A. Anota

A longitudinal analysis of patient satisfaction with care and quality of life in ambulatory oncology based on the OUT-PATSAT35 questionnaire

BackgroundIn the oncology setting, there has been increasing interest in evaluating treatment outcomes in terms of quality of life and patient satisfaction. The aim of our study was to investigate the determinants of patient satisfaction, especially the relationship between quality of life and satisfaction with care and their changes over time, in curative treatment of cancer outpatients.MethodsPatients undergoing ambulatory chemotherapy or radiotherapy in two centers in France were invited to complete the OUT-PATSAT35, at the beginning of treatment, at the end of treatment, and three months after treatment. This questionnaire evaluates patients’ perception of doctors and nurses, as well as other aspects of care organization and services. Additionally, for each patient, socio-demographic and clinical characteristics, and self-reported quality of life data (EORTC QLQ-C30) were collected.ResultsOf the 691 patients initially included, 561 answered the assessment at all three time points. By cross-sectional analysis, at the end of the treatment, patients who experienced a deterioration of their global health reported less satisfaction on most scales (pu2009≤u20090.001). Three months after treatment, the same patients had lower satisfaction scores only in the evaluation of doctors (pu2009≤u20090.002). Furthermore, longitudinal analysis showed a significant relationship between a deterioration in global health and a decrease in satisfaction with their doctor and, conversely, between an improvement in global health and an increase in satisfaction on the overall satisfaction scale. Global health at baseline was largely and significantly associated with all satisfaction scores measured at the following assessment time points (pu2009<u20090.0001). Younger age (<55xa0years), radiotherapy (versus chemotherapy) and head and neck cancer (versus other localizations) were clinical factors significantly associated with less satisfaction on most scales evaluating doctors.ConclusionsPre-treatment self-evaluated global health was found to be the major determinant of patient satisfaction with care. The subsequent deterioration of global health, during and after treatment, emphasized the decrease in satisfaction scores, mainly in the evaluation of doctors. Early initiatives aimed at improving the delivery of care in patients with poor health status should lead to improved perception of the quality of care received.

Item response theory and factor analysis as a mean to characterize occurrence of response shift in a longitudinal quality of life study in breast cancer patients

BackgroundThe occurrence of response shift (RS) in longitudinal health-related quality of life (HRQoL) studies, reflecting patient adaptation to disease, has already been demonstrated. Several methods have been developed to detect the three different types of response shift (RS), i.e. recalibration RS, 2) reprioritization RS, and 3) reconceptualization RS. We investigated two complementary methods that characterize the occurrence of RS: factor analysis, comprising Principal Component Analysis (PCA) and Multiple Correspondence Analysis (MCA), and a method of Item Response Theory (IRT).MethodsBreast cancer patients (nu2009=u2009381) completed the EORTC QLQ-C30 and EORTC QLQ-BR23 questionnaires at baseline, immediately following surgery, and three and six months after surgery, according to the “then-test/post-test” design. Recalibration was explored using MCA and a model of IRT, called the Linear Logistic Model with Relaxed Assumptions (LLRA) using the then-test method. Principal Component Analysis (PCA) was used to explore reconceptualization and reprioritization.ResultsMCA highlighted the main profiles of recalibration: patients with high HRQoL level report a slightly worse HRQoL level retrospectively and vice versa. The LLRA model indicated a downward or upward recalibration for each dimension. At six months, the recalibration effect was statistically significant for 11/22 dimensions of the QLQ-C30 and BR23 according to the LLRA model (pu2009≤u20090.001). Regarding the QLQ-C30, PCA indicated a reprioritization of symptom scales and reconceptualization via an increased correlation between functional scales.ConclusionsOur findings demonstrate the usefulness of these analyses in characterizing the occurrence of RS. MCA and IRT model had convergent results with then-test method to characterize recalibration component of RS. PCA is an indirect method in investigating the reprioritization and reconceptualization components of RS.

