Effectiveness Of Different Treatment Approaches Research Articles

Uric Acid as a Biomarker for Mood Disorders: A Comparative Study of Blood Uric Acid Levels Correlating With the Symptom Severity and Treatment Response.

Background Bipolar affective disorder (BPAD) and major depressive disorder (MDD) are two mood disorders whose pathophysiology may involve the purinergic system. Elevated uric acid levels, associated with this system, can impact various behaviors in individuals affected by these conditions. In addition to genetic predisposition, blood uric acid levels can be impacted by various factors, including metabolic syndrome, the consumption of psychoactive medications, and other underlying kidney conditions such as gout. Objective The study aims to investigate the relationship between blood uric acid levels and mental health conditions, specifically BPAD subtypes (manic and depressive) and MDD. The study also examines changes in blood uric acid levels following treatment and evaluates the effectiveness of different treatment approaches in reducing uric acid levels. Methodology To be eligible to participate, individuals must have a confirmed diagnosis of BPAD (manic or depressive type) or MDD, according to the International Classification of Diseases (ICD-10). Blood uric acid levels were measured at both baseline and follow-up assessments. Symptoms were assessed weekly using standardized rating scales (Young Mania Rating Scale (YMRS) and Hamilton Rating Scale for Depression (HAM-D)) until treatment response was achieved, which was defined as a 50% reduction in initial scores on both scales. We used ANOVA to examine the differences among the three patient groups and paired sample t-tests to examine the changes in means before and after treatment conditions. Results A significant positive correlation was found between the severity of illness and serum uric acid levels across all three patient groups: those with BPAD-mania, BPAD-depression, and MDD. Notably, patients with BPAD-mania patients had significantly higher serum uric acid levels (5.2±0.9 mg/dL) compared to those with BPAD-depression (4.8±1.0 mg/dL) and MDD (4.0±1.1 mg/dL). After treatment, all patient groups exhibited a decrease in serum uric acid levels. The reduction in serum uric acid levels was pronounced in all patient groups, with decreases of 3.1±0.8 mg/dL in patients with BPAD-mania, 3.1±0.9 mg/dL in those with BPAD-depression, and 3.5±1.1 mg/dL in those with MDD. The study showed that the reduction in serum uric acid levels was significantly correlated with the severity of illness in patients with BPAD-mania, but not in those with BPAD-depression or MDD. Furthermore, the study found that treatment with lithium carbonate, sodium valproate, or carbamazepine was equally effective in reducing serum uric acid levels, regardless of the mood stabilizer used. Conclusion The study supports that dysfunction in the purine system might play a significant role in the development and progression of BPAD, suggesting that this phenomenon is not solely due to chronicity or medication exposure. This study also introduces a fresh perspective on the underlying biological processes that contribute to the development of BPAD and also sheds light on new treatment regimens targeting uric acid reduction in treating patients with bipolar disorder.

Approach to a patient with iatrogenic coronary dissection during percutaneous intervention

This case report presents a conservative management approach to a potentially hazardous complication, iatrogenic coronary artery dissection (ICAD), occurring during percutaneous coronary intervention (PCI). The patient, a 60-year-old woman with a history of chronic hypertension and type II diabetes mellitus, presented with non-ST-segment elevation myocardial infarction. During the PCI, an antegrade coronary dissection was observed, leading to the termination of the procedure. Post-procedure, the patient was managed conservatively with standard coronary artery disease treatment. Follow-up angiography showed complete healing of the dissection area, and no adverse cardiovascular events occurred during a 3-month follow-up period. This report highlights the importance of early diagnosis and careful management of ICAD. It stresses the need for further research into long-term outcomes and effectiveness of different treatment approaches for patients with ICAD.

Bone disease in mucopolysaccharidosis type I: Morphological, structural and biomechanical characterization, and effect of different treatment approaches

Bone disease in mucopolysaccharidosis type I: Morphological, structural and biomechanical characterization, and effect of different treatment approaches

Comparison of Treatment Outcomes for Tennis Elbow: Corticosteroid Injection and Casting vs. Acupuncture and Physiotherapy

