Evaluation Of Health-related Quality Of Life Research Articles

Influence of Early Postoperative Basal Insulin Treatment and Post-Transplant Diabetes Mellitus Risk on Health-Related Quality of Life in Kidney Transplant Recipients-An Analysis of Data From a Randomized Controlled Trial.

Health-related quality of life (HRQOL) improves after kidney transplantation (KT) but declines over time. Studies on the effect of early postoperative basal insulin therapy on HRQOL after KT, especially KTRs at high risk of developing post-transplant diabetes mellitus (PTDM) are missing. Data from a randomized controlled trial on 148 non-diabetic KTRs were analyzed. HRQOL using the KDQOL-SF™ was compared in KTRs who either received early postoperative basal insulin therapy or standard-of-care and in KTRs at risk of developing PTDM. Determinants of HRQOL outcomes were investigated using multivariable linear regression analysis. In total, 148 patients completed the KDQOL-SF at baseline. Standard-of-care or early basal insulin therapy after KT did not influence HRQOL. Overall, KT improved the mental (MCS) and physical component summary (PCS) scores at 6-month after KT, which remained stable during further follow-up visits. However, patients at high-risk for PTDM had significantly greater impairment in the PCS score (baseline, 24months) without differences in MCS scores. In the multivariable regression analysis, allograft function and hemoglobin levels were associated with decreased MCS and PCS scores, respectively. A limitation of the study is the fact that only around 50% of the ITP-NODAT study patients participated in the HRQOL evaluation. Still, our data clearly show that early basal insulin therapy does not affect HRQOL after KT but is negatively influenced by classical clinical factors and PTDM-risk at 24months after KT. The latter might be influenced by older age.

Effects of the Multicomponent Cognitive Training Program BrainProtect in Cognitively Healthy Adults: A Randomized Controlled Trial.

Cognitive integrity is a fundamental driver of health. The exact structure of strategies against cognitive impairment is still under debate. To compare the short-term effects of a multicomponent cognitive training (BrainProtect) with those of general health counseling (GHC) on cognitive abilities and health-related quality of life (HRQoL) in healthy adults in Germany. In this parallel randomized controlled trial (RCT), 132 eligible cognitively healthy adults (age ≥50 years, Beck Depression Inventory ≤9/63; Montreal Cognitive Assessment ≥26/30) were randomized to either GHC (N = 72) or to intervention with BrainProtect (intervention group, IG; N = 60). IG participants received 8 weekly sessions of 90 min of the group-based BrainProtect program focusing on executive functions, concentration, learning, perception, and imagination, plus nutritional and physical exercise units. Before and after intervention, all participants underwent neuropsychological testing and HRQoL evaluation, blinded for pretest. No significant training effect was observed for the primary endpoint of global cognition as assessed by CERAD-Plus-z Total Score (p = 0.113; ηp2 = 0.023). Improvements in several cognitive subtests were shown in the IG (N = 53) compared to the GHC (N = 62) without adverse events. Differences reached significance for verbal fluency (p = 0.021), visual memory (p = 0.013), visuo-constructive functions (p = 0.034), and HRQoL (p = 0.009). Significance was lost after adjustment, though several changes were clinically relevant. BrainProtect did not significantly impact global cognition in this RCT. Nevertheless, the results of some outcomes indicate clinically meaningful changes, so that a strengthening of the cognitive performance by BrainProtect cannot be excluded. Further studies with larger sample size are needed to confirm these findings.

Translation and validation of the Korean version of the QUAlity of Life Assessment in Spina bifida for Teenagers (QUALAS-T-K)

Purpose The Quality of Life Assessment in Spina bifida for Teenagers (QUALAS-T) is a tool used to evaluate health-related quality of life (HRQOL) in adolescents with spina bifida (SB). The purpose of this study was to translate the QUALAS-T into Korean and validate its Korean version (QUALAS-T-K). Methods Translation and validation processes were carried out in accordance with a specified protocol, including forward and back translation, a content validity study, and a main study. The tool’s reliability was evaluated based on its internal consistency and stability. Factor analysis was conducted, and convergent validity was confirmed using the KIDSCREEN-27. Results Of the 59 participants, 35 had lipomyelomeningoceles. Confirmatory factor analysis confirmed that QUALAS-T-K had the same structure as QUALAS-T. The QUALAS-T-K showed excellent internal consistency (α: 0.872–0.893, ω: 0.875–0.885), test–retest reliability (ICC:0.84–0.92), and weak to strong correlations with the KIDSCREEN-27. Conclusions The QUALAS-T-K, developed by reflecting on the characteristics of SB and considering the applicability of Korean cultural characteristics and clinical practice, is a convenient and reliable tool with excellent internal consistency and stability. This could be a useful tool in clinical and research settings for HRQOL evaluation of adolescents with SB.

