Acceptability and feasibility of an online information linker service for caregivers who have a child with genetic epilepsy: a mixed-method pilot study protocol.

INTRODUCTION: Developmental and epileptic encephalopathies (DEEs) are rare epilepsy conditions that collectively impact 1 in 2000 children. They are highly genetically heterogeneous, resulting in significant barriers to accurate and adequate information for caregivers. This can lead to increased distress and dissatisfaction with the healthcare system. To address this gap, we developed 'GenE Compass' to provide caregivers with the highest-quality possible, understandable and relevant information in response to specific questions about their child's DEE. Using a mixed-method design, we will now pilot GenE Compass to evaluate the acceptability to caregivers and clinicians, feasibility and impact to caregivers. METHODS AND ANALYSIS: We will recruit 88 caregivers (estimated final sample of 50 at follow-up) who have a child under 18 years of age with a suspected or confirmed DEE diagnosis. Following consent and a baseline questionnaire (questionnaire 1 (Q1)), participants will be able to submit questions to GenE Compass over a 3-month period. After 3 months, participants will complete a follow-up questionnaire (Q2) and an optional telephone interview to answer the research questions. Primary outcomes are acceptability of GenE Compass and feasibility of delivering the intervention (eg, cost of the intervention, number of questions submitted and time taken to respond to questions). Secondary outcomes include the impact of GenE Compass on caregivers' quality of life, information searching behaviours, perceptions of their child's illness and activation. ETHICS AND DISCUSSION: The study protocol (V.2, dated 16 September 2021) has been approved by the Sydney Children's Hospitals Network Human Research Ethics Committee (ETH11277). The results will be disseminated in peer-reviewed journals and at scientific conferences. A lay summary will be disseminated to all participants. TRIAL REGISTRATION NUMBER: ACTRN12621001544864.

The cost-effectiveness of progesterone in preventing miscarriages in women with early pregnancy bleeding: an economic evaluation based on the PRISM trial.

OBJECTIVES: To assess the cost-effectiveness of progesterone compared with placebo in preventing pregnancy loss in women with early pregnancy vaginal bleeding. DESIGN: Economic evaluation alongside a large multi-centre randomised placebo-controlled trial. SETTING: Forty-eight UK NHS early pregnancy units. POPULATION: Four thousand one hundred and fifty-three women aged 16-39 years with bleeding in early pregnancy and ultrasound evidence of an intrauterine sac. METHODS: An incremental cost-effectiveness analysis was performed from National Health Service (NHS) and NHS and Personal Social Services perspectives. Subgroup analyses were carried out on women with one or more and three or more previous miscarriages. MAIN OUTCOME MEASURES: Cost per additional live birth at ≥34 weeks of gestation. RESULTS: Progesterone intervention led to an effect difference of 0.022 (95% CI -0.004 to 0.050) in the trial. The mean cost per woman in the progesterone group was £76 (95% CI -£559 to £711) more than the mean cost in the placebo group. The incremental cost-effectiveness ratio for progesterone compared with placebo was £3305 per additional live birth. For women with at least one previous miscarriage, progesterone was more effective than placebo with an effect difference of 0.055 (95% CI 0.014-0.096) and this was associated with a cost saving of £322 (95% CI -£1318 to £673). CONCLUSIONS: The results suggest that progesterone is associated with a small positive impact and a small additional cost. Both subgroup analyses were more favourable, especially for women who had one or more previous miscarriages. Given available evidence, progesterone is likely to be a cost-effective intervention, particularly for women with previous miscarriage(s). TWEETABLE ABSTRACT: Progesterone treatment is likely to be cost-effective in women with early pregnancy bleeding and a history of miscarriage.

Ultrasound bladder wall thickness and detrusor overactivity: a multicentre test accuracy study.

OBJECTIVE: Women with overactive bladder (OAB) often undergo urodynamics before invasive treatments are considered. Ultrasound measurement of bladder wall thickness (BWT) is a less invasive, less expensive and widely available test. It has the potential to diagnose the presence of detrusor overactivity (DO). We aimed to evaluate the accuracy of BWT in the diagnosis of DO. DESIGN: Prospective cohort study. SETTING: Twenty-two UK clinics (university and district general hospitals). METHODS: Consecutive eligible women with OAB symptoms had transvaginal ultrasound to estimate BWT (index test). The reference standard for the diagnosis of DO was urodynamic testing with multichannel subtracted cystometry. MAIN OUTCOME MEASURES: The sensitivity, specificity and likelihood ratios using a BWT threshold of ≥5 mm were used to indicate the presence of DO, and the area under the receiver operating characteristics (ROC) curve to give an overall estimate of BWT accuracy. RESULTS: Between March 2011 and 2013, 644/687 (94%) women recruited had both tests. The mean age was 52.7 years (standard deviation 13.9) and DO was diagnosed in 399/666 (60%) women. BWT had a sensitivity of 43% [95% confidence interval (CI) 38-48%], specificity of 62% (95% CI 55-68%), and likelihood ratios of 1.11 (95% CI 0.92-1.35) and 0.93 (95% CI 0.82-1.06) for positive and negative tests, respectively. The area under the ROC curve was 0.53 (95% CI 0.48-0.57). Extensive sensitivity analyses and subgroup analyses were carried out, but did not alter the interpretation. CONCLUSIONS: BWT is not a good replacement test for urodynamics in women with overactive bladder. TWEETABLE ABSTRACT: Bladder wall thickness is not a good replacement test for urodynamics in women with overactive bladder.