'It's not meant to be for life, but it carries on': a qualitative investigation into the psychosocial needs of young retinoblastoma survivors.

OBJECTIVE AND DESIGN: Retinoblastoma (Rb) is a rare childhood eye cancer, with 45% of individuals impacted by heritable disease and the remainder impacted non-heritably. The condition can leave survivors with life-long psychological and social challenges. This qualitative study examined the psychosocial needs of teenagers and young adults living beyond Rb. SETTING: A qualitative, exploratory study was conducted using focus groups with teenagers and interviews with young adults. Participants were recruited via the Childhood Eye Cancer Trust and the two national Rb treatment centres in the UK. Reflexive thematic analysis was used to analyse data using exploratory and inductive methods. PARTICIPANTS: 32 young survivors of Rb (10 heritable, 21 non-heritable, 1 unknown; 23 unilateral, 9 bilateral) aged between 13 and 29 years (12 male, 20 female). RESULTS: Data were rich and spanned the life course: three key themes were generated, containing eight subthemes. Theme 1 describes participants' experiences of childhood and trauma, including survivor guilt, memories from treatment and impact on personality. Theme 2 focuses on the challenges of adolescence, including the psychological impact of Rb, the impact on identity, and the sense of normality and adaptation to late effects. The third theme considered adulthood and the development of acceptance, a state of being widely considered unachievable during childhood, as well as the 'work' needed to feel supported, including seeking out information, peer support and therapeutic strategies. CONCLUSIONS: This study provides in-depth insight into the experiences of life beyond Rb. Findings highlight the need for specific psychosocial interventions informed by codesign.

A systematic review of combined surgery and brachytherapy approaches for children and young people with relapsed and refractory rhabdomyosarcoma (Local-REFoRMS).

Approximately one third of children with rhabdomyosarcoma relapse or have refractory disease. Treatment approaches include a combination of systemic therapies and local therapies, directed at tumour site(s). This review was conducted to evaluate the effectiveness and safety of the combination of surgery and brachytherapy as local therapy for treating children and young people with relapsed/refractory rhabdomyosarcoma. This review identified studies based on a previous systematic review looking at the treatments for children and young people under 18 years old with relapsed/refractory rhabdomyosarcoma. Studies conducted after 2000 were included. Survival outcomes, relapse rates, adverse events and functional outcomes were extracted. From 16,965 records identified in the baseline systematic review, 205 included the words 'AMORE' or 'brachytherapy', and were screened for eligibility in this substudy. Thirteen studies met the inclusion criteria for Local-REFoRMS, including over 55 relapsed and refractory rhabdomyosarcoma patients. Most studies were retrospective cohort studies conducted within Europe. Most patients had embryonal disease within the head and neck or bladder/prostate regions, and received local therapy for first relapse. Approximately one quarter of patients relapsed following surgery and brachytherapy, with local relapses occurring more than metastatic relapse. Adverse events and functional outcomes were infrequently reported, but related to the site of surgery and brachytherapy. Study quality was limited by inconsistent reporting and potential selection bias. Outcomes following surgery and brachytherapy for a selected group of relapsed and refractory rhabdomyosarcoma show reasonable benefits, but reporting was often unclear and based on small sample sizes.

A systematic review of early phase studies for children and young people with relapsed and refractory rhabdomyosarcoma: The REFoRMS-SR project.

Rhabdomyosarcoma is the commonest soft tissue sarcoma in children. Around one-third of children with rhabdomyosarcoma experience relapse or have refractory disease, which is associated with a poor prognosis. This systematic review of early phase studies in pediatric relapsed/refractory rhabdomyosarcoma was conducted to inform future research and provide accurate information to families and clinicians making difficult treatment choices. Nine databases and five trial registries were searched in June 2021. Early phase studies of interventions for disease control in patients under 18 years old with relapsed/refractory rhabdomyosarcoma were eligible. No language/geographic restrictions were applied. Studies conducted after 2000 were included. Survival outcomes, response rates, quality of life and adverse event data were extracted. Screening, data extraction and quality assessment (Downs and Black Checklist) were conducted by two researchers. Owing to heterogeneity in the included studies, narrative synthesis was conducted. Of 16,965 records screened, 129 published studies including over 1100 relapsed/refractory rhabdomyosarcoma patients were eligible. Most studies evaluated systemic therapies. Where reported, 70% of studies reported a median progression-free survival ≤6 months. Objective response rate was 21.6%. Adverse events were mostly hematological. One-hundred and seven trial registry records of 99 studies were also eligible, 63 of which report they are currently recruiting. Study quality was limited by poor and inconsistent reporting. Outcomes for children with relapsed/refractory rhabdomyosarcoma who enroll on early phase studies are poor. Improving reporting quality and consistency would facilitate the synthesis of early phase studies in relapsed/refractory rhabdomyosarcoma (PROSPERO registration: CRD42021266254).

