Health-related Quality Of Life Impact Research Articles

Health-related quality of life and clinical characteristics of hidradenitis suppurativa in a paediatric population

Hidradenitis Suppurativa (HS) is a chronic, Skin Immune-Mediated Inflammatory Disease (sIMID), with a high impact on Quality of Life (QoL). Data on clinical management, comorbidities, psychosocial burden, and psychiatric disorders in paediatric patients with HS is scarce. The aim of our study was to compare clinical characteristics and Patient-Reported Outcome Measures (PROMs) for Health-Related Quality of Life (HRQoL) in adult and paediatric patients with HS. Data were collected on 601 patients with HS, 60 of whom (10.0%) were paediatric. The psychological and HRQoL impact was generally higher in adults than in children, but it was noteworthy also in paediatric patients. Early diagnosis and interventions based on education for the patients and their families and psychological support are the key to optimal disease management and adherence. Our study underlines the need for screening for HS-associated psychiatric disorders and for using age-specific tools to evaluate HRQoL. A multidisciplinary approach involving several specialists is needed, as well as liaison with primary care specialists to improve care, and management of comorbidities and psychological issues in patients with HS.

The Development and Content Validation of the Sjögren's Related Quality of Life Instrument (SRQoL).

Several clinical outcome assessment (COA) instruments assess Sjögren's disease (Sjögren's) symptoms, but do not provide comprehensive assessment of the health-related quality of life (HRQoL) impact of Sjögren's. This study aimed to develop a patient-reported outcome (PRO) instrument for the assessment of HRQoL, intended for use in clinical trials and clinical practice in the assessment of treatment benefit. Review of study sponsor proprietary data and qualitative interviews informed the development of a conceptual model, the Sjögren's Related Quality of Life (SRQoL) and patient global impression of severity (PGI-S) and change (PGI-C) items. Combined concept elicitation and cognitive debriefing interviews with patients with Sjögren's explored their HRQoL impact experience and content validity of the SRQoL and PGI items. Twenty participants were interviewed about their Sjögren's experience. Following inductive analysis of interviews, concepts were categorized into eight domains: emotional well-being (e.g., worry and stress; n = 20/20; 100%), sleep (e.g., daytime sleepiness and waking up during the night; n = 20/20; 100%), activities of daily living (e.g., difficulty looking at screens and difficulty driving; n = 20/20; 100%), cognition (e.g., concentration difficulties and word finding difficulties; n = 19/20; 95.0%), physical functioning (e.g., difficulty walking and difficulty exercising; n = 19/20; 95.0%), social and family functioning (e.g., dependent on others and relationship difficulties; n = 17/20; 85.0%), work (n = 15/20; 75.0%), and sexual functioning (n = 12/20; 60.0%). SRQoL and PGI items, instructions, response options, and recall period were well understood and relevant to participants. The SRQoL is a new PRO instrument to assess Sjögren's impact on HRQoL, developed in accordance with regulatory guidance. This study provides considerable insight into the patient experience of Sjögren's and evidence to support the content validity of the SRQoL. Future research should evaluate the psychometric properties of the SRQoL to support its use in clinical trials and clinical practice and further validate its use as an assessment of treatment benefit.

Diverse phenotype of Ménière’s disease associated with family history, thyroid disorder, migraine and associated disorders

ObjectiveTo better understand the clinical phenotype of Ménière’s disease (MD), we examined family history, thyroid disorder, migraine, and associated disorders in complaints of people living with MD. MethodWe designed the study as a retrospective and examined data gathered from 912 participants with MD. Their data were originally collected by the Finnish Ménière Federation (FMF). The survey data included individual case histories for environmental factors, comorbidities, disease-specific complaints, impact-related questions, cognitive complaints, health-related quality of life (HRQoL), and sense of coherence (SOC). ResultsWe observed significant differences between those with and without sporadic occurrence, family history, thyroid disorder, and migraine-associated complaints. Family history explained 20% of variability in patient complaints. Patients with a family history of MD whose disease started at younger age experienced balance problems, more severe vertigo spells, more severe vestibular drop attacks (VDA), and less nausea, although they had good SOC. Thyroid disorder explained 14% of variability in patient complaints. MD patients with a thyroid disorder comorbidity suffered more often from constant dizziness, balance problems, greater impact of hearing problems, cognitive complaints, and poor HRQoL. Migraine explained 12% of variability in patients’ complaints and was associated with poor SOC and cognitive balance problems. MD patients with both thyroid disorder and migraine used antidepressants more often than other groups. Logistic regression analysis showed comorbidities of ischemic brain disorder (among 7.1%), kidney insufficiency (among 1.2%), and diabetes (among 7.3%) had statistically significant but restricted association with balance and gait problems, VDA, and reduced HRQoL. ConclusionsFamily history of MD and thyroid disorder or migraine comorbidities in MD influence the complaint pattern and partially explain complex symptom profiles, including symptoms of cognitive problems. Confounders play a minimal role in complaint profile and impact of MD whereas comorbidities influence the complaint structure and partly explain the complex symptom profile in MD.

