Health economic evaluation of blended collaborative care for older multimorbid heart failure patients: study protocol.

BACKGROUND: Integrated care, in particular the 'Blended Collaborative Care (BCC)' strategy, may have the potential to improve health-related quality of life (HRQoL) in multimorbid patients with heart failure (HF) and psychosocial burden at no or low additional cost. The ESCAPE trial is a randomised controlled trial for the evaluation of a BCC approach in five European countries. For the economic evaluation of alongside this trial, the four main objectives were: (i) to document the costs of delivering the intervention, (ii) to assess the running costs across study sites, (iii) to evaluate short-term cost-effectiveness and cost-utility compared to providers' usual care, and (iv) to examine the budgetary implications. METHODS: The trial-based economic analyses will include cross-country cost-effectiveness and cost-utility assessments from a payer perspective. The cost-utility analysis will calculate quality-adjusted life years (QALYs) using the EQ-5D-5L and national value sets. Cost-effectiveness will include the cost per hospital admission avoided and the cost per depression-free days (DFD). Resource use will be measured from different sources, including electronic medical health records, standardised questionnaires, patient receipts and a care manager survey. Uncertainty will be addressed using bootstrapping. DISCUSSION: The various methods and approaches used for data acquisition should provide insights into the potential benefits and cost-effectiveness of a BCC intervention. Providing the economic evaluation of ESCAPE will contribute to a country-based structural and organisational planning of BCC (e.g., the number of patients that may benefit, how many care managers are needed). Improved care is expected to enhance health-related quality of life at little or no extra cost. TRIAL REGISTRATION: The study follows CHEERS2022 and is registered at the German Clinical Trials Register (DRKS00025120).

Health-related quality of life in patients with diabetic foot ulcers in the Arab world: a systematic review.

OBJECTIVE: To conduct a systematic review to identify the impact of diabetic foot ulceration (DFU) on health-related quality of life (HRQoL) in individuals within the Arab world. METHOD: A PRISMA-guided systematic search for HRQoL studies in Arab populations was conducted in CINAHL, PubMed, Scopus and EBSCO. Relevant studies were critically appraised using the STROBE statement checklist. RESULTS: A total of five studies were included. Three studies originated from Saudi Arabia, one from Jordan and one from Tunisia. The studies consistently demonstrated lower (poorer) HRQoL in patients with DFU, and worse HRQoL compared with both patients with diabetes and no DFU, and with healthy subjects. CONCLUSION: This review confirmed the negative impact of DFU on HRQoL in individuals with diabetes. It also highlights the scarcity of HRQoL studies from the Arab world. However, given that all studies included were conducted between 2013-2019, this could reflect a growing interest in DFU and HRQoL in the Arab world, and could potentially indicate that more studies will follow. In light of this, there is a need for a renewed focus on the completion of a high-quality standardised approach to research in this region.

Monopolar diathermy versus a vessel-sealing device for reducing postoperative drain output after simple mastectomy: randomized clinical trial.

BACKGROUND: Electrosurgical devices are commonly used during mastectomy for simultaneous dissection and haemostasis, and can provide potential benefits regarding vessel and lymphatic ligation. The aim of this prospective RCT was to assess whether using a vessel-sealing device (LigaSure™) improves perioperative outcomes compared with monopolar diathermy when performing simple mastectomy. METHODS: Patients were recruited prospectively and randomized in a 1 : 1 manner to undergo simple mastectomy using either LigaSure™ or conventional monopolar diathermy at a single centre. The primary outcome was the number of days the drain remained in situ after surgery. Secondary outcomes of interest included operating time and complications. RESULTS: A total of 86 patients were recruited (42 were randomized to the monopolar diathermy group and 44 were randomized to the LigaSure™ group). There was no significant difference in the mean number of days the drain remained in situ between the monopolar diathermy group and the LigaSure™ group (7.75 days versus 8.23 days; P = 0.613) and there was no significant difference in the mean total drain output between the monopolar diathermy group and the LigaSure™ group (523.50 ml versus 572.80 ml; P = 0.694). In addition, there was no significant difference in the mean operating time between the groups, for simple mastectomy alone (88.25 min for the monopolar diathermy group versus 107.20 min for the LigaSure™ group; P = 0.078) and simple mastectomy with sentinel lymph node biopsy (107.20 min for the monopolar diathermy group versus 114.40 min for the LigaSure™ group; P = 0.440). CONCLUSION: In this double-blinded single-centre RCT, there was no difference in the total drain output or the number of days the drain remained in situ between the monopolar diathermy group and the LigaSure™ group. REGISTRATION NUMBER: EudraCT 2018-003191-13 BEAUMONT HOSPITAL REC 18/66.

