Health-related Quality Of Life Parameters Research Articles

Safety and efficacy of monthly high-dose vitamin D3 supplementation in children and adolescents with sickle cell disease.

Little is known about the impact of vitamin D supplementation on hand grip strength (HGS) and health-related quality of life (HRQoL) in children and adolescents with sickle cell disease (SCD). We aimed to evaluate the safety and efficacy of monthly high-dose vitamin D3 supplementation and its implications on bone mineral density (BMD), HGS, and HRQoL in patients with SCD and healthy controls. The study included 42 children with SCD and 42 healthy matched controls. The study participants were supplemented with high-dose monthly oral vitamin D3. Changes in the serum level of 25(OH) vitamin D3, maximum HGS, and BMD from baseline to 6months were assessed, and the HRQoL questionnaire and Childhood Health Assessment Questionnaire (CHAQ) were used to evaluate the functional capacity. At baseline, SCD subjects had poorer growth status indicated by negative Z scores. Suboptimal BMD was detected by significantly lower Z score, and lower HGS and worse HRQL parameters were found compared to the controls (P < 0.001). Median 25(OH) vitamin D3 was significantly lower in SCD patients compared to controls (16.5 vs. 28ng/mL, respectively (P < 0.001)). After 6months of vitamin D supplementation, there was significant improvement in the DEXA Z-score (P < 0.001), limitation of physical health (P = 0.02), pain scores (P < 0.001), and CHAQ grades (P = 0.01) in SCD patients. A significant improvement in HGS (P < 0.001 and P = 0.005) as well as the CHAQ score (P < 0.001 and P = 0.003) was detected in the SCD group and controls, respectively. There were no reported clinical adverse events (AEs) or new concomitant medications (CMs) during the study duration, and safe levels of Ca and 25 (OH) D3 were observed at 3 and 6months for both groups. There was a significant positive correlation between HGS and total physical score (r = 0.831, P < 0.001) and a negative correlation with CHAQ score (r = - 0.685, P < 0.001). We also detected a significant positive correlation between vitamin D levels at 6months and HGS (r = 0.584, P < 0.001), pain score (r = 0.446, P < 0.001), and a negative correlation with CHAQ score (r = - 0.399, P < 0.001). Conclusion: Monthly oral high-dose vitamin D supplementation was safe and effective in improving vitamin D levels, HGS, and HRQoL in SCD children and healthy subjects, and BMD scores in SCD patients. Further randomized controlled trials are warranted to assess an optimal dosing strategy and to investigate the impact on clinically significant outcomes in children and adolescents with SCD and their healthy counterparts. Trial registration: ClinicalTrials.gov , identifier NCT06274203, date of registration: 23/02/2024, retrospectively registered. What is known: • Several studies have reported a high prevalence of vitamin D deficiency and suboptimal bone mineral density (BMD) in sickle cell disease (SCD) patients. • Musculoskeletal dysfunction is reported in SCD patients with a negative impact on physical activity and health-related quality of life (HRQL). • Little is known regarding the impact of vitamin D3 supplementation in children and adolescents with SCD. What is new: • We found that monthly oral high-dose vitamin D3 supplementation was safe, tolerated, and effective in improving serum vitamin D levels, HGS, BMD scores, and HRQL in SCD patients.

The changes in health-related quality of life in stable coronary artery disease patients and intermediante coronary lesions after elective percutaneous coronary intervention

The aim – to analyze the factors, associated with the change in health-related quality of life (HRQoL) in stable coronary artery disease (SCAD) patients with intermediate coronary lesions at 3-months follow-up after elective percutaneous coronary intervention (PCI).Materials and methods. A prospective single-center study enrolled 84 SCAD patients (mean age 64±8 years; males – 73 %) with intermediate coronary lesions (stenosis 50-90 %) (by invasive coronary angiography [ICA]), consecutively enrolled and hospitalized with the aim of elective PCI to be performed. HRQoL was evaluated by SF-36 (physical health summary [PHsum] and mental health summary [МНsum]) and SAQ (Seattle Angina Questionnaire»; the domains «physical limitation» [PL], «angina stability» [AS], «angina frequency» [AF], «treatment satisfaction» [TS] and «disease perception» [DP]), – at baseline and 3-months follow-up. The enrolled sample was subdivided in a dichotomous manner into the HRQoL «improvement» or «non-improvement» patterns, based on the cut-off-values (COVs) of clinically meaningful change of the certain HRQoL parameter score at 3-months follow-up after PCI.Results and discussion. We revealed an improvement of average (median) HRQoL parameters at 3-months follow-up after PCI, namely PHsum (score increase from 38.8 to 43.1; р &lt; 0.001), SAQ-AS (25 to 75; р &lt; 0.001), SAQ-AF (70 to 80; р &lt; 0.001), SAQ-TS (58.8 to 70.6; р &lt; 0.001) and SAQ-DP (45.8 to 66.7; р &lt; 0.001). The clinically meaningful HRQoL improvement ranged from 14 % to 71 % cases, depending on the certain HRQoL parameter and the COVs of its change at follow-up after PCI. The worse baseline HRQoL, older age and higher baseline total cholesterol level were the factors, associated with the increased probability of HRQoL to be meaningfully improved after revascularization. In addition, a presence of diabetes mellitus, a history of acute cerebrovascular accident, and the ICA data on multivessel disease and in-stent restenosis after previous PCI, – all were associated with the lower odds of HRQoL «improvement» status to be achieved.Conclusions. A PCI favored the HRQoL improvement in SCAD patients at 3-months follow-up. The clinical and angiographic factors, associated with the post-procedure change in HRQoL, should be considered for a better selection of SCAD patients with intermediate coronary lesions for PCI.

