OBJECTIVE: We comprehensively characterized a large pediatric cohort with focal cortical dysplasia (FCD) type 1 to expand the phenotypic spectrum and to identify predictors of postsurgical outcomes. METHODS: We included pediatric patients with histopathological diagnosis of isolated FCD type 1 and at least 1 year of postsurgical follow-up. We systematically reanalyzed clinical, electrophysiological, and radiological features. The results of this reanalysis served as independent variables for subsequent statistical analyses of outcome predictors. RESULTS: All children (N = 31) had drug-resistant epilepsy with varying impacts on neurodevelopment and cognition (presurgical intelligence quotient [IQ]/developmental quotient scores = 32-106). Low presurgical IQ was associated with abnormal slow background electroencephalographic (EEG) activity and disrupted sleep architecture. Scalp EEG showed predominantly multiregional and often bilateral epileptiform activity. Advanced epilepsy magnetic resonance imaging (MRI) protocols identified FCD-specific features in 74.2% of patients (23/31), 17 of whom were initially evaluated as MRI-negative. In six of eight MRI-negative cases, fluorodeoxyglucose-positron emission tomography (PET) and subtraction ictal single photon emission computed tomography coregistered to MRI helped localize the dysplastic cortex. Sixteen patients (51.6%) underwent invasive EEG. By the last follow-up (median = 5 years, interquartile range = 3.3-9 years), seizure freedom was achieved in 71% of patients (22/31), including seven of eight MRI-negative patients. Antiseizure medications were reduced in 21 patients, with complete withdrawal in six. Seizure outcome was predicted by a combination of the following descriptors: age at epilepsy onset, epilepsy duration, long-term invasive EEG, and specific MRI and PET findings. SIGNIFICANCE: This study highlights the broad phenotypic spectrum of FCD type 1, which spans far beyond the narrow descriptions of previous studies. The applied multilayered presurgical approach helped localize the epileptogenic zone in many previously nonlesional cases, resulting in improved postsurgical seizure outcomes, which are more favorable than previously reported for FCD type 1 patients.
- MeSH
- Child MeSH
- Electroencephalography * methods MeSH
- Epilepsy MeSH
- Focal Cortical Dysplasia MeSH
- Cohort Studies MeSH
- Infant MeSH
- Humans MeSH
- Magnetic Resonance Imaging * MeSH
- Malformations of Cortical Development, Group I * surgery complications diagnostic imaging MeSH
- Malformations of Cortical Development surgery complications diagnostic imaging MeSH
- Adolescent MeSH
- Positron-Emission Tomography MeSH
- Child, Preschool MeSH
- Drug Resistant Epilepsy * surgery diagnostic imaging physiopathology MeSH
- Treatment Outcome MeSH
- Check Tag
- Child MeSH
- Infant MeSH
- Humans MeSH
- Adolescent MeSH
- Male MeSH
- Child, Preschool MeSH
- Female MeSH
- Publication type
- Journal Article MeSH
BACKGROUND AND OBJECTIVE: Bladder cancer (BCa) imposes a substantial economic burden on health care systems and patients. Understanding these financial implications is crucial for effective resource allocation and optimization of treatment cost effectiveness. Here, we aim to systematically review and analyze the financial burden of BCa from the health care and patient perspectives. METHODS: A Preferred Reporting Items for Systematic Reviews and Meta-analyses (PRISMA)-compliant systematic review was conducted, searching PubMed/Medline, Embase, and public sources for studies evaluating the financial impact of BCa, encompassing costs, cost effectiveness, and financial toxicity (FT). KEY FINDINGS AND LIMITATIONS: Non-muscle-invasive BCa (NMIBC) incurs significant costs for surveillance and treatment, with costs exceeding $200 000 after 5 yr for high-risk NMIBC patients progressing after bacillus Calmette-Guerin (BCG) treatment (including inpatient, outpatient, and physician service expenses). Muscle-invasive BCa generates substantial costs from radical cystectomy (RC) and neoadjuvant chemotherapy, averaging $30 000-40 000 from surgical costs of RC, with additional expenses in case of complications. Trimodal therapy has higher costs (1-yr management cost >$200 000) than RC because of higher outpatient, radiology, and medication costs. Metastatic BCa incurs the highest financial burden, with systemic therapy costs ranging from $40 000 to over $100 000 per five-cycle course, increasing further with combination therapies (ie, enfortumab vedotin and pembrolizumab), treatment-related toxicity, and supportive care. FT is particularly prevalent among younger, less educated, and minority populations. CONCLUSIONS AND CLINICAL IMPLICATIONS: BCa treatment, particularly in advanced stages, imposes a substantial economic burden. Innovations in care, while improving oncologic outcomes, necessitate detailed cost-effectiveness assessments. Addressing these economic challenges is essential for optimizing BCa management, targeting patients at a higher risk of FT, and improving patient quality of life.