Background & Objectivs: Today, despite the existence of numerous treatments for tennis elbow, the search for more effective methods continues due to the limited efficacy of these existing approaches. This study aimed to compare the results of tennis elbow treatment in two groups: patients receiving corticosteroid injection and casting, and patients receiving acupuncture and physiotherapy. Materials & Methods: This quasi-experimental study, conducted in Jiroft in 2021, investigated the effectiveness of different treatment approaches for tennis elbow. Fifty patients diagnosed with tennis elbow were randomly divided into two groups of 25 each. One group received a combination of corticosteroids and casts, while the other group received acupuncture and physiotherapy. A pain line was used to assess pain levels based on VAS criteria. Data were analyzed using SPSS-26 statistical software, employing Chi-square, Fisher’s Exact, Mann-Whitney, one-way ANOVA, Kruskal-Wallis, and Kolmogorov-Smirnov tests. Results: The majority of patients were female (66%) and housewives (46%). There was a significant difference in pain outcomes between the two groups: physiotherapy alone and physiotherapy combined with corticosteroids and casts. This difference was observed across various conditions (pain at rest, pain during activity, and amount of pain evoked during activity) (p <0.05). In simpler terms, corticosteroids and casts were more effective in reducing pain from tennis elbow compared to acupuncture and physiotherapy. Additionally, corticosteroids and casts had a greater effect on hand movement (supination, pronation, extension, and flexion) compared to acupuncture and physiotherapy. Conclusion: Our findings indicate that treatment of tennis elbow with a combination of corticosteroid injection and casting is more effective than acupuncture or physiotherapy alone. Corticosteroid injections themselves are also an effective way to relieve tennis elbow pain.

Making progress against rare cancers: A case study on neuroendocrine tumors.

In April 2023, the National Cancer Institute offered a roadmap for cancer research to achieve Cancer Moonshot goals. To reach these goals requires making progress for all cancers, not just those that are most common. Achieving progress against rare cancers, as well as common cancers, requires involvement of large clinical research networks. In 2020, the Patient-Centered Outcomes Research Institute (PCORI) launched an initiative on Conducting Rare Disease Research using PCORnet, the National Patient-Centered Clinical Research Network. The purpose of this commentary is to introduce the broader community of cancer researchers to the PCORnet NET-PRO study (comparing the effects of different treatment approaches for neuroendocrine tumors on patient-reported outcomes) thereby demonstrating how researchers can use the PCORnet infrastructure to conduct large-scale patient-centered studies of rare cancers.

Exploring the Literature on Narcolepsy: Insights into the Sleep Disorder That Strikes during the Day

Narcolepsy is a chronic sleep disorder that disrupts the regulation of a person’s sleep–wake cycle, leading to significant challenges in daily functioning. It is characterized by excessive daytime sleepiness, sudden muscle weakness (cataplexy), sleep paralysis, and vivid hypnagogic hallucinations. A literature search was conducted in different databases to identify relevant studies on various aspects of narcolepsy. The main search terms included “narcolepsy”, “excessive daytime sleepiness”, “cataplexy”, and related terms. The search was limited to studies published until May 2023. This literature review aims to provide an overview of narcolepsy, encompassing its causes, diagnosis, treatment options, impact on individuals’ lives, prevalence, and recommendations for future research. The review reveals several important findings regarding narcolepsy: 1. the classification of narcolepsy—type 1 narcolepsy, previously known as narcolepsy with cataplexy, and type 2 narcolepsy, also referred to as narcolepsy without cataplexy; 2. the genetic component of narcolepsy and the complex nature of the disorder, which is characterized by excessive daytime sleepiness, disrupted sleep patterns, and potential impacts on daily life activities and social functioning; and 3. the important implications for clinical practice in the management of narcolepsy. Healthcare professionals should be aware of the different types of narcolepsies and their associated symptoms, as this can aid in accurate diagnosis and treatment planning. The review underscores the need for a multidisciplinary approach to narcolepsy management, involving specialists in sleep medicine, neurology, psychiatry, and psychology. Clinicians should consider the impact of narcolepsy on a person’s daily life, including their ability to work, study, and participate in social activities, and provide appropriate support and interventions. There are several gaps in knowledge regarding narcolepsy. Future research should focus on further elucidating the genetic causes and epigenetic mechanisms of narcolepsy and exploring potential biomarkers for early detection and diagnosis. Long-term studies assessing the effectiveness of different treatment approaches, including pharmacological interventions and behavioral therapies, are needed. Additionally, there is a need for research on strategies to improve the overall well-being and quality of life of individuals living with narcolepsy, including the development of tailored support programs and interventions.

