Economics of hematopoietic stem cell transplant in immune-mediated neurologic autoimmune diseases.

Autologous hematopoietic stem cell transplantation (AHSCT) is a therapeutic procedure for autoimmune diseases which suppresses inflammation and resets the immune system, thereby halting disease activity and disability progression in treatment-resistant patients. This chapter reviews existing guidelines and health economic evaluations of AHSCT for multiple sclerosis (MS) and presents a cost-utility analysis from the UK NHS and personal social services perspective comparing AHSCT with disease-modifying therapies (DMTs) in patients with highly active relapsing-remitting MS (RRMS) based on the only published randomized controlled trial, "MIST," in this population. Over a 5-year time horizon, AHSCT was dominant (more effective and less costly) over the DMTs in MIST. At a threshold of £20,000 per QALY, there was a 100% probability that AHSCT was cost-effective. This result is explained by the high ongoing costs of DMTs compared with the up-front cost of AHSCT, combined with the high effectiveness of AHSCT. When compared with natalizumab, the result did not change; AHSCT remained dominant. These results support current guideline recommendations regarding AHSCT for highly active RRMS. The cost-effectiveness of AHSCT in progressive and aggressive MS and other immune-mediated neurologic diseases remains uncertain due to a lack of health economic analyses, reflecting the limited clinical evidence base.

Cost-effectiveness of neuropsychological rehabilitation for acquired brain injuries: Update of Stolwyk et al.'s (2019) review.

Acquired brain injuries (ABI), resulting from stroke or traumatic brain injury, cause a range of neuropsychological impairments and many patients continue to experience neuropsychological deficits years after onset. The increasing average age of the population highlights the importance of effective management strategies for the consequences of ABI. Despite the well-documented impact of rehabilitation interventions, the cost-effectiveness of neuropsychological rehabilitation remains largely unknown. This study conducted a scoping review to update the findings of Stolwyk et al. (Neuropsychological Rehabilitation, 2021, 31, 316), focusing on the economic evaluations of neuropsychological rehabilitation for individuals with ABI. Following the PIO framework, PRISMA ScR guidelines, and systematic review reporting checklist, the review screened 1027 articles and included eight studies published between 2019 and 2024. The studies encompassed either language rehabilitation or general neuropsychological programs, including neuropsychological interventions. The economic analyses, including two cost-effectiveness, five cost-utility, and one cost-benefit study, mostly adhered to CHEERS guidelines, enhancing the transparency and methodological rigour of their reporting. These studies demonstrated varying degrees of cost-effectiveness for interventions targeting post-stroke language disorders and neuropsychological rehabilitation for ABI, with significant cost savings and health benefits observed, particularly for home-based rehabilitation interventions. The included studies suffered from a short time horizon, limiting the ability to capture the long-term economic impacts and effectiveness of the interventions. Future research should focus on longer-term follow-up data and include broader search strategies to enhance understanding and optimise health care interventions. A comprehensive implementation of these economic analyses is crucial for informing policymakers, enabling them to introduce rehabilitative interventions based on solid evidence.

Analyzing quality of life among people with opioid use disorder from the National Institute on Drug Abuse Data Share initiative: implications for decision making.

PURPOSE: We aimed to estimate health state utility values (HSUVs) for the key health states found in opioid use disorder (OUD) cost-effectiveness models in the published literature. METHODS: Data obtained from six trials representing 1,777 individuals with OUD. We implemented mapping algorithms to harmonize data from different measures of quality of life (the SF-12 Versions 1 and 2 and the EQ-5D-3 L). We performed a regression analysis to quantify the relationship between HSUVs and the following variables: days of extra-medical opioid use in the past 30 days, injecting behaviors, treatment with medications for OUD, HIV status, and age. A secondary analysis explored the impact of opioid withdrawal symptoms. RESULTS: There were statistically significant reductions in HSUVs associated with extra-medical opioid use (-0.002 (95% CI [-0.003,-0.0001]) to -0.003 (95% CI [-0.005,-0.002]) per additional day of heroin or other opiate use, respectively), drug injecting compared to not injecting (-0.043 (95% CI [-0.079,-0.006])), HIV-positive diagnosis compared to no diagnosis (-0.074 (95% CI [-0.143,-0.005])), and age (-0.001 per year (95% CI [-0.003,-0.0002])). Parameters associated with medications for OUD treatment were not statistically significant after controlling for extra-medical opioid use (0.0131 (95% CI [-0.0479,0.0769])), in line with prior studies. The secondary analysis revealed that withdrawal symptoms are a fundamental driver of HSUVs, with predictions of 0.817 (95% CI [0.768, 0.858]), 0.705 (95% CI [0.607, 0.786]), and 0.367 (95% CI [0.180, 0.575]) for moderate, severe, and worst level of symptoms, respectively. CONCLUSION: We observed HSUVs for OUD that were higher than those from previous studies that had been conducted without input from people living with the condition.

