scholarly journals The Prognostic Value for Survival of Patient-Reported Outcomes in Hematological Malignancies: A Systematic Review

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 4793-4793
Author(s):  
Avrita Campinha-Bacote ◽  
Peter C Trask ◽  
Fabio Efficace
Keyword(s):  
Quality Of Life ◽  
Prognostic Value ◽  
Patient Reported Outcomes ◽  
Hematological Malignancies ◽  
Multivariate Analyses ◽  
Life Questionnaire ◽  
Quality Of Life Questionnaire ◽  
Patient Reported ◽  
Pre Treatment

Abstract Introduction: Patient-reported outcomes (PROs) are now critical to more comprehensively evaluate treatment outcomes in oncology. There is convincing evidence indicating that, similarly to traditional biomarkers, PROs may also predict survival outcomes. This systematic review examined state of the art literature on the prognostic value of PROs for survival in hematological malignancies. Methods: We searched PubMed and Google Scholar from 2000-2016 for studies assessing the predictive relationship between PROs and overall survival (OS) in Acute Myelogenous Leukemia (AML), Chronic Lymphocytic Lymphoma (CLL), Diffuse Large B-Cell Lymphoma (DLBCL), indolent Non-Hodgkin's Lymphoma (iNHL), Follicular Lymphoma (FL), marginal Zone Lymphoma (mZL), Myelodysplastic Syndrome (MDS), and Multiple Myeloma (MM). Search terms included but were not limited to: quality of life, physical functioning, predictive/prognostic, survival, patient-reported outcomes, and fatigue. Studies were excluded if they did not use multivariate analyses, did not examine OS as an endpoint, were case reports, did not report results for PROs, were reported in an abstract only, or used only clinician-reported outcomes as a predictor. A pre-defined data extraction form (DEF) was used to assess the following data from each article: disease, sample size, demographics, type of PRO measures included, clinical parameters included as predictors, type of analysis, and outcomes assessed. Results: 223 articles were reviewed of which 13 met the inclusion criteria (6 AML/MDS, 3 MM, 2 aggressive lymphomas, 1 mixed heme diagnoses, and 1 DLBCL). PROs in these studies were captured with the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire - Core30 (EORTC QLQ-C30) or the EORTC myeloma module 24 item (MY24), a health-related quality of life questionnaire for patients with myelodysplastic syndromes (QOL-E), Functional Assessment of Cancer Treatment (FACT) or Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F), and the MD Anderson Symptom Inventory (MDASI). PROs that were independently associated with OS in multivariate analyses from the different studies were: physical function, fatigue, pain, difficulty with strenuous activity, and impaired physical performance in AML patients; physical function and fatigue in MM; fatigue in MDS; global/total QOL and functional well-being in aggressive lymphomas; drowsiness in a mixed group of heme diagnoses; and global QOL, function, and symptoms/signs in DLBCL (see Table). Baseline PROs were obtained following diagnosis of de novo or relapsed disease in 3 studies, within 6 months of registration in 1 study, and prior to any treatment in 6 studies. Conclusions: PROs related to physical functioning, fatigue, and performance were the most common predictors of OS in hematological malignancies and remained significant indicators even after accounting for clinical variables. This review highlights the prognostic value of PROs in both newly diagnosed and relapsed patients, and argues for their use in clinical decision-making and risk assessment at the time of diagnosis and pre-treatment. It also raises the question of how baseline PROs should be used in assessing new treatments within clinical trials in order to identify patients who may respond better to treatments or be in need of pre-treatment adjuvant therapy. Table Table. Disclosures Campinha-Bacote: Genentech, Inc.: Employment. Trask:Genentech, Inc.: Employment, Equity Ownership. Efficace:Lundbeck: Research Funding; Seattle Genetics: Consultancy; Bristol Myers Squibb: Consultancy; TEVA: Consultancy, Research Funding.

