BACKGROUND: Ulcerative colitis (UC) is a chronic disorder with a considerable negative impact on health-related quality of life (HRQoL), which has been recently recognized as an important treatment target. The purpose of this study is to compare the efficacy of different biologics and small molecule therapies in achieving better patient-reported outcomes and HRQoL in patients with UC.
METHODS: We performed a systematic review and network meta-analysis of the EMBASE, MEDLINE, and Cochrane Central databases from inception until February 1, 2024. The primary endpoint was clinical remission in the patient-reported outcome (PRO-2) score in UC patients who were treated with different biologics or small molecules during induction and maintenance phases. PRO-2 score is the sum of both stool frequency and rectal bleeding subscores. The secondary outcome was improvement of HRQoL defined as an increase in Inflammatory Bowel Disease Questionnaire score of ≥16 points from baseline or any change in total score from baseline. A random effects model was used, and outcomes were reported as odds ratio with 95% confidence interval. Interventions were ranked per the SUCRA (surface under the cumulative ranking curve) score.
RESULTS: A total of 54 studies were included in the primary outcome analysis and 15 studies were included in the secondary outcome analysis. The primary analysis showed that during the induction phase all of included drugs were better than placebo in improving the PRO-2 score. Interestingly, upadacitinib was found to be superior to most medications in improving PRO-2 scores. The secondary analysis showed that guselkumab ranked first in the improvement of the Inflammatory Bowel Disease Questionnaire score, followed by upadacitinib during the induction phase.
CONCLUSIONS: Upadacitinib ranked first in PRO-2 clinical remission during the induction and maintenance phases. Guselkumab, mirikizumab, tofacitinib, and upadacitinib were the only novel medications that were superior to placebo in improving HRQoL in UC, with guselkumab ranking the highest, followed by tofacitinib and upadacitinib. During maintenance of remission, tofacitinib ranked highest in improving HRQoL.
Patient-reported outcome (PRO-2) and disease impact on health-related quality of life (HRQoL) have been recognized as important treatment targets in ulcerative colitis. In this systematic review and network meta-analysis, we compared different biologics and small molecules in achieving these outcomes. We found that upadacitinib ranked first in PRO-2 clinical remission during induction and maintenance phases. Guselkumab, tofacitinib, and upadacitinib were the only novel medications that were superior to placebo in improving HRQoL during induction in ulcerative colitis, with guselkumab ranking the highest, followed by tofacitinib and upadacitinib. During maintenance of remission, tofacitinib ranked highest in improving HRQoL.

Atopic dermatitis (AD) is a chronic, inflammatory skin disease affecting all age groups, particularly children. This systematic review provides an overview of the humanistic and economic disease burden in the pediatric population with AD in Spain. The evidence, collected from 11 observational studies published over the past 10 years, exhibits the most common characteristics of the patients, disease burden, patient-reported outcomes, use of resources, and treatment patterns. The burden of AD extends beyond physical symptoms, with associated comorbidities such as asthma and impaired health-related quality of life and mental health disorders, particularly in severe cases. Traditional therapies, primarily topical corticosteroids, face adherence and efficacy challenges. Despite promising innovative treatments and available biological therapies, their use is still limited in the pediatric population. The findings of the present review highlight the scarce scientific evidence on the economic burden of pediatric AD, as well as the most updated humanistic evidence on this disease. At the same time, the need for individualized care and innovative therapeutic interventions to address the multifaceted challenges of pediatric AD in Spain is evident.

The Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data (SISAQOL) initiative was established in 2016 to assess the quality and standardization of patient-reported outcomes (PRO) data analysis in randomized controlled trials (RCTs) on advanced breast cancer. The initiative identified deficiencies in PRO data reporting, including nonstandardized methods for handling missing data. This study evaluated the reporting of health-related quality of life (HRQOL) in Japanese cancer RCTs to provide insights into the state of PRO reporting in Japan. The study reviewed PubMed articles published from 2010 to 2018. Eligible studies included Japanese cancer RCTs with ≥50 adult patients (≥50% were Japanese) with solid tumors receiving anticancer treatments. The evaluation criteria included clarity of the HRQOL hypotheses, multiplicity testing, primary analysis methods, and reporting of clinically meaningful differences. Twenty-seven HRQOL trials were identified. Only 15% provided a clear HRQOL hypothesis, and 63% examined multiple HRQOL domains without adjusting for multiplicity. Model-based methods were the most common statistical methods for the primary HRQOL analysis. Only 22% of the trials explicitly reported clinically meaningful differences in HRQOL. Baseline assessments were reported in most trials, but only 26% reported comparisons between the treatment groups. HRQOL analysis was based on the intention-to-treat population in 19% of the trials, and 74% reported compliance at follow-up; however, 41% did not specify how missing values were handled. Although the rates of reporting clinical hypotheses and clinically meaningful differences were relatively low, the current state of HRQOL evaluation in the Japanese cancer RCT appears comparable to that of previous studies.

A recent evidence gaps assessment of the clinical, health-related quality of life, and economic burden associated with α-thalassemia is lacking. We conducted a systematic literature review (SLR) following the methodological and reporting requirements of the Preferred Reporting Items for Systematic Reviews and Meta-Analyses and the Cochrane Handbook for Systematic Reviews, using available literature over the past decade. This SLR identified a considerable evidence gap with regard to understanding the current burden of α-thalassemia as evident from paucity of studies published in the past 10 years. The limited data available still indicate that patients with α-thalassemia experience substantial morbidity and quality of life/economic burden that is generally comparable to patients with β-thalassemia.

UNASSIGNED: β-Thalassemia major (β-TM) represents one of the most important hemoglobinopathies worldwide. Remarkable improvements have been achieved in supportive therapy based on blood transfusions and iron chelation, and nowadays, this approach is capable of assuring a long life in these patients in industrialized countries. The only curative treatment is represented by hematopoietic stem cell transplantation (HSCT). However, this treatment may be burdened by deterioration in the Health-Related Quality of Life (HRQoL). This paper aimed to evaluate the role of HRQoL in transplanted β-TM patients with a systematic review and meta-analysis.
UNASSIGNED: PubMed database, Web of Science, and Scopus were systematically searched for studies published between January 1st, 2000 to September 2020. The following terms were entered in the database queries: β-thalassemia, HRQoL, and HSCT. The study was carried out according to the Preferred Reporting Items for Systematic and Meta-analyses (PRISMA) statement.
UNASSIGNED: We identified a total of 33 potential studies. Among these, 10 were finally considered in the systematic review and 5 in the meta-analysis. Overall, good scores in the principal domains of HRQoL were reported by transplanted patients. These data were confirmed by results of meta-analysis that showed significant difference between transplanted and β-TM patients treated with conventional therapy in the physical and emotional dimension, with a medium effect size [d=0.65, 95% CI (0.29-1.02), z = 3.52, p =0.0004, I2=75%; and d=0.59, 95% CI (0.43-0.76), z = 6.99, p <0.00001, I2=0%, respectively].
UNASSIGNED: HRQoL is generally good in β-TM transplanted patients and may significantly contribute in deciding whether or not to transplant a β-TM patient treated with conventional therapy.

