Chronic hepatitis B beyond clinical burden: Psychosocial effects and impact on quality of life.

Chronic hepatitis B (CHB) infection has far-reaching implications for patients' quality of life (QOL), regardless of cirrhosis or complications. However, limited research exists on the psychosocial impact of CHB, particularly in asymptomatic, noncirrhotic patients. Noncirrhotic CHB patients without comorbidities affecting QOL were evaluated in hepatology clinics. Patients underwent a standardized interview and completed the Short Form-12 (SF-12) questionnaire. Multiple social and psychological parameters were compared between patients receiving chronic treatment and untreated controls with HBeAg-negative chronic infections. A total of 41 CHB patients were included in the study, with 41% in the treatment group and 59% in the control group. Both groups showed a male predominance, and their average age, ethnic distribution and family status were comparable. High unemployment rates and discrimination at work due to HBV status were prevalent, with many patients feeling their career choices were influenced by HBV, particularly among untreated patients. Treated patients reported significantly lower scores in general health perception (41% vs. 13%; p = .06) and limitation of usual activity secondary to CHB (29% vs. 0%; p = .008), resulting in a substantial impact on overall health-related QOL. This study highlights the psychosocial implications of CHB in asymptomatic, noncirrhotic patients, underscoring the importance of comprehensive support and increased awareness. Additionally, addressing challenges related to long-term treatment, such as adherence and cost, may have the potential to improve patient well-being and outcomes.

Patient-reported quality of life and working status outcomes in ambulatory patients with idiopathic inflammatory myopathy.

OBJECTIVE: To investigate the health-related quality of life (HR-QoL), work productivity and activity impairment and associated factors among patients with idiopathic inflammatory myopathy (IIM). METHODS: This was an observational, cross-sectional study. The 189 ambulatory patients with IIM were recruited from May 2019 to May 2022. HR-QoL was measured by the European Quality of Life 5-Dimension (EQ-5D) questionnaire. The Work Productivity and Activity Impairment (WPAI) questionnaire was used to evaluate work productivity and activity impairment. The IIM-related parameters were assessed by the 8-item Manual Muscle Test (MMT-8), Myositis Disease Activity Assessment visual analogue scale (MYOACT), Myositis Damage Index (MDI), Disease Activity Score (DAS) and Physician/Patient Global Assessment (PhGA/PtGA). Quantile regression and ordinal logistic regression were performed to identify the factors, considering EQ-5D or WPAI scores as dependent variables, respectively. RESULTS: Of the 189 IIM patients enrolled, 60% had DM, 13% had PM and 27% had clinical amyopathic DM. The median EQ-5D score was 1.00 (95% CI 0.73, 1.00), 28% were employed and 45% of overall work was impaired due to health problems. EQ-5D values were positively associated with MMT-8 and negatively with MYOACT, DAS, MDI-global and PhGA/PtGA. For the WPAI, activity impairment was associated with a lower MMT-8 score, older onset age and higher PhGA only in 25th-75th percentile. Increased PtGA was associated with increased activity and overall working productivity impairment in most quantiles (P<0.05). CONCLUSION: Multiple disease characteristics were associated with reduced HR-QoL or working productivity impairment in patients with IIM, especially for PtGA.

A systematic review of patient-reported outcome measures in patients with anti-neutrophil cytoplasmic antibody associated vasculitis.

OBJECTIVES: 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. A total of 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. CONCLUSION: 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 some limitations and may not fully capture the AAV patient's perspective on disease and treatment burden.

Effect of bimekizumab on patient-reported disease impact in patients with psoriatic arthritis: 1-year results from two phase 3 studies.