Thus far, health-related quality of life estimates for patients with opioid use disorder in the United States are limited, and importantly, they were not generated from studies among people living with the condition. This study extracted data from six clinical trials providing data among 1,777 people with opioid use disorder, made publicly available by the National Institutes of Health, to produce estimates of health-related quality of life. Our study found higher health-related quality of life estimates as compared to previous studies, modest impact of medications for opioid use disorder and strong impact of withdrawal symptoms on this outcome. These higher values among people with opioid use disorder might reflect the very negative perception of this condition among members of the general population (among whom these estimates have been generated previously). However, these relatively high estimates could also reflect an adaptation to the condition or a lack of awareness of associated-health damage in the context of dependence. The low number of observations providing data on medications for opioid use disorder led to high uncertainty around related estimates of health-related quality of life, but our findings could also reflect real experiences by patients in the absence of the positive effects of non-medication opioids, which deserve more attention in clinical practice. Our study suggests that systematically measuring withdrawal symptoms and representing these in health economic models might provide a more accurate representation of health-related quality of life among people with opioid use disorder and therefore of the impact and cost-effectiveness of interventions.

Surgery for Esotropia in a Case of Nanophthalmos.

Nanophthalmos, characterized by an abnormally small ocular globe, presents significant challenges in the management of strabismus due to its unique anatomical constraints. This detailed case report highlights the intricacies and outcomes of strabismus surgery in a patient with nanophthalmos, providing valuable insights into the surgical considerations and adaptations required for this rare condition. The subject of this case, a young girl diagnosed with esotropia and high hyperopia, underwent unilateral medial rectus muscle recession in an attempt to correct her esotropia. Despite the careful surgical approach and postoperative management, a two-year follow-up revealed a limited response to the intervention, with improvements in visual acuity but continued presence of esotropia and lack of stereopsis development. This case sheds light on several key considerations in the surgical treatment of strabismus in nanophthalmos patients, including the potential for reduced surgical effectiveness due to the small globe size, the importance of accurate preoperative assessment, and the challenges in predicting surgical outcomes. Additionally, it discusses the implications of these findings for future surgical planning, the potential need for revision surgeries, and the broader research context, emphasizing the necessity for a deeper understanding of the biomechanical and anatomical particularities of nanophthalmos in the context of strabismus surgery. The report concludes with recommendations for improving surgical strategies and patient outcomes, advocating for more comprehensive studies and a tailored approach to treating strabismus in individuals with nanophthalmos.

Use of electroconvulsive therapy during postpartum: A retrospective chart review.

Background: There is limited data on use of electroconvulsive therapy (ECT) for management of psychiatric disorders during the postpartum period from India. Aim: We aimed to assess the demographic and clinical profile of patients receiving ECT during the postpartum period for various psychiatric disorders using a retrospective study design. Methods: ECT register of the department was reviewed for the period of January 2019 to December 2023 to identify the patients who received ECT during the postpartum period. The treatment records of these patients were evaluated to extract the demographic and clinical profile. Results: During the study period, 10 patients received ECT during the postpartum period. The mean age of the study sample was 27 (standard deviation [SD]: 2.9) years. Majority of the patients were inpatients (70%) at the time of receiving ECT. Five patients were diagnosed with first-episode depression with postpartum onset, and two patients had postpartum-onset psychotic disorder. One patient was diagnosed with recurrent depressive episode and one with bipolar disorder, current episode mania with psychotic symptom, at the time of receiving ECT. The mean number of ECTs during the ECT course was 6.7 (SD: 3.09). Nine out of the 10 patients showed good response to ECT. Conclusion: Although ECT is less frequently used for management of postpartum psychiatric disorders, its use is associated with significant clinical improvement.

Cost-effectiveness analysis of pharmaceutical care in adult critically ill patients: based on a prospective cohort study.

Background: The medication regimen for critically ill patients is complex and dynamic, leading to a high incidence of drug-related problems. This study aimed to assess the effectiveness and economic efficiency of pharmaceutical care for these patients. Methods: In this prospective cohort study conducted in a tertiary hospital, adult patients were assigned either to a clinical pharmaceutical care group or a control group based on existing clinical grouping rules. Health outcomes and economic indicators were collected, followed by a cost-effectiveness analysis. Results: The acceptance rate for clinical pharmacist interventions was 89.31%. The pharmaceutical care group exhibited significant reductions in the rate of medication errors (40.65% vs. 61.69%, P < 0.001) and adverse drug events (44.52% vs. 56.45%, P = 0.020). The usage rates for special-grade antibiotics (85.16% vs. 91.13%, P = 0.009) and proton pump inhibitors (77.42% vs. 88.71%, P = 0.002) were also lower in the pharmaceutical care group. Secondary outcomes did not show significant differences in total hospital stay (21 days vs. 22 days, P = 0.092). However, ICU stay was significantly shorter (9 days vs. 11 days, P = 0.003) in the pharmaceutical care group. Cost-effectiveness analysis demonstrated that each 1% reduction in adverse drug events associated with ICU pharmaceutical care saved $226.75 in ICU hospitalization costs and $203.42 in total ICU drug costs. A 1% reduction in the medication error rate saved $128.57 in ICU hospitalization costs and $115.34 in total ICU drug costs. Conclusions: Pharmaceutical care significantly reduces adverse drug events and medication errors, promotes rational use of medications, decreases the length of ICU stay, and lowers treatment costs in critically ill patients, establishing a definitive advantage in terms of cost-effectiveness.

