Controversy on the CONVINCE study findings: the PRO take / Controvérsia sobre os achados do estudo CONVINCE: a perspectiva PRO

ABSTRACT The CONVINCE study, recently published in the New England Journal of Medicine, reveals a groundbreaking 23% reduction in the relative risk of all-cause mortality among end-stage kidney patients undergoing high convective volume hemodiafiltration. This significant finding challenges the conventional use of high-flux hemodialysis and offers hope for improving outcomes in chronic kidney disease patients. While some controversies surround the study's findings, including concerns about generalizability and the causes of death, it is essential to acknowledge the study's design and its main outcomes. The CONVINCE study, part of the HORIZON 2020 project, enrolled 1360 patients and demonstrated the superiority of hemodiafiltration in reducing all-cause mortality overall, as well as in specific patient subgroups (elderly, short vintage, non-diabetic, and those without cardiac issues). Interestingly, it was shown that hemodiafiltration had a protective effect against infection, including COVID-19. Future research will address sustainability, dose scaling effects, identification of subgroups especially likely to benefit and cost-effectiveness. However, for now, the findings strongly support a broader adoption of hemodiafiltration in renal replacement therapy, marking a significant advancement in the field.

RESUMO O estudo CONVINCE, publicado recentemente no New England Journal of Medicine, revela uma redução inovadora de 23% no risco relativo de mortalidade por todas as causas entre pacientes renais em estágio terminal submetidos à hemodiafiltração de alto volume de convecção. Esse achado significativo desafia o uso convencional da hemodiálise de alto fluxo e oferece esperança de melhoria dos desfechos em pacientes com doença renal crônica. Embora algumas controvérsias cerquem os achados do estudo, incluindo preocupações sobre a generalização e as causas de óbito, é essencial reconhecer o desenho do estudo e seus principais desfechos. O estudo CONVINCE, parte do projeto HORIZON 2020, inscreveu 1.360 pacientes e demonstrou a superioridade da hemodiafiltração na redução da mortalidade por todas as causas em geral, bem como em subgrupos específicos de pacientes (idosos, HD de curta duração, não diabéticos e aqueles sem problemas cardíacos). Curiosamente, demonstrou-se que a hemodiafiltração teve um efeito protetor contra infecções, incluindo a COVID-19. Pesquisas futuras abordarão sustentabilidade, efeitos de escalonamento da dose, identificação de subgrupos especialmente propensos a se beneficiar e a relação custo-benefício. No entanto, por ora, os achados apoiam fortemente uma adoção mais ampla da hemodiafiltração na terapia renal substitutiva, marcando um avanço significativo na área.

Outcomes of Distal Third Femur Fractures in Patients 18 Years and Older: A Pilot Study.

INTRODUCTION: The selection of the most optimal fixation method for fractures of the distal femur, whether intramedullary nail (NL), lateral locking plate (PL), or nail/plate (NP) is not always clear. This study retrospectively evaluates surgical patients with distal femur fractures and introduces a pilot study using cluster analysis to identify the most optimal fracture fixation method for a given fracture type. METHODS: This is a retrospective cohort study of patients 18 years and older with an isolated distal femur fracture who presented to our Level-1 trauma center between January 1, 2012, and December 31, 2022, and obtained NL, PL, or NP implants. Patients with polytrauma and those without at least six months of follow-up were excluded. A chart review was used to obtain demographics, fracture classification, fixation method, and postoperative complications. A cluster analysis was performed. The following factors were used to determine a successful outcome: ambulatory status pre-injury and 6-12 months postoperatively, infection, non-union, mortality, and implant failure. RESULTS: A total of 169 patients met inclusion criteria. No statistically significant association between the fracture classification and fixation type with overall outcome was found. However, patients treated with an NP (n = 14) had a success rate of 92.9% vs only a 68.1% success rate in those treated with a PL (n = 116) (p = 0.106). The most notable findings in the cluster analysis (15 total clusters) included transverse extraarticular fractures demonstrating 100% success if treated with NP (n = 6), 50% success with NL (n=2), and 78.57% success with PL fixation (n=14). NP constructs in complete articular fractures demonstrated success in 100% of patients (n = 5), whereas 77.78% of patients treated with NL (n = 9) and 61.36% of those treated with PL (n = 44). CONCLUSIONS: Plate fixation was the predominant fixation method used for distal third femur fractures regardless of fracture classification. However, NP constructs trended towards improved success rates, especially in complete intraarticular and transverse extraarticular fractures, suggesting the potential benefit of additional fixation with these fractures. Cluster analysis provided a heuristic way of creating patient profiles in patients with distal third femur fractures. However, a larger cohort study is needed to corroborate these findings to ultimately develop a clinical decision-making tool that also accounts for patient specific characteristics.

