What hope means for families of children with rare diseases in clinical trials

Children taking part in clinical trials are often described as being given ‘hope’. Is this useful as a coping mechanism or does give rise to misconceptions and possibly false hope?

In this extended abstract, Northumbria University graduate tutor and paediatric nurse Claire Camara outlines the main findings from a literature review

Background

Due to the extended survival of children and young people that modern medicine has enabled (Moreira et al 2013, Ferro et al 2014) there is a rising prevalence of life-limiting conditions in the UK (Fraser et al 2011, Norman and Fraser 2014). Duchenne muscular dystrophy (DMD) is one such condition, in which muscle wasting gradually affects all skeletal muscles (Thomas et al 2014).

In recent years the number of clinical trials attempting to establish new treatments for DMD has increased (Franson et al 2019). While it is widely accepted that clinical trials are necessary in paediatrics for the development of safe and effective treatments (Naka et al 2017) it is also well established that this must be coupled with careful ethical consideration (Barned et al 2018).

For nurses working with children and young people who have conditions which have no licensed curative treatment, the word ‘hope’ is frequently heard. Interest in the role of ‘hope’ as a concept in paediatric clinical trials for DMD then became the focus for a review of the literature.  

Methods

A working group was formed with three children’s nurses working in early phase clinical trials and one children’s nursing graduate tutor working at Northumbria University.  

We took a systematic approach to review the available literature, search terms were agreed using a population, exposure, outcomes (PEO) strategy (Bettany-Saltikov 2012). This included defining the patient, condition, exposure and outcomes. Synonyms were also identified and included.

CINAHL, PubMed and SCOPUS databases were reviewed and 731 articles were found of which 17 were reviewed fully. Other articles were excluded for not being DMD focused, exclusively focused on the scientific drug or treatment development, or being results of early laboratory-based studies.

Findings

Hope appears regularly in many areas of a child or young person’s participation in early phase research. This may be hope of direct benefit from trialled treatment (Schaffer et al 2009), hope from the participant that one day what they are enduring will help others as those before have helped them (Hunfeld and Passchier 2012, Schaffer et al 2009), or hope from clinicians that they have something to offer their patients (Bendixen et al 2016). 

Literature may be presented in a hopeful way or it can be perceived as such by families of a hopeful disposition (Woods et al 2014). Medical and pharmaceutical companies may use language for results based on animal models. This can influence potential participants and their beliefs surrounding how far the study has progressed (Aartsma-rus 2011). Hope has the potential to influence decisions made by all parties.

Implications for practice

Clinical research trials will always be a necessity for children and young people with conditions for which there is no licensed curative treatment. 

For children’s nurses working with children and young people who are considering taking part in clinical trials there is a need to be mindful of the messages we give and the clarity of informed decisions made by parents and carers.

Conclusion

Whether the concept of hope is helpful is contested because it may lead to therapeutic misconceptions, but it can also be a coping mechanism for families who face no curative options. While this literature review has been focused on hope in DMD trials, similarities could be drawn to other long-term or life-limiting conditions.

References

• Aartsma-rus A (2011) The risks of therapeutic misconception and individual patient (n=1) “trials” in rare diseases such as Duchenne dystrophy. Neuromuscular Disorders. 21, 1, 13-15.  doi: 10.1016/j.nmd.2010.09.012
• Barned C, Dobson J, Stinzi A et al (2018) Children’s perspectives on the benefits and burdens of research participation. Empirical Bioethics. 9, 1, 19-28. doi: 10.1080/23294515.2018.1430709 
• Bendixen RM, Morgenroth LP, Clinard KL (2016) Engaging participants in rare disease research: a qualitative study of Duchenne muscular dystrophy. Clinical Therapeutics. 38, 6, 1474-1484.e1472. doi: 10.1016/j.clinthera.2016.04.001
• Bettany-Saltikov J (2012) How to do a systematic literature review in nursing. Open University Press, Maidenhead, 71-74
• Ferro M, Gorter JW, Boyle MH (2014) Trajectories of depressive symptoms during the transition to young adulthood: the role of chronic illness. Journal of Affective Disorders. 174, 594-601 doi: 10.1016/j.jad.2014.12.014
• Franson T, Kinnett K, Cripe TP (2019) Unique burdens of pediatric clinical trials in Duchenne muscular dystrophy, April 20- 21, 2017, Bethesda, Maryland, USA. Therapeutic Innovation & Regulatory Science. 53, 1, 154-163.  doi: 10.1177/2168479018764650
• Fraser LK, Miller M, Aldridge J et al (2011) Life-limiting and life-threatening conditions in children and young people in the United Kingdom; national and regional prevalence in relation to socioeconomic status and ethnicity
• Hunfeld JA, Passchier J (2012) Participation in medical research: a systematic review of the understanding and experience of children and adolescents. Patient Education and Counseling. 87, 3, 268-276.  doi: 10.1016/j.pec.2011.09.006
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• Naka F et al (2017) Clinical trials: Kids are not just little people. Clinics in Dermatology. 35, 6, 583-593. doi: 10.1016/j.clindermatol.2017.08.008
• Norman P, Fraser L (2014) Prevalence of life-limiting conditions in children and young people in England: time trends by area type. Health and Place. 26, 171-179. doi: 10.1016/j.healthplace.2014.01.002
• Schaffer R, Henderson G, Churchill L et al (2009). ‘Parents' Online Portrayals of Pediatric Treatment and Research Options’, Journal of Empirical Research on Human Research Ethics, 4(3), 73-87. doi: 10.1525/jer.2009.4.3.73
• Thomas PT, Rajaram P, Nalini A (2014) Psychosocial challenges in family caregiving with children suffering from Duchenne muscular dystrophy. Health and Social Work. 39, 3, 144- 152. doi: 10.1093/hsw/hlu027
• Woods S, Hagger L, McCormack P (2014) Therapeutic misconception: hope, trust and misconception in paediatric research. Health Care Analysis. 22, 1, 3-21. doi: 10.1007/s10728-012-0201-8

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