Lifecycle evaluation of medical devices: supporting or jeopardizing patient outcomes? A comparative analysis of evaluation models.

OBJECTIVES: Lack of evidence regarding safety and effectiveness at market entry is driving the need to consider adopting a lifecycle approach to evaluating medical devices, but it is unclear what lifecycle evaluation means. This research sought to explore the tacit meanings of "lifecycle" and "lifecycle evaluation" as embodied within evaluation models/frameworks used for medical devices. METHODS: Drawing on qualitative evidence synthesis methods and using an inductive approach, novel methods were developed to identify, appraise, analyze, and synthesize lifecycle evaluation models used for medical devices. Data was extracted (including purpose; audience; characterization; outputs; timing; and type of model) from key texts for coding, categorization, and comparison, exploring embodied meaning across four broad perspectives. RESULTS: Fifty-two models were included in the synthesis. They demonstrated significant heterogeneity of meaning, form, scope, timing, and purpose. The "lifecycle" may represent a single stage, a series of stages, a cycle of innovation, or a system. "Lifecycle evaluation" focuses on the overarching goal of the stakeholder group, and may use a single or repeated evaluation to inform decision-making regarding the adoption of health technologies (Healthcare), resource allocation (Policymaking), investment in new product development or marketing (Trade and Industry), or market regulation (Regulation). The adoption of a lifecycle approach by regulators has resulted in the deferral of evidence generation to the post-market phase. CONCLUSIONS: Using a "lifecycle evaluation" approach to inform reimbursement decision-making must not be allowed to further jeopardize evidence generation and patient safety by accepting inadequate evidence of safety and effectiveness for reimbursement decisions.

Validation and comparison of two new scoring systems for the prediction of complicated versus uncomplicated appendicitis.

OBJECTIVES: To validate the Atema and APSI scoring systems in the diagnosis of complicated vs uncomplicated appendicitis. To compare these scoring systems with computed tomography (CT) imaging alone to establish which method provides most accurate prediction of complicated vs uncomplicated appendicitis. METHODS: This was a retrospective review of a sample of 160 patients that underwent appendicectomy and CT imaging for suspected appendicitis between 2018 and 2021 in a tertiary university teaching hospital. Each scoring system was applied to all patients and results analysed and compared with the effectiveness of CT imaging, RESULTS: 32.5% (n = 52) were found to have complicated appendicitis and 67.5% (n = 108) uncomplicated appendicitis. Application of the Atema score to our cohort of patients resulted in a sensitivity 76.9% [CI (64.2, 87.5), specificity 58.7% [CI (48.9, 68.1)], PPV 47.1% [CI (40.5, 53.8) and NPV 84.2% [CI (76.0, 89.9)]. By comparison, the APSI yielded a sensitivity 50.9% [CI (36.6, 65.4)], specificity 76.1% [CI (67.0, 87.8)], PPV 50% [CI (39.2, 60.6)] and NPV 76% [CI (71.1, 81.7)]. Radiology prediction of complicated vs uncomplicated appendicitis with CT imaging showed sensitivity 46% [CI (32.2, 60.5)], specificity 79%; [CI (69.8, 86)], PPV 51% [CI (39.6, 62.5)] and NPV 75% [CI (69.8, 79.9)]. CONCLUSION: By comparing the APSI and Atema et al. scoring systems with CT reporting in our hospital, it appears that the Atema may confer some benefit in stratifying patient risk of complicated versus uncomplicated appendicitis. Further larger scale prospective studies are required.