- MeSH
- Cost-Benefit Analysis MeSH
- Cystectomy economics adverse effects MeSH
- Humans MeSH
- Neoplasm Metastasis MeSH
- Urinary Bladder Neoplasms * economics therapy pathology MeSH
- Health Care Costs * MeSH
- Cost of Illness * MeSH
- Check Tag
- Humans MeSH
- Publication type
- Journal Article MeSH
- Review MeSH
- Systematic Review MeSH
BACKGROUND AND OBJECTIVES: Enhanced Recovery After Surgery (ERAS) guidelines for Radical Cystectomy (RC) were published over ten years ago. Aim of this systematic review is to update ERAS recommendations for patients undergoing RC and to give an expert opinion on the relevance of each single ERAS item. METHODS: A systematic review was performed to identify the impact of each single ERAS item on RC outcomes. Embase and Medline (through Pubmed) were searched systematically. Relevant articles were selected and graded. For each ERAS item, a level of evidence was determined. An e-Delphi consensus was then performed amongst an international panel with renowned experience in RC to provide recommendations based on expert opinion. KEY FINDINGS AND LIMITATIONS: Preoperative medical optimization and avoiding bowel preparation are highly recommended. Robotic-assisted RC with intracorporeal urinary diversion is moderately recommended and can help in applying other ERAS items, such as early mobilization. Medical thromboprophylaxis should be administered and nasogastric tube should be removed at the end of surgery. Perioperative fluid restriction as well as opioid-sparing anesthesia protocols should be implemented. Generally, consensus was reached on most ERAS items, with the exception of epidural anesthesia (no consensus), resection site drainage (consensus against), and type of urinary drainage. Limitations include the lack of a multidisciplinary approach to the present consensus, giving however a highly specialized surgical opinion on ERAS. CONCLUSIONS: and clinical implications: The current study updates ERAS recommendations for patients undergoing RC and suggests application of ERAS by a panel of experts in the field.
- MeSH
- Early Ambulation MeSH
- Surgeons MeSH
- Cystectomy * methods MeSH
- Urinary Diversion methods MeSH
- Humans MeSH
- Urinary Bladder Neoplasms surgery MeSH
- Robotic Surgical Procedures MeSH
- Practice Guidelines as Topic MeSH
- Enhanced Recovery After Surgery * MeSH
- Check Tag
- Humans MeSH
- Publication type
- Journal Article MeSH
- Review MeSH
- Systematic Review MeSH
BACKGROUND: Endoscopic retrograde cholangiopancreatography (ERCP) still has a relatively high complication rate, underscoring the importance of high-quality training. Despite existing guidelines, real-world data on training conditions remain limited. This pan-European survey aims to systematically explore the perceptions surrounding ERCP training. METHODS: A survey was distributed through the friends of United European Gastroenterology (UEG) Young Talent Group network to physicians working in a UEG member or associated states who regularly performed ERCPs. RESULTS: Of 1035 respondents from 35 countries, 649 were eligible for analysis: 228 trainees, 225 trainers, and 196 individuals who regularly performed ERCP but were neither trainees nor trainers. The mean age was 43 years, with 72.1% identifying as male, 27.6% as female, and 0.3% as non-binary. The majority (80.1%) agreed that a structured training regimen is desirable. However, only 13.7% of trainees and 28.4% of trainers reported having such a structured program in their institutions. Most respondents (79.7%) supported the concept of concentrating training in centers meeting specific quality metrics, with 64.1% suggesting a threshold of 200 annual ERCPs as a prerequisite. This threshold revealed that 36.4% of trainees pursued training in lower-volume centers performing <200 ERCPs annually. As many as 70.1% of trainees performed <50 annual ERCPs, whereas only 5.0% of trainers performed <50 ERCPs annually. A low individual trainee caseload (<50 ERCPs annually) was more common in lower-volume centers than in higher-volume centers (82.9% vs. 63.4%). CONCLUSIONS: The first pan-European survey investigating ERCP training conditions reveals strong support for structured training and the concentration of training efforts within centers meeting specific quality metrics. Furthermore, this survey exposes the low availability of structured training programs with many trainees practicing at lower-volume centers and 71% of all trainees having little hands-on exposure. These data should motivate to standardize ERCP training conditions further and ultimately improve patient care throughout Europe.