Cross-cultural adaptation and validation of a self-reporting tool to assess health-related quality of life for Egyptians with extremity bone sarcomas in childhood or adolescence

BackgroundValidated self-reporting tools are required to evaluate the functional outcome and health-related quality of life (HRQOL) for those who had extremity bone sarcomas in their childhood or adolescence. Our study pursued cross-cultural adaptation and validation of the pediatric Toronto Extremity Salvage Score (pTESS) and Toronto Extremity Salvage Score (TESS) to assess the functional outcome for Egyptian children and adult survivors following surgeries of extremity bone sarcomas. In the modified versions of pTESS and TESS, mental domains were added to allow the evaluation of HRQOL using a specific instrument for childhood bone cancer.MethodsThe internal consistency and test–retest reliability of the studied forms were assessed with Cronbach’s alpha and Intra-class coefficients (ICC), respectively. For convergent validity, correlations between scores of the generic Pediatric Quality of Life Inventory (PedsQL 4.0) and pTESS /TESS scores were reported. Factor Analysis was feasible for pTESS-leg; due to the insufficient samples, only the average inter-item correlation coefficients were reported for the remaining versions.ResultsOut of 233 participants, 134 responded to pTESS-leg, 53 to TESS-leg, 36 to pTESS-arm, and only 10 to TESS-arm. All versions showed excellent internal consistency (Cronbach’s alpha >0.9), good test–retest reliability (ICC >0.8), moderate to strong correlations with PedsQL, and acceptable average inter-item correlation coefficients (≥0.3). Three factors were extracted for the pTESS-leg, in which all mental items were loaded on one separate factor with factor loadings exceeding 0.4. Active chemotherapy, less than one year from primary surgery, or tibial tumors were associated with significantly inferior pTESS/TESS scores in the lower extremity group.ConclusionThe Egyptian pTESS and TESS are valid and reliable self-reporting tools for assessing the functional outcome following surgeries for extremity bone sarcomas. The modified pTESS and TESS versions, which include additional mental domains, enabled the assessment of the overall health status of our population. Future studies should include a larger sample size and evaluate the ability of pTESS/TESS to track progress over time.

Relationship between C2 slope with sagittal parameters and clinical function of degenerative cervical kyphosis

PurposeTo explore the relationship between C2 slope with sagittal parameters and clinical function of degenerative cervical kyphosis (DCK).MethodsA retrospective analysis of 127 patients with degenerative cervical spondylosis treated in our spinal deformity center from January 2019 to June 2022. Patients were categorized into two groups and compared based on C2-7 angle (C2-7 ≥ 5° as kyphosis group, C2-7 < 5° as lordosis group). Pearson correlation or Spearman correlation was used to analyze the relationship between C2S and conventional radiological parameters and health -related quality-of-life (HRQOL) outcomes as measured by the EuroQol 5 dimension questionnaire (EQ5D), NRS, and the neck disability index (NDI). The cutoff value of C2S was determined by a receiver operating characteristic (ROC) curve.ResultsThere were 127 patients who met inclusion criteria (79 men and 48 women). Average 56.00 ± 10.27 years old (range 31–81 years old). C2S of kyphosis group is higher than non-kyphosis group. Aggravating cervical kyphosis increases cSVA positively. For all patients, C2S demonstrated a significant correlation with the O-C2 angle, C2-7 angle, cSVA, and TS-CL (p < 0.05). NRS, NDI and EQ5D-VAS scores revealed a significant correlation with C2S and cSVA (p < 0.01). For the subgroup of patients presenting with DCK, ROC curves demonstrated the cutoff values of C2S as 26.3°, and 30.5°, according to a cSVA of 40 mm, and severe disability expressed by NDI, respectively.ConclusionOn the basis of retaining the consistency of cranio-cervical and cervico-thoracic structure, C2S can better analyze the sagittal alignment of DCK patients than TS-CL and has good practicability in clinical application and HRQOL evaluation.

Changes in patient-reported outcomes in patients with non-idiopathic pulmonary fibrosis fibrotic interstitial lung disease and progressive pulmonary fibrosis.

