Impaired Health-related Quality Of Life Research Articles

Health-Related Quality of Life (HRQoL) in Patients with Diffuse Large B-Cell Lymphoma (DLBCL) Treated with Polatuzumab Vedotin, Rituximab, Cyclophosphamide, Doxorubicin and Prednisone (Pola-R-CHP) Versus Rituximab, Cyclophosphamide, Doxorubicin, Vincristine and Prednisone (R-CHOP) in the Phase III POLARIX Study

Background: Patients (pts) with lymphoma report impaired HRQoL as a result of disease- and treatment-related symptoms (Hlubocky FJ, et al. Leuk Lymphoma 2013). However, there are limited dynamic data for quality of life using patient-reported outcome (PRO) measures from large prospective clinical trials in pts with previously untreated DLBCL. The Phase III POLARIX study (NCT03274492) demonstrated superior progression-free survival (PFS) and a similar safety profile with Pola-R-CHP vs R-CHOP in pts with previously untreated DLBCL (Tilly H, et al. N Engl J Med 2022). In this analysis, PROs were used to measure physical functioning, fatigue, lymphoma-related symptom scores, and treatment-related symptom scores to fully characterize the pt experience in POLARIX. Methods: In total, 879 pts with previously untreated DLBCL were randomized 1:1 to Pola-R-CHP (n=440) or R-CHOP (n=439). Lymphoma symptom scores were assessed using the Functional Assessment of Cancer Therapy-Lymphoma Subscale (FACT-LymS; Hlubocky FJ, et al. Leuk Lymphoma 2013). Physical functioning, fatigue, constipation, diarrhea, nausea, and vomiting were assessed using the European Organisation for Research and Treatment of Cancer Quality of Life-Core 30 questionnaire (EORTC QLQ-C30; Aaronson NK, et al. J Natl Cancer Inst 1993). Questionnaires were administered to patients without progression during treatment on Day 1 of Cycles 1 (baseline), 2, 3, and 5, at end of treatment (EOT), biannually for 2 years after EOT, then annually for the following 3 years. Questionnaire compliance and summary statistics at each assessment time point were estimated. Percentage of pts with clinically meaningful improvements, worsening, and no changes from baseline for physical functioning (≥7-point increase, ≥10-point decrease, and in between, respectively), fatigue (≥9-point increase, ≥10-point decrease, and in between, respectively), and lymphoma symptom scores (≥3-point increase, ≥3-point decrease, and in between, respectively) were reported based on validated thresholds (Aaronson NK, et al. J Natl Cancer Inst 1993; Hlubocky FJ, et al. Leuk Lymphoma 2013). Changes over time in physical functioning, fatigue, lymphoma symptom scores, and treatment-related symptom scores for constipation, diarrhea, nausea and vomiting were assessed. Results: Questionnaire response rates were high (>80%) throughout the study. At baseline, scores for lymphoma symptoms, physical functioning, and fatigue were similar in both treatment arms; levels of constipation, diarrhea, nausea, and vomiting were similarly low. Both Pola-R-CHP and R-CHOP induced a rapid improvement in lymphoma symptom scores in most pts after Cycle 1 (pts with clinically meaningful improvement at any time point: 82.3% vs 81.3%, respectively), and this was sustained with improvement plateauing by the first assessment after EOT (Figure). Improvements in physical functioning and fatigue were similar with Pola-R-CHP vs R-CHOP (pts with clinically meaningful improvement at any time point in physical functioning: 42.4% vs 39.6%; fatigue: 74.8% vs 68.2%, respectively); improvements plateaued by the first assessment after EOT. Between Cycle 2 and EOT, diarrhea symptoms were more frequently reported in pts treated with Pola-R-CHP (range: 17-34%) than with R-CHOP (range: 18-24%), and constipation symptoms were reported more frequently with R-CHOP (range: 21-42%) than with Pola-R-CHP (range: 23-35%); diarrhea and constipation symptoms returned to baseline levels by EOT. Nausea and vomiting symptoms were reported infrequently in both treatment arms (nausea: range 12-33% in Pola-R-CHP, 11-30% in R-CHOP; vomiting: range 3-11% in Pola-R-CHP, 4-10% in R-CHOP) and returned to baseline levels at EOT. Conclusions: Both regimens led to rapid and sustained improvements in symptoms and improved HRQoL. Lymphoma symptom scores were improved in the majority of pts after Cycle 1, with improvement maintained in most pts. Physical functioning and fatigue scores were similar at baseline between arms, and improved during and after treatment. During chemotherapy, treatment-related symptoms (i.e. nausea, vomiting, constipation, diarrhea) were observed in a minority of pts, with a return to baseline after treatment completion. These data may represent a benchmark for patient-reported HRQoL in frontline DLBCL in the modern era. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