Characteristics and Hospital Outcomes of 1403 Patients Hospitalized at Community Hospitals With Ankylosing Spondylitis.

Background: In this study, we aimed to assess the hospital course, outcomes after hospitalization, and predictors of outcomes in patients with ankylosing spondylitis (AS). Methods: We included 1403 patients with AS between 2016 and 2021 who were identified using International Classification of Disease (ICD) codes from a large for-profit healthcare system database. Demographics and clinical characteristics were compared between those who had a favorable outcome, defined as being discharged to home without readmission within 3 months of discharge, versus those who had an unfavorable outcome. A stepwise logistic regression was used to identify demographic and clinical characteristics associated with home discharge and readmission. Results: The mean age for all AS patients was 56.06 ± 17.01 years, which was younger in the favorable outcome group, and 82.47% of patients were discharged to home after the average length of stay of 3.72 ± 4.09 days, also shorter in the favorable outcome group. Of 1403 patients, 37.56% were readmitted within 3 months of discharge, at a lower rate in the group with home discharge. Opioids were the most commonly used medication during hospitalization (67.07%), prescribed at a lower rate in the favorable outcome group. Medical coverage by Medicare and Medicaid, fall at admission, hospital-acquired anemia, steroid, acetaminophen, muscle relaxant use, and an increased dose of morphine milligram equivalent at discharge were significantly associated with decreased odds of home discharge. Surgical procedures during admission, gastrointestinal complications, discharge to inpatient rehabilitation units, and use of benzodiazepine were associated with an increased risk of readmission within 3 months. Conclusion: Recognizing factors that put patients with AS at risk of unfavorable outcomes is useful information to improve patient care during hospitalization.

Impact of pulmonary hypertension on outcomes after TEER in patients suffering from mitral regurgitation.

AIM: Data on associations of invasively determined hemodynamic parameters with procedural success and outcomes in patients suffering from mitral regurgitation (MR) undergoing transcatheter edge-to-edge repair of the mitral valve (M-TEER) is limited. METHODS AND RESULTS: We enrolled 239 patients with symptomatic MR of grade 2 + , who received M-TEER. All patients underwent extensive pre-interventional invasive hemodynamic measurements via right heart catheterization (mean pulmonary arterial pressure (mPAP), systolic- (PAPsys) and diastolic pulmonary arterial pressure (PAPdia), pulmonary arterial wedge pressure (PAWP), a-wave, v-wave, pulmonary vascular resistance (PVR), transpulmonary pressure gradient (TPG), cardiac index (CI), stroke volume index (SVI)). mPAP and PAWP at baseline were neither associated with procedural success, immediate reduction of MR, nor residual MR after 6 months of follow-up. The composite outcome (All-cause mortality (ACM) and/or heart failure induced rehospitalization (HFH)) and HFH differed significantly after M-TEER when stratified according to mPAP, PAWP, PAPdia, a-wave and v-wave. ACM was not associated with the afore mentioned parameters. Neither PVR, TPG, CI nor SVI were associated with the composite outcome and HFH, respectively. In multivariable analyses, PAWP was independently associated with the composite outcome and HFH. PVR and SVI were not associated with outcomes. CONCLUSION: PAWP at baseline was significantly and independently associated with HFH and might serve as a valuable parameter for identifying patients at high risk for HFH after M-TEER. ACM and procedural success were not affected by pulmonary arterial pressure before M-TEER. We suggest that the post-capillary component of PH serves as the driving force behind the risk of HFH.

Feasibility of using an automated call service to collect quality of life and functional outcome data in trauma patients.