BACKGROUND: Heavy menstrual bleeding (HMB) and dysmenorrhea (DM) are common gynecological problems.
OBJECTIVE: To systematically review the needs, quality of life (QOL), and effectiveness of self-management strategies among young women (12-25 years) with DM or HMB.
METHODS: Relevant terms were searched through PubMed, EBSCO, Google Scholar, ProQuest, and Ovid between 2010 and 2022.
METHODS: Qualitative and quantitative studies published in peer-reviewed journals, females aged 12-25, exploring DM or HMB, reporting supportive care needs, quality of life, self-treatment strategies, and/or treatment-seeking behavior.
METHODS: Abstracts were reviewed for eligibility by two researchers. Included studies were extracted and assessed for quality independently by two authors, with discrepancies resolved through consensus or the involvement of a third researcher. Data extracted included study details, menstrual history, symptoms, self-care strategies, and quality of life. The Joanna Briggs Institute checklists were used for quality assessment.
RESULTS: The search returned 285 190 studies, of which 55 were eligible for inclusion. Prevalence rates of HMB and DM were in the ranges 4%-63% and 42%-94%, respectively. Over 80% of young women with DM and HMB experienced physical and psychological problems, including pelvic pain, sleep issues, mood disturbance, diarrhea, and nausea. Academic performance and daily activities were severely affected. Most (>62%) named their mothers as their primary source of information, and friends as the secondary source (10%-65%). Few studies explored needs, but they could be inferred and fell under school-related and social needs. QOL was poorer in those who had DM than those who did not. Pain was the most common issue that drove young women to find treatment. More than 70% used medication to reduce pain. More than half chose home remedies (e.g., heat therapy, massages, herbal tea, hot drinks). No studies provided information about the efficacy and dosage of medication and herbal remedies.
CONCLUSIONS: HMB and DM have a large impact on daily living, with large areas of unmet need. Limited access to information impairs the management of symptoms and consequent QOL.

BACKGROUND: Evaluating the Health-related quality of life (HRQoL) of individuals with Parkinson\'s disease (PD) holds significant importance in clinical and research settings. The EQ-5D is a widely recognized tool for comprehensive measurement of HRQoL using utility values. This study aims to systematically review and synthesize EQ-5D utility values from existing literature on patients with PD and their caregivers.
METHODS: We conducted a systematic search for studies that provided EQ-5D utility scores for patients with PD, using PubMed-Medline, Scopus, and Embase and selected the studies. The selected studies underwent systematic review, including an assessment of their quality. We performed a meta-analysis using a random-effect model and conducted a meta-regression analysis to investigate sources of heterogeneity among the studies.
RESULTS: The search result of 13,417 articles that were reviewed, 130 studies with 33,914 participants were selected for systematic review, and 79 studies were included for meta-analysis. The pooled EQ-5D utility values and visual analog score (VAS) among PD were 62.72% (60.53-64.93, I2 = 99.56%) and 0.60 (0.55-0.65, I2 = 99.81%), respectively. The pooled scores for caregivers\' EQ-VAS and EQ-5D utility were 70.10% (63.99-76.20, I2 = 98.25%) and 0.71 (0.61-0.81, I2 = 94.88%), respectively. Disease duration (P < 0.05) showed a negative correlation with EQ-5D utility values on meta-regression.
CONCLUSIONS: The pooled utility values of PD and their caregivers help to understand their HRQoL and aid in conducting health economics research. The negative association between disease duration and utility values highlights the evolving nature of HRQoL challenges, suggesting the need for appropriate long-term disease management.

