Journal of Patient-Reported Outcomes最新文献

{"title":"Embedding patient voice within European healthcare: achievements and future directions of the health outcomes observatory.","authors":"Preston Long, Rahim Lalji, Margaret R Andrews, Afroditi Avgerinou, Olga Alekseeva, Francesco Patalano, Nataliya Kemenyash, Lisa R Otto, Gemma Galan, Linetta Koppert, Meni Styliadou, Tanja Stamm","doi":"10.1186/s41687-026-01221-y","DOIUrl":"10.1186/s41687-026-01221-y","url":null,"abstract":"","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"10 1","pages":""},"PeriodicalIF":2.9,"publicationDate":"2026-10-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13650199/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149138255","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Beyond performance: the role of illness perception and internal comparison standards in patient-reported physical function assessments.","authors":"Nina Schnaderbeck, Mia Grote, Sandra Nolte, Audrey Y Brinker, Alexander Obbarius, Felix Fischer, Matthias Rose, Gregor Liegl","doi":"10.1186/s41687-026-01227-6","DOIUrl":"10.1186/s41687-026-01227-6","url":null,"abstract":"<p><strong>Background: </strong>Previous research has yielded inconsistent findings regarding the relationship between patient-reported physical function (PF) and physical performance. Patient-reported PF has been discussed to be more strongly associated with psychological variables, which could explain discrepancies with performance assessments. The aim of this study was to investigate differential association patterns of illness perception and internal comparison standards with patient-reported versus performance-based PF.</p><p><strong>Methods: </strong>A total of N = 106 patients from three medical specialties (Cardiology, n = 22; Rheumatology, n = 21; Psychosomatic medicine, n = 63) were included in this cross-sectional study. Patient-reported PF was assessed using a 20-item short form of the Patient-Reported Outcomes Measurement Information System (PROMIS), the PROMIS-PF SF-20a, while performance-based PF was measured using the Physical Performance Test (PPT), comprising 9 physical performance tasks. Illness perception was assessed using the Brief Illness Perception Questionnaire (BIPQ), while social and temporal comparisons were assessed using single-item scales. Multiple linear regression analyses were conducted to examine the relationships of illness perception and internal comparison standards with both patient-reported and performance-based PF.</p><p><strong>Results: </strong>Physical performance was strongly associated with patient-reported PF (Pearson's r = .74), accounting for more than 50% of the variance in the regression model with patient-reported PF as dependent variable. Illness perception and internal comparison standards also contributed significantly to the prediction of patient-reported PF beyond actual performance. Specifically, more negative illness perceptions, as well as poorer self-assessments compared to peers (social comparisons) and compared to one's own best functioning (temporal comparisons), were associated with lower patient-reported PF. Illness perception was also significantly associated with performance-based PF, however, this association was very small. Social and temporal comparisons were not associated with performance-based PF. Consistent with this pattern, the additional explained variance by psychological variables was substantially larger for patient-reported PF than for performance-based PF.</p><p><strong>Conclusions: </strong>While patient-reported functioning was highly associated with actual performance, illness perception as well as social and temporal comparisons appeared to play an additional role in the self-assessment of PF. In contrast, psychological variables showed only negligible effects when performance-based PF was used as the outcome. These findings highlight the importance of considering psychological variables when interpreting patient-reported PF. Future research is needed to explore the role of further psychological factors not considered in this study to further improve our unders","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"10 1","pages":""},"PeriodicalIF":2.9,"publicationDate":"2026-10-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13653012/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149149786","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Targeted literature review to understand the patient experience of essential thrombocythemia and evaluate existing clinical outcome assessment measures.","authors":"Amy Howerter, Xiaoqin Yang, Kelly McQuarrie, Lucy Lloyd-Price, Joseph Gyorkey, Stacie Hudgens","doi":"10.1186/s41687-026-01048-7","DOIUrl":"10.1186/s41687-026-01048-7","url":null,"abstract":"<p><strong>Background: </strong>The use of Clinical Outcome Assessments (COA) instruments in blood cancers such as myeloproliferative neoplasms (MPN) is widely documented, however little is known about Essential Thrombocythemia (ET). A targeted literature review was conducted to identify the concepts of interest to the ET population and to develop a conceptual framework. Using this framework, identification and review of COA instruments will help determine if there are existing instruments fit for purpose for use in ET studies.