Steroid myopathy in patients with myasthenia gravis: a literature review

Steroid myopathy is a common drug-induced non-inflammatory myopathy that affects patients requiring long-term glucocorticoid treatment for various autoimmune, inflammatory and oncological diseases. According to the neurology clinical practice guidelines, non-fluorinated glucocorticoids are the first-line pathogen-directed therapy for a number of dysimmune neuromuscular disorders, including myasthenia gravis. Long-term high-dose steroid treatment regime for myasthenia gravis leads to both acute and chronic development of glucocorticoids-induced proximal muscle weakness and atrophy. Steroid myopathy, along with other undesirable side effects of glucocorticoids therapy, impact health-related quality of life, patient satisfaction and adherence to treatment. Hence, further studies are required to expand our knowledge of clinical evaluation, diagnostic testing and prevention approaches for glucocorticoids-induced myopathy. The aim of this literature review is to analyze existing data on pathogenesis, diagnostic tools and treatment strategies for steroid myopathy.

Psychometric Properties of the EQ-5D-5L in Patients with Alopecia Areata.

For many decision makers in Health Technology Assessment the EQ-5D-5L is the standard measure of health-related quality of life (HRQL). However, evidence has shown the limitations of the measure in certain disease areas, including dermatology. Alopecia areata (AA) is associated with a significant HRQL impact, partly due to the emotional impact of hair loss. This study explores the psychometric properties of the EQ-5D-5L in people with AA in reference to the short-form 36 version 2 (SF-36v2), the Alopecia Areata Patient Priority Outcomes (AAPPO), the Severity of Alopecia Tool (SALT) and the Patient Global Impressions of Change (PGI-C). Data from participants with AA enrolled in the ALLEGRO-2b/3 trial (NCT03732807) of ritlecitinib were analysed. Participants completed the AAPPO measure (an AA-specific measure assessing emotional symptoms and activity limitations), PGI-C, EQ-5D-5L and SF-36v2 across 48-weeks of follow up. Extent of scalp hair loss was assessed using the SALT. Ceiling effects, known groups validity, convergent validity and responsiveness were examined. Known groups were defined by SALT score and a PGI-C defined response from baseline. Exploratory factor analysis was also performed. Data were available from 612 adult participants. Ceiling effects were observed for the EQ-5D-5L (55.3-61.2%) and analyses suggested that the EQ-5D did not capture important differences between patients that the SF-36v2 did. The EQ-5D-5L very weakly correlated with SALT score, whereas the AAPPO correlated more strongly with the extent of hair loss. Compared with the EQ-5D-5L, the AAPPO was better able to discriminate between known groups defined by SALT and PGI-C. An exploratory factor analysis suggested that the EQ-5D-5L had limitations in content validity compared with the AAPPO. The EQ-5D-5L may not adequately measure the burden of AA on patients' HRQL. Insensitivity to the burden of AA suggests that the EQ-5D-5L may not measure treatment-related benefit with hair regrowth. Data from other measures could be considered if they are shown to be more relevant.

Demonstrating responsiveness of the pediatric cardiac quality of life inventory in children and adolescents undergoing arrhythmia ablation, heart transplantation, and valve surgery.

Pediatric Cardiac Quality of Life Inventory (PCQLI) is a disease-specific pediatric cardiac health-related quality of life (HRQOL) instrument that is reliable, valid, and generalizable. We aim to demonstrate PCQLI responsiveness in children undergoing arrhythmia ablation, heart transplantation, and valve surgery before and after cardiac intervention. Pediatric cardiac patients 8-18 years of age from 11 centers undergoing arrhythmia ablation, heart transplantation, or valve surgery were enrolled. Patient and parent-proxy PCQLI Total, Disease Impact and Psychosocial Impact subscale scores were assessed pre- and 3-12 months follow-up. Patient clinical status was assessed by a clinician post-procedure and dichotomized into markedly improved/improved and no change/worse/much worse. Paired t-tests examined change over time. We included 195 patient/parent-proxies: 12.6 ± 3.0 years of age; median follow-up time 6.7 (IQR = 5.3-8.2) months; procedural groups - 79 (41%) ablation, 28 (14%) heart transplantation, 88 (45%) valve surgery; clinical status - 164 (84%) markedly improved/improved, 31 (16%) no change/worse/much worse. PCQLI patient and parent-proxies Total scores increased (p ≤ 0.013) in each intervention group. All PCQLI scores were higher (p < 0.001) in the markedly improved/improved group and there were no clinically significant differences in the PCQLI scores in the no difference/worse/much worse group. The PCQLI is responsive in the pediatric cardiac population. Patients with improved clinical status and their parent-proxies reported increased HRQOL after the procedure. Patients with no improvement in clinical status and their parent-proxies reported no change in HRQOL. PCQLI may be used as a patient-reported outcome measure for longitudinal follow-up and interventional trials to assess HRQOL impact from patient and parent-proxy perspectives.