Health-related quality of life (HRQoL) captures different aspects of the fibrotic interstitial lung disease (FILD) evaluation from the patient's perspective. However, little is known about how HRQoL changes in patients with non-idiopathic pulmonary fibrosis (IPF) FILD, especially in those with progressive pulmonary fibrosis (PPF). The aim of this study is to clarify whether HRQoL deteriorates in patients with non-IPF FILD and to evaluate the differences in the changes in HRQoL between those with and without PPF. We collected data from consecutive patients with non-IPF FILD and compared annual changes in HRQoL over 2 years between patients with PPF and those without. The St George's respiratory questionnaire (SGRQ) and COPD assessment test (CAT) were used to assess HRQoL. Changes in the SGRQ and CAT scores for 24 months from baseline were evaluated with a mixed-effect model for repeated measures. A total of 396 patients with non-IPF FILD were reviewed. The median age was 65 years and 202 were male (51.0%). The median SGRQ and CAT scores were 29.6 and 11, respectively. Eighty-six (21.7%) showed PPF. Both SGRQ and CAT scores were significantly deteriorated in patients with PPF compared to those without PPF (p < 0.01 for both). Clinically important deterioration in the SGRQ and CAT scores were observed in 40.0 and 35.7% of patients with PPF and 11.7 and 16.7% of those without, respectively. PPF was significantly associated with clinically important deterioration in the SGRQ score (odds ratio 5.04; 95%CI, 2.61-9.76, p < 0.01) and CAT score (odds ratio 2.78; 95%CI, 1.27-6.06, p = 0.02). The SGRQ and CAT scores were significantly deteriorated in patients with non-IPF FILD and PPF. Considering an evaluation of HRQoL would be needed when assessing PPF.

Quality of Life Among Children With Type 1 Diabetes Mellitus in Alahsa: A Cross-Sectional Study.

Diabetes mellitus (DM) is the most common endocrine disease in children, and its annual prevalence is increasing worldwide. Health-related quality of life (HRQoL) is a crucial indicator of chronic illnesses such as DM. This study aimed to assess the HRQoL and determine the associated factors among children and adolescents with type 1 DM in Alahsa region in 2022. An analytical cross-sectional study was conducted in the DiabeterSA center using the Arabic version of the Pediatric Quality of Life Inventory (PedsQl 3.2). All patients aged 5-16 years and diagnosed with type 1 DM were included in the study. Face-to-face interviews were conducted during the patients' routine visits to the outpatient clinic from September 2022 to January 2023. A total of 254 children aged 5-16 years (mean age: 10.87 ± 2.89 years) were recruited. The mean HRQoL total score reported by the children and adolescents was 72.61 ± 15.36. Older age, higher family socioeconomic status, excellent school performance, and higher parental education level, particularly in mothers, were significantly associated with higher total HRQoL scores. In the regression analysis, male sex (β = -0.157,P = 0.018), older age (β = 0.246,P <0.001), and excellent school performance (β = -0.290,P < 0.001) were identified as independent predictors of the HRQoL total score. Nearly19% of the participants achieved glycemic control. The quality of life of children and adolescents with type 1 DM in Alahsa region was relatively good. Increased age, good family economic status, and higher parent education levels positively influenced the participants' quality of life. Therefore, regular evaluation of HRQoL is recommended for children and adolescents with type 1 DM to identify problems and initiate an appropriate intervention for improving child health and the health care system.

PD15-02 LONG-TERM HEALTH RELATED QUALITY OF LIFE IN PROSTATE CANCER PATIENTS REQUIRING RADIOTHERAPY AFTER RADICAL PROSTATECTOMY

PD15-02 LONG-TERM HEALTH RELATED QUALITY OF LIFE IN PROSTATE CANCER PATIENTS REQUIRING RADIOTHERAPY AFTER RADICAL PROSTATECTOMY

Improvements in body mass index of children with cystic fibrosis following implementation of a standardized nutritional algorithm: A quality improvement project.