</p><p><strong>Methodology: </strong>Targeted reviews in May and October 2023 synthesized the literature on ET symptoms and impacts to derive a conceptual model and identify suitable COAs. Eligible articles consisted of ET only populations and extended to MPN samples that included ET patients. A gap analysis was conducted on selected instruments.</p><p><strong>Results: </strong>Of the 890 abstracts reviewed, 206 were included in the analysis. An ET conceptual model that encompassed disease-defining concepts (fatigue most common), proximal, distal, and general life impacts was created. MPN specific measures such as the Myelofibrosis Symptom Assessment Form v4.0 (MFSAF), and Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS; MPN-10), European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core Questionnaire (EORTC QLQ-C30), Patient-Reported Outcomes Measurement Information System (PROMIS) - Fatigue, and Brief Fatigue Inventory (BFI) were deemed the most appropriate for the gap analysis. Each had strengths and limitations related to conceptual coverage, evidence for measurement properties, and feasibility for use in clinical trials.</p><p><strong>Conclusions: </strong>The MFSAF v4.0, PROMIS-Fatigue, and EORTC QLQ-C30 are considered appropriate for use in deriving trial endpoints in ET; however further work is needed to assess the fitness-for-purpose in the ET population as most validation studies were performed in mixed MPN or oncology samples.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"10 1","pages":""},"PeriodicalIF":2.9,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13650201/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149138298","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Hand and shoulder diagnoses are associated with substantial bodily pain compared to vascular complications and long-term glycaemic control in people with long-term type 1 diabetes.","authors":"Anne Karin Molvær, Iversen Marjolein Memelink, Jannicke Igland, Mark Peyrot, Grethe S Tell, Tore Julsrud Berg","doi":"10.1186/s41687-026-01199-7","DOIUrl":"https://doi.org/10.1186/s41687-026-01199-7","url":null,"abstract":"<p><strong>Background: </strong>People with type 1 diabetes are burdened by musculoskeletal hand and shoulder diagnoses and vascular late complications. We aimed to explore associations of musculoskeletal diagnoses, 30-year HbA1c and vascular complications with pain, fatigue, depression, and quality of life (QoL) in an increasing group of long-term survivors of type 1 diabetes.</p><p><strong>Methodology: </strong>This exploratory, observational study included 104 participants with type 1 diabetes ≥ 45 years' duration who completed questionnaires assessing pain (RAND-36 bodily pain), fatigue (Fatigue Questionnaire), depression (Patient Health Questionnaire), overall QoL (WHOQoL-BREF) and diabetes-specific QoL (Audit of Diabetes-Dependent QoL). We used linear regression to estimate associations between hand and shoulder diagnoses, 30-year HbA1c ≥ 64 mmol/mol (8%) and vascular complications with PROs.</p><p><strong>Results: </strong>Compared to 30-year HbA1c and vascular complications, combined hand and shoulder diagnoses showed the strongest estimated association with bodily pain; [B (95% confidence interval)] [-17.4 (-32.8, -2.1)], followed by 30-year HbA1c ≥ 64 mmol/mol [10.9 (-21.9, 0.1)], microvascular complications alone [-11.3 (-24.8, 2.1)], and micro- and macrovascular complications combined [-5.9 (-21.9, 10.2)]. Vascular complications, particularly microvascular, were associated with worse overall QoL and diabetes-specific QoL.</p><p><strong>Conclusions: </strong>Musculoskeletal hand and shoulder diagnoses show stronger associations with bodily pain than vascular complications, while vascular complications appear more important for QoL in this cohort of long-term survivors with type 1 diabetes. Estimates where however uncertain due to limited sample size.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"10 1","pages":""},"PeriodicalIF":2.9,"publicationDate":"2026-09-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13631153/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149115974","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The QOLA study: psychometric validation of the Quality of Life of Parents of Children with Achondroplasia (QOLA) questionnaire - a multinational study in Germany, Italy, and Portugal.","authors":"Adekunle Adedeji, Stefanie Witt, Lea Maric, Maria Salomao, Florian Innig, Inês Alves, Chiara Provasi, Marco Sessa, Klaus Mohnike, Julia Quitmann","doi":"10.1186/s41687-026-01207-w","DOIUrl":"10.1186/s41687-026-01207-w","url":null,"abstract":"<p><strong>Background: </strong>Parents of children with achondroplasia experience multidimensional impacts on quality of life (QoL), yet validated condition-specific instruments for assessing these outcomes across countries are scarce. This study evaluated the psychometric properties of the Quality of Life of Parents of Children with Achondroplasia (QOLA) questionnaire in three European settings.