Development of a novel patient reported outcome measure for health-related quality of life in amyotrophic lateral sclerosis (PROQuALS): study protocol

BackgroundPatient reported outcome measures (PROMs) can be used to assess the impact of health conditions upon an individual’s health-related quality of life (HRQoL). Whilst PROMs have been used to quantify the HRQoL impact of amyotrophic lateral sclerosis (ALS), existing instruments may not fully capture what matters to people living with ALS (plwALS) or be appropriate to be used directly to inform the cost-effectiveness of new treatments. This highlights a need for a new condition-specific PROM that can both capture what’s important to plwALS and be used in economic evaluation. This study has two key aims: 1) to produce a novel PROM for measuring HRQoL in plwALS (PROQuALS). 2) to value a set of items from the novel PROM to generate an associated preference-weighted measure (PWM) that will enable utility values to be generated.MethodsA mixed-methods study design will be conducted across three stages. Stage 1 involves concept elicitation and the generation of draft PROM content from a robust and comprehensive systematic review of HRQoL in ALS, with input from plwALS. Stage 2 consists of cognitive debriefing of the draft PROM content to ascertain its content validity (Stage 2a), followed by a psychometric survey (Stage 2b) to assess statistical performance. Evidence from Stage 2 will be used to make decisions on the final content and format of the novel PROM. Stage 3 will involve valuation and econometric modeling using health economics methods to generate preference weights, so a PWM derived from the novel PROM can be used in the cost-effectiveness analyses of treatments. Patient and clinical advisory groups will have critical, collaborative input throughout the project.DiscussionThe novel PROM will be designed to comprehensively assess important aspects of HRQoL to plwALS and to quantify HRQoL in terms of subjective impact. The PROQuALS measure will be available for use in research and healthcare settings. The associated PWM component will extend and enable the use of PROQuALS in cost-effective analyses of new treatments for ALS.Trial registrationNot applicable.

Can minimally invasive transforaminal lumbar interbody fusion achieve a better clinical and radiological outcome than traditional open technique in isthmic spondylolisthesis?

BackgroundSpondylolisthesis is a prevalent condition in the lumbar spine that can cause low back pain, leg pain, neurogenic claudication, and impact health-related quality of life in symptomatic individuals.Aimto assess the results of minimally invasive TLIF (MIS-TLIF) versus open-TLIF and the impact of correcting spino-pelvic parameters on the Health-Related Quality of Life (HRQoL) in patients with low-grade isthmic spondylolisthesis. The primary objective was to compare the effectiveness of both methods in correcting spinopelvic parameters. The secondary objectives involved comparing clinical improvement, operating time, blood loss, complications, and postoperative hospital stays between the two procedures.Patients and methodsSeventy-two patients with low-grade isthmic spondylolisthesis were enrolled in this retrospective cohort-control study, with a minimum follow-up period of 18 months. Disability was assessed using the Oswestry Disability Index (ODI), while back and leg discomfort were rated using the Visual Analogue Scale (VAS) for each patient. The measurements comprised the sacral slope (SS), pelvic tilt (PT), pelvic incidence (PI), and Meyerding slip grades. We measured lumbar lordosis (LL), and segmental lordosis.ResultsThe seventy-two patients were 60 female and 12 males. There was no statistically significant difference in the duration of operation between the two groups. In the MIS group, there was a notable reduction in blood loss, higher radiation exposure, and a shorter hospital stay (P < 0.001). The back VAS showed more favorable outcomes in the MIS-TLIF, while the leg VAS showed better results in the Open-TLIF in the early outcome. Both procedures significantly reduced leg and back pain VAS scores and ODI, with no notable difference between the two groups at the final follow-up. Post-surgery, the pelvic incidence (PI) and lumbar lordosis (LL) matched properly in all patients, showing a rise in LL and sacral slope along with a decrease in pelvic tilt.ConclusionBoth open-TLIF and MIS-TLIF are effective methods for correcting spino-pelvic parameters and improving HRQoL in patients with low-grade isthmic spondylolisthesis. The rapid improvement in back pain experienced by these patients favored the use of MIS-TLIF. However, the cost-effectiveness of this approach must be carefully evaluated.

