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PubMed | Hematology and Bone Marrow Transplant Unit, San Gerardo Hospital, UO Medicina Interna e Onco Ematologica P.O. Umberto I, University of Rome La Sapienza and 13 more.
Type: Journal Article | Journal: BMJ supportive & palliative care | Year: 2016

The primary objective of this study was to evaluate the health-related quality of life (HRQOL) in lower-risk, transfusion-dependent patients with myelodysplastic syndromes (MDS) treated with deferasirox. A secondary objective was to investigate the relationship between HRQOL, serum ferritin levels and transfusion dependency.This was a prospective multicentre study enrolling 159 patients, of whom 152 received at least one dose of deferasirox. HRQOL was assessed with the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30) at baseline and then at 3, 6, 9 and 12months. Primary analysis was performed estimating mean HRQOL scores over time by a linear mixed model on selected scales.The median age of treated patients was 72years (range 24-87years). No statistically significant changes over time were found in mean scores for global health status/quality of life (p=0.564), physical functioning (p=0.409) and fatigue (p=0.471) scales. Also, no significant changes were found for constipation (p=0.292), diarrhoea (p=0.815) and nausea and vomiting (p=0.643). Serum ferritin levels were not associated with HRQOL outcomes. A higher patient-reported baseline pain severity was an independent predictive factor of an earlier achievement of transfusion independence with a HR of 1.032 (99% CI 1.004 to 1.060; p=0.003).HRQOL of transfusion-dependent patients with MDS receiving deferasirox therapy remains stable over time. HRQOL assessment might also provide important predictive information on treatment outcomes.NCT00469560.


PubMed | University Utrecht, Catharina Hospital and Data Center and Health Outcomes Research Unit
Type: | Journal: European journal of cancer (Oxford, England : 1990) | Year: 2016

Patient-reported outcomes (PROs) are amongst the most relevant outcome measures in pancreatic cancer care and research. However, it is unknown which out of the numerous PROs are most important to patients and health care professionals (HCPs) in this setting. The aim of this study was to identify a core set of PROs to be incorporated in a nationwide prospective multidisciplinary pancreatic cancer registry.We performed a two-round Delphi survey among 150 patients diagnosed with pancreatic or periampullary cancer (treated either with curative intent or in palliative setting) and 78 HCPs (surgeons, medical oncologists, gastroenterologists, radiotherapists, nurses, and dietitians) in The Netherlands. In round 1, participants were invited to rate the importance of 53 PROs, which were extracted from 17 different PRO measures and grouped into global domains, on a 1-9 Likert scale. PROs rated as very important (score 7-9) by the majority ( 80%) of curative and/or palliative patients as well as HCPs were considered sufficiently important to be incorporated in the core set. PROs not fulfilling these criteria in round 1 were presented again to the participants in round 2 along with individual and group feedback.A total of 97 patients (94%) in curative-intent setting, 38 patients (81%) in palliative setting and 73 HCPs (94%) completed both rounds 1 and 2. After the first round, 7 PROs were included in the core set: general quality of life, general health, physical ability, satisfaction with caregivers, satisfaction with services and care organisation, coping and defecation. After the second round, 10 additional PROs were added: appetite, ability to work/do usual activities, medication use, weight changes, fatigue, negative feelings, positive feelings, fear of recurrence, relationship with partner/family, and pancreatic enzyme replacement therapy use.This study provides a core set of PROs selected by patients and HCPs, which may be incorporated in pancreatic cancer care and research. Validation outside the Dutch context is recommended for generalisation and use in international studies.