</p><p><strong>Methods: </strong>A multinational, multicentre observational psychometric validation study was conducted in Germany, Italy, and Portugal. Parents/primary caregivers (N = 149) completed the 43-item QOLA (eight conceptual domains) and the EUROHIS-QOL-8 at baseline (T1); a subsample completed follow-up assessments (T2; n = 77) for test-retest analyses. Data quality (missingness, floor/ceiling effects), internal consistency (Cronbach's α; composite reliability), temporal stability (Pearson correlations; intraclass correlation coefficients), and convergent validity (correlations with EUROHIS-QOL-8) were examined. Exploratory factor analysis informed item reduction and domain aggregation, followed by confirmatory factor analysis of the reduced model.</p><p><strong>Results: </strong>Item-level missingness was minimal, and no substantial floor/ceiling effects were observed. Eight-domain scores showed acceptable to excellent internal consistency (α = 0.706-0.905) and moderate-to-good temporal stability (single-measure ICCs = 0.64-0.75). Convergent validity was supported by negative associations between QOLA domains and generic QoL (r = .15-0.69). Exploratory and confirmatory analyses supported a parsimonious three-factor, 20-item model, comprising Healthcare System Trust, Caregiving Burden and Daily Impact, and Worries About the Future and Social Relationships, with acceptable fit (CFI = 0.935, TLI = 0.925, RMSEA = 0.066), strong reliability (α = 0.852-0.918; CR = 0.864-0.914), and good test-retest stability (r = .670-0.782; average-measure ICCs = 0.801-0.877).</p><p><strong>Conclusions: </strong>The QOLA demonstrates robust reliability and validity across three countries and offers both an eight-domain profile and a three-factor summary for assessing parental QoL in achondroplasia.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"10 1","pages":""},"PeriodicalIF":2.9,"publicationDate":"2026-09-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13615216/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149064927","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Psychometric evaluation of the PROMIS^® Medication Adherence Scale among patients prescribed oral anticancer medication for multiple myeloma.","authors":"Sarah M Belcher, Paul Scott, Susan M Sereika, Catherine Bender, Jacqueline Dunbar-Jacob, Margaret Q Rosenzweig, Benyam Muluneh, Lindsay M Sabik, Valire Copeland, Mounzer Agha, Abigail A Lustyik, Emily S He, Corrine Bozich, Grace Booze, Petra Duran Basso, J Devin Peipert","doi":"10.1186/s41687-026-01204-z","DOIUrl":"10.1186/s41687-026-01204-z","url":null,"abstract":"<p><strong>Background: </strong>Oral anticancer medications are standard care for cancer. Medication adherence influences health outcomes, but valid, reliable measures assessing self-reported medication adherence are limited. Psychometric properties of the PROMIS^® Medication Adherence Scale (PMAS) were evaluated in patients prescribed oral anticancer medications for multiple myeloma.</p><p><strong>Methodology: </strong>This was a secondary analysis from a longitudinal observational study examining medication adherence, symptoms, quality of life, and financial hardship among individuals prescribed oral anticancer medications for multiple myeloma. PMAS measured self-reported medication adherence. Self-report and medical record data assessed participant characteristics and adherence correlates. Objective medication adherence indices were generated from continuous electronic event monitored data. Internal consistency reliability was estimated using Cronbach's alpha. Dimensionality was assessed with confirmatory factor analysis. Construct validity was assessed considering adherence correlates. Spearman rank-order correlations summarized associations between PMAS and electronic event monitored data.</p><p><strong>Results: </strong>PMAS items had limited variability, with high adherence over time. Reliability of PMAS scores was adequate (T1 α = 0.82, 95% CI: 0.75, 0.87; T2 α = 0.84, 95% CI: 0.78, 0.89). Confirmatory factor analysis fit was better for sub-scales (Medication Beliefs and Knowledge and Medication Taking Behaviors) than Total scale, particularly for the Medication Beliefs and Knowledge subscale. Adherence correlates were observed as expected between PMAS and age, self-reported cognitive function, symptom severity, and depression. Weak/moderate positive associations were found between PMAS and electronic event monitored data.</p><p><strong>Conclusions: </strong>Reliability, two-factor dimensionality, and construct validity were supported, with some evidence of concurrent criterion validity with electronic event monitored adherence data. Additional validation testing is needed to support findings. Evidence supports the feasibility of longitudinal oral anticancer medication adherence assessment monitoring using PMAS.