Health-Related Quality of Life, Disease Impacts, and Health Equity Concerns in Adults with Sickle Cell Disease with Recurrent Vaso-Occlusive Crises: Preliminary Results from a Global Longitudinal Survey

Introduction: Sickle cell disease (SCD) is characterized by vaso-occlusive crises (VOCs) and a progressive clinical course, often leading to end-organ damage and accelerated mortality. Individuals with SCD with recurrent VOCs experience disruptions to daily life due to recurrent pain crises and debilitating chronic pain and fatigue, which affect their ability to work and substantially impair their health-related quality of life (HRQoL). Nonetheless, HRQoL and disease impacts in this population are not well quantified. Here, we report preliminary findings from a prospective longitudinal study evaluating the HRQoL, work productivity impacts, and health equity concerns among adult participants with SCD with recurrent VOCs. Methods: A mixed-methods study was conducted to quantify the symptoms, HRQoL, and work impacts of adults with SCD with recurrent VOCs. The study consisted of qualitative interviews and an online longitudinal survey administered at 3 timepoints (time 0, 3 months, and 6 months) in the US, the UK, France, Germany, and Italy. The survey included multiple patient-reported outcomes measures (PROMs): the Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT), composed of the Functional Assessment of Cancer Therapy-General (FACT-G) and a BMT subscale (BMTS); EuroQol 5-Dimension 5-Level (EQ-5D-5L); 11-point pain Numerical Rating Scale (NRS); Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me); and Work Productivity and Activity Impairment (WPAI) questionnaire. Symptoms, comorbidities, treatment experiences, out-of-pocket costs, time spent managing SCD, and participants' health equity concerns were also collected and evaluated. Survey invitations were sent to patient communities via patient advocacy groups. Participants were required to meet the eligibility criteria and provide consent. Eligibility criteria included experiencing ≥2 VOCs/year, in each of the 2 years before study enrollment, that required interaction with a healthcare provider and administration of pain medication or a red blood cell transfusion. Descriptive analyses were conducted for participant characteristics and PROMs based on survey findings at time 0. Data are reported as mean (standard deviation [SD]), unless noted. Results: The survey was completed by 142 participants with SCD (aged ≥18 years) living in the US (n=85) and the UK, Germany, France, and Italy (n=57) at time 0. Overall, 71.8% were female (n=102), the mean age was 35.5 years (10.4), and 15.5% (n=22) had full-time employment (≥32 hours/week). In the prior 12 months, participants reported a mean of 5.9 (4.8) VOCs that required interaction with a healthcare provider and pain medication and spent a mean of 14.6 (24.7) days in the hospital. In the past month, 62.0% (n=88) were taking hydroxyurea to manage their condition. The mean FACT-G total score was 62.2 (18.5) (0-108 scale; 108=best HRQoL), which is lower than in the US general population (80.1 [18.1]). The largest impacts were observed on the physical (-23.9% relative difference) and emotional well-being domains (-16.3% relative difference) vs the US general population. Participants reported a mean EQ-5D-5L visual analog scale score of 58.7 (19.4), with 89.9% reporting pain/discomfort, 74.1% anxiety/depression, 71.2% mobility impairment, and 46.8% self-care impairment. Significant HRQoL impairments were also observed on the ASCQ-Me vs the SCD reference population, with the largest differences observed on the pain (41.5 [9.0]), stiffness (41.1 [8.2]), and emotional (43.5 [7.6]) impact domains. Among those employed (n=47), 42.0% (27.8%) reported work impairment (presenteeism), 24.7% (27.1%) absenteeism, and 53.4% (27.5%) work productivity loss on the WPAI. Among all respondents (n=137), the mean activity impairment was 52.6% (26.0%). On healthcare equity concerns, 68.3% (n=97) felt that they had been treated unfairly while seeking care, with most attributing the unfair treatment to their race/ethnic background (67.0%) and/or requesting more pain medicine (64.9%). Results were consistent in a subset of 18-to-35-year-old participants. Conclusions: Adults with SCD with recurrent VOCs currently receiving treatment experience significant HRQoL impact, impaired work productivity, and health equity concerns, indicating substantial humanistic burden and unmet needs in this patient population.

Health-related quality of life 6months after pediatric intensive care unit admission for bronchiolitis: a prospective single-center cohort study.