Treatment of multiple traumatized adolescents by enhancing regulation skills and reducing trauma related symptoms: rationale, study design, and methods of randomized controlled trial (the Mars-study)

BackgroundThere is ongoing debate regarding the treatment of severe and multiple traumatized children and adolescents with post-traumatic stress disorder (PTSD). Many clinicians favor a phase-based treatment approach (i.e., a stabilization phase prior to trauma-focused therapy) over immediate trauma-focused psychological treatment, despite the lack of scientific evidence. Research on the effects of different treatment approaches is needed for children and adolescents with (symptoms of complex) PTSD resulting from repeated sexual and/or physical abuse during childhood.ObjectiveThis paper describes the rationale, study design, and methods of the MARS-study, a two-arm randomized controlled trial (RCT) that aims to compare the results of phase-based treatment with those of immediate trauma-focused treatment and determine whether immediate trauma-focused treatment is not worse than phase-based treatment in reducing PTSD symptoms.MethodsParticipants are individuals between 12 and 18 years who meet the diagnostic criteria for PTSD due to repeated sexual abuse, physical abuse, or domestic violence during childhood. Participants will be blindly allocated to either the phase-based or immediate trauma-focused treatment condition. In the phase-based treatment condition, participants receive 12 sessions of the Dutch version of Skill Training in Affective and Interpersonal Regulation (STAIR-A), followed by 12 sessions of EMDR therapy. In the immediate trauma-focused condition, the participants receive 12 sessions of EMDR therapy. The two groups are compared for several outcome variables before treatment, mid-treatment (only in the phase-based treatment condition), after 12 trauma-focused treatment sessions (post-treatment), and six months post-treatment (follow-up). The main parameter is the presence and severity of PTSD symptoms (Clinician-Administered PTSD Scale for Children and Adolescents, CAPS-CA). The secondary outcome variables are the severity of complex PTSD symptoms (Interpersonal Problems as measured by the Experiences in Close Relationship-Revised, ECR-RC; Emotion Regulation as measured by the Difficulties in Emotion Regulation Scale, DERS; Self Esteem as measured by the Rosenberg Self Esteem Scale, RSES), changes in anxiety and mood symptoms (Revised Anxiety and Depression Scale; RCADS), changes in posttraumatic cognitions (Child Posttraumatic Cognitions Inventory, CPTCI), changes in general psychopathology symptoms (Child Behavior Checklist, CBCL), and Quality of Life (Youth Outcome Questionnaire, Y-OQ-30). Furthermore, parental stress (Opvoedingsvragenlijst, OBVL) and patient-therapist relationship (Feedback Informed Treatment, FIT) will be measured, whereas PTSD symptoms will be monitored in each session during both treatment conditions (Children’s Revised Impact of Event Scale, CRIES-13).DiscussionTreating (symptoms of complex) PTSD in children and adolescents with a history of repeated sexual and/or physical abuse during childhood is of great importance. However, there is a lack of consensus among trauma experts regarding the optimal treatment approach. The results of the current study may have important implications for selecting effective treatment options for clinicians working with children and adolescents who experience the effects of exposure to multiple interpersonal traumatic events during childhood.Trial registrationsThe study was registered on the “National Trial Register (NTR)” with the number NTR7024. This registry was obtained from the International Clinical Trial Registry Platform (ICTRP) and can be accessed through the ICTRP Search Portal (https://trialsearch.who.int/).

Evaluation of the effectiveness of different treatment approaches in preventing coronal discoloration caused by regenerative endodontic treatment.

This study aimed to evaluate the effectiveness of Teethmate desensitizer, a dentin bonding agent (DBA), Nd:YAG laser, and Er:YAG laser, which provides dentin tubule occlusion in the pulp chamber with different mechanisms, in preventing tooth discoloration due to regenerative endodontic treatment. One hundred five extracted maxillary human incisors with single roots and single canals were included in the study. The apical third of each tooth was resected below the enamel-cementum junction (CEJ) to obtain a standard root length as 10 ± 1mm. Root canal preparation was performed using the ProTaper Next files up to X5. Root canals were prepared with Gates Glidden (# 2-4) burs to simulate the immature root apex and an apical diameter of 1.1 ± 0.1mm was obtained. The teeth were randomly divided into 7 groups (n = 15): DBA, Teethmate, Nd:YAG, Er:YAG, Biodentine, Blood, and Negative Control. Relevant dentin tubule occlusion methods were applied to DBA, Teethmate, Nd:YAG, and Er:YAG groups. Following dentin tubule occlusion procedures, Biodentine was placed on the blood clot after filling the root canals with blood up to 4mm below the CEJ. No dentin tubule occlusion procedure was applied for Blood and Biodentine groups. Color measurement was performed with the spectrophotometer Vita Easyshade Advance before treatment, immediately after treatment, and at days 7, 30, and 90. Data were converted to L*a*b color values of Commission International de I'Eclairage (CIE L*a*b) and ΔE values were calculated. Two-way ANOVA and post hoc Tukey test (p = 0.05) were performed for statistical analysis. A clinically detectable color change was observed in all groups except for the negative control (ΔE ≥ 3,3). It was observed that Biodentine used alone has a potential for discoloration. It was determined that as the contact time with blood increased, tooth discoloration increased. However, no significant difference was found between dentin tubule occlusion methods in preventing color change (p > 0.05). It was determined that no dentin tubule occlusion method could 100% prevent discoloration caused by RET. DBA and Teethmate, which do not have a significant difference in terms of preventing color change, are considered to be suitable for dentin tubule occlusion due to their ease of application and low cost compared to Nd:YAG laser and Er:YAG laser.