Patient-Reported Outcomes After Hepatic Resection of Colorectal Cancer Metastases

2012 ◽  
Vol 30 (12) ◽  
pp. 1364-1370 ◽  
Author(s):  
Jonathan R. Rees ◽  
Jane M. Blazeby ◽  
Peter Fayers ◽  
Elizabeth A. Friend ◽  
Fenella K.S. Welsh ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Liver Metastases ◽  
Hepatic Resection ◽  
Patient Reported Outcomes ◽  
Hepatic Metastases ◽  
Life Questionnaire ◽  
Quality Of Life Questionnaire ◽  
Patient Reported ◽  
The Impact

Purpose Hepatic resection of colorectal carcinoma (CRC) liver metastases is increasing, but evidence for the impact of surgery on patient-reported outcomes (PROs) is limited. This study aimed to describe comprehensively the impact of liver surgery for CRC hepatic metastases on PROs. Patients and Methods Consecutive patients selected for hepatic resection completed the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire–C30 and Quality of Life Questionnaire–Liver Metastases C21 before and 3, 6, and 12 months after surgery. For functional scales, mean scores with 95% CIs were calculated at each time point, with differences in scores of at least 10 points considered clinically significant. Responses to symptom scales and items were categorized as minimal or severe. Proportions and 95% CIs for each symptom category were calculated. Results Hepatic surgery was planned in 241 patients but abandoned in nine because of unresectable disease. There were two postoperative deaths, 58 complications (25.2%), and 32 patients (14.9%) with disease recurrence. Questionnaire compliance was excellent (> 95% at all time points). After surgery, most functional aspects of health decreased, and the proportions of patients with severe symptoms increased; role function deteriorated significantly, and 30% of patients reported severe activity/vigor problems. Functional scales recovered by 6 months and were maintained at 1 year. Postoperative symptoms returned to baseline levels at 12 months, but 32.1% of patients reported severe problems with sexual dysfunction and 11.9% with abdominal pain. Conclusion These findings provide new evidence regarding outcomes of liver resection for CRC metastases. It is recommended that patients be reassured that surgery has a minimal and short-lived detrimental impact on health.

scholarly journals The 27-Item Multiple Sclerosis Quality of Life Questionnaire: A New Brief Measure Including Treatment Burden and Work Life

2021 ◽  
Author(s):  
Helen Beckmann ◽  
Christoph Heesen ◽  
Matthias Augustin ◽  
Christine Blome
Keyword(s):  
Quality Of Life ◽  
Multiple Sclerosis ◽  
Construct Validity ◽  
Convergent Validity ◽  
Life Questionnaire ◽  
Quality Of Life Questionnaire ◽  
Patient Reported ◽  
Disease Specific

Abstract Background: Treatment- and work-related aspects have been neglected in health-related quality of life (HRQOL) measures in multiple sclerosis (MS). We aimed to develop a brief instrument covering all important impairment-, activity-, participation-, and treatment-related aspects for use in research and practice. Methods: The 27-item Multiple Sclerosis Quality of Life Questionnaire (MS-QLQ27) was developed using open item collection, a multidisciplinary expert panel, and cognitive pretesting. It was evaluated for reliability, construct validity, and responsiveness with 100 patients presenting with relapse (84 at follow-up ~14 days later). Construct validity was analyzed by correlating the MS-QLQ27 with the disease-specific Hamburg Quality of Life Questionnaire in MS (HAQUAMS) and generic HRQOL instuments. The Expanded Disability Status Scale (EDSS) was used to analyze known-groups validity. Responsiveness was determined as the correlation of changes in MS-QLQ27 scores with changes in validation criteria. Results: Internal consistency was high (Cronbach α = 0.94 at baseline and 0.93 at follow-up). Convergent validity was supported by direction and magnitude of associations with disease-specific and generic instruments. Correlations with change in convergent criteria were strong, indicating responsiveness. The HAQUAMS showed the strongest associations with the MSQLQ27. The MS-QLQ27 showed the highest effect size compared with other patient-reported outcomes and the EDSS. It successfully distinguished between levels of disease severity. Conclusions: These results indicate that the MS-QLQ27 is a reliable, valid, and highly responsive instrument for assessing HRQOL during relapse evolution in MS. Its advantages are that it is brief yet comprehensive, covering work- and treatment-related aspects not addressed in previous measures.

scholarly journals Quality of life trajectories during the first three years after diagnosis of breast cancer: the NEON-BC study