Significant differences in several HRQoL domains were found after PICU admission for bronchiolitis compared to normative data. Whereas the domains lung and stomach problems showed significantly impaired scores, most domains revealed HRQoL levels comparable with healthy peers. This study may contribute to the optimization of HRQoL PICU outcomes by highlighting specific HRQoL domains to focus on at admission and during follow-up. •With the decline in PICU mortality, HRQoL became an important outcome measure. Yet, the currently limited number of studies on HRQoL outcomes often involve heterogeneous patient groups. • Bronchiolitis is one of the most frequent reasons for PICU admission, and although a significant part of children admitted for bronchiolitis has a medical history, compared with other reasons for PICU admission, this patient group is relatively homogeneous in terms of age, disease course, and treatment. •In the present study, six months after PICU admission for bronchiolitis, children scored differently on multiple HRQoL domains compared to healthy peers. • Significantly impaired HRQoL scores were reported on lung and stomach problems in comparison to normative data.

Prospects of Probiotic Adjuvant Drugs in Clinical Treatment.

In modern society, where new diseases and viruses are constantly emerging, drugs are still the most important means of resistance. However, adverse effects and diminished efficacy remain the leading cause of treatment failure and a major determinant of impaired health-related quality of life for patients. Clinical studies have shown that the disturbance of the gut microbial structure plays a crucial role in the toxic and side effects of drugs. It is well known that probiotics have the ability to maintain the balance of intestinal microecology, which implies their potential as an adjunct to prevent and alleviate the adverse reactions of drugs and to make medicines play a better role. In addition, in the past decade, probiotics have been found to have excellent prevention and alleviation effects in drug toxicity side effects, such as liver injury. In this review, we summarize the development history of probiotics, discuss the impact on drug side effects of probiotics, and propose the underlying mechanisms. Probiotics will be a new star in the world of complementary medicine.

Associations between patient-reported late effects and systemic cytokines in long-term survivors of head and neck cancer treated with radiotherapy

PurposeHead and neck cancer (HNC) treatment may lead to late effects and impaired health-related quality of life of survivors. Knowledge on long-term late effects after radiotherapy (RT) and potential underlying biological mechanisms is lacking. We assessed the prevalence of xerostomia, dysphagia, and chronic fatigue (CF) in HNC survivors ≥ 5 years post-RT, and examined associations between pro-inflammatory cytokines and late effects.MethodsIn a cross-sectional study, 263 HNC survivors treated between 2007 and 2013 were enrolled. They completed validated questionnaires assessing xerostomia and dysphagia (the EORTC QLQ-H&N35), and CF (the Fatigue Questionnaire), and underwent blood sampling and clinical examination. Pro-inflammatory cytokines were analyzed in 262 survivors and 100 healthy age- and gender-matched controls.ResultsMedian time since treatment was 8.5 years. The proportions of survivors reporting xerostomia, dysphagia, and CF were 58%, 31%, and 33%, respectively, with a preponderance of females. We found no significant associations between IL-6, IL-8, IP-10, TARC, TNF, or ENA-78 and the three late effects. The odds of having elevated levels of IL-6 and IP-10 were significantly higher in the survivors compared to the controls.ConclusionsMore than one-third of long-term HNC survivors experienced xerostomia, dysphagia, and CF. Persistent inflammation, with elevated systemic cytokines, was not associated with these late effects, although HNC survivors had higher levels of some cytokines than the controls.Implications for Cancer SurvivorsThis study provides new knowledge on late effects that can serve as grounds for informing patients with HNC about risk of late effects more than 5 years after RT.

Adjuvant trastuzumab without chemotherapy for treating early HER2-positive breast cancer in older patients: A propensity score-adjusted analysis of a prospective cohort study

PurposeTo gauge the effects of treatment practices on prognosis for older patients with HER2-positive early breast cancer, particularly to determine whether adjuvant trastuzumab alone can offer benefit over no adjuvant therapy. This is a prospective cohort study which accompanies the RESPECT that is a randomized-controlled trial (RCT). MethodsPatients who declined the RCT were treated based on the physician's discretion. We studied the 1) trastuzumab-plus-chemotherapy group, 2) trastuzumab-monotherapy group, and 3) non-trastuzumab group (no therapy or anticancer therapy without trastuzumab). The primary endpoint was disease-free survival (DFS), which was compared using the propensity-score method. Relapse-free survival (RFS) and health-related quality of life (HRQoL) were assessed. ResultsWe enrolled 123 patients aged over 70 years (median: 74.5). Treatment categories were: trastuzumab-plus-chemotherapy group (n = 36, 30%), trastuzumab-monotherapy group (n = 52, 43%), and non-trastuzumab group (n = 32, 27%). The 3-year DFS was 96.7% in trastuzumab-plus-chemotherapy group, 89.2% in trastuzumab-monotherapy group, and 82.5% in non-trastuzumab group. DFS in non-trastuzumab group was lower than in trastuzumab-plus-chemotherapy and trastuzumab-monotherapy groups (propensity-adjusted hazard ratio; HR: 3.29; 95% CI: 1.15–9.39; P = 0.026). The RFS in non-trastuzumab group was lower than in trastuzumab-plus-chemotherapy and trastuzumab-monotherapy groups (propensity-adjusted HR = 7.80; 95% CI: 2.32–26.2, P < 0.0001). There were no significant intergroup differences in the proportions of patients showing HRQoL deterioration at 36 months (P = 0.717). ConclusionTrastuzumab-treated patients had better prognoses than patients not treated with trastuzumab without deterioration of HRQoL. Trastuzumab monotherapy could be considered for older patients who reject chemotherapy.

