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A fixed-dose combination of calcipotriene 0.005%/betamethasone dipropionate 0.064% (Cal/BD) aerosol foam (Enstilar, LEO Pharma) is the only topical therapy approved for the acute (reactive) and proactive management of psoriasis. Although treatment with Cal/BD foam has been characterized in a clinical context, further evidence is needed to determine its optimal use in clinical practice. A group of experts discussed the value of the Cal/BD foam as a topical treatment for mild-to-moderate psoriasis in combination with systemic treatments. The reported experiences support effectiveness of the Cal/BD foam in daily clinical practice, with an improvement in patient quality of life.

Purpose: To evaluate the efficacy of systemic tumor necrosis factor-alpha inhibitors (TNFi) in the treatment of non-infectious uveitis (NIU). Methods: This Swiss multicenter retrospective cohort study included patients with NIU requiring TNFi during the period from 2001 to 2018. Risk factors for the occurrence of new complications were identified using Cox regression analysis and hazard ratios (HR). Results: Seventy-one patients (126 eyes; mean age 40.6 ± 14.4 years, mean duration of uveitis 46.0 ± 61.8 months) were followed for 40.2 ± 17.3 months after addition of TNFi. Under TNFi, visual acuity improved from 0.2 ± 0.3 to 0.1 ± 0.3 logMAR (p < 0.001). The portion of patients under systemic corticosteroids decreased from 81.7% to 25.4% (p < 0.001), while that for conventional synthetic disease-modifying anti-rheumatic drugs insignificantly decreased from 63.4% to 50.7% (p > 0.05). In 80.2% of eyes, complications were present at baseline with epiretinal gliosis (39.7%), cataract (41.3%) and macular edema (ME; 27.8%) being the most common. New complications under TNFi were encountered in 49.2% of eyes, also including recurrence (5 eyes) or new onset of ME (14 eyes). The need for switching of TNFi was associated with further complications (HR 3.78, p = 0.012). Conclusion: Although the efficacy and tolerability of TNFi in a real-life setting are favorable, treatment is often initiated late, i.e., after many eyes have already developed complications. Even with TNFi, new complications, particularly ME, cannot be completely avoided. Further research is needed to assess the impact of earlier initiation of TNFi therapy. [ABSTRACT FROM AUTHOR]

Introduction: Smart healthcare technologies (SHCTs) exhibit the great potential to support older Hong Kong adults with their health problems. Although there are various SHCTs in the Hong Kong market, and some adoption predictors have been proposed and investigated, little is known about older users' views on and real-life experiences with these technologies. This exploratory study examined the experiences, functional needs, and barriers of three kinds of SHCT (i.e., smart wearable devices, smart health monitors, and healthcare applications) with older adults in real life. Methods: A convenience sampling method was applied to recruit twenty-two older adults from the Hong Kong community. The interview was designed in semi-structured and conducted in a face-to-face setting. The content analysis was used to summarize the older adults' functional needs and barriers in real life. Results: We found older adults mainly applied SHCTs to address physical health, but there are few technological solutions for mental health in practice. There are four types of barriers in using SHCT. However, social support in Hong Kong community greatly helps reduce the barriers in technology use. Based on the findings, we discussed the possible solutions based on the social and technology perspective. Conclusion: Current technologies still could not fully address older adults' needs for healthy aging, and various barriers still hinder the actual adoption. By deeply understanding and considering the social context, technology innovation can facilitate the adoption of SHCT and promote a healthy aging society. [ABSTRACT FROM AUTHOR]

Individuals who cope well with challenges may engage in social situations more successfully. We examined the association between coping behaviors, self-perceived competence, and social anxiety in everyday social situations. A total of 133 participants between the ages of 10 and 16 years (82 autistic, 51 neurotypical) carried a mobile device that prompted them seven times each day for 7 days to record what they were doing and their perceived competence and social anxiety in that situation. We used the Coping Inventory to measure overall coping abilities (i.e. coping with the self and the environment). Multilevel analyses found that autistic adolescents were more likely than neurotypical peers to experience social anxiety while engaged in maintenance or productive activities with adults. While their ability to cope with the self was positively associated with perceived social competence, it was negatively associated with the ability to cope with environmental challenges. Furthermore, autistic adolescents with poor ability to cope with environmental challenges were more likely than those who coped well to be socially anxious when engaged in leisure activities with peers. Findings from this study can guide evaluation and intervention to improve the social experiences of autistic adolescents by helping them increase coping skills. Individuals who cope well with challenges may engage in social situations more successfully. We examined how well autistic adolescents coped, depending on how competent they felt and how much anxiety they experienced during social activities. We included 133 individuals (82 autistic, 51 neurotypical) between the ages of 10 and 16 years. Participants carried a mobile device that prompted them seven times a day for 7 days to record what they were doing, how competent they felt and how much anxiety they experienced. We used the Coping Inventory to understand how well participants coped with environmental challenges and met their needs for growth. Autistic adolescents were more likely than neurotypical peers to feel anxious while doing activities with adults. Autistic adolescents who had more difficulty coping with challenges were more likely to feel anxious when doing leisure activities with peers. Interestingly, autistic adolescents who coped better with challenges tended to feel less competent in social situations. However, those better able to meet their needs for growth tended to perceive their social competence positively. These findings can help practitioners develop strategies and programs to reduce the negative social experiences of autistic adolescents by helping them cope better. [ABSTRACT FROM AUTHOR]

Aim: Rheumatoid arthritis (RA) poses a significant health challenge and is characterized by chronic immune-mediated inflammation and potential joint damage. This study explores the real-life effectiveness of baricitinib, a Janus kinase inhibitor, in treating patients with RA. The goal of this study was to assess its impact on disease activity and factors influencing treatment outcomes. Material and Methods: Ninety patients with RA diagnosed between September 2021 and 2023 at Necmettin Erbakan University Meram Medical Faculty Hospital were retrospectively analyzed. Baricitinib, prescribed during this period, was evaluated for its impact on C-reactive protein (CRP), Disease Activity Score-28 with CRP (DAS28/CRP), and overall treatment continuation rates. Demographic and clinical data, including rheumatoid factor (RF) and anti-cyclic citrullinated peptide (anti-CCP) markers, were collected. Results: Significant reductions in CRP and DAS28/CRP levels were observed over a 12-week follow-up after baricitinib use. Positive detection rates for RF and anti-CCP were 60% and 57.8%, respectively. Baricitinib demonstrated a high continuation rate (82%) at an average of 9.37 months. No significant differences were found in the continuation rates based on the prior use of conventional or biological disease-modifying anti-rheumatic drugs (DMARDs). Conclusion: Comparisons with existing studies support the efficacy of baricitinib in improving disease activity. Our findings align with the literature, emphasizing positive outcomes in patients with prior DMARD experience. Unlike some studies reporting higher discontinuation risks, our results highlight a favorable safety profile. The study's limitations include a short follow-up period, which warrants further investigation with larger cohorts. In conclusion, baricitinib exhibits promising real-life effectiveness in RA treatment, emphasizing its role as a valuable therapeutic option. [ABSTRACT FROM AUTHOR]

BACKGROUND: Topical therapies represent the first-line treatment for mild-to-moderate psoriasis. Among various topical options, the fixed-dose combination of calcipotriene (Cal) and betamethasone dipropionate (BD) foam (Enstilar®, LEO Pharma, Ballerup, Denmark) showed superior efficacy to Cal and BD monotherapy and ointment and gel formulations. In addition, the Cal/BD foam is the only topical treatment allowed for either reactive treatment of relapse or twice-weekly maintenance use. Since treatment acceptability is crucial to optimize adherence, this paper presents a case series from a multicenter experience using the Cal/BD foam, to further characterize the use of this therapeutic approach. In addition, a narrative review of studies evaluating the acceptability of the Cal/BD foam, even compared with other formulations, is provided. CASE SERIES: The case series involved adult patients with mild-to-moderate psoriasis treated with the Cal/BD foam from October 2021 to June 2022. A clinical and dermoscopic evaluation of plaques was provided for all patients. Data from the clinical practice report complete clinical resolution of plaques in most patients after 4 weeks of active treatment with the Cal/BD foam, and the dermoscopic clearance after a maximum of 8 weeks. Full adherence to treatment was also reported. Literature evidence suggests that the Cal/BD foam is easy to apply and presents high cosmetic acceptance, rapid onset of action, high efficacy, optimal safety, and a high patient preference. The high satisfaction obtained with Cal/BD foam suggests that this formulation is better accepted than others. CONCLUSIONS: The Cal/BD foam represents a valuable approach for managing mild-to-moderate psoriasis, both in short and long-term treatment. [ABSTRACT FROM AUTHOR]