Cost-Effectiveness Analysis of Ultra-Short Whole Breast Radiation Therapy Alone vs. Endocrine Only or Combined Treatment for Low-Risk ER-Positive Early-Stage Breast Cancer in Women Age 70 and Older

<h3>Purpose/Objective(s)</h3> The optimal management of early-stage, low-risk, hormone-positive breast cancer in women age 70 and older remains controversial. Recent trials have shown whole-breast radiation courses of only 5 fractions have similar outcome as longer courses, reducing the cost and inconvenience of treatment. We therefore performed a cost-utility analysis to compare ultra-short whole breast irradiation (U-WBI) to endocrine therapy (ET) alone and to combined ET and U-WBI. <h3>Materials/Methods</h3> We simulated the cost-effectiveness and acute and late effects of different treatment approaches for women age 70 years or older with pT1-2N0 ER-positive invasive ductal carcinoma treated with lumpectomy with negative margins using a Markov microsimulation model. The strategies were: U-WBI performed with a 3D technique over 5 fractions without a boost ("RT Alone"), adjuvant ET (anastrozole for 5 years) without RT ("AI Alone"), or the combination of the two. The FAST-Forward trial served as the basis for this analysis. The combination strategy was calibrated to match the trial results. The relative effectiveness of the RT Alone and AI Alone strategies were inferred from historical randomized trials. Costs were obtained from 2022 Medicare rates. Utilities and other costs were obtained from the literature. The primary endpoint was the cost-effectiveness of the 3 strategies over a 10-year horizon as measured by the incremental cost-effectiveness ratio (ICER), with a value of $100,000/QALY deemed "cost-effective". Deterministic and probabilistic sensitivity analyses were performed to evaluate parameter uncertainty. <h3>Results</h3> The model was validated using 500,000 simulated patients, each treated with the 3 strategies. The model results agreed well with the pre-specified target outcomes. On average, RT Alone was the least expensive strategy ($15,200), with AI Alone slightly more expensive, ($16,761), and combination therapy the most costly ($20,416). The most effective strategy was the combination (9.497 QALY), with small differences between RT Alone (9.453) and AI Alone (9.420). Therefore, the AI Alone strategy was more costly and less effective than RT Alone (dominated), and the combination therapy was slightly more expensive than the usual definition of "cost-effective" (ICER $117,721 relative to RT Alone). Probabilistic sensitivity analysis demonstrated RT Alone to be cost-effective in 52% of trials, with combination in 35% and AI Alone in 13%. <h3>Conclusion</h3> U-WBI without ET represents a cost-effective strategy for low-risk women in the United States, with slightly higher QALY outcome compared to AI Alone. Combination therapy improves outcome very modestly. Hence, many patients may prefer RT Alone in order to avoid the possible side effects of ET. Discussion of these options must account for patients' weighing of these issues.

All-Cause Mortality in People with Co-Occurring Insomnia Symptoms and Sleep Apnea: Analysis of the Wisconsin Sleep Cohort.

