Group Of Patients Research Articles

Surgical treatment for recurrent patellar dislocation with severe torsional deformities:Double-level derotational osteotomy may not have a clear advantage over single-level derotational osteotomy in improving clinical and radiological outcomes.

The purpose of this study was toinvestigate whether double-level (femur + tibia) derotational osteotomy is superior to single-level femoral derotational osteotomy for recurrent patellar dislocation with severe femoral and tibial rotational deformities (femoral anteversion >30° and external tibial torsion >30°). Between January 2015 and June 2020, a total of 115 knees with recurrent patellar dislocation treated with combined medial patellofemoral ligament reconstruction (MPFL-R) and derotational osteotomies were evaluated after a minimum follow-up of 2 years. Among these cases, 15 knees that underwent double-level derotational osteotomy were included in the double-level group, which was propensity-matched in a 1:2 ratio to a single-level group of patients who underwent single-level femoral derotational osteotomy (30 knees). The clinical and radiological outcomes were evaluated and compared between the groups. Furthermore, the foot progression angle was measured preoperatively and 2 years after surgery. The patient-specific variables did not differ significantly between the double-and the single-level groups after propensity score matching. The postoperative mean foot progression angle was significantly lower in the double-level groupthan in the single-level group (9° ± 8° vs. 15° ± 11°; p = 0.014); however, there were no statistically significant differences between the groups in terms of any other clinical and radiological assessments. For patients with severe femoral and tibial torsional deformities (femoral anteversion >30° and external tibial torsion >30°), the double-level derotational osteotomy is superior to single-level osteotomy in maintaining normal foot progression angle, but it does not show an advantage in terms of patient-reported outcomes, radiological resultsand redislocation rate at minimum 2 years of follow-up. Furthermore, concomitant excessive external tibial torsion (>30°) did not have an adverse effect on clinical outcomes in patients who underwent derotational distal femoral osteotomy with MPFL-R due to excessive femoral anteversion. Level III.

Metabolomic profiling of Prader-Willi syndrome compared with essential obesity

IntroductionPrader-Willi syndrome (PWS) is a rare disease, which shows a peculiar clinical phenotype, including obesity, which is different from essential obesity (EOB). Metabolomics might represent a valuable tool to reveal the biochemical mechanisms/pathways underlying clinical differences between PWS and EOB. The aim of the present (case-control, retrospective) study was to determine the metabolomic profile that characterizes PWS compared to EOB.MethodsA validated liquid chromatography-tandem mass spectrometry (LC-MS/MS) targeted metabolomic approach was used to measure a total of 188 endogenous metabolites in plasma samples of 32 patients with PWS (F/M = 23/9; age: 31.6 ± 9.2 years; body mass index [BMI]: 42.1 ± 7.0 kg/m2), compared to a sex-, age- and BMI-matched group of patients with EOB (F/M = 23/9; age: 31.4 ± 6.9 years; BMI: 43.5 ± 3.5 kg/m2).ResultsBody composition in PWS was different when compared to EOB, with increased fat mass and decreased fat-free mass. Glycemia and HDL cholesterol were higher in patients with PWS than in those with EOB, while insulinemia was lower, as well as heart rate. Resting energy expenditure was lower in the group with PWS than in the one with EOB, a difference that was missed after fat-free mass correction. Carrying out a series of Tobit multivariable linear regressions, adjusted for sex, diastolic blood pressure, and C reactive protein, a total of 28 metabolites was found to be associated with PWS (vs. non-PWS, i.e., EOB), including 9 phosphatidylcholines (PCs) ae, 5 PCs aa, all PCs aa, 7 lysoPCs a, all lysoPCs, 4 acetylcarnitines, and 1 sphingomyelin, all of which were higher in PWS than EOB.ConclusionsPWS exhibits a specific metabolomic profile when compared to EOB, suggesting a different regulation of some biochemical pathways, fundamentally related to lipid metabolism.

Testing the disintegration and texture-related palatability predictions for orodispersible tablets using an instrumental tool coupled with multivariate analysis: Focus on process variables and analysis settings