Numerous literature reviews highlight a considerable volume of quantitative publications in the field of sports coach behavior. While qualitative approaches on real-life experiences of coaches are less common, they help to comprehend the intricacies of high-performance sports. Examining coaches' experiences in a professional setting through the 'enactive phenomenology framework' proves particularly intriguing, because this approach aims to elucidate behaviors within dual practical and meaningful dimensions. This case study aimed to provide insight into the practices of experienced rhythmic gymnastics coaches by observing their typical activities during training sessions in competitive periods. The objective is to elucidate their interactions with both the environment and gymnasts, shedding light on how they shape their roles within a specific context: the domain of gymnastics and artistic sports, predominantly featuring female participants. Such access should help to better understand how rhythmic gymnastics coaches act and interact with gymnasts during training in order to improve learning and coaching. Four coaches participated in this study. The theoretical framework used was enactive phenomenology. Data sources included observations and audiovisual recordings of the coaches' behaviour and communication, and enactive interviews conducted a posteriori with the coaches. Our findings highlight four typical rhythmic gymnastics coaches' actions (guiding, checking, encouraging and pacing) which are closely linked to the time and space invested over the course of training. This case study demonstrates the existence of recurring features in the way rhythmic gymnastics coaches interact with gymnasts. The strategies identified carry a meaning for the coaches that is anchored in the desire to both help and control the gymnasts. All of these actions are continuously adjusted during the training session, depending on the coach's perceptions of the gymnasts and the choreography, as well as her own feelings. This study offers a reflection on the practices of rhythmic gymnastics coaches in order to understand their intrinsic logical sequences, which would allow a modernisation of professional practices and questioning of training approaches, with a focus on the real experience of the actors. [ABSTRACT FROM AUTHOR]

Background and Aim: The aims of the present study were to evaluate the real-life efficacy and tolerability of glecaprevir (GLE)/pibrentasvir (PIB) in the treatment of patients with chronic hepatitis C (CHC). Materials and Methods: Between May 2019 and May 2022, 686 patients with CHC, treated with GLE/PIB combination from 21 participating centers in Turkiye, were enrolled in the study. Results: All patients were Caucasian, and their median age was 56 years. At the start of GLE/PIB treatment, the median serum Hepatitis C virus RNA and serum alanine amino transaminase (ALT) levels were 6.74 log10 IU/mL and 47 U/L, respectively. Fifty-three percent of the patients were infected with genotype 1b, followed by genotype 3 (17%). Diabetes was the more common concomitant disease. The sustained virological response (SVR12) was 91.4% with intent-to-treat analysis and 98.5% with per protocol analysis. The SVR12 rates were statistically significant differences between the patients who were i.v. drug users and non-user (88.0% vs. 98.8%, p=0.025). From the baseline to SVR12, the serum ALT levels and Model for End-Stage Liver Disease score were significantly improved (p<0.001 and p=0.014, respectively). No severe adverse effect was observed. Conclusion: GLE/PIB is an effective and tolerable treatment in patients with CHC. [ABSTRACT FROM AUTHOR]

Background: The correction of iron deficiency (ID) with ferric carboxymaltose (FCM) is a recommended intervention in heart failure (HF) with reduced ejection fraction. Our aim is to evaluate, in a real-life setting, the clinical significance of ID screening and FCM treatment in acute decompensated HF (ADHF). Methods: In a cohort of ADHF patients, the prevalence of ID and FCM administration were investigated. Among the 104 patients admitted for ADHF, in n = 90 (median age 84, 53.5% with preserved left ventricular ejection fraction—LVEF), a complete iron status evaluation was obtained. ID was detected in n = 73 (81.1%), 55 of whom were treated with in-hospital FCM. The target dose was reached in n = 13. Results: No significant differences were detected in terms of age, sex, comorbidities, or LVEF between the FCM-supplemented and -unsupplemented patients. During a median follow-up of 427 days (IQR 405–466) among the FCM-supplemented patients, only 14.5% received FCM after discharge; the mortality and rehospitalizations among FCM-supplemented and -unsupplemented patients were similar (p = ns). In a follow-up evaluation, ID was still present in 75.0% of the FCM-supplemented patients and in 69.2% of the unsupplemented patients (p = ns). Conclusions: In this real-life ADHF cohort, FCM was administered at lower-than-prescribed doses, thus having no impact on ID correction. The significance of our findings is that only achieving the target dose of FCM and pursuing outpatient treatment can correct ID and produce long-term clinical benefits. [ABSTRACT FROM AUTHOR]

Purpose: To present our real-life experience with dabrafenib and trametinib (D-T) treatment in patients with BRAF V600E-mutated ATC in Argentina. Patients y methods: We included five patients from four different hospitals. The median age was 70 years, and 60% were male. The performance status at diagnosis was grade 0 in 60% and grade 2 in 40% of patients. Four patients could undergo total thyroidectomy; in one of them, surgical treatment was amenable due to the indication of D-T as neoadjuvant therapy. From the total cohort, the best response to treatment was complete response in 40%, partial response in 20%, and stable disease in 20%. The median duration of response was 20 weeks, ranging from 16 to 92 weeks. All patients experienced at least one adverse event (AE). Grade ≥3 AEs were observed in two (40%) patients. They were upper gastrointestinal bleeding and subclavian vein thrombosis. The median follow-up was 20 weeks (range: 16 to 92). Conclusion: This report contributes to illustrate the feasibility and effectiveness of D-T treatment in five patients with loco-regionally advanced and metastatic BRAF V600E-mutated ATC in a real-life setting. A multidisciplinary approach and rapid molecular-tailored testing are essential to begin this therapeutic option. [ABSTRACT FROM AUTHOR]

The present multi-center, long-term, real-life study made an attempt to assess the efficacy of risankizumab in the treatment of moderate-to-severe plaque psoriasis. The study comprised 185 patients from 10 Polish dermatologic departments undergoing risankizumab treatment. The disease severity was measured using the Psoriasis Area and Severity Index (PASI) before the start of the risankizumab treatment and next at the defined timepoints, i.e., 4, 16, 28, 40, 52 and 96 weeks of treatment. The percentage of patients achieving PASI90 and PASI100 responses as well as the PASI percentage decrease at the defined timepoints were calculated, and correlations with clinical characteristics and therapeutic effect were analyzed. The number of patients evaluated at the defined timepoints was: 136, 145, 100, 93, 62, and 22 at 4, 16, 28, 40, 52 and 96 weeks of treatment, respectively. At 4, 16, 28, 40, 52 and 96 weeks, the PASI90 response was achieved in 13.2%, 81.4%, 87.0%, 86.0%, 88.7% and 81.8% of patients, whereas the PASI100 response was achieved in 2.9%, 53.1%, 67.0%, 68.8%, 71.0% and 68.2% of patients, respectively. Our study revealed a significant negative correlation between a decrease in the PASI and the presence of psoriatic arthritis as well as the patient's age and duration of psoriasis at several timepoints throughout the observation period. [ABSTRACT FROM AUTHOR]

Background: The PedsQL is widely used to retrospectively evaluate quality of life (QoL) in autistic adolescents. However, concerns have arisen regarding its ability to reflect real-time QoL, considering their challenges in recollecting past experiences., Objective: We examined the overall and social QoLs of autistic adolescents compared to neurotypical peers using the PedsQL and the experience sampling method (ESM), an ecological momentary assessment of QoL in real-time. Additionally, we explored the relationship between these measures in each group., Methods: A total of 175 participants, including 117 autistic and 58 neurotypical adolescents aged 10-16, completed the PedsQL and an ESM protocol with a mobile device to record their real-time experiences seven times a day for seven days. We performed multiple linear regression and multilevel analyses to compare QoLs between groups and the association between the two measures., Results: Autistic adolescents scored significantly lower than neurotypical peers on PedsQL overall and social QoL but not on the real-time experiences collected with ESM. Among neurotypical adolescents, we found significant associations between the Social Functioning score of the PedsQL and various aspects of real-time social experiences recorded with ESM. For autistic adolescents, only the real-time experience of 'loneliness' during social engagement was associated with Social Functioning on the PedsQL., Conclusions: The retrospective PedsQL does not entirely capture the real-time QoL via ESM. However, relying solely on ESM may overlook situations where participants opt out or could not complete surveys. Thus, using both retrospective and real-time assessments to examine QoL among autistic adolescents is recommended., Competing Interests: Declaration of competing interest The author(s) declared no potential conflicts of interest with respect to the research, authorship, and/or publication of this article., (Copyright © 2024. Published by Elsevier Inc.)