PurposeInsomnia symptoms and sleep apnea frequently co-occur and are associated with worse sleep, daytime function, mental health and quality of life, compared to either insomnia or obstructive sleep apnea (OSA) alone. This study aimed to investigate the association of symptoms of co-morbid insomnia and sleep apnea (COMISA) with all-cause mortality.Patients and MethodsWisconsin Sleep Cohort data were analysed to assess potential associations between COMISA symptoms and all-cause mortality. Nocturnal insomnia symptoms were defined as difficulties initiating sleep, maintaining sleep, and/or early morning awakenings “often” or “almost always”, and/or regular sedative-hypnotic medicine use. OSA was defined as an apnea-hypopnea index ≥5/hr sleep. Participants were classified as having neither insomnia symptoms nor OSA, insomnia symptoms alone, OSA alone, or COMISA symptoms. Associations between the four groups and all-cause mortality over 20 years of follow-up were examined via multivariable adjusted Cox regression models.ResultsAmong 1115 adult participants (mean ± SD age 55 ± 8 years, 53% males), 19.1% had COMISA symptoms. After controlling for sociodemographic and behavioral factors, COMISA symptoms were associated with an increased risk of all-cause mortality compared to no insomnia symptoms or OSA (HR [95% CI]; 1.71 [1.00–2.93]). OSA alone (0.91 [0.53, 1.57]) and insomnia symptoms alone (1.04 [0.55, 1.97]) were not associated with increased mortality risk.ConclusionCo-morbid insomnia symptoms and sleep apnea is associated with increased all-cause mortality risk. Future research should investigate mechanisms underpinning COMISA and the effectiveness of different treatment approaches to reduce mortality risk for this common condition.

Comparative effect of different treatment approaches on lumbar hyperlordosis, respiratory muscle strength, and balance in diplegic children: A randomized controlled clinical trial

The aim of this study was to compare the effect of different treatment approaches (pressure biofeedback training of the abdominal drawing-in maneuver with and without prone hip extension, whole-body vibration, and core stability exercises) on lumbar hyperlordosis, respiratory muscle strength, and balance in children with diplegic cerebral palsy. Forty-five children, ranged in age from 8 to 12 years, were randomly allocated into three groups (A, B, C). All children underwent a specialized exercise program for one hour, three days/ week for three successive months. In addition, group A received pressure biofeedback training, group B underwent the whole-body vibration, and group C underwent the core stability exercises. The lumbar lordotic angle, active lumbar and hip range of motion, respiratory muscle strength, and balance were measured before and after three months of treatment. We found a significant decrease in lumbar lordotic angle along with a significant increase in active lumbar and hip range of motion of group A and group C post-treatment (p &lt; 0.001) in favor of group A, while non-significant change was found in group B (p &gt; 0.05). A significant improvement was recorded in the three groups' respiratory muscle strength and stability indices in favor of group B (p &lt; 0.001). Pressure biofeedback training and core stability exercises are the best adjunctive to traditional physical therapy to improve lumbar hyperlordosis, respiratory muscle strength, and balance together in children with diplegic cerebral palsy. Moreover, whole-body vibration is the best adjunctive to improve respiratory muscle strength and balance.

3D assessment of facial contours of patients wearing either complete denture or implant-supported fixed dentures.

Total edentulousness leads to soft tissue changes causing esthetic problems. Being aware of the significant effects of different treatment approaches on facial soft tissue can help to obtain more satisfying esthetic results. The current study's objective was to evaluate three-dimensional facial soft tissue changes in edentulous patients rehabilitated with a complete denture (CD) and implant-supported fixed partial denture (FPD). Fourteen edentulous patients, mean age of 47.6 years (42-63), were assessed before prosthetic treatments (T0), after CD (T1), and after FPD (T2). 3dMDface System (3dMD LLC) obtained the images and transferred them to 3dMD Vultus software (3dMD Vultus software Version 2.3.0.2). Nine linear, five angular, and one topographical measurement were performed for facial soft tissue analysis. For statistical analysis of facial soft tissue, ANOVA was used with a level of significance set at 5% (p <.05). Significant differences were observed in lower lip height (Sto-Sl) and nasolabial angle (Prn Sn Ls) with CD. There is no significant difference with the implant-supported fixed partial dentures in these measurements when compared with pretreatment. Significant differences were observed in the lower lip angle (Chr Li Chl) with the implant-supported fixed partial dentures. There is no significant difference with CD in this measurement when compared with pretreatment. According to the paired comparisons, a significant difference was observed in philtrum height (Ls-Sn) between the treatment approaches. CD and implant-supported fixed partial dentures, both treatment approaches, cause facial soft tissue changes, which may help to improve the facial esthetic. The effects of these treatment methods on facial soft tissues are not significantly different, except the philtrum height.

Multidisciplinary approach to the diagnosis and treatment of patients with potentially resectable colorectal cancer liver metastasis: results of a multicenter study.

