Cost-effectiveness of Prednisolone to Treat Bell Palsy in Children: An Economic Evaluation Alongside a Randomized Controlled Trial.

BACKGROUND AND OBJECTIVES: Bell palsy is the third most frequent diagnosis in children with sudden-onset neurologic dysfunction. The cost-effectiveness of treating Bell palsy with prednisolone in children is unknown. We aimed to assess the cost-effectiveness of prednisolone in treating Bell palsy in children compared with placebo. METHODS: This economic evaluation was a prospectively planned secondary analysis of a double-blinded, randomized, placebo-controlled superiority trial (Bell Palsy in Children [BellPIC]) conducted from 2015 to 2020. The time horizon was 6 months since randomization. Children aged 6 months to <18 years who presented within 72 hours of onset of clinician-diagnosed Bell palsy and who completed the trial were included (N = 180). Interventions were oral prednisolone or taste-matched placebo administered for 10 days. Incremental cost-effectiveness ratio comparing prednisolone with placebo was estimated. Costs were considered from a health care sector perspective and included Bell palsy-related medication cost, doctor visits, and medical tests. Effectiveness was measured using quality-adjusted life-years (QALYs) based on Child Health Utility 9D. Nonparametric bootstrapping was performed to capture uncertainties. Prespecified subgroup analysis by age 12 to <18 years vs <12 years was conducted. RESULTS: The mean cost per patient was A$760 in the prednisolone group and A$693 in the placebo group over the 6-month period (difference A$66, 95% CI -A$47 to A$179). QALYs over 6 months were 0.45 in the prednisolone group and 0.44 in the placebo group (difference 0.01, 95% CI -0.01 to 0.03). The incremental cost to achieve 1 additional recovery was estimated to be A$1,577 using prednisolone compared with placebo, and cost per additional QALY gained was A$6,625 using prednisolone compared with placebo. Given a conventional willingness-to-pay threshold of A$50,000 per QALY gained (equivalent to US$35,000 or £28,000), prednisolone is very likely cost-effective (probability is 83%). Subgroup analysis suggests that this was primarily driven by the high probability of prednisolone being cost-effective in children aged 12 to <18 years (probability is 98%) and much less so for those <12 years (probability is 51%). DISCUSSION: This provides new evidence to stakeholders and policymakers when considering whether to make prednisolone available in treating Bell palsy in children aged 12 to <18 years. TRIAL REGISTRATION INFORMATION: Australian New Zealand Clinical Trials Registry ACTRN12615000563561.

Prevalence of high flow nasal cannula therapy use for management of infants with bronchiolitis in Australia and New Zealand.

AIM: To determine the prevalence of high flow nasal cannula (HFNC) therapy in infants presenting to hospital in Australia and New Zealand with bronchiolitis over four bronchiolitis seasons. Secondary aims were to determine temporal trends in HFNC use, and associations between HFNC, hospital length of stay (LOS) and intensive care unit (ICU) admission. METHODS: A planned sub-study of a multi-centre international cluster randomised controlled trial investigating knowledge translation strategies for a bi-national bronchiolitis guideline. Demographics, management and outcomes data were collected retrospectively for infants presenting with bronchiolitis to 26 hospitals between 1 May 2014 and 30 November 2017. Prevalence data are presented as absolute frequencies (95% confidence interval (CI)) with differences between groups for continuous and categorical variables analysed using linear and logistic regression, respectively. RESULTS: 11 715 infants were included with 3392 (29.0%, 95% CI (28.1-29.8%)) receiving oxygen therapy; of whom 1817 (53.6%, 95% CI (51.9-55.3%)) received HFNC. Use of oxygen therapy did not change over the four bronchiolitis seasons (P = 0.12), while the proportion receiving HFNC increased (2014, 336/2587 (43.2%); 2017, 609/3720 (57.8%); P ≤ 0.001). Infants who received HFNC therapy were not substantially different to infants who received oxygen therapy without HFNC. HFNC use was associated with increases in both hospital LOS (P < 0.001) and ICU admissions (P < 0.001). CONCLUSION: Use of HFNC therapy for infants with bronchiolitis increased over 4 years. Of those who received oxygen therapy, the majority received HFNC therapy without improvement in hospital LOS or ICU admissions. Strategies to guide appropriate HFNC use in infants with bronchiolitis are required.