Orodispersible tablets (ODTs) represent a growing category of dosage forms intended to increase the treatment acceptability for special groups of patients. ODTs are designed to rapidly disintegrate in the oral cavity and to be administered without water. In addition, ODTs are easy to manufacture using standard excipients and pharmaceutical equipment.This study adds to previously published research that developed an instrumental tool to predict oral disintegration and texture-related palatability of ODTs with different formulations. The current study aimed to challenge the predictive capacity of the models under variable process conditions. The studied process parameters with potential impact on the pharmaceutical properties, texture profiles, and palatability were the compression pressure, punch shape and diameter. Subsequently, for all the placebo and drug-loaded ODTs, the in vivo disintegration time and texture-related palatability were determined with healthy volunteers.Previously developed regression models were applied to predict the formulation's disintegration time and texture-related palatability characteristics of ODTs obtained under different experimental conditions. The influence of process variables on the predictive performance of the models was estimated by calculating the residuals as the difference between the predicted and observed values for the investigated response. Increasing the speed of the analyser`s probe from 0.01 mm/s to 0.02 mm/s led to an improved differentiation of the texture profiles. The in vivo disintegration time and texture-related palatability scores were only influenced by the mechanical resistance and the tablet shape. Lower score was observed for the larger diameter tablets (10 mm). Overall, the prediction of the disintegration time at 0.02 mm/s was more accurate, except for stronger tablets. The best prediction of texture-related palatability was achieved for the 10 mm tablets, tested at 0.01 mm/s speed.The same model achieved good predictions of the oral disintegration time for all API-loaded formulations, which confirmed the ability of the texture analysis to capture process-related variability. Drug loading decreased the predictive capacity of the texture-related palatability because of the taste effect.

Causes and clinical symptoms of anaphylactic reactions in children: experience of the first «Pediatric Moscow Anaphylaxis Registry» in the Russian Federation

BACKGROUND: In Russian, there was a problem of the lack of registry of anaphylaxis, as the most important tool for monitoring this group of patients. Since 2022, the first "Pediatric Moscow Anaphylaxis Registry" has started working at the Morozov Children's Hospital. AIMS: Analysis of data from the Pediatric Moscow Anaphylaxis Registry to determine frequency, causes andclinical characteristics of anaphylaxis among children of the Moscow region. MATERIALS AND METHODS: Statistical analysis of the questionnaire data of 69 children aged 0-18, who were prospectively included in the Register during emergency hospitalization due to anaphylaxis, was carried out. RESULTS: The incidence of anaphylaxis among children hospitalized with acute allergic reactions from May 2022 to September 2023 was 5.8%. Higher percentage of anaphylaxis (38%) were observed among adolescents aged 13-18, three patients developed anaphylaxis in the first year of life (minimum age 1 month). Boys (70%) prevailed among patients under the age of 13, and girls (56%) prevailed among adolescents aged 13-18 (p=0.042). 75% anaphylaxis developed at home. Food anaphylaxis was dominant (75%-91%). Tree nuts were the most common trigger of food anaphylaxis – 35%, and confectionery products - 17%. 56% of children experienced symptoms from 4 or more organ systems, the most common symptoms were: skin/mucous (94%), respiratory (78%) and laryngeal symptoms (64%). Emergency care, diagnosed anaphylaxis in 22%, administration of epinephrine was 54%. The frequency of epinephrine use among children under 3 years was lower compared to adolescents aged 13-18 (25% vs 76%, p=0.003). CONCLUSIONS:Data from the "Pediatric Moscow Register of Anaphylaxis" showed a high incidence of anaphylaxis among patients with acute allergic reactions. More than 1/3 of anaphylaxis occurs in adolescents aged 13-18 years, anaphylaxis occurs in the first month of life. Most often, anaphylaxis occurs at home, which requires training patients to eliminate the trigger. The dominant cause of anaphylaxis in children in the Russian, regardless of age, are food allergens, tree nuts induce food anaphylaxis in more than 1/3 of cases. The emergency care diagnoses anaphylaxis in 22% of cases, epinephrine is administered in 54%, minimal use of epinephrine was among children under 3 years of age.

Arnold-Chiari Syndrome – a review

Arnold Chiari syndrome, also known as Chiari malformation, is a group of deformations of the posterior cranial fossa and hindbrain, consisting in the lowering of the cerebellum or its tonsils towards the occipital foramen. This leads to a reduction in the volume reserve within the foramen magnum, pressure on surrounding structures and obstruction of the flow of cerebrospinal fluid. The process is most often asymptomatic. Symptoms include headaches, neck pain, but also fainting, sinus bradycardia, coordination disorders and many others. Chiari malformation is often diagnosed incidentally. The basis of diagnosis is the detection of characteristic morphological features in magnetic resonance imaging. Surgical treatment is successfully used among patients with severe Arnold-Chiari syndrome and progression of clinical symptoms. Materials and methods Literature included in the PubMed, BioMed Central and Polish Medical Platform databases searched by means of the words such as Gamma Knife, CyberKnife, radiosurgery, stereotactic radiotherapy. Sources quoted in selected works were also used. Summary Arnold-Chiari malformation is often discovered incidentally. If the features observed on MRI suggest the above diagnosis, the patient should be referred to a neurologist. After excluding other possible causes of the imaged morphological features and performing a dedicated magnetic resonance imaging examination, the neurosurgeon decides whether to qualify for the procedure. In a selected group of patients, significant improvement can be expected after surgical treatment.