Sense of coherence or self-efficacy as predictors of health-related quality of life in sickle cell disease patients

Patients with sickle cell disease (SCD) suffer from impaired health-related quality of life (HRQoL). This study aimed to determine the level of HRQoL, sense of coherence (SOC), and self-efficacy (SE) in a sample of SCD patients, and to explore predictors of their physical and mental HRQoL. A cross-sectional descriptive study was conducted on 83 SCD patients of one university hospital. The data of the study was collected through Persian versions of the Short-Form Health Survey SF-36 (RAND 36-item), the Sense of Coherence Scale (SOC-13), and the Sickle Cell Self-Efficacy Scale (SCSES). The mean age of the patients was 26.34 ± 8.19 years old. Patients’ mean scores for the Physical Component Summary (PCS), Mental Component Summary (MCS), SOC, and SCSES were 40.57 ± 17.18 (range: 0–100), 50.44 ± 17.95 (range: 0–100), 52.40 ± 15.35 (range: 13–91), 26.40 ± 6.96 (range: 9–45), respectively. Regression models showed that the level of the patients’ SOC, was the main predictor of the MCS (β = 0.37, p < 0.001). However, the level of the patients’ SE was the main predictor of the PCS (β = 0.30, p = 0.004). Also, “blood transfusion history” in patients was a common predictor for both the PCS (β = − 0.28, p = 0.008) and the MCS (β = − 0.29, p = 0.003). These results can assist nurses and clinicians to plan clinical interventions for SCD patients by focusing on increasing the level of the SOC and SE and improving SCD patients’ HRQoL. Furthermore, measuring the level of the SOC and self-efficacy as screening tests are useful to find patients with a greater risk of impaired HRQoL.

Surviving rectal cancer at the cost of a colostomy: global survey of long-term health-related quality of life in 10 countries.

Colorectal cancer management may require an ostomy formation; however, a stoma may negatively impact health-related quality of life (HRQoL). This study aimed to compare generic and stoma-specific HRQoL in patients with a permanent colostomy after rectal cancer across different countries. A cross-sectional cohorts of patients with a colostomy after rectal cancer in Denmark, Sweden, Spain, the Netherlands, China, Portugal, Australia, Lithuania, Egypt, and Israel were invited to complete questionnaires regarding demographic and socioeconomic factors along with the Colostomy Impact (CI) score, European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ-C30) and five anchor questions assessing colostomy impact on HRQoL. The background characteristics of the cohorts from each country were compared and generic HRQoL was measured with the EORTC QLQ-C30 presented for the total cohort. Results were compared with normative data of reference European populations. The predictors of reduced HRQoL were investigated by multivariable logistic regression, including demographic and socioeconomic factors and stoma-related problems. A total of 2557 patients were included. Response rates varied between 51-93 per cent. Mean time from stoma creation was 2.5-6.2 (range 1.1-39.2) years. A total of 25.8 per cent of patients reported that their colostomy impairs their HRQoL 'some'/'a lot'. This group had significantly unfavourable scores across all EORTC subscales compared with patients reporting 'no'/'a little' impaired HRQoL. Generic HRQoL differed significantly between countries, but resembled the HRQoL of reference populations. Multivariable logistic regression showed that stoma dysfunction, including high CI score (OR 3.32), financial burden from the stoma (OR 1.98), unemployment (OR 2.74), being single/widowed (OR 1.35) and young age (OR 1.01 per year) predicted reduced stoma-related HRQoL. Overall HRQoL is preserved in patients with a colostomy after rectal cancer, but a quarter of the patients interviewed reported impaired HRQoL. Differences among several countries were reported and socioeconomic factors correlated with reduced quality of life.