The Pediatric Emergency Research Network: A Decade of Global Research Cooperation in Pediatric Emergency Care.

OBJECTIVES: The Pediatric Emergency Research Network (PERN) was launched in 2009 with the intent for existing national and regional research networks in pediatric emergency care to organize globally for the conduct of collaborative research across networks. METHODS: The Pediatric Emergency Research Network has grown from 5- to 8-member networks over the past decade. With an executive committee comprising representatives from all member networks, PERN plays a supportive and collaborative rather than governing role. The full impact of PERN's facilitation of international collaborative research, although somewhat difficult to quantify empirically, can be measured indirectly by the observed growth of the field, the nature of the increasingly challenging research questions now being addressed, and the collective capacity to generate and implement new knowledge in treating acutely ill and injured children. RESULTS: Beginning as a pandemic response with a high-quality retrospective case-controlled study of H1N1 influenza risk factors, PERN research has progressed to multiple observational studies and ongoing global randomized controlled trials. As a recent example, PERN has developed sufficient network infrastructure to enable the rapid initiation of a prospective observational study in response to the current coronavirus disease 2019 pandemic. In light of the ongoing need for translation of research knowledge into equitable clinical practice and to promote health equity, PERN is committed to a coordinated international effort to increase the uptake of evidence-based management of common and treatable acute conditions in all emergency department settings. CONCLUSIONS: The Pediatric Emergency Research Network's successes with global research, measured by prospective observational and interventional studies, mean that the network can now move to improve its ability to promote the implementation of scientific advances into everyday clinical practice. Achieving this goal will involve focus in 4 areas: (1) expanding the capacity for global randomized controlled trials; (2) deepening the focus on implementation science; (3) increasing attention to healthcare disparities and their origins, with growing momentum toward equity; and (4) expanding PERN's global reach through addition of sites and networks from resource-restricted regions. Through these actions, PERN will be able to build on successes to face the challenges ahead and meet the needs of acutely ill and injured children throughout the world.

First-line oxygen therapy with high-flow in bronchiolitis is not cost saving for the health service.

BACKGROUND: Bronchiolitis is the most common reason for hospital admission in infants. High-flow oxygen therapy has emerged as a new treatment; however, the cost-effectiveness of using it as first-line therapy is unknown. OBJECTIVE: To compare the cost of providing high-flow therapy as a first-line therapy compared with rescue therapy after failure of standard oxygen in the management of bronchiolitis. METHODS: A within-trial economic evaluation from the health service perspective using data from a multicentre randomised controlled trial for hypoxic infants (≤12 months) admitted to hospital with bronchiolitis in Australia and New Zealand. Intervention costs, length of hospital and intensive care stay and associated costs were compared for infants who received first-line treatment with high-flow therapy (early high-flow, n=739) or for infants who received standard oxygen and optional rescue high-flow (rescue high-flow, n=733). Costs were applied using Australian costing sources and are reported in 2016-2017 AU$. RESULTS: The incremental cost to avoid one treatment failure was AU$1778 (95% credible interval (CrI) 207 to 7096). Mean cost of bronchiolitis treatment including intervention costs and costs associated with length of stay was AU$420 (95% CrI -176 to 1002) higher per infant in the early high-flow group compared with the rescue high-flow group. There was an 8% (95% CrI 7.5 to 8.6) likelihood of the early high-flow oxygen therapy being cost saving. CONCLUSIONS: The use of high-flow oxygen as initial therapy for respiratory failure in infants with bronchiolitis is unlikely to be cost saving to the health system, compared with standard oxygen therapy with rescue high-flow.

A Cost-Effectiveness Analysis Comparing Clinical Decision Rules PECARN, CATCH, and CHALICE With Usual Care for the Management of Pediatric Head Injury.

