What do families want to improve in the management of paediatric febrile neutropenia during anti-cancer treatment? Report of a patient/public involvement group

Background This study reports how parents and young people who had an experience of febrile neutropenia (FN) improved the design of a trial to inform the management of this condition. Five parents, a young person who had completed treatment and three clinician-researchers contributed. Methods The group was formed after an invitation on social media and met via video conference. Many participants were from an existing childhood-cancer parent-involvement group. The initial questions asked during discussion were about the importance of the topic, the views on the need for a trial, which important outcomes should be measured and the practical aspects which would make it easier or more difficult for people to take part in it. The conversation occurred for an entire afternoon, was audio and video recorded, transcribed, analysed and checked by those involved. The fifth parent added to this via email. Results The group altered the trial structure by proposing randomising of each child to one of the two management methods through the whole of their anti-cancer treatment, rather than randomising the study sites or the child at each visit. They felt that even if people declined taking part in the study in the first weeks of diagnosis, their views might change and they should be allowed to consent later. They also proposed methods of collecting important patient and family data, enriching the medical information gained in the study. Active follow-up, negotiated for each individual family, was also suggested. Conclusion Trials for improving the management of FN in children and young people who are undergoing anti-cancer treatments should consider individual-patient randomisation, collection of ‘quality of life’ and ‘experience of care’ aspects using digital and paper-based methods, engage families in shared decision-making about management options and ensure adequate supportive information is available and accessible to all patients regardless of background, geographical location or age.

The article demonstrates the merit of having a proper plan about how you are going to involve patients and parents: the way you are going to find people; the need for a structure for the discussions; how to capture the contributions; and to describe the differences and impact these have made. This type of structural clarity is missing from much of the writing about PPI.
First of all, the reach was by social media rather than handpicking a few select individuals to sit on a committee or attend a discussion group. Although many of the respondents were known to the researchers, this openness gave opportunities for others to join and probably helped raise awareness. This shows the extent to finding different means of PPI (Inclusiveness and Working Together).
Using video conferencing means that people can fit involvement into their lives rather than always travel to a meeting. The fact that the team had a contingencies to enable a telephone conference element and contribution by email can be viewed as inclusive and helpful communication.
The structure for the discussion of Importance, Value, Outcomes and Advice could be outlined a little stronger in the article as it offers a useful framework for other to follow and adapt.
Similarly the clustering of the issues into study specific themes are again helpful guides.
The real power of this article lies in the examples of how this involvement changed the actual design of the study in a fundamental manner -ie the randomise method (Impact). The clarity with which the group recommended the use of clinical data was also interesting as it is an issue worrying many researchers.
The recording and reporting (Communications and Impact) of the dialogue between the team and those affected by paediatric febrile neutropenia is well composed offering a mix of quotes, comment and analysis.
The description of being 'in a cupboard' perfectly captures the line that this exercise brought about a 'deeper understanding'.
Finally, I like the fact that the group have shown a willingness to continue to be involved and that they will be included on the Trial Steering Group (Governance). The authors should be encouraged to continue writing about how the patient voice is helping to shape and influence this work. It will be interesting to see how both the patients and team are supported and what they feel they have learned from the experience over time (Support and Learning).

REVIEWER
Reviewer name: Louca-Mai Brady Institution and Country: Kingston University, UK Competing interests: I sit on a study steering committee with Bob Philips.

07-Nov-2018
GENERAL COMMENTS This is a really interesting article which I think could make a worthwhile contribution to the literature on patient and public involvement in health and social care as well as paediatric oncology. As the former is my area of expertise rather than the latter the following comments are focused on this submission as an article on PPI.
I think the article needs to locate this work in relation to the PPI literature and particularly the literature on children and young people's involvement e.g. why the research team wanted to do PPI, evidence on benefits and good practice. Some recent references are included below which may be useful + I would suggest following the GRIPP2 guidance as this is now the accepted standard on reporting PPI.
The article reports clearly how the public involvement group was set up and run but I think does so fairly uncritically. Some reflection on the cons as well as the pros of the approach taken and emerging learning would be helpful. Is there anything the authors think could have been done better or differently regarding who was involved and how? E.g was the young person who attended a child of one of the adult contributors and if so what were the implications of this? Why was only one young person involved?
Were there advantages as well as disadvantages of having adults and young people meeting together? Would the authors have liked to have ongoing involvement/more than one meeting with the group and if so what were the barriers to this?
In the abstract and generally: as well as how the PPI group informed the trial plans -are there any general lessons on PPI with parents and young people in the design stage of research and generally? Language is inconsistent for those involved: public contributors are referred to variously as 'volunteers', 'the PPI group, and 'the group' & the research team as 'the trialists', 'trial group' 'clinical trialists' and 'clinical academics'. PPI group members/public contributors and researchers might be simpler.
Key messages: 'What is known': PPI has done a lot more than modify designs and information leaflets (ref: point about drawing on literature above. 'What this study adds': add a point on how this article adds to the literature on PPI/PPI with families and CYP Methods: as above more discussion on the pros and cons of, for example, involving parents and young people in the same group + video as opposed to face to face meetings. Much of the existing evidence on young people's involvement seems to say that face-toface is better so I'm interested in why the authors found this not to be the case with this project and whether they this approach might potentially exclude some, while enabling the involvement of others?
The authors say that 'the participants volunteered their time and were not paid' but NIHR INVOLVE guidance is that public contributors should be paid for their time. This should probably be mentioned or briefly discussed.
'A patient/public engagement group of experts through experience in the development of a study' -could this wording be clarified/simplified? Also in this paragraph explain why ethical review wasn't required for readers who may be unfamiliar with the NIHR INVOLVE/HTA guidance on this. 'Study-specific themes': I agree that 'PPI is important in modifying....presumptions and beliefs' and this would be a good point to draw in some of the literature. Similarly where the authors state that 'this seems to speak of a greater public awareness being required'.
I was interested in knowing more about plans for PPI when the trial is underway, if and how the group informed these plans and any ongoing involvement in the study (e.g. as coapplicants). This is only mentioned in passing in the final sentence but I think merits further discussion. Response summary: We would like to thank the reviewer for these very positive and constructive comments. We've adapted our manuscript in response to these, and those of the other reviewer, to use more acknowledged terminology to enhance clear communication. We are particularly pleased our work was accessible to someone from outside the 'niche' world of paediatric oncology.
The elements raised by Reviewer 2: Comments to the Author This is a really interesting article which I think could make a worthwhile contribution to the literature on patient and public involvement in health and social care as well as paediatric oncology. As the former is my area of expertise rather than the latter the following comments are focused on this submission as an article on PPI I think the article needs to locate this work in relation to the PPI literature and particularly the literature on children and young people's involvement e.g. why the research team wanted to do PPI, evidence on benefits and good practice. Some reflection on the cons as well as the pros of the approach taken and emerging learning would be helpful. how the PPI group informed the trial plans Language is inconsistent for those involved Response summary: We would like to greatly thank the reviewer for their work in improving our article, providing a great selection of evidence to deepen our work and thoroughly appreciate the work we are attempting to do. We have described more critically our method, highlighting the weaknesses and suggested reasons for success, developed the background and where this advances our knowledge of PPI further, and put in a more consistent approach to 'naming' as well as expanding what actually occurred in the information sharing part of the group discussion.