OBJECTIVES: To evaluate 1-year bimekizumab efficacy in PsA from the patient perspective using the 12-item PsA Impact of Disease (PsAID-12) questionnaire. METHODS: BE OPTIMAL (NCT03895203; biologic DMARD [bDMARD]-naïve), BE COMPLETE (NCT03896581; inadequate response/intolerance to TNF inhibitors [TNFi-IR]) and BE VITAL (NCT04009499; open-label extension) assessed bimekizumab 160 mg every 4 weeks in patients with PsA. Post hoc analyses of patient-reported disease impact, assessed by the PsAID-12 questionnaire, are reported to 1 year (collected to Week 40 in BE COMPLETE). RESULTS: Overall, 1,112 total patients were included (698 bimekizumab, 414 placebo). Rapid improvements observed with bimekizumab treatment at Week 4 continued to Week 16 and were sustained to 1 year. At 1 year, mean (SE) change from baseline in PsAID-12 total score was comparable between bimekizumab-randomized patients and patients who switched to bimekizumab at Week 16 (bDMARD-naïve bimekizumab -2.3 [0.1], placebo/bimekizumab -2.2 [0.1]; TNFi-IR bimekizumab -2.5 [0.1], placebo/bimekizumab -2.2 [0.2]). Proportions of bimekizumab-randomized patients achieving clinically meaningful within-patient improvement (≥3-point decrease from baseline) at Week 16 were sustained to 1 year (bDMARD-naïve 49.0%; TNFi-IR 48.5%) and were similar for placebo/bimekizumab patients (bDMARD-naïve 44.4%; TNFi-IR 40.6%). Across studies and arms, 35.3% to 47.8% of patients had minimal or no symptom impact at 1 year. Improvements were observed to 1 year across all single-item domains, including pain, fatigue and skin problems. CONCLUSION: Bimekizumab treatment resulted in rapid and sustained clinically meaningful improvements in disease impact up to 1 year in bDMARD-naïve and TNFi-IR patients with PsA. TRIAL REGISTRATION: BE OPTIMAL: NCT03895203; BE COMPLETE: NCT03896581; BE VITAL: NCT04009499 (ClinicalTrials.gov).

Quality of life and decision regret in patients with late-hypothyroidism after radioiodine treatment for Graves' disease.

OBJECTIVE: Patients with Graves' disease often engage in shared decision-making to select an individualised treatment regimen from multiple options. Radioactive iodine (RAI) is one of the treatment choices for their condition, aims to improve quality of life and well-being. Likewise, dissatisfaction with treatment outcomes can result in decision regret. We employed validated questionnaires to assess the prospective quality of life, decision regret and relative factors involved in decision-making of patients with late hypothyroidism after RAI therapy. METHODS: A questionnaire survey was conducted among patients in hypothyroidism status for more than 1 year after RAI therapy. Disease-specific and generic QoL were assessed using the short form of thyroid-related patient-reported outcome (ThyPRO-39) questionnaire. Patient satisfaction regarding their decision to undergo RAI was assessed using the Decision Regret Scale (DRS) and patients were asked about the importance of relative factors in decision-making. RESULTS: Of 254 patients who responded to the survey, the mean age of patients was 45.3 years (range: 18-78 years) and the median time from RAI therapy to survey was 4 years (range: 1-30 years). Patients' median and mean DRS score were 34.4 and 38.8 (range: 0-100), respectively. A total of 100 (39.4%) patients express absent-to-mild regret (score: 0-25), 154 (60.6%) patients express moderate-to-severe regret (score: >25). The mean score of the absent-to-mild regret group were significantly higher than those of the moderate-to-severe regret group on most ThyPRO-39 scales. A statistically significant positive correlation was observed between DRS score and most ThyPRO-39 scale score. There was a significant positive association between higher DRS score and longer time intervals after RAI treatment, a brief duration of hyperthyroidism, and the significance of long-time outpatient follow-up. More decision regret was negatively associated Iodine-free diet, ineffectiveness of ATD, fear of surgery. CONCLUSION: Impairment of quality of life was positively correlated with decision regret in patients with late-hypothyroidism after radioiodine therapy. Patients with insufficient information support before decision-making are more likely to have higher decision regret after treatment. Our findings suggest that health providers should fully communicate with patients and provide information support in multiple dimensions during the shared-decision-making process.

A phase 2 open-label extension study of prekallikrein inhibition with donidalorsen for hereditary angioedema.