A collaboration between the University of Michigan (UM) Cystic Fibrosis Center (CFC) and Marmara University (MU) CFC was initiated in MU through conducting Quality Improvement projects (QIP). The global aim was to improve nutritional status of children with CF (cwCF), with a specific aim to increase the mean BMI percentile (BMIp) for cwCF by 10 percentile points in 12 months. Body mass index (BMI) percentiles of cwCF were categorized as: nutritionally adequate (BMIp ≥ 50%); at risk (BMIp 25%-49%); urgently at risk (BMIp 10%-25%); critically at risk (BMIp < 10%). Appropriate interventions were made according to BMIp category every three months. Forced expiratory volume in one-second percent predicted (FEV1pp), and health-related quality of life (HRQoL) were evaluated. One hundred and eight-two cwCF with a mean age of 9.1 ± 4.3 years were included in the project. Baseline BMIp increased from 25.6 to 37.2 at the 12th month (p < 0.001). In the critically at-risk group BMIp increased from 3.6 to 20.5 (p < 0.001), in the urgently at risk group from 15.9 to 30.8 (p < 0.001), in the at risk group from 37.0 to 44.2 (p < 0.079) and in the nutritionally adequate group the increase was from 66.8 to 69.5 (p < 0.301). FEV1pp also improved significantly, from 81.3 ± 20.6 to 85.9 ± 20.8 (p < 0.001). Physical functioning, eating problems, and respiratory symptoms domains of the HRQoL evaluation improved (p < 0.05). This project has led to significant improvements in BMIp, FEV1pp and HRQoL of cwCF; similar projects could easily be implemented by centers in other developing countries.

Amylo-AFFECT-QOL, a self-reported questionnaire to assess health-related quality of life and to determine the prognosis in cardiac amyloidosis.

Self-reported questionnaires are useful for estimating the health-related quality of life (HR-QoL), impact of interventions, and prognosis. To our knowledge, no HR-QoL questionnaire has been developed for cardiac amyloidosis (CA). This study aimed to validate Amylo-AFFECT-QOL questionnaire to assess HR-QoL and its prognostic value in CA. A self-reported questionnaire, "Amylo-AFFECT" had been designed and validated for CA symptoms evaluation and screening by physicians. It was adapted here to assess HR-QoL (Amylo-AFFECT-QOL) and its prognostic value in CA. To validate the theoretical model, internal consistency and convergent validity were assessed, particularly correlations between Amylo-AFFECT-QOL and the HR-QoL Minnesota Living Heart Failure (MLHF) questionnaire. Amylo-AFFECT-QOL was completed by 515 patients, 425 of whom (82.5%) had CA. Wild-type and hereditary transthyretin amyloidosis (ATTRwt and ATTRv) and immunoglobulin light-chain amyloidosis (AL) were diagnosed in 47.8, 14.7, and 18.8% of cases, respectively. The best HR-QoL evaluation was obtained with five dimensions: "Heart failure," "Vascular dysautonomia," "Neuropathy," "Ear, gastrointestinal, and urinary dysautonomia," and "Skin or mucosal involvement." The global Amylo-AFFECT-QOL and MLHF scores showed significant positive correlations (rs = 0.72, p < 0.05). Patients with a final diagnosis of CA had a global Amylo-AFFECT-QOL score significantly higher than the control group composed by patients with other diagnoses (22.2 ± 13.6 vs. 16.2 ± 13.8, respectively, p-value < 0.01). According to the Amylo-AFFECT-QOL global results, ATTRv patients' QoL was more affected than AL patients' QoL or ATTRwt patients' QoL. Patients with a higher HR-QoL score had a greater risk of death or heart transplant after 1 year of follow-up (log-rank < 0.01). Amylo-AFFECT-QOL demonstrates good psychometric properties and is useful for quantifying HR-QoL and estimating CA prognosis. Its use may help to improve overall management of patients with CA.

Subjective HRQoL in Patients with Sleep Apnea Syndrome Who Underwent PAP Therapy in a Rehabilitation Setting: A Longitudinal Study

Obstructive sleep apnea (OSA) is often associated with decreased health-related quality of life (HRQoL). The aims of this study were to evaluate HRQoL, the clinical and psychological profile of suspected or verified OSA patients, and the impact of PAP therapy at 1-year follow-up. At T0, OSA-suspected subjects underwent clinical, HRQoL, and psychological assessment. At T1, OSA patients underwent PAP therapy in a multidisciplinary rehabilitation setting. At 1 year follow-up, OSA patients were evaluated for the second time. At T0, OSA patients (n = 283) and suspected OSA subjects (n = 187) differed for AHI, BMI, and ESS. At T0, the PAP-treatment group (n = 101) showed moderate-severe anxious (18.7%) and depressive (11.9%) symptoms. At 1 year follow-up (n = 59), the sleep breathing pattern had normalized and there was a reduction of ESS scores and anxious symptoms. There was also an improvement in HRQoL (0.6 ± 0.4 vs. 0.7 ± 0.5, p = 0.032; 70.4 ± 19.0 vs. 79.2 ± 20.3, p = 0.001) and in satisfaction with sleep quantity (52.3 ± 31.7 vs. 71.4 ± 26.2, p = 0.001), sleep quality (48.1 ± 29.7 vs. 70.9 ± 27.1, p = 0.001), mood (58.5 ± 24.9 vs. 71.0 ± 25.6, p = 0.001), and physical resistance (61.6 ± 28.4 vs. 67.8 ± 27.4, p = 0.039). Considering the impact of PAP treatment on patients' psychological and HRQoL evaluations that we observed, our data are valuable for unveiling different profiles characterizing this clinical population.