Background: Belimumab was the first biological drug approved for Systemic Lupus Erythematosus (SLE). There is not a review focusing on all real-life experience with belimumab to date that could help to describe how this drug behaves in the Spanish clinical setting. Objective: To describe the characteristics of SLE patients treated with belimumab added to standard of care in real-clinical setting in Spain. Methods: We conducted a comprehensive scoping review of real-world data (RWD) according to PRISMA Scoping Reviews Checklist and the framework proposed by Arksey and O'Malley. PubMed and EMBASE were searched without language restriction and hand searches of relevant articles were examined. Results: We included data from 222 patients treated with belimumab for SLE included in 19 RWD studies conducted in Spain. The mean age was 40.9 years, 84.2% were female, and baseline scores SELENA-SLEDAI ranged between 5.9 and 12. Lupus nephritis basal prevalence was of 2.7%. The main reason for belimumab initiation was previous treatments lack of efficacy (69.7%) and the most common laboratory abnormalities were hypocomplementemia (40.9%), ANA + (34.2%), and anti-DNA (33.3%). The addition of belimumab to standard therapy was associated with a reduction of daily glucocorticoids intake in 1.4–11.1 mg at 6 months. Belimumab discontinuation was observed in 18.6% of patients. Conclusion: Our study helps to further explore the profile of SLE patients most likely to be treated with belimumab. Key Points • Scientific evidence in SLE provided by randomized controlled trials sometimes differs from the actual treatment of SLE patients in routine clinical practice. • There is a lack of published "real-world" data on SLE treatment with belimumab in Spain. • This scoping review intends to describe and analyze the clinical characteristics of SLE patients receiving belimumab in a real-life setting in Spain. • These "real-world" clinical experience can provide a more realistic view of the overall patterns of SLE care compared with clinical trials. [ABSTRACT FROM AUTHOR]

Simple Summary: Intensive induction strategies are rarely used for older patients in community on-cology practice, with comorbidities being the major cause of contraindication. We conducted a multicentric retrospective study to evaluate activity and safety in a real-life setting of hypomethylating drugs (HMAs) in patients older than 75 years with AML. In multivariate analysis, age (≥80), Charlson comorbidity index (≥3), creatinine clearance and the type of best response (≥PR) during treatment maintained independent significance in predicting survival. Furthermore, our data show that HMAs have similar efficacy compared to pivotal trials and are well tolerated in a setting of very elderly patients with several co-comorbidities. Elderly patients represent the most challenging and hard-to-treat patient population due to dismal characteristics of the disease, such as secondary-acute myeloid leukemia (AML), enrichment of unfavorable molecular genes (TP53) and comorbidities. We conducted a multicentric retrospective study to evaluate activity and safety in a real-life setting of hypomethylating drugs (HMAs) in patients older than 75 years with AML. Between September 2010 and December 2021, 220 patients were treated, 164 (74.5%) received AZAcitidine and 56 DECitabine; most patients (57.8%), received more than four cycles of HMAs. The best response obtained was CR in 51 patients (23.2%), PR in 23 (10.5%) and SD in 45 (20.5%); overall transfusion independence was obtained in 47 patients (34%), after a median of 3.5 months. The median OS (mOs) was 8 months (95% CI 5.9–10.2), with 1- and 2-years OS of 39.4% (95% CI 32.7–46) and 17.4% (95% CI 11.7–23.1), respectively; similar mOS was observed according to HMA treatment (AZA 8.3 vs. DEC 7.8 months, p = 0.810). A subset of 57 long survivors (44 in AZA group and 13 in DEC group) received at least 12 cycles of HMAs, their mOS was 24.3 months. In multivariate analysis, age (≥80), Charlson comorbidity index (≥3), creatinine clearance and the type of best response (≥PR) during treatment maintained independent significance in predicting survival. Infectious complications, most frequently pneumonia (35) and septic shock (12), were lethal in 49 patients (22.2%). Our data show that HMAs have similar efficacy compared to pivotal trials and are well tolerated in a setting of very elderly patients with several co-comorbidities. [ABSTRACT FROM AUTHOR]

Background: COVID-19 (coronavirus disease 2019) outbreak has spread rapidly around the world, continues to show its effect, and it is not clear how long it will continue. For the diagnosis of COVID-19, it is important to ensure the comfort of the patients and to protect the healthcare workers (HCWs) by reducing the use of protective equipment. Aims: To evaluate or assess whether the samples taken by the patient for COVID-19 testing during this pandemic period can be used in real-life experience. Methods: Three different samples (nasopharyngeal taken by the healthcare worker, nasopharyngeal, and saliva taken by the patient) from 132 patients were evaluated for the diagnosis of COVID-19. The sensitivity and specificity of the samples in the diagnosis of COVID-19 were compared with real-life experience. Results: Paired analyzes were performed by comparing each sample taken by the healthcare worker with the sample taken by the patient. The sensitivity of the three samples (nasopharyngeal taken by the healthcare worker, nasopharyngeal, and saliva taken by the patient) in the diagnosis of the COVID-19 was (100%, 98.7%, and 96.1%, respectively) accepted to be accurate. Conclusions: The sample taken by the paramedic was compatible compared to the real-life experience for the samples taken by the patient in the COVID-19 pandemic period. During the pandemic that is unknown when it will end, this study demonstrated that taking the sample of the patient alone for the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) test is a beneficial approach to the protection of the healthcare worker, reducing the need for protective equipment, increasing the patient's comfort and rapid sampling. [ABSTRACT FROM AUTHOR]

Summary: Denosumab leads to improvements in BMD levels and is a well-tolerated agent according to results of randomized controlled studies but results in real-life setting are important to evaluate drug adherence and real-life efficiency. In this study, we present the results of 305 patients that were treated with denosumab in our clinic. Introduction: The long-term efficacy of anti-osteoclastic drugs in treatment of osteoporosis is well known. Denosumab, a novel human monoclonal antibody, is an anti-osteoclastic agent that has been shown to lead to reductions in vertebral, nonvertebral, and hip fracture risk in randomized and observational studies. Real-life data of this agent is increasing. In this study, we presented our real-life data about the 2-year follow-up of patients under denosumab treatment. Methods: Osteoporotic patients who were treated with at least one denosumab injection between 2014 and 2020 years were included. Clinical and demographic data, bone turnover markers, and radiological reports (bone mineral densitometry (BMD), vertebral x-ray) were obtained from patient files retrospectively. Results: A total of 305 patients (f/m: 275/30, 68.1 ± 11.05 years) were included. The median injection number was 4 (1–10). Two hundred seventy-three patients (89.8%) were persistent on treatment at the 12th month; 175 patients (57.3%) were persistent at 24th month. Sixty-eight patients (22%) were not using denosumab anymore, 55 of the patients were not continuing by doctor desicion and 13 were not continuing due to patient-related causes. Median BMD levels significantly increased from 0.809 (0.2–1.601, IQR: 0.136) to 0.861 (0.517–1.607, IQR: 0.14) in L1–L4 and from 0.702 (0.349–0.997, IQR: 0.125) to 0.745 (0.508–1.008, IQR: 0.137) in femur area at the 24th month of treatment. An improvement of 8.04% in L1–L4 BMD and 4.5% in femur neck BMD levels at the 24th month of treatment was observed. There was a significant decrease in bone turnover markers at the 24th month of treatment. Conclusion: In our group of patients under denosumab treatment, 53% of persistence was found at 24 months and associated with improvement in BMD levels without any significant side effects except one case with urticarial reaction. Denosumab leads to improvements in BMD levels and is a well-tolerated agent in a real-life setting comparable to results of randomized controlled studies in patients with different comorbidities. [ABSTRACT FROM AUTHOR]