Evaluating the Population-Based Usage and Benefit of Digitally Collected Patient-Reported Outcomes and Experiences in Patients With Chronic Diseases: The PROMchronic Study Protocol.

BACKGROUND: Chronic diseases are associated with a high disease burden. Under- and overprovision of care as well as quality variation between health care providers persists, while current quality indicators rarely capture the patients' perspective. Capturing patient-reported outcome measures (PROMs) as well as patient-reported experience measures (PREMs) is becoming more and more important to identify gaps in care provision, prioritize services most valuable to patients, and aid patients' self-management. OBJECTIVE: This study aims to measure the potential benefits and effectiveness of using electronic patient-reported outcome measures (ePROMs) and electronic patient-reported experience measures in a structured and population-based manner to enhance health care for chronic disease patients in Germany. METHODS: This prospective cohort study aims to evaluate the potential benefits of PROM usage in patients with chronic diseases. We evaluate whether (1) digitally collected PROMs and PREMs can be used for health system performance assessment by generating a representative response of chronically diseased individuals with asthma, chronic obstructive pulmonary disease, diabetes, and coronary artery disease across Germany, and (2) based on the PROMs and PREMs, low-value care can be identified. As patient-reported outcomes (PROs) are rarely presented back to patients, (3) this study also examines patients' reactions to their PROM scores in the form of digital PRO feedback. For these purposes, randomly selected patients from a nationwide German insurer are digitally surveyed with generic and disease-specific PROMs and PREMs, as well as additional questions on their health-related behavior, 4 times over 1 year. Individual PRO feedback is presented back to patients longitudinally and compared to a peer group after each survey period. Patient-reported data is linked with health insurance data. Response rates, changes in health and experience outcomes over time, self-reported changes in health behavior, and health care system usage will be analyzed. RESULTS: The PROMchronic study explores the usage of PROMs in patients with chronic diseases. Data collection began in October 2023, after the initial invitation letter. All the 200,000 potential patients have been invited to participate in the study. Data have not yet been analyzed. Publication of the interim results is planned for the autumn of 2024, and the results are planned to be published in 2025. CONCLUSIONS: We aim to fill the research gap on the population-based usage of PROMs and PREMs in patients with chronic diseases and add to the current understanding of PROM data-sharing with patients. The study's results can thereby inform whether a health care system-wide approach to collecting PROMs and PREMs can be used to identify low-value care, assess quality variation within and across chronic conditions, and determine whether PRO feedback is helpful and associated with any changes in patients' health behaviors. TRIAL REGISTRATION: German Clinical Trials Register DRKS00031656; https://drks.de/search/en/trial/DRKS00031656. INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): DERR1-10.2196/56487.

Platelet response to romiplostim amongst patients with newly diagnosed, persistent, and chronic immune thrombocytopenia in routine clinical practice in Denmark, Sweden, and Norway.

Patient characteristics and platelet responses at romiplostim initiation according to the duration of immune thrombocytopenia (ITP) are poorly understood. Amongst romiplostim-exposed adults with ITP lasting ≥6 months during 2009-2018 in Denmark, Sweden, and Norway, we examined characteristics at romiplostim initiation, romiplostim dosage, and durable platelet response (≥75% of measurements ≥50 × 109/L at 14-24 weeks) for subcohorts with newly diagnosed (duration <3 months), persistent (3-12 months), or chronic (>12 months) ITP initiating romiplostim. The 285 romiplostim initiators comprised 81 (28%) with newly diagnosed, 47 (16%) with persistent, and 157 (55%) with chronic ITP. More patients with newly diagnosed ITP than longer ITP duration, had low comorbidity levels, two or more prior ITP therapies, and previous bleeding requiring hospitalisation. The median romiplostim doses were similar across subcohorts. During treatment, median platelet counts were similar across subcohorts (75-76 × 109/L), and the durable platelet response was 64.6%, 52.9%, and 52.7% for newly diagnosed, persistent, and chronic ITP, respectively. After treatment cessation, the median platelet count was 138 × 109/L, 68 × 109/L, and 71 × 109/L, respectively. In conclusion, newly diagnosed patients, compared with romiplostim initiators with longer disease duration, had more severe ITP, higher frequency of durable platelet response, and higher median platelet count after cessation.

Ivory tower in MD/PhD programmes: sticky floor, broken ladder and glass ceiling.