Identifying and Validating an Angiogenesis-related Signature for the Prognosis of Head and Neck Squamous Cell Carcinoma.

The present study aimed todevelop a prognostic model for HNSCC treatment on the basis of angiogenesis-related signatures. Head and Neck Squamous Cell Carcinoma (HNSCC) is the most frequent malignancy with poor prognostic outcomes in the head and neck. Angiogenesis plays a critical role in tumorigenesis and is expected to be an effective therapeutic target. The RNA-seq dataset TCGA-HNSCC and the hallmark gene set were used for angiogenesis-related RiskScore model construction. The RNA-seq data was downloaded from The Cancer Genome Atlas (TCGA), and the hallmark gene set was used to measure the angiogenesis score using the GSVA R package. Then, the optimal cutoff point for prognostic classification was calculated by the survminer package, and Weighted Gene Co-expression Network Analysis (WGCNA) was used to identify angiogenesis gene modules . Multi/univariable and Lasso Cox analyses were performed to develop the RiskScore model, and the classifier efficiency was evaluated by the Receiver Operating Characteristic curve (ROC). Furthermore, a nomogram was designed for survival probability prediction, and the immune infiltration and immunotherapy differences among different risk patients were assessed. After calculating the angiogenesis score, we found that this indicator and patients' prognosis were closely correlated, especially when patients with a high angiogenesis score had a poor prognosis. Then, WGCNA identified a blue gene module positively correlated with angiogenesis. Multivariate and Lasso Cox analysis further identified 9 risk model genes for developing a RiskScore, which was used to divide low- and high- -risk groups of patients. Those with a high risk tended to show poor prognosis, immune infiltration, and higher immune escape. Finally, a nomogram was developed to optimize the risk model, and it exhibited excellent short- and long-term survival prediction performance. We constructed a reliable RiskScore model for the prognostic prediction of HNSCC patients, contributing to precise therapeutic intervention of the cancer.

Characterization of discordance between mismatch repair deficiency and microsatellite instability testing may prevent inappropriate treatment with immunotherapy.

In the Drug Rediscovery Protocol (DRUP), patients with cancer are treated based on their tumor molecular profile with approved targeted and immunotherapies outside the labeled indication. Importantly, patients undergo a tumor biopsy for whole-genome sequencing (WGS) which allows for a WGS-based evaluation of routine diagnostics. Notably, we observed that not all biopsies of patients with dMMR/MSI-positive tumors as determined by routine diagnostics were classified as microsatellite-unstable by subsequent WGS. Therefore, we aimed to evaluate the discordance rate between routine dMMR/MSI diagnostics and WGS and to further characterize discordant cases. We assessed patients enrolled in DRUP with dMMR/MSI-positive tumors identified by routine diagnostics, who were treated with immune checkpoint blockade (ICB) and for whom WGS data were available. Patient and tumor characteristics, study treatment outcomes, and material from routine care were retrieved from the patient medical records and via Palga (the Dutch Pathology Registry), and were compared with WGS results. Initially, discordance between routine dMMR/MSI diagnostics and WGS was observed in 13 patients (13/121; 11%). The majority of these patients did not benefit from ICB (11/13; 85%). After further characterization, we found that in six patients (5%) discordance was caused by dMMR tumorsthat did not harbor an MSI molecular phenotype by WGS. In six patients (5%), discordance was false due to the presence of multiple primary tumors (n = 3, 2%) and misdiagnosis of dMMR status by immunohistochemistry (n = 3, 2%). In one patient (1%), the exact underlying cause of discordance could not be identified. Thus, in this group of patients limited to those initially diagnosed with dMMR/MSI tumors by current routine diagnostics, the true assay-based discordance rate between routine dMMR/MSI-positive diagnostics and WGS was 5%. To prevent inappropriate ICB treatment, clinicians and pathologists should be aware of the risk of multiple primary tumors and the limitations of different tests. © 2024 The Pathological Society of Great Britain and Ireland.

The double burden of severe mental illness and cancer: a population-based study on colorectal cancer care pathways from screening to end-of-life care.