Aim: To determine the clinical outcomes and evaluate the perspectives of children with Type 1 diabetes (T1D) and their parents managing their child on hybrid closed‐loop (HCL) therapy. Methods: Children with T1D on HCL attending a tertiary diabetes centre between April 2019 and July 2021 were included. A retrospective analysis of glycaemic data was conducted to determine the clinical outcomes. Time spent in closed loop, time in target glucose range (TIR 3.9–10 mmol/L), hypoglycaemia and hyperglycaemia were collected at baseline, 4 weeks, 3 and 6 months post‐HCL. User experience was assessed by questionnaires administered to parents of children with T1D. Results: Seventy‐one children, mean (SD) age of 12.2 (3.2) years were commenced on HCL. Ten (14%) discontinued HCL use, with 60% discontinuing within the first 6 months. Glycaemic outcomes were analysed in 52 children. Time spent in closed loop was 78 (21) % at 4 weeks, declined to 69 (28) % at 3 months (P = 0.037) and 63 (34) % at 6 months (P = 0.001). The mean %TIR increased from 59.8 at baseline to 67.6 at 3 months and 65.6 at 6 months with a mean adjusted difference of 7.8% points [95% CI 3.6, 11.9] and 5.5% points [95% CI 1.4, 9.5], respectively. There was a reduction in time > 10 mmol/L and time < 3.9 mmol/L from baseline to 6 months. Although families faced challenges with technology, better glucose control with reduced glycaemic fluctuations were reported. Conclusions: HCL therapy is associated with improved glycaemia; however, adequate support and education are required for best outcomes. [ABSTRACT FROM AUTHOR]

Background. Carfilzomib, lenalidomide, and dexamethasone (KRd) have been approved for the treatment of relapsed and refractory multiple myeloma (RRMM) based on ASPIRE clinical trial. Aim. Analysis of efficacy and safety of KRd in routine clinical practice. Materials and methods. The prospective analysis included patients with MM who received at least one line of previous therapy. The inclusion criteria were relapse/progression; refractoriness; lack of very good partial response (VGPR) and more after the first line of therapy. Since February 2016, we used KRd like in ASPIRE trial, since October 2019, carfilzomib has been used at a dose of 56 mg/m2 on days 1, 8 and 15. Autologous hematopoietic stem cell transplantation (autoHSCT), consolidation (KRd) and maintenance therapy (Rd) were regarded as one line of therapy. Results and discussion. We evaluated 77 patients with median age at the time of diagnosis is 55 (3072) years. For 56% (n=43) of patients KRd was applied as the second line (group 1), for 44% (n=34) as the third and more (group 2). In 23/43 patients from group 1, an early change in therapy was made due to insufficient effectiveness (after 24 courses of VCD or PAD). KRd served as a "bridge" to autoHSCT in 25 (32%) patients (21 of 25 in group 1). Another 7 patients underwent collection of autoHSC (all from group 1). The overall response rate (ORR) was 80.5%, with 33.8% complete response (CR) and 26% VGPR. ORR in group 1 was 98% versus 65.6% in group 2; 24-month overall survival (OS) was 70%, progression free survival (PFS) 49.8%. In group 1, 24-month OS was 85.6% versus 50.0% in group 2, 24-month PFS was 67.8% versus 25.5% (p=0.01). Conclusion. Our analysis confirmed the high efficiency of KRd in the treatment of RRMM in real-life practice. Early correction of therapy with insufficient effectiveness of the first line made it possible to implement the strategy of high-dose consolidation and autoHSCT in a larger percentage of patients with MM.

Psoriasis is one of the commonest inflammatory skin diseases determining a very high impact on patients' quality of life and daily activities and relationships. Several biologic therapies have been approved through the years for the treatment of moderate‐to‐severe plaque psoriasis, and efficacy and safety profile have been analyzed in clinical trials. Ixekizumab is an immunoglobulin G subclass 4 monoclonal antibody that selectively targets and binds IL‐17A with high specificity and affinity. Inhibiting IL‐17A activity, ixekizumab reduces and turns down levels of inflammation, resulting in the clinical improvement of the disease. Long‐term efficacy and safety profile of ixekizumab have been investigated and reported in the UNCOVER trials, but in literature there are only few studies based on real life experience. We present the efficacy and safety profile of ixekizumab in a cohort of 779 patients affected by moderate‐to‐severe plaque psoriasis and treated with ixekizumab in 11 Italian dermatology hospitals, with a follow‐up of care until 192 weeks. [ABSTRACT FROM AUTHOR]

Abstract Practical, safe, and effective hemostatic approach to orthopedic surgery using Extended Half‐Life factor IX in hemophilia B. By intraindividual comparison, we found a lower FIX consumption, number of infusions, and cost compared to plasma‐derived FIX.

Recent epidemiological studies highlighted a possible link between PsO and demyelinating disorders such as MS.[2] Herein, we reported the case of a 40-year-old female who suffered from PsO and MS successfully treated with a combination therapy of secukinumab and fingolimod. Keywords: fingolimod; multiple sclerosis; psoriasis; real-life experience; secukinumab EN fingolimod multiple sclerosis psoriasis real-life experience secukinumab e100 e102 3 02/17/23 20230201 NES 230201 Dear Editor, Psoriasis (PsO) is a chronic inflammatory disease characterized by erythemato-desquamative plaques with systemic involvement (psoriatic arthritis, metabolic syndrome, cardiovascular disorders, Crohn's disease, and depression).[1] Multiple sclerosis (MS) is an auto-immune neurological disease characterized by inflammation, demyelination, gliosis, and neuronal loss. [Extracted from the article]

This study compared the in-the-moment experiences among mothers of children with Autism Spectrum Disorders (ASD) by their household status (i.e., single versus coupled) and region of residence (i.e., regional versus major city area). An experience sampling method was employed to collect data, and a total of 40 mothers used an iOS device to record activity types and in-the-moment experiences for one week during school term. Mann–Whitney U test and multilevel analysis were conducted to compare the experiences of these mothers. The analyses found the following results: 1) mothers spent most time in childcare and least time in self-care activities; 2) coupled mothers were more likely to feel supported; 3) coupled mothers were less likely to feel supported in domestic tasks; 4) mothers from major city were more likely to feel challenged in self-care activities; and 5) mothers from major city were more likely to feel supported in productivity tasks. Limited but significant differences between single and coupled mothers, as well as mothers from regional and major city areas, were found. Future research direction and service provisions were suggested. This study shows that all mothers spent the most time on childcare and the least amount of time on self-care activities. In-the-moment experiences between single and coupled mothers, as well as mothers from major cities and mothers from regional areas, differ somewhat; however, this study builds evidence to support that these mothers' experiences are similar. The result of the study indicates that single mothers require extra support as they carry similar levels of responsibilities as coupled mothers, but without the support of a partner. Promoting a sense of control may assist all mothers to fully engage in parenting activities. [ABSTRACT FROM AUTHOR]

Background: In node-negative HER2-overexpressed breast cancers, adjuvant paclitaxel plus trastuzumab treatment is a successful de-escalation approach with excellent survival outcomes. Methods: All patients with HER2+ breast cancer treated in our centers were retrospectively reviewed. Results: We analyzed 173 patients who were treated with adjuvant paclitaxel plus trastuzumab. The mean tumor size was 2.2 cm. There were eight invasive disease events or death: four distant recurrences (2.3%), three locoregional recurrences (1.7%) and one death without documented recurrence after a 52 month follow-up. The 3-year disease-free survival and recurrence-free interval rate was 96.6%. Conclusion: This real-life experience with adjuvant paclitaxel plus trastuzumab demonstrated few distant recurrences and is compatible with the APT trial findings. [ABSTRACT FROM AUTHOR]

