Adolescents with Rett syndrome at critical care pathway junctures: Examining clinicians' decision to initiate invasive long-term ventilation.

BACKGROUND: The initiation of invasive long-term ventilation (I-LTV) for an adolescent with Rett Syndrome (RTT) involves many serious bioethical considerations. In moving towards a more inclusive model of patient participation, transparency surrounding the main influencing factors around this decision is important. OBJECTIVE: We aimed to identify the main drivers influencing a clinician's decision to support initiation of I-LTV for an adolescent with RTT. METHOD: We used an anonymous online vignette-based factorial survey. The survey was distributed internationally through eight professional multi-disciplinary organisations to reach clinicians working in paediatrics. RESULTS: We analysed 504 RTT vignettes completed by 246 clinicians using mixed effect regression modelling. The main three significant influencing factors identified were: parental agreement with the decision to support initiation, the family's support network, and proximity to a tertiary care centre. Additional comments from participants focused on family support, and the importance of on-going communication with the family. CONCLUSION: As the rights of those with disabilities improve and participation of adolescents in decision-making becomes more established, effective communications with the family around goals of care and particular sensitivity and reflective practice around methods of consensus building will likely contribute to a positive decision-making process at this difficult time.

Assessing the impact of risk-based data monitoring on outcomes for a paediatric multicentre randomised controlled trial.

BACKGROUND/AIMS: Regulatory guidelines recommend that sponsors develop a risk-based approach to monitoring clinical trials. However, there is a lack of evidence to guide the effective implementation of monitoring activities encompassed in this approach. The aim of this study was to assess the efficiency and impact of the risk-based monitoring approach used for a multicentre randomised controlled trial comparing treatments in paediatric patients undergoing cardiac bypass surgery. METHODS: This is a secondary analysis of data from a randomised controlled trial that implemented targeted source data verification as part of the risk-based monitoring approach. Monitoring duration and source to database error rates were calculated across the monitored trial dataset. The monitored and unmonitored trial dataset, and simulated trial datasets with differing degrees of source data verification and cohort sizes were compared for their effect on trial outcomes. RESULTS: In total, 106,749 critical data points across 1,282 participants were verified from source data either remotely or on-site during the trial. The total time spent monitoring was 365 hours, with a median (interquartile range) of 10 (7, 16) minutes per participant. An overall source to database error rate of 3.1% was found, and this did not differ between treatment groups. A low rate of error was found for all outcomes undergoing 100% source data verification, with the exception of two secondary outcomes with error rates >10%. Minimal variation in trial outcomes were found between the unmonitored and monitored datasets. Reduced degrees of source data verification and reduced cohort sizes assessed using simulated trial datasets had minimal impact on trial outcomes. CONCLUSIONS: Targeted source data verification of data critical to trial outcomes, which carried with it a substantial time investment, did not have an impact on study outcomes in this trial. This evaluation of the cost-effectiveness of targeted source data verification contributes to the evidence-base regarding the context where reduced emphasis should be placed on source data verification as the foremost monitoring activity.

The factorial survey as an approach to investigate clinical decision-making: examining influences on a clinician's decision to initiate life-sustaining clinical technology for a child with spinal muscular atrophy type 1.

Background: Spinal Muscular Atrophy (SMA) type 1 is a debilitating condition with a poor prognosis, though therapeutic advances are promising. Long-term ventilation is a common management strategy as respiratory function deteriorates. Without consensus on best practice respiratory management, the decision to initiate invasive LTV (I-LTV) for this group of young children involves many ethical considerations. Understanding the main influencing factors on a clinician's likelihood to initiative I-LTV for a child with chronic critical illness is important to maintain transparency and trust with the family during this challenging time. Methods: A factorial survey was used to identify the factors that influence a clinician to support initiation of I-LTV for children with SMA type 1. Factorial survey content was based on literature and evidence-based practice and the content was subject to extensive pretesting and pilot testing. An anonymous survey was disseminated (Oct 2021-Jan 2022), via eight international professional organisations, to clinicians with experience caring for children at the time of initiation of I-LTV. Results: 251 participants answered 514 vignettes on SMA type 1. The greatest influencing factor on clinician's likelihood to initiate I-LTV was parental agreement with the need to initiate I-LTV. Additional qualitative comments from participants support this finding. Clinicians also highlighted the important role of innovative therapies as well as the availability of supports for families when considering initiation however these findings were context based. Conclusions: The factorial survey approach provides a valuable way of identifying influencers on decision-making in sensitive situations. The findings demonstrate the acceptance of the centrality of parental influence in decisions on care delivery. Effective communication with the child's family is key to ensuring shared understanding and agreement of goals of care. More international research is needed on the long-term effects of novel treatments, as well as impact on quality of life and influence of geographical location, to inform decision-making.

