and Tom was motivated to undergo surgery after seeing the X-ray and talking with his doctor. Jay made a decision to have testosterone injections to help him grow: ‘well I literally said that I’d like to be a bit taller’ (Interview, 22 June 2011); the use of steroids slows the progress of muscle weakness in DMD, but it also delays puberty and growth (Bianchi et al., 2011). Despite Jay’s affirmative decision, on the day of his first injection, the doctors gave him time to rethink treatment; ‘when we were actually there [hospital], “are you actually sure you want to” … I had to think it over’ (Interview, 22 June 2011). Jay gave this more thought and decided he was happy to have the injections, giving him control over the treatment. Children and young people benefit from being treated as partners in their healthcare, and for those with long-term conditions, their competence is experience and context dependent (Alderson et al., 2006; Hagger and Woods, 2005), drawing on an embodied understanding of their health.
Medical research decision-making
Experiences of working with parents and doctors to make healthcare decisions are likely to influence how the participants approach medical research decisions, although the freedom to make personally appropriate choices was raised. As we discussed a scenario in which a young man is making a decision to take part in medical research, Adam observed that not being fully involved could be disempowering:
Do you think it’s important for the young man to make up his own mind about taking part?
Yeah definitely because if they don’t want to do it then they’ll be more worried about it and more sort of scared about it if they haven’t been able to make up their own mind … they might not have control over the situation. (Interview, 24 November 2011)
Referring to the same scenario, Tom also felt that the young man should reach his own decision: ‘because it’s them that it’s happening to’ (Interview, 29 June 2011). Involvement in decision-making is a significant marker of maturity for Ollie, as he explained when asked what his motivation would be if he took part in medical research: To show other Duchenne boys that it’s OK to have it done and it would be great for them to let them have it done, to check what it does … it would help me prove my skills as a young adult. (Interview, 10 November 2011)
Rick has decided to take part in a clinical trial and he reached this decision after asking his doctors some questions about the trial drug: ‘one of the doctors was showing me how it will work, like a diagram of what the drug does and … how it will help you’ (Second interview, 1 February 2012). Rick felt that it was his own decision to make, commenting that his mother was ‘letting me make my own decision’, although he appreciated parental support: ‘they’re both trying to help’ (Second interview, 1 February 2012) and he foresaw that as he matured he would still consult them. Making informed decisions about taking part in medical research requires the child or young person’s willing commitment and an understanding of the processes involved, and the participants’ capacities were apparent as they talked about managing their health and making decisions. They expressed appreciation for parental support and the advice of doctors, and they reported approaching decisions based on this help while also making known their own preferences.
The participants confound notions of helpless dependency, and simultaneous to this, they redefine idealisations of carefree childhoods; they deal with disablist attitudes, and institutional presumptions of low competence, and they negotiate these reductive categories while coping with increasing muscle weakness. This complexity of experiences and the independence and agency that the participants have recounted destabilise fixed boundaries of how disabled children and young people can live and flourish.
Discussion
Adults may assume that childhood disability correlates to incompetence and an inability to engage in decision-making (Davis and Watson, 2000), but such assumptions overlook contextualised competence and the possibilities for agency. Ill and disabled children’s competence can be positively influenced when time is spent listening, giving them choices and encouraging them to take an active role in treatment (Alderson, 1992). Increasing muscle weakness imposes constraints on those with DMD and they are dependent on others for physical care; however, decision-making is a constructive way of achieving independence (Dreyer et al., 2010). The participants are moral beings (Carnevale, 2004) who have expressed a desire to be actively involved in their lives, and it is apparent that this nurtures resilience, self-confidence and self-reliance (Boyden, 2003; Guell, 2007; Speraw, 2009). Ensuring that disabled children and young people have opportunities to speak up and express their views gives them protection through keeping them involved and developing decision-making skills and capacities (Cave, 2011; Lansdown, 2001).
Judging a child’s abilities according to age and developmentally based assumptions (Christensen and Prout, 2002) does not sufficiently allow for the contextual and social components of cognitive development (Hagger and Woods, 2005). Nor can it account for the many variables in ability levels and how children make sense of their world (Banks et al., 2001). Indeed, it has been observed that even young children demonstrate competence in managing healthcare information when they have experience of long-term conditions such as type 1 diabetes (Alderson et al., 2006). Recognising their rights and capacity for active involvement in healthcare decisions and their sociocultural worlds situates them as agents, rather than passive recipients of adult care (Prout and James, 1997; Tisdall, 2012). The participants’ contributions to this study demonstrate their capacities as ‘social actors who are competent at making decisions about their own lives’ (Skelton, 2008: 32). Acknowledging and accommodating these abilities is important; as the participants mature, they may face life-changing choices about their health and social care and, pertinent to this study, taking part in medical research. The findings indicate how the participants can discuss decisions relating to healthcare and medical research and their abilities are variable, rather than strictly conforming to developmentally based categories.
Hearing directly from this under-researched group provides insight on growing up with a degenerative disability, contributing to understandings of disabled children and young people’s competence and ability to be meaningfully involved in their lives. The findings suggest that in theorising and engaging with disabled children and young people, it is constructive to challenge assumptions of their innate vulnerability and to regard them as socially engaged actors with unique insight. Indeed, the participants’ contributions demonstrate a capacity for defining their own priorities and wishes, shaped within an acceptance of their physical limitations. This study demonstrates how disabled children and young people should be recognised as capable agents whose experiences span multiple and complex social spheres in ways that contest the limited categorisations others may ascribe to them.
Conclusion
Restrictive attitudes towards all children and young people can be intensified if they are also disabled, and healthcare policy sometimes makes assumptions about disabled children’s vulnerability, missing the situated, fluid nature of competency (Davis and Watson, 2000). The shift to greater involvement of disabled children and young people in their healthcare can be limited by low expectations and presumptions of immaturity and dependence (Meyer, 2007; Tisdall, 2012; Viper, 2012). The conflation of disability and care-intensive needs with vulnerability and incompetence disempowers them, failing to recognise self-determination. Regarding disabled children as passive denies them a voice, not because they are incompetent, but because their ability to make choices goes unrecognised and their opinions are ignored (Davis and Watson, 2000).
Disabled children and young people have first-hand knowledge of their healthcare, and their views and experiences should be considered when implementing policy and practice (Mayall, 2000). Their contributions can inform and improve provision, with constructive involvement ensuring they feel in control as agents of their own development (Boyden, 2003; Guell, 2007; Speraw, 2009). The participants have provided insight on how decision-making and competence develop within relational and social settings, with their decisions emerging from these contexts (Gibbons, 2008). The development of independence and agency is somewhat contingent upon how adults involve children and young people in decision-making (Alderson, 2007). Interviews with the boys demonstrate their ability to actively engage in their lives, and participation requires they be recognised as participants in society (Phillips and Coppock, 2014). They have gained the skills to negotiate and communicate with their parents and doctors, and can effectively engage in making medical research decisions that are informed by their knowledge and experiences.
Footnotes
Funding
The author(s) disclosed receipt of the following financial support for the research, authorship and/or publication of this article: Doctoral study funded by the Economic and Social Research Council and by TREAT-NMD.