Impact of TG4010 Vaccine on Health-Related Quality of Life in Advanced Non-Small-Cell Lung Cancer: Results of a Phase IIB Clinical Trial

Background This study describes the effect of TG4010 vaccine on Health related Quality of Life (HRQOL) in patients with stage IIIb and IV non–small-cell lung cancer (NSCLC). Methods 148 patients with advanced NSCLC expressing MUC1 were randomly assigned to receive TG4010 plus chemotherapy or chemotherapy alone. HRQOL was assessed with the Functional Assessment of Cancer Therapy-Lung (FACT-L) at baseline and every 6 weeks until disease progression. Time until definitive deterioration (TUDD) of the four well-being dimensions of the FACT-L physical (PWB), functional (FWB), emotional (EWB) and social well-being (SWB) and the Lung Cancer Subscale (LCS) domains were analyzed for a 5-point minimal clinically important difference. Results No difference of TUDD of HRQOL has been found between treatment arms. No prognostic factors have been found to have a significant impact on the TUDD of PWB, SWB and LCS domains. The gender, the performance status and the smoking habits seemed to be associated with a shorter TUDD of EWB domain. The smokers and the former smokers seemed to present a shorter TUDD of FWB domain. Conclusion This study suggests that adding therapeutic vaccination with TG4010 to standard chemotherapy in patients with advanced NSCLC is associated with a similar evolution in HRQOL compared to chemotherapy alone.

Methodology of health-related quality of life analysis in phase III advanced non-small-cell lung cancer clinical trials: a critical review

BackgroundHealth-related quality of life (HRQoL) is recognized as a component endpoint for cancer therapy approvals. The aim of this review was to evaluate the methodology of HRQoL analysis and reporting in phase III clinical trials of first-line chemotherapy in advanced non-small cell lung cancers (NSCLC).MethodsA search in MEDLINE databases identified phase III clinical trials in first-line chemotherapy for advanced NSCLC, published between January 2008 to December 2014. Two authors independently extracted information using predefined data abstraction forms.ResultsA total of 55 phase III advanced NSCLC trials were identified. HRQoL was declared as an endpoint in 27 studies (49xa0%). Among these 27 studies, The EORTC questionnaire Quality of Life Questionnaire C30 was used in 13 (48xa0%) of the studies and The Functional Assessment of Cancer Therapy-General was used in 12 (44xa0%) trials. The targeted dimensions of HRQoL, the minimal clinically important difference and the statistical approaches for dealing with missing data were clearly specified in 13 (48.1xa0%), 9 (33.3xa0%) and 5 (18.5xa0%) studies, respectively. The most frequent statistical methods for HRQoL analysis were: the mean change from baseline (33.3xa0%), the linear mixed model for repeated measures (22.2xa0%) and time to HRQoL score deterioration (18.5xa0%). For each targeted dimension, the results for each group, the estimated effect size and its precision were clearly reported in 4 studies (14.8xa0%), not clearly reported in 11 studies (40.7xa0%) and not reported at all in 12 studies (44.4xa0%).ConclusionsThis review demonstrated the weakness and the heterogeneity of the measurement, analysis, and reporting of HRQoL in phase III advanced NSCLC trials. Precise and uniform recommendations are needed to compare HRQoL results across publications and to provide understandable messages for patients and clinicians.