Prospective Analysis of Radiation-Induced Contrast Enhancement and Health-Related Quality of Life After Proton Therapy for Central Nervous System and Skull Base Tumors

Intracerebral radiation-induced contrast enhancement (RICE) can occur after photon as well as proton beam therapy (PBT). This study evaluated the incidence, characteristics, and risk factors of RICE after PBT delivered to, or in direct proximity to, the brain and its effect on health-related quality of life (HRQoL). Four hundred twenty-one patients treated with pencil beam scanning PBT between 2017 and 2021 were included. Follow-up included clinical evaluation and contrast-enhanced magnetic resonance imaging at 3, 6, and 12 months after treatment completion and annually thereafter. RICE was graded according to Common Terminology Criteria for Adverse Events version 4, and HRQoL parameters were assessed via European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ)-C30 questionnaires. The median follow-up was 24 months (range, 6-54), and median dose to 1% relative volume of noninvolved central nervous system (D1%CNS) was 54.3 Gy relative biologic effectiveness (RBE; range, 30-76 Gy RBE). The cumulative RICE incidence was 15% (n = 63), of which 10.5% (n = 44) were grade 1, 3.1% (n = 13) were grade 2, and 1.4% (n = 6) were grade 3. No grade 4 or 5 events were observed. Twenty-six of 63 RICE (41.3%) had resolved at the latest follow-up. The median onset after PBT and duration of RICE in patients in whom the lesions resolved were 11.8 and 9.0 months, respectively. On multivariable analysis, D1%CNS > 57.6 Gy RBE, previous in-field radiation, and diabetes mellitus were identified as significant risk factors for RICE development. Previous radiation was the only factor influencing the risk of symptomatic RICE. After PBT, general HRQoL parameters were not compromised. In a matched cohort analysis of 54/50 patients with and without RICE, no differences in global health score or functional and symptom scales were seen. The overall incidence of clinically relevant RICE after PBT is very low and has no significant negative effect on long-term patient QoL.

Efficacy and Safety of the Anti-Tissue Factor Pathway Inhibitor Marstacimab in Participants with Severe Hemophilia without Inhibitors: Results from the Phase 3 Basis Trial

Background:Marstacimab (PF-06741086) is a monoclonal antibody targeted to the tissue factor pathway inhibitor protein to improve hemostasis via the extrinsic pathway of blood coagulation. Previous phase 1/2 studies demonstrated the efficacy and safety of long-term administration of marstacimab up to 450 mg weekly for reducing bleeding episodes in adults with severe hemophilia A (HA) or hemophilia B (HB), with or without inhibitors, compared with on-demand (OD) therapy. We evaluated the efficacy and safety of marstacimab in participants with severe HA or moderately severe to severe HB without inhibitors compared with previous factor replacement therapy. Methods: BASIS (NCT03938792) is an open-label, multicenter, pivotal phase 3 study that enrolled male participants aged ≥12 to ˂75 y with severe HA (factor [F] VIII ˂1%) or moderately severe to severe (FIX ≤2%) HB, with or without inhibitors. Following screening, participants entered a 6-month observational phase (OP) and were categorized by factor replacement treatment: (1) OD or (2) routine prophylaxis (RP). Participants who completed the OP crossed over to 12-month active treatment phase (ATP) and received a single subcutaneous loading dose of 300 mg followed by once weekly 150 mg marstacimab. Primary endpoints were annualized bleeding rate (ABR) for treated bleeds and safety outcomes. Secondary endpoints included incidence of various types of breakthrough bleeds and health-related quality of life (HRQoL) measures. Participants who completed the ATP were eligible to enroll in the long-term extension (LTE) study. Informed consent/ethics committee approvals were obtained. Results for participants without inhibitors are presented. Results: Participants (N=128; 108 adults, 20 adolescents) with HA or HB without inhibitors entered the OP (OD: HA n=29, HB n=8; RP: n=72, HB n=19); of these, 116 entered the ATP. The median age was 30 [range, 13-66] y, most participants were White (50.8%) or Asian (47.7%) and predominately from Europe and India (51.6%). At baseline, 89 participants (69.5%; OD: n=36; RP: n=53) had ≥1 target joint. Mean (range) duration of marstacimab treatment was 12.1 (11.5-13.1) months for OD and 11.6 (0.9-12.8) months for RP. Eighty-eight participants entered the LTE (OD: HA n=22, HB n=7; RP: HA n=45; HB n=13). Data were not available by the cutoff date for 1 RP participant and was not included in the LTE safety analysis set. The mean (range) treatment duration in the LTE was 8.0 (1.2-14.5) months for OD and 6.5 (1.1-16.1) months for RP. In the phase 3 study, the OD group reported 12 (36.4%) adverse events (AEs) during ATP vs 9 (24.3%) in OP whereas the RP group reported 62 (74.7%) AEs in ATP vs 20 (22.0%) in OP. Both groups reported more treatment-related AEs during ATP ( Table 1). ADAs developed in 23/112 participants (20.5% incidence), of which titers were low and resolved in 22 participants by end of study. One RP participant discontinued due to a non-treatment-related SAE, and no deaths or thromboembolic events were recorded in the phase 3 study or the LTE. Mean (95% CI) ABR for treated bleeds was reduced for OD (91.6% [88.1-94.1%]) and RP (35.2% [5.6-55.6%]) participants over the 12-month ATP and marstacimab demonstrated superiority vs OD (P&amp;lt;0.001) and non-inferiority and superiority vs RP (P=0.0376) therapy. Marstacimab was also associated with significant reductions in ABR across all breakthrough bleed categories vs OD, and numerical reductions vs RP (non-inferiority). Overall, mean ABR declined over the first 6 months of ATP, which continued to Month 12 (data not shown). Bleed rates for an additional 16 months of follow-up in the LTE were consistent with those observed during the first 12 months of treatment in the phase 3 study ( Table 2). The ABR reductions observed with marstacimab during ATP were consistent across hemophilia types and age groups for OD and were generally consistent across hemophilia types and age groups for RP, with all point estimates for a difference &amp;lt;2.5 (non-inferiority margin for the ABR of treated bleeds). HRQoL parameters demonstrated non-significant improvements vs OD therapy and non-inferiority vs RP therapy. Conclusion: Compared with previous OD or RP therapy, once weekly subcutaneous marstacimab was safe and effective for reducing bleeding events in participants with severe HA or moderately severe to severe HB without inhibitors beyond 12 months in the phase 3 study and up to an additional 16 months in the LTE study.