With the recent economic and technological rapid change, a major shift in the workforce nature is expected shortly. Construction management is currently focused on increasing productivity and optimizing structures costs. However. new management strategies rely on quality management, global manufacturing. building information modeling, and many others. Those new strategies require managers with soft skills and can operate in situations. With that in mind, cooperative education (Co-op) has a pivotal role in formulating the relationship between the universities and industry for better reflecting on the recently needed industrial requirements in curriculum content. In this paper. the role of the Co-op programs iii university-to-career transition has been explored through a case study of the engineering management students of Prince Sultan University (Riyadh, KSA). Direct and Indirect assessment studies were performed to investigate the nature of the Co-op programs offered to the five student batches (2015-2019) of the Construction Management program (CMP), the students' gained experiences upon completion of the Co-op. the program's overall quality and efficiency, and the Co-op students' readiness to start their career. The 5A's indicators model has been used for the indirect assessment, while the SEF direct assessment tool was proposed to evaluate the student learning outcomes achieved by the end of the program, considering the evaluation of Employers, Faculty, and the students' self-assessment. The assessment results showed that the real-life experiences gained by Co-op students improved their job readiness. It also increased their chances of getting a job even before graduation. The study found that 70% of PSU construction management Co-op students got jobs through the Co-op. Nevertheless. the study also addressed several weak points associated with the offered Co-op, such as the unreliability of the employers' evaluation and ignoring the student self-assessment in the currently utilized direct assessment method. [ABSTRACT FROM AUTHOR]

Primary or secondary immune deficiency (ID) is a risk factor, although rare, to develop Waldenström macroglobulinemia (WM). We aimed to better understand the incidence of this occurrence in the real-life and the outcome of either entity. We conducted a review of 194 WM in the Poitou-Charentes registry and identified 7 (3.6%) with a prior history of ID. Across the 7 WM with ID, 4 progressed to active WM disease and required treatment for WM with a median time between WM diagnosis and the first treatment of 1.5 years (range 0–3). The median time from ID to WM occurrence was 8 years (1–18). WM could develop from ID, although a rare event. Our first action was to systematically decrease immunosuppression with long-term control of ID. Half of indolent WM remained indolent despite ID and for remaining WM none appeared of poor risk WM. [ABSTRACT FROM AUTHOR]

Caregivers of adolescents with autism spectrum disorder commonly experience stigma. However, how stigma influences social interactions of adolescents with autism spectrum disorder is unclear. We aimed to explore the impact of caregiver stigma on real-life social experiences of Taiwanese adolescents with autism spectrum disorder. In the context of everyday activities, 76 adolescents with autism spectrum disorder who were not intellectually disabled (69 males, aged 10–16 years) carried a mobile device that prompted them 7 times, randomly, each day for 7 days to record with whom they were interacting, what they perceived, and how they felt about the interactions. Caregivers completed the Affiliate Stigma Scale to measure caregiver stigma. Multilevel analyses revealed that participants whose caregivers perceived high levels of stigma were more likely than those whose caregivers experienced less stigma to interact with family members and less likely to be interested in interacting with people at school. However, those participants also experienced more anxiety while interacting with family members. The findings shed light on ways that caregiver stigma impacted the social experiences of adolescents with autism spectrum disorder and suggest that, in promoting social participation for adolescents with autism spectrum disorder, researchers and service providers must support caregivers to manage stigma. Caregivers of people with autism spectrum disorder commonly experience stigma. As a result, they may avoid contact with others, in turn, influencing their child's social participation. This study aimed to explore the impact of stigma perceived by the caregivers on the everyday social experience of Taiwanese adolescents with autism spectrum disorder. We asked 76 adolescents with autism spectrum disorder who did not have intellectual disability (69 males, aged 10–16 years) to carry a mobile device for 7 days. The device prompted them 7 times each day to record who they were interacting with, what they perceived, and how they felt about their social interactions. In addition, we asked their caregivers to complete the Affiliate Stigma Scale to measure their experience of stigma. We found that participants whose caregivers perceived high levels of stigma were more likely to spend time with family members and less likely to be interested in interacting with people at school. Those participants also were more likely to experience anxiety while interacting with family. Our study suggests that it is important for clinicians to implement support services for adolescents with autism spectrum disorder and help caregivers in managing stigma to promote their child's social participation. [ABSTRACT FROM AUTHOR]

Purpose: Colorectal cancer is the third leading diagnosis accounting for nearly 10% of all new cancers worldwide. The distinct features among BRAF mutant colorectal cancers make these tumor groups hard to treat for oncologists. The median overall survival (OS) of these types of cancers is reported to be 9 to 14 months. Methods: The study was declared on the Turkish Oncology Study Group Conference and approved. The patients' data was received from the centers who confirmed to participate. The BRAF-mutated patients were included in the study. The demographic features (age, gender, etc.), type of mutation, tumor localizations, histology, microsatellite instability (MSI) status, metastasis patterns chemotherapeutic agents and progression, and death times were recorded. Results: Thirty-nine patients were enrolled in the study. Sixteen patients had concurrent KRAS mutations, while 7 had NRAS mutations. Most of the patients received doublet chemotherapies in combination with anti-VEGF agents in the first and second line of the treatment. There was a significant difference in OS according to the stage which showed a decreased survival in stage IV patients at the time of diagnosis. Concurrent KRAS mutation resulted in increased OS. The median OS was 47 and 24 months favoring the KRAS mutant group. The patients whose primary tumor operated had better survival when compared with other patients. The median OS of the operated group was 47 months, while the non-operated group was 24 months. Liver metastasis was related to worse prognosis at the time of diagnosis in univariate analysis. Conclusion: In our study we found a high concurrent RAS mutation ratio in a BRAF mutant patient group which was different from prior studies. The concurrent mutations resulted in a favorable outcome in terms of OS which is also different from the current knowledge. More prospective studies are needed especially BRAF-mutated patient population and especially with concurrent RAS mutations. [ABSTRACT FROM AUTHOR]

There is a paucity of data on long‐term (≥5‐year) outcomes of ustekinumab therapy for psoriasis in real‐life clinical practice. This observational, retrospective study aimed to evaluate the long‐term efficacy and safety profile of ustekinumab in 52 adult patients with moderate‐to‐severe chronic plaque psoriasis who were treated with ustekinumab for at least 28 weeks and a maximum of 105 months in our tertiary referral center in Turkey, between 2010 and 2019. Response to therapy was assessed using Psoriasis Area and Severity Index (PASI). Logistic regression analysis was performed to determine significant associations (p‐value <0.05) with response to treatment. The PASI50, PASI75, PASI90, and PASI100 response rates were 97.8%, 88.9%, 53.3%, and 35.5%, respectively, at year 1 and 100%, 80.0%, 60.0%, and 40.0%, respectively, at year 5. Non‐obesity was independently associated with PASI90 response at year 2 (p = 0.043), while biologic‐naivety was independently associated with PASI90 responses at year 2 (p = 0.047) and year 3 (p = 0.021). An absolute PASI score of ≤3 was achieved by 82.2% and 80.0% of the patients at year 1 and year 5, respectively. Nine patients received adjuvant therapy and nine underwent ustekinumab dose escalation. These strategies were effective for recapturing clinical response in most patients. Ustekinumab was generally well‐tolerated with no dose‐related and cumulative toxicity, or drug interaction over a mean of 33.5 ± 21.1 months. The main reasons for discontinuation were secondary failure and loss to follow‐up. Our 9‐year real‐life clinical experience demonstrates that ustekinumab is an efficacious and safe treatment option for long‐term therapy of moderate‐to‐severe plaque psoriasis. [ABSTRACT FROM AUTHOR]