This study sought to review colorectal cancer liver metastasis (CRLM) patients at multiple centers to analyze the factors affecting the success of conversion therapy in patients whose CRLM was initially evaluated as potentially resectable, to explore the effect of different treatment approaches on patient survival, and to provide a scientific reference for clinical treatment of CRLM. Fifty patients whose CRLM was initially evaluated as potentially resectable at 3 large Chinese general hospitals were enrolled in this retrospective study. Statistical analyses were carried out on the general data and pathological characteristic data to examine the clinical efficacy of the treatment approaches. The factors affecting the success of conversion therapy were analyzed by logistic regression. Additionally, follow-up appointments were conducted to examine survival, and survival curves were plotted using the Kaplan-Meier estimator. The effect of different clinical and pathological characteristics on CRLM patients was analyzed. Seventeen patients achieved no evidence of disease (NED) status through surgical resection/ablation after undergoing conversion therapy. The multifactor analysis demonstrated that the number of liver metastases was the primary risk factor affecting the efficacy of conversion therapy (P<0.05). Survival analysis results showed statistically significant difference in overall survival (OS) between the NED group and theinconspicuous/progressivegroup (P<0.0001). Also, there was a statistically significant difference in the progression-free survival (PFS) between the NED group and theinconspicuous/progressive group (P<0.0001). Patients in the surgical resection group had better OS and PFS than those in the ablation group (P<0.0001 and P<0.01, respectively). The monofactor analysis demonstrated that the number and maximum diameter of liver metastases, serum Carcino-Embryonic Antigen (CEA) level, and BRAF V600E mutation status were factors affecting the OS of CRLM patients (P<0.05), of which BRAF V600E mutation was the primary determinant (P<0.05). Among the patients whose CRLM was initially evaluated as unresectable, those who underwent surgical resection of the primary lesions and liver metastases after receiving conversion therapy had the best prognosis. Thus, a thorough evaluation should be conducted to determine the effect of and survival factors affecting conversion therapy in the treatment of liver metastases.

Treatment of anastomotic leakage after rectal cancer resection: The TENTACLE–Rectum study

AimAnastomotic leakage is a severe complication after low anterior resection (LAR) for rectal cancer and occurs in up to 20% of patients. Most research focuses on reducing its incidence and finding predictive factors for anastomotic leakage. There are no robust data on severity and treatment strategies with associated outcomes. The aims of this work were (1) to investigate the factors that contribute to severity of anastomotic leakage and to compose an anastomotic leakage severity score and (2) to evaluate the effects of different treatment approaches on prespecified outcome parameters, stratified for severity score and other leakage characteristics.MethodTENTACLE–Rectum is an international multicentre retrospective cohort study. Patients with anastomotic leakage after LAR for primary rectal cancer between 1 January 2014 and 31 December 2018 will be included by each centre. We aim to include 1246 patients in this study. The primary outcome is 1‐year stoma‐free survival (i.e. patients alive at 1 year without a stoma). Secondary outcomes include number of reinterventions and unplanned readmissions within 1 year, total length of hospital stay, total time with a stoma, the type of stoma present at 1 year (defunctioning, permanent), complications related to secondary leakage and mortality. For aim (1) regression models will be used to create an anastomotic leakage severity score. For aim (2) the effectiveness of different treatment strategies for leakage will be tested after correction for severity score and leakage characteristics, in addition to other potential related confounders.ConclusionTENTACLE–Rectum will be an important step towards drawing up evidence‐based recommendations and improving outcomes for patients who experience severe treatment‐related morbidity.

Psychometric properties of the Czech versions of the Impact of Pediatric Epilepsy Scale (IPES) and Quality of Life in Epilepsy Inventory for Adolescents (QOLIE-AD-48)

ObjectiveIn order to introduce a complex system of monitoring and evaluation of the heath-related quality of life (HRQoL) of children and adolescents with epilepsy (CWE) and their families in the Czech Republic, we aimed to validate the Czech versions of two appropriate instruments – the Impact of Pediatric Epilepsy Scale (IPES) and the Quality of Life in Epilepsy Inventory for Adolescents (QOLIE-AD-48). MethodsThe verification of the 11-item IPES was carried out in the group of parents of 248 CWE aged 2–18 years. One hundred and thirty-five CWE from the given group aged 11–18 years then completed the 48-item QOLIE-AD-48. Internal consistency, test–retest reliability (with a three-month interval) and the factorial structure of the Czech versions were determined and compared with the original instruments. ResultsWe found that the Czech version of the IPES exhibited very good psychometric properties including high internal consistency (Cronbach’s alpha, α, of 0.93), high test–retest reliability (intraclass correlation coefficient, ICC, of 0.76) and the same 3-factor structure as the original instrument. The superiority of this 3-factor solution over the alternate 2-factor model proposed for some language versions of the IPES was determined using confirmatory factor analysis. We found 8 items in the Czech version of the QOLIE-AD-48 belonging to original Attitudes towards epilepsy and Social support subscales that do not fit well with the Czech version due to their low correlation with the total score and insufficient test–retest reliability and should be omitted. For the remaining 40 items, we have determined high internal consistency (α = 0.95) and test–retest reliability (ICC = 0.82). Confirmatory factor analysis revealed that the 6-factor solution derived from the original instrument (without two removed subscales) was appropriate for the Czech version. The individual subscales exhibited high internal consistency with α = 0.61–0.91. The external validation of both instruments was confirmed based on a significant correlation between test results and physicians’ reports of the characteristics of the child’s epilepsy. ConclusionsThe Czech versions of both instruments studied are reliable and valid, and can be used in the next research focusing on the effect of different treatment approaches on the HRQoL of CWE and their families.