Health-related quality of life outcomes following Roux-en-Y gastric bypass versus one anastomosis gastric bypass.

Roux-en-Y gastric bypass (RYGB) and one anastomosis gastric bypass (OAGB) are effective standard bariatric surgeries with comparable weight loss and remission of obesity-related comorbidities. As procedure-specific health-related quality of life (HrQoL) outcomes have not been directly compared thus far, we conducted this questionnaire-based study. Two hundred forty patients after undergoing either RYGB or OAGB between 2011 and 2016 were contacted and asked to fill out SF36 and BAROS questionnaires. All statistical analysis was performed with Microsoft Excel and GraphPad Prism. Primary objectives were procedure-dependent differences in HrQoL. Secondary objectives were weight loss and remission of comorbidities. One hundred nineteen of 240 contacted patients (49.6%) replied, 58 after RYGB (48.7%) and 61 after OAGB (51.3%). Follow-up period was < 24months in 52 and > 24months in 64 evaluable patients. The mean age was 46years (range 23 to 71). Regarding the < 24months groups, both physical and psychological SF36 sum scales were comparably high. Only the subcategory "general health perception" was significantly better after RYGB. Significantly higher excess weight loss (EWL) after RYGB (88.81%) compared to OAGB (66.25%) caused significantly better global < 24months BAROS outcomes, whereas remission of comorbidities and HrQoL was similar. Both > 24months groups showed high SF36-HrQoL sum scales. Global mean BAROS results after > 24months were "very good" in both procedures. EWL in RYGB (80.81%) and in OAGB (81.36%) were comparably excellent. Concerning SF36 and BAROS evaluated HrQoL in early and late postoperative phases, both procedures demonstrated comparable and relevant improvements. Further (preferably randomized) studies should include evaluation of preoperative HrQoL.

Health-related quality of life after curative treatment for muscle-invasive bladder cancer.

Muscle-invasive bladder cancer (MIBC) is an aggressive disease for which the gold-standard treatment is radical cystectomy (RC) in combination with cisplatin-based neoadjuvant chemotherapy. Bladder-sparing strategies such as trimodal therapy (TMT) have also emerged to improve health-related quality of life (HRQoL) of patients. However, an improved understanding of the effect of all these treatment modalities on HRQoL is essential to provide personalized patient care. Different combinations of generic, cancer-specific and bladder cancer-specific questionnaires can be used as instruments for HRQoL evaluation in patients with MIBC before and after curative treatments, which can largely affect multiple domains of HRQoL including general health as well as physical, functional, social and emotional well-being. However, diagnosis of MIBC per se is also likely to affect HRQoL, and the perspective of cure after RC or TMT could induce a return to baseline HRQoL values for most of these domains. A considerable amount of data on HRQoL after RC is available, but conflicting results have been reported regarding the effect of urinary diversion (ileal conduit or orthotopic neobladder) and surgical approach (open or robotic surgery) on patient quality of life. Data on HRQoL after TMT are scarce, and additional comparative studies including patients receiving RC (especially using ileal orthotopic neobladder) are needed.

Characterizing the Clinical, Health-Related Quality of Life and Economic Burden of Alpha-Thalassemia: A Systematic Literature Review and Evidence Gaps Assessment

