Assessment Of Health-related Quality Of Life In Patients Research Articles

International validation of a health-related quality-of-life questionnaire for Hodgkin lymphoma: the EORTC QLQ-HL27

AbstractHodgkin lymphoma (HL) has become 1 of the most curable cancers. Therefore, rigorous assessment of health-related quality of life (HRQoL) and symptom burden of these patients is essential to support informed clinical decisions. The European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Group previously developed the EORTC Quality of Life Questionnaire (QLQ) Hodgkin Lymphoma 27. This paper reports the final results of an international study by the EORTC group to develop a HRQoL disease-specific measure for these patients: the EORTC QLQ-HL27. Patients with a confirmed diagnosis of HL (N = 381) were enrolled from 12 countries and completed the EORTC QLQ-C30, QLQ-HL27, and a debriefing questionnaire at baseline (any time after diagnosis). A subset completed a retest (n = 126) or responsiveness-to-change analyses (RCA) second measurement (n = 98). Psychometrics were evaluated. Confirmatory factor analysis showed an acceptable fit of the 27 items of the QLQ-HL27 on its 4 scales (symptom burden, physical condition/fatigue, emotional impact, and worries about health/functioning). Test–retest reliability, convergent validity, known-group comparisons, and RCA find satisfactory results. Symptom burden and fatigue was higher among patients on treatment (with 36%-83% reporting at least a few problems) compared with those who had completed treatment (19%-61% reporting at least a few problems). Prevalence of worries about health and functioning (reporting at least some worry) was similar for patients on treatment (51%-81%) vs those who had completed treatment (52%-78%). Implementation of the EORTC QLQ-HL27 in research and clinical applications will increase sensitivity of HRQoL assessment in patients with HL. High quality data generated through use of this questionnaire are expected to facilitate clinical decision making in the HL setting.

Health-related quality of life in children and adolescents with pleuropulmonary blastoma: A report from the International PPB/DICER1 Registry.

Pleuropulmonary blastoma (PPB) is the most common lung cancer of infancy and early childhood and is associated with germline DICER1 variants. Type I and Ir PPB are cystic lesions treated surgically, with a subset of children with type I receiving chemotherapy. Type II and III are more aggressive lesions, treated with surgery, intensive chemotherapy and potentially radiation. We sought to assess health-related quality of life (HRQoL) in children with PPB and known germline DICER1 variants. Children with a diagnosis of PPB or germline DICER1 pathogenic variant without history of PPB or other DICER1-related neoplasm (DICER1+ only) were enrolled in the International PPB/DICER1 Registry. Parent reports for participants aged 2-17years for the PedsQL v.4 and PedsQL Multidimensional Fatigue Scale v.3 were collected. Fatigue, physical, and psychosocial function scores were compared. Analysis included 84 participants (PPB type Ir=20, type I=15, type II/III=27, DICER1+ only=22). Total fatigue scores of participants with type I and II/III PPB were lower compared to DICER1+ only, with effect size larger in type II/III (-0.82 vs. -0.40). Total psychosocial and physical functioning scores were lower in participants with type I and type II/III PPB compared to DICER1+ only, with larger effects noted in type II/III. Female sex was suggestive of worse HRQoL for both type I/Ir and type II/III cohorts. These data demonstrate the importance of regular HRQoL assessment in patients with a history of PPB as well as the importance and feasibility of studying HRQoL in children with rare tumors.

International validation of the EORTC QLQ-CLL17 questionnaire for assessment of health-related quality of life for patients with chronic lymphocytic leukaemia.

Selecting the most appropriate chronic lymphocytic leukaemia (CLL) treatment is challenging. Patient-reported health-related quality of life (HRQoL) is therefore a critical aspect to consider. This international study by the European Organization for Research and Treatment of Cancer (EORTC) tested the psychometric properties of a newly developed measure for CLL patients: the EORTC QLQ-CLL17 to supplement the core questionnaire (EORTC QLQ-C30). Patients with CLL (n=341) from 12 countries completed the QLQ-C30, QLQ-CLL17 and a debriefing questionnaire. Sociodemographic and clinical data were recorded from medical records. A high percentage (30%-66%) reported symptoms and/or worries (e.g. aches/pains in muscles, lack of energy and worry/fears about health). Confirmatory factor analysis showed an acceptable to good fit of the 17 items on the three scales (i.e. symptom burden, physical condition/fatigue and worries/fears about health and functioning). Completion took on average 8min. Test-retest and convergent validity was demonstrated. The QLQ-CLL17 differentiated between patients with an Eastern Cooperative Oncology group (ECOG) performance of 0 versus 1-3 (p's<0.01 and clinically relevant). The newly developed EORTC QLQ-CLL17 will increase sensitivity of HRQoL assessment in patients with CLL. Implementation of this questionnaire both in clinical research and practice will help to generate unique clinically relevant data to better inform CLL treatment decision-making.