Health-related quality of life with gilteritinib vs placebo posttransplant for FLT3-ITD+ acute myeloid leukemia

AbstractThe Blood and Marrow Transplant (BMT) Clinical Trials Network conducted a phase 3 randomized trial comparing gilteritinib with placebo after allogeneic hematopoietic cell transplantation (HCT) for FLT3-ITD+ acute myeloid leukemia (AML). The primary analysis demonstrated no statistically significant difference in relapse-free survival (RFS); however, patients with FLT3-ITD measurable residual disease (MRD) peri-HCT had significantly longer RFS with gilteritinib. This analysis investigates the effect of post-HCT gilteritinib vs placebo on health-related quality of life (HRQOL). HRQOL was measured with Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT), FACT-Leukemia (FACT-Leu), and EuroQOL-5 Dimensions (EQ-5D-5L) at post-HCT randomization; day 29; months 3, 6, 12, 18, 24; and/or end of therapy. HRQOL and clinically meaningful differences were summarized using descriptive statistics and compared using mixed model repeated measures to evaluate longitudinal change from baseline and stratified Cox model to evaluate time to improvement. HRQOL completion rate was acceptable (>70%) across all time points and measures. There were no differences in HRQOL scores at any time point between cohorts. Clinically meaningful and time to improvement in HRQOL were similar in both arms. Despite higher treatment-emergent adverse effects with gilteritinib, response to the question of being “bothered by side effects of treatment” did not differ between groups. Subgroup analysis of MRD-positive and negative patients demonstrated no differences in HRQOL between arms. For patients with FLT3-ITD+ AML undergoing HCT, gilteritinib maintenance was not associated with any difference in HRQOL or patient-reported impact of side effects. This trial was registered at www.ClinicalTrials.gov as #NCT02997202.

Health-related quality of life is an independent predictor of mortality and hospitalisations in transthyretin amyloid cardiomyopathy: a prospective cohort study

PurposeTransthyretin amyloid cardiomyopathy (ATTR-CM) is associated with severely impaired health-related quality of life (HRQL). HRQL is an independent predictor of outcome in heart failure (HF), but data on patients with ATTR-CM is scarce. This study therefore aims to evaluate the association of HRQL with outcome in ATTR-CM.MethodsPatients from our prospective ATTR-CM registry were assessed using the Kansas City cardiomyopathy questionnaire (KCCQ), the Minnesota living with HF questionnaire (MLHFQ), and the EuroQol five dimensions questionnaire (EQ-5D). Cox regression analysis was utilised to assess the impact of HRQL on all-cause mortality.Results167 patients [80 years; interquartile range (IQR): 76–84; 80.8% male] were followed for a median of 27.6 (IQR: 9.7–41.8) months. The primary endpoint of all-cause mortality was met by 43 (25.7%) patients after a median period of 16.2 (IQR: 9.1–28.1) months. In a univariate Cox regression for mortality, a 10-point change in the KCCQ implied a hazard ratio (HR) of 0.815 [95%-confidence interval (CI): 0.725–0.916; p = 0.001], in the EQ-5D VAS of 0.764 (95%-CI: 0.656–0.889; p < 0.001), and 1.163 (95%-CI: 1.114–1.433; p < 0.001) in the MLHFQ. After adjustment for established biomarkers of HF, all-cause mortality was predicted independently by the EQ-5D VAS (HR: 0.8; 95%-CI: 0.649–0.986; p = 0.037; per 10 points) and the MLHFQ (HR: 1.228; 95%-CI: 1.035–1.458; p = 0.019; per 10 points).ConclusionHRQL is a predictor of outcome in ATTR-CM. The EQ-5D VAS and the MLHFQ predict survival independent of biomarkers of HF.

Development and psychometric properties of the Functional Assessment of Cancer Therapy-Cutaneous T-Cell Lymphoma (FACT-CTCL).