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"10 1","pages":""},"PeriodicalIF":2.9,"publicationDate":"2026-09-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13615244/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149064872","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"When do we need to take action? Cut-off values for the hamburg inventory for measuring quality of life in oncological patients (HELP-6).","authors":"Theresa Schrage, Mirja Görlach, Christian Stephan Betz, Carsten Bokemeyer, Nicolaus Kroeger, Volkmar Mueller, Andreas Krüll, Holger Schulz, Christiane Bleich","doi":"10.1186/s41687-026-01198-8","DOIUrl":"10.1186/s41687-026-01198-8","url":null,"abstract":"<p><strong>Purpose: </strong>Results of patient-reported outcome measurements need to be comprehensible and conclusive for a successful integration into treatment in cancer care. The aim of this study was to determine cut-off values for the recently developed \"Hamburg Inventory for Measuring Quality of Life in Oncological Patients-6\" (HELP-6), a short measurement for Health-related Quality of Life (HrQoL) in routine care in oncological patients.</p><p><strong>Methods: </strong>Oncological in- and out-patients with differing cancer entities participated in this study. The survey included the HELP-6 and standardized questionnaires (Distress-Thermometer, PHQ-4, Fact-G, PDI-G). Six receiver operating characteristics analyses were performed, one for each scale of the instrument (Emotional Health, Physical Ailment, Social Functionality, Autonomy, Dignity, Resources).</p><p><strong>Results: </strong>For five of the six scales cut-offs could be determined. Areas under the Curve were between 0.7 and 0.8. However, the Dignity scale demonstrated poor discriminative validity, failing to distinguish adequately between severity levels of patient groups.</p><p><strong>Conclusion: </strong>By applying the HELP-6 with the cut-off values established in this study, clinicians can more readily interpret patients' HrQoL, thereby supporting effective clinical decision-making. This feature is especially valuable in routine care, where streamlined monitoring of patient quality of life is paramount. Further conceptualization and validation of the Dignity scale, as well as its integration into patient-reported outcome measures, are warranted.</p><p><strong>Trial registration: </strong>The complete study including the development of the instrument was registered at Open Science Framework ***redacted for peer-review***.</p><p><strong>Clinical trial number: </strong>not applicable.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"10 1","pages":""},"PeriodicalIF":2.9,"publicationDate":"2026-09-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13598032/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149026191","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cultural adaptation of the Work Disability Functional Assessment Battery (WD-FAB) into German (Germany): hybrid application of two adaptation approaches.","authors":"You-Shan Feng, Sandra Meyer-Moock, Maresa Buchholz, Elizabeth Marfeo, Julia Porcino, Susanne Weinbrenner, Jörg Gehrke, Lewis E Kazis, Thomas Kohlmann","doi":"10.1186/s41687-026-01181-3","DOIUrl":"10.1186/s41687-026-01181-3","url":null,"abstract":"<p><strong>Background: </strong>The Work Disability Functional Assessment Battery (WD-FAB) was developed based on the International Classification of Functioning, Disability and Health (ICF) framework and administered using a computer adaptive test (CAT) to assess individuals' functional status. The framework and assessment of functional status is especially pertinent in Germany as one factor in determining work disability. This paper reports on the translation and cultural adaptation of the WD-FAB from English (USA) to German (Germany).</p><p><strong>Methodology: </strong>A combination of forward-backward and dual-panel approaches was used to translate the WD-FAB into German. During the reconciliation process after forward and backward translations, a selection of items was discussed with the forward/backward certified translators, discussed with developers of the WD-FAB and selected for dual-panel discussion. A field test with rehabilitation patients was conducted to finalize the adaptation.</p><p><strong>Results: </strong>401 unique items from the original WD-FAB and version 3.0 (the majority of the items were the same across versions) were translated into German. After forward translation, the majority of the items were easily reconciled. Some items (identified by YF and TK) could not be easily reconciled and required additional examination. Based on the reasons for difficulties in forward translation, items were selected for the translation panel discussions (n = 69), discussed with developers (n = 49) and/or further discussed with the two forward translators (n = 18). After the first reconciliation and backward translation, 31 items were discussed with all translators again and 4 were again discussed with developers. 77 rehabilitation patients participated in the field test, 29 of whom provided feedback suggesting further revisions. Through the entire adaptation process, the items that were problematic involved those dealing with \"anger\" and \"stress\". Furthermore, environmental differences between Germany and the USA limited the adaptation of some physical functioning items.