Health Outcomes Research Unit

Rome, Italy

Health Outcomes Research Unit

Rome, Italy
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Sodergren S.C.,University of Southampton | White A.,University of Southampton | Efficace F.,Health Outcomes Research Unit | Sprangers M.,University of Amsterdam | And 2 more authors.
Critical Reviews in Oncology/Hematology | Year: 2014

Tyrosine kinase inhibitors (TKIs) have revolutionised the treatment of advanced gastrointestinal stromal tumours (GISTs). Imatinib is approved as first line therapy and sunitinib is used in cases of imatinib resistance or intolerance. Compared with conventional treatments, TKIs are delivered over longer periods of time and are more specific in their targets (i.e., molecularly targeted), thus presenting different side effect profiles. We review the safety profiles of imatinib and sunitinib, documenting a total of 95 side effects including patient based as well as medically defined outcomes. Gastrointestinal complaints, particularly diarrhoea and nausea, oedema, fatigue and haematological disorders, notably anaemia, are amongst the most prevalent side effects. While there is overlap between the side effect profiles of imatinib and sunitinib, important differences emerge in the frequencies of oedema, hypertension, thyroid functioning, muscle and joint pains, as well as skin and oral conditions. Awareness of potential side effects is informative to both clinician and patient in terms of treatment decision making and can have important implications for treatment adherence and clinical outcome. © 2014 Elsevier Ireland Ltd.


Lo-Coco F.,University of Rome Tor Vergata | Lo-Coco F.,Santa Lucia Foundation | Avvisati G.,Biomedical University of Rome | Vignetti M.,Data Center | And 39 more authors.
New England Journal of Medicine | Year: 2013

BACKGROUND: All-trans retinoic acid (ATRA) with chemotherapy is the standard of care for acute promyelocytic leukemia (APL), resulting in cure rates exceeding 80%. Pilot studies of treatment with arsenic trioxide with or without ATRA have shown high efficacy and reduced hematologic toxicity. METHODS: We conducted a phase 3, multicenter trial comparing ATRA plus chemotherapy with ATRA plus arsenic trioxide in patients with APL classified as low-to-intermediate risk (white-cell count, ≤10×109per liter). Patients were randomly assigned to receive either ATRA plus arsenic trioxide for induction and consolidation therapy or standard ATRA- idarubicin induction therapy followed by three cycles of consolidation therapy with ATRA plus chemotherapy and maintenance therapy with low-dose chemotherapy and ATRA. The study was designed as a noninferiority trial to show that the difference between the rates of event-free survival at 2 years in the two groups was not greater than 5%. RESULTS: Complete remission was achieved in all 77 patients in the ATRA-arsenic trioxide group who could be evaluated (100%) and in 75 of 79 patients in the ATRA-chemotherapy group (95%) (P = 0.12). The median follow-up was 34.4 months. Two-year event-free survival rates were 97% in the ATRA-arsenic trioxide group and 86% in the ATRA-chemotherapy group (95% confidence interval for the difference, 2 to 22 percentage points; P<0.001 for noninferiority and P = 0.02 for superiority of ATRA-arsenic trioxide). Overall survival was also better with ATRA-arsenic trioxide (P = 0.02). As compared with ATRA-chemotherapy, ATRA-arsenic trioxide was associated with less hematologic toxicity and fewer infections but with more hepatic toxicity. CONCLUSIONS: ATRA plus arsenic trioxide is at least not inferior and may be superior to ATRA plus chemotherapy in the treatment of patients with low-to-intermediate-risk APL. Copyright © 2013 Massachusetts Medical Society.