EuroQOL-5D-3L does not adequately map quality-of-life deterioration in severely affected patients with epilepsy

IntroductionThe steadily increasing impact of health-related quality of life (HRQOL) on reasonable diagnostic and therapeutic decisions makes the correct mapping of HRQOL indispensable in modern epileptology. The aim of this study was to address the reliability of the often-used generic HRQOL screening questionnaire EuroQOL 5-dimension, 3-level (EQ-5D-3L) by comparing its normalized index value (calculated via the time trade-off method) and visual analog scale (VAS) to the gold standard of the extensive Quality of Life in Epilepsy Inventory (QOLIE-31). QOLIE-10 scores were compared with the extensive QOLIE-31 and EQ-5D-3L TTO. MethodsWe conducted a retrospective analysis of a monocentric study of 184 patients with epilepsy. Bivariate Spearman correlation analysis and Fisher’s r-to-z transformation were used to compare the strengths of correlations of EQ-5D-3L, QOLIE-10 and QOLIE-31 with different epilepsy-specific domains (disease severity, drug interactions, emotional well-being, stigmatization, seizure-related anxiety, cognitive impairment). ResultsThe different metrics of EQ-5D-3L, QOLIE-10 and QOLIE-31 showed moderate to very strong intra- and inter-metric correlations for overall HRQOL. Quality of Life in Epilepsy Inventory-31 VAS and EQ-5D-3L VAS did not show any significantly different strengths of correlations with respect to the domains studied. In contrast, the correlation strength of the normalized EQ-5D-3L index value differed significantly from the QOLIE-31 T-score for several domains, for example, for drug-related adverse events, neuropsychological deficits, symptoms of depression and seizure worry. In seizure-free patients, EQ-5D-3L VAS and EQ-5D-3L index values correlated significantly less with the domain of “cognitive impairment” than the QOLIE-31 T-score. In patients without relevant neuropsychological deficits, the strengths of correlations with the assessed domains did not differ significantly between EQ-5D-3L metrics and the QOLIE-31 T-score. The HRQOL mapping probability of QOLIE-10 was inferior to QOLIE-31 and comparable to EQ-5D-3L regarding the analyzed domains. ConclusionIn contrast to the EQ-5D-3L VAS, EQ-5D-3L index values do not adequately map health-related quality of life in severely affected patients with epilepsy and therefore should not be used as screening tools. The QOLIE-31 T-score remains the gold standard for HRQOL assessment in patients with epilepsy.

Validation of the QLQ-CX24 questionnaire for the assessment of quality of life in Mexican women with cervical cancer

ObjectiveCervical cancer is the fourth most frequent neoplasm among women in terms of incidence and mortality. Health-related quality of life (HRQL) is an important outcome in oncology. The QLQ-CX24 instrument...

Cross-Sectional Survey of Symptoms and Health-Related Quality of Life of Adults with De Novo Acute Myeloid Leukemia (AML) in Clinical Practice