Introduction: In a randomized prospective trial, adjuvant rituximab was more efficacious than corticosteroids alone in the treatment of pemphigus; however, real-life data are limited. Rituximab treatment for pemphigus has only recently been introduced to the Israeli health basket. Previously, patients received rituximab if they paid out of pocket or through private insurance, separating patients into 2 treatment groups, mostly based on economic capability. Methods: A retrospective cohort study of the 12-month clinical response of pemphigus vulgaris/foliaceus patients. We compared patients after a single cycle (1,000 mg on days 0 and 15 or weekly 375 mg/m2 for 4 weeks) of adjuvant rituximab with systemic corticosteroids ± steroid-sparing agents, to patients who were prescribed rituximab, could not obtain it, and received systemic corticosteroids ± steroid-sparing agents. Results: Forty-five patients were included (adjuvant rituximab, n = 29; immunosuppression alone, n = 16). At baseline, rituximab patients had a higher mean pemphigus disease area index (PDAI) (p = 0.07) and higher mean daily dosages of prednisone (1.51 vs. 1.16 mg/kg, p = 0.39). All patients but 1 in the rituximab group continued systemic steroids, and 31% in the rituximab group versus 50% in the immunosuppression-alone group received systemic adjuvants. At 12 months, partial or complete remission rates (on or off maximum 40 mg/day prednisone equivalent) were nonsignificantly higher in the rituximab group (62 vs. 50%, p = 0.53); however, patients on rituximab showed faster remissions (3.4 ± 1.9 vs. 5.9 ± 3.6 months; p = 0.03) with a trend for a greater PDAI reduction (p = 0.051). Adverse events were comparable. Conclusions: In this real-life study, a single cycle of rituximab achieved more remissions and sooner compared to conventional immunosuppression, but the differences were not significant, probably due to a small sample size and severe baseline disease in the rituximab group. Future real-life studies on larger groups are needed. [ABSTRACT FROM AUTHOR]

Carfilzomib, lenalidomide, and dexamethasone (KRd) have been approved for the treatment of relapsed and refractory multiple myeloma (RRMM) based on ASPIRE clinical trial. However, its effectiveness and safety profile in real clinical practice should be further assessed. We retrospectively evaluated 130 consecutive RRMM patients treated with KRd between December 2015 and August 2018, in 9 Hematology Departments of Rete Ematologica Pugliese (REP). The overall response rate (ORR) was 79%, with 37% complete response (CR). Treatment with KRd led to an improvement in response regardless of age, refractory disease, and number and type of previous therapies. After a median follow-up of 18 months, median PFS was 24 months and 2y-PFS was 54%. PFS was longer in patients achieving a very good partial response (VGPR) with median PFS of 32.4 months. The relapses after prior autologous transplant (ASCT) positively impact median PFS. Several baseline disease characteristics, such as III ISS scoring or elevated LDH, and prior exposure to lenalidomide were found to negatively impact PFS. Primary refractory or relapsed myeloma patients have been treated with KRd as bridge to ASCT with a great benefit. Thirty-four (83%) reached at least a partial response after KRd and 21 (61%) performed ASCT. In transplanted patients, median PFS was not reached and 2y-PFS was 100%. The treatment discontinuation rate due to adverse events (AEs) was 18%, most commonly for lenalidomide (11%). Overall, in 10% of patients, a KRd dose reduction was necessary at least once (2.5% for carfilzomib and 8% for lenalidomide). The most frequent AE was neutropenia (44%) and anemia (41%). Infections occurred in 14% of patients. Cardiovascular events occurred in 11% of patients. Elderly patients have tolerated therapy very well, without additional side effects compared to younger patients, except for cardiac impairment. Our analysis confirmed that KRd is effective in RRMM patients. It is well tolerated and applicable to the majority of patients outside clinical trials. A longer PFS was shown in patients achieving VGPR, in those lenalidomide naïve and in patients relapsing after previous ASCT. Previous ASCT should not hamper the option for KRd therapy. Accordingly, KRd should be used as bridge regimen to ASCT with remarkable improvement in response and PFS rates. Further clinical studies are needed. [ABSTRACT FROM AUTHOR]

Background: Multiple sclerosis (MS) is an autoimmune, neuroinflammatory, and neurodegenerative disease of the central nervous system. B cells have recently emerged as a promising target to significantly reduce inflammatory disease activity in MS, with successful trial studies using antiCD20 therapies. However, real-life data about safety and efficacy are limited.Objectives: To analyze the clinical and radiological inflammatory activity, adherence to therapy, and safety of rituximab (RTX) in an MS patients' sample, treated from 2015 to 2018 in our center PATIENTS AND METHODS: Retrospective study on prospectively collected data about relapses, disability progression, and radiological activity (new T2 lesions and Gd-enhancing lesions) were recorded and used to assess no evidence of disease activity (NEDA) at 12 months. RTX-related adverse events were recorded. RTX was administered intravenously at a dosage of 1000 mg twice 2 weeks apart, then every 6 months.Results: Sixty-nine patients were included. Fifty-three (76.8%) had a relapsing-remitting, two a primary progressive course, and 14 a secondary progressive course. The mean follow-up period was 16 ± 9.7 months. Thirty-five (50.7%) patients had relapses in the year prior to RTX therapy, with a mean annualized relapse rate of 0.75, significantly reduced to 0.36 at 12 months (p < 0.001). Among the 36 patients included in the study who had an MRI available at 12 months, MRI activity was reduced from 88% (n = 32) to 8.3% (n = 3) at follow-up (p < 0.001). Twelve (17.4%) patients suspended RTX during the study.Conclusions: Our real-life experience confirms that off-label therapy with RTX may represent a valid, cost-effective therapeutic option in MS. [ABSTRACT FROM AUTHOR]

Objective: To gain information about multiple dexamethasone intravitreal implant (DEX-I) injections in diabetic macular edema (DME) eyes in real-life clinical settings. Methods: Patients with DME treated with multiple (≥5) DEX-I injections between January 1, 2014, and December 31, 2018, were retrospectively enrolled regardless of previous treatment with anti-VEGF agents. All patients were evaluated with best-corrected visual acuity (BCVA) in logMAR, ocular fundus, and spectral domain optical coherence tomography (SD-OCT) at baseline and at 3 months after the last DEX-I injection. Multiple DEX-I injections were administered when necessary in case of DME recurrence. Main efficacy measures were changes in BCVA and central retinal thickness (CRT) from baseline to 3 months after the last DEX-I injection; main secondary measures were an increase in intraocular pressure (IOP), the need for cataract surgery, endophthalmitis, and vitreous hemorrhage. Results: Seventeen patients (18 eyes) with DME and mean age (± SD) of 54.3 ± 8.16 years were treated with DEX-I injections between 2014 and 2018. The majority of eyes (77.8%) had been treated with a mean of 6.3 ± 3.2 anti-VEGF agents before switching to DEX-I. During a mean follow-up period of 37.6 months and after a mean number of 5.9 DEX-I injections, visual acuity improved or stabilized in 77.8% of all eyes, accompanied by a significant reduction in CRT. An increase in IOP was recorded in 38.8% of all patients, while a surgical procedure was needed for cataract in 73.3% of all phakic patients. Conclusions: In this real-life experience in Italy, multiple DEX-I treatments showed good efficacy with no new safety concerns. The follow-up duration of >3 years and a greater number of DEX-I intravitreal injections compared to other observations confirm the positive balance between risks and benefits of DEX-I in the long term. [ABSTRACT FROM AUTHOR]

Objective: The objective of this paper is to assess the prevalence of the main clinical manifestations and laboratory features at disease onset and during the ensuing 10 years of a large cohort of patients with antiphospholipid syndrome (APS) from a single center. Methods: The study included all consecutive APS patients followed longitudinally in our center from 2003 to 2013. Descriptive statistics for demographics, clinical and laboratory features and mortality were performed. Results: A total of 160 patients were included. Most of them, 128 (78.8%), were women and the mean (SD) age at diagnosis was 39.1 (14.0) years. The majority of them, 104 (65.0%), had primary APS, 36 (22.5%) had APS associated with systemic lupus erythematous, and 20 (12.5%) had APS associated with other autoimmune disease. During the study period, thrombotic events occurred in 27 (16.9%) patients, the most common being strokes, nonbacterial thrombotic endocarditis and deep venous thrombosis. Regarding obstetric morbidity, 18 women (14.3%) became pregnant and 90% of pregnancies succeeded in having live births. The most common obstetric complication was early pregnancy loss (15% of pregnancies). Prematurity (11.1% of live births) and intrauterine growth restriction (5.6% of live births) were the most frequent fetal morbidities. Ten (6.3%) patients died and the most frequent causes of death were severe thrombosis, hemorrhage, and cancer. Three (0.9%) cases of catastrophic APS occurred. The survival probability at 10 years was 93.8%. Conclusions: Patients with APS develop significant morbidity and mortality despite current treatment. It is imperative to identify prognostic factors and therapeutic measures to prevent these complications. [ABSTRACT FROM AUTHOR]