PubMed | Queen's University of Belfast, University of New South Wales, Ontario Cancer Institute, Health Outcomes Research Unit and 7 more.
Type: Journal Article | Journal: BMJ open | Year: 2016

Emerging evidence suggests that patient-reported outcome (PRO)-specific information may be omitted in trial protocols and that PRO results are poorly reported, limiting the use of PRO data to inform cancer care. This study aims to evaluate the standards of PRO-specific content in UK cancer trial protocols and their arising publications and to highlight examples of best-practice PRO protocol content and reporting where they occur. The objective of this study is to determine if these early findings are generalisable to UK cancer trials, and if so, how best we can bring about future improvements in clinical trials methodology to enhance the way PROs are assessed, managed and reported.Trials in which the primary end point is based on a PRO will have more complete PRO protocol and publication components than trials in which PROs are secondary end points.Completed National Institute for Health Research (NIHR) Portfolio Cancer clinical trials (all cancer specialities/age-groups) will be included if they contain a primary/secondary PRO end point. The NIHR portfolio includes cancer trials, supported by a range of funders, adjudged as high-quality clinical research studies. The sample will be drawn from studies completed between 31 December 2000 and 1 March 2014 (n=1141) to allow sufficient time for completion of the final trial report and publication. Two reviewers will then review the protocols and arising publications of included trials to: (1) determine the completeness of their PRO-specific protocol content; (2) determine the proportion and completeness of PRO reporting in UK Cancer trials and (3) model factors associated with PRO protocol and reporting completeness and with PRO reporting proportion.The study was approved by the ethics committee at University of Birmingham (ERN_15-0311). Trial findings will be disseminated via presentations at local, national and international conferences, peer-reviewed journals and social media including the CPROR twitter account and UOB departmental website (http://www.birmingham.ac.uk/cpro0r).PROSPERO CRD42016036533.


Efficace F.,Health Outcomes Research Unit | Baccarani M.,University of Bologna | Breccia M.,University of Rome La Sapienza | Alimena G.,University of Rome La Sapienza | And 21 more authors.
Blood | Year: 2011

The main objective of this study was to investigate whether patients with chronic myeloid leukemia (CML) in treatment with long-term therapy imatinib have a different health-related quality-of-life (HRQOL) profile compared with the general population. In total, 448 CML patients were enrolled, and the SF-36 Health Survey was used to compare generic HRQOL profiles. Symptoms were also assessed. HRQOL comparisons were adjusted for key possible confounders. The median age of patients was 57 years and the median time of imatinib treatment was 5 years (range 3-9 years). The largest HRQOL differences were found in younger patients. In particular, patients aged between 18 and 39 years had marked impairments in role limitations because of physical and emotional problems, respectively: -22.6 (P < .001), -22.3 (P < .001). Patients with CML age 60 or older had a HRQOL profile very similar to that reported by the general population. Women had a worse profile than men when each were compared with their peers in the general population. Fatigue was the most frequently reported symptom. The HRQOL of CML patients is comparable with that of population norms in many areas, however, younger and female patients seem to report the major limitations. © 2011 by The American Society of Hematology.


Calvert M.,University of Birmingham | Brundage M.,Queen's University | Jacobsen P.B.,H. Lee Moffitt Cancer Center and Research Institute | Schunemann H.J.,McMaster University | Efficace F.,Health Outcomes Research Unit
Health and Quality of Life Outcomes | Year: 2013

To inform clinical guidelines and patient care we need high quality evidence on the relative benefits and harms of intervention. Patient reported outcome (PRO) data from clinical trials can " empower patients to make decisions based on their values" and " level the playing field between physician and patient" While clinicians have a good understanding of the concept of health-related quality of life and other PROs, evidence suggests that many do not feel comfortable in using the data from trials to inform discussions with patients and clinical practice. This may in part reflect concerns over the integrity of the data and difficulties in interpreting the results arising from poor reporting.The new CONSORT PRO extension aims to improve the reporting of PROs in trials to facilitate the use of results to inform clinical practice and health policy. While the CONSORT PRO extension is an important first step in the process, we need broader engagement with the guidance to facilitate optimal reporting and maximize use of PRO data in a clinical setting. Endorsement by journal editors, authors and peer reviewers are crucial steps. Improved design, implementation and transparent reporting of PROs in clinical trials are necessary to provide high quality evidence to inform evidence synthesis and clinical practice guidelines. © 2013 Calvert et al.; licensee BioMed Central Ltd.