OBJECTIVE: Achieving gender equity in academic medicine is not only a matter of social justice but also necessary in promoting an innovative and productive academic community. The purpose of this study was to assess gender distribution in dual MD/PhD academic programme faculty members across North America. METHODS: Academic metrics were analysed to quantify the relative career success of academic faculty members in MD/PhD programmes. Measured parameters included academic and leadership ranks along with nominal research factors such as peer-reviewed research publications, H-index, citation number and years of active research. RESULTS: Χ² analysis revealed a statistically significant (p<0.0001, χ²=114.5) difference in the gender distribution of faculty and leadership across North American MD/PhD programmes. Men held 74.2% of full professor positions, 64% of associate professor positions, 59.4% of assistant professor positions and 62.8% of lecturer positions. Moreover, men occupied a larger share of faculty leadership roles with a statistically significant disparity across all ranks (p<0.001, χ²=20.4). A higher proportion of men held positions as department chairs (79.6%), vice chairs (69.1%) and programme leads (69.4%). CONCLUSION: Gender disparity was prevalent in the MD/PhD programmes throughout North America with women achieving a lower degree of professional stature than men. Ultimately, steps must be taken to support women faculty to afford them better opportunities for academic and professional advancement.

Effectiveness of de-implementation of low-value healthcare practices: an overview of systematic reviews.

BACKGROUND: Reducing low-value care (LVC) is crucial to improve the quality of patient care while increasing the efficient use of scarce healthcare resources. Recently, strategies to de-implement LVC have been mapped against the Expert Recommendation for Implementing Change (ERIC) compilation of strategies. However, such strategies' effectiveness across different healthcare practices has not been addressed. This overview of systematic reviews aimed to investigate the effectiveness of de-implementation initiatives and specific ERIC strategy clusters. METHODS: We searched MEDLINE (Ovid), Epistemonikos.org and Scopus (Elsevier) from 1 January 2010 to 17 April 2023 and used additional search strategies to identify relevant systematic reviews (SRs). Two reviewers independently screened abstracts and full texts against a priori-defined criteria, assessed the SR quality and extracted pre-specified data. We created harvest plots to display the results. RESULTS: Of 46 included SRs, 27 focused on drug treatments, such as antibiotics or opioids, twelve on laboratory tests or diagnostic imaging and seven on other healthcare practices. In categorising de-implementation strategies, SR authors applied different techniques: creating self-developed strategies (n = 12), focussing on specific de-implementation strategies (n = 14) and using published taxonomies (n = 12). Overall, 15 SRs provided evidence for the effectiveness of de-implementation interventions to reduce antibiotic and opioid utilisation. Reduced utilisation, albeit inconsistently significant, was documented in the use of antipsychotics and benzodiazepines, as well as in laboratory tests and diagnostic imaging. Strategies within the adapt and tailor to context, develop stakeholder interrelationships, and change infrastructure and workflow ERIC clusters led to a consistent reduction in LVC practices. CONCLUSION: De-implementation initiatives were effective in reducing medication usage, and inconsistent significant reductions were observed for LVC laboratory tests and imaging. Notably, de-implementation clusters such as change infrastructure and workflow and develop stakeholder interrelationships emerged as the most encouraging avenues. Additionally, we provided suggestions to enhance SR quality, emphasising adherence to guidelines for synthesising complex interventions, prioritising appropriateness of care outcomes, documenting the development process of de-implementation initiatives and ensuring consistent reporting of applied de-implementation strategies. REGISTRATION: OSF Open Science Framework 5ruzw.

Cost-effectiveness analysis of NVX-CoV2373 COVID-19 vaccination for elderly people in Japan.

Background: NVX-CoV2373 is one of the vaccines marketed for COVID-19 prevention in Japan. Information on its cost-effectiveness is important for making well-informed decisions on the future of Japan's COVID-19 vaccination programme from the public healthcare payer's perspective. The aim of this study was to evaluate the cost-effectiveness of NVX-CoV2373 vaccination in the elderly Japanese population. Methods: Two analysis populations that included elderly Japanese individuals (aged ≥ 65 years) were defined in this study: those who had not received a COVID-19 vaccine or had not completed a primary vaccination series (i.e., first two vaccinations) with an approved COVID-19 vaccine (analysis population 1), and those who had received two primary vaccinations with an approved COVID-19 vaccine (analysis population 2). A literature-informed Markov model for each analysis population was developed to evaluate the cost-effectiveness of vaccination with NVX-CoV2373 against no vaccination with NVX-CoV2373 from the public healthcare payer's perspective as a base-case analysis and from the societal perspective as a scenario analysis. Vaccine efficacy was estimated from a phase 3 study of NVX-CoV2373 (EudraCT number: 2020-004123-16). Cost-effectiveness was assessed using a willingness-to-pay threshold of Japanese yen (JPY) 5 million per quality-adjusted life-year (QALY). Deterministic and probabilistic sensitivity analyses were also performed. Results: For analysis population 1, NVX-CoV2373 primary and booster vaccinations would reduce costs by JPY 37,647 and prolong QALYs by 0.01601. Therefore, NVX-CoV2373 primary and booster vaccinations were considered to be dominant over no vaccination. For analysis population 2, an NVX-CoV2373 booster vaccination would increase costs by JPY 5010 and prolong QALYs by 0.00550, with the incremental cost-effectiveness ratio of JPY 910,566 per QALY gained. Conclusions: Our analyses suggest that a vaccination strategy with NVX-CoV2373 is cost-effective in the elderly population (aged ≥ 65 years) of Japan.