Practical, safe, and effective hemostatic approach to orthopedic surgery using Extended Half‐Life factor IX in hemophilia B. By intraindividual comparison, we found a lower FIX consumption, number of infusions, and cost compared to plasma‐derived FIX. [ABSTRACT FROM AUTHOR]

A comparative analysis of the explicit and implicit foundations of the natural, social, and humanitarian sciences explores the genesis of the foundations of science and their relationship to common sense, everyday consciousness, real-life experience, and human intuition. The analysis takes into account the conventional division of the sciences into theoretical (fundamental) and practical (applied). The main examples selected are physics from the natural sciences and psychology from the social and humanitarian sciences. The article considers the connection between the conformity of the foundations of theory to scientific standards and its survival. It shows that the natural sciences developed by means of the supplantation of initial postulates and paradigms, while the principal external criterion vis-à-vis social-science theories is their survivability. This assertion is juxtaposed with the fact that in the natural sciences applied technologies that change the world are a direct result of fundamental-research results, whereas in the social and humanitarian sciences, this connection is much weaker. [ABSTRACT FROM AUTHOR]

Imatinib, the first BCR/ABL kinase inhibitor approved for the treatment of chronic myeloid leukemia (CML), has changed the long-term outcome of patients affected by this disease. The aim of our analysis was to report, after a median follow-up of 10.2 years (range 5.8-14.8), the long-term outcome, efficacy, and safety of imatinib treatment (frontline and after interferon failure) in a single institution cohort of 459 patients with CML in chronic phase treated outside of clinical trials. The 10-year overall survival of the whole cohort was 77.1%, while the 10-year probability of dying due to CML and other causes was 7.8% and 16%, respectively. The prognostic value of the BCR-ABL1 ratio at 3 months (⩽ 10%) and of complete cytogenetic response and major molecular response at 1 year was confirmed also in the real-life practice. The EUTOS long-term survival score better stratified the baseline risk of dying of CML compared with other risk scores. Two hundred thirty-six (51.4%) patients achieved a deep molecular response during imatinib treatment after a median time of 4.57 years, and 95 (20.6%) had a stable deep molecular response maintained for at least 2 consecutive years. Imatinib was associated with a low rate of serious cardiovascular events and second neoplasia. This 10-year real-life follow-up study shows that imatinib maintains efficacy over time and that long-term administration of imatinib is not associated with notable cumulative or late toxic effects. [ABSTRACT FROM AUTHOR]

Abstract Introduction REHAP is a voluntary, observational Spanish registry of patients with pulmonary arterial hypertension. We analyzed the experience (use and effectiveness) with inhaled iloprost (inh-ILO) in real-life conditions during a 3-year period. Methods Patients included were those with PAH ≥14 years recruited during 1998–2016 who had received inh-ILO. Variables were collected at the beginning of treatment (0 ± 3 months) and 12 ± 3/36 ± 6 months follow-up. Effectiveness was assessed in the intent-to-treat population as changes in functional class and/or physical performance and transplant-free survival from the beginning of treatment. Stopping inh-ILO-related survival was also assessed. Subanalyses included treatment strategy (first-line therapy –monotherapy or upfront combination- or sequential therapy) and risk of clinical worsening/death. Results Inh-ILO was the most frequently used prostanoid in Spain, rendering 267 patients eligible for analysis. Median age was 54 years; 61% were WHO FC III. Sixty (23%) patients started inh-ILO as monotherapy, 27 (10%) as upfront combination and 180 (67%) sequentially. At 3-year follow-up significant clinical improvements were observed; however, transplant-free survival rate was 54%, being poorer in patients at high risk (63% vs. 85% in low risk patients; P < 0.001) and similar in the three treatment strategies. Only 25% patients remained on inh-ILO. Three-year after stopping inh-ILO-related survival rate was 24.7%. Conclusion Data from the REHAP collected during 3 years shows that inh-ILO has low effectiveness independently of the treatment strategy used, with a 3-year survival rate of 54% despite significant clinical improvements, probably due to the use in high-risk patients. Discontinuation rate was as high as 75%. Highlights • Iloprost is a mainstay in the treatment of pulmonary arterial hypertension (PAH). • Our study evidences the low effectiveness of iloprost (monotherapy or combination). • Three-year survival rate of patients is 54%, despite clinical improvements. • Discontinuation rate was as high as 75%. • ~15% of patients had severe PAH (WHO FC IV): they were not appropriately treated. [ABSTRACT FROM AUTHOR]

Exemestane (Exe) in combination with Everolimus (Eve) represents an important treatment option for patients diagnosed with hormone receptor positive (HR+), human epidermal growth factor receptor 2 negative (HER2-) metastatic breast cancer (MBC), which was previously treated with non-steroidal aromatase inhibitors (NSAI). Data from unselected populations may be useful for defining the optimal therapeutic algorithm within a clinical setting. Data from 264 HR+, HER2- MBC patients who received Exe-Eve treatment in combination, following the failure of NSAIs was retrospectively analyzed. Different lines of endocrine treatment (ET) were investigated to evaluate the efficacy and toxicity of the treatment within the 'everyday clinical practice' population. The disease control rate (DCR) was 73.1%, with no statistically significant difference among the different settings. At a median follow-up of 42 months, the median progression free survival (PFS) was 11.6, 9.7 and 7.5 months for patients treated with Exe-Eve as first, second or third line therapy, respectively. There was a statistically significant correlation with younger age, no previous adjuvant chemotherapy (CT), no previous adjuvant endocrine therapy (ET), HT duration =36 months, involvement of liver and/or lung, no prior CT for metastatic disease and PS=0 at the start of treatment. The median overall survival (OS) was 33.0 months; at a median follow-up of 67 months, the median OS was 43.1, 31.7 and 27.9 months in patients treated with Exe-Eve in first, second or third line therapy, respectively. On multivariate analysis, diabetes and previous CT for metastatic disease were revealed to correlate with a worse outcome. Conversely, the presence of mucositis was significantly associated with long-term survival. Overall, Exe-Eve was typically well tolerated and the majority toxicities were G1 or 2, while treatment discontinuation due to unacceptable toxicity was only required in 5.7% of patients. Despite the limitations due to the observational nature of this study, the findings suggest that treatment with Exe-Eve is an active and safe therapeutic option for endocrine-sensitive MBC patients in a real-world clinical setting, regardless of treatment lines. [ABSTRACT FROM AUTHOR]

Introduction: Sorafenib (SOR) has proved to be effective in patients with advanced hepatocellular carcinoma (HCC), since overall survival was higher in phase III clinical trials; however, disease progression can occur. Objectives: The study aimed to describe real-life experience in advanced HCC treatment with SOR at a university hospital in Brazil and to estimate the number of patients with indication of second-line therapy. Methods: This is a retrospective study that included cases of HCC with prescription of SOR based on real-life practice between 2011 and 2016. Demographic, clinical, and laboratory data were collected. Results: From 572 patients with HCC, SOR was prescribed in 103 cases. From them, 62.1% were classified as Child-Pugh (CP)-A, 54.4% as Barcelona Clinic Liver Cancer (BCLC)-C, and 74 (71.8%) started treatment. Overall survival was 25.5 (95% CI 17.0–34.1) months and 1-year survival was greater in patients who received SOR than in non-treated (88.7 vs. 44.4%, p < 0.001). There was no difference in survival between BCLC-B and C (p = 0.405), as well as CP-A and B (p = 0.919). In 21.6% of the patients, a second-line therapy with regorafenib was indicated. Conclusion: In this real-life study, SOR significantly increased the survival rate by 1 year in patients with advanced HCC regardless of BCLC staging and CP score. Second-line therapy would be indicated in 21.6% of cases. [ABSTRACT FROM AUTHOR]