OBJECTIVE: Describing the characteristics and sources of health state utility values and reporting practice in the literature of cost-utility analyses facilitates an understanding of the level of the transparency, validity, and generalizability of cost-utility analyses. Improving the quality of reporting will support investigators in describing the incremental value of emerging glaucoma interventions.
OBJECTIVE: To describe the state of practice among published glaucoma cost-utility analysis studies, focusing on valuation of health and the quality of reporting.
METHODS: We searched several databases including Medline, CINHAL, Embase, Web of Science, Scopus, Biosis previews, the Health Economic Evaluations Database, and the NHS Economic Evaluation Database (NHS EED). We included full-text, English, published cost-utility analyses of glaucoma interventions with quality-adjusted life years (QALYs) as the primary outcome measure to calculate incremental cost-utility ratios. Excluded studies were non-English language, reviews, editorials, protocols, or other types of economic studies (cost-benefit, cost-minimization, cost-effectiveness). Study characteristics, operational definitions of glaucoma health states and health state utilities were extracted. The original source of the health utility was reviewed to determine the scale of measurement and the source of preference weighting. Items from the Systematic Review of Utilities for Cost-Effectiveness (SpRUCE checklist) were used to assess the reporting and quality of health utilities in glaucoma CUA.
RESULTS: 43 CUAs were included, with 11 unique sources of health utilities. A wide range of health utilities for the same Hodapp-Parrish-Anderson glaucoma health states were reported; ocular hypertension (0.84-0.95), mild (0.68-0.94), moderate (0.57-0.92), advanced (0.58-0.88), severe/blind (0.46-0.76), and bilateral blindness (0.26-0.5). Most studies reported the basis for using health utilities (34, 79%) and any assumptions or adjustments applied to the health utilities (22, 51%). Few studies reported a framework for assessing the relevance of health utilities to a decision context (8, 19%). Even fewer (3, 7%) applied a systematic search strategy to identify health utilities and used a structured assessment of quality for inclusion. Overall, reporting has not improved over time.
CONCLUSIONS: This review describes that few CUAs describe important rationale for using health state utility values. Including additional details on the search, appraisal, selection, and inclusion process of health utility values improves transparency, generalizability and supports the assessment of the validity of study conclusions. Future investigations should aim to use health utilities on the same scale of measurement across health states and consider the source and relevance to the decision context/purpose of conducting that cost-utility study.

OBJECTIVE: (1) To identify instruments used to assess quality of life (QoL) in children and adolescents with neuromuscular diseases; (2) To identify the psychometric properties contained in these instruments.
METHODS: This is a scoping review in which the electronic databases Embase, Scielo, Scopus, Pubmed and Lilacs were used as well as grey literature. The following terms were used in the search for articles published in the last 10 years: children, adolescents, neuromuscular disease, and quality of life.
RESULTS: In total, 15 articles were included and evaluated, indicating 7 instruments used to assess QoL (PedsQL™ Inventory 3.0 Neuromuscular Module, the PedsQL™ 4.0, the PedsQL DMD Module, the PedsQL ™ MFS, the SOLE, the KIDSCREEN and the LSI-A). The number of items ranged from 17 to 45. In addition, 6 instruments showed psychometric properties, but only 2 showed good and high quality, either in internal reliability or reproducibility.
CONCLUSIONS: Our results were able to map the main QoL assessment instruments of children and adolescents with neuromuscular disease and the most cited instrument was the PedsQL™ Inventory 3.0 Neuromuscular Module. Larger studies that assess psychometric properties and that are validated for most diseases are needed.

OBJECTIVE: ANCA associated vasculitis (AAV) is associated with significant morbidity, fatigue, pain and poor health-related quality of life (HRQoL). This review aims to assess the comprehensiveness of existing patient reported outcome measures (PROMs) used in AAV and identify associations with poorer HRQoL outcomes.
METHODS: A literature review of studies using PROMs, including those labelled HRQoL in people with AAV as a primary or secondary study outcome were screened and reviewed up to July 2023. Quality was assessed using the Critical Appraisal Skills Programme.
RESULTS: A total of 30 articles were included which utilised 22 different PROM tools. 76.7% (n = 23) used the SF-36 or a variation as a generic measure of health status and or HRQoL. Two studies developed a disease specific PROM. The AAV-PRO showed good psychometric properties but potential limitations in capturing all relevant aspects of the disease experience for AAV patients. Factors associated with poorer HRQoL included: neurological and sinonasal involvement, women and younger patients. 86.6% of studies showed no meaningful relationships between the SF-36 and BVAS, VDI or disease duration. Depression and anxiety were common and socioeconomic factors such as unemployment were significantly associated with poorer mental health outcomes. Glucocorticoids were found to be independently associated with worse SF-36 scores.
CONCLUSIONS: Generic PROMs are useful in measuring significant changes but lack sensitivity to specific symptoms and unique AAV-related issues, while existing disease specific PROMs have limitations and may not fully capture AAV patient\'s perspective on disease and treatment burden.