- MeSH
- Cholangiopancreatography, Endoscopic Retrograde * standards adverse effects MeSH
- Adult MeSH
- Gastroenterology * education MeSH
- Clinical Competence standards MeSH
- Middle Aged MeSH
- Humans MeSH
- Surveys and Questionnaires statistics & numerical data MeSH
- Education, Medical, Graduate * standards methods MeSH
- Check Tag
- Adult MeSH
- Middle Aged MeSH
- Humans MeSH
- Male MeSH
- Female MeSH
- Publication type
- Journal Article MeSH
- Research Support, Non-U.S. Gov't MeSH
- Geographicals
- Europe MeSH
BACKGROUND: Psychotherapy outcomes are typically measured in terms of symptom relief. However, this method might overlook important changes from clients' perspectives when they are asked to report on them. A more client-centred approach might bring a deeper understanding of psychotherapy outcomes. We aimed to evaluate the outcomes identified by clients within qualitative psychotherapy research. METHODS: The PsycArticles, PsycInfo, and MEDLINE Complete databases were searched for English language studies published until Nov 11, 2023. Additional studies were identified through references in the primary studies and previous meta-analyses or systematic reviews. Search terms were related to psychotherapy and counselling, clients' or patients' experiences, psychotherapy outcomes and changes, post-treatment perspectives, and types of qualitative methods. Qualitative studies on client-identified outcomes of individual psychotherapy were included. Findings related to clients' perceptions of psychotherapy outcomes were extracted (by ML and checked by TR and LT) and analysed (by all authors) using the descriptive-interpretative meta-analytic approach. All authors have personally experienced psychotherapy as clients. This study was pre-registered with PROSPERO (CRD42021277330). FINDINGS: We included 177 studies in the qualitative meta-analysis, from 24 countries, including descriptions from 2908 clients. Most of the studies were of good quality; they covered a wide range of therapeutic approaches and diagnoses. The descriptions of psychotherapy outcomes were classified into 60 meta-categories and grouped into ten clusters. These clusters related to clients' relational and social functioning; their emotional functioning; self-awareness, self-understanding, and more adaptive cognitive processing; behavioural functioning; developing their own resources; clients' attitudes towards themselves; generally embracing life; symptom and problem change; and more general wellbeing. The tenth cluster was outcomes that could not be clearly attributed to psychotherapy, which was considered outside the scope of this study. INTERPRETATION: The meta-analysis showed that clients value outcome dimensions beyond symptom reduction, such as deeper self-understanding, enhanced self-agency, and greater social engagement. By examining psychotherapy outcomes across various diagnoses and therapeutic approaches, we highlight limitations in traditional outcome measures, showing the need for more comprehensive, client-centred assessment tools and the value of incorporating qualitative methods into understanding dimensions of change. FUNDING: European Union.