PB2078 A RELATIVE ADVANTAGE OF STEM CELL TRANSPLANTATION (SCT) IN PATIENTS WITH LESS ADVANCED MYELODYSPLASTIC DSYNDROMES (MDS)

Background:An optimal treatment approach to patients with less advanced MDS is still a question of debate. We present results of a long term retrospecive study of outcome of MDS patients treated in a single center with different therapeutic modalities.Aims:A long term effect of different treatment approaches was evaluated in patients with RA or RARS subtype of MDS (according to the initial FAB classification) or with MDS‐SLD,MDS‐MLD,MDS‐RS or MDS with isolated del(5q) (according to WHO 2016 classification).MethodsThe data obtained from a long‐term follow up for a period of 30 years (1988‐2018) were analyzed in a group of 174 patients with primary myelodysplastic syndrome (MDS) and factors affecting prolonged survival were detected using different statistical methods including Kaplan Maier plots and multivariate analysis.Results:A median survival of untreated RA and RARS patients (43.5 and 48.3 months respectively) was significantly different from that in patients with advanced MDS (3.4 months) as well as estimated 3 years survival of 60.3% for RA vs. 58.3% for RARS vs. 0 months for advanced disease. An analysis of different treatment approaches revealed median survival of 43.8 months in 150 patients treated with best supportive care only (BSC) (median age 61.0 years) in comparison to 83.0 months in 53 transplanted patients (median age 40.0 years) (p &lt; 0.01 for both age and survival). However, estimated 3 years survival was not statistically different between BSC and SCT treated patients (59.3 vs. 67.9%). A difference became significant after 5 years (43.3% for BSC vs. 54.7% for SCT and after 10 years of follow‐up (26% for BSC vs. 49.1% for SCT). The explanation of the differences may be a relatively high rate of transplantation related mortality (30.2%) on one hand and increasing rate of mortality after 5 years of follow‐up in BSC patients (20.7% vs. 3.8% in SCT patients) on the other hand. The reason of death after 5 years in BSC patients was mostly related to complications not directly connected with MDS, however, a late progression of the disease was observed in 9 BSC treated patients (63.5‐376.5 months after diagnosis of MDS). Two transplanted patients developed secondary malignancy 5 and 8 years after SCT. A significant difference in survival was observed between patients transplanted with &lt; 5% bone marow blasts and those transplanted at the time of initial progression of the disease (111.5 months vs. 26.0 months, p = 0.001). A specific subgroups of patients responded to special treatment modalities. Estimated 5 years survial was 63.6% for patients with sEPO &lt; 100 IU/l and &lt; 2 TU of RBC/month treated with rHuEPO, 75.0% for patients with isolated del(5q) treated with lenalidomide and 83.3% for patients with hypoplastic MDS tretaed with ATG.Summary/Conclusion:The results of our retrospective analysis showed that younger patients with less advanced MDS without excess of blasts and adverse prognostic factors (profound cytopenia with transfusion dependency and signs of bone marrow failure, bone marrow fibrosis, adverse cytogenetics) should be transplanted as soon as possible after diagnosis before disease progression. A more conservative approach to patients with early disease who are not indicated for SCT was justified by a relatively high number of patients surviving 5 years with supportive care only.

Social decision making in autism: On the impact of mirror neurons, motor control, and imitative behaviors.