Composite quality measures of abdominal surgery at a population level: systematic review.

BACKGROUND: Measurement of surgical quality at a population level is challenging. Composite quality measures derived from administrative and clinical information systems could support system-wide surgical quality improvement by providing a simple metric that can be evaluated over time. The aim of this systematic review was to identify published studies of composite measures used to assess the overall quality of abdominal surgical services at a hospital or population level. METHODS: A search was conducted in PubMed and MEDLINE for references describing measurement instruments evaluating the overall quality of abdominal surgery. Instruments combining multiple process and quality indicators into a single composite quality score were included. The identified instruments were described in terms of transparency, justification, handling of missing data, case-mix adjustment, scale branding and choice of weight and uncertainty to assess their relative strengths and weaknesses (PROSPERO registration: CRD42022345074). RESULTS: Of 5234 manuscripts screened, 13 were included. Ten unique composite quality measures were identified, mostly developed within the past decade. Outcome measures such as mortality rate (40 per cent), length of stay (40 per cent), complication rate (60 per cent) and morbidity rate (70 per cent) were consistently included. A major challenge for all instruments is the reliance of valid administrative data and the challenges of assigning appropriate weights to the underlying instrument components. A conceptual framework for composite measures of surgical quality was developed. CONCLUSION: None of the composite quality measures identified demonstrated marked superiority over others. The degree to which administrative and clinical data influences each composite measure differs in important ways. There is a need for further testing and development of these measures.

Hospital response to Activity-Based Funding and price incentives: Evidence from Ireland.

Activity-Based Funding (ABF) is a funding policy incentivising hospitals to deliver more efficient care. ABF can be complemented by additional price incentives to further drive hospital efficiency. In 2016, ABF was introduced for public patients admitted to Irish public hospitals. Additionally, a price incentive to perform laparoscopic cholecystectomy as day-case surgery was introduced in 2018. Private patient activity in public hospitals was subject to neither ABF nor price incentive. Using national Hospital In-Patient-Enquiry activity data 2013-2019, we evaluated the impact of ABF and the price incentive for laparoscopic cholecystectomy surgery in Ireland. We exploit variation in hospital payment for public and private patients treated in public acute Irish hospitals and employ a Propensity Score Matching Difference-in-Differences approach. We estimate the funding change impacts across outcomes measuring the proportion of day-case admissions and length of stay. We found no significant impact for either outcomes linked to ABF introduction. Similarly, no impacts linked to the price incentive were observed. It appears providers of laparoscopic cholecystectomy in Irish public hospitals did not react to the new funding mechanisms. The implementation of the funding policies did not improve hospital efficiency. Further strengthening of these new funding mechanisms are required to deliver more efficient care.

Costs associated with adverse drug reactions in an older population admitted to hospital: a prospective cohort study.

PURPOSE: This study examines healthcare costs associated with adverse drug reactions (ADR) in an older population admitted acutely to an Irish tertiary hospital. METHODS: Prospective cohort study involving older persons admitted to hospital with and without an ADR. Data was collected at baseline, during hospitalisation and post-discharge. Participants provided information on healthcare resource use three months before admission (baseline) and three months after discharge (follow-up). For each healthcare resource, unit costs were derived and applied. The average cost (standard deviation (SD)) associated with the hospital admission for the ADR and non-ADR are presented. In addition, baseline and follow-up care costs were compared using difference-in-difference analysis and presented with 95% confidence intervals (CI). Costs by preventability and severity of ADR are also presented. RESULTS: A total of n = 230 participants were included (n = 93 ADR and n = 137 without ADR). The average cost associated with hospital admission for an ADR was €9538 (SD €10442) and €9828 (SD €11770) for non-ADR. The additional follow-up costs (difference-in-difference) associated with the ADR was estimated at €2047 (95% CI: -€889 to €4983). The mean incremental follow-up cost of definite preventable ADRs was estimated at €1648 (95% CI: -€4310 to €7605), possible preventable ADRs €2259 (95 CI: -€1194 to €5712) and unavoidable ADRs €1757 (95% CI: -€3377 to €6890). The mean incremental follow-up cost associated with moderate severe ADRs was estimated at €1922 (95% CI: -€1088 to €4932) and €3580 (95% CI: -€4898 to €12,058) for severe ADRs.  CONCLUSION: ADRs leading to hospital admission are associated with modest incremental healthcare costs during and three months after admission. Severe and possibly preventable ADRs were associated with higher costs.