Objectives: Following up trauma patients after discharge, to evaluate their subsequent quality of life and functional outcomes, is notoriously difficult, time consuming, and expensive. Automated systems are a conceptually attractive solution. We prospectively assessed the feasibility of using a series of automated phone calls administered by Emmi Patient Engagement to survey trauma patients after discharge. Methods: Recruitment into the study was incorporated into the patient discharge process by nursing staff. For this pilot, we included trauma patients discharging home and who were able to answer phone calls. A script was created to evaluate the Extended Glasgow Outcome Scale and the EuroQol EQ-5D to assess functional status and quality of life, respectively. Call attempts were made at 6 weeks, 3 months, 6 months, and 1 year after discharge. Results: A total of 110 patients initially agreed to participate. 368 attempted patient encounters (calls or attempted calls) took place, with 104 (28.3%) patients answering a least one question in the study. 21 unique patients (19.1% of those enrolled) completed 27 surveys. Conclusions: Automated, scripted phone calls to survey patients after discharge are not a feasible way of collecting functional and quality of life data. Level of evidence: Level II/prospective.

Associated Factors of Cardiopulmonary Resuscitation Outcomes; a Cohort Study on an Adult In-hospital Cardiac Arrest Registry.

Introduction: In-hospital cardiac arrest (IHCA) remains a substantial cause of morbidity and mortality for hospitalized patients worldwide. This study aimed to identify associated factors of return of spontaneous circulation (ROSC) and survival with favorable neurological outcomes of IHCA patients. Method: A two-year retrospective cohort study was conducted at a university-based tertiary care hospital in Bangkok, Thailand, studying adult patients aged ≥ 18 years with IHCA from January 2021 to December 2022. The primary endpoint was sustained ROSC, and the secondary endpoint was survival with favorable neurological outcomes defined as Cerebral Performance Categories (CPC) Scale of 1 or 2 at discharge. Pre-arrest and intra-arrest variables were collected and analyzed using multivariable logistic regression to identify independent factors associated with the outcomes. Results: During the study period, 156 patients were included in the study. 105 (67.3%) patients achieved sustained ROSC after the CPR, 28 patients (18.0%) were discharged alive, and 15 patients (9.6%) survived with a favorable neurological outcome at hospital discharge. Overall, sustained ROSC was higher in patients who had IHCA during the day shift (odds ratio (OR): 4.11; 95% confidence interval (CI): 1.05-16.06) and electrocardiogram (ECG) monitoring prior to arrest (OR: 6.38; 95% CI: 1.18-34.54). In contrast, higher adrenaline doses administrated, and increased CPR duration reduced the odds of sustained ROSC (OR: 0.72; 95% CI: 0.54-0.94 and OR: 0.92; 95% CI: 0.85-0.98, respectively). Arrest due to cardiac etiology was associated with increased discharged survival with favorable neurological outcomes (OR: 13.43; 95% CI: 2.00-89.80), while a higher Good Outcome Following Attempted Resuscitation (GO-FAR) score reduced the odds of the secondary outcome (OR: 0.89; 95% CI: 0.81-0.98). Conclusion: The sustained ROSC was higher in IHCA during the daytime shift and under prior ECG monitoring. The administration of higher doses of adrenaline and prolonged CPR durations decreased the likelihood of achieving sustained ROSC. Furthermore, patients with cardiac-related causes of cardiac arrest exhibited a higher rate of survival to hospital discharge with favorable neurological outcomes.

Patient adherence and response time in electronic patient-reported outcomes: insights from three longitudinal clinical trials.