Cancer is one of the main causes of death in persons with severe mental illness (SMI). Although their cancer incidence is similar, or sometimes even potentially lower compared to the general population, their cancer mortality remains higher. The role of healthcare provision and care equity in this mortality is increasingly being addressed in research, but available studies are limited in their scope. In this context, our aim was to compare colorectal cancer (CRC) care pathways from screening to end-of-life care in patients with and without pre-existing SMI on a national scale. This research leverages real-world data from the French national health claims database, covering the entire population, to assess cancer screening, diagnosis, treatment and post-treatment follow-up as well as quality of care (QOC) pathways among patients with incident CRC in 2015-2018, considering whether they had pre-existing SMI. We matched patients with SMI with three patients without - on age, sex, region of residence, year of cancer incidence and cancer type and location at presentation - as well as nationally established quality of CRC care indicators and regression models adjusting for relevant socio-economic, clinical and care provider-related covariates. Among patients with incident CRC, 1,532 individuals with pre-existing SMI were matched with individuals without SMI. After adjusting for covariates, both colon and rectal cancer patients with SMI were less likely to participate in the national CRC screening programme and to receive advanced diagnostic examinations (e.g., colonoscopies and several complementary diagnostic examinations). They also had lower odds of receiving combined treatments (e.g., neoadjuvant chemotherapy, radiotherapy and excision) and of having access to targeted therapy or capecitabine but higher odds for invasive care (e.g., stoma). Colon cancer patients with SMI were also more likely to have no treatment at all, and rectal cancer patients with SMI were less likely to receive post-treatment follow-up. Suboptimal QOC was observed for both groups of patients, but to a higher extent for patients with SMI, with statistically significant differences for indicators focusing on diagnosis and post-treatment follow-up. Our findings reveal discrepancies across the care continuum of CRC between individuals with and without SMI and provide initial avenues on where to focus future efforts to address them, notably at the entry and exit stages of cancer care pathways, while calling for further research on the mechanisms preventing equity of physical healthcare for individuals with SMI.

Investigation on the treatment of empagliflozin in glycogen storage disease type Ib

Objective: To investigate the safety, efficacy and effective dose of empagliflozin in the treatment of glycogen storage disease type Ⅰb (GSD Ⅰb). Method: This was a cross sectional study. A total of 28 children with GSDⅠb who started oral empagliflozin treatment from January 2021 to June 2023 in the WeChat group of patients with glycogen storage disease were selected as the study objects. Clinical data such as general situation, current situation of medication and adverse reactions of the children were collected through questionnaires from June 18 to 30, 2023. The differences of symptoms and laboratory tests before and after empagliflozin treatment were compared by using paired chi-square test and Wilcoxon signed rank sum test. Results: Totally 28 children with GSD Ⅰb were from 12 different provinces, autonomous regions and municipalities in China. There were 14 males and 14 females, Empagliflozin treatment was started at the age of 4.8(2.4, 10.8) years, the time of treatment was 14.5(11.3, 21.5) months, the initial dosage was (0.23±0.11) mg/(kg·d), and the maintenance dosage was (0.28±0.12) mg/(kg·d). Empagliflozin showed positive effects on neutropenia, severity of inflammatory bowel disease like symptoms(Z=-3.70, -2.65, both P<0.05), The proportion of recurrent oral ulcers, recurrent bacterial infections and anemia was significantly lower than that before medication 18% (5/28) vs. (46% (13/28), 14% (4/28) vs. 46% (13/28), 21% (6/28) vs. 46% (13/28), χ²=4.05, 5.26, 3.05, all P<0.05). GCSF was once used in 5 children with GSD Ⅰb, all of them had completely stopped GCSF after empagliflozin treatment. The most common adverse events during empagliflozin treatment were hypoglycemia (5 children) and urinary infection (3 children). All 28 patients had no serious adverse reactions. Conclusions: Empagliflozin can increase the neutrophil count of children with GSD Ⅰb, and had a favorable effect on symptoms such as recurrent oral ulcers, and recurrent infection. The common adverse events during empagliflozin treatment were hypoglycemia and urinary infection.

THORACIC SEGMENTAL SPINAL ANAESTHESIA IN UPPER ABDOMINAL SURGERIES AND SIMPLE MASTECTOMY-A CASE SERIES

Objective: To observe safety and efficacy of Segmental thoracic spinal anesthesia (STSA) in upper abdominal and breast surgeries. Methods: 14 cases were selected, out of which 6 laparoscopic cholecystectomy (LC), 4 open cholecystectomy (OC), 3 emergency exploratory laparotomy and 1 simple mastectomy. Under full aseptic precaution, segmental thoracic spinal anesthesia was administered through midline approach in T8-9 space for upper abdominal surgeries and T5-6 space in a simple mastectomy. Using 25G Quincke spinal needle 1.5 ml [7.5 mg] of 0.5% Isobaric Levobupivacaine mixed with 0.4 ml (20µg) Fentanyl was given in abdominal surgeries and 1.2 ml [6 mg] of 0.5% Isobaric Levobupivacaine mixed with 0.4 ml (20µg) Fentanyl in simple mastectomy. Results: Age group of patients ranged from 25 to 65 y, Male: Female ratio of 4:10, Body Mass Index (BMI) range of 22 to 24.9 with physiological status of ASA I to ASA III. Hemodynamically, hypotension was observed in every patient after 5-10 min of STSA well managed with fluid and vasopressors with no bradycardia. Average width of sensory block was T3 to L1-2 in upper abdominal surgeries and T1 to T8 in a simple mastectomy. Motor block was Modified Bromage Scale (MBS) of 0 to 1 during and postoperatively. Regarding analgesia, Visual Analogue Scale (VAS) score was 0 intraoperatively and 0-1 postoperatively. Sedation score was 2 both intraoperatively and postoperatively with no significant complications and good patient and surgeon satisfaction. Conclusion: Segmental thoracic spinal anaesthesia is a safe and effective procedure with good patient and surgeon satisfaction.