Efficace F.,Data Center and Health Outcomes Research Unit | Feuerstein M.,Sloan Kettering Cancer Center | Fayers P.,University of Aberdeen | Cafaro V.,Data Center and Health Outcomes Research Unit | And 3 more authors.
European Urology | Year: 2014

Context Patient-reported outcomes (PRO) data from randomised controlled trials (RCTs) are increasingly used to inform patient-centred care as well as clinical and health policy decisions. Objective The main objective of this study was to investigate the methodological quality of PRO assessment in RCTs of prostate cancer (PCa) and to estimate the likely impact of these studies on clinical decision making. Evidence acquisition A systematic literature search of studies was undertaken on main electronic databases to retrieve articles published between January 2004 and March 2012. RCTs were evaluated on a predetermined extraction form, including (1) basic trial demographics and clinical and PRO characteristics; (2) level of PRO reporting based on the recently published recommendations by the International Society for Quality of Life Research; and (3) bias, assessed using the Cochrane Risk of Bias tool. Studies were systematically analysed to evaluate their relevance for supporting clinical decision making. Evidence synthesis Sixty-five RCTs enrolling a total of 22 071 patients were evaluated, with 31 (48%) in patients with nonmetastatic disease. When a PRO difference between treatments was found, it related in most cases to symptoms only (n = 29, 58%). Although the extent of missing data was generally documented (72% of RCTs), few reported details on statistical handling of this data (18%) and reasons for dropout (35%). Improvements in key methodological aspects over time were found. Thirteen (20%) RCTs were judged as likely to be robust in informing clinical decision making. Higher-quality PRO studies were generally associated with those RCTs that had higher internal validity. Conclusions Including PRO in RCTs of PCa patients is critical for better evaluating the treatment effectiveness of new therapeutic approaches. Marked improvements in PRO quality reporting over time were found, and it is estimated that at least one-fifth of PRO RCTs have provided sufficient details to allow health policy makers and physicians to make critical appraisals of results. Patient summary In this report, we have investigated the methodological quality of PCa trials that have included a PRO assessment. We conclude that including PRO is critical to better evaluating the treatment effectiveness of new therapeutic approaches from the patient's perspective. Also, at least one-fifth of PRO RCTs in PCa have provided sufficient details to allow health policy makers and physicians to make a critical appraisal of results. © 2013 Published by Elsevier B.V. on behalf of European Association of Urology.


Efficace F.,Data Center and Health Outcomes Research Unit | Jacobs M.,University of Amsterdam | Pusic A.,Sloan Kettering Cancer Center | Greimel E.,Medical University of Graz | And 5 more authors.
European Journal of Cancer | Year: 2014

Aim The aim for this study is to investigate the methodological quality and potential impact on clinical decision making of patient reported outcome (PRO) assessment in randomised controlled trials (RCTs) in the gynaecological cancer sites. Methods A systematic review identified RCTs published between January 2004 and June 2012. Relevant studies were evaluated using a pre-determined extraction form which included: (1) Trial demographics and clinical and PRO characteristics; (2) level of PRO reporting and (3) bias, assessed using the Cochrane Risk of Bias tool. All studies were additionally analysed in relation to their relevance in supporting clinical decision making. Results Fifty RCTs enrolling 24,991 patients were identified. In eight RCTs (16%) a PRO was the primary end-point. Twenty-one studies (42%) were carried out in a multi-national context. Where statistically significant PRO differences between treatments were found, it related in most cases to both symptoms and domains other than symptoms (n = 17, 57%). The majority of studies (n = 42, 84%) did not mention the mode of administration nor the methods of collecting PRO data. Statistical approaches for dealing with missing data were only explicitly mentioned in nine RCTs (18%). Sixteen RCTs (32%) were considered to be of high-quality and thus able to inform clinical decision making. Higher-quality PRO studies were generally associated with RCTs that were at a low risk of bias. Conclusion This study showed that RCTs with PROs were generally well designed and conducted. In a third the information was very informative to fully understand the pros and cons of PROs treatment decision-making. © 2014 Elsevier Ltd. All rights reserved.