PURPOSE: Patient-reported outcome measures (PROMs) are used to collect data on disease symptoms in support of clinical trial endpoints. Clinical studies can last a year or more, and the patients' adherence and response time to daily at-home questionnaires may vary significantly over time. The aim of this study was to understand patterns and changes in patients' completion of daily PROMs during longitudinal clinical studies. METHODS: Data were collected from 1342 patients randomized into three respiratory clinical trials (NCT03401229, NCT03347279, and NCT03406078). PROMs were completed by patients using electronic handheld devices that collected the starting and completion times. A Bayesian generalized linear mixed-effects model was used to identify unbiased coefficients associated with PROM adherence and response time using patient, site, and calendar features as covariates. RESULTS: Adherence decreased over time after randomization, and the rate of decrease was higher in younger patients. The 14-day pre-randomization adherence was correlated with adherence throughout the study. Patients were also more adherent during working days compared to non-working days. Oldest patients took twice as long to complete PROMs throughout the study; however, the response time for all patients decreased during the first month of the study regardless of age. Response time increased 7 days before and after the date of a scheduled clinic visit and when a patient-reported higher symptom burden. CONCLUSION: Detailed analyses of adherence and response time for daily PROMs in clinical trials can provide significant insights about trends of patient behavior in longitudinal clinical studies with high baseline adherence.

Evaluating the real-world effectiveness of belimumab in patients with SLE using SLE-related laboratory values and rheumatoid arthritis-derived disease activity measures: RAPID3, swollen joint count and tender joint count.

OBJECTIVE: To investigate the real-world impact of intravenous belimumab treatment among patients with SLE using rheumatoid arthritis-derived disease activity measures and SLE-related laboratory values. METHODS: This retrospective cohort study used US electronic medical record data from the United Rheumatology Normalised Integrated Community Evidence (UR-NICE) database. Adult patients with SLE who initiated intravenous belimumab between 1 January 2012 and 3 December 2019 (index), had 12 months of pre-index and 24 months of post-index clinical activity, and had ≥6 infusions of belimumab during the 24 months post-index were included. The primary outcome measure was time to first improvement of minimally important difference (MID) for Routine Assessment of Patient Index Data 3 (RAPID3), Patient Pain Index (PPI), swollen joint count, tender joint count (TJC), complement C3 and C4 and anti-double-stranded DNA antibodies during the on-treatment follow-up period of up to 24 months. The secondary outcome measure evaluated the trajectories of these outcome measures for up to 24 months of belimumab treatment. RESULTS: Of 495 patients included, between 21.0% and 52.1% had ≥1 record for each of the disease activity measures or laboratory values in the pre-index and post-index periods and were included in analyses for that measure. The proportion of patients achieving MID for each measure increased rapidly within 3 months, with continued gradual improvement throughout the remaining on-treatment period, up to 24 months. After 6 months, 52.3% and 55.3% of patients had achieved MID in RAPID3 and PPI, respectively. Outcome measure trajectories indicated improved disease activity with belimumab treatment, particularly in RAPID3, TJC and laboratory values. CONCLUSIONS: In this real-world effectiveness study, belimumab therapy for SLE resulted in clinically meaningful improvements in rheumatoid arthritis-derived disease activity measures within 3 months of treatment, with patients who remained on belimumab therapy experiencing improvement even up to 24 months of observation.

Attraction and achievement as two attributes of gamification in healthcare: an evolutionary concept analysis.

PURPOSE: This study conducted a conceptual analysis of gamification in healthcare utilizing Rogers' evolutionary concept analysis methodology to identify its attributes and provide a method for its applications in the healthcare field. Gamification has recently been used as a health intervention and education method, but the concept is used inconsistently and confusingly. METHODS: A literature review was conducted to derive definitions, surrogate terms, antecedents, influencing factors, attributes (characteristics with dimensions and features), surrogate concepts, consequences, implications, and hypotheses from various academic fields. A total of 56 journal articles in English and Korean, published between August 2 and August 7, 2023, were extracted from databases such as PubMed Central, the Institute of Electrical and Electronics Engineers, the Association for Computing Machinery Digital Library, the Research Information Sharing Service , and the Korean Studies Information Service System, using the keywords "gamification" and "healthcare." These articles were then analyzed. RESULTS: Gamification in healthcare is defined as the application of game elements in health-related contexts to improve health outcomes. The attributes of this concept were categorized into two main areas: attraction and achievement. These categories encompass various strategies for synchronization, enjoyable engagement, visual rewards, and goal-reinforcing frames. CONCLUSION: Through a multidisciplinary analysis of the concept's attributes and influencing factors, this paper provides practical strategies for implementing gamification in health interventions. When developing a gamification strategy, healthcare providers can reference this analysis to ensure the game elements are used both appropriately and effectively.