Establishing an expert opinion framework for lung volume reduction in Ireland: a Delphi consensus technique.

BACKGROUND: Lung volume reduction (LVR) is an effective treatment option offered to patients with emphysema. There is no formalised LVR referral network in Ireland. A rigorous approach to agreeing and implementing a LVR referral framework in an Irish context is required. A Delphi process was used to provide a basis for a framework of multi-disciplinary teams (MDTs) which can provide LVR as a management option. A Delphi process offers a framework for understanding variations and developing a consensus from expert opinion. AIM: The aim of this study was to develop consensus on recommendations for LVR referral guidelines in an Irish context and provide a national scope based on current practice and evidence. DESIGN: In accordance with Guidance on Conducting and Reporting Delphi Studies, a consensus-building Delphi study was performed. Thirty-three statements informed from review of research literature were identified and presented to participants. Evaluation of the statements was performed by an expert panel using a 5-point Likert scale. ≥ 70% agreement was defined as consensus and items with a consensus rating of < 70% were revised during the process. In total, Delphi questionnaires were distributed to 18 experts with a response rate of 78% (n = 14) and a follow-up response-rate of 50% (n = 7). SETTING/PARTICIPANTS: The expert panel in Ireland consisted of representatives from respiratory medicine, cardiothoracic surgery and allied-health professionals with expertise in COPD care. RESULTS: Of the initial 33 statements in five dimensions, there were consensus regarding 31 statements. CONCLUSIONS: The 31 statements agreed through this Delphi study clarify a coherent direction for development of a LVR framework in Ireland. The Delphi study methodology described is a useful process to reach consensus among multi-disciplinary experts.

Assessing the healthcare costs associated with venous leg ulcer compression bandages - A scoping review.

AIM: To determine the monetary costs identified in economic evaluations of treatment with compression bandages among adults with venous leg ulcers (VLU). METHOD: A scoping review of existing publications was conducted in February 2023. The Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines were used. RESULTS: Ten studies met the inclusion criteria. To place the costs of treatment into context, these are reported in conjunction with the healing rates. Three comparisons were made: 1.4 layer compression versus no compression (3 studies). One study reported that 4 layer compression was more expensive than usual care (£804.03 vs £681.04, respectively), while the 2 other studies reported the converse (£145 vs £162, respectively) and all costs (£116.87 vs £240.28 respectively). Within the three studies, the odds of healing were statistically significantly greater with 4 layer bandaging (OR: 2.20; 95% CI: 1.54-3.15; p = 0.001).; 2.4 layer compression versus other compression (6 studies). For the three studies reporting the mean costs per patient associated with treatment (bandages alone), over the treatment period, analysis identified a mean difference (MD) in costs for 4 layer vs comparator 1 (2 layer compression, short-stretch compression, 2 layer compression hosiery, 2 layer cohesive compression, 2 layer compression) of -41.60 (95% CI: 91.40 to 8.20; p = 0.10). The OR of healing for 4 layer compression vs comparator 1 (2 layer compression, short-stretch compression, 2 layer compression hosiery, 2 layer cohesive compression, 2 layer compression) is: 0.70 (95% CI: 0.57-0.85; p = 0.004). For 4 layer vs comparator 2 (2 layer compression) the MD is: 14.00 (95% CI: 53.66 to -25.66; p < 0.49). The OR of healing for 4 layer compression vs comparator 2 (2 layer compression) is: 3.26 (95% CI: 2.54-4.18; p < 0.00001). For comparator 1 (2 layer compression, short-stretch compression, 2 layer compression hosiery, 2 layer cohesive compression, 2 layer compression) vs comparator 2 (2 layer compression) the MD in costs is: 55.60 (95% CI: 95.26 to -15.94; p = 0.006). The OR of healing with Comparator 1 (2 layer compression, short-stretch compression, 2 layer compression hosiery, 2 layer cohesive compression, 2 layer compression) is: 5.03 (95% CI:4.10-6.17; p < 0.00001). Three studies presented the mean annual costs per patient associated with treatment (all costs). The MD is 172 (150-194; p = 0.401), indicating no statistically significant difference in costs between the groups. All studies showed faster healing rates in the 4 layer study groups. 3. Compression wrap versus inelastic bandage (one study). Compression wrap was less expensive than inelastic bandage (£201 vs £335, respectively) with more wounds healing in the compression wrap group (78.8%, n = 26/33; 69.7%, n = 23/33). CONCLUSION: The results for the analysis of costs varied across the included studies. As with the primary outcome, the results indicated that the costs of compression therapy are inconsistent. Given the methodological heterogeneity among studies, future studies in this area are needed and these should use specific methodological guidelines to generate high-quality health economic studies.