Efficace F.,Data Center and Health Outcomes Research Unit | Fayers P.,University of Aberdeen | Pusic A.,Sloan Kettering Cancer Center | Cemal Y.,Sloan Kettering Cancer Center | And 7 more authors.
Cancer | Year: 2015

BACKGROUND: The main objectives of this study were to identify the number of randomized controlled trials (RCTs) including a patient-reported outcome (PRO) endpoint across a wide range of cancer specialties and to evaluate the completeness of PRO reporting according to the Consolidated Standards of Reporting Trials (CONSORT) PRO extension. METHODS: RCTs with a PRO endpoint that had been performed across several cancer specialties and published between 2004 and 2013 were considered. Studies were evaluated on the basis of previously defined criteria, including the CONSORT PRO extension and the Cochrane Collaboration's tool for assessing the risk of bias of RCTs. Analyses were also conducted by the type of PRO endpoint (primary vs secondary) and by the cancer disease site. RESULTS: A total of 56,696 potentially eligible records were scrutinized, and 557 RCTs with a PRO evaluation, enrolling 254,677 patients overall, were identified. PROs were most frequently used in RCTs of breast (n = 123), lung (n = 85), and colorectal cancer (n = 66). Overall, PROs were secondary endpoints in 421 RCTs (76%). Four of 6 evaluated CONSORT PRO items were documented in less than 50% of the RCTs. The level of reporting was higher in RCTs with a PRO as a primary endpoint. The presence of a supplementary report was the only statistically significant factor associated with greater completeness of reporting for both RCTs with PROs as primary endpoints (β =.19, P =.001) and RCTs with PROs as secondary endpoints (β =.30, P <.001). CONCLUSIONS: Implementation of the CONSORT PRO extension is equally important across all cancer specialties. Its use can also contribute to revealing the robust PRO design of some studies, which might be obscured by poor outcome reporting. © 2015 American Cancer Society.


PubMed | University of Amsterdam, University of Sydney, University of Bristol, Auckland City Hospital and Data Center and Health Outcomes Research Unit
Type: | Journal: European journal of cancer (Oxford, England : 1990) | Year: 2016

To determine the completeness of reporting of patient-reported outcomes (PROs) of head and neck cancer (HNC) and thyroid cancer randomised-controlled trials (RCTs) and identify PRO measures used.A systematic literature search was conducted for HNC and thyroid cancer RCTs with PRO end-points (January 2004-June 2015). Two investigators independently extracted data, assessed adherence to the International Society for Quality of Life Research (ISOQOL) PRO reporting standards and concordance between hypotheses and PRO measures used. Data wereentered into the Patient-Reported Outcomes Measurements Over Time in Oncology (PROMOTION) Registry.Sixty-six RCTs were included, 56 (85%) HNC and 10 (15%) thyroid cancer. Twenty-two (33%) included a primary and 44 (67%) included a secondary PRO end-point. A total of 40 unique PRO measures were used. Adherence to the ISOQOL PRO reporting standards was higher for RCTs with primary PRO end-points than for secondary PRO end-points: (mean adherence of 43% and 29% respectively). Completeness of PRO reporting did not improve with time: r=.13, p=.31. ISOQOL checklist items poorly reported included: PRO hypothesis (reported for eight RCTs, 12%), justification chosen of PRO measures (n=16, 24%), rates of missing PRO data (n=19, 29%), and generalisability of results (n=12, 18%). Encouragingly, PROs were identified in 55 RCT abstracts (83%) and PRO results interpreted for 30 RCTs (45%).Reporting of PRO end-points was more comprehensive in RCTs with primary rather than secondary PRO end-points. Improvement is needed in the transparent reporting of PRO studies, particularly regarding data collection, analyses and generalisability of PRO results.


PubMed | University of Amsterdam, Sloan Kettering Cancer Center, University of Bristol, University of California at Los Angeles and 3 more.
Type: Journal Article | Journal: Cancer | Year: 2015

The main objectives of this study were to identify the number of randomized controlled trials (RCTs) including a patient-reported outcome (PRO) endpoint across a wide range of cancer specialties and to evaluate the completeness of PRO reporting according to the Consolidated Standards of Reporting Trials (CONSORT) PRO extension.RCTs with a PRO endpoint that had been performed across several cancer specialties and published between 2004 and 2013 were considered. Studies were evaluated on the basis of previously defined criteria, including the CONSORT PRO extension and the Cochrane Collaborations tool for assessing the risk of bias of RCTs. Analyses were also conducted by the type of PRO endpoint (primary vs secondary) and by the cancer disease site.A total of 56,696 potentially eligible records were scrutinized, and 557 RCTs with a PRO evaluation, enrolling 254,677 patients overall, were identified. PROs were most frequently used in RCTs of breast (n = 123), lung (n = 85), and colorectal cancer (n = 66). Overall, PROs were secondary endpoints in 421 RCTs (76%). Four of 6 evaluated CONSORT PRO items were documented in less than 50% of the RCTs. The level of reporting was higher in RCTs with a PRO as a primary endpoint. The presence of a supplementary report was the only statistically significant factor associated with greater completeness of reporting for both RCTs with PROs as primary endpoints ( = .19, P = .001) and RCTs with PROs as secondary endpoints ( = .30, P < .001).Implementation of the CONSORT PRO extension is equally important across all cancer specialties. Its use can also contribute to revealing the robust PRO design of some studies, which might be obscured by poor outcome reporting.