Qualitative interviews of patients with COPD and muscle weakness enrolled in a clinical trial evaluating a new anabolic treatment: patient perspectives of disease experience, trial participation and outcome assessments.

BACKGROUND: Chronic obstructive pulmonary disease (COPD) and muscle weakness can cause impaired physical function, significantly impacting patients' health-related quality of life (HRQoL). Loss of muscle strength is usually assessed through clinical and performance outcome (PerfO) assessments, which consists of tasks performed in a standardized manner, providing evidence of a patient's functional ability. However, evidence documenting the patient experience of COPD and muscle weakness is limited. METHODS: This two-stage qualitative study used semi-structured interviews in patients aged 45-80 years with COPD (post-bronchodilator forced expiratory volume in 1s [FEV1]/forced vital capacity ratio < 0.70, and FEV1% predicted of 30-80%) and muscle weakness. In Stage 1, 30-minute concept elicitation interviews were conducted with participants recruited across three US sites to explore impacts on physical functioning and activities of daily living. In Stage 2, interviews were performed with participants exiting a Phase IIa trial investigating the efficacy of a selective androgen receptor modulator (GSK2881078) on leg strength, whereby PerfOs were used to evaluate strength and physical functioning endpoints. These participants completed either 60-minute in-depth (n = 32) or 15-minute confirmatory (n = 35) interviews exploring trial experience, completion of outcome measures, disease experience and treatment satisfaction. RESULTS: In Stage 1 (n = 20), most participants described their muscles as weak (83.3%). Difficulties with walking (100%) and lifting heavy objects (90%) were reported. In Stage 2, 60-minute interviews, all participants (n = 32) reported a positive trial experience. Most participants reported that the home exercise program was easy to fit into daily life (77.8%), the PROactive daily diary was easy to complete (100%) and wearable sensors were easy to use (65.6%). However, technical issues were reported (71%), and few participants (19.4%) found physical assessments easy to complete. Improvements in muscle strength and functional limitations were reported by most participants. The shorter 15-minute confirmatory interviews (n = 35) supported the in-depth interview results. CONCLUSION: The qualitative interviews generated in-depth evidence of key concepts relevant to patients with COPD and muscle weakness and support the assessments of patient strength and physical function as outcome measures in this population in future studies. TRIAL NUMBER: GSK Stage 1: 206869; Stage 2: 200182, NCT03359473; Registered December 2, 2017, https://clinicaltrials.gov/ct2/show/NCT03359473 .

New consensus definition on defining and measuring care for children with paediatric feeding disorder.

AIM: This study addresses the absence of a definition of care for children with feeding disorders, limited agreement on key performance indicators (KPIs), and the lack of data linked to those KPIs. METHODS: Clinicians, consumers and researchers involved in outpatient feeding care in New South Wales (NSW), Australia were invited to participate in a two-Phase study. In Phase 1, a modified Delphi method was used. Two rounds of voting resulted in a new consensus definition of a multidisciplinary paediatric feeding clinic. Three further rounds voting determined relevant KPIs. In Phase 2, the KPIs were piloted prospectively in 10 clinics. RESULTS: Twenty-six clinicians, consumers and researchers participated in Phase 1. Participation across five voting rounds declined from 92% to 60% and a valid definition and KPI set were created. In Phase 2, the definition and KPIs were piloted in 10 clinics over 6 weeks. Data for 110 patients were collected. The final KPI set of 28 measures proposed covers clinical features, patient demographics and medical issues, parent-child interaction and outcome measures. CONCLUSIONS: A new definition of a multidisciplinary paediatric feeding clinic is now available, linked to a standardised KPI set covering relevant performance measures. These proved viable in baseline data collection for 10 clinics across NSW. This sets a foundation for further data collection, systematic measurement of care provision and outcomes, and research needed to deliver care improvement for children with paediatric feeding disorder.

Primary liver transplantation vs transplant after Kasai portoenterostomy in children with biliary atresia: A retrospective Brazilian single-center cohort.