BACKGROUND: Hereditary angioedema (HAE) is a potentially fatal disease characterized by unpredictable, recurrent, often disabling swelling attacks. In a randomized phase 2 study, donidalorsen reduced HAE attack frequency and improved patient quality-of-life (ISIS721744-CS2, NCT04030598). We report the 2-year interim analysis of the phase 2 open-label extension (OLE) study (ISIS 721744-CS3, NCT04307381). METHODS: In the OLE, the on-treatment study period consisted of fixed (weeks 1-13, donidalorsen 80 mg subcutaneously every 4 weeks [Q4W]) and flexible (weeks 17-105, donidalorsen 80 mg Q4W, 80 mg every 8 weeks [Q8W], or 100 mg Q4W) dosing periods. The primary outcome was incidence and severity of treatment-emergent adverse events (TEAEs). The secondary outcomes included efficacy, pharmacodynamic, and quality-of-life assessments. RESULTS: Seventeen patients continued in the OLE study. No serious TEAEs or TEAEs leading to treatment discontinuation were reported. Mean monthly HAE attack rate was 96% lower than the study run-in baseline rate (mean, 0.06/month; 95% confidence interval [CI], 0.02-0.10; median, 0.04 on-treatment vs. mean, 2.70/month; 95% CI, 1.94-3.46; median, 2.29 at baseline). Mean monthly attack rate for Q8W dosing (n = 8) was 0.29 (range, 0.0-1.7; 95% CI, -0.21 to 0.79; median, 0.00). Mean plasma prekallikrein and D-dimer concentrations decreased, and Angioedema Quality of Life Questionnaire total score improved from baseline to week 105 with donidalorsen. CONCLUSION: The 2-year interim results of this phase 2 OLE study of donidalorsen in patients with HAE demonstrated no new safety signals; donidalorsen was well tolerated. There was durable efficacy with a 96% reduction in HAE attacks.

Satisfaction with continuous glucose monitoring is associated with quality of life in young people with type 1 diabetes regardless of metabolic control and treatment type.

AIMS: While continuous glucose monitoring (CGM) and associated technologies have positive effects on metabolic control in young people with type 1 diabetes (T1D), less is known about their impact on quality of life (QoL). Here, we quantified CGM satisfaction and QoL in young people with T1D and their parents/caregivers to establish (i) the relationship between QoL and CGM satisfaction and (ii) the impact of the treatment regimen on QoL. METHODS: This was a cross-sectional study of children and adolescents with T1D on different treatment regimens (multiple daily injections, sensor-augmented pumps and automated insulin delivery). QoL was assessed with the KINDL instrument, and CGM satisfaction with the CGM-SAT questionnaire was evaluated in both youths with T1D and their parents. RESULTS: Two hundred and ten consecutively enrolled youths with T1D completed the KINDL and CGM-SAT questionnaires. The mean total KINDL score was greater than neutral in both subjects with T1D (3.99 ± 0.47) and parents (4.06 ± 0.40), and lower overall CGM-SAT scores (i.e., higher satisfaction) were significantly associated with higher QoL in all six KINDL subscales (p < 0.05). There were no differences in KINDL scores according to delivery technology or when participants were grouped according to optimal and sub-optimal glucose control. CONCLUSIONS: Higher satisfaction with recent CGMs was associated with better QoL in all dimensions. QoL was independent of both the insulin delivery technology and glycaemic control. CGM must be further disseminated. Attention on perceived satisfaction with CGM should be incorporated with the clinical practice to improve the well-being of children and adolescents with T1D and their families.

Urinary function in female patients after traditional, organ-sparing and nerve-sparing radical cystectomy for bladder cancer: a systematic review and pooled analysis.

OBJECTIVES: To determine and summarize the available data on urinary, sexual, and health-related quality-of-life (HRQOL) outcomes after traditional radical cystectomy (RC), reproductive organ-preserving RC (ROPRC) and nerve-sparing RC (NSRC) for bladder cancer (BCa) in female patients. METHODS: The PubMed, SCOPUS and Web of Science databases were searched to identify studies reporting functional outcomes in female patients undergoing RC and urinary diversion for the treatment of BCa. The outcomes of interest were voiding function (for orthotopic neobladder [ONB]), sexual function and HRQOL. The following independent variables were derived and included in the meta-analysis: pooled rate of daytime and nighttime continence/incontinence, and intermittent self-catheterization (ISC) rates. Analyses were performed separately for traditional, organ- and/or nerve-sparing surgical approaches. RESULTS: Fifty-three studies comprising 2740 female patients (1201 traditional RC and 1539 organ-/nerve-sparing RC, and 264 nerve-sparing-alone RC) were eligible for qualitative synthesis; 44 studies comprising 2418 female patients were included in the quantitative synthesis. In women with ONB diversion, the pooled rates of daytime continence after traditional RC, ROPRC and NSRC were 75.2%, 79.3% and 71.2%, respectively. The pooled rate of nighttime continence after traditional RC was 59.5%; this rate increased to 70.7% and 71.7% in women who underwent ROPRC and NSRC, respectively. The pooled rate of ISC after traditional RC with ONB diversion in female patients was 27.6% and decreased to 20.6% and 16.8% in patients undergoing ROPRC and NSRC, respectively. The use of different definitions and questionnaires in the assessment of postoperative sexual and HRQOL outcomes did not allow a systematic comparison. CONCLUSIONS: Female organ- and nerve-sparing surgical approaches during RC seem to result in improved voiding function. There is a significant need for well-designed studies exploring sexual and HRQOL outcomes to establish evidence-based management strategies to support a shared decision-making process tailored towards patient expectations and satisfaction. Understanding expected functional, sexual and quality-of-life outcomes is necessary to allow individualized pre- and postoperative counselling and care delivery in female patients planned to undergo RC.