Pharmacokinetic Variability of Rufinamide and Stiripentol in Children with Refractory Epilepsy: A Retrospective Study of Therapeutic Drug Monitoring from the National Epilepsy Centers in Denmark and Norway.

Rufinamide and stiripentol, orphan drugs used in Lennox-Gastaut and Dravet syndromes, respectively, are antiseizure medications (ASMs), often administered to children; however, pharmacokinetic studies are lacking. The authors compared the pharmacokinetic variability of these drugs with respect to the dose, serum concentrations, comedication, age, and duration of treatment. Children and adolescents (<18 years) whose serum concentrations were measured were retrospectively identified from the therapeutic drug monitoring (TDM) databases at 2 national epilepsy centers in Norway and Denmark (2012-2021). Data from 165 patients (56% boys/44% girls) treated with rufinamide and 52 patients (50% boys/50% girls) treated with stiripentol were included. For rufinamide, the median age was 10 (range 2-17) years, dose 23 (3-73) mg/d, and serum concentration 34 (3-227) µmol/L [8.1 mg/L (0.71-54.0 mg/L)]. For stiripentol, the median age was 8.5 (range 1-17) years, dose 37 (18-76) mg/d, and serum concentration 33 (4-113) µmol/L [7.7 mg/L (0.93-26.3 mg/L)]. The concomitant use of 1-9 other ASMs during the data collection was noted. Pharmacokinetic variability, calculated as the concentration/(dose/kg) ratio, ranged from 0.26 to 11.31 (µmol/L)/(mg/kg) for rufinamide and 0.17-1.52 (µmol/L)/(mg/kg) for stiripentol. The intraindividual coefficients of variation ranged widely, from 5% to 110% for rufinamide and 11%-117% for stiripentol. The treatment period was at least 5 years in 50% of patients. No statistically significant effects of age, sex, or ASM comedication were observed, possibly due to the small sample size and heterogeneous groups with variable seizure situations, comorbidities, and changes in comedication and physiology. This study demonstrates considerable pharmacokinetic variability in and between patients for both drugs and similar use in terms of age, burden of comedication and retention rates. TDM may be useful in the clinical setting to monitor and optimize treatment in this vulnerable patient group.

Defining Benchmarks for Pelvic Exenteration Surgery: A Multicentre Analysis of Patients with Locally Advanced and Recurrent Rectal Cancer.

To establish globally applicable benchmark outcomes for pelvic exenteration (PE) in patients with locally advanced primary (LARC) and recurrent rectal cancer (LRRC), using outcomes achieved at highly specialised centres. PE is established as the standard of care for selected patients with LARC and LRRC. There are currently no available benchmarks against which surgical performance in PE can be compared for audit and quality improvement. This international multicentre retrospective cohort study included patients undergoing PE for LARC or LRRC at 16 highly experienced centres between 2018 and 2023. Ten outcome benchmarks were established in a lower-risk subgroup. Benchmarks were defined by the 75th percentile of the results achieved at the individual centres. 763 patients underwent PE, of which 464 patients (61%) had LARC and 299 (39%) had LRRC. 544 patients (71%) who met predefined lower risk criteria formed the benchmark cohort. For LARC patients, the calculated benchmark threshold for major complication rate was ≤44%; comprehensive complication index (CCI): ≤30.2; 30-day mortality rate: 0%; 90-day mortality rate: ≤4.3%; R0 resection rate: ≥79%. For LRRC patients, the calculated benchmark threshold for major complication rate was ≤53%; CCI: ≤34.1; 30-day mortality rate: 0%; 90-day mortality rate: ≤6%; R0 resection rate: ≥77%. The reported benchmarks for PE in patients with LARC and LRRC represent the best available care for this patient group globally and can be used for rigorous assessment of surgical quality and to facilitate quality improvement initiatives at international exenteration centres.

Clinical and echocardiographic characterization of patients hospitalized for severe tricuspid valve regurgitation: a single tertiary-care center experience with two-year follow-up.