Health-related quality of life among persons with initial mild, moderate, and severe or critical COVID-19 at 1 and 12 months after infection: a prospective cohort study

BackgroundCurrently, there is limited evidence about the long-term impact on physical, social and emotional functioning, i.e. health-related quality of life (HRQL) after mild or moderate COVID-19 not requiring hospitalization. We compared HRQL among persons with initial mild, moderate or severe/critical COVID-19 at 1 and 12 months following illness onset with Dutch population norms and investigated the impact of restrictive public health control measures on HRQL.MethodsRECoVERED, a prospective cohort study in Amsterdam, the Netherlands, enrolled adult participants after confirmed SARS-CoV-2 diagnosis. HRQL was assessed with the Medical Outcomes Study Short Form 36-item health survey (SF-36). SF-36 scores were converted to standard scores based on an age- and sex-matched representative reference sample of the Dutch population. Differences in HRQL over time were compared among persons with initial mild, moderate or severe/critical COVID-19 using mixed linear models adjusted for potential confounders.ResultsBy December 2021, 349 persons were enrolled of whom 269 completed at least one SF-36 form (77%). One month after illness onset, HRQL was significantly below population norms on all SF-36 domains except general health and bodily pain among persons with mild COVID-19. After 12 months, persons with mild COVID-19 had HRQL within population norms, whereas persons with moderate or severe/critical COVID-19 had HRQL below population norms on more than half of the SF-36 domains. Dutch-origin participants had significantly better HRQL than participants with a migration background. Participants with three or more COVID-19 high-risk comorbidities had worse HRQL than part participants with fewer comorbidities. Participants who completed the SF-36 when restrictive public health control measures applied reported less limitations in social and physical functioning and less impaired mental health than participants who completed the SF-36 when no restrictive measures applied.ConclusionsTwelve months after illness onset, persons with initial mild COVID-19 had HRQL within population norms, whereas persons with initial moderate or severe/critical COVID-19 still had impaired HRQL. Having a migration background and a higher number of COVID-19 high-risk comorbidities were associated with worse HRQL. Interestingly, HRQL was less impaired during periods when restrictive public health control measures were in place compared to periods without.

Approach to Evaluation and Comprehensive Care for Primary Ovarian Insufficiency

Idiopathic primary ovarian insufficiency (POI) is a reproductive endocrine disorder defined as the loss of ovarian function before age 40. The condition is characterized by infertility, estrogen deficiency, and premature menopause. POI has significant long-term physical and psychosocial sequelae. Because POI is underrecognized and challenging to diagnose, patients often experience a “diagnostic odyssey” that contributes to impaired health-related quality of life. This review provides a state-of-the-science overview of POI, approach to diagnosis and management, and interprofessional aspects of care. We highlight unanswered questions and future directions and underscore the nurse practitioner’s role in providing holistic care for women with POI.

Health-Related Quality of Life and Mental Well-Being during the COVID-19 Pandemic in Five Countries: A One-Year Longitudinal Study

This study aimed to investigate socioeconomic and health-related determinants and recent life events and their relation to changes in health-related quality of life (HRQoL) and mental well-being during the first year of the COVID-19 pandemic. A web-based survey was administered repeatedly to participants from Greece, Italy, the Netherlands, the United Kingdom, and the United States. Primary outcome measures were HRQoL (measured by EQ-5D-5L) and mental well-being (measured by WHO-5). Linear regression analyses were performed to estimate the impact of determinants on HRQoL and well-being. In total, 6765 respondents completed the questionnaire at T1 (April-May 2020) and T2 (May-June 2021). Regarding results, 33% showed improved HRQoL at T2, whereas 31% deteriorated. In terms of mental well-being, 44% improved and 41% deteriorated. The greatest deterioration in HRQoL and mental well-being from T1 to T2 was observed with an increasing number of chronic conditions. The effect of negative life events on HRQoL and mental well-being was larger than the effect of positive life events. We conclude that slightly more respondents showed improved rather than deteriorated HRQoL and mental well-being, with some variation by outcome measure and country.

Health Related Quality of Life Outcomes Following Stereotactic Body Radiotherapy in Patients with Oligo-Metastatic Disease: An Individual Patient Data Meta-Analysis