Patients with Mycosis Fungoides (MF)/Sézary Syndrome (SS) can experience impacted health-related quality of life (HRQoL). To validate the CTCL-S, a novel subscale of the Functional Assessment of Cancer Therapy - General (FACT-G), in patients with MF/SS. Qualitative interviews were conducted with expert clinicians and MF/SS patients. Thematic analysis identified the most common concerns, and 19 items were selected.MF/SS patients were recruited from a single center. FACT-G, CTCL-S (collectively "FACT-CTCL"), Skindex29, and Visual Analogue Scale-Pruritis (VAS itch) were administered. A subset repeated FACT-CTCL and VAS itch after ≈2 weeks. Patient demographics and clinical characteristics were obtained via review of the electronic medical record.Psychometric properties were assessed. Internal consistency was estimated using Cronbach's alpha (α). Convergent and discriminant validity were assessed by comparing CTCL-S to disease stage, age, VAS itch, FACT-G, and SkinDex29. Exploratory factor analysis (EFA) was used to preliminarily assess CTCL-S dimensionality. Test-retest repeatability was summarized using intraclass correlation coefficient (ICC), within-subject standard deviation (wSD), and within-subject coefficient of variation. Seventy-two patients completed the initial survey, and 35 repeated the FACT-CTCL and VAS itch after ≈2 weeks. Two-thirds were male, most were white (78%). The majority (85%) had MF, 15% SS, and 75% early (stage IA-IIA) and 25% advanced (≥ stage IIB) disease. Preliminary EFA found a single predominant factor, supporting a hypothesis of unidimensionality of the CTCL-S. Internal consistency of the CTCL-S was high (α: 0.95 [95% CI: 0.93-0.96]). There was no significant change in CTCL-S average test-retest scores (ICC of 0.93 (p = 0.63)). CTCL-S was significantly lower in advanced vs early stage disease (median[IQR]: 34[26, 48] vs. 59[44, 68], p < 0.001) and strongly correlated with VAS itch (Spearman's r (rs): -0.70, 95% CI: -0.81, -0.55), FACT-G (rs: 0.77, 95% CI: 0.65, 0.85), and Skindex29 (rs: -0.90, 95% CI: -0.94, -0.84), supporting convergent validity. CTCL-S scores had little correlation with age (rs: 0.19, 95% CI: -0.05, 0.41, p = 0.12), supporting discriminant validity. The FACT-CTCL is a disease specific instrument for assessing HRQoL with high reproducibility and good performance in a cohort of patients with MF/SS.

Stereotactic Body Proton Therapy Versus Conventionally Fractionated Proton Therapy for Early Prostate Cancer: A Randomized, Controlled, Phase 3 Trial

PurposeWe aimed to determine if ultrahypofractionated proton therapy delivered via stereotactic body proton therapy (SBPT) is noninferior to conventionally fractionated proton therapy (CFPT) in patients with early prostate cancer. Methods and MaterialsThis study was a multicenter, randomized, controlled, noninferiority phase 3 trial that included patients with histologically confirmed low-risk prostate adenocarcinoma defined by Gleason score grouping 1, Prostate-specific antigen <10 ng/mL, and clinical stage T1-T2a N0 M0 according to 7th edition of the American Joint Committee on Cancer tumor-node-metastasis cancer staging system. Eligible participants were randomly assigned initially at a 1:1 ratio and later at a 2:1 ratio to SBPT (38 Gy in 5 fractions) or CFPT (79.2 Gy in 44 fractions). The primary endpoint was freedom from failure (FFF) at 2 years from the date of randomization. Noninferiority for FFF was determined based on 1-sided confidence intervals. Toxicities were compared at different time points using Fisher's exact test. Health-related quality-of-life (HRQoL) was analyzed at different time points using a mixed-effects linear model. This trial is registered with ClinicalTrials.gov, NCT01230866, and is closed to accrual. ResultsBetween December 10, 2010, and September 29, 2020, 144 patients were enrolled and 135 were randomly assigned (90 to the SBPT group and 45 to the CFPT group). The median follow-up was 5 years (IQR, 3.9-5.2). The 2-year FFF was 100% for both groups, with the 1-sided 5-year risk difference in FFF between groups reported as 2.63% (90% CI, −1.70% to 6.96%), favoring the SBRT arm, thus fulfilling the prespecified criteria for noninferiority of SBPT compared with CFPT. Rates of gastrointestinal and genitourinary G2 and G3 toxicities did not differ significantly between groups. Further, HRQoL metrics did not differ significantly between groups over the study's median follow-up. ConclusionsSBPT is noninferior to CFPT regarding FFF, with similar long-term genitourinary and gastrointestinal toxicity rates and minimal impact in patient-reported HRQoL over time.

The role of symptom reduction in improving health-related quality of life through brief cognitive behavioral therapy

Objective Brief cognitive behavior therapy (bCBT) is effective in reducing symptoms of depression and anxiety disorders and improving health-related quality of life (HRQoL). However, the mechanisms through which cognitive behavior therapy impact HRQoL are not well understood. This study evaluated whether anxiety and depression symptom reduction is a mechanism of treatment for HRQoL outcomes. Method: Using secondary data from a multisite, pragmatic, randomized trial, this study evaluated bCBT vs enhanced usual care in 16 VA community-based outpatient clinics. Ordinary least-squares path analysis testing multiple mediators was used to evaluate the role of change in depression and anxiety symptoms in the relationship between treatment condition and HRQoL. Results: Receiving bCBT (vs. enhanced usual care) was significantly negatively associated with change (reduction) in depression and anxiety scores. The indirect effect of treatment on mental HRQoL was significant with change in depression scores as mediator. A similar pattern was observed for physical HRQoL and change in anxiety scores as mediator. Conclusion: Findings suggest reduction of depression and anxiety symptoms as a mechanism through which bCBT for depression promoted improvements in HRQoL, with important implications for understanding how CBT impacts functioning, as well as the utility of bCBT in nontraditional mental health settings. Trial registration: ClinicalTrials.gov identifier: NCT02466126.