Tricuspid regurgitation (TR) is a common acquired valvular heart disease. Recently new transcatheter treatment options for severe TR have emerged that could change the management of this condition, which warrants better characterization of this specific patient group. The aim of the study was to describe the clinical and echocardiographic characteristics of patients with severe TR and to evaluate their short- and mid-term prognosis. This retrospective, observational single-center study enrolled consecutive patients with severe TR hospitalized between January 2016 and September 2021 in the 1st Chair and Department of Cardiology, Medical University of Warsaw, Poland. The severity of heart failure (HF) was evaluated using New York Heart Association (NYHA) classification. Echocardiographic assessment was performed by experienced sonographer. EuroSCORE II and TRI-SCORE were computed for each patient. 12- and 24-month clinical outcomes were reported. There were 172 patients [93 female (54.1%)] included. The mean age was 76.4 (± 10.5) years. The most common comorbidities included: atrial fibrillation (84.9%), hypertension (68.0%), chronic kidney disease (54.1%), coronary artery disease (45.3%), diabetes mellitus (30.9%). The median EuroSCORE II and TRI-SCORE values were 4.68% and 14.0%, respectively. The median follow-up was 24 months. The overall mortality was 29.7% at 1 year and 47.3% at 2 years. TR grade, TAPSE <17 mm, TAPSE/SPAP <0.26 and right atrial area were significant factors associated with mortality. Patients presenting with severe TR are characterized by a large comorbidity burden and poor prognosis, despite intensive heart failure management.

Evaluating the psychometric properties of the simplified Chinese version of PROMIS-29 version 2.1 in patients with hematologic malignancies

The Patient-Reported Outcomes Measurement Information System 29-item Profile version 2.1 (PROMIS-29 V2.1) is a widely utilized self-reported instrument for assessing health outcomes from the patients’ perspectives. This study aimed to evaluate the psychometric properties of the PROMIS-29 V2.1 Chinese version among patients with hematological malignancy. Conducted as a cross-sectional, this research was approved by the Medical Ethical Committee of the Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College (registration number QTJC2022002-EC-1). We employed convenience sampling to enroll eligible patients with hematological malignancy from four tertiary hospitals in Tianjin, Shandong, Jiangsu, and Anhui province in China between June and August 2023. Participants were asked to complete a socio-demographic information questionnaire, the PROMIS-29 V2.1, and the Functional Assessment of Cancer Therapy-General (FACT-G). We assessed the reliability, ceiling and floor effects, structural, convergent discriminant and criterion validity of the PROMIS-29 V2.1. A total of 354 patients with a mean age of 46.93 years was included in the final analysis. The reliability of the PROMIS-29 V2.1 was affirmed, with Cronbach’s α for the domains ranging from 0.787 to 0.968. Except sleep disturbance, the other six domains had ceiling effects, which were seen on physical function (26.0%), anxiety (37.0%), depression (40.4%), fatigue (18.4%), social roles (18.9%) and pain interference (43.2%), respectively. Criterion validity was supported by significant correlations between the PROMIS-29 V2.1 and FACT-G scores, as determined by the Spearman correlation test (P < 0.001). Confirmatory factor analysis (CFA) indicated a good model fit, with indices of χ2/df (2.602), IFI (0.960), and RMSEA (0.067). The Average Variance Extracted (AVE) values for the seven dimensions of PROMIS-29 V2.1, ranging from 0.500 to 0.910, demonstrated satisfactory convergent validity. Discriminant validity was confirmed by ideal √AVE values. The Chinese version of the PROMIS-29 V2.1 profile has been validated as an effective instrument for assessing symptoms and functions in patients with hematological malignancy, underscoring its reliability and applicability in this specific patient group.

Modifications to Enhance Outcomes of Family-Based Treatment for Anorexia Nervosa: A Scoping Review

Family-based treatment (FBT) is recommended for anorexia nervosa (AN) in young people (YP). However, a substantial proportion of YP undergoing FBT do not recover. Several modifications to standard FBT have been tested to improve recovery rates. This review provides an updated overview of empirically tested modifications to FBT for AN in YP and estimates whether such modifications increase the percentage recovering. Computerized searches performed in five databases resulted in the inclusion of 43 papers (representing 40 original studies), highlighting that a variety of modifications to standard FBT have been tested and appear promising. However, only 11 studies compared the results of a modification to standard FBT. In conclusion, some modifications, such as parent-focused treatment, the addition of home treatment, or interventions for families at risk of non-response, appear to have the potential to improve the recovery rate, either at the group or subgroup level. Other modifications, such as FBT-based guided self-help, virtually delivered FBT, or FBT delivered during in- or day-patient stays, enable the dissemination of FBT principles to other contexts and to patient groups with limited access. Small additions to FBT, such as a workshop or parent-to-parent consultation, do not seem to improve the recovery rate.

Protocol for the 'Supporting Young Cancer Survivors who Smoke' study (PRISM): Informing the development of a smoking cessation intervention for childhood, adolescent and young adult cancer survivors in England.