Longitudinal cohort study investigating neurodevelopmental and socioemotional outcomes in school-entry aged children after open heart surgery in Australia and New Zealand: the NITRIC follow-up study protocol.

INTRODUCTION: Despite growing awareness of neurodevelopmental impairments in children with congenital heart disease (CHD), there is a lack of large, longitudinal, population-based cohorts. Little is known about the contemporary neurodevelopmental profile and the emergence of specific impairments in children with CHD entering school. The performance of standardised screening tools to predict neurodevelopmental outcomes at school age in this high-risk population remains poorly understood. The NITric oxide during cardiopulmonary bypass to improve Recovery in Infants with Congenital heart defects (NITRIC) trial randomised 1371 children <2 years of age, investigating the effect of gaseous nitric oxide applied into the cardiopulmonary bypass oxygenator during heart surgery. The NITRIC follow-up study will follow this cohort annually until 5 years of age to assess outcomes related to cognition and socioemotional behaviour at school entry, identify risk factors for adverse outcomes and evaluate the performance of screening tools. METHODS AND ANALYSIS: Approximately 1150 children from the NITRIC trial across five sites in Australia and New Zealand will be eligible. Follow-up assessments will occur in two stages: (1) annual online screening of global neurodevelopment, socioemotional and executive functioning, health-related quality of life and parenting stress at ages 2-5 years; and (2) face-to-face assessment at age 5 years assessing intellectual ability, attention, memory and processing speed; fine motor skills; language and communication; and socioemotional outcomes. Cognitive and socioemotional outcomes and trajectories of neurodevelopment will be described and demographic, clinical, genetic and environmental predictors of these outcomes will be explored. ETHICS AND DISSEMINATION: Ethical approval has been obtained from the Children's Health Queensland (HREC/20/QCHQ/70626) and New Zealand Health and Disability (21/NTA/83) Research Ethics Committees. The findings will inform the development of clinical decision tools and improve preventative and intervention strategies in children with CHD. Dissemination of the outcomes of the study is expected via publications in peer-reviewed journals, presentation at conferences, via social media, podcast presentations and medical education resources, and through CHD family partners. TRIAL REGISTRATION NUMBER: The trial was prospectively registered with the Australian New Zealand Clinical Trials Registry as 'Gene Expression to Predict Long-Term Neurodevelopmental Outcome in Infants from the NITric oxide during cardiopulmonary bypass to improve Recovery in Infants with Congenital heart defects (NITRIC) Study - A Multicentre Prospective Trial'. TRIAL REGISTRATION: ACTRN12621000904875.

Technology solutionism in paediatric intensive care: clinicians' perspectives of bioethical considerations.

BACKGROUND: The use of long-term life-sustaining technology for children improves survival rates in paediatric intensive care units (PICUs), but it may also increase long-term morbidity. One example of this is children who are dependent on invasive long-term ventilation. Clinicians caring for these children navigate an increasing array of ethical complexities. This study looks at the meaning clinicians give to the bioethical considerations associated with the availability of increasingly sophisticated technology. METHODS: A hermeneutic phenomenological exploration of the experiences of clinicians in deciding whether to initiate invasive long-term ventilation in children took place, via unstructured interviews. Data were analysed to gain insight into the lived experiences of clinicians. Participants were from PICUs, or closely allied to the care of children in PICUs, in four countries. RESULTS: Three themes developed from the data that portray the experiences of the clinicians: forming and managing relationships with parents and other clinicians considering, or using, life sustaining technology; the responsibility for moral and professional integrity in the use of technology; and keeping up with technological developments, and the resulting ethical and moral considerations. DISCUSSION: There are many benefits of the availability of long-term life-sustaining technology for a child, however, clinicians must also consider increasingly complex ethical dilemmas. Bioethical norms are adapting to aid clinicians, but challenges remain. CONCLUSION: During a time of technological solutionism, more needs to be understood about the influences on the initiation of invasive long-term ventilation for a child. Further research to better understand how clinicians, and bioethics services, support care delivery may positively impact this arena of health care.