The health-related quality of life in patients with diabetic foot ulcers in the Kingdom of Bahrain.

AIMS: The aims of this study were to assess the health-related quality of life (HRQoL) of adult Bahraini patients with diabetic foot ulcers (DFU) and to explore factors associated with poor HRQoL. METHODS: Cross-sectional HRQoL data were obtained from a sample of patients in active treatment for DFU at a large public hospital in Bahrain. Patient-reported HRQOL was measured using the following instruments: DFS-SF, CWIS and EQ-5D. RESULTS: The patient sample included 94 patients, with a mean age of 61.8 (SD: 9.9) years, 54 (57.5%) were males, and 68 (72.3%) were native Bahrainis. Poorer HRQoL was found among patients who were unemployed, divorced/widowed, and those with a shorter duration of formal education. Additionally, patients with severe DFUs, persisting ulcers, and a longer duration of diabetes reported statistically significantly poorer HRQoL. CONCLUSIONS: Findings from this study demonstrate a low level of HRQoL among Bahraini patients with DFUs. A longer duration of diabetes, in addition to ulcer severity and status statistically significantly influence HRQoL.

Patient and hospital-level factors associated with time to surgery after hip fracture in Ireland: Analysis of national audit data 2016-2020.

INTRODUCTION: In hip fracture care, time to surgery (TTS) is a commonly used quality indicator associated with patient outcomes including mortality. This study aimed to identify patient and hospital-level characteristics associated with TTS in Ireland. METHODS: National data from the Irish Hip Fracture Database (IHFD) (2016-2020) were analysed along with hospital-level characteristics obtained from a 2020 organisational survey. Generalised linear model regression was used to explore the association of TTS with case-mix, surgical details, hospital-level staffing and specific protocols recommended to expedite surgery. RESULTS: A total of 14,951 patients with surgically treated hip fracture from 16 hospitals were included (Mean age= 80.6 years (SD=8.8), 70.4% female). Mean TTS was 40.9 h (SD=60.3 h). Case-mix factors associated with longer TTS were male sex and higher American Society of Anaesthesiologists (ASA) grade. Other factors found to be associated with longer TTS included low pre-morbid mobility, inter-hospital transfer, weekday presentation, pre-operative medical physician assessment, intracapsular fracture type, arthroplasty surgery, general anaesthesia, consultant grade of surgeon and lower hospital-level orthopaedic surgical capacity. The oldest age-group and pre-fracture nursing home residence were associated with shorter TTS when adjusted for other case-mix factors. None of four explored protocols for expediting surgery were associated with TTS. CONCLUSION: Patients with more comorbidity experience longer surgical delay after hip fracture in Ireland, in line with international research. Low availability of senior orthopaedic surgeons in Ireland may be delaying hip fracture surgery. Pathway of presentation, including via inter-hospital transfer or hospital bypass, is an important factor that requires further exploration. Further research is required to identify successful system-level protocols and interventions that may expedite hip fracture surgery within this setting.