Introduction: The care of adults with acute myeloid leukemia (AML) is complex, involving repeated courses of chemotherapy; many patients suffer from sequelae of cytopenias and treatment-related toxicities that impair their health-related quality of life (HRQoL). Because knowledge regarding symptom burden and HRQoL is largely derived from academic centers' experience, there are uncertainties regarding how this compares to the real-world experience.Methods: The Adelphi AML Disease-Specific Program (DSP), a real-world, cross-sectional survey, was conducted in the US between February-May 2015; 61 hematologist/oncologists completed patient record forms for the next 6-8 adult patients with primary (de novo) AML with whom they consulted. Physicians reported demographics, clinical background, and treatment history for each patient. Patients were invited to complete the Functional Assessment of Cancer Therapy Leukemia (FACT-Leu) and EuroQoL 5 Dimensions (EQ-5D) questionnaires, which assess patient HRQoL and functional health status, respectively. Patients were categorized as newly diagnosed (NEW: currently on 1st line AML treatment), relapsed/refractory (RR: currently on 2nd line or later AML treatment), or not currently receiving active AML treatment (NO). Analysis of variance was used for quantitative outcomes, while chi-square and Fisher's exact tests were used for categorical outcomes, to determine statistical differences between patient groups. Effect sizes were calculated using Cohen's d. Minimally important differences (MIDs), required to suggest clinical importance on FACT-Leu and EQ-5D, were also examined.Results: Physician-completed data on 415 patients (median age: 61 years, 55% male) were obtained (NEW: 82%; RR: 12%; NO: 6%). At the time of most recent consultation, 46% of patients were currently in full or part-time employment, 31% required some caregiver support and 27% were considered asymptomatic. Among those who experienced symptoms, fatigue (24%), bruising (10%), loss of appetite (10%), and nausea (9%) were most common.When comparing subgroups, 58% of NO patients and 66% of RR patients were still in full or part-time employment compared with 42% of NEW patients; however, RR and NO patients were more likely than NEW patients to require some degree of caregiver support (RR: 48%, NO: 50%, NEW: 27%). The current symptom most commonly reported was fatigue (23% NEW, 32% RR, and 15% NO).Seventy-five patients (median age: 60 years, 53% male) provided self-reported information (68% NEW, 23% RR, and 9% NO). Results from the FACT-Leu indicated that physical well-being (PWB), leukemia-related well-being (LRW), well-being as measured by the Trial Outcome Index (TOI), and FACT-Leu Total Score were significantly affected by stage of treatment (PWB: p<0.001, LRW: p=0.002, TOI: p=0.011; Total: p=0.035), with NO patients consistently scoring lowest in all domains. Large differences in mean domain scores, exceeding the MIDs, were observed across the patient subgroups for the FACT-Leu. Large effect sizes were seen between NEW and NO patients for PWB (d=1.27 standard deviation units), LRW (d=1.35), and the TOI (d=1.02). Between RR and NO patients, large effect sizes were seen for functional well-being (FWB: d=0.80), and LRW (d=0.95). Between NEW and RR patients, a large effect was seen for PWB (d=0.83) and a moderate effect for TOI (d=0.54). In general, differences between NEW and RR patients were small to moderate, suggesting HRQoL was similar for the two patient populations, and differences between the NEW and NO group were moderate to large.Results from the EQ5D did not show a significant difference in the utility score between NEW (M=0.74), RR (M=0.73) and NO (M=0.76) patients. The effect sizes for any differences in EQ5D utility score were also small, whereas the difference between NO and RR patients for the EQ5D Visual Analogue Scale was moderate (d=0.57).Conclusions: In this novel real-world analysis of AML patients, differences in symptomatology, level of caregiver involvement, and HRQoL were observed across NEW, RR, and NO patients. Regarding HRQoL, the disease-specific FACT-Leu appears more sensitive than the EQ-5D, indicating that NEW patients overall fare better than RR patients, who fare better than NO patients. These results suggest effective and tolerable treatments that delay progression of the disease are important to conserve patients' HRQoL. DisclosuresMamolo:Pfizer Inc: Employment, Equity Ownership. Cappelleri:Pfizer Inc: Employment, Equity Ownership. Hoang:Pfizer Inc: Employment, Equity Ownership. Kim:Pfizer: Employment, Equity Ownership. Hadfield:Adelphi Real World: Employment. Middleton:Adelphi Real World: Employment. Rider:Adelphi Real World: Employment. Walter:Aptevo Therapeutics: Research Funding; ADC Therapeutics: Research Funding.

Validation of the QLQ-EN24 instrument for the assessment of health-related quality of life for women with endometrial cancer in México.

The impact of disease activity or treatments on health-related quality of life (HRQL) is crucial in Oncology, but adequate instruments for this assessment are scarce. Our aim is to validate the Mexican-Spanish version of the QLQ-EN24 questionnaire to evaluate HRQL in women with endometrial cancer (EC). This is a prospective study of Mexican women with EC, attending a single cancer centre, who responded the QLQ-C30 and QLQ-EN24 instruments; usual psychometric analysis were performed as well as the association of HRQL scales and relevant clinical data. Correlation analysis was performed with the Spearman's method, reliability analysis with the Cronbach's alpha, known-group comparisons with the Kruskal-Wallis test, and survival analysis with the Kaplan-Meier method and Log-rank test. One hundred and eighty-nine women with EC were assessed. Most functional scales reported high values, and most symptom scales, low. Questionnaire compliance rates were high and internal consistency tests demonstrated adequate convergent and divergent validity. Cronbach's α coefficients of the five multi-item scales the QLQ-EN24 instruments were from 0.659 to 0.887. Scales of the QLQ-C30 and QLQ-EN24 instruments distinguished among clinically distinct groups of patients, particularly based on serum albumin levels. The Urological symptoms, Gastrointestinal symptoms, Body image, Pelvic pain and Taste change scales were significantly associated with OS. The Mexican-Spanish version of the QLQ-EN24 questionnaire is reliable and valid for the assessment of HRQL in patients with EC and can be broadly used in multi-national clinical trials. However, conclusions derived from scales evaluating sexual function should be handled carefully.