INNV-17. TREATMENT DECISION AND QUALITY OF LIFE OF PATIENTS WITH NEWLY DIAGNOSED GLIOBLASTOMA RECEIVING TUMOR TREATING FIELDS (TTFIELDS) THERAPY IN ROUTINE CLINICAL CARE: RESULTS FROM THE TIGER STUDY

Abstract BACKGROUND TTFields therapy, concurrent with temozolomide, is approved for treatment of newly diagnosed glioblastoma (ndGBM), following results from the pivotal EF-14 study. The multicenter, prospective TIGER study (NCT03258021) investigated real-world use of TTFields therapy in patients with ndGBM during routine clinical care in Germany. Data on patient treatment decisions and health-related quality of life (HRQoL) are reported. METHODS Reasons for patient decision to receive TTFields therapy were evaluated via questionnaire (scoring scale: 1 [not at all] to 5 [strongly]) at baseline. In patients who decided to receive TTFields therapy, HRQoL was assessed via the EORTCQLQC30/BN20 questionnaire at baseline, and 2 and 4 months after initiating therapy. HRQoL parameters included: global health status and functioning, insomnia, headaches, fatigue, nausea/vomiting, pain, itchy skin, hair loss, seizures, dyspnea, appetite loss, constipation, diarrhea, visual disorder, leg weakness, drowsiness, bladder control, motor dysfunction, communication deficit, future uncertainty, and financial difficulties. RESULTS Overall, 710 patients (259 female; 451 male) were enrolled (Aug 2017–Nov 2019). Mean age was 59 years (range: 19–85). In total, 582 (82%) patients decided to start TTFields therapy, while 128 (18%) declined; the proportion of women was similar in each group (36% and 38%, respectively). Predominant reasons for deciding to start TTFields therapy were the prospect of treatment success (mean score: 4.5) and doing something to fight their disease (4.2). Predominant reason for deciding against TTFields therapy was carrying the device (4.4). During 4-month study follow-up, there was no decline or improvement in any HRQoL parameter, except for more itchy skin, consistent with EF-14 data. CONCLUSION TIGER is the largest non-interventional study to date on TTFields therapy use in routine clinical care. Most patients opted to receive TTFields therapy, with fighting their disease and hope for treatment success as major motivators. HRQoL was not impaired during follow-up, except for skin itchiness.

An economic evaluation of first-line cryoballoon ablation versus antiarrhythmic drug therapy for the treatment of paroxysmal atrial fibrillation from a German healthcare service payer perspective