Cost-Effectiveness Analysis of Pharmacogenomics-Guided Versus Standard Dosing of Warfarin in Patients with Mechanical Prosthetic Heart Valve.

Background: Warfarin is the only approved anticoagulant for antithrombotic treatment in patients with mechanical prosthetic heart valves (MPHV). However, dosing warfarin is challenging due to its narrow therapeutic window and highly variable clinical outcomes. Both low and high doses of warfarin can lead to thrombotic and bleeding events, respectively, with these complications being more severe in individuals with sensitive genetic polymorphisms. Incorporating genetic testing could enhance the accuracy of warfarin dosing and minimize its adverse events. Objectives: This study aims to evaluate the utilities and cost-effectiveness of pharmacogenomics-guided versus standard dosing of warfarin in patients with MPHV in Iran. Methods: In this economic evaluation study, a cost-effectiveness analysis was conducted to compare pharmacogenomics-guided versus standard warfarin dosing. Data related to quality of life (QoL) were collected through a cross-sectional study involving 105 randomly selected MPHV patients using the EuroQol-5D (EQ-5D) Questionnaire. Costs were calculated with input from clinical experts and a review of relevant guidelines. Additional clinical data were extracted from published literature. The pharmacoeconomic threshold set for medical interventions within Iran's healthcare system was $1,500. A decision tree model was designed from the perspective of Iran's healthcare system with a one-year study horizon. Sensitivity analyses were also performed to assess the uncertainty of input parameters. Results: The utility scores derived from the questionnaire for standard and pharmacogenomics-guided warfarin treatments were 0.68 and 0.76, respectively. Genotype-guided dosing of warfarin was more costly compared to the standard dosing ($246 vs $69), and the calculated incremental cost-effectiveness ratio (ICER) was $2474 per quality-adjusted life year (QALY) gained. One-way sensitivity analyses showed that our model is sensitive to the percentage of time in the therapeutic range (PTTR), the cost of genetic tests, and the utility of both pharmacogenomics-guided and standard dosing arms. However, the probabilistic sensitivity analysis demonstrates the robustness of our model. Conclusions: Warfarin dosing with pharmacogenomics testing is currently not cost-effective. However, if the cost of genotyping tests decreases to $118, the ICER would become cost-effective.

Clinical efficacy of gamma-oryzanol combined with Femoston for perimenopausal syndrome.

BACKGROUND: Perimenopausal syndrome (PMS) is a chronic disease associated with estrogen deficiency. Because of the unsatisfactory outcomes of current conventional treatments for this condition, its treatment must be continuously explored and optimized. AIM: To assess the clinical effectiveness of γ-oryzanol in combination with Femoston for PMS. METHODS: A total of 119 patients with PMS were selected from June 2023 to December 2023, which included 59 and 60 patients in the control and observation group, respectively. The control and observation groups were treated with Femoston and γ-oryzanol + Femoston, respectively. Comparative analyses were performed in terms of clinical effectiveness, safety (dizziness and headache, nausea and vomiting, and breast tenderness), sex hormones [estradiol (E2), luteinizing hormone (LH), and follicle-stimulating hormone (FSH)], lumbar spine (L1-4) and bilateral femoral bone mineral density (BMD), and sleep quality (sleeping time and frequency of awakenings from sleep). RESULTS: Compared with the control group, the observation group had statistically higher total effective rates of treatment; lower overall incidence of adverse events; higher post-treatment E2 levels and L1-4 and bilateral femoral BMD; and lower LH and FSH levels, sleeping time, and frequency of awakenings from sleep after treatment. CONCLUSION: Therefore, for the treatment of PMS, γ-oryzanol combined with Femoston is significantly better than Femoston alone in terms of clinical effectiveness, exhibiting more pronounced clinical advantages in improving safety, sex hormone levels, BMD, and sleep quality.

Effectiveness of Educational Videos Comparing Single Versus Multiple Topics: A Cluster Randomized Controlled Trial.