Development of a factorial survey for use in an international study examining clinicians' likelihood to support the decision to initiate invasive long-term ventilation for a child (the TechChild study).

BACKGROUND: The decision to initiate invasive long-term ventilation for a child with complex medical needs can be extremely challenging. TechChild is a research programme that aims to explore the liminal space between initial consideration of such technology dependence and the final decision. This paper presents a best practice example of the development of a unique use of the factorial survey method to identify the main influencing factors in this critical juncture in a child's care. METHODS: We developed a within-subjects design factorial survey. In phase 1 (design) we defined the survey goal (dependent variable, mode and sample). We defined and constructed the factors and factor levels (independent variables) using previous qualitative research and existing scientific literature. We further refined these factors based on expert feedback from expert clinicians and a statistician. In phase two (pretesting), we subjected the survey tool to several iterations (cognitive interviewing, face validity testing, statistical review, usability testing). In phase three (piloting) testing focused on feasibility testing with members of the target population (n = 18). Ethical approval was obtained from the then host institution's Health Sciences Ethics Committee. RESULTS: Initial refinement of factors was guided by literature and interviews with clinicians and grouped into four broad categories: Clinical, Child and Family, Organisational, and Professional characteristics. Extensive iterative consultations with clinical and statistical experts, including analysis of cognitive interviews, identified best practice in terms of appropriate: inclusion and order of clinical content; cognitive load and number of factors; as well as language used to suit an international audience. The pilot study confirmed feasibility of the survey. The final survey comprised a 43-item online tool including two age-based sets of clinical vignettes, eight of which were randomly presented to each participant from a total vignette population of 480. CONCLUSIONS: This paper clearly explains the processes involved in the development of a factorial survey for the online environment that is internationally appropriate, relevant, and useful to research an increasingly important subject in modern healthcare. This paper provides a framework for researchers to apply a factorial survey approach in wider health research, making this underutilised approach more accessible to a wider audience.

The Meaning Given to Bioethics as a Source of Support by Physicians Who Care for Children Who Require Long-Term Ventilation.

The role and potential of bioethics input when a child requires the initiation of technology dependence to sustain life is relatively unknown. In particular, little is understood about the meaning physicians give to bioethics as a source of support during the care of children in pediatric intensive care who require long-term ventilation (LTV). We used a hermeneutic phenomenological approach to underpin the collection and analysis of data. Unstructured interviews of 40 physicians in four countries took place during 2020. We found that elements of trust, communication and acceptance informed the physicians' perceptions of the relationship with bioethics. These ranged from satisfaction to disappointment with their input into critical decisions. Bioethics services have potential to help physicians gain clarity over distressing and complex care decisions, yet physicians perceive the service inconsistently as a means of support. This research provides a sound basis to guide more beneficial interactions between clinicians and bioethics services.

Initiating technology dependence to sustain a child's life: a systematic review of reasons.

BACKGROUND: Decision-making in initiating life-sustaining health technology is complex and often conducted at time-critical junctures in clinical care. Many of these decisions have profound, often irreversible, consequences for the child and family, as well as potential benefits for functioning, health and quality of life. Yet little is known about what influences these decisions. A systematic review of reasoning identified the range of reasons clinicians give in the literature when initiating technology dependence in a child, and as a result helps determine the range of influences on these decisions. METHODS: Medline, EMBASE, CINAHL, PsychINFO, Web of Science, ASSIA and Global Health Library databases were searched to identify all reasons given for the initiation of technology dependence in a child. Each reason was coded as a broad and narrow reason type, and whether it supported or rejected technology dependence. RESULTS: 53 relevant papers were retained from 1604 publications, containing 116 broad reason types and 383 narrow reason types. These were grouped into broad thematic categories: clinical factors, quality of life factors, moral imperatives and duty and personal values; and whether they supported, rejected or described the initiation of technology dependence. The majority were conceptual or discussion papers, less than a third were empirical studies. Most discussed neonates and focused on end-of-life care. CONCLUSIONS: There is a lack of empirical studies on this topic, scant knowledge about the experience of older children and their families in particular; and little written on choices made outside 'end-of-life' care. This review provides a sound basis for empirical research into the important influences on a child's potential technology dependence.