Childhood, adolescent and young adult (CAYA) cancer survivors are vulnerable to adverse late-effects. For CAYA cancer survivors, tobacco smoking is the most important preventable cause of ill-health and early death. Yet, effective strategies to support smoking cessation in this group are lacking. The PRISM study aims to undertake multi-method formative research to explore the need for, and if appropriate, inform the future development of an evidence-based and theory-informed tobacco smoking cessation intervention for CAYA cancer survivors. PRISM involves three phases of: 1) an environmental scan using multiple strategies to identify and examine a) smoking cessation interventions for CAYA cancer survivors that are published in the international literature and b) current smoking cessation services in England that may be available to, or tailorable to, CAYA cancer survivors; 2) a qualitative study involving semi-structured interviews with CAYA cancer survivors (aged 16-29 years and who are current or recent ex-smokers and/or current vapers) to explore their views and experiences of smoking, smoking cessation and vaping; and 3) stakeholder workshops with survivors, healthcare professionals and other stakeholders to consider the potential for a smoking cessation intervention for CAYA cancer survivors and what such an intervention would need to target and change. Findings will be disseminated to patient groups, healthcare professionals and researchers, through conference presentations, journal papers, plain English summaries and social media. PRISM will explore current delivery of, perceived need for, and barriers and facilitators to, smoking cessation advice and support to CAYA cancer survivors from the perspective of both survivors and healthcare professionals. A key strength of PRISM is the user involvement throughout the study and the additional exploration of survivors' views on vaping, a behaviour which often co-occurs with smoking. PRISM is the first step in the development of a person-centred, evidence- and theory-based smoking cessation intervention for CAYA cancer survivors who smoke, which if effective, will reduce morbidity and mortality in the CAYA cancer survivor population.

Obesity, liver steatosis and metabolic syndrome: The hidden enemies in transfusion-dependent thalassaemia.

In their paper, the authors quantified liver iron concentration (LIC) and hepatic steatosis (HS) using MRI-T2* technology in transfusion-dependent thalassaemia (TDT) patients and healthy controls and found that the prevalence of HS among patients with TDT was 36.4%. In comparison with healthy controls, the hepatic fat fraction (FF) was significantly higher in the TDT population (p = 0.013). Active hepatitis C virus infection, body mass index (BMI) and LIC were independent predictors of HS. An inverse correlation between hepatic FF and high-density lipoprotein cholesterol (p = 0.042) and a significant association of high glycaemia level (p = 0.037) with higher hepatic FF and a significant relationship (p = 0.026) between HS and higher BMI (though in a 'lean' group of patients) in TDT patients indicated that 'metabolic syndrome' was present in this subset with TDT. The impact of metabolic syndrome on TDT, including cardiac disease unrelated to iron overload, needs further study. Commentary on: Ricchi etal. Liver steatosis in patients with transfusion-dependent thalassaemia. Br J Haematol 2024 (Online ahead of print). doi: 10.1111/bjh.19496.

The volume of steal phenomenon is associated with neurological deterioration in patients with large-vessel occlusion minor stroke not eligible for thrombectomy.

A significant number of patients who present with mild symptoms following large-vessel occlusion acute ischemic stroke (LVO-AIS) are currently considered ineligible for EVT. However, they frequently experience neurological deterioration during hospitalization. This study aimed to investigate the association between neurological deterioration and hemodynamic impairment by assessing steal phenomenon derived from blood oxygenation-level dependent cerebrovascular reactivity (BOLD-CVR) in this specific patient cohort. From the database of our single-center BOLD-CVR observational cohort study (June 2015-October 2023) we retrospectively identified acute ischemic stroke patients with admission NIHSS < 6, a newly detected large vessel occlusion of the anterior circulation and ineligible for EVT. Neurological deterioration during hospitalization as well as outcome at hospital discharge were rated with NIHSS score. We analyzed the association between these two outcomes and BOLD-CVR-derived steal phenomenon volume through regression analysis. Additionally, we investigated the discriminatory accuracy of steal phenomenon volume for predicting neurological deterioration. Forty patients were included in the final analysis. Neurological deterioration occurred in 35% of patients. In the regression analysis, a strong association between steal phenomenon volume and neurological deterioration (OR 4.80, 95% CI 1.32-31.04, p = 0.04) as well as poorer NIHSS score at hospital discharge (OR 3.73, 95% CI 1.52-10.78, p = 0.007) was found. The discriminatory accuracy of steal phenomenon for neurological deterioration prediction had an AUC of 0.791 (95% CI 0.653-0.930). Based on our results we may distinguish two groups of patients with minor stroke currently ineligible for EVT, however, showing hemodynamic impairment and exhibiting neurological deterioration during hospitalization: (1) patients exhibiting steal phenomenon on BOLD-CVR imaging as well as hemodynamic impairment on resting perfusion imaging; (2) patients exhibiting steal phenomenon on BOLD-CVR imaging, however, no relevant hemodynamic impairment on resting perfusion imaging. The presence of BOLD-CVR derived steal phenomenon may aid to further study hemodynamic impairment in patients with minor LVO-AIS not eligible for EVT.

