Using existing data from clinical registries to support clinical trials and other prospective studies has the potential to improve research efficiency. However, little has been reported about staff experiences and lessons learned from implementation of this method in pediatric cardiology.

We describe the process of using existing registry data in the Pediatric Heart Network Residual Lesion Score Study, report stakeholders’ perspectives, and provide recommendations to guide future studies using this methodology.

The Residual Lesion Score Study, a 17-site prospective, observational study, piloted the use of existing local surgical registry data (collected for submission to the Society of Thoracic Surgeons-Congenital Heart Surgery Database) to supplement manual data collection. A survey regarding processes and perceptions was administered to study site and data coordinating center staff.

Survey response rate was 98% (54/55). Overall, 57% perceived that using registry data saved research staff time in the current study, and 74% perceived that it would save time in future studies; 55% noted significant upfront time in developing a methodology for extracting registry data. Survey recommendations included simplifying data extraction processes and tailoring to the needs of the study, understanding registry characteristics to maximise data quality and security, and involving all stakeholders in design and implementation processes.

Use of existing registry data was perceived to save time and promote efficiency. Consideration must be given to the upfront investment of time and resources needed. Ongoing efforts focussed on automating and centralising data management may aid in further optimising this methodology for future studies.

On January 13, 2018, a false ballistic missile alert that lasted 38 minutes was issued across Oahu, Hawaii, United States. As a result of a system failure, an erroneous text message was sent that stated, “Ballistic missile threat inbound to Hawaii. Seek immediate shelter.”

The research team wanted to know the degree of reported anxiety triggered by the event and if knowledge, attitudes, or behaviors for individual/family emergency preparedness (EP) changed post-event.

A 50-question survey that asked about individual and family EP pre- and post-event, and the level of anxiety triggered by the event, was administered to a convenience sample of full-time adult residents of Oahu. The study was conducted over a 6-8 week period post-event. Statistical analysis was used to identify factors associated with an increasing level of EP post-event and reported event-triggered anxiety.

209 participants completed the survey (29% male, 71% female) with about one half living with children. One third were essential workers. Key factors that correlate with increasing various areas of EP post-event include higher educational, receipt of electronic emergency alerts, prior emergency training, and higher reported connectedness to community. Those with higher event anxiety were more likely to develop and practice an EP plan post-event, encourage EP with friends, and report a higher level of community connectedness. The elderly were more likely to have higher levels of EP before and after the event but were less likely to receive emergency alert notifications or have EP training.

While the event was very unfortunate, it did seem to stimulate citizen disaster EP among some groups. Additional research should explore the utility of increasing EP education for communities immediately after disasters, tailoring this education for groups, and targeting the elderly for participation in the emergency alert system.

Prion diseases are rare dementias that most commonly occur sporadically, but can be inherited or acquired, and for which there is no cure. We sought to understand which prion disease symptoms are most problematic for carers, to inform the development of outcome measures.

Self-completed questionnaire with follow-up of a subset of participants by structured interview.

A nested study in the UK National Prion Monitoring Cohort, a longitudinal observational study.

71 carers, of people with different prion diseases with a wide range of disease severity, identified 236 of their four most problematic symptoms by questionnaire which were grouped into ten domains. Structured interviews were then done to qualitatively explore these experiences. Eleven family carers of people with prion disease were selected, including those representative of a range of demographics and disease subtypes and those who cared for people with prion disease, living or recently deceased. Interviews were transcribed and formally studied.

The six most problematic symptom domains were: mobility and coordination; mood and behavior; personal care and continence; eating and swallowing; communication; and cognition and memory. The prevalence of these symptoms varied significantly by disease stage and type. A formal analysis of structured interviews to explore these domains is reported.

We make suggestions about how healthcare professionals can focus their support for people with prion disease. Clinical trials that aim to generate evidence regarding therapies that might confer meaningful benefits to carers should consider including outcome measures that monitor the symptomatic domains we have identified as problematic.

In resource-constrained facilities or during resuscitation, immediate paediatric weight estimation remains a fundamental challenge. We aimed to develop and validate weight estimation models based on ulna length and forearm width and circumference measured by simple and portable tools; and to compare them against previous methods (advanced paediatric life support (APLS), Theron and Traub–Johnson formulas).