STUDY OBJECTIVE: To determine the cost-effectiveness of 3 clinical decision rules in comparison to Australian and New Zealand usual care: the Children's Head Injury Algorithm for the Prediction of Important Clinical Events (CHALICE), the Pediatric Emergency Care Applied Research Network (PECARN), and the Canadian Assessment of Tomography for Childhood Head Injury (CATCH). METHODS: A decision analytic model was constructed from the Australian health care system perspective to compare costs and outcomes of the 3 clinical decision rules compared with Australian and New Zealand usual care. The study involved multicenter recruitment from 10 Australian and New Zealand hospitals; recruitment was based on the Australian Pediatric Head Injury Rules Study involving 18,913 children younger than 18 years and with a head injury, and with Glasgow Coma Scale score 13 to 15 on presentation to emergency departments (EDs). We determined the cost-effectiveness of the 3 clinical decision rules compared with usual care. RESULTS: Usual care, CHALICE, PECARN, and CATCH strategies cost on average AUD $6,390, $6,423, $6,433, and $6,457 per patient, respectively. Usual care was more effective and less costly than all other strategies and is therefore the dominant strategy. Probabilistic sensitivity analyses showed that when simulated 1,000 times, usual care dominated all clinical decision rules in 61%, 62%, and 60% of simulations (CHALICE, PECARN, and CATCH, respectively). The difference in cost between all rules was less than $36 (95% confidence interval -$7 to $77) and the difference in quality-adjusted life-years was less than 0.00097 (95% confidence interval 0.0015 to 0.00044). Results remained robust under sensitivity analyses. CONCLUSION: This evaluation demonstrated that the 3 published international pediatric head injury clinical decision rules were not more cost-effective than usual care in Australian and New Zealand tertiary EDs. Understanding the usual care context and the likely cost-effectiveness is useful before investing in implementation of clinical decision rules or incorporation into a guideline.

Implementing evidence-based practices in the care of infants with bronchiolitis in Australasian acute care settings: study protocol for a cluster randomised controlled study.

BACKGROUND: Bronchiolitis is the most common reason for admission to hospital for infants less than one year of age. Although management is well defined, there is substantial variation in practice, with infants receiving ineffective therapies or management. This study will test the effectiveness of tailored, theory informed knowledge translation (KT) interventions to decrease the use of five clinical therapies or management processes known to be of no benefit, compared to usual dissemination practices in infants with bronchiolitis. The primary objective is to establish whether the KT interventions are effective in increasing compliance to five evidence based recommendations in the first 24 h following presentation to hospital. The five recommendations are that infants do not receive; salbutamol, antibiotics, glucocorticoids, adrenaline, or a chest x-ray. METHODS/DESIGN: This study is designed as a cluster randomised controlled trial. We will recruit 24 hospitals in Australia and New Zealand, stratified by country and provision of tertiary or secondary paediatric care. Hospitals will be randomised to either control or intervention groups. Control hospitals will receive a copy of the recent Australasian Bronchiolitis Guideline. Intervention hospitals will receive KT interventions informed by a qualitative analysis of factors influencing clinician care of infants with bronchiolitis. Key interventions include, local stakeholder meetings, identifying medical and nursing clinical leads in both emergency departments and paediatric inpatient areas who will attend a single education train-the-trainer day to then deliver standardised staff education with the training materials provided and coordinate audit and feedback reports locally over the study period. Data will be extracted retrospectively for three years prior to the study intervention year, and for seven months of the study intervention year bronchiolitis season following intervention delivery to determine compliance with the five evidence-based recommendations. Data will be collected to assess fidelity to the implementation strategies and to facilitate an economic evaluation. DISCUSSION: This study will contribute to the body of knowledge to determine the effectiveness of tailored, theory informed interventions in acute care paediatric settings, with the aim of reducing the evidence to practice gaps in the care of infants with bronchiolitis. TRIAL REGISTRATION: Australian New Zealand Clinical Trials Registry ACTRN12616001567415 (retrospectively registered on 14 November 2016).