Abstract Background Three recent randomized controlled trials demonstrated that, in patients with symptomatic paroxysmal atrial fibrillation (PAF), first-line pulmonary vein isolation (PVI) with cryoballoon ablation reduces atrial arrhythmia recurrence when compared to initial antiarrhythmic drug (AAD) therapy. Purpose To evaluate the cost-effectiveness of initial rhythm control therapy from a German healthcare service payer perspective. Methods The cost-effectiveness model (CEM) structure consisted of a hybrid decision tree and Markov model, where the decision tree informed the initial health state allocation in the first cycle of the Markov model and had a one-year time horizon. The Markov model had a 40-year time horizon using a three-month cycle length. Health benefits were expressed in quality-adjusted life years (QALYs). Costs and benefits were discounted at 3.5% p.a. Individual patient-level data from 703 participants with untreated PAF enrolled into Cryo-FIRST (NCT01803438), STOP AF First (NCT03118518) and EARLY-AF (NCT02825979) were used to estimate efficacy, resource use and health-related quality of life parameters. Cost inputs were sourced from diagnosis-related groups and the Institute for the Hospital Remuneration System (InEK). Where parameters could not be derived, inputs were sourced from available published literature or determined through clinical expert opinion. Probabilistic sensitivity analyses were conducted to explore the impact of any assumptions on model outputs. Results In those treated with cryoablation, the three-month rate of AF recurrence was reduced by 46.7% (p&amp;lt;0.001) on average. Similarly, the average monthly rate of receiving an ablation following initial treatment was reduced by 72.8% (p&amp;lt;0.001). Cryoablation was also associated with a 4.3% (p=0.025) increase in health-related quality of life at 12 months, assessed through the standard EQ-5D-3L instrument. There was no difference in the rate of AF resolution in those who failed initial treatment. CEM results are shown in Table 1. Analysis shows that cryoablation is cost-effective, incurring a cost of ∼€1,000 per patient over a lifetime compared to AADs, while offering an increase in QALYs. Cryoablation attains an average ICER of ∼€5,500, with a 94.1% probability of being cost-effective at a willingness-to-pay threshold of €35,000 per QALY gained. Through 5,000 iterations, the probabilistic sensitivity analysis indicates that cryoablation has ∼20% probability of being cost-saving. Individuals are expected to receive a total of ∼1.2 ablations over a lifetime, regardless of initial treatment. Although, those initially treated with cryoablation as opposed to AADs experience a 45% reduction in time spent in AF health states. Conclusion Initial rhythm control with cryoballoon ablation in PAF is a cost-effective treatment option in a German healthcare setting.Table 1:Key results (per patient)

Long-Term Follow-Up of Health-Related Quality of Life and Short-Term Intervention with CFTR Modulator Therapy in Adults with Cystic Fibrosis: Evaluation of Changes over Several Years with or without 33 Weeks of CFTR Modulator Therapy

Background: Longitudinal data on changes in health-related quality of life (HRQoL) in adult people with cystic fibrosis (pwCF) and the longitudinal effects of Elexacaftor/Tezacaftor/Ivacaftor therapy (ETI) on HRQoL or HRQoL domains are currently scarce. This study aimed to investigate the effects of ETI on HRQoL and compare them with those of pwCF who did not receive highly effective CFTR modulators over a longer period. Methods: Baseline assessment and follow-up data for 5.6 years in pwCF with (n = 21) and 6.5 years in pwCF without (n = 6) ETI (≥18 years) were evaluated. The assessment of HRQoL and clinical parameters was identical at both time points. HRQoL was assessed using the CFQ-R, and clinical outcomes included BMI, ppFEV1, and FEV1 z-score. Results: ETI was found to improve all HRQoL domains at more than four points over time, and their increases were significant except for vitality, digestion, treatment burden, and social functioning (p &lt; 0.05). Without ETI, psychosocial domains remained almost constant, whereas most physical domains decreased over time. Conclusions: The results of the present study show that ETI therapy has a positive effect on HRQoL and clinical outcomes over time but not in pwCF without ETI treatment. Furthermore, our results suggest that disease progression over time affects the physical domains of HRQoL more than the psychosocial domains. Due to the small sample size and the heterogeneity of the study population (CFTR mutation genotype), the results should be interpreted with some caution.

Adult spinal deformity correction surgery using age-adjusted alignment thresholds: clinical outcomes and mechanical complication rates. A systematic review of the literature.

Adult spinal deformity (ASD) surgery gives good clinical outcomes but has a high rate of mechanical complications (MC). In 2016, Lafage described the age-adjusted alignment thresholds (AAAT) to adapt the correction in relation to patient's age proposing less aggressive corrections for the elderly population. The aim of this review was to clarify the effectiveness of AAAT to achieve good health-related quality of life (HRQoL) and their relationship with post-operative MC. We performed a review of the literature, including articles reporting data on post-operative HRQoL and MC rates in relation to the AAAT. Data were stratified according to whether they matched the AAAT, dividing the population in undercorrected (U), matched (M) and overcorrected (O). The quality of the included studies was assessed using the GRADE and MINORS systems. Six articles reporting data from 1,825 patients were included. The different categories (U, M and O) had homogeneous pre-operative sagittal parameters (p > 0.05) that became statistically different after surgeries (p < 0.05). Proximal junctional kyphosis (PJK) was more frequent in the O group compared to U (p = 0.05). Post-operative HRQoL parameters were similar in the 3 groups (p > 0.05). The quality of the included studies was generally low with a high bias risk. The results extrapolated from this review are interesting, as for the same HRQoL the U group had a lower MC rate. Unfortunately, the results are inconsistent, mainly because of the low quality of the included studies and the lack of reporting of some important patient- and surgery-related factors.