Background Dental caries and gingivitis are preventable diseases that remain highly prevalent among children globally and, while transmissible through the transfer of oral bacteria typically from mother to child, differ from communicable diseases that are spread through direct contact, air, or vectors. Unlike communicable diseases, dental caries and gingivitis can be effectively prevented through proper oral hygiene practices and dietary modifications. Oral health education (OHE) intends to improve oral hygiene practices and reduce oral health problems. However, evidence of the impact of multiple topics in OHE on preschool children is lacking. This study aimed to examine the effects of single- versus multiple-topic OHE delivered via video presentations on the plaque and gingival status of preschool children. Methods A parallel five-arm cluster randomized controlled trial was conducted on healthy preschool children aged five and six years. Children with chronic illnesses, disabilities, or conditions that could affect their oral health or ability to participate in the OHE intervention were excluded. OHE interventions were given to children from eight of the 10 classes assigned as intervention groups, while two classes served as the control group and received no intervention. The intervention groups received one, two, or three oral health topics using specially developed animation videos, in Malay language: toothbrushing technique (T), toothbrushing technique and the effects of sugar consumption on oral health (TS), toothbrushing technique and pathogenesis of dental caries and gingivitis (TP), and toothbrushing technique, the effects of sugar consumption on oral health, and pathogenesis of dental caries and gingivitis (TSP). Plaque and gingival scores, along with oral health knowledge, attitude, and skills (KAS), were recorded before and after the intervention. The CONSORT guidelines were followed in reporting. The analyses included descriptive statistics, one-way ANOVA, effect sizes, and multivariate analysis of covariance (MANCOVA) at a 5% significance level. Results A total of 160 participants were equally distributed into five groups (n = 32). There were no baseline differences in plaque or gingival scores. All groups showed significantly lower plaque and gingival scores post-intervention (p < 0.05), with effect sizes ranging from -1.1 to -0.7. No changes in oral health (KAS) were observed. The intervention groups had significantly lower plaque and gingival scores compared to the control group (p < 0.05), but no differences were found between them after adjusting for baseline KAS (p > 0.05). Conclusions This study suggests that including multiple topics in OHE programs for preschool children may not necessarily improve oral health outcomes. Simplified OHE may be more advantageous in terms of time, cost, human resources, and organization.

Real-world effectiveness and safety of Baloxavir Marboxil or Oseltamivir in outpatients with uncomplicated influenza A: an ambispective, observational, multi-center study.

Introduction: Baloxavir Marboxil is a per oral small-molecule antiviral for the treatment of influenza. While the efficacy and safety of Baloxavir Marboxil have been thoroughly characterized across an extensive clinical trial, studies on the effectiveness of Baloxavir Marboxil in a real-world setting are still scarce. Methods: We conducted an ambispective, observational, multi-center study that enrolled uncomplicated in-fluenza outpatients treated with Baloxavir Marboxil or Oseltamivir in East China. The primary endpoint was time from treatment to alleviation of all influenza symptoms (TTAIS). The secondary endpoints included time from treatment to alleviation of fever (TTAF) and household transmission during the duration of influenza. Results: A total of 509 patients were enrolled. The median TTAIS in the Baloxavir Marboxil group and the Oseltamivir group was 28.0 h (IQR, 20.0 to 50.0) and 48.0 h (IQR, 30.0 to 67.0), respectively. The median TTAF in the Baloxavir Marboxil group and the Oseltamivir group was 18 h (IQR, 10.0-24.0) and 30.0 h (IQR, 19.0-48.0). In the COX multivariable analysis, Baloxavir Marboxil reduced the duration of influenza symptoms (HR = 1.36 [95%CI:1.12-1.64], p = 0.002) and the duration of fever (HR = 1.93 [95%CI:1.48-2.52], p < 0.001) compared to Oseltamivir. When antiviral drugs were given within 12-48 h after symptom onset, the Baloxavir Marboxil group had a significantly shorter TTAIS compared to the Oseltamivir group. There was no significant difference in the rate of adverse events between the two group (p = 0.555). Discussion: Baloxavir Marboxil was superior to Oseltamivir in alleviating influenza symptoms in outpatients with uncomplicated influenza. Our findings suggested that compared to Oseltamivir, Baloxavir Marboxil might be more appropriate for patients with influenza 12- 48 h after symptom onset.

Efficacy and safety of radical cystectomy with ileal conduit for muscle-invasive bladder cancer in the elderly: a multicenter retrospective study.

Objective: Radical cystectomy with ileal conduit is the current mainstay of treatment for muscle-invasive bladder cancer and is also a high-risk procedure. Existing studies have limited targeted assessment of the efficacy and safety of this procedure, and the patient population appropriate for this procedure is still poorly defined. We sought to longitudinally analyze differences in the efficacy and safety of radical cystectomy with ileal conduit by age subgroups to assess whether the age factor should be used as an exclusion criterion when selecting this procedure. Materials and methods: We retrospectively examined the clinicopathological data of patients with MIBC treated with RC with IC at the Cancer Hospital of Harbin Medical University between February 2014 and October 2023. Additionally, we utilized clinical and pathological data from the SEER database (2000-2020) for external validation of our findings. Patients were categorized into elderly (≥70 years at diagnosis) and non-elderly (<70 years) groups. Statistical analyses included t-tests, non-parametric tests for continuous data, chi-square tests for categorical data, and Kaplan-Meier survival analysis. Results: In this study, 152 patients were included: 119 were categorized as non-elderly and 33 as elderly. For external validation, data from 416 patients in the SEER database were analyzed, with 172 classified as non-elderly and 244 as elderly. The results indicated that elderly patients were more likely to require ICU transfer postoperatively but exhibited a lower incidence of stoma inflammation. Additionally, both the data from our center and the external validation from the SEER database showed a concordance in cancer-specific survival (CSS) between the elderly and non-elderly groups. The efficacy of RC with IC was comparable in both elderly and non-elderly patients. Conclusion: For longitudinal age subgroups, RC with IC for both elderly and non-elderly MIBC had good efficacy and safety, and good quality of life after surgery. Although there are surgical and perioperative risks in elderly patients, there is no significant difference compared with non-elderly patients. In elderly patients requiring RC for bladder cancer, IC should remain the preferred mode of urinary diversion, and old age should not be used as an absolute exclusion criterion for IC.