BACKGROUND: Biliary atresia (BA) is the most common indication for pediatric liver trans plantation, although portoenterostomy is usually performed first. However, due to the high failure rate of portoenterostomy, liver transplantation has been advocated as the primary procedure for patients with BA. It is still unclear if a previous portoenterostomy has a negative impact on liver transplantation outcomes. AIM: To investigate the effect of prior portoenterostomy in infants un dergoing liver transplantation for BA. METHODS: This was a retrospective cohort study of 42 pediatric patients with BA who underwent primary liver transplantation from 2013 to 2023 at a single tertiary center in Brazil. Patients with BA were divided into two groups: Those under going primary liver transplantation without portoenterostomy and those undergoing liver transplantation with prior portoenterostomy. Continuous variables were compared using the Student's t-test or the Kruskal-Wallis test, and categorical variables were compared using the χ2 or Fisher's exact test, as appropriate. Multivariable Cox regression analysis was performed to determine risk factors for portal vein thrombosis. Patient and graft survival analyses were conducted with the Kaplan-Meier product-limit estimator, and patient subgroups were compared using the two-sided log-rank test. RESULTS: Forty-two patients were included in the study (25 [60%] girls), 23 undergoing liver transplantation without prior portoenterostomy, and 19 undergoing liver transplantation with prior portoenterostomy. Patients with prior portoenterostomy were older (12 vs 8 months; P = 0.02) at the time of liver transplantation and had lower Pediatric End-Stage Liver Disease scores (13.2 vs 21.4; P = 0.01). The majority of the patients (35/42, 83%) underwent living-donor liver transplantation. The group of patients without prior portoenterostomy appeared to have a higher incidence of portal vein thrombosis (39 vs 11%), but this result did not reach statistical significance. Prior portoenterostomy was not a protective factor against portal vein thrombosis in the multivariable analysis after adjusting for age at liver transplantation, graft-to-recipient weight ratio, and use of vascular grafts. Finally, the groups did not significantly differ in terms of post-transplant survival. CONCLUSION: In our study, prior portoenterostomy did not significantly affect the outcomes of liver transplantation.

The effectiveness and safety of centralized early rehabilitation care for critically ill children with severe acquired brain injury: A retrospective cohort and implementation study.

BACKGROUND: Most children with neurocritical illness are at risk of physical, neurocognitive, and psychosocial sequelae and need centralized early rehabilitation care. OBJECTIVE: To identify the effectiveness and safety of centralized early rehabilitation care for children with severe acquired brain injury. METHODS: This is a mixed methods study-an implementation study and single-center retrospective cohort study with historical control. All children with severe acquired brain injury hospitalized in a specialized rehabilitation center in a comprehensive tertiary pediatric hospital between September 2016 and August 2020 were included. Patients treated in the centralized early rehabilitation unit were compared to historical controls dispersed in the normal inpatient rehabilitation ward. The effectiveness outcomes were measured by the Pediatric Cerebral Performance Category (PCPC) scale and the incidence of newly onset comorbidities. The safety outcomes were indicated by the mortality rate and the incidence of unexpected referrals. RESULTS: One hundred seventy-five patients were included. The delta PCPC scores of the first 4 weeks of inpatient rehabilitation in the intervention group were significantly lower than the control group (Z = -2.395, p = 0.017). The PCPC scores at 1 year in the intervention group were significantly reduced as compared to the control group (Z = -3.337, p = 0.001). The incidence of newly onset pneumonia/bronchitis was also decreased in the intervention group (χ2 = 4.517, p = 0.034). No death of patients was recorded, and there was no significant difference in unexpected referral rate between the two groups (χ2 = 0.374, p = 0.541). CONCLUSIONS: The centralized pediatrics early rehabilitation unit is effective and safe for children with severe acquired brain injury. Further multicenter prospective implementation studies on effectiveness, safety, and economic evaluation are needed.

Medical Students' Perspectives Regarding the Use of a Slit-Lamp Smartphone Adapter for Clinical Slit-Lamp Photography.