What matters to you? Engaging with children in the James Lind Alliance Children's Cancer Priority Setting Partnership.

BACKGROUND: Previous priority setting exercises have sought to involve children, but in the final reporting, it is evident that few children had been engaged through the process. A primary aim in the Children's Cancer Priority Setting Partnership was to find out from children what they want research to focus on. We report on our experience to inform methods of engagement with children in future James Lind Alliance Priority Setting Partnerships and similar exercises. METHODS: We followed the James Lind Alliance process, collecting and shortlisting questions via online surveys with adult survivors of childhood cancer, carers, and professionals, and holding a final workshop. Alongside this, a parallel process to collect and prioritise questions from children was undertaken. We created animations for parents/carers to explain the project and surveys to children, gathered questions via online surveys and held a workshop with children to identify their priorities. RESULTS: Sixty-one children and young people with cancer and 10 siblings, aged 3-21 years, submitted 252 potential questions/topics via the surveys. Submissions were refined into 24 summary questions. These questions were discussed at a workshop with eight children; they also added more questions on topics of importance to them. Workshop participants prioritised the Top 5 questions; top priority was, 'How can we make being in hospital a better experience for children and young people? (like having better food, internet, toys, and open visiting so other family members can be more involved in the child's care)'. The Top 5 also included cancer prevention, treatments closer to home, early diagnosis, and emotional support. These questions were taken to the final workshop at which the Top 10 priorities were decided, all five children's priorities were reflected in the final Top 10. CONCLUSIONS: We have demonstrated that it is possible to successfully involve children directly in setting priorities for future research. Future priority setting exercises on topics relevant to children, should seek to include their views. The Children's Cancer Top 10 priorities reflect the voices of children and should inform the funding of future research.

Priority Setting Partnerships find out what areas of research are important to patients, families, and the professionals who care for them. Few Priority Setting Partnerships have involved children, so what matters to them may not have been well-represented. The Children's Cancer Priority Setting Partnership aimed to find out directly from children what research we should do. We collected questions/topics for research from children using online surveys. We made animations to explain the project and surveys to children. Two-hundred and fifty-two questions were sent in by 61 children and young people with cancer and 10 siblings. We grouped similar questions together into 24 summary questions. Summary questions were discussed at a workshop with eight children. Workshop participants added more questions on topics that mattered to them, and decided their Top 5 questions. The top question was, 'How can we make being in hospital a better experience for children and young people? (like having better food, internet, toys, and open visiting so other family members can be more involved in the child's care)'. The Top 5 questions included: preventing cancer, having treatments nearer home, early diagnosis, and emotional support. These questions were taken to the final project workshop, this was with adults, including childhood cancer survivors, where the Top 10 priorities were decided. All five children's priorities were included in the Top 10. We have shown it is possible to successfully involve children in setting research priorities. Future priority setting exercises on topics that affect children should actively seek and include their views.

Research priorities for children's cancer: a James Lind Alliance Priority Setting Partnership in the UK.

OBJECTIVES: To engage children who have experienced cancer, childhood cancer survivors, their families and professionals to systematically identify and prioritise research questions about childhood cancer to inform the future research agenda. DESIGN: James Lind Alliance Priority Setting Partnership. SETTING: UK health service and community. METHODS: A steering group oversaw the initiative. Potential research questions were collected in an online survey, then checked to ensure they were unanswered. Shortlisting via a second online survey identified the highest priority questions. A parallel process with children was undertaken. A final consensus workshop was held to determine the Top 10 priorities. PARTICIPANTS: Children and survivors of childhood cancer, diagnosed before age 16, their families, friends and professionals who work with this population. RESULTS: Four hundred and eighty-eight people submitted 1299 potential questions. These were refined into 108 unique questions; 4 were already answered and 3 were under active study, therefore, removed. Three hundred and twenty-seven respondents completed the shortlisting survey. Seventy-one children submitted questions in the children's surveys, eight children attended a workshop to prioritise these questions. The Top 5 questions from children were taken to the final workshop where 23 questions in total were discussed by 25 participants (young adults, carers and professionals). The top priority was 'can we find effective and kinder (less burdensome, more tolerable, with fewer short and long-term effects) treatments for children with cancer, including relapsed cancer?' CONCLUSIONS: We have identified research priorities for children's cancer from the perspectives of children, survivors, their families and the professionals who care for them. Questions reflect the breadth of the cancer experience, including diagnosis, relapse, hospital experience, support during/after treatment and the long-term impact of cancer. These should inform funding of future research as they are the questions that matter most to the people who could benefit from research.