To date, belimumab is the only biological drug approved for the treatment of patients with active refractory SLE. We compared and critically analyzed the results of 11 observational clinical-practice-based studies, conducted in SLE referral centers. Despite the differences in endpoints and follow-up duration, all studies remarked that belimumab provides additional benefits when used as an add-on to existing treatment, allowing a higher rate of patients to reach remission and to taper or discontinue corticosteroids. In the OBSErve studies, 2-9.6% of patients discontinued corticosteroids and 72-88.4% achieved a ≥ 20% improvement by physician’s judgment at 6 months. In Hui-Yuen’s study, 51% of patients attained response by simplified SRI at month 6. In Sthoeger’s study, 72.3% of patients discontinued corticosteroids and 69.4% achieved clinical remission by PGA after a median follow-up of 2.3 years. In the multicentric Italian study, 77 and 68.7% of patients reached SRI-4 response at months 6 and 12, respectively. In all the studies, disease activity indices decreased over time. Retention rates at 6, 9, and 12 months were 82-94.1, 61.2-83.3, and 56.7-79.2%, respectively. The main limitations of these studies include the lack of a control group, the short period of observation (6-24 months) and the lack of precise restrictions regarding concomitant medication management. This notwithstanding, these experiences provide a more realistic picture of real-life effectiveness of the drug compared with the randomized controlled clinical trials, where stringent inclusion/exclusion criteria and changes in background therapy could limit the inference of data to the routine clinical care. [ABSTRACT FROM AUTHOR]

Purpose: Randomized clinical trials studying the effects of VEGF inhibition on wet age-related macular degeneration (wAMD) are designed so that the effects of individually varying risk factors on the treatment response are eliminated. The influence of these risk factors can be studied in large data sets from real-life experience. Patients and methods: All 2,255 patients diagnosed with wAMD requiring anti-VEGF treatment in at least one eye over more than 9 years in a defined Danish population with 0.9 million inhabitants were studied. The predictive value of eye laterality, sex, current smoking status, type of anti-VEGF compound, membrane position, membrane type, leakage area, number of injections, number of visits, age, time to follow-up, visual acuity, and central retinal thickness (CRT) at baseline on change in CRT after three monthly injections with anti-VEGF compound followed by treatment pro re nata for up to 12 months was assessed. Results: After 12 months, 67 patients had died, 903 had had stable CRT for at least 6 months, and 1,285 patients had not achieved stable CRT. The reduction in CRT was -84.8±118.3 µm, whereas the increase in visual acuity was 2.2±14.7 Early Treatment Diabetic Retinopathy Study letters. The risk factors included contributed to 64% of the variation in CRT reduction. High age and high CRT at baseline predicted high CRT reduction, whereas more injections, treatment with ranibizumab, and male sex predicted a low CRT reduction. Conclusion: Age, sex, and type of anti-VEGF medication can be used to plan treatment and inform patients about the expected response of anti-VEGF treatment in wAMD. [ABSTRACT FROM AUTHOR]

Background and Aim The introduction of sofosbuvir has revolutionized the treatment of chronic hepatitis C. This study was planned to observe whether the efficacy and tolerability of sofosbuvir-based regimens demonstrated in phase 3 clinical trial results translate into real-life clinical practice. Methods This prospective, non-randomized observational study conducted in Dayanand Medical College and Hospital, Punjab, included all consecutive treatment-naïve patients with chronic hepatitis C (genotypes 1-5) who were treated with sofosbuvir-based regimens. Response to therapy was assessed at week 4 (rapid virological response), week 12 or 24 (end of treatment response), and 12 weeks after cessation of therapy (sustained virological response [SVR]). Results Of 947 patients diagnosed with chronic hepatitis C virus and considered for treatment with direct-acting antivirals, 736 patients (77.1%) opted for treatment (age 45.1 ± 10.1 years, 64% men, genotype 3 [80%], genotype 1 [14.7%], and genotype 4 [4.9%]). Viral load was high (>600 000 IU/mL) in 361/736 (49%); 330 patients (44.8%) had cirrhosis (80 [14.3%] were decompensated). Patients with genotypes 1, 4, and 5 ( n = 135) were treated with triple drug regime (pegylated interferon, ribavirin, and sofosbuvir) for 12 weeks. Patients with genotype 3 ( n = 589) were treated either with dual therapy (sofosbuvir and ribavirin) for 24 weeks ( n = 405) or triple therapy for 12 weeks ( n = 184). SVR was achieved in 453/473 (95.8%). SVR rates did not differ among different genotypes but were higher in non-cirrhotics. Conclusion Sofosbuvir-based treatment regimens achieve high SVR rates in real-life cohort of Indian patients with chronic hepatitis C infection (including those with cirrhosis). [ABSTRACT FROM AUTHOR]

Whether preschool children with autism spectrum disorder (ASD) have motivation for social engagement is a matter of great concern. However, the current findings derived in laboratory settings are still controversial, probably because the motivation for social engagement depends on the specific social contexts. Therefore, we compared the motivation for real-life social engagement between preschool children with ASD and typically developing (TD) peers from the caregivers' perspectives using the self-determination theory (SDT) and experience sampling methodology (ESM). The SDT helped us to examine the impact of living contexts on the motivation for social engagement, and the ESM allowed us to record objective and subjective experiences in real time across natural settings. Thirty preschool children with ASD and 30 matched TD peers were recruited. Caregivers carried mobile devices to record their children's social experiences repeatedly over seven days. Multilevel modelling was used for analysis. Regardless of ASD, preschool children were more likely to be autonomous and intrinsically motivated while engaging in social play/leisure activities, but less likely to be autonomous, competent, and intrinsically motivated in non-play/leisure activities involving social interactions. The ASD group was also less likely to care about others or to be cared about in social play/leisure activities. The findings highlight that the motivation for social engagement in preschool children with ASD is situation-specific and emphasize the importance of providing a supportive environment and encouraging engagement in social play/leisure activities. • The motivation for social engagement in children with ASD is situation-specific. • Preschool children were autonomous and intrinsically motivated in social activities. • Children with ASD were intrinsically motivated to engage in social activities. [ABSTRACT FROM AUTHOR]

Introduction: Single-tablet regimens (STRs) can increase treatment success and even improve the quality of life of human immunodeficiency virus (HIV) patients. In this study, we aim to analyze the real-life efficacy and tolerability data of people living with HIV (PLWH) initiated on or switched to bictegravir/emtricitabine/tenofovir alafenamide fumarate (BIC/FTC/TAF) as first-line treatment., Materials and Methods: This retrospective analysis was performed in HIV-1-positive patients who were initiated BIC/FTC/TAF in the HIV clinic between June 2020 and June 2022. Patients who received BIC/FTC/TAF for at least 12 months were included in this study. Virological suppression, laboratory parameters, side effects, and immunological response were analyzed at one, three, six, nine, and 12 months., Results: A total of 116 patients, 66 (56.9%) treatment-experienced and 50 (43.1%) naive, were evaluated within the scope of the study. In the naive patient group, baseline HIV-RNA, CD4+ and CD8+ T cell counts, CD4/CD8 ratio, and estimated glomerular filtration rate (eGFR) values were significantly different in different follow-up months. The number of patients with HIV-1 RNA levels below 50 copies/mL was 55.9% in the first month, 73.7% in the third month, 90.2% in the sixth month, and 100% in the ninth and 12th months., Conclusion: In our real-life observational study, BIC/FTC/TAF treatment achieved rapid viral suppression, maintained viral suppression in virally suppressed patients, and was effective for immunological recovery in both treatment-experienced and naive HIV patients. No serious side effects were observed. Our study has proved the potential of BIC/FTC/TAF as an important option in the treatment of HIV patients., Competing Interests: The authors have declared that no competing interests exist., (Copyright © 2023, Balcı et al.)

This study explored the everyday life experiences of individuals with an autism spectrum disorder (ASD). Fourteen Australians and 16 Taiwanese (aged 16-45 years) with Asperger syndrome/high functioning autism recorded what they were doing, level of interest/involvement, emotional reactions and preference for being alone 7 times/day for 7 days. Multilevel analyses showed that 'solitary/parallel leisure' and 'social activities' were positively associated with interest and involvement. Engaging in these two activities and interacting with friends were positively associated with enjoyment. However, engaging in 'social activities' and having less severe ASD symptoms were associated with in-the-moment anxiety. Severity of ASD and social anxiety moderated experience in social situations. The findings highlight the importance of considering the in-the-moment experience of people with ASD. [ABSTRACT FROM AUTHOR]