Background This study aimed to investigate medical students' perspectives regarding the ease and utility of smartphone slit-lamp photography. Methodology In this prospective experimental study, fourth and fifth-year medical students who were in or had finished ophthalmology rotation were included to attempt slit-lamp smartphone anterior segment photography on adult patients after a brief hands-on instruction course. Each medical student attempted to record five supervised slit-lamp videos of the anterior segment of five patients using the described adapter and their own smartphone. The time taken until photography was calculated for each attempt. After the fifth attempt, each medical student rated the ease of the use of this method of slit-lamp photography as well as their perspective regarding its utility as a potential means of medical education and telemedical consultations on a five-point Likert scale. Results A total of 33 medical students participated, with each successfully recording five slit-lamp examinations using their smartphones. The time used for the application of the adapter until the image capture ranged from 6 to 278 seconds (average = 39.51 ± 34.7 seconds) and markedly improved by the fifth attempt (30.5 ± 25.7 seconds) compared to the first attempt (67.3 ± 49.3 seconds). Learning this skill was perceived to be relatively easy (2.2 ± 1), with high potential in clinical education (4.6 ± 0.75) and teleconsultations (4.7 ± 0.65). Conclusions Smartphone slit-lamp photography is a relatively easy process. It can be quickly acquired by medical students and has the potential to enhance their medical education and telemedical consultation capabilities.

Mortality rates in Norwegian HEMS-a retrospective analysis from Central Norway.

BACKGROUND: Helicopter Emergency Medical Services (HEMS) provide rapid and specialized care to critically ill or injured patients. Norwegian HEMS in Central Norway serves an important role in pre-hospital emergency medical care. To grade the severity of patients, HEMS uses the National Advisory Committee for Aeronautics' (NACA) severity score. The objective of this study was to describe the short- and long term mortality overall and in each NACA-group for patients transported by HEMS Trondheim using linkage of HEMS and hospital data. METHODS: The study used a retrospective cohort design, aligning with the STROBE recommendations. Patient data from Trondheim HEMS between 01.01.2017 and 31.12.2019 was linked to mortality data from a hospital database and analyzed. Kaplan Meier plots and cumulative mortality rates were calculated for each NACA group at day one, day 30, and one year and three years after the incident. RESULTS: Trondheim HEMS responded to 2224 alarms in the included time period, with 1431 patients meeting inclusion criteria for the study. Overall mortality rates at respective time points were 10.1% at day one, 13.4% at 30 days, 18.5% at one year, and 22.3% at three years. The one-year cumulative mortality rates for each NACA group were as follows: 0% for NACA 1 and 2, 2.9% for NACA 3, 10.1% for NACA 4, 24.7% for NACA 5 and 49.5% for NACA 6. Statistical analysis with a global log-rank test indicated a significant difference in survival outcomes among the groups (p < 2⋅10- 16). CONCLUSION: Among patients transported by Trondheim HEMS, we observed an incremental rise in mortality rates with increasing NACA scores. The study further suggests that a one-year follow-up may be sufficient for future investigations into HEMS outcomes.

Disease flares with baricitinib dose reductions and development of flare criteria in patients with CANDLE/PRAAS.

OBJECTIVES: Patients with chronic atypical neutrophilic dermatosis with lipodystrophy and elevated temperature/proteasome-associated autoinflammatory syndrome (CANDLE/PRAAS) respond to the janus kinase inhibitor 1/2 inhibition with baricitinib at exposures higher than in rheumatoid arthritis. Baricitinib dose reductions to minimise exposure triggered disease flares which we used to develop 'flare criteria'. METHODS: Of 10 patients with CANDLE/PRAAS treated with baricitinib in an open-label expanded-access programme, baricitinib doses were reduced 14 times in 9 patients between April 2014 and December 2019. Retrospective data analysis of daily diary scores and laboratory markers collected before and after the dose reductions were used to develop 'clinical' and 'subclinical' flare criteria. Disease flare rates were compared among patients with <25% and >25% dose reductions and during study visits when patients received recommended 'optimized' baricitinib doses (high-dose visits) versus lower than recommended baricitinib doses (low-dose visits) using two-sided χ2 tests. RESULTS: In the 9/10 patients with CANDLE with dose reduction, 7/14 (50%) times the dose was reduced resulted in a disease flare. All four dose reductions of >25% triggered a disease flare (p <0.05). Assessment of clinical and laboratory changes during disease flares allowed the development of disease flare criteria that were assessed during visits when patients received high or low doses of baricitinib. Disease flare criteria were reached during 43.14% of low-dose visits compared with 12.75% of high-dose visits (p <0.0001). Addition of an interferon score as an additional flare criterion increased the sensitivity to detect disease flares. CONCLUSION: We observed disease flares and rebound inflammation with baricitinib dose reductions and proposed flare criteria that can assist in monitoring disease activity and in designing clinical studies in CANDLE/PRAAS.