264. A RETROSPECTIVE CHART REVIEW OF 85 PATIENTS UNDERGOING THE REFLUXSTOP PROCEDURE TO MANAGE GASTROESOPHAGEAL REFLUX DISEASE: SAFETY AND EFFICACY

Abstract Background RefluxStop is a novel surgical device that manages gastroesophageal reflux disease (GERD) by restoring the normal structure of the antireflux barrier. This is accomplished by reinstating the acute angle of His and anchoring the gastroesophageal sphincter adequately in the intraabdominal cavity. This study reports the safety and efficacy of 79 patients in the postoperative period. Methods A retrospective chart review was performed on 79 patients that underwent the RefluxStop procedure at a single institution between July 2021 and November 2022 to manage GERD symptoms after informed consent was obtained. Measures were evaluated to assess the feasibility, safety, and clinical outcomes during the follow-up period. Results 79 patients (age 49.8 ± 14 years), 46 of which were male, had a mean BMI of 25.8 ± 4.5. Associated conditions noted included esophagitis (45.6%), Barrett’s esophagus (17.7%), and hiatal hernia (57%). Hiatal hernias were large in 35% of cases, with an overall average size of 2.9 cm. Baseline symptoms included general GERD symptoms (35.4%), heartburn (29.1%), cough (13.9%), regurgitation (11.4%), retrosternal burning (10.1%), hoarseness (5.1%), abdominal pain or pressure (3.8%), nausea (2.5%), and others. Baseline GERD health-related quality of life (GERD-HRQL) score was 21.5 ± 5.2 and patients were receiving proton pump inhibitors (PPIs) for a mean of 4.7 ± 6.8 years. After surgery (10.7 ± 3.1 months), all subjects experienced significant improvement in GERD-related symptoms including dysphagia. Only 2.5% of patients required use of PPIs and no subjects required postoperative esophageal dilatation or reoperation. Additionally, there were no severe perioperative complications. Postoperatively, the mean GERD-HRQL score was significantly reduced to 1.6 ± 3.2 (92.6% improvement). Conclusion This study adds to a growing body of evidence that the RefluxStop procedure is safe and effective in management of GERD patients. Notably, a considerable improvement in PPI usage and health-related quality of life parameters were appreciated. Additional studies are required to further validate the role of this procedure in disease management.

Immediate versus conventional loading of mandibular implant-retained overdentures: a 3-year follow-up of a randomized controlled trial.

There is a scarcity of randomized clinical trials (RCT) that report medium- and long-term results and a lack of consensus in the literature on the predictability of immediately loaded unsplinted narrow diameter implants supporting mandibular overdentures. This RCT compared the performance of conventional (CL) and immediate loading (IL) of mandibular overdentures retained by two narrow-diameter implants for 3years. Patients from an RCT treated with CL or IL were invited to attend to 2- and 3-year follow-ups. Clinical, radiographic, functional, and oral health-related quality of life parameters were evaluated. Prosthetic maintenance events, biological complications, and success and survival rates were also recorded. The data were tested by multilevel mixed-effects linear regression analysis and chi-squared tests. The 1-year survival rates of 90% in the CL group and 85% in the IL group were maintained as no implants were lost between 1 and 3years. The marginal bone loss (MBL) in the IL group was significantly lower after year 3 (-0.04; p < 0.01). Significant changes were found only for the intra-group comparisons in the third year of function: (i) CL and IL presented similar progression of implant stability, MBL, and posterior bone area resorption; (ii) while CL started deteriorating of masticatory function, IL still exhibited functional evolution and (iii) oral comfort domain in the CL and pain domain in the IL were improved. Although IL experienced the lowest MBL after 3years, the outcomes showed that both loading protocols result in predictable medium-term rehabilitation when monitored annually. It can be expected that in the third year of function, patients with immediate loading may present more complaints related to general performance even with acceptable masticatory function and self-reported improvements in oral comfort.

Health-related quality of life and associated factors in elderly individuals with dyslipidemia in rural Northern China.

The purpose of the study was to investigate health-related quality of life (HRQOL) and its associated factors among elderly individuals with dyslipidemia in rural Northern China. A cross-sectional study was conducted on 457 elderly individuals. The EQ-5D-5L questionnaire was used to assess HRQOL. Sociodemographic, anthropometric, lifestyle and health information was collected using a structured and standardized questionnaire. The Tobit regression model and multiple linear regression analysis were used to assess factors associated with HRQOL parameters including utility index and visual analogue score (EQ-VAS). The mean age of the participating individuals was 68.04 ± 5.98years. The median utility index and EQ-VAS scores were 0.942 (P25-P75: 0.876-1.000) and 80 (P25-P75: 70-90), respectively. Pain/discomfort was the most frequently reported complaint among the five health dimensions (50.5%). Illiterate and primary education levels, unemployed, widowed, smoking, secondhand smoke exposure, with hypertension or DM, chronic disease core knowledge scores < 8, and daily sedentary time ≥ 6h were associated with lower HRQOL. Pain/discomfort dimension was the most affected problems. Education, occupation, marital status, smoking, secondhand smoke exposure, chronic disease core knowledge, comorbidities, and daily sedentary time were significantly associated with HRQOL in the present study.