Validation of the Mexican-Spanish Version of the EORTC QLQ-OV28 Instrument for the Assessment of Quality of Life in Women with Ovarian Cancer

Health-related quality of life (HRQL) is an important outcome measure in Oncology. To validate the Mexican-Spanish version of the QLQ-OV28 questionnaire to assess HRQL in women with ovarian cancer (OC). The QLQ-C30 and QLQ-OV28 instruments were applied to women with OC attending a cancer center in Mexico. The usual psychometric analyses were performed; the Spearman's method was used for correlation analysis, reliability analysis with the Cronbach's alpha, known-group comparisons with the Kruskal-Wallis test, responsiveness was tested employing repeated measures ANOVA, and the association of scale scores and overall survival (OS) were analyzed with the Kaplan-Meier method and Cox's model. Two hundred fifty-two women with OC were included in this cohort. The instruments were well accepted and compliance rates were high; patients responded both instruments in <30min. The QLQ-OV28 internal consistency tests demonstrated good convergent (Correlation coefficients [CC] 0.154‒0.694) and divergent validity (CC 0.003‒0.69). Cronbach's α coefficients of six of eight scales of the QLQ-OV28 instruments were >0.7 (range, 0.567‒0.857). Scales QLQ-OV28 instruments distinguished among clinically distinct groups of patients, particularly after basal serum albumin and basal Ca‒125 levels. The evaluation of responsiveness demonstrated that two scales of the QLQ-OV28 were sensitive to change over time during induction chemotherapy. Six scales of the QLQ-OV28 were associated with OS. The Mexican-Spanish version of the QLQ-OV28 questionnaire is reliable and valid for the assessment of HRQL in patients with OC and can be broadly used in clinical trials.

Cross-cultural adaptation and validation of the RhinAsthma Patient Perspective (RAPP) in the Polish population

IntroductionThe RhinAsthma Patient Perspective (RAPP) was developed in Italian to assess the Health Related Quality of Life (HRQoL) impairment in patients with asthma and allergic rhinitis (AR) in daily practice.AimTo cross-culturally validate the Polish version.Material and methodsThe Polish version was administered to patients suffering from asthma and rhinitis in a prospective observational study. Polish RAPP, along with SF-12, ACT, and a Symptomatologic VAS was filled in twice, with a 4-week interval between visits. At visit 2, a Global Rating Scale (GRS) was completed to assess any change in health status. Internal consistency, validity, reliability, discriminant ability and responsiveness to change as well as Minimal Important Difference were determined.ResultsThe factor and confirmatory analysis revealed a unidimensional structure of RAPP. Internal consistency was satisfactory with Cronbach’s α (visit 1 = 0.85, visit 2 = 0.89). High reliability (ICC = 0.89 and a CCC = 0.94) was found. Validity analyses showed good correlations of the Polish RAPP with Physical and Mental Component Scores of SF-12. In addition, RAPP adequately discriminated patients on the basis of the asthma control level and rhinitis severity (p < 0.03 for all the analyses), and demonstrated to be sensitive to change. MID value was 1 point.ConclusionsThe study confirmed the reliability and validity of the Polish version of RAPP demonstrating that it is a useful tool in the assessment of HRQoL in patients with asthma and comorbid allergic rhinitis, in clinical practice.

Maintenance of Health-Related Quality of Life in the Phase 4 BYOND Study of Bosutinib for Pretreated Chronic Phase Chronic Myeloid Leukemia

Maintenance of Health-Related Quality of Life in the Phase 4 BYOND Study of Bosutinib for Pretreated Chronic Phase Chronic Myeloid Leukemia

A Global Systematic Literature Review on the Burden of Illness in Transfusion-Dependent β-Thalassemia