</p><p><strong>Conclusions: </strong>Despite some differences in the cultural and structural context, we successfully adapted the WD-FAB item banks to German. Future validation of the measure is necessary to understand whether the items have similar psychometric measurement properties to those of the original WD-FAB.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"10 1","pages":""},"PeriodicalIF":2.9,"publicationDate":"2026-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13558539/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148920836","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Impact of facial telangiectasias and anemia on quality of life in patients with hereditary hemorrhagic telangiectasia: a cross-sectional study using FACE-Q.","authors":"Nadia Sadok, Franziska Bielefeld, Carl Nikola Martin, Eva-Marie Skoda, Katharina Klinger, Felicia Toppe, Marie Carolin Schleupner, Lukas Boosfeld, Björn Kropf, Noemi Voß, Antonia Lakomek, Anna-Lena Valina, Christina Kaiser, Julia Garvert, Stephan Lang, Freya Droege","doi":"10.1186/s41687-026-01179-x","DOIUrl":"10.1186/s41687-026-01179-x","url":null,"abstract":"<p><strong>Background: </strong>Hereditary hemorrhagic telangiectasia (HHT) is an autosomal dominant disorder characterized by telangiectasias and visceral arteriovenous malformations. While the clinical manifestations are well described, its psychosocial impact-particularly with regard to visible facial telangiectasias and anemia-related appearance changes-remains insufficiently studied. This study aimed to assess appearance-related quality of life in individuals with hereditary hemorrhagic telangiectasia.</p><p><strong>Methods: </strong>In this cross-sectional, single-center study, adults with hereditary hemorrhagic telangiectasia were recruited at a specialized HHT center between November 2023 and May 2024. Participants completed validated patient-reported outcome measures, including the Facial Assessment and Cosmetic Enhancement (FACE-Q) questionnaire and the Patient-Reported Outcomes Measurement Information System (PROMIS-29 +2) profile. Besides that, a standardized questionnaire assessing demographic and clinical characteristics, subjective perceptions of pallor, and cosmetic disturbance was administrated. Disease severity was assessed using the Epistaxis Severity Score and the Toronto Severity Index. Analyses were performed for the overall cohort and stratified by sex to explore potential sex differences in health-related quality of life and symptom perception. Multivariate linear regression analyses were performed to identify independent predictors of FACE-Q subscale outcomes.</p><p><strong>Results: </strong>A total of 116 participants with HHT were included (mean age 60 ± 14 years; 62% were female). Subjective appearance perception showed strong effects: perceiving pallor or tiredness due to low hemoglobin levels was associated with greater appearance-related psychological distress (adjusted β = -9.09, 95% CI -17.23 to -0.94), whereas perceiving facial telangiectasias as cosmetically disturbing was associated with multiple appearance-related outcomes, including lower social function (adjusted β = -9.51, 95% CI -18.04 to -0.99), greater appearance-related psychological distress (adjusted β = -9.33, 95% CI -16.51 to -2.16), and lower satisfaction with the nose (adjusted β = -17.39, 95% CI -28.94 to -5.84). In contrast, objective clinical parameters and overall disease severity assessed by the Epistaxis Severity Score and the Toronto Severity Index were not independently associated with appearance-related quality of life.</p><p><strong>Conclusions: </strong>In HHT, appearance-related quality of life is driven predominantly by subjective perception and psychosocial factors.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"10 1","pages":""},"PeriodicalIF":2.9,"publicationDate":"2026-08-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13530060/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148866988","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Fatigue: assess and address it.","authors":"Tanja A Kuut, Hans Knoop, Hedy A van Oers, Lotte Haverman","doi":"10.1186/s41687-026-01188-w","DOIUrl":"10.1186/s41687-026-01188-w","url":null,"abstract":"<p><p>Severe fatigue is highly prevalent in patients across different medical conditions and can only be partially explained by disease-specific factors. Also, fatigue is often seen by patients as one of the most disabling symptoms. Despite its substantial impact, fatigue is often not systematically discussed in clinical practice. Effective generic treatments exist for patients with severe fatigue. When fatigue is not assessed, patients may be withhold the opportunity to get advice on how to manage fatigue or receive effective interventions to achieve meaningful improvement. Therefore, designated health care professionals involved in patient care should be able to recognize and address fatigue as a key patient-reported outcome.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"10 1","pages":""},"PeriodicalIF":2.9,"publicationDate":"2026-08-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13506999/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148819780","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}