Impact of a pandemic shock on unmet medical needs of middle-aged and older adults in 10 countries.

OBJECTIVE: The objective is to explore the impact of the pandemic shock on the unmet medical needs of middle-aged and older adults worldwide. METHODS: The COVID-19 pandemic starting in 2020 was used as a quasiexperiment. Exposure to the pandemic was defined based on an individual's context within the global pandemic. Data were obtained from the Integrated Values Surveys. A total of 11 932 middle-aged and older adults aged 45 years and above from 10 countries where the surveys conducted two times during 2011 and 2022 were analysed. We used logistic regression models with the difference-in-difference method to estimate the impact of pandemic exposure on unmet medical needs by comparing differences before and after the pandemic across areas with varying degrees of severity. RESULTS: Among the 11 932 middle-aged and older adults, 3647 reported unmet medical needs, with a pooled unmet rate of 30.56% (95% CI: 29.74% to 31.40%). The pandemic significantly increased the risk of unmet medical needs among middle-aged and older adults (OR: 2.33, 95% CI: 1.94 to 2.79). The deleterious effect of the pandemic on unmet medical needs was prevalent among middle-aged adults (2.53, 2.00 to 3.20) and older adults (2.00, 1.48 to 2.69), as well as among men (2.24, 1.74 to 2.90) and women (2.34, 1.82 to 3.03). The results remained robust in a series of sensitivity analyses. CONCLUSION: These findings suggest that efforts should be made by policymakers and healthcare professionals to balance healthcare resources to adequately address the comprehensive healthcare demands of individuals regarding multiple health issues, taking into account the challenges posed by pandemics.

Psychometric evaluation of the near activity visual questionnaire presbyopia (NAVQ-P) and additional patient-reported outcome items.

BACKGROUND: The Near Visual Acuity Questionnaire Presbyopia (NAVQ-P) is a patient-reported outcome (PRO) measure that was developed in a phakic presbyopia population to assess near vision function impacts. The study refined and explored the psychometric properties and score interpretability of the NAVQ-P and additional PRO items assessing near vision correction independence (NVCI), near vision satisfaction (NVS), and near vision correction preference (NVCP). METHODS: This was a psychometric validation study conducted using PRO data collected as part of a Phase IIb clinical trial (CUN8R44 A2202) consisting of 235 randomized adults with presbyopia from the US, Japan, Australia, and Canada. Data collected at baseline, week 2, and months 1, 2, and 3 during the 3-month trial treatment period were included in the analyses to assess item (question) properties, NAVQ-P dimensionality and scoring, reliability, validity, and score interpretation. RESULTS: Item responses were distributed across the full response scale for most NAVQ-P and additional PRO items. Confirmatory factor analysis supported the pre-defined unidimensional structure and calculation of a NAVQ-P total score as a measure of near vision function. Item deletion informed by item response distributions, dimensionality analyses, item response theory, and previous qualitative findings, including clinical input, supported retention of 14 NAVQ-P items. The 14-item NAVQ-P total score had excellent internal consistency (α = 0.979) and high test-retest reliability (Intraclass Correlation Coefficients > = 0.898). There was good evidence of construct-related validity for all PROs supported by strong correlations with concurrent measures. Excellent results for known-groups validity and ability to detect change analyses were also demonstrated. Anchor-based and distribution-based methods supported interpretation of scores through generation of group-level and within-individual estimates of meaningful change thresholds. A meaningful within-patient change in the range of 8-15-point improvement on the NAVQ-P total score (score range 0-42) was recommended, including a more specific responder definition of 10-point improvement. CONCLUSIONS: The NAVQ-P, NVCI, and NVS are valid and reliable instruments which have the ability to detect change over time. Findings strongly support the use of these measures as outcome assessments in clinical/research studies and in clinical practice in the presbyopia population.