Introduction: Adults with transfusion-dependent (TD) anemia due to β-thalassemia experience significant side effects and increased mortality due to iron overload resulting from repeated red blood cell (RBC) transfusions. To characterize the burden associated with TD anemia in adults with β-thalassemia, a systematic literature review (SLR) was conducted to gather data on the clinical, health-related quality of life (HRQoL), economic, and epidemiological burdens, as well as treatment patterns for patients with TD anemia due to β-thalassemia. Methods: Systematic searches (covering the period from database inception to April 2019) were executed in MEDLINE®, Embase®, and the Cochrane Library for observational studies on the burden of illness in adult patients with TD anemia associated with β-thalassemia. Conference proceedings from the last 2 years were searched to identify recent data not yet published in full text. References were screened at the abstract and full-text levels by 2 independent reviewers, with methodology following Cochrane guidelines. Results: Searches of electronic databases yielded 1,252 citations. After removing duplicates, 874 abstracts were reviewed, with 72 publications progressing to full-text review. Five publications from database searches that met the eligibility criteria were included. An additional 11 references were identified for inclusion from hand searching and conference proceedings, resulting in 16 total references reporting on 13 unique study populations (Table, Figure). Study population sizes ranged from 14 to 1,548 patients, with mean ages varying from 19 to 43 years when reported. Definitions of transfusion dependence were reported in 5 studies; 3 studies defined transfusion dependence as receipt of ≥ 8 RBC transfusions over a 1-year period. Clinical outcomes were rarely reported across studies; 1 study reported survival over time (overall survival [OS] of 96% at 1 year, 86% at 2 years, and 71% at 3 years, with no significant differences by sex). Use of iron chelation therapy (ICT) varied from 63.6% to 100% in 5 studies; 1 study reported that 72% of patients received oral iron chelators, 30% received injectable iron chelators, and 14% received both oral and injectable ICT. Mean total annual cost per person for treating patients with β-thalassemia who received regular RBC transfusions was $128,062, significantly higher than for matched controls ($5,438; P &lt; 0.001). The main cost drivers were ICT (48%) and RBC transfusions (31%). Hospitalizations related to transfusions varied widely within and across studies. Instruments that were used to assess patient HRQoL included the SF-36, BPI Short Form, TranQoL, and FACT-An; results were inconsistent across studies. Most studies indicated that β-thalassemia impacted HRQoL relative to the general population. Relative to normative US data (50), patients with β-thalassemia had lower SF-36 scores in role-physical (47.33), social functioning (46.79), role-emotional (46.78), physical component summary (46.8), and for general health (41.5). Patients were found to experience transfusion-related pain throughout the transfusion cycle, with increased pain associated with higher transfusion frequency. A cross-sectional study found that families of patients with β-thalassemia experienced substantial caregiver burden, including employment and financial problems affecting nearly half of families and a high level of parental anxiety. Epidemiological studies were limited to sparse mortality and comorbidity data and did not present data on the incidence or prevalence of adults with TD anemia in β-thalassemia. Mortality in patients with thalassemia major was 12.8%; primary comorbidities were osteoporosis (16%-100%) followed by cardiac disease (30%), hypogonadism (22%), hypothyroidism (12%) and depression (2%). Conclusions: TD β-thalassemia affects multiple areas of a patient's life and well-being. Although limited data were available for the adult population, studies suggested a significant burden affecting the HRQoL and economic outcomes of patients with TD β-thalassemia in countries around the world. To better understand the burden of illness in TD β-thalassemia, further high-quality observational studies on treatment patterns and epidemiology, as well as those on the clinical, HRQoL, and economic burdens, are needed to close the evidence gaps identified by this SLR. Disclosures Turner: Evidera: Employment; Celgene Corporation: Consultancy. Deshpande:Celgene Corporation: Consultancy; Evidera: Employment. Chitnis:Celgene Corporation: Consultancy; Evidera: Employment. Byrnes:Evidera: Employment. Pelligra:Evidera: Employment; Celgene Corporation: Research Funding. Rines-MacEachern:Evidera: Employment. Morison:Celgene Corporation: Employment. Tang:Celgene Corporation: Employment, Equity Ownership.

HSR19-109: The Healthy Days Measure in a Metastatic Castration-Resistant Prostate Cancer Population Treated With Novel Oral Therapies