- MeSH
- Mental Disorders therapy psychology MeSH
- Qualitative Research MeSH
- Humans MeSH
- Psychotherapy * methods MeSH
- Treatment Outcome MeSH
- Check Tag
- Humans MeSH
- Publication type
- Journal Article MeSH
- Meta-Analysis MeSH
- Research Support, Non-U.S. Gov't MeSH
BACKGROUND: A third of endovascularly treated patients with stroke experience incomplete reperfusion (expanded Thrombolysis in Cerebral Infarction [eTICI] <3), and the natural evolution of this incomplete reperfusion remains unknown. We systematically reviewed the literature and performed a meta-analysis on the natural evolution of incomplete reperfusion after endovascular therapy. METHODS: A systematic review of MEDLINE, Embase, and PubMed up until March 1, 2024, using a predefined strategy. Only full-text English-written articles reporting rates of either favorable (ie, delayed reperfusion (DR) or no new infarct) or unfavorable progression (ie, persistent perfusion deficit or new infarct) of incompletely reperfused tissue were included. The primary outcome was the rate of DR and its association with functional independence (modified Rankin Scale score, 0-2) at 90 days postintervention. Pooled odds ratios with 95% CIs were calculated using a random-effects model. RESULTS: Six studies involving 950 patients (50.7% female; median age, 71 years; interquartile range, 60-79) were included. Four studies assessed the evolution of incomplete reperfusion on magnetic resonance imaging perfusion imaging, while 2 studies used diffusion-weighted imaging and noncontrast computed tomography imaging, where new infarct was used to denote unfavorable progression. Five studies defined incomplete reperfusion as eTICI 2b50 or 2c. DR occurred in 41% (interquartile range, 33%-51%) of cases 24 hours postintervention. Achieving DR was associated with a higher likelihood of functional independence at 90 days (odds ratio, 2.5 [95% CI, 1.9-3.4]). CONCLUSIONS: Nearly half of eTICI <3 patients achieve DR, leading to favorable clinical outcomes. This subgroup may derive limited or potentially harmful effects from pursuing additional reperfusion strategies (eg, intra-arterial lytics or secondary thrombectomy). Accurately predicting the evolution of incomplete reperfusion could optimize patient selection for adjunctive reperfusion strategies at the end of an intervention. REGISTRATION: URL: https://www.clinicaltrials.gov; Unique identifiers: NCT05499832.
- MeSH
- Endovascular Procedures * methods MeSH
- Ischemic Stroke * surgery diagnostic imaging therapy MeSH
- Middle Aged MeSH
- Humans MeSH
- Reperfusion methods MeSH
- Aged MeSH
- Thrombolytic Therapy methods MeSH
- Check Tag
- Middle Aged MeSH
- Humans MeSH
- Male MeSH
- Aged MeSH
- Female MeSH
- Publication type
- Journal Article MeSH
- Meta-Analysis MeSH
- Systematic Review MeSH
OBJECTIVES: Mindfulness-based interventions (MBIs) for persons with dementia (PwD) have yielded mixed results, possibly attributable to the fact that little is known about the validity and reliability of trait mindfulness self-report measures in PwD. This narrative review sought to identify studies involving self-reported trait mindfulness and other clinical measures that may hold information on the convergent validity and reliability of these measures in PwD. METHODS: Scientific databases were searched for studies involving PwD and mindfulness assessments. RESULTS: N = 426 studies from PubMed and N = 156 from PsychInfo databases were reviewed. Four cross-sectional studies were identified that allowed inferences about the validity of mindfulness measures. A qualitative review indicated that convergent validity with other measures varied with sample heterogeneity and cognitive impairment. Merely one MBI included self-reported trait mindfulness, however without reporting sample-specific validity or reliability. CONCLUSIONS: Despite efforts to implement MBIs in PwD, information on basic methodological psychometric issues is minimal. Future studies ought to address the validity and reliability of self-reported mindfulness in detail across different stages of dementia. CLINICAL IMPLICATIONS: Results of MBIs need to be considered cautiously. Basic information about psychometric properties of mindfulness self-report measures is required and these measures need to be included systematically in MBIs.