Decision regret analysis in early URSL vs medical expulsive therapy 1 for ureteric calculi ≤ 1cm.

BACKGROUND: The study assesses the decisional regret following Shared Decision-making (SDM) in patients selecting either early ureteroscopic lithotripsy (URSL) or medical expulsive therapy (MET) for ureteric stones ≤ 1 cm, with the aim to evaluate their decisional Conflict, satisfaction, and regret regarding their opted treatment choices. METHODS: Adults aged more than 18 years with one stone up to 1 cm in either ureter were included. After SDM, the patients were allocated into their opted group viz. URSL or MET. Patients in each group were reassessed at "treatment completion". Cambridge Ureteric Stone PROM (CUSP) questionnaire for HRQoL, Decision Regret Scale and the OPTION scale (SDM) were filled at treatment completion. FINDINGS: 111 patients opted for MET, while 396 patients opted for early URSL. Mean stone size was larger in URSL group (7.16 ± 1.63 mm vs. 5.50 ± 1.89; p < 0.001). Decisional conflict was higher in patients opting for URSL (77.3% vs. 57.7%; p < 0.001). Stone-free rate at four weeks was higher in URSL group (87.1%vs68.5%, p < 0.001). Decisional regret was higher in patients opting for MET (33.24 ± 30.89 vs. 17.26 ± 12.92; p = 0.002). Anxiety, was higher in patients opting for MET (6.94 ± 1.89 vs. 5.85 ± 1.54; p < 0.001). Urinary symptoms and interference in patients' travel plans and work-related activities were more in URSL group (6.21 ± 1.57 vs. 5.59 ± 1.46; p < 0.001 and 6.56 ± 1.59 vs. 6.05 ± 1.72; p < 0.001 respectively). INTERPRETATION: After SDM, decisional regret is higher in patients opting for MET mainly due protracted treatment duration with increased pain and anxiety during the treatment course and the need for additional procedure for attaining stone clearance and the. Despite higher decisional conflict, a larger proportion of patients opt for early URSL with the aim of avoiding anxiety and achieving early stone clearance.

The personal impact of living with a myeloproliferative neoplasm.

OBJECTIVE: The aim of this study is to gain insight into the physical, psychological and social impact of having a myeloproliferative neoplasm (MPN), a rare type of cancer with an often chronic course. METHODS: An online survey was conducted among 455 Dutch MPN patients (62.7% female, age M 63) to explore the impact of the disease by measuring the MPN symptom burden (MPN-SAF TSS) and quality of life (QoL) (EORTC QLQ-C30) and its subscales within a hierarchical QoL model. We examined differences in MPN symptom burden and QoL in relation to sociodemographic and disease-related factors. Hierarchical regression analysis was used to explain variances in QoL. RESULTS: Most patients (97%) experienced MPN-related health complaints, with a significantly higher MPN symptom burden in women (M 31.50) compared to men (M 24.10). Regarding to fatigue and cognitive functioning MPN patients suffered more compared to a reference group of other cancers. MPN subtype or type of treatment did not show significant differences in MPN symptom burden or QoL. However, experiencing side effects, complications or comorbidities significantly negatively affected MPN symptom burden and QoL. 48.8% of patients reported that MPN affected their ability to work. The explained variance in overall QoL was 58%, most importantly by disease progression, comorbidities, MPN symptom burden and role, emotional and social functioning. CONCLUSION: This study revealed that having an MPN has a negative impact on several domains of QoL. Symptom assessment and support should be included in the healthcare management of MPN patients.