Background: Health-related quality of life (HRQoL) is an important patient-reported outcome to assess in patients with prostate cancer (PC). A variety of general and disease-specific measures have been developed, but there is a lack of evidence on performance of the Centers for Disease Control and Prevention (CDC) Healthy Days (HDs) measure in a population with metastatic disease relative to other validated scales. Objective: To report and understand general HRQoL using the HDs instrument in patients with metastatic PC taking novel oral therapies and compare results to validated scales. Methods: Patients currently enrolled in a Medicare Advantage or Prescription Drug plan treated with enzalutamide or abiraterone acetate were eligible for inclusion. Patients undertook a telephone-based survey including 2 HRQoL questionnaires (Short-Form 36, version 2 [SF-36] survey and the CDC HDs measures), the Service Satisfaction with Cancer Care (SCA) instrument, and the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue). Higher scores indicated higher HRQoL on all surveys, except the HDs scale, where higher numbers indicated lower HRQoL (ie, unhealthy days). Pearson correlations with SF-36 and HDs measures and SCA and FACIT-Fatigue were assessed. Results: 1,745 patients were eligible to participate; 269 completed the survey and were included in the analysis. The mean (standard deviation) age was 74.4 (±7.3) years and 82% were Caucasian. Patients had lower physical HRQoL and a higher number of physically unhealthy days, compared to mental health impacts on both scales. The mean Physical Component Summary (PCS) and Mental Component Summary (MCS) norm-based scores for the SF-36 were 39.9 (±10.6) and 53.4 (±10.4). The mean number of physically and mentally unhealthy days reported in a 30-day period were 9.2 (±11.3) and 3.9 (±7.7), respectively. The unadjusted correlations between scores were all statistically significant (P&lt;.05). Conclusions: The SF-36 and HDs measures both indicated physical health was impacted more than mental health in these patients. The correlations with both HRQoL measures and the other scales were similar in terms of magnitude and significance. This indicates that the HDs instrument may be a useful measure for assessment of general HRQoL in patients with metastatic PC and provides an understanding of general HRQoL in this population that can be utilized to identify opportunities for enhancing patient-centered care.

The King’s Brief Interstitial Lung Disease (KBILD) questionnaire: an updated minimal clinically important difference

IntroductionThe King’s Brief Interstitial Lung Disease (KBILD) is a 15-item validated health-related quality of life (HRQOL) questionnaire. The method of scoring the KBILD has recently changed to incorporate a logit-scale...

Importance of health-related quality of life and its current assessment tools in patients with systemic lupus erythematosus

The paper presents an overview of studies assessing health-related quality of life (HRQoL) in patients with systemic lupus erythematosus (SLE). It describes the HRQoL concept and prerequisites for its creation. The classification of questionnaires used in patients with SLE is considered in relation to the study objectives. The main key triad (fatigue, pain, and depression), which causes a reduction in HRQoL in SLE patients, is derived on the basis of many studies. The paper provides brief comparative characteristics of eight specific HRQoL questionnaires in SLE patients: Symptom checklist (SSC), SLEQol, LupusQol, L-QoL, LupusPRO, and Lupus Impact Tracker (LIT), LUP-QoL, and SMILEY. Special attention is paid to HRQoL assessment in patients receiving different treatment options: glucocorticoids, cytostatic and anti-B-cell therapy.

Systematic review reveals lack of quality in reporting health-related quality of life in patients with gastroenteropancreatic neuroendocrine tumours.

BackgroundGastroenteropancreatic neuroendocrine tumours (GEP-NET) are often slow-growing and patients may live for years with metastasised disease. Hence, along with increasing overall and progression-free survival, treatments aim at preserving patients’ well-being and health-related quality of life (HRQoL). However, studies on systematic HRQoL assessment in patients with GEP-NET are scarce. Therefore, the purpose of the current review is to systematically evaluate the methodological quality of the identified studies.MethodsA targeted database search was performed in PubMed, EMBASE, and CENTRAL. Data extraction was conducted by two independent researchers according to predefined criteria. For study evaluation, the Minimum Standard Checklist for Evaluating HRQoL Outcomes in Cancer Clinical Trials and the CONSORT Patient-Reported Outcome extension were adapted.ResultsThe database search yielded 48 eligible studies. We found the awareness for the need of HRQoL measurement to be growing and application of cancer-specific instruments gaining acceptance. Overall, studies were too heterogeneous in terms of patient characteristics and treatment interventions to draw clear conclusions for clinical practice. More importantly, a range of methodological shortcomings has been identified which were mainly related to the assessment and statistical analysis, as well as the reporting and interpretation of HRQoL data.ConclusionDespite an increasing interest in HRQoL in GEP-NET patients, there is still a lack of knowledge on this issue. A transfer of HRQoL results into clinical practice is hindered not only by the scarceness of studies, but also by the often limited quality of HRQoL processing and reporting.Electronic supplementary materialThe online version of this article (doi:10.1186/s12955-016-0527-2) contains supplementary material, which is available to authorized users.

Abstract A77: Validation of the EORTC QLQ-OV28 instrument for the assessment of quality of life in women with ovarian carcinoma in Mexico.