Body Composition and Phase Angle: How to Improve Nutritional Evaluation in Juvenile Dermatomyositis Patients.

(1) Background: This study aimed to assess body composition (BC) using bioelectrical impedance and food intake in juvenile dermatomyositis (JDM) patients. Associations between BC and physical activity, disease activity/cumulative damage and health-related quality of life parameters were also evaluated; (2) Methods: This was a cross-sectional study with 30 consecutive JDM patients (18 female and 12 male) and 24 healthy volunteers (14 female and 10 male) of both sexes followed at our pediatric rheumatology unit. The gathering of anthropometric and dietary data, and the performance of physical activity and bioelectrical impedance were undertaken in face-to-face meetings and through questionnaires. Clinical and therapeutic data were collected from medical records according to information from routine medical appointments; (3) Results: The frequency of high/very high body fat was significantly higher in controls compared with JDM patients (66.7% vs. 91.7%; p = 0.046). The median phase angle was significantly lower in patients compared with controls (5.2 ± 1.3 vs. 6.1 ± 1.0; p = 0.016). Body fat and lean mass were positively correlated with disease duration (rs = +0.629, p < 0.001 and rs = +0.716, p < 0.001, respectively) and phase angle (PhA) (rs = +0.400, p = 0.029 and rs = +0.619, p < 0.001, respectively). JDM patients with PhA ≥ 5.5 presented higher lean mass when compared with patients with PhA < 5.5 (p = 0.001); (4) Conclusions: Bioelectrical impedance can be a useful auxiliary exam in the medical and nutritional follow-up of JDM patients, because it seems to impact functional ability. These findings may assist professionals when advising JDM patients about the importance of physical activity and healthy eating in the preservation of lean mass.

Randomized, double-blind, placebo-controlled study to evaluate the effect of treatment with an SPMs-enriched oil on chronic pain and inflammation, functionality, and quality of life in patients with symptomatic knee osteoarthritis: GAUDI study

BackgroundSpecialized pro-resolving mediators (SPMs), including 18-HEPE, 17-HDHA, and 14-HDHA are recognized as potentially therapeutic in inflammatory diseases because SPMs regulate the inflammation process, which leads to, for example; swelling and the sensation of pain. In osteoarthritis (OA), chronic pain is described as the symptom that reduces patients´ quality of life (QoL). The GAUDI study evaluated the efficacy of SPMs supplementation in reducing pain in the symptomatic knee of OA patients.MethodsThis randomized, multicenter, double-blind, and placebo-controlled parallel-group pilot study was performed in Spain and conducted on adults 18–68 years old diagnosed with symptomatic knee OA. Patients were enrolled in the study for up to 24 weeks, which included a 12-week intervention period and a follow-up visit on week 24. The primary endpoint was pain change measured through a Visual Analog Scale (VAS). Secondary endpoints included: Pain change evaluation, stiffness, and function according to the WOMAC index; assessment of constant, intermittent, and total pain according to the OMERACT-OARSI score; evaluation of changes in health-related QoL parameters; the use or not of concomitant, rescue, and anti-inflammatory medication; and safety and tolerability assessments.ResultsPatients were enrolled in the study from May 2018 to September 2021. VAS pain score was evaluated in the per protocol population (n = 51 patients), in which we observed a statistically significant reduction after 8 weeks (p = 0.039) and 12 weeks (p = 0.031) of treatment in patients consuming SPMs (n = 23 subjects) vs. placebo (n = 28 subjects). In line with the OMERACT-OARSI score, intermittent pain was reduced after 12 weeks with statistical significance (p = 0.019) in patients treated with SPMs (n = 23 subjects) vs. placebo (n = 28 subjects). Functional status as WOMAC score did not significantly change after SPMs or placebo consumption. Notably, patients consuming SPMs showed improvements in all five aspects of the EUROQoL-5, including a significant improvement in the usual-activities dimension. None of the patients required rescue medication, nor were any adverse events reported.ConclusionsThese findings suggest that sustained SPMs consumption reduces pain in OA patients while also improving their Quality of Life. These results also support the safety profile of SPMs supplementation.Trial registration NCT05633849. Registered 1 December 1 2022. Retrospectively registered, https://clinicaltrials.gov/ct2/show/study/NCT05633849

A peer support program results in greater health benefits for peer leaders than other participants: evidence from the Kerala diabetes prevention program

BackgroundPeer support programs are promising approaches to diabetes prevention. However, there is still limited evidence on the health benefits of peer support programs for lay peer leaders.PurposeTo examine whether a peer support program designed for diabetes prevention resulted in greater improvements in health behaviors and outcomes for peer leaders as compared to other participants.Methods51 lay peer leaders and 437 participants from the Kerala Diabetes Prevention Program were included. Data were collected at baseline, 12 months, and 24 months. We compared behavioral, clinical, biochemical, and health-related quality of life parameters between peer leaders and their peers at the three time-points.ResultsAfter 12 months, peer leaders showed significant improvements in leisure time physical activity (+ 17.7% vs. + 3.4%, P = 0.001) and health-related quality of life (0.0 vs. + 0.1, P = 0.004); and a significant reduction in alcohol use (-13.6% vs. -6.6%, P = 0.012) and 2-hour plasma glucose (-4.1 vs. + 9.9, P = 0.006), as compared to participants. After 24 months, relative to baseline, peer leaders had significant improvements in fruit and vegetable intake (+ 34.5% vs. + 26.5%, P = 0.017) and leisure time physical activity (+ 7.9% vs. -0.9%, P = 0.009); and a greater reduction in alcohol use (-13.6% vs. -4.9%, P = 0.008), and waist-to-hip ratio (-0.04 vs. -0.02, P = 0.014), as compared to participants. However, only the changes in fruit and vegetable intake and waist-to-hip ratio were maintained between 12 and 24 months.ConclusionBeing a peer leader in a diabetes prevention program was associated with greater health benefits during and after the intervention period. Further studies are needed to examine the long-term sustainability of these benefits.