Transcranial electrical stimulation (TES) in patients with atopic dermatitis

BACKGROUND: Atopic dermatitis is the most common diseases in dermatology, among the main pathogenetic mechanisms of development of which a complex interaction of a genetically determined defect in the barrier function of the skin, an overactive state of the immune system is considered, and a neurogenic theory of disease development is not excluded, which is based on the relationship of anxiety-stress reactions and neuropsychiatric stresses with the occurrence of pathological changes on the skin. Modern atopic dermatitis therapy is characterized by the appointment of a large number of medications according to clinical recommendations, and the associated increase in allergic and toxic-allergic reactions dictates the need to search for new non-drug treatments for this disease. In order to prevent the pathological effects of stress, as well as to increase resistance to its damaging effects, activation of opioidergic mechanisms may be useful. AIM: To evaluate the effectiveness and acceptability of the transcranial electrical stimulation technique in patients with atopic dermatitis. MATERIALS AND METHODS: A controlled study was conducted among 60 patients with moderate atopic dermatitis, 33 women (55.0%) and 27 men (45.0%) aged 19 to 72 years. Transcranial electrical stimulation was carried out by a pulsed bipolar electrical stimulator with an acoustic effect "TRANSAIR-07", stimulation was carried out by an electric current with a frequency of 77.5 Hz and a duration of 3.5 ms. Patients underwent 10 procedures, 1 time a day, lasting 30 minutes, with a current of 1−3 mA. SCORAD, EASI, IGA, DLQI, HADS, and BDI scales were used to assess the dynamics of clinical manifestations. The assessment was carried out on the first and last day of treatment. RESULTS: During statistical data processing, when comparing the scales in the first and last days of the procedures, depending on the patient groups: the 1st group was the main one, received transcranial electrical stimulation treatment in combination with therapy, according to clinical recommendations, the 2nd group was the control group, received treatment according to clinical recommendations, without the use of transcranial electrical stimulation, significant improvement of itching indicators, clinical manifestations and improvement of quality of life after 4 weeks of therapy in both groups. A statistically more significant dynamics of the indicators of the main clinical scales in favor of combination therapy was established: according to the SCORAD scale ― in the main group from 47.50±2.25 to 21.00±1.00, in the control group from 47.00±2.00 to 39.00±1.75; DLQI ― in the main group from 8.00±2.00 to 2.00±0.75, in the control group ― from 8.50±1.50 to 5.00±1.00. CONCLUSION: The paper presents the positive results of our own research and presents data confirming the clinical effectiveness of transcranial electrostimulation in patients with atopic dermatitis compared with the traditional drug treatment of this disease, which allows us to recommend the treatment regimen we have proposed for widespread implementation in the practice of a dermatovenerologist.

The changes in fear of childbirth in pregnancy during and before the COVID-19 pandemic

We aimed to investigate how factors such as age, education level, planned delivery method and fear of childbirth were affected in pregnant women before and during the pandemic. This cross-sectional study compared a pre-pandemic pregnant group (July 2019 and December 2019) and a pandemic group (November 2020 and May 2021) of patients at Kütahya Health Sciences University Evliya Çelebi Training and Research Hospital. A total of 696 pregnant women in their second trimester were included in the study. All of them were literate and voluntarily agreed to participate in the study. Data were collected with the Wijma delivery expectancy/experience questionnaire (WDEQ-A), and the outpatient doctor asked the questions face-to-face. The mean age of the pregnant women participating in the study was 31.6 ± 6.8 years. While the total Wijma score was 62.1 ± 25.1 in the pre-pandemic group, it was 61.3 ± 26.4 in the pandemic group, and there was no significant difference between the two groups (p = 0.738). Upon analyzing the fear of childbirth among groups based on education level, no statistically significant differences were observed between the pre-pandemic and pandemic periods within any of the groups. While 25.7% (n = 179) of all participants had a normal fear of childbirth, 22% (n = 153) had a mild fear of childbirth, 27% (n = 188) had a moderate fear of childbirth, and 25.3% (n = 176) had a severe fear of childbirth (Wijma score of 85 and above). When the pre-pandemic and the pandemic period were compared, the fear of childbirth was unchanged in pregnant women at all education levels (p = 0.079, p = 0.957, p = 0.626, p = 0.539, p = 0.202). When comparing fear of childbirth before and after the pandemic, it was found that patients with a high school education level have a significantly higher fear of childbirth. To alleviate the fear of childbirth in pregnant women who have completed high school, training or psychosocial support interventions may be prioritized.