Health-related quality of life in children with childhood acute myeloid leukemia in China: A five-year prospective study

PurposeThis study aims to identify the key factors influencing health-related quality of life (HRQoL) of pediatric acute myeloid leukemia (AML) patients following their initial diagnosis and examine their impact on the five-year survival prognosis. MethodsA chart review and follow-up were conducted for children with AML who participated in a prospective cohort study between 2017 and 2020. We identified factors influencing HRQoL through Pediatric Quality of Life Inventory™ (PedsQL™ 4.0), PedsQL™ Cancer Module 3.0 (CM 3.0) and PedsQL™ Family Impact Module 2.0 (FIM 2.0), as well as assessed the impact of impaired HRQoL on the overall outcomes of patients. ResultsSixty-four subjects enrolled in the study had complete HRQoL outcome data, and 61 of them completed the 5-year follow-up. In CM 3.0, age was positively associated with parental proxy reports (p = 0.040), whereas divorced families were negatively associated with child self-reports (p = 0.045). A positive medical history correlates with FIM 2.0 (p = 0.025). Residence (p = 0.046), the occupation of caregivers (p = 0.014), disease severity (p = 0.024), and the only child (p = 0.029) exhibited statistically significant associations with the impairment of HRQoL. Impaired HRQoL scores shown by the PedsQL™4.0 parent proxy report (p = 0.013) and FIM 2.0 (p = 0.011) were associated with a reduced 5-year survival rate. ConclusionsThis study demonstrated that early impairment of HRQoL in pediatric acute myeloid leukemia patients has predictive value for long-term prognosis. Once validated, these findings may provide some guidance to clinicians treating children with AML.

#2155 Evaluating the health-related quality of life of patients undergoing haemodialysis: highlights from a multicentric nationwide study

Abstract Background and Aims Haemodialysis (HD) represents the most common renal replacement therapy for end-stage renal disease worldwide. Despite recent advances in HD the health-related quality of life (HRQoL) should be monitored in conjunction with clinical variables to optimize a comprehensive treatment. Method From June to July 2022, all patients from 47 clinics were included. Among other key relevant clinical variables, HRQoL was assessed by using the Kidney Disease Quality of Life Short Form (KDQOL-SF™ 1.3).It`s generic part is summarised in the physical component summary (PCS) and mental component summary (MCS). The specific part evaluates 11 domains: symptom/problem list; effects of kidney disease on daily life; burden of kidney disease; work status; cognitive function; quality of social interactions; sexual function; sleep; social support; dialysis staff encouragement; and patient satisfaction. All scores range from 0 (worst) to 100 (best). To ensure comparability of results, mean ± SD values are outlined, even though non-parametric tests were used to test associations. Results A total of 3,595 patients (67.3% male) completed the KDQOL (out of 4259 HD patients treated during study period). Most of them (67.18%) were over 60yo of age, and 65.67% had a severe comorbidity index score. In addition, 50.54% had a native AV fistula and only 3.92% were incident HD. PCS scores reported were higher in over 75yo (47.68 ± 10.63) rather than normative population (40.16 ± 11.62), indicating an overall good physical function. However, MCS values (33.17 ± 9.36) were indicative of substantial impact. Gender differences were noted across almost all disease-specific and generic domains (all p &amp;lt; 0.05). Severe comorbidity scores were associated with lower scores in certain renal specific domains and MCS. Finally, incident HD patients reported lower satisfaction and poorer perceived overall health (p-values = 0.001). Conclusion Although PCS scores reported were reasonable, a remarkable impact on mental health was found. Moreover, a differential impact of patient's HRQoL was captured based on gender and other variables highlighting the need of individualised evaluation and management of HD patients.

Impact of outpatient adaptation to home mechanical ventilation on health-related quality of life in patients with COPD: the OutVent study.