Introduction: The burden of beta-thalassemia (β-thal) is well documented but, to our knowledge, there are no systematic literature reviews (SLRs) on the burden of alpha-thalassemia (α-thal). To fill this gap, we conducted an SLR to characterize the clinical (complications, treatment patterns, or mortality), health-related quality of life (HRQoL), and economic burden associated with α-thal and report on evidence gaps. Methods: An SLR was conducted for publications on clinical burden, HRQoL, HCRU/costs and economic evaluations in thalassemia. MEDLINE, Embase, EconLit and Evidence-Based Medicine Reviews and relevant conference websites (2019-2021) were searched. Real-world, observational studies and economic evaluations were selected that reported on α-thal and any outcome data of interest; for clinical burden, only studies in adults (≥18 years) were included. Results: Across 7,881 search hits in thalassemia, 9 studies reported relevant data on α-thal (see Table 1).1-9 No evidence was identified on economic evaluations. Only three studies were exclusively in α-thal patients (sample size 50-86) and were limited to deletional (DEL) HbH and/or non-deletional (non-DEL) HbH, including Constant Spring (CS).2, 4, 9 These studies found that among adults with DEL and/or non-DEL HbH, almost 1 in 3 had moderate-to-severe iron overload (31%),2 and 2 out of 3 had iron overload of unspecified severity (66%).9 Advanced liver fibrosis was present among 1 in 5 patients (20%); 9% with probable cirrhosis.2 Complications including moderate-to-severe liver iron overload (53% vs. 25%, P=0.03) and median liver iron concentration (median [range]: 7.8 [1.7-17.7] vs. 2.9 [0.1-15.0] mg/g dry weight, P=0.002) were significantly higher in non-DEL versus DEL HbH adult patients with non-transfusion dependent thalassemia (NTDT).2 In addition, among HbH/CS transfusion-dependent thalassemia (TDT) and NTDT adult patients, iron overload was further exacerbated among those who were splenectomized compared to those who were not (80% vs. 52%, P=0.037).9 The other studies reporting clinical data were from a mixed (α-thal and β-thal) population where data for α-thal was reported separately (6 studies with sample sizes ranging from 2 to 54 with α-thal).1, 3, 5-8 Across these, the data also demonstrated a high burden of clinical complications for HbH and/or HbH/CS including iron overload (52.2%, 1 study)3, cholelithiasis (27.7% to 52.2%, 3 studies3, 6, 8), abnormal liver function (27.7%, 1 study8), osteoporosis (0.0% to 20.4%, 3 studies3, 6, 8) and hyperuricemia (60.0%, 1 study).1 Complication rates among α-thal patients across studies can be seen in Figure 1. With regard to treatment patterns, among patients with DEL and/or non-DEL HbH, the majority had historical/occasional transfusion (46.7% to 87.0%, 3 studies)2, 3, 6, followed by iron chelation therapy (6.0% to 52.2%, 5 studies)2, 3, 6, 8, 9 and splenectomy (10.0% to 14.8%, 3 studies).2, 6, 8 A single study on HRQoL in TD HbH and β-thal (age range: 2 to 18 years) found that HRQoL total score, and subdomains, psychological, emotional, social, and school functioning (as measured by Pediatric Quality of Life Inventory) were similar between HbH and β-thal patients, except for physical functioning.7 For physical functioning, homozygous β-thal patients had the best HRQoL, followed by HbH patients, with β-thal/Hb E patients demonstrating the poorest HRQoL (mean [SD]: 76.52 [15.23] vs. 61.16 [18.39] vs. 56.88 [17.17], respectively, P=0.008).7 This study was limited by a small sample size (n=49) with only 5 HbH patients.7 Lastly, one study on HCRU among adult and pediatric patients with DEL HbH and HbH/CS, found that patients with HbH/CS had a significantly increased number annual clinic visits, by a factor of 1.7, and hospital admissions, by a factor of 3.9, versus those with HbH (P<0.001).4 Conclusions: To our knowledge, this was the first SLR investigating the clinical, HRQoL and economic burden of α-thal. Most of the evidence was in HbH and HbH/CS adult patients, and complications, with no data on economic evaluations, and limited data on HRQoL and HCRU/costs. Our SLR highlights the need for further research to fully characterize the burden of this disease. Where reported, DEL and/or non-DEL HbH adult patients experience clinical complications across a range of conditions and generally, children and adolescents with HbH experience similar HRQoL burden as those with β-thal. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

Health-related quality of life in Japanese patients with multiple sclerosis.

Neurological disabilities, especially physical issues, can adversely affect the daily lives of people with multiple sclerosis (MS) and negatively impact their health-related quality of life (HRQOL). On the other hand, physical and psychiatric symptoms are variable in people with MS, and QOL can be influenced by cultural and educational background. This study aimed to evaluate the association of HRQOL with disabilities, fatigue, and depression in Japanese subjects with MS. Evaluation of HRQOL, fatigue, and depression was performed in 184 Japanese individuals with MS, using the Functional Assessment of MS (FAMS), Fatigue Severity Scale (FSS), and Beck Depression Inventory-Second Edition (BDI-II), respectively. Multiple linear regression analysis demonstrated negative correlations of the Expanded Disability Status Scale (EDSS) with scores on the FAMS subscales of mobility, symptoms, thinking and fatigue, total FAMS, and additional concerns. The FSS score had negative correlations with mobility, symptoms, emotional well-being, thinking and fatigue, total FAMS, and additional concerns. There were negative correlations between BDI-II scores and all items of FAMS. HRQOL had relatively close correlations with disabilities and fatigue, and depression had an especially close relationship with HRQOL.