Comparative analysis of the safety and effectiveness of Nirmatrelvir-Ritonavir and Azvudine in older patients with COVID-19: a retrospective study from a tertiary hospital in China.

Introduction: Numerous studies have explored the treatment outcomes of Nirmatrelvir-Ritonavir and Azvudine in older patients with COVID-19. However, direct comparisons between these two drugs are still relatively limited. This study aims to compare the safety and effectiveness of these two drugs in Chinese older patients with early infection to provide strategies for clinical treatment. Methods: Older COVID-19 patients (age ≥65) hospitalized during the winter 2022 epidemic in China were included and divided into Nirmatrelvir-Ritonavir and Azvudine. Demographics, medication information, laboratory parameters, and treatment outcomes were collected. All-cause 28-day mortality, delta cycle threshold (ΔCt), nucleic acid negative conversion time, and incidence of adverse events were defined as outcomes. Propensity score matching (PSM), Kaplan-Meier, Cox proportional hazards model, subgroup analysis, and nomograms were selected to evaluate the outcomes. Results: A total of 1,508 older COVID-19 patients were screened. Based on the inclusion and exclusion criteria, 1,075 patients were eligible for the study. After PSM, the final number of older COVID-19 patients included in the study was 375, and there were no significant differences in demographic characteristics between the two groups (p > 0.05). Compared to the Azvudine group, the Nirmatrelvir-Ritonavir group showed a higher incidence of multiple adverse events (12.8% vs 5.2%, p = 0.009). The incidence of adverse events related to abnormal renal function was higher in the Nirmatrelvir-Ritonavir group compared to the Azvudine group (13.6% vs 7.2%, p = 0.045). There were no significant differences between the two groups in terms of all-cause 28-day mortality (HR = 1.020, 95% CI: 0.542 - 1.921, p = 0.951), whereas there were significant differences in nucleic acid negative conversion time (HR = 1.659, 95% CI: 1.166 - 2.360, p = 0.005) and ΔCt values (HR = 1.442, 95% CI: 1.084 - 1.918, p = 0.012). Conclusion: Azvudine and Nirmatrelvir-Ritonavir have comparable effectiveness in reducing mortality risk. Azvudine may perform better in nucleic acid negative conversion time and virus clearance and shows slightly better safety in older patients. Further studies with a larger sample size were needed to validate the result.

Evaluating Pregnancy Rates in Fertility Awareness-Based Methods for Family Planning: Simulated Comparison of Correct Use to Avoid, Method-Related, and Total Pregnancy Rates.

Fertility awareness-based methods (FABMs), also known as natural family planning (NFP), enable couples to identify the days of the menstrual cycle when intercourse may result in pregnancy ("fertile days"), and to avoid intercourse on fertile days if they wish to avoid pregnancy. Thus, these methods are fully dependent on user behavior for effectiveness to avoid pregnancy. For couples and clinicians considering the use of an FABM, one important metric to consider is the highest expected effectiveness (lowest possible pregnancy rate) during the correct use of the method to avoid pregnancy. To assess this, most studies of FABMs have reported a method-related pregnancy rate (a cumulative proportion), which is calculated based on all cycles (or months) in the study. In contrast, the correct use to avoid pregnancy rate (also a cumulative proportion) has the denominator of cycles with the correct use of the FABM to avoid pregnancy. The relationship between these measures has not been evaluated quantitatively. We conducted a series of simulations demonstrating that the method-related pregnancy rate is artificially decreased in direct proportion to the proportion of cycles with intermediate use (any use other than correct use to avoid or targeted use to conceive), which also increases the total pregnancy rate. Thus, as the total pregnancy rate rises (related to intermediate use), the method-related pregnancy rate falls artificially while the correct use pregnancy rate remains constant. For practical application, we propose the core elements needed to assess correct use cycles in FABM studies. Summary: Fertility awareness-based methods (FABMs) can be used by couples to avoid pregnancy, by avoiding intercourse on fertile days. Users want to know what the highest effectiveness (lowest pregnancy rate) would be if they use an FABM correctly and consistently to avoid pregnancy. In this simulation study, we compare two different measures: (1) the method-related pregnancy rate; and (2) the correct use pregnancy rate. We show that the method-related pregnancy rate is biased too low if some users in the study are not using the method consistently to avoid pregnancy, while the correct use pregnancy rate obtains an accurate estimate. Short Summary: In FABM studies, the method-related pregnancy rate is biased too low, but the correct use pregnancy rate is unbiased.