- MeSH
- Dementia * psychology therapy MeSH
- Humans MeSH
- Psychometrics methods MeSH
- Reproducibility of Results MeSH
- Aged MeSH
- Mindfulness * methods MeSH
- Self Report * standards MeSH
- Check Tag
- Humans MeSH
- Aged MeSH
- Publication type
- Journal Article MeSH
- Review MeSH
Necrotizing enterocolitis (NEC) is a severe intestinal condition primarily affecting preterm neonates. It has a high mortality rate, particularly in infants with a birthweight of below 1,500 g or for those requiring surgical intervention. The European Reference Network for Inherited and Congenital Anomalies (ERNICA) has developed a clinical practice guideline to aid clinical decision-making pertaining to the surgical treatment and management of NEC in preterm neonates. This guideline was developed in accordance with the Guidelines 2.0 checklist and GRADE methodology. A multidisciplinary group of Europe's top experts collaborated with patient representatives to develop this guideline. After selecting critical points in care for which recommendations are required, a systematic review of the literature and critical appraisal of the evidence was performed. The Evidence to Decision framework was used as a guide to structure the consensus meetings and draft the recommendations. The panel developed seven recommendations and three good practice statements on the following topics: indications for surgery, peritoneal drainage, surgical technique, management of extensive NEC, enteral feeding, and neurodevelopmental outcomes in premature neonates with NEC. The certainty of evidence was graded as (very) low for most recommendations. However, the panel weighed up the benefits and harms in light of all relevant arguments and expert opinion. This guideline provides recommendations on caring for premature neonates with NEC. These recommendations can assist clinicians in their care decisions and can inform families on treatment options and relevant considerations. This guideline will be revised every 5 years to ensure it remains up to date.
- MeSH
- Enteral Nutrition MeSH
- Clinical Decision-Making MeSH
- Humans MeSH
- Evidence-Based Medicine MeSH
- Enterocolitis, Necrotizing * surgery diagnosis MeSH
- Infant, Premature MeSH
- Infant, Newborn MeSH
- Check Tag
- Humans MeSH
- Infant, Newborn MeSH
- Publication type
- Journal Article MeSH
- Practice Guideline MeSH
- Systematic Review MeSH
- Geographicals
- Europe MeSH
Necrotizing enterocolitis (NEC) is one of the most devastating intestinal diseases observed in preterm in the first days of life. Researchers have recently focused on potential predictive biomarkers for early and concomitant diagnoses. Thus, we inquired about the linkage of intestinal dysbiosis, one of the most important factors in NEC development to the gut microbiota. In this study, the systematic differences in the bacterial composition between neonates affected by NEC and healthy newborns were highlighted by metagenomic analysis. The next-generation sequencing of the V3-V4 variable region of the 16S rRNA gene and gene-specific qPCR analyzed the untargeted gut microbiota. Total bacteria, total and fecal coliform loads in stool samples with NEC were higher than control. OTU-level relative abundances of NEC infant was characterized by Firmicutes and Bacteroidetes at phylum levels. At the genus level, NEC stool was identified by the lack of Klebsiella and the presence of Roseburia, Blautia, and Parasutterella. Finally, Clostridium fessum was the predominant species of Clostridium genus in disease and healthy specimens at the species level, whereas Clostridium jeddahitimonense was at NEC diagnosis. Despite a strong relationship between pathophysiology and characterization of gut microbiota at a clinical diagnosis of NEC, our results emphasize the broad difficulty in identifying potential biomarkers.