Cross-sectional analysis of anthropometric measurements. Four ulna- and forearm-based weight estimation models were developed in the training set (n 1016). Assessment of bias, precision and accuracy was examined in the validation set (n 457).

National Children’s Study-Formative Research in Anthropometry (2011–2012).

Multi-racial/ethnic infants and children aged <6 years (n 1473).

Developed Models 1–4 had high predictive precision (R2=0·91–0·97). Mean percentage errors between predicted and measured weight were significantly smaller across the developed models (0·1–0·7 %) v. the APLS, Theron and Traub–Johnson formulas (−1·7, 9·2 and −4·9 %, respectively). Root-mean-squared percentage error was overall smaller among Models 1–4 v. the three existing methods (range=7·5–8·7 v. 9·8–13·3 %). Further, Models 1–4 were within 10 and 20 % of actual weight in 72–87 and 95–99 % of the weight estimations, respectively, which outperformed any of the three existing methods.

Ulna length, forearm width and forearm circumference by simple and portable tools could serve as valid and reliable surrogate measures of weight among infants and children aged <6 years with improved precision over the existing age- or length-based methods. Further validation of these models in physically impaired or non-ambulatory children is warranted.

Children with CHD and acquired heart disease have unique, high-risk physiology. They may have a higher risk of adverse tracheal-intubation-associated events, as compared with children with non-cardiac disease.

We sought to evaluate the occurrence of adverse tracheal-intubation-associated events in children with cardiac disease compared to children with non-cardiac disease. A retrospective analysis of tracheal intubations from 38 international paediatric ICUs was performed using the National Emergency Airway Registry for Children (NEAR4KIDS) quality improvement registry. The primary outcome was the occurrence of any tracheal-intubation-associated event. Secondary outcomes included the occurrence of severe tracheal-intubation-associated events, multiple intubation attempts, and oxygen desaturation.

A total of 8851 intubations were reported between July, 2012 and March, 2016. Cardiac patients were younger, more likely to have haemodynamic instability, and less likely to have respiratory failure as an indication. The overall frequency of tracheal-intubation-associated events was not different (cardiac: 17% versus non-cardiac: 16%, p=0.13), nor was the rate of severe tracheal-intubation-associated events (cardiac: 7% versus non-cardiac: 6%, p=0.11). Tracheal-intubation-associated cardiac arrest occurred more often in cardiac patients (2.80 versus 1.28%; p<0.001), even after adjusting for patient and provider differences (adjusted odds ratio 1.79; p=0.03). Multiple intubation attempts occurred less often in cardiac patients (p=0.04), and oxygen desaturations occurred more often, even after excluding patients with cyanotic heart disease.

The overall incidence of adverse tracheal-intubation-associated events in cardiac patients was not different from that in non-cardiac patients. However, the presence of a cardiac diagnosis was associated with a higher occurrence of both tracheal-intubation-associated cardiac arrest and oxygen desaturation.

Globally, health systems are struggling with reliably appraising the safety and efficacy of rapidly changing digital health interventions whilst allowing useful innovations to be rapidly adopted. Assessment and regulation of the large number of health apps should be proportional to their clinical risk, but there is large uncertainty about suitable criteria to assess risk (1). We aimed to identify criteria for assessing clinical risks associated with different types of health apps.

Our work builds on previous studies that identified some of the risks that health apps can pose and contextual factors that can moderate these risks (2,3). This work is grounded in a review of existing literature; wide consultation of stakeholders; participation in multi-agency policy discussion; and sense-checking successive versions of the framework that evolved over time. We combined different risk domains for apps (technical safety, usability, intervention quality, and engagement) with their functions (learning, behaviour and cognition change, communication, record keeping, and clinical decision support).

We developed a comprehensive generic risk framework that app users, developers, commissioners, regulators and other stakeholders worldwide can use to guide assessment of the likely risks posed by a specified health app in a specific context. We also propose questions that should help determine whether these risks have been addressed.

Apps are very promising in health care but are very numerous, complex, rapidly evolving and with overlapping functions. A rigorous risk framework should help stakeholders to deal with the large quantity of health apps, classify and manage clinical risks, and improve patient safety by applying generic risk assessment criteria. Further work is needed to test and develop the criteria we propose, especially as apps that integrate different functions are emerging, which will make risk assessment more complex.