Abstract Objective: Health-related quality of life (HRQL) is an important outcome measurement in Oncology. Our aim was to validate the Mexican-Spanish version of the European Organization for Research and Treatment of Cancer (EORTC) QLQ-OV28 questionnaire to assess HRQL in patients with Ovarian cancer (OC). Patients and Methods: The QLQ-C30 and QLQ-OV28 instruments were applied to Mexican patients with OC at a cancer referral centre. Reliability and validity tests were performed, and test-retest analysis was carried out in a subgroup of patients. Results: Two hundred fifty-two women were included in this cohort. Questionnaire compliance rates were high and the instrument was well accepted; internal consistency tests demonstrated good convergent and divergent validity. Cronbach's α; coefficients of 8 of 9 multi-item scales of the QLQ-C30 and of 6 of 8 scales of the QLQ-OV28 instruments were &amp;gt;0.7 (range, 0.22 to 0.89). Scales of the QLQ-C30 and QLQ-OV28 instruments distinguished among clinically distinct groups of patients; some were highly sensitive to change over time after the use of induction chemotherapy, and some were associated to overall survival. Conclusion: The Mexican-Spanish version of the QLQ-OV28 questionnaire is reliable and valid for the assessment of HRQL in patients with OC and can be broadly used in clinical trials. Citation Format: Dolores Gallardo-Rincon, Wendy Munoz-Montano, David Cantu de Leon, Jaime Coronel-Martinez, Flavia Morales, Luis F. Onate-Ocana. Validation of the EORTC QLQ-OV28 instrument for the assessment of quality of life in women with ovarian carcinoma in Mexico. [abstract]. In: Proceedings of the AACR Special Conference on Advances in Ovarian Cancer Research: Exploiting Vulnerabilities; Oct 17-20, 2015; Orlando, FL. Philadelphia (PA): AACR; Clin Cancer Res 2016;22(2 Suppl):Abstract nr A77.

Pediatric Urologists' Personal Point-of-view of Health Related Quality of Life

To assess pediatric urologists' understanding and use of health-related quality of life (HRQOL) resources in clinical practice. HRQOL is becoming an increasingly vital part of patient care. Although HRQOL has been evaluated and instruments have been developed for its assessment for several pediatric conditions, it is underrepresented in pediatric urology. A SurveyMonkey 7-item questionnaire was sent to the members of the Society for Pediatric Urology. The questions inquired as to knowledge of surveys available, views on appropriate age for beginning discussion of HRQOL, patients' and providers' ability to assess patient HRQOL, and the need for disease-specific surveys. One-hundred and eighteen providers (37.6%) completed the survey. Remarkably, 85.6% of the responders believe disease-focused HRQOL instruments would be useful for specific urologic diseases; however, only 14.4% of the providers state use of any HRQOL instrument. Only 5 unique measures were reported. The majority felt the age at which patients can start reporting his or her HRQOL is 6-8 (41.3%) or 9-11 (31.2%) years. A preponderance of providers believed both themselves and parents could "somewhat" evaluate the patient HRQOL, but the majority believe it is important to inquire about parents' perceptions of patient HRQOL. The overwhelming majority of pediatric urologists believe HRQOL is important but only a minority utilize the available instruments. Further studies are needed to develop instruments to fill this essential gap.

Cross cultural adaptation of the English version of the IOF-QLQ to Polish, to assess the health-related quality-of-life of patients after a distal radius fracture.

BackgroundA distal radius fracture (DRF) is a common injury that can cause significant pain and lead to a prolonged decrease in physical, emotional, and social functioning. In modern randomized clinical trials, assessing outcomes after a DRF, health-related quality-of-life (HRQoL) is a “must-be” endpoint. Additionally, HRQoL assessments are essential in the clinical decision-making process. The aim of this study to cross-culturally adapt the International Osteoporosis Foundation Quality of Life Questionnaire (IOF QLQ) for patients with a DRF to Polish.MethodsA standard forward-backward translation procedure and pilot-testing were used to prepare the Polish version of the IOF QLQ for use in this case–control study. Patients were eligible if they were between 18–80 years and were within 1–3 days after a non-comminuted DRF. The study group was gender and aged matched with healthy controls. All DRF patients filled out the Polish version of the IOF QLQ, the SF-36 and a demographic questionnaire. Assessment points were set as soon as possible after the fracture, 7 days, 6 weeks, 3, 6, 12, and 18 months after the fracture. Standard validity and reliability analyses were performed.ResultsNinety-seven patients (73 women – 75.3 %) with a mean age of 62.4 ± 7.1 years agreed to take part in the study. The control group consisted of 81 patients (60 women – 74.1 %) with a mean age 63.9 ± 8.2 years. No significant differences were found between the mean age of patients and controls (p = 0.19). Cronbach’s alpha coefficients showed positive internal consistency (0.79–0.89). The interclass correlations for the IOF QLQ domains and the overall score ranged from 0.85 to 0.92. Satisfactory convergent and discriminant validity of the IOF QLQ was seen.ConclusionsThe Polish version of the IOF QLQ for patients with a DRF is a reliable and valid tool for measuring HRQoL. It can be fully recommended for use in clinical settings in the Polish population. When combined with the SF-36 the IOF QLQ allows to obtain a comprehensive HRQoL assessment in patients with a DRF.