Home mechanical ventilation (HMV) is indicated in patients with severe hypercapnic COPD. Initiation of HMV commonly occurs during an inpatient period, but there has been increasing interest for outpatient adaptation. This study aimed to evaluate the outpatient initiation and adaptation of HMV and its impact on health-related quality of life (HRQoL) in patients with severe COPD. A single-group pre-test-post-test study was conducted in an outpatient ventilation clinic of a tertiary hospital in Portugal. Patients with severe COPD and symptoms of chronic respiratory failure with daytime partial pressure of carbon dioxide (P CO2 ) ≥50 mmHg in a stable condition or with persistent hypercapnia ≥53 mmHg >14 days following an exacerbation with mechanical ventilation were included. After 3 months of HMV, patients completed the severe respiratory insufficiency (SRI), the S3-noninvasive ventilation (S3-NIV) and a patient experience questionnaire. 53 patients (73.6% male, median 71 (p25-p75 61-77) years), with a median forced expiratory volume in 1 s of 35 (29-40)% and a median baseline P CO2 of 53.5 (51.9-56.5) mmHg completed the study. At 3 months patients had a median HMV usage of 6.5 h and decreased their P CO2 by 6.0 mmHg. After 3 months, there was a significant improvement in the SRI summary scale (+5.7), above the minimal clinically import difference of five. Patients who used HMV for more than 5 h had higher S3-NIV total score (6.8 versus 5.7, p=0.04) and S3-NIV sleep and NIV-related side effects subscore (7.1 versus 5.7, p=0.03). Our findings might indicate that outpatient initiation and adaptation of HMV has a positive impact in short-term HRQoL in patients with COPD and that this approach is perceived as a positive experience by the patients.

A qualitative study to explore the burden of disease in activated phosphoinositide 3-kinase delta syndrome (APDS).

Activated phosphoinositide 3-kinase delta syndrome (APDS) is an ultra-rare primary immunodeficiency, with only 256 cases reported globally. This study aimed to explore the disease burden of APDS from the perspective of individuals with APDS and their caregivers. Qualitative interviews were conducted with healthcare providers (HCPs), individuals with APDS and caregivers, to explore the symptoms and health-related quality of life (HRQoL) impact of APDS. Some individuals and caregivers also completed a narrative account exercise. All interviews were audio recorded and transcribed. Data were analysed using thematic analysis and saturation was recorded. Semi-structured qualitative interviews were conducted with healthcare providers (HCPs), individuals with APDS and caregivers. Individuals and caregivers had the option of completing a narrative account exercise. Six HCPs participated in an interview. Seven participants completed the narrative account exercise (N = 5 caregivers and N = 2 individuals with APDS) and 12 took part in an interview (N = 4 caregivers and N = 8 individuals with APDS). Themes identified from HCPs interviews included symptoms, clinical manifestations, HRQoL impacts and treatments/management of APDS. The narrative account exercise identified similar themes, but with the addition to the journey to diagnosis. These themes were explored during the individual/caregiver interviews. Reported clinical manifestations and symptoms of APDS included susceptibility to infections, lymphoproliferation, gastrointestinal (GI) disorders, fatigue, bodily pain, and breathing difficulties. HRQoL impacts of living with APDS included negative impacts to daily activities, including work, education and social and leisure activities, physical functioning, as well as emotional well-being, such as concern for the future, and interpersonal relationships. Impacts to caregiver HRQoL included negative impacts to physical health, work, emotional well-being, interpersonal relationships and family life and holidays. The management of APDS included the use of healthcare services and medications including immunoglobulin replacement therapy (IRT), rapamycin, prophylactic antibiotics, leniolisib, as well as medical procedures due to complications. APDS has a high disease burden and there is an unmet need for licensed, more targeted treatments which modify disease progression. This study was the first to describe the day-to-day experience and HRQoL impact of APDS from the perspective of individuals living with the condition, caregivers and treating physicians.

Empirical Development of a Behavioral Intervention for African American/Black and Latino Persons with Unsuppressed HIV Viral Load Levels: An Application of the Multiphase Optimization Strategy (MOST) Using Cost-Effectiveness as an Optimization Objective.

We used results from an optimization randomized controlled trial which tested five behavioral intervention components to support HIV antiretroviral adherence/HIV viral suppression, grounded in the multiphase optimization strategy and using a fractional factorial design to identify intervention components with cost-effectiveness sufficiently favorable for scalability. Results were incorporated into a validated HIV computer simulation to simulate longer-term effects of combinations of components on health and costs. We simulated the 32 corresponding long-term trajectories for viral load suppression, health related quality of life (HRQoL), and costs. The components were designed to be culturally and structurally salient. They were: motivational interviewing counseling sessions (MI), pre-adherence skill building (SB), peer mentorship (PM), focused support groups (SG), and patient navigation (short version [NS], long version [NL]. All participants also received health education on HIV treatment. We examined four scenarios: one-time intervention with and without discounting and continuous interventions with and without discounting. In all four scenarios, interventions that comprise or include SB and NL (and including health education) were cost effective (< $100,000/quality-adjusted life year). Further, with consideration of HRQoL impact, maximal intervention became cost-effective enough to be scalable. Thus, a fractional factorial experiment coupled with cost-effectiveness analysis is a promising approach to optimize multi-component interventions for scalability. The present study can guide service planning efforts for HIV care settings and health departments.

Development of Patient and Caregiver Conceptual Models Investigating the Health-Related Quality of Life Impacts of Malignant Pleural Mesothelioma.