SummaryThe Mirror Neuron System (MNS) plays a crucial role in action perception and imitative behavior, which is suggested to be impaired in Autism Spectrum Disorders (ASDs). In this review, we discuss the plausibility and empirical evidence of a neural interaction between the MNS, action perception, empathy, imitative behavior, and their impact on social decision making in ASDs. To date, there is no consensus regarding a particular theory in ASDs and its underlying mechanisms. Some theories have completely focused on social difficulties, others have emphasized sensory aspects. Based on the current studies, we suggest a multilayer neural network model including the MNS on a first layer and transforming this information to a higher layer network responsible for reasoning. Future studies with ASD participants combining behavioral tasks with neuroimaging methods and transcranial brain stimulation as well as computational modeling can help validate and complement this suggested model. Moreover, we propose applying the behavioral paradigms, and the neurophysiological markers mentioned in this review article for evaluating psychiatric treatment approaches in ASDs. The investigation of modulating effects of different treatment approaches on the neurophysiological markers of the MNS can help find specific subgroups of ASDs patients and support tailored psychiatric interventions.

Early estimates of epidemic final sizes

ABSTRACTEarly in a disease outbreak, it is important to be able to estimate the final size of the epidemic in order to assess needs for treatment and to be able to compare the effects of different treatment approaches. However, it is common for epidemics, especially of diseases considered dangerous, to grow much more slowly than expected. We suggest that by assuming behavioural changes in the face of an epidemic and heterogeneity of mixing in the population it is possible to obtain reasonable early estimates.

Consensus in Traditional Chinese Medical Diagnosis in Open Populations.

Objectives: An acceptable level of diagnostic agreement is a prerequisite for consistent administration of treatment. It is critical for investigating effectiveness of different treatment approaches using multiple practitioners. To the best of our knowledge, no previous investigation of diagnostic consensus using open populations in Chinese medicine (CM) has been reported. Investigations restricted to individual medical conditions, such as have been usually studied, do not reveal any information as to what occurs in real world clinical settings. This knowledge gap led to the current study being conducted. Design/Location/Subjects/Interventions: Investigating diagnostic agreement specifically in Traditional Chinese Medicine (TCM) in an open population, two or three practitioners diagnosed 35 subjects at the University of Technology, Sydney (UTS), TCM clinic. The practitioners were restricted to a list of the 56 most frequently used TCM diagnoses at the UTS clinic. Up to three diagnostic patterns per subject could be selected, with nominated patterns scored between 1 and 5. Outcome measures: Agreement was determined with two criteria, both expressed as simple percentages: pattern and linearly weighted agreements. Results: The results showed that 23% of practitioners obtained pattern agreement, while 19% demonstrated weighted agreement. Conclusion: There appears to be very low diagnostic agreement between practitioners. This is an important finding. If unchallenged by further investigation, the recognition of such poor diagnostic consensus may lead to rejection of TCM theory before it has been adequately assessed. Diagnostic agreement must be improved so that future investigations into treatment effectiveness or mechanisms of action are made on a valid basis. Additionally, the current TCM diagnostic format must be altered to allow the application of chance-removed statistics or the calculation of a standard error with open populations. This article is the first of a series of three that report problems in TCM diagnostic reliability and proposes solutions to the issues outlined.

Effects of combined therapies on the survival of pleural mesothelioma patients treated in Brescia, 1982-2006.

During the 1990's, the traditional unimodal treatments (surgery, radiotherapy, chemotherapy, immunotherapy) for malignant pleural mesothelioma started to be combined in bimodal or multimodal strategies. However, recent population-based analyses of the survival of patients with malignant pleural mesothelioma indicate that even these treatments have not led to significant improvements in prognosis, which remains very poor. The present study assessed the survival of patients given combined treatments and multimodal therapies in a specialized hospital department. The study population comprised 530 patients diagnosed with malignant pleural mesothelioma from 1982 to 2006: 343 of them were residents in the province of Brescia (Lombardy, Northern Italy) and 187 were residents outside the province, with a follow-up to 31 December 2009. Kaplan-Meier survival analyses and Cox proportional risks model were used to test sex, age at diagnosis, histological type and treatments, as prognostic factors. The estimated median survival for the whole group of patients was 317 days (257 for residents and 398 for non-residents), and respectively 310 and 340 days in the groups diagnosed in the periods 1982-2000 and 2001-2006. Multivariate analysis confirmed that the prognosis was better for younger patients and cases of epithelioid type malignant pleural mesothelioma, whereas for patients receiving any single treatment the prognosis was not significantly better than for those given palliative care alone. However, patients receiving combined treatments or the multimodality approach had significantly longer median survival and the relative risk of death was respectively 0.57 and 0.61 compared to untreated patients (or those only given symptomatic therapy). This is the first study in Italy to assess the effectiveness of different treatment approaches in a significant number of patients treated in one hospital. Further studies are needed to confirm the improvement in prognosis - even if modest--on larger numbers of patients and taking into account the different stages of the disease.