<h3>Purpose/Objective(s)</h3> Health-related quality of life (HRQOL) outcomes are lacking in patients with oligo-metastatic disease (OMD) receiving stereotactic body radiotherapy (SBRT). The aim of this individual patient data meta-analysis was to determine the effect of SBRT on the HRQOL of patients with OMD. <h3>Materials/Methods</h3> As part of a previously described systematic review (PROSPERO 2021 CRD42021238506), the primary outcome of this individual patient data meta-analysis was change in HRQOL at 12-months from baseline in patients with OMD who received SBRT (versus not), reported as standardized mean difference (SMD). Random effects models implemented in RevMan (Cochrane Collaboration) were used for meta-analyses of scores. <h3>Results</h3> Of 7556 publications identified, four studies met inclusion criteria (2 single arm interventional studies and 2 randomized controlled trials [RCTs]). Individual patient data were available from three studies (175 patients). In the two RCTs, there was no statistically significant difference in the SMD between patients who received SBRT and those that did not (0.09 [95% CI -0.32, 0.5], P=0.66). On meta-analysis of patients (N=107) who received SBRT, SMD was -0.23 (95% CI [-0.42, -0.04] versus -0.25 (95% CI [-0.57, 0.07]) in those who did not (N=37) receive SBRT, demonstrating a small deterioration from baseline. For all patients (SBRT and SOC) measured by the Global Health/Quality of Life (GHQOL) EORTC QLQ-C30 (N=80), a small deterioration was found in both the GHQOL change score from baseline to 12-months (-0.27, 95% CI -0.49, -0.05) and in those who received SBRT alone (N=57) (-0.27, 95% CI -0.52, -0.02). <h3>Conclusion</h3> In patients with OMD, there is no difference in HRQOL at 12-months from baseline between patients who received SBRT and those that did not. However, a small HRQOL deterioration was found in both groups of patients. More robust assessment and reporting of HRQOL in studies of OMD is needed to better inform patient decision-making.

Evaluation design of the patient-centred pathways of early palliative care, supportive ecosystems and appraisal standard (InAdvance): a randomised controlled trial

BackgroundPalliative care aims to contribute to pain relief, improvement with regard to symptoms and enhancement of health-related quality of life (HRQoL) of patients with chronic conditions. Most of the palliative care protocols, programmes and units are predominantly focused on patients with cancer and their specific needs. Patients with non-cancer chronic conditions may also have significantly impaired HRQoL and poor survival, but do not yet receive appropriate and holistic care. The traditional focus of palliative care has been at the end-of-life stages instead of the relatively early phases of serious chronic conditions. The ‘Patient-centred pathways of early palliative care, supportive ecosystems and appraisal standard’ (InAdvance) project implements and evaluates early palliative care in the daily clinical routine addressing patients with complex chronic conditions in the evolution towards advanced stages. The objective of the current study is to evaluate the acceptability, feasibility, effectiveness and cost-effectiveness of this novel model of palliative care in the relatively early phases in patients with chronic conditions.MethodsIn this study, a single blind randomised controlled trial design will be employed. A total of 320 participants (80 in each study site and 4 sites in total) will be randomised on a 1:1 basis to the Palliative Care Needs Assessment (PCNA) arm or the Care-as-Usual arm. This study includes a formative evaluation approach as well as a cost-effectiveness analysis with a within-trial horizon. Study outcomes will be assessed at baseline, 6 weeks, 6 months, 12 months and 18 months after the implementation of the interventions. Study outcomes include HRQoL, intensity of symptoms, functional status, emotional distress, caregiving burden, perceived quality of care, adherence to treatment, feasibility, acceptability, and appropriateness of the intervention, intervention costs, other healthcare costs and informal care costs.DiscussionThe InAdvance project will evaluate the effect of the implementation of the PCNA intervention on the target population in terms of effectiveness and cost-effectiveness in four European settings. The evidence of the project will provide step-wise guidance to contribute an increased evidence base for policy recommendations and clinical guidelines, in an effort to augment the supportive ecosystem for palliative care.Trial registrationISRCTN, ISRCTN24825698. Registered 17/12/2020.

Long-term changes of health-related quality of life in patients with peripheral vascular malformations – a prospective observational study

The aim of this observational study was to assess health-related quality of life (HRQOL) changes in patients with vascular malformations, over a period of almost eight years, and to assess clinical and demographic characteristics possibly associated with HRQOL changes. Eighty out of 111 patients who were included in a previously published comparative HRQOL study accepted inclusion in this follow-up study. HRQOL at baseline and follow-up was assessed with the Short-Form 36-item questionnaire (SF-36). Median observation time was 7.9 years. Linear mixed models and linear regression models were applied to assess HRQOL change and possible associations with demographic and clinical variables. The median age of the patient cohort at baseline evaluation (n=111) was 27.0 years. Ninety-six out of 111 (86.5%) patients were diagnosed with venous malformations. Significantly higher SF-36 scores at follow-up were found for the physical domains Role limitations due to (RLDT) physical problems (difference=13.5; 95% CI [1.6, 25.3]) and Bodily pain (difference=11.3; 95% CI [3.8, 18.8]). No deterioration of HRQOL was found in any domain. In multivariate analyses, female gender, muscle/bone involvement, and higher age were associated with a positive relative change in SF-36 in the domains Physical functioning, RLDT physical problems, and RLDT emotional problems, respectively. Invasive treatment was not associated with long-term HRQOL change. Over a period of almost eight years, significant improvement of SF-36 scores was observed in the physical domains RLDT physical problems and bodily pain. Female gender, muscle/bone involvement, and higher age were associated with HRQOL improvement in certain domains.