Subclinical cognitive impairment in chronic kidney disease is associated with frailty and reduced quality of life.

Cognitive impairment (CI) in chronic kidney disease (CKD) is highly prevalent and is associated with multiple limitations to patients as well as a higher mortality, more days of hospitalisation and a lower quality of life. Frailty in CKD is associated with adverse health outcomes and is also highly prevalent. The aim of our study was to determine the prevalence and characteristics of CI and relate the findings to frailty, mobility, muscle strength and health-related quality of life (HRQOL). Non-dialysis patients with CKD stages 3-5 were prospectively evaluated for inclusion. Excluded were patients with other cognitive disorders, signs of overt uraemic encephalopathy, severe infection and hyponatraemia. All patients underwent psychometric testing (five different tests): assessments of mobility, strength and frailty and an evaluation of HRQOL. Based on the number of pathological psychometric test results, we established two different definitions of CI: subclinical uraemic encephalopathy 1 (SUE1: one pathological test) and subclinical uraemic encephalopathy 2 (SUE2: two or more pathological test results). Sixty-two patients were included [median age 66years (interquartile range 57-75), male 55%]. Most patients had CKD stage 3 (48%; stage 4: 32%; stage 5: 19%). CI was highly prevalent (SUE1: 60%; SUE2: 42%) and associated with a higher risk of falls (pathological tandem gait test; SUE1: 50% versus 16%, P=.023; SUE2: 69% versus 15%, P=.001), lower muscle strength (SUE2-pathological: 39% versus 7%, P=.008), frailty (SUE1: 59% versus 28%, P=.038; SUE2: 67% versus 33%, P=.028) and HRQOL. CI is highly prevalent in non-dialysis CKD patients. Even mild CI is associated with decreased mobility, muscle strength and HRQOL and increased frailty.

The Relationship between Health-Related Quality of Life, Subjective Scar Estimation, and Activity Performance in Adult Burn Patients 6 and 12 Months after Injury

A burn injury affects a person’s health-related quality of life (HRQoL) in different ways and might influence their daily life for months and years afterward. The aim of this study was to examine how activity performance and subjective scar estimation relate to self-rated health and whether this changes in the first year post-burn. Fifty consecutive patients who were scheduled for follow-up at the Burn Center’s outpatient clinic in Uppsala were included. Assessments of HRQoL (EQ-5D), activity performance (DASH), and subjective scar evaluation (POSAS) were conducted at 6 and 12 months post-burn. The results show a statistically significant correlation between self-rated HRQoL and activity performance (p = 0.001) and between self-rated HRQoL and subjective scar estimation (p = 0.000) at 6 but not at 12 months post-burn. A possible explanation of the lack of correlation at one year post-burn might be the patient´s expectations of his or her recovery. In future research, it would be interesting to investigate the long-term correlations between quality of life and activity performance.

Abstract 8 Health-Related Quality of Life (HRQL) Following Transplantation with Omidubicel Versus Umbilical Cord Blood (UCB) in Patients with Hematologic Malignancies: Results from a Phase III Randomized, Multicenter Study