Bredart A.,University Pierre and Marie Curie | Bredart A.,University of Paris Descartes | Kop J.-L.,University of Lorraine | Efficace F.,Health Outcomes Research Unit | And 6 more authors.
Psycho-Oncology | Year: 2015

Background Cancer care is increasingly provided in the outpatient setting, requiring specific monitoring of care quality. The patients' perspective is an important indicator of care quality and needs to be assessed with well designed, psychometrically sound questionnaires. We performed a systematic literature review of currently available patient satisfaction measures for use in cancer outpatient care settings. Methods We carried out MEDLINE/PubMed, PsycINFO, CINAHL, and Scopus searches of papers published over the past 15 years that describe cancer patient satisfaction questionnaires for use in the outpatient setting. We used the adapted COSMIN checklist to assess the quality of the questionnaires' measurement properties. Results A total of 6677 citations were identified and 76 relevant articles were read, of which 55 were found either not to be relevant or to provide insufficient psychometric information. The remaining 21 studies pertained to 14 patient satisfaction questionnaires. Continuity and transition, accessibility, and involvement of family/friends were less frequently addressed despite their relevance in outpatient oncology. Almost half of the psychometric studies did not provide information on item level missing data. Most internal consistency estimates (Cronbach's α) were satisfactory. Few studies reported test-retest assessment (n = 5), used confirmatory factor analysis (n = 2), or assessed fit to a graded response item response theory model (n = 3). Only three questionnaires were cross-culturally validated. Conclusion Important aspects of care may be missed by current patient satisfaction questionnaires for use in the cancer outpatient setting. Additional evidence is needed of their psychometric performance, especially for cross-cultural comparative assessments. © 2014 John Wiley & Sons, Ltd.


Vivat B.,Brunel University | Young T.,Mount Vernon Cancer Center | Efficace F.,Health Outcomes Research Unit | Siguradottir V.,University of Iceland | And 6 more authors.
Palliative Medicine | Year: 2013

Background: No existing stand-alone measures of spiritual wellbeing have been developed in cross-cultural and multiple linguistic contexts. Aim: Cross-cultural development of a stand-alone European Organisation for Research and Treatment of Cancer (EORTC) measure of spiritual wellbeing for palliative care patients with cancer. Design: Broadly following EORTC Quality of Life Group (QLG) guidelines for developing questionnaires, the study comprised three phases. Phase I identified relevant issues and obtained the views of palliative care patients and professionals about those issues. Phase II operationalised issues into items. Phase III pilot-tested those items with palliative care patients. Amendments to the guidelines included an intermediate Phase IIIa, and debriefing questions specific to the measure. Setting/participants: Phase III pilot-testing recruited 113 people with incurable cancer from hospitals and hospices in six European countries and Japan. Results: A provisional 36-item measure ready for Phase IV field-testing, the EORTC QLQ-SWB36, has been developed. Careful attention to translation and simultaneous development in multiple languages means items are acceptable and consistent between different countries and languages. Phase III data from 113 patients in seven countries show that the items are comprehensible across languages and cultures. Phase III patient participants in several countries used the measure as a starting point for discussing the issues it addresses. Conclusion: The EORTC QLG's rigorous cross-cultural development process ensures that the EORTC QLQ-SWB36 identifies key issues for spiritual wellbeing in multiple cultural contexts, and that items are comprehensible and consistent across languages. Some cross-cultural differences were observed, but data were insufficient to enable generalisation. Phase IV field-testing will investigate these differences further. © 2012 The Author(s).