Benefits of dual-release hydrocortisone treatment on central adiposity and health-related quality of life in secondary adrenal insufficiency

PurposePatients with secondary adrenal insufficiency (SAI) have an increased morbidity and an impaired health-related quality of life (HRQoL), which seems to primarily depend on the sub-optimal replacement of hypoadrenalism with standard glucocorticoid (GC) therapy, and on the inadequate correction of other associated pituitary deficiencies. A dual-release hydrocortisone (DR-HC) formulation has shown to exert positive effects on morbidity and HRQoL, mainly in patients with primary adrenal insufficiency. We assessed the variations of anthropometric and metabolic parameters and HRQoL in patients with SAI after switching from cortisone acetate (CA) or hydrocortisone (HC) to DR-HC.MethodsTwenty-one patients (17 M, 4 F) treated with CA (n = 16; 25 mg/day twice a day) or HC (n = 5; 20 mg/day three times a day), were evaluated for waist circumference, BMI, fasting glucose, HbA1c, insulin, HOMA-IR index, serum lipids, electrolytes, blood pressure and HRQoL at baseline, at 3, 6 and 12 months after switching from CA/HC to DR-HC.ResultsThe study showed a significant reduction of waist circumference and BMI (p = 0.04, for both), after 3 and 6months of DR-HC treatment, respectively. No significant changes were observed for fasting glucose, insulin, HOMA-IR index, HbA1c, total cholesterol, triglycerides, LDL cholesterol, electrolytes, and blood pressure. However, HDL cholesterol significantly decreased (p = 0.003). An improvement of AddiQoL total score was observed during DR-HC treatment (p = 0.01), mainly for the category “emotions”. No predictors resulted for these changes.ConclusionDR-HC treatment provides some benefits in patients with SAI, reducing central adiposity and improving HRQoL; however, worsening of HDL cholesterol is observed during treatment with DR-HC.

Trajectories of Health-related quality of life in patients with Advanced Cancer during the Last Year of Life: findings from the COMPASS study

BackgroundPatients with advanced cancer prioritise health-related quality of life (HrQoL) in end-of-life care, however an understanding of pre-death HrQoL trajectories is lacking. We aimed to delineate and describe the trajectories of physical, social, emotional and functional HrQoL during last year of life among advanced cancer patients. We assessed associations between these trajectories and patient socio-demographic characteristics, healthcare use and place of death.MethodsWe used data from 345 decedents from a prospective cohort study of 600 patients with a solid advanced cancer receiving secondary care at public hospitals in Singapore. Patients were surveyed every three months until death and HrQoL was assessed using the Functional Assessment of Cancer Therapy – General (FACT-G) questionnaire. Interviews were conducted between July 2016 and December 2019. Group-based multi-trajectory modelling was used to assess potential heterogeneity in the four HrQoL dimensions during patients’ last year of life.ResultsWe identified four distinct trajectories of HrQoL − (1) overall high HrQoL (47% of sample), (2) progressively decreasing HrQoL (32%), (3) asymmetric decline in HrQoL (13%), (4) overall low HrQoL (8%). Compared to patients with secondary or above education, those with primary education or less (β = 1.39, SE = 0.55, p-value = 0.012) were more likely to have “progressively decreasing HrQoL” or “overall low HrQoL” in contrast to “overall high HrQoL”. Compared to patients with ‘overall high HrQoL’, those with ‘overall low HrQoL’ had longer length of hospital stay during the last year of life (β = 0.47, SE = 0.21, p-value = 0.026) and were more likely to die in a hospice/care home (β = 1.86, SE = 0.66, p-value = 0.005).ConclusionOur results showed heterogeneity in deterioration of HrQoL among patients with advanced cancer in the last year of life. Systematic monitoring of HrQoL, early identification and referral of high-risk patients to palliative care may provide timely relief and mitigate the steep decline in their HrQoL.Trial Registration: NCT02850640.

Health-Related Quality of Life and mental health of families with children and adolescents affected by rare diseases and high disease burden: the perspective of affected children and their siblings

BackgroundAdvances in genetic and pharmaceutical technology and pediatric care have enabled treatment options for an increasing number of rare diseases in affected children. However, as current treatment options are primarily of palliative nature, the Health-Related Quality of Life (HRQoL) and mental health of this impaired population and their siblings are of increasing importance. Among children and adolescents with rare diseases, those who are technology-dependent carry a high disease burden and are selected as the target population in our study. In a cross-sectional observational design, the children’s HRQoL was assessed with the DISABKIDS (DCGM-37) as well as KIDSCREEN-27, while mental health was assessed with the Strengths and Difficulties Questionnaire (SDQ) by both the affected children, their parents, and siblings.ResultsResults of the study sample were compared to normative data. Affected children scored significantly lower than the norm on almost all HRQoL subscales as reported by parent and child. From the parental perspective, more mental health subscales were significantly impaired compared to the child’s perspective. Siblings showed no impairment in HRQoL as well as significantly fewer behavioral problems and higher prosocial behavior regarding their mental health compared to the norm.ConclusionChildren and adolescents with rare diseases seem particularly impaired in social and emotional aspects of HRQoL and mental health. Interventions may focus primarily on promoting social skills, fostering prosocial behavior and peer relationships.