- MeSH
- Bacteria * classification genetics isolation & purification MeSH
- DNA, Bacterial genetics MeSH
- Dysbiosis microbiology MeSH
- Feces * microbiology MeSH
- Humans MeSH
- Metagenomics MeSH
- Enterocolitis, Necrotizing * microbiology MeSH
- Infant, Premature MeSH
- Infant, Newborn MeSH
- RNA, Ribosomal, 16S * genetics MeSH
- Gastrointestinal Microbiome * MeSH
- High-Throughput Nucleotide Sequencing MeSH
- Check Tag
- Humans MeSH
- Male MeSH
- Infant, Newborn MeSH
- Female MeSH
- Publication type
- Journal Article MeSH
BACKGROUND AND OBJECTIVE: Non-muscle-invasive bladder cancer (NMIBC) poses a significant clinical challenge, particularly when failing bacillus Calmette-Guérin (BCG) therapy, necessitating alternative treatments. Despite radical cystectomy being the recommended treatment, many patients are unfit or unwilling to undergo this invasive procedure, highlighting the need for effective bladder-sparing therapies. This review aims to summarize and report the evidence on the efficacy and to estimate the costs of bladder-preserving strategies used in NMIBC recurrence after failure of intravesical BCG therapy. METHODS: We systematically searched online databases for prospective studies investigating intravesical therapy, systemic therapy, or combination of both in patients treated previously with BCG. Owing to significant heterogeneity across the studies, a meta-analysis was inappropriate. A sensitivity analysis was performed in an exploratory manner. We used a decision-analytic Markov model to compare novel U.S. Food and Drug Administration-approved treatments with a 2-yr time horizon. KEY FINDINGS AND LIMITATIONS: A total of 57 studies published between 1998 and 2024, with 68 unique study arms and consisting of 2589 patients, were identified. The 3-mo overall response rate (ORR) across all studies, complete response rate (CRR) in concomitant carcinoma in situ (CIS) or CIS only disease, and recurrence-free rate (RFR) in papillary disease were estimated to be 52.4% (95% confidence interval [CI]: 45.4-59.2), 52.8% (95% CI: 42.9-62.6), and 26.4% (95% CI: 13.3-45.6), respectively. The 12-mo ORR, CRR, and RFR were estimated to be 78% (95% CI: 52.9-91.8), 27.8% (95% CI: 21.3-35.4), and 25.4% (95% CI: 18.2-34.2), respectively. The progression rate was estimated to be 13% (95% CI: 9-18.2). The mean proportion of patients treated with radical cystectomy was estimated to be 24.7 (range 0-85.7). The reported toxicity grades were overall mild, with a median of 3.4% (range 0-33.3%) participants experiencing a dose limiting toxicity. Compared with using radical cystectomy to treat patients failing BCG therapy, at a willingness-to-pay threshold of 100 000 USD, nadofaragene firadenovec was cost effective, with an incremental cost-effectiveness ratio (ICER) of 10 014 USD per quality-adjusted life year (QALY), while nogapendekin alfa inbakicept was less cost effective than nadofaragene firadenovec (ICER of 44 602 USD per QALY). Pembrolizumab, which dominated, was both less costly and more effective than the other strategies. CONCLUSIONS AND CLINICAL IMPLICATIONS: Salvage bladder-sparing therapies show a response rate of around 50% at 3 mo in patients with NMIBC failing BCG. However, long-term data are heterogeneous. Nevertheless, recently developed agents show promising tumor control activity. In the rapidly evolving landscape of urothelial cancer, some of these treatment strategies might be cost effective and improve patients' quality of life. The findings of our review highlight the need for novel, more effective therapeutic strategies. PATIENT SUMMARY: In this study, we reviewed the evidence on the efficacy of bladder-preserving strategies used in patients with bladder cancer recurrence after failing bacillus Calmette-Guérin (BCG) therapy. We found that these strategies show a response rate of around 50% at 3 mo. However, long-term data are heterogeneous. Nevertheless, recently developed agents show promising tumor control activity. In the rapidly evolving landscape of urothelial cancer, some of these treatment strategies might be cost effective and improve patients' quality of life.
- MeSH
- Adjuvants, Immunologic * therapeutic use economics MeSH
- Cost-Benefit Analysis * MeSH
- Administration, Intravesical MeSH
- BCG Vaccine * therapeutic use economics MeSH
- Neoplasm Invasiveness MeSH
- Humans MeSH
- Neoplasm Recurrence, Local MeSH
- Non-Muscle Invasive Bladder Neoplasms MeSH
- Urinary Bladder Neoplasms * drug therapy pathology therapy economics MeSH
- Treatment Failure MeSH
- Check Tag
- Humans MeSH
- Publication type
- Journal Article MeSH
- Review MeSH
- Systematic Review MeSH