IntroductionOmidubicel, an advanced cell therapy used for allogeneic hematopoietic stem cell transplant has demonstrated faster hematopoietic recovery, shorter hospitalization, and lower rates of bacterial, viral, and invasive fungal infections compared with umbilical cord blood (UCB) in a phase III randomized trial (NCT02730299;Blood 2021;138:1429).ObjectiveThe objective was to compare changes in health-related quality of life (HRQL) between treatment groups in the phase III trial.MethodsPatients who received protocol-defined treatment and provided HRQL evaluations at baseline and ≥1 follow-up visit were analyzed. Outcomes included Functional Assessment of Cancer Therapy General (FACT-G) domain scores for physical, social/family, functional and emotional well-being, and EQ-5D-3L index scores, at days 42, 100, 180, and 365 post-transplant. HRQL changes from baseline were compared using mixed effect models with repeated measures, adjusting for age, sex, race, region, primary diagnosis, HCT comorbidity index, and baseline HRQL score. Average HRQL over time was compared using the area under the curve (AUC) of mean HRQL trajectories in each treatment group.ResultsSeventy-five patients (omidubiceln = 37, UCB n = 38) provided HRQL data for inclusion and were representative of the full randomized population (N = 125) at baseline. Median age was 38 years, and 41% were female. During the first year post-transplant, patients receiving omidubicel had numerically superior average FACT-G domain and EQ-5D-3L index scores compared with UCB, with mean differences across time points ranging from 1.4 to 3.1 for physical well-being, 0 to 1.3 for social/family well-being, 0.5 to 1.4 for emotional well-being, 1.6 to 3.2 for functional well-being, and 0.03 to 0.09 for the EQ-5D-3L index score. Minimal clinically important differences (MCIDs) were exceeded at ≥1 time point for mean physical and functional well-being (MCIDs = 2 units) and for the EQ-5D-3L (MCID = 0.07 units). Initial mean declines in HRQL occurred for all measures at day 42 and were consistently numerically smaller in the omidubicel group than in the UCB group. Averaging across the first year post-transplant, patients receiving omidubicel had significantly improved HRQL for physical and functional well-being domains (P < .05 for comparison of AUCs).DiscussionAlong with faster time to engraftment, lower infection risk, and shorter hospitalization, omidubicel was associated with meaningfully greater preservation or improvement of important HRQL domains compared with UCB.

RETRACTED ARTICLE: Neurocognitive impairment and patient–proxy agreement on health-related quality of life evaluations in recurrent high-grade glioma patients

PurposeThe rate of missing data on patient-reported health-related quality of life (HRQOL) in brain tumor clinical trials is particularly high over time. One solution to this issue is the use of proxy (i.e., partner, relative, informal caregiver) ratings in lieu of patient-reported outcomes (PROs). In this study we investigated patient–proxy agreement on HRQOL outcomes in high-grade glioma (HGG) patients.MethodsGeneric and disease-specific HRQOL were assessed using the EORTC QLQ-C30 and QLQ-BN20 in a sample of 501 patient–proxy dyads participating in EORTC trials 26101 and 26091. Patients were classified as impaired or intact, based on their neurocognitive performance. The level of patient–proxy agreement was measured using Lin’s concordance correlation coefficient (CCC) and the Bland–Altman limit of agreement. The Wilcoxon signed-rank test was used to evaluate differences between patients’ and proxies’ HRQOL.ResultsPatient–proxy agreement in all HGG patients (N = 501) ranged from 0.082 to 0.460. Only 18.8% of all patients were neurocognitively intact. Lin’s CCC ranged from 0.088 to 0.455 in cognitively impaired patients and their proxies and from 0.027 to 0.538 in cognitively intact patients and their proxies.ConclusionWhile patient–proxy agreement on health-related quality of life outcomes is somewhat higher in cognitively intact patients, agreement in high-grade glioma patients is low in general. In light of these findings, we suggest to cautiously consider the use of proxy’s evaluation in lieu of patient-reported outcomes, regardless of patient’s neurocognitive status.

Stigma and its association with health-related quality of life in adults with epilepsy

ObjectiveTo explore the presence of felt and enacted stigma in people with epilepsy (PWE), members of a self-governing epilepsy organization and to evaluate the influence of both types of stigma on health-related quality of life (HRQoL) in PWE. MethodsParticipants were 55 PWE (age range: 18–53 years), members of a non-governmental organization (further “NGO”). The sociodemographic and epilepsy-related variables were collected through structured interviews designed for the purpose of the study. Felt stigma was assessed with the Epilepsy Stigma Scale (ESS) and enacted stigma with the Questionnaire for episodes of discrimination against PWE. Quality of Life in Epilepsy Inventory (QOLIE-31) (Serbian version) was used for the evaluation of HRQoL. ResultsThe mean ESS score was 33.93 ± 14.50. Felt stigma was significantly associated with the male gender and the number of antiepileptic drugs (AEDs). Participants’ mean score on the Questionnaire for episodes of discrimination was 2.80 ± 2.78. Enacted stigma was significantly correlated with male gender and number of AEDs, as well as with older age/longer duration of the disorder. Felt stigma scores were positively correlated with enacted stigma scores (p < 0.001), but only felt stigma scores were negatively related to QOLIE-31 overall score (p = 0.01). The combination of AEDs, seizure frequency, and felt stigma best explained the HRQoL in PWE (p < 0.001). ConclusionFelt and enacted stigma are moderately related, but only felt stigma appears to be a significant predictor of the deteriorating HRQoL in this sample. Interventions targeting felt stigma should be considered a part of comprehensive epilepsy care as well as educating the wider community about epilepsy.