Reproduction in life and death: should cancer patients with a poor prognosis be offered fertility preservation interventions?

In the context of a cancer diagnosis, fertility preservation interventions are used to mitigate the potential impact of gonadotoxic cancer treatment upon fertility. They provide patients with cancer the option to freeze their reproductive material to have their own biological child following treatment. The evidence suggests some clinicians are less likely to have fertility preservation discussions with patients who have an aggressive or metastatic cancer which has a poor prognosis. Although this is contrary to current policy recommendations, there is a lack of guidance relating to offering fertility preservation in the context of a poor prognosis to support clinicians. Controversy surrounds posthumous reproduction, and whether the wishes of the cancer patient, when living and deceased should take precedence over others' wellbeing. We consider the question of whether cancer patients with a poor prognosis should be offered FP from an ethics perspective. We structure the paper around key arguments to which multiple ethical principles might pertain, first establishing a central argument in favour of offering fertility preservation based on respect for autonomy, before exploring counterarguments. We conclude by proposing that a defeasible assumption should be adopted in favour of offering fertility preservation to all cancer patients who might benefit from it. It is important to recognise that patients could benefit from fertility preservation in many ways, and these are not limited to having a parenting experience. The burden of proof rests on the clinician in collaboration with their multi-disciplinary team, to show that there are good grounds for withholding the offer.

Regional perspectives on the coordination and delivery of paediatric end-of-life care in the UK: a qualitative study.

BACKGROUND: Provision of and access to paediatric end-of-life care is inequitable, but previous research on this area has focused on perspectives of health professionals in specific settings or children with specific conditions. This qualitative study aimed to explore regional perspectives of the successes, and challenges to the equitable coordination and delivery of end-of-life care for children in the UK. The study provides an overarching perspective on the challenges of delivering and coordinating end-of-life care for children in the UK, and the impact of these on health professionals and organisations. Previous research has not highlighted the successes in the sector, such as the formal and informal coordination of care between different services and sectors. METHODS: Semi-structured interviews with Chairs of the regional Palliative Care Networks across the UK. Chairs or co-Chairs (n = 19) of 15/16 Networks were interviewed between October-December 2021. Data were analysed using thematic analysis. RESULTS: Three main themes were identified: one standalone theme ("Communication during end-of-life care"); and two overarching themes ("Getting end-of-life services and staff in the right place", with two themes: "Access to, and staffing of end-of-life care" and "Inconsistent and insufficient funding for end-of-life care services"; and "Linking up healthcare provision", with three sub-themes: "Coordination successes", "Role of the networks", and "Coordination challenges"). Good end-of-life care was facilitated through collaborative and network approaches to service provision, and effective communication with families. The implementation of 24/7 advice lines and the formalisation of joint-working arrangements were highlighted as a way to address the current challenges in the specialism. CONCLUSIONS: Findings demonstrate how informal and formal relationships between organisations and individuals, enabled early communication with families, and collaborative working with specialist services. Formalising these could increase knowledge and awareness of end of life care, improve staff confidence, and overall improve professionals' experiences of delivering care, and families' experiences of receiving it. There are considerable positives that come from collaborative working between different organisations and sectors, and care could be improved if these approaches are funded and formalised. There needs to be consistent funding for paediatric palliative care and there is a clear need for education and training to improve staff knowledge and confidence.

Consultant-led UK paediatric palliative care services: professional configuration, services, funding.

OBJECTIVES: To systematically gather information on the professional team members, services provided, funding sources and population served for all consultant-led specialised paediatric palliative care (SPPC) teams in the UK. METHODS: Two-part online survey. RESULTS: Survey 1: All 17 medical leads from hospital-based or hospice-based SPPC teams responded to the survey (100% response rate).Only six services met the NICE guidance for minimum SPPC team.All services reported providing symptom management, specialist nursing care, end-of-life planning and care, and supporting discharges and transfers to home or hospice for the child's final days-hours. Most services also provided care coordination (n=14), bereavement support (n=13), clinical psychology (n=10) and social work-welfare support (n=9). Thirteen had one or more posts partially or fully funded by a charity.Survey 2: Nine finance leads provided detailed resource/funding information, finding a range of statutory and charity funding sources. Only one of the National Health Service (NHS)-based services fully funded by the NHS. CONCLUSIONS: One-third of services met the minimum criteria of professional team as defined by NICE. Most services relied on charity funding to fund part or all of one professional post and only one NHS-based service received all its funding directly from the NHS.