Healthcare Costs in the Year Before and After Alzheimer's Disease Diagnosis: A Danish Register-Based Matched Cohort Study.

BACKGROUND: Alzheimer's disease (AD) carries a significant economic burden, with costs peaking around the time of diagnosis. However, the cost of diagnosis, including the time leading up to it, has not been studied thoroughly. Furthermore, regionalized healthcare structure could result in differences in the pre-diagnostic costs for people with suspected AD. OBJECTIVE: This study set out to estimate the excess healthcare costs before and after AD diagnosis compared to a matched non-AD population and to investigate regional variation in AD healthcare costs in Denmark. METHODS: We used a register-based cohort of 25,523 matched pairs of new cases of AD and non-AD controls. The healthcare costs included costs on medication, and inpatient-, outpatient-, and primary care visits. Generalized estimating equations were employed to estimate the excess healthcare cost attributable to diagnosing AD, and the variation in costs across regions. RESULTS: Mean excess costs attributable to AD were € 3,284 and € 6,173 in the year before and after diagnosis, respectively. Regional differences in healthcare costs were identified in both the AD and control groups and were more pronounced in patients with AD (PwAD). CONCLUSION: PwAD incur higher healthcare costs across all cost categories in the year before and after diagnosis. Regional differences in healthcare utilization by PwAD may reveal potential variation in access to healthcare. These findings suggest that a more standardized and targeted diagnostic process may help reduce costs and variation in access to healthcare.

Integrated care for older multimorbid heart failure patients: protocol for the ESCAPE randomized trial and cohort study.

ESCAPE: Evaluation of a patient-centred biopsychosocial blended collaborative care pathway for the treatment of multimorbid elderly patients. THERAPEUTIC AREA: Healthcare interventions for the management of older patients with multiple morbidities. AIMS: Multi-morbidity treatment is an increasing challenge for healthcare systems in ageing societies. This comprehensive cohort study with embedded randomized controlled trial tests an integrated biopsychosocial care model for multimorbid elderly patients. HYPOTHESIS: A holistic, patient-centred pro-active 9-month intervention based on the blended collaborative care (BCC) approach and enhanced by information and communication technologies can improve health-related quality of life (HRQoL) and disease outcomes as compared with usual care at 9 months. METHODS: Across six European countries, ESCAPE is recruiting patients with heart failure, mental distress/disorder plus ≥2 medical co-morbidities into an observational cohort study. Within the cohort study, 300 patients will be included in a randomized controlled assessor-blinded two-arm parallel group interventional clinical trial (RCT). In the intervention, trained care managers (CMs) regularly support patients and informal carers in managing their multiple health problems. Supervised by a clinical specialist team, CMs remotely support patients in implementing the treatment plan-customized to the patients' individual needs and preferences-into their daily lives and liaise with patients' healthcare providers. An eHealth platform with an integrated patient registry guides the intervention and helps to empower patients and informal carers. HRQoL measured with the EQ-5D-5L as primary endpoint, and secondary outcomes, that is, medical and patient-reported outcomes, healthcare costs, cost-effectiveness, and informal carer burden, will be assessed at 9 and ≥18 months. CONCLUSIONS: If proven effective, the ESCAPE BCC intervention can be implemented in routine care for older patients with multiple morbidities across the participating countries and beyond.

To develop, implement and evaluate an integrated care pathway for patients presenting with pneumothorax.