Impact of the occurrence of a response shift on the determination of the minimal important difference in a health-related quality of life score over time

BackgroundAn important challenge of the longitudinal analysis of health-related quality of life (HRQOL) is the potential occurrence of a Response Shift (RS) effect. While the impact of RS effect on the longitudinal analysis of HRQOL has already been studied, few studies have been conducted on its impact on the determination of the Minimal Important Difference (MID). This study aims to investigate the impact of the RS effect on the determination of the MID over time for each scale of both EORTC QLQ-C30 and QLQ-BR23 questionnaires in breast cancer patients.MethodsPatients with breast cancer completed the EORTC QLQ-C30 and the EORTC QLQ-BR23 questionnaires at baseline (time of diagnosis; T0), three months (T1) and six months after surgery (T2). Four hospitals and care centers participated in this study: cancer centers of Dijon and Nancy, the university hospitals of Reims and Strasbourg At T1 and T2, patients were asked to evaluate their HRQOL change during the last 3xa0months using the Jaeschke transition question. They were also asked to assess retrospectively their HRQOL level of three months ago.The occurrence of the RS effect was explored using the then-test method and its impact on the determination of the MID by using the Anchor-based method.ResultsBetween February 2006 and February 2008, 381 patients were included of mean age 58xa0years old (SDu2009=u200911). For patients who reported a deterioration of their HRQOL level at each follow-up, an increase of RS effect has been detected between T1 and T2 in 13/15 dimensions of QLQ-C30 questionnaire, and 4/7 dimensions of QLQ-BR23 questionnaire. In contrast, a decrease of the RS effect was observed in 8/15 dimensions of QLQ-C30 questionnaire and in 5/7 dimensions of QLQ-BR23 questionnaire in case of improvement. At T2, the MID becameu2009≥u20095 points when taking into account the RS effect in 10/15 dimensions of QLQ-C30 questionnaire and in 5/7 dimensions of QLQ-BR23 questionnaire.ConclusionsThis study highlights that the RS effect increases over time in case of deterioration and decreases in case of improvement. Moreover, taking the RS into account produces a reliable and significant MID.

Health-related quality-of-life as co-primary endpoint in randomized clinical trials in oncology

Overall survival (OS) has been considered as the most relevant primary endpoint but trials using OS often require large numbers of patients and long-term follow-up. Therefore composite endpoints, which are assessed earlier, are frequently used as primary endpoint but suffer from important limitations specially a lack of validation as surrogate of OS. Therefore, Health-related quality of life (HRQoL) could be considered as an outcome to judge efficacy of a treatment. An alternative approach would be to combine HRQoL with composite endpoints as co-primary endpoint to ensure a clinical benefit for patients of a new therapy. The decision rules of such design, the procedure to control the Type I error and the determination of sample size remain questions to debate. Here, we discusses HRQoL as co-primary endpoints in randomized clinical trials in oncology and provide some solutions to promote such design.

Evaluation properties of the French version of the OUT-PATSAT35 satisfaction with care questionnaire according to classical and item response theory analyses

PurposeThe present study investigates the properties of the French version of the OUT-PATSAT35 questionnaire, which evaluates the outpatients’ satisfaction with care in oncology using classical analysis (CTT) and item response theory (IRT).MethodsThis cross-sectional multicenter study includes 692 patients who completed the questionnaire at the end of their ambulatory treatment. CTT analyses tested the main psychometric properties (convergent and divergent validity, and internal consistency). IRT analyses were conducted separately for each OUT-PATSAT35 domain (the doctors, the nurses or the radiation therapists and the services/organization) by models from the Rasch family. We examined the fit of the data to the model expectations and tested whether the model assumptions of unidimensionality, monotonicity and local independence were respected.ResultsA total of 605 (87.4xa0%) respondents were analyzed with a mean age of 64xa0years (range 29–88). Internal consistency for all scales separately and for the three main domains was good (Cronbach’s α 0.74–0.98). IRT analyses were performed with the partial credit model. No disordered thresholds of polytomous items were found. Each domain showed high reliability but fitted poorly to the Rasch models. Three items in particular, the item about “promptness” in the doctors’ domain and the items about “accessibility” and “environment” in the services/organization domain, presented the highest default of fit. A correct fit of the Rasch model can be obtained by dropping these items. Most of the local dependence concerned items about “information provided” in each domain. A major deviation of unidimensionality was found in the nurses’ domain.ConclusionsCTT showed good psychometric properties of the OUT-PATSAT35. However, the Rasch analysis revealed some misfitting and redundant items. Taking the above problems into consideration, it could be interesting to refine the questionnaire in a future study.