2019 ◽  
Author(s):  
Luisa Lopes-Conceição ◽  
Mariana Brandão ◽  
Natália Araújo ◽  
Milton Severo ◽  
Teresa Dias ◽  
...  
Keyword(s):  
Breast Cancer ◽  
Quality Of Life ◽  
Sleep Quality ◽  
Patient Reported Outcomes ◽  
Life Questionnaire ◽  
European Organization ◽  
Life Trajectories ◽  
Patient Reported ◽  
Anxiety Depression

Abstract Background We aimed to identify and characterize quality of life trajectories up to 3 years after breast cancer diagnosis. Methods A total of 460 patients were evaluated at baseline (before treatments), and after 1- and 3-years. Patient-reported outcomes, including quality of life (European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30, QLQ-C30), anxiety, depression and sleep quality, were assessed in all evaluations. Model-based clustering was used to identify quality of life trajectories. Results We identified four trajectories without intersection during 3 years. The two trajectories characterized by better quality of life depicted relatively stable scores; in the other trajectories, quality of life worsened until 1 year, though in one of them the score at 3 years improved. Sociodemographic and clinical characteristics at baseline did not differ between trajectories, except for mastectomy, which was higher in the worst trajectory. Anxiety, depression and poor sleep quality increased from the best to the worst trajectory. Conclusions The type of surgery and the variation of other patient-reported outcomes were associated with the course of quality of life over 3 years. More research to understand the heterogeneity of individual trajectories within these major patterns of variation is needed.

Patient-reported outcomes from a phase 2 study of copanlisib in patients with relapsed/refractory indolent B-cell non-Hodgkin lymphoma (iNHL).

2017 ◽  
Vol 35 (15_suppl) ◽  
pp. e18123-e18123
Author(s):  
Karen N. Keating ◽  
Florian Hiemeyer ◽  
Jose E. Garcia-Vargas ◽  
Barrett H. Childs ◽  
Martin H. Dreyling ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Patient Reported Outcomes ◽  
Background Information ◽  
Life Questionnaire ◽  
Health Related Quality ◽  
Imaging Results ◽  
Patient Reported ◽  
Related Quality ◽  
Health Related

e18123 Background: Information on the degree to which treatment of iNHL impacts health-related quality of life in terms of disease- and treatment-related symptoms is limited. Copanlisib is an investigational pan-Class I phosphatidylinositol 3-kinase (PI3K) inhibitor. In a phase II study in patients with relapsed/refractory iNHL, copanlisib treatment resulted in a high response rate and durable responses. We report here on patient-reported outcomes (PRO) from this trial (NCT01660451, part B). Methods: Patients with 4 subtypes of iNHL were eligible: follicular, marginal zone, small lymphocytic and lymphoplasmacytoid/Waldenstrӧm macroglobulinemia. Patients had to have received ≥2 prior lines of treatment, including rituximab and an alkylating agent. Copanlisib (60 mg, I.V.) was intermittently administered on days 1, 8 and 15 of a 28-day cycle. Health-related quality of life questionnaire FACT-Lym questionnaire (with focus on Total and Lymphoma subscale [FACT-LymS] and LOCF-method applied) was evaluated with Hodges-Lehmann estimate for change in median until week 16, including 95% confidence intervals. EQ-5D and visual analogue scale (EQ-5D VAS) were statistically described. All questionnaires were compiled at baseline and every other cycle in the first year of the study (aligned with radiological imaging). Results: A total of 141 iNHL patients were treated, with completed questionnaires received from 132 patients at baseline and 86 at week 16. The mean±SD (and median) for FACT-Lym LymS score at baseline was 45.27 ± 9.55 (46.5), and 47.20 ± 8.69 (49.0) at 16 weeks. The estimated change in median was +1.0 with 95% CI: [0.5; 2.5] and min: -13.0, max: 23.0, representing an improvement in lymphoma-related symptoms. Accordingly, the FACT-Lym total score was 126.59 ± 22.96 (127.5) at baseline and 127.99 ± 23.60 (130.8) at 16 weeks. Estimated change in median was 0.0 with 95% CI: [-0.5; 3.2] and min: -38.0, max 42.5. Conclusions: This study demonstrated the favorable impact of copanlisib treatment on disease-related symptoms and HRQoL in patients with relapsed/refractory iNHL.