Malignant pleural mesothelioma (MPM) is a rare and usually fatal malignancy frequently linked to occupational asbestos exposures and associated with poor prognosis and considerable humanistic burden. The study aimed to develop conceptual models of the health-related quality of life (HRQoL) impact on patients with and receiving treatment for MPM, and the burden on their caregivers. This multi-country study (Australia and United Kingdom) adopted a qualitative methodology to conduct semi-structured, independent interviews with people with MPM (n = 26), current caregivers (n = 20), and caregivers of people who had recently died because of MPM (n = 4). Participants were recruited using a purposive sampling approach and interviews conducted via telephone between January 2021 and January 2022. Transcripts were analysed using thematic analysis and used to construct conceptual models. Patient analysis yielded four overarching themes: (1) debilitating burden of breathlessness and fatigue; (2) physical mesothelioma symptoms experienced by patients; (3) distress of MPM on the self and family; and (4) treatment is worth 'having a go' despite the potential impact on symptoms. Caregiver analysis yielded five core themes: (1) daily life limited by caregiving duties; (2) emotional well-being and the need for support; (3) the relational role shift to caregiver; (4) time spent providing care negatively impacts work and productivity; and (5) positive aspects and outcomes of caregiving. This study highlights the substantial daily and emotional HRQoL impact that MPM symptoms have on patients and caregivers. Both groups reduced work, productivity, and social and leisure activities. There was evidence of positive HRQoL impacts as a result of immunotherapy and radiotherapy, but less for chemotherapy. Caregiver impacts were intensified during the end-of-life period and persisted following patient death. Evident is a need for increased psychological support, information, and advice for caregivers, increased during the end-of-life period.

A quantitative cross-sectional study of the burden of caring for patients with Lennox-Gastaut syndrome, Dravet syndrome, and tuberous sclerosis complex-associated epilepsy in Japan

IntroductionLennox-Gastaut syndrome (LGS), Dravet syndrome (DS), and tuberous sclerosis complex (TSC)-associated epilepsy are rare conditions associated with severe childhood-onset epilepsy. Caregivers play a critical role in the patients’ care and may experience significant psychosocial and socioeconomic burden. This cross-sectional study determined the burden of caring for patients with these rare epilepsy conditions in Japan. MethodsA quantitative online survey was used to assess patients’ and caregivers’ characteristics and the caregivers’ emotional state, among others. Several validated questionnaires were used: the Hospital Anxiety and Depression Scale (HADS; 0–21 score) assessed the caregivers’ emotional wellbeing, the Pediatric Quality of Life Inventory Family Impact Module (PedsQL FIM; 0–100 score) assessed the health-related quality of life (HRQoL) of the caregivers and their families, and the Work Productivity and Activity Impairment General Health (WPAI:GH; 0–100 % score) questionnaire assessed work productivity. ResultsA total of 36 caregivers responded (median [interquartile range (IQR)] age 43.5 [39.5, 48.3] years; 33/36 [92 %] female; 13/36 [36 %] working part-time and 13/36 [36 %] not working). Participants cared for 7/36 (19 %), 19/36 (53 %), and 10/36 (28 %) patients with LGS, DS, and TSC, respectively (median [IQR] age, 11.0 [6.8, 16.3] years; age at first seizure, 0 [0, 0] years). Patients received a median (IQR) of 4 (3, 5) treatment drug types. Patients experienced median (IQR) 3.0 (0, 21.0) epileptic seizures in the previous week; 28/36 (78 %) had severe intellectual disabilities, and 34/36 (94 %) had developmental delays. Caregivers reported stress (17/36 [47 %]), sleep problems (13/36 [36 %]), and anxiety (12/36 [33 %]). They spent a median (IQR) of 50.0 (17.5, 70.0) hours caregiving in the previous week, with 3.0 (1.0, 11.0) hours of seizure-specific care. Caregivers reported that their lives would be easier with a median (IQR) of 1.5 (0, 5.0) hours fewer per week caring for patients during/following seizures. Median HADS scores were 9.5 (‘suspected anxiety diagnosis’) and 7.5 (‘no depression’) for caregivers, and PedsQL FIM Total median score was 60.1, indicating HRQoL impairment for the caregiver and their family. WPAI:GH scores for paid workers indicated important work impairment. Higher caregiving hours (≥ 21 h vs. < 21 h in the previous week) resulted in higher caregiver burden as indicated by the HADS Total score (p = 0.0062) and PedsQL FIM Total score (p = 0.0007). ConclusionsCaregivers of patients with LGS, DS, or TSC in Japan experience a significant time burden, reduced HRQoL, and high level of work/activity impairment. Caregivers provide round-the-clock care to patients and rely on family and specialized caring services to help manage the increased caregiving time, which tends to be associated with greater emotional burden and HRQoL impact.