Additional Hepatic Arterial Infusion Chemotherapy to Sorafenib Was Cost-Effective for Hepatocellular Carcinoma with Major Portal Vein Tumor Thrombosis.

Purpose: The combination of sorafenib and hepatic arterial infusion chemotherapy (SoHAIC) has shown to enhance overall survival rates in patients with advanced hepatocellular carcinoma and major portal vein tumor thrombosis (HCC-Vp3-4) compared to sorafenib alone. Our objective was to evaluate the cost-effectiveness of SoHAIC versus sorafenib for the treatment of HCC-Vp3-4, taking into account the viewpoint of Chinese healthcare payers. Methods: This pharmacoeconomic study employed a Markov model to assess the cost-effectiveness of treating HCC-Vp3-4 with SoHAIC in comparison to sorafenib. The patient characteristics were drawn from individuals from the trial conducted between June 2017 and November 2019, with cost and health value data sourced from published literature. The primary outcome measure in this research was the incremental cost-effectiveness ratio (ICER), which indicates the additional cost per quality-adjusted life year (QALY). The willingness-to-pay (WTP) threshold per QALY was set at $30,492.00. Furthermore, 1-way sensitivity and probabilistic sensitivity analyses were carried out to validate the consistency of the results. Results: In the baseline scenario, sorafenib resulted in 0.42 QALY at a cost of $10,507.89, while SoHAIC generated 1.66 QALY at a cost of $32,971.56. When comparing SoHAIC to sorafenib, the ICER was $18,237.20 per QALY, which was below the WTP threshold per QALY. Furthermore, the 1-way sensitivity analysis demonstrated that the ICER remained within the WTP threshold despite fluctuations in variables. In the probabilistic sensitivity analysis, SoHAIC had a 98.8% probability of being cost-effective at the WTP threshold, considering a wide range of parameters. Conclusion: In this cost-effectiveness evaluation, SoHAIC demonstrated cost-effectiveness over sorafenib for HCC with major portal vein tumor thrombosis, as observed from the perspective of a Chinese payer.

The Effectiveness of Medical Adherence Mobile Health Solutions for Individuals With Epilepsy: Protocol for a Systematic Review.

BACKGROUND: Epilepsy requires continuous management and treatment to optimize patient outcomes. The advancement of digital health has led to the development of various mobile health (mHealth) tools designed to enhance treatment adherence among individuals with epilepsy. These solutions offer crucial support through features such as reminders, educational resources, personalized feedback, assistance with managing costs, shared decision-making, and access to supportive communities. To design effective medication adherence mHealth solutions, it is essential to evaluate the effectiveness of existing mHealth tools, understand the unique circumstances of different patients, and identify the roles of health care professionals within the digital care pathway. Existing studies on epilepsy primarily focus on self-management, whereas the effectiveness and usability of medical adherence mHealth solutions often remain overlooked. Furthermore, the involvement of health care professionals in digital care pathways for epilepsy as well as the impact of adherence mHealth solutions on the patient experience have not been adequately explored. OBJECTIVE: This study aims to assess the effectiveness of current mHealth solutions designed to improve medical adherence among patients with epilepsy. Furthermore, the study will examine the experiences of patients using mHealth solutions for maintaining medical adherence in epilepsy care. Finally, this review intends to determine the roles of health care professionals within mHealth systems aimed at supporting adherence to medication among patients with epilepsy. METHODS: A systematic literature review has been selected as the appropriate method to address the research questions, adhering to the PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analyses) guidelines. The inclusion and exclusion criteria have been carefully selected, and both qualitative and quantitative analyses will be used to analyze the results. The expected results will mainly focus on the comparison, classification, and analysis of the effectiveness of current medical adherence mHealth tools. Moreover, the patient experiences using available medical adherence mHealth tools for epilepsy will be assessed. Finally, the role of health care professionals in the epilepsy digital care pathway will be explored, with emphasis on medical adherence. RESULTS: The initial search, full-text screening, and data extraction have been carried out. Thirty-three papers were included in the final stage of the review. The study is expected to be completed by October 2024. CONCLUSIONS: To enhance the digital care pathway for epilepsy, a medical adherence mHealth solution should be personalized, manage medications, include an alarm system, track seizures, support consultations, and offer updated treatment plans. This study aims to understand how findings from the research questions can improve mHealth solutions for individuals with epilepsy. Insights from this research on the effectiveness of current mHealth adherence solutions will provide guidance for developing future mHealth systems, making them more efficient and effective in managing epilepsy. TRIAL REGISTRATION: PROSPERO CRD4202347400; https://tinyurl.com/48mfx22e. INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): DERR1-10.2196/55123.