Decrease in Health-Related Quality of Life and Post-COVID-19 Syndrome in Health Care Workers After SARS-CoV-2 Infection: A Cohort Study.

The aim of this study is to assess the persistence of symptoms, the prevalence of post-COVID-19 syndrome, and the health-related quality of life (HRQOL) among health care workers (HCWs) 6 months after severe acute respiratory syndrome coronavirus 2 infection. A prospective cohort study was conducted. All HCWs with confirmed COVID-19 from January to June 2021 were invited to participate. Health-related quality of life was evaluated in three moments: before COVID-19, after COVID-19 (on return to work), and after 6 months. Persistence of symptoms post-COVID-19 was also assessed. There was a worsening in all dimensions of HRQOL. After 6 months, self-rated health on EuroQol visual analog scale did not return to pre-COVID-19 values. At total, 36.2% of HCWs were diagnosed with post-COVID-19 syndrome. There was a significant deterioration in HRQOL among HCWs who had COVID-19 and a high frequency of post-COVID-19 syndrome.

Cough suppression and HRQoL in adult people with cystic fibrosis: an unexplored correlation

BackgroundCough suppression assessed by embarrassment about coughing has been shown in adolescents with cystic fibrosis (CF) and negatively affects health-related quality of life (HRQoL) and clinical indicators of disease severity in adolescent females. However, whether cough suppression exists in adults has been studied as little as its effects on clinical and psychological outcomes beyond adolescence.MethodsSeventy-one subjects completed the self-reported 'Cystic Fibrosis Questionnaire-Revised (CFQ-R + 14)' and a self-report questionnaire about cough suppression, health-related perspectives, and therapy adherence. The status of CF disease was quantified in terms of the percentage of predicted forced expiratory volume in one second (ppFEV1), body mass index (BMI), Pseudomonas aeruginosa, pancreatic status, and CF-related diabetes (CFRD). Additional demographic data for sex, age, graduation, employment, and marital status were assessed.ResultsCS exists in adult CF and is associated with impaired HRQoL but not the overall CF disease status regarding BMI, ppFEV1, or health-related perspectives. Despite a higher prevalence of cough suppression in women, no effect of sex regarding either outcome measure was observed.ConclusionThe results of this study suggest that mental health indicators have an impact on cough suppression.

Confidence in treatment is contributing to quality of life in autoimmune liver diseases. The results of ERN RARE-LIVER online survey.

Autoimmune liver diseases (AILDs) are associated with impaired health-related quality of life (HrQoL). The aim of this project was to identify potentially modifiable factors related to HrQoL in a large transnational cohort of patients with AILDs. A cross-sectional online survey was conducted on patients with autoimmune hepatitis (AIH), primary biliary cholangitis (PBC) or primary sclerosing cholangitis from 15 European countries. HrQoL was measured with EQ-5D-5L and EQ visual analogue scale (EQ-VAS) and analysed in relation to demographic, psychosocial, disease- and treatment-related factors. A Patient Health Questionnaire-2 score >3 indicated relevant depression. Multivariable linear regression analyses were used to identify potentially modifiable factors associated with HrQoL and confidence in treatment whilst adjusting for known confounders. A group of 1178 European patients (79% female, mean age 48 ± 14 years) participated in the study. HrQoL was impaired in all three diseases (mean EQ-5D-5L=0.75, mean EQ VAS=68.9), most markedly in PBC (mean EQ-5D-5L=0.73, mean EQ-VAS=66.2). Relevant depression, which was detected in 17% of patients, was prominently associated with impaired HrQoL. In the regression analysis, treatment confidence was identified as an important modifiable factor positively contributing to HrQoL. This influence was observable even after adjusting for other covariates including depression. Management in a transplant centre, treatment with azathioprine in AIH, and with ursodeoxycholic acid in PBC, was associated with increased treatment confidence. Finally, improved patient-physician relationships contributed to treatment confidence. Treatment confidence is a relevant modifiable determinant of HrQoL and should be further investigated to improve the standards of care for patients with AILDs.