Efficace F.,Data Center and Health Outcomes Research Unit | Rosti G.,University of Bologna | Cottone F.,Data Center and Health Outcomes Research Unit | Breccia M.,University of Rome La Sapienza | And 4 more authors.
Leukemia Research | Year: 2014

The main objective of this study was to outline key characteristics, including health-related quality of life (HRQOL) and symptoms, in 175 chronic myeloid leukemia (CML) patients reporting intentional or unintentional reasons for not fully adhering to imatinib therapy. There was a significant higher proportion of males in the unintentional group (P= 0.037). Also, in this group patients were on average younger (P= 0.046). Patients reporting intentional reasons had generally a worse HRQOL profile and a higher symptom severity than those who reported unintentional reasons for non-adherence. This study suggests that patients with suboptimal adherence are not a homogenous group, thus generalized approaches to improve medication-taking behaviors are not recommended. © 2013 Elsevier Ltd.


PubMed | University of Amsterdam, University of Cagliari and Data Center and Health Outcomes Research Unit
Type: Journal Article | Journal: Critical reviews in oncology/hematology | Year: 2015

Health-related quality of life (HRQOL) and other patient-reported outcomes (PROs) are crucial for a comprehensive evaluation of treatment effectiveness. A systematic review of randomized controlled trials (RCTs) with a PRO endpoint conducted in patients with leukemia and myelodysplastic syndromes (MDS) was performed. Eligible studies were evaluated independently, according to a pre-defined coding scheme, by two reviewers. Thirteen RCTs, enrolling overall 3380 patients were identified. There were four RCTs involving acute myeloid leukemia patients (AML), one with acute lymphoid leukemia (ALL), five with chronic lymphocytic leukemia (CLL) and three with MDS. Six RCTs accurately documented PRO methodology assessment and were thus considered likely to robustly inform clinical decision-making. Of these, three RCTs dealt with AML, two with CLL, one with MDS. A growing number of RCTs in leukemia and MDS have included a PRO component in recent years. Inclusion of PROs in RCTs is feasible and can provide unique information to facilitate clinical decision-making.


PubMed | Data Center and Health Outcomes Research Unit
Type: Journal Article | Journal: British journal of haematology | Year: 2015

The primary objective of this study was to investigate factors associated with fatigue severity in newly diagnosed patients with higher-risk myelodysplastic syndromes (MDS). The secondary objectives were to assess symptom prevalence and to examine the relationships between fatigue, quality of life (QoL) and overall symptom burden in these patients. The analyses were conducted in 280 higher-risk MDS patients. Pre-treatment patient-reported fatigue was evaluated with the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scale and QoL was assessed with the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30). Female gender (P=0018), poor performance status (i.e., ECOG of 2-4) (P<0001) and lower levels of haemoglobin (Hb) (P=0026) were independently associated with higher fatigue severity. The three most prevalent symptoms were as follows: fatigue (92%), dyspnoea (63%) and pain (55%). Patients with higher levels of fatigue also had greater overall symptom burdens. The mean global QoL scores of patients with the highest versus those with the lowest levels of fatigue were 292 [standard deviation (SD), 183] and 690 (SD, 188), respectively and this difference was four times the magnitude of a clinically meaningful difference. Patient-reported fatigue severity revealed the effects of disease burden on overall QoL more accurately than did degree of anaemia. Special attention should be given to the female patients in the management of fatigue.

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