Efficace F.,Health Outcomes Research Unit | Taphoorn M.,VU University Amsterdam | Taphoorn M.,Medical Center Haaglanden
Journal of Neuro-Oncology | Year: 2012

Health-related quality of life (HRQOL) and other types of patient-reported outcomes (PROs) are nowimportant outcome measures in cancer clinical trials. A number of potentially less toxic drugs are available, and newer treatments can potentially offer cancer patients the possibility to be treated with less aggressive approaches, making PROs more critical in evaluating treatment effectiveness. However, assessing PROs in clinical trials requires careful consideration of a number of methodological issues. Robust methodology and accurate reporting of results are crucial to provide the scientific community and health care providers with a transparent message about the impact of a given drug or a new medical approach on patients' health status. This paper provides basic guidance onmethodological issues to be addressed when designing and reporting HRQOL in clinical trials and presents examples of relevant brain cancer studies. © Springer Science+Business Media, LLC. 2012.


PubMed | Sloan Kettering Cancer Center, University Institute of Health Sciences, Health Outcomes Research Unit and The Surgical Center
Type: Journal Article | Journal: Journal of cancer research and clinical oncology | Year: 2015

Randomised controlled trials (RCTs) are the most robust study design measuring outcomes of colorectal cancer (CRC) treatments, but to influence clinical practice trial design and reporting of patient-reported outcomes (PROs) must be of high quality. Objectives of this study were as follows: to examine the quality of PRO reporting in RCTs of CRC treatment; to assess the availability of robust data to inform clinical decision-making; and to investigate whether quality of reporting improved over time.A systematic review from January 2004-February 2012 identified RCTs of CRC treatment describing PROs. Relevant abstracts were screened and manuscripts obtained. Methodological quality was assessed using International Society for Quality of Life Research-patient-reported outcome reporting standards. Changes in reporting quality over time were established by comparison with previous data, and risk of bias was assessed with the Cochrane risk of bias tool.Sixty-six RCTs were identified, seven studies (10 %) reported survival benefit favouring the experimental treatment, 35 trials (53 %) identified differences in PROs between treatment groups, and the clinical significance of these differences was discussed in 19 studies (29 %). The most commonly reported treatment type was chemotherapy (n = 45; 68 %). Improvements over time in key methodological issues including the documentation of missing data and the discussion of the clinical significance of PROs were found. Thirteen trials (20 %) had high-quality reporting.Whilst improvements in PRO quality reporting over time were found, several recent studies still fail to robustly inform clinical practice. Quality of PRO reporting must continue to improve to maximise the clinical impact of PRO findings.


La Nasa G.,R Binaghi Hospital | La Nasa G.,University of Cagliari | Caocci G.,R Binaghi Hospital | Caocci G.,University of Cagliari | And 10 more authors.
Blood | Year: 2013

The principal aim of our study was to investigate whether patients transplanted more than 20 years ago for β-thalassemia major had a different health-related quality of life (HRQoL) compared with the general population. The Medical Outcomes Study 36-Item Short-Form Health Survey (SF-36) and the Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) were received from 109 ex-thalassemia patients who underwent hematopoietic stem cell transplantation (HSCT) during the 1980s and 1990s. Adjusted comparisons were performed separately for patient age at HSCT and the presence or absence of graft-versus-host disease (GVHD). Sociodemographic and clinical variables were also analyzed. The median age of our cohort at HSCT and the time of the survey was 12 years (range, 1-36) and 34 years (range, 21-48), respectively, with a median follow-up age of 22.8 years (range, 11.7-30.3). Statistical analysis of data collected more than 20 years after HSCT showed that the long-term HRQoL of ex-thalassemia patients was very similar to that of the general population. Clinical meaningful differences were only found for the general health (GH) scale (-8.9; 95% CI, -15.0 to 2.7, P = .005). Mental health, education level, employment status, marital status, living arrangements, and birth rate were compatible with normal living patterns. The development of GVHD and older age at transplantation were important impairing factors. Additional analyses performed to evaluate HRQoL in an age-sex-matched cohort of 124 patients receiving conventional treatment of β-thalassemia revealed poorer outcomes compared with the cohort of transplanted patients. © 2013 by The American Society of Hematology.

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