Precision of weight measurement in critically ill infants: a technical report.

Objectives: To investigate the precision of weight measurements in critically ill infants in a paediatric intensive care unit (PICU). Design: Prospective cohort study. Setting: Royal Children's Hospital PICU. Participants: Mechanically ventilated infants admitted to the Royal Children's Hospital PICU between September 2020 and February 2021. Main outcome measures: Mean percentage difference and agreement of consecutive weight measurements. Results: Thirty infants were enrolled, of which 17 were receiving post-surgical care for congenital heart disease and four were receiving extracorporeal membrane oxygenation (ECMO). The median age was 13 days (interquartile range [IQR], 3.1-52.4 days). The mean difference in weight was 1.3% (standard deviation [SD], 1.0%), and the test-retest agreement intraclass correlation was 0.99 (95% CI, 0.99-0.99; P < 0.01). The percentage difference between measurements was ≤ 2.5% in 26/30 (87%) children, and the range was < 0.1% to 3.6%. In 26 children not receiving ECMO, the mean difference in weight was 1.1% (SD, 1.0%). There were no complications. Conclusions: Weighing mechanically ventilated, critically ill infants in intensive care can be performed safely, with a mean difference between consecutive weights of 1.3%, making it a potentially useful additional measure of fluid accumulation.

A systematic concept analysis of 'technology dependent': challenging the terminology.

There are an increasing number of children who are dependent on medical technology to sustain their lives. Although significant research on this issue is taking place, the terminology used is variable and the concept of technology dependence is ill-defined. A systematic concept analysis was conducted examining the attributes, antecedents, and consequences of the concept of technology dependent, as portrayed in the literature. We found that this concept refers to a wide range of clinical technology to support biological functioning across a dependency continuum, for a range of clinical conditions. It is commonly initiated within a complex biopsychosocial context and has wide ranging sequelae for the child and family, and health and social care delivery.Conclusion: The term technology dependent is increasingly redundant. It objectifies a heterogenous group of children who are assisted by a myriad of technology and who adapt to, and function with, this assistance in numerous ways. What is Known: • There are an increasing number of children who require medical technology to sustain their life, commonly referred to as technology dependent. This concept analysis critically analyses the relevance of the term technology dependent which is in use for over 30 years. What is New: • Technology dependency refers to a wide range of clinical technology to support biological functioning across a dependency continuum, for a range of clinical conditions. It is commonly initiated within a complex biopsychosocial context and has wide-ranging sequelae for the child and family, and health and social care delivery. • The paper shows that the term technology dependent is generally portrayed in the literature in a problem-focused manner. • This term is increasingly redundant and does not serve the heterogenous group of children who are assisted by a myriad of technology and who adapt to, and function with, this assistance in numerous ways. More appropriate child-centred terminology will be determined within the TechChild project.

'Don't put the parent out': parents' perspectives of being present during an inter-hospital transfer.

AIMS AND OBJECTIVES: To understand Irish parents' experiences of the inter-hospital transfer of their acutely ill child from a regional hospital to a tertiary children's hospital. BACKGROUND: Much of the literature on inter-hospital transfer focuses on the medical outcome of children who have been transferred for specialist care. While parental presence during this journey is the focus of discussion papers, little is known about the experience of parents as service users. DESIGN: A qualitative design using a Husserlian phenomenological approach was adopted for this study. METHODS: Semi-structured interviews were conducted with ten parents between March and October 2014 to explore their experiences of parental presence during the transfer of an acutely ill child. Data were analysed using the modified Van Kaam method. RESULTS: Two key themes emerged 'don't put the parent out' and 'being a parent'. Given a choice, all parents interviewed would have wished to stay with their child during transfer and the findings portray the specific contribution of parents during the journey. CONCLUSIONS: Not being permitted to travel with their acutely ill child had a negative effect on parents' perceptions of their ability to continue to parent their child during transfer to specialist paediatric services. To address this there is a need for parents to have the choice, when at all possible, to stay with their child at this time. RELEVANCE TO CLINICAL PRACTICE: There is a need to maximise the facilitation of parental presence in an Irish context through the introduction of guidelines to support staff involved in the transfer of an acutely ill child.