The impact of disease activity on health-related quality of life in patients with systemic lupus erythematosus

Background and objectiveThe aim of this research was to investigate the relationship between disease activity and health-related quality of life (HRQoL) in patients with systemic lupus erythematosus (SLE) considering the increased interest in the management of this disease. Materials and methodsHRQoL was measured at clinic visits during a 12-month follow-up period using questionnaires on fatigue (FACIT-FATIGUE); quality of life, EuroQol 5-dimension (EQ-5D-5L) health questionnaire with 5 levels; disability, Health Assessment Questionnaire (HAQ), and a Global Health Status (GHS) scale. Disease activity, organ damage and other clinical factors that could affect HRQoL were recorded. The association between disease activity and HRQoL was assessed using Bayesian linear regression models with monotonic effects. ResultsData from 70 patients at the baseline visit and 42 patients with 1 year of follow-up were analyzed. At baseline, 28.57% of patients presented Systemic Lupus Erythematosus Disease Activity Index (SLEDAI)>6. In the 70 baseline patients, disease activity was associated with HRQoL in all four parameters. In the 42 patients with 12 months of follow-up, the positive association of disease activity with GHS, FACIT-FATIGUE and EQ-5D-5L and the negative association with HAQ was maintained.Patients who are smokers and those receiving immunosuppressant therapy presented low GHS and FACIT-FATIGUE scores. Moreover, older age at inclusion was significantly associated to low GHS, while low leucocyte and 25-OH-vitamin D levels were associated to fatigue perception in SLE patients. ConclusionOur results showed a statistically significant association between disease activity and HRQoL parameters.

Revision surgery following long lumbopelvic constructs for adult spinal deformity: prospective experience from two dedicated databases.

Pan Lumbar Arthodesis (PLA) are often required for Adult Spinal Deformity (ASD) correction, reducing significantly the compensatory capacity in case of postoperative sagittal malalignment. Few papers have investigated outcomes and complications in this vulnerable subset of patients. The objective of this study was to assess revision surgery rate for PLA in ASD, its risk factors and impact on clinical outcomes. Retrospective multicenter review of prospective ASD data from 7 hospitals covering Europe and Asia. ASD patients included in two prospective databases having a posterior instrumentation spanning the whole lumbar region with more than 2-years of follow-up were reviewed. Demographic, surgical, radiographic parameters and Health-Related Quality of Life (HRQoL) scores were analyzed. Univariate and multivariate regression models analyzed risk factors for revision surgery as well as surgical outcomes. Patients with Early versus Late and PJK versus Non-PJK mechanical complications were also compared. Out of 1359 ASD patients included in the database 589 (43%) had a PLA and 357 reached 2-years mark. They were analyzed and compared to non-PLA patients. Average age was 67 and 82% were females. 100 Patients (28.1%) needed 114 revision surgeries (75.4% for mechanical failures). Revised patients were more likely to have a nerve system disorder, higher BMI and worst immediate postoperative alignment (as measured by GAP Parameters). These risk factors were also associated with earlier mechanical complications and PJK. Deformity and HRQoL parameters were comparable at baseline. Non-revised patients had significantly better clinical outcomes at 2-years (SRS 22 scores, ODI, Back pain). Multivariate analysis could identify nerve system disorder (OR 4.8; CI 1.8-12.6; p = 0.001), postoperative sagittal alignment (GAP Score) and high BMI (OR 1.07; CI 1.01-1.13; p = 0.004) as independent risk factors for revisions. Revision surgery due to mechanical failures is relatively common after PLA leading to worse clinical outcomes. Prevention strategies should focus on individualized restoration of sagittal alignment and better weight control to decrease stress on these rigid constructs in non-compliant spines. Nerve system disorders independently increase revision risk in PLA. Prognosis.

Investigating survival, quality of life and cognition in PROton versus photon therapy for IDH-mutated diffuse grade 2 and 3 GLIOmas (PRO-GLIO): a randomised controlled trial in Norway and Sweden

IntroductionThe use of proton therapy increases globally despite a lack of randomised controlled trials demonstrating its efficacy and safety. Proton therapy enables sparing of non-neoplastic tissue from radiation. This is...