BACKGROUND AND PURPOSE: Pneumothorax is a common presentation to acute healthcare services in Ireland, however there is wide variation in management approaches between centres. There is robust evidence to demonstrate that ambulatory management of pneumothorax is feasible and safe. The purpose of this study was to evaluate whether the implementation of an integrated care pathway (ICP) for pneumothorax patients with a focus on ambulatory care would be economically beneficial for the healthcare system. METHODS: This study developed, implemented and evaluated an ICP for all patients presenting with pneumothorax, with a specific focus on ambulatory management for suitable patients. The ICP was designed to be utilised in the Irish healthcare setting, and was evaluated using a prospective multi-centre observational study, with a rigorous economic analysis at the centre of study design. MAIN FINDINGS: Implementation of the ICP resulted in a statistically significant reduction in inpatient length of stay of 2.84 days from 7.4 to 4.56 days (p = 0.001). The incremental per patient cost reduction of treating a patient according to the pneumothorax ICP was 2314 euro. There were no adverse events related to drain insertion at the study sites. CONCLUSIONS: This study demonstrates therefore that standardisation of care for pneumothorax patients with a focus on ambulatory management are economically beneficial for the publicly-funded healthcare service. It is envisaged that this work will be used to inform healthcare policy at a national level across Ireland.

Treatment of inborn errors of immunity patients with inflammatory bowel disease phenotype by allogeneic stem cell transplantation.

Patients with inborn errors of immunity (IEI) can suffer from treatment-refractory inflammatory bowel disease (IBD) causing failure to thrive and consequences of long-term multiple immunosuppressive treatments. Allogeneic haematopoietic stem cell transplantation (alloHSCT) can serve as a curative treatment option. In this single-centre retrospective cohort study we report on 11 paediatric and young adult IEI patients with IBD and failure to thrive, who had exhausted symptomatic treatment options and received alloHSCT. The cohort included chronic granulomatous disease (CGD), lipopolysaccharide-responsive and beige-like anchor protein (LRBA) deficiency, STAT3 gain-of-function (GOF), Wiskott-Aldrich syndrome (WAS), dedicator of cytokinesis 8 (DOCK8) deficiency and one patient without genetic diagnosis. All patients achieved stable engraftment and immune reconstitution, and gastrointestinal symptoms were resolved after alloHSCT. The overall survival was 11/11 over a median follow-up of 34.7 months. Graft-versus-host disease (GVHD) was limited to grade I-II acute GVHD (n = 5), one case of grade IV acute GVHD and one case of limited chronic GVHD. Since treatment recommendations are limited, this work provides a centre-specific approach to treatment prior to transplant as well as conditioning in IEI patients with severe IBD.

A comparison of four quasi-experimental methods: an analysis of the introduction of activity-based funding in Ireland.

BACKGROUND: Health services research often relies on quasi-experimental study designs in the estimation of treatment effects of a policy change or an intervention. The aim of this study is to compare some of the commonly used non-experimental methods in estimating intervention effects, and to highlight their relative strengths and weaknesses. We estimate the effects of Activity-Based Funding, a hospital financing reform of Irish public hospitals, introduced in 2016. METHODS: We estimate and compare four analytical methods: Interrupted time series analysis, Difference-in-Differences, Propensity Score Matching Difference-in-Differences and the Synthetic Control method. Specifically, we focus on the comparison between the control-treatment methods and the non-control-treatment approach, interrupted time series analysis. Our empirical example evaluated the length of stay impact post hip replacement surgery, following the introduction of Activity-Based Funding in Ireland. We also contribute to the very limited research reporting the impacts of Activity-Based-Funding within the Irish context. RESULTS: Interrupted time-series analysis produced statistically significant results different in interpretation, while the Difference-in-Differences, Propensity Score Matching Difference-in-Differences and Synthetic Control methods incorporating control groups, suggested no statistically significant intervention effect, on patient length of stay. CONCLUSION: Our analysis confirms that different analytical methods for estimating intervention effects provide different assessments of the intervention effects. It is crucial that researchers employ appropriate designs which incorporate a counterfactual framework. Such methods tend to be more robust and provide a stronger basis for evidence-based policy-making.

A field trial on the effect of cross-fostering and weaning age on daily gain and disease resilience in weaned pigs.