Patient-reported outcomes enhance the survival prediction of traditional disease risk classifications: An international study in patients with myelodysplastic syndromes

Current prognostic systems for myelodysplastic syndromes (MDS) are based on clinical, pathologic, and laboratory indicators. The objective of the current study was to develop a new patient‐centered prognostic index for patients with advanced MDS by including self‐reported fatigue severity into a well‐established clinical risk classification: the International Prognostic Scoring System (IPSS).

La qualité de vie relative à la santé dans les essais cliniques de phase III en oncologie : de l’administration du questionnaire à l’analyse statistique

Endpoints refer to clinical and biological measurements that assess efficacy of therapeutic strategies. As the American Society of Clinical Oncology states, active treatment in cancer is generally undertaken with the goal of providing improved quantity and/or quality of patient survival. Health-related quality of life (HRQoL) reflects the patient-perceived evaluation of ones health, including physical, emotional, and social dimensions as well as symptoms due to disease or treatment. HRQoL is recognized as a component endpoint for cancer therapy approvals by the American Society of Clinical Oncology and the FDA. Many self-completion HRQoL questionnaires have been developed and validated. Two main statistical methods have been developed to longitudinally analyze HRQoL. The first is the linear mixed model for repeated measure (LMM). The second is a survival approach, which estimates the time to HRQoL deterioration. However, there is no guideline for methods of analyzing and reporting longitudinal changes in HRQoL scores. Moreover, HRQoL could also be combined with other endpoints like progression-free survival as co-primary endpoint, but the use of co-primary endpoints in cancer clinical trials is a new approach and methodological researches must be pursued to promote such designs.

Cross-cultural adaptation and validation of the French version of the Expanded Prostate cancer Index Composite questionnaire for health-related quality of life in prostate cancer patients

BackgroundHealth-related quality of life (HRQoL) has been positioned as one of the major endpoints in oncology. Thus, there is a need to validate cancer-site specific survey instruments. This study aimed to perform a transcultural adaptation of the 50-item Expanded Prostate cancer Index Composite (EPIC) questionnaire for HRQoL in prostate cancer patients and to validate the psychometric properties of the French-language version.MethodsThe EPIC questionnaire measures urinary, bowel, sexual and hormonal domains. The first step, corresponding to transcultural adaptation of the original English version of the EPIC was performed according to the back translation technique. The second step, comprising the validation of the psychometric properties of the EPIC questionnaire, was performed in patients under treatment for localized prostate cancer (treatment group) and in patients cured of prostate cancer (cured group). The EORTC QLQ-C30 and QLQ-PR25 prostate cancer module were also completed by patients to assess criterion validity. Two assessments were performed, i.e., before and at the end of treatment for the Treatment group, to assess sensitivity to change; and at 2 weeks’ interval in the Cured group to assess test-retest reliability. Psychometric properties were explored according to classical test theory.ResultsThe first step showed overall good acceptability and understanding of the questionnaire. In the second step, 215 patients were included from January 2012 to June 2014: 125 in the Treatment group, and 90 in the Cured group. All domains exhibited good internal consistency, except the bowel domain (Cronbach’s αu2009=u20090.61). No floor effect was observed. Test-retest reliability assessed in the cured group was acceptable, expect for bowel function (intraclass coefficientu2009=u20090.68). Criterion validity was good for each domain and subscale. Construct validity was not demonstrated for the hormonal and bowel domains. Sensitivity to change was exhibited for 5/8 subscales and 2/4 summary scores for patients who experienced toxicities during treatment.ConclusionsThe French EPIC questionnaire seems to have adequate psychometric properties, comparable to those exhibited by the original English-language version, except for the construct validity, which was not available in original version.