Effects of a Diabetes Sports Summer Camp on the Levels of Physical Activity and Dimensions of Health-Related Quality of Life in Young Patients with Diabetes Mellitus Type 1: A Randomized Controlled Trial

Physical activity (PA) is considered an important part of the treatment of children with diabetes mellitus type 1 (T1DM). Furthermore, health-related quality of life (HRQoL) affects both the physical and mental health of patients with T1DM. The purpose of the study was to evaluate through a randomized controlled trial the impact of participation in a summer diabetes sports camp on the PA and HRQoL of children and adolescents with T1DM. Eighty-four children and adolescents with T1DM were randomly assigned into an intervention (M = 12.64, SD = 1.82, 30 female) and a control group (M = 12.67, SD = 2.50, 30 female). Intervention group participants attended a ten-day summer diabetes sports camp which included an intensive program of PA (6 h of daily PA), educational and entertaining activities as well as education on the importance of PA in the management of the disease. At baseline and at the end of the study, participants completed measures of physical activity, self-esteem, depression, health status, intention to change behavior, and life satisfaction. Results of the two-way repeated measures analysis showed no statistically significant group differences in PA levels (p < 0.05) and HRQoL parameters (p < 0.05 for all parameters). In conclusion, the results did not support the effectiveness of a 10-day diabetes sports camp on PA levels and HRQoL for children with T1DM. Longer interventions may be more effective in exerting positive influence on trait parameters of children with T1DM's quality of life. Participation in such programs on multiple occasions should be evaluated in the future.

The role of SGLT-2 inhibitors on health-related quality of life, exercise capacity, and volume depletion in patients with chronic heart failure: a meta-analysis of randomized controlled trials.

Improving health-related quality of life (HRQoL) is essential in treating heart failure (HF). Evidence of sodium-glucose cotransporter-2 (SGLT-2) inhibitors on HRQoL and exercise capacity needs to be systematically analyzed. This meta-analysis aimed to summarize the effects of SGLT-2 inhibitors on HRQoL, exercise capacity, and volume depletion in patients with HF. Randomized controlled trials were searched from PubMed, EMBASE, and the Cochrane Central Register of Controlled Trials. The intervention arm was the SGLT-2 inhibitor group, and the control group was the placebo group. HRQoL outcomes were the Kansas City Cardiomyopathy Questionnaires (KCCQ)-OSS (Overall Summary Score), KCCQ-CSS (Clinical Summary Score), and KCCQ-TSS (Total Symptom Score). Exercise capacity was a 6-min walk test distance (6MWTD). The last search was conducted in May 2022. Two researchers independently screened articles, extracted data, and evaluated the quality of included trials. The Cochrane risk-of-bias tool was used to assess the quality of each study. Random or fixed-effect models were used in statistical methods. I2 statistics were used to assess heterogeneity. Eight studies (6,213 patients) were included. Compared to the placebo group, SGLT-2 inhibitors significantly improved HRQoL parameters of the KCCQ-CSS score [mean difference (MD) 5.17, 95% confidence interval (95% CI) 4.61-5.73, P < 0.01] and the KCCQ-OSS score (MD 4.00, 95% CI 3.44-4.56, P < 0.01). SGLT-2 inhibitors also significantly improved exercise capacity 6MWTD (MD 21.90, 95% CI 6.54-37.25, P = 0.005). There were no significant differences in KCCQ-TSS (MD 1.95, 95% CI - 1.10 to 5.01, P = 0.21) and volume depletion [odds ratio (OR) 1.15, 95% CI 0.94-1.42, P = 0.18] between the treatment and placebo groups. SGLT-2 inhibitors could improve HRQoL and exercise capacity in patients with chronic HF. SGLT-2 inhibitors did not have an impact on volume depletion.

The impact of disease activity on health-related quality of life in patients with systemic lupus erythematosus

Background and objectiveThe aim of this research was to investigate the relationship between disease activity and health-related quality of life (HRQoL) in patients with systemic lupus erythematosus (SLE) considering the increased interest in the management of this disease. Materials and methodsHRQoL was measured at clinic visits during a 12-month follow-up period using questionnaires on fatigue (FACIT-FATIGUE); quality of life, EuroQol 5-dimension (EQ-5D-5L) health questionnaire with 5 levels; disability, Health Assessment Questionnaire (HAQ), and a Global Health Status (GHS) scale. Disease activity, organ damage and other clinical factors that could affect HRQoL were recorded. The association between disease activity and HRQoL was assessed using Bayesian linear regression models with monotonic effects. ResultsData from 70 patients at the baseline visit and 42 patients with 1 year of follow-up were analyzed. At baseline, 28.57% of patients presented Systemic Lupus Erythematosus Disease Activity Index (SLEDAI)>6. In the 70 baseline patients, disease activity was associated with HRQoL in all four parameters. In the 42 patients with 12 months of follow-up, the positive association of disease activity with GHS, FACIT-FATIGUE and EQ-5D-5L and the negative association with HAQ was maintained.Patients who are smokers and those receiving immunosuppressant therapy presented low GHS and FACIT-FATIGUE scores. Moreover, older age at inclusion was significantly associated to low GHS, while low leucocyte and 25-OH-vitamin D levels were associated to fatigue perception in SLE patients. ConclusionOur results showed a statistically significant association between disease activity and HRQoL parameters.