Alternative management strategies that can increase disease resilience and reduce antibiotics in weaned pigs are needed. Our objective was to compare the effect of two nursing strategies and weaning ages on weight gain, clinical health and antibiotic treatments in weaned pigs not provided with medical zinc oxide in feed. A 2×2 factorial trial was conducted in three Danish commercial herds. Nursing strategies tested were "cross-fostering allowed" (CF) vs. "cross-fostering not allowed" (non-CF). Weaning ages tested were four (24-29 days) vs. five weeks (31-35 days). Pigs were followed from weaning until 33-35 days post-weaning. Herd staff made decisions on and registered antibiotic treatment, removal of pigs to sick pens and mortality. Pigs were weighed at weaning (N = 3139) and on day 33-35 post-weaning (N = 2898). Clinical examinations were carried out on day 4, 7 and 33-35 post-weaning. The effect of nursing strategy and weaning age on weight gain, clinical health and antibiotic treatments was analysed by mixed linear and logistic models. We found that pigs weaned at five weeks of age gained 103.6 g more daily compared to pigs weaned at four weeks during the 33-35 days post-weaning (<0.001). Weaning age affected diarrhoea prevalence, but the effect differed between herds. For pigs weaned at five weeks compared to four weeks of age, the odds for diarrhoea one week post-weaning was 0.7 times lower in one herd whereas the odds for diarrhoea were 2.0 and 1.4 times higher in the two other herds, respectively (P < 0.05). In all herds, we found fewer runted (OR=0.28, P < 0.001) and thin (OR=0.23, P < 0.001) pigs 33-35 days post-weaning in pigs weaned at five weeks of age compared to four weeks. Furthermore, in all herds, CF pigs were more likely to be removed to a sick pen or to die. In total, 5.9%, 13.6% and 64.9% of the studied weaned pigs were treated with antibiotics in the three herds, respectively. Treatment prevalence did not associate to weaning age or nursing strategy and did not in all cases appear to be linked with diarrhoea prevalence. The results indicate that a higher weaning age and less cross-fostering to some extend increase disease resilience post-weaning but herd specific factors interacted with the effects. The prevalence of pigs treated with antibiotics were herd dependent and may relate more to management decisions than to disease level.

A protocol to evaluate the impact of embedding Public and Patient Involvement in a structured PhD program for stroke care.

Background: Embedding Public and Patient Involvement (PPI) in postgraduate research has been recognized as an important component of post-graduate training, providing research scholars with an awareness and a skillset in an area which prepares them for future roles as healthcare researchers. Improving Pathways for Acute STroke And Rehabilitation (iPASTAR) is a structured PhD training program [Collaborative Doctoral Award (CDA)] which aims to design a person-centered stroke pathway throughout the trajectory of stroke care, to optimize post-stroke health and wellbeing. PPI is embedded at all stages. Purpose: The iPASTAR research programme was strongly informed by a round-table PPI consultation process with individuals who experienced stroke and who provided broad representation across ages, gender, geographical locations (urban and rural) and the PhD themed areas of acute care, early supported discharge and lifestyle-based interventions after stroke. Four PhD scholars taking part in the CDA-iPASTAR now work collaboratively with four stroke champions, supported by a wider PPI advisory panel. Methods: This study will evaluate the process and impact of embedding PPI during a PhD program. We will conduct a longitudinal mixed-methods evaluation, conducting focus groups at 24, 36, and 48 months to explore the experiences of the key stakeholders involved. The participants will include PhD scholars, PPI partners (PPI Advisory Group and PPI Champions), PhD supervisors and a PPI manager. An independent researcher will conduct the evaluation. We will include focus groups, individual interviews and participant reflections. Qualitative data will be analyzed using thematic and content analysis, quantitative data will be analyzed using descriptive statistics. Discussion: PPI and patient voice initiatives bring together researchers, family, and people with health care issues into meaningful dialogue and allow the development of a patient-voice learning network. Embedding PPI training within a PhD program can build meaningful capacity in PPI partnerships in stroke research.