scholarly journals Measuring health-related quality of life in chronic headache: A comparative evaluation of the Chronic Headache Quality of Life Questionnaire and Headache Impact Test (HIT-6)

Cephalalgia ◽  
2021 ◽  
pp. 033310242110060
Author(s):  
Kirstie L Haywood ◽  
Felix Achana ◽  
Vivien Nichols ◽  
Gemma Pearce ◽  
Barbara Box ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Chronic Headache ◽  
Impact Test ◽  
Life Questionnaire ◽  
Specific Patient ◽  
Quality Of Life Questionnaire ◽  
Patient Reported ◽  
Smallest Detectable Change ◽  
Headache Impact

Objective To compare the quality and acceptability of a new headache-specific patient-reported measure, the Chronic Headache Quality of Life Questionnaire (CHQLQ) with the six-item Headache Impact Test (HIT-6), in people meeting an epidemiological definition of chronic headaches. Methods Participants in the feasibility stage of the Chronic Headache Education and Self-management Study (CHESS) (n = 130) completed measures three times during a 12-week prospective cohort study. Data quality, measurement acceptability, reliability, validity, responsiveness to change, and score interpretation were determined. Semi-structured cognitive interviews explored measurement relevance, acceptability, clarity, and comprehensiveness. Results Both measures were well completed with few missing items. The CHQLQ’s inclusion of emotional wellbeing items increased its relevance to participant’s experience of chronic headache. End effects were present at item level only for both measures. Structural assessment supported the three and one-factor solutions of the CHQLQ and HIT-6, respectively. Both the CHQLQ (range 0.87 to 0.94) and HIT-6 (0.90) were internally consistent, with acceptable temporal stability over 2 weeks (CHQLQ range 0.74 to 0.80; HIT-6 0.86). Both measures responded to change in headache-specific health at 12 weeks (CHQLQ smallest detectable change (improvement) range 3 to 5; HIT-6 2.1). Conclusions While both measures are structurally valid, internally consistent, temporally stable, and responsive to change, the CHQLQ has greater relevance to the patient experience of chronic headache. Trial registration number: ISRCTN79708100. Registered 16th December 2015, http://www.isrctn.com/ISRCTN79708100

The Prognostic Significance of Patient-Reported Outcomes in Cancer Clinical Trials

2008 ◽  
Vol 26 (8) ◽  
pp. 1355-1363 ◽  
Author(s):  
Carolyn C. Gotay ◽  
Crissy T. Kawamoto ◽  
Andrew Bottomley ◽  
Fabio Efficace
Keyword(s):  
Quality Of Life ◽  
Clinical Trials ◽  
Patient Reported Outcomes ◽  
Prognostic Significance ◽  
Cancer Clinical Trials ◽  
Life Questionnaire ◽  
European Organization ◽  
Patient Reported ◽  
The Impact

Purpose Patient-reported outcomes (PROs), routinely collected as a part of cancer clinical trials, have been linked with survival in numerous clinical studies, but a comprehensive critical review has not been reported. This study systematically assessed the impact of PROs on patient survival after a cancer diagnosis within the context of clinical trials. Design Cancer clinical trials that assessed baseline PROs and mortality were identified through MEDLINE (through December 2006) supplemented by the Cochrane database, American Society of Clinical Oncology/European Society for Medical Oncology abstracts and hand searches. Inclusion criteria were publication in English language and use of multivariate analyses of PROs that controlled for one or more clinical factors. Two raters reviewed each study, abstracted data, and assessed study quality; two additional raters verified abstractions. Results In 36 of 39 studies (N = 13,874), at least one PRO was significantly associated with survival (P < .05) in multivariate analysis, with varying effect sizes. Studies of lung (n = 12) and breast cancer (n = 8) were most prevalent. The most commonly assessed PRO was quality of life, measured by the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire C30 in 56% of studies. Clinical variables adjusted for included performance status (PS), treatment arm, stage, weight loss, and serum markers. Results indicated that PROs provide distinct prognostic information beyond standard clinical measures in cancer clinical trials. Conclusion PROs might be considered for stratification purposes in future trials, as they were often better predictors of survival than PS. Studies are needed to determine whether interventions that improve PROs also increase survival and to identify explanatory mechanisms through which PROs relate to survival.

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