Quality of life in multiple myeloma: clinical validation of the Mexican-Spanish version of the QLQ-MY20 instrument.

Health-related quality of life (HRQL) has become an important outcome measurement in hematology. Our aim was to validate the quality of life questionnaire (QLQ)-MY20 instrument in patients with multiple myeloma (MM) in Mexico. The Mexican-Spanish versions of the QLQ-C30 and QLQ-MY20 instruments were applied to patients with MM at a cancer referral centre. Reliability and validity tests were performed. Test-retest was carried out in selected patients. Ninety-eight patients with MM were included in this study. Questionnaire compliance rates were high, and the instrument was well accepted; internal consistency tests demonstrated good convergent and divergent validity. Cronbach's α coefficients of seven of nine multi-item scales of the QLQ-C30 and of all three multi-item scales of the QLQ-MY20 instruments were >0.7 (range, 0.36-0.89). The scales of the QLQ-C30 and QLQ-MY20 instruments distinguished among clinically distinct groups of patients; 9 of 15 scales of the QLQ-C30 and all 4 scales of the QLQ-MY20 presented responsiveness after change over time. The Mexican-Spanish version of the QLQ-MY20 questionnaire is reliable and valid for the assessment of HRQL in patients with MM and can be used in clinical trials in the Mexican community.

Health Related Quality of Life of Bosutinib in Imatinib-Resistant or Imatinib-Intolerant Patients with Advanced Chronic Myeloid Leukemia

Abstract 4448 Purpose:Bosutinib is a dual Src/Abl tyrosine kinase inhibitor (TKI), which has demonstrated efficacy in a phase I/II study of patients with Advanced Phase Chronic Myeloid Leukemia (CML). The objective was to evaluate the effect of bosutinib on health -related quality of life (HRQoL) in patients with advanced CML after failure with imatinib. Methods:Patient reported HRQoL was an exploratory objective in the clinical trial and measured using the 44-item Functional Assessment of Cancer Therapy-Leukemia (FACT-Leu). The FACT-Leu is a modular approach to assess patient HRQoL using a core set of general cancer questions as well as a cancer site specific leukemia subscale with 5 domains: Social Well-being (SWB), Emotional Well-being (EWB), Physical Well-being (PWB), Functional Well-being (FWB) and Leukemia Subscale (LeuS); and 3 summary scales: FACT-General, FACT Trial Outcome Index (TOI) and FACT-Leu Total. The item responses for each scale are summed to provide scores; higher scores indicate better HRQoL. The FACT-Leu was completed at weeks 4, 8, 12 and every 12 weeks thereafter, as well as treatment completion. Within cohort comparisons were assessed using paired t-tests. Results:Of the 164 patients with advanced leukemia included in the trial, 76 had accelerated phase (AP) CML and 64 blast phase (BP) CML. At 24 weeks, AP patients reported statistically significant improvements in PWB (p=0.02), EWB (p<0.001), LeuS (p<0.001), FACT-G (p<0.001), FACT-Leu (p<0.001) and FACT-TOI (p<0.001) with the PWB, FACT-G, LeuS, TOI and FACT-Leu exceeding minimally important differences (MID) at 24 weeks. Blast phase patients reported significant improvements in PWB (p=0.02), EWB (p=0.02), FWB (p=0.04), LeuS (p=0.01), FACT-G (p<0.001), FACT-Leu (p<0.001) and FACT-TOI (p=0.01) at 24-weeks with all scales exceeding MID except the SWB. At 48-weeks the AP patients continued to have statistically significant improvements in PWB (p=0.05), EWB (p=0.02), and FACT-G (p=0.03) and PWB, FACT-G, TOI and FACT-Leu exceeded the MID at 48 weeks. There were no statistically significant deteriorations in HRQoL through week 48 (Figure 1) Conclusions:These data suggest that CML patients treated with bosutinib demonstrate improved HRQoL. Confirmation in a controlled study is needed. [Display omitted] Disclosures:Whiteley:Pfizer Inc: Employment, Equity Ownership. Reisman:Pfizer Inc: Employment, Equity Ownership. Kelly:Pfizer Inc: Employment, Equity Ownership. Cortes:Novartis, Bristol Myers Squibb, Pfizer, Ariad, Chemgenex: Consultancy, Research Funding. Cella:Pfizer Inc: Honoraria, Research Funding.