Italian Group for Adult Hematologic Diseases GIMEMA

Rome, Italy

Italian Group for Adult Hematologic Diseases GIMEMA

Rome, Italy
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Efficace F.,Italian Group for Adult Hematologic Diseases GIMEMA | Gaidano G.,University of Piemonte Orientale | Lo-Coco F.,University of Rome Tor Vergata
Blood | Year: 2017

In less than 2 decades, major clinical advances have been made in various areas of hematologic malignancies. Clinicians and patients now frequently face challenging choices regarding various treatments that are often similar in regard to safety or clinical effectiveness; hence, medical decision making has grown in complexity. For example, several novel drugs have been developed as oral agents, introducing an additional challenge in patient management, such as ensuring an optimal adherence to therapy in order to maximize drug effectiveness. This rapidly changing scenario provides a rationale for a more systematic collection of patient-reported outcomes (PRO) in clinical research and routine care. In the former case, PRO may help to better understand overall treatment effectiveness of a new drug being tested. In the latter case, it may aid in making more informed, individualized treatment decisions in daily practice by obtaining more accurate information on the actual symptom burden experienced by the patient. In any case, evaluating PRO requires making several, and often challenging, decisions depending, for example, on the population being studied and the specific setting. Therefore, if PROs are to fulfill their potential of generating clinically meaningful data that robustly inform patient care, special attention should be given to methodological rigor. We outline the value of a more systematic and rigorous implementation of PRO assessment in the current hematology arena, by providing some real world examples of how PRO data have contributed in better understanding the value of new therapies. We also discuss practical considerations in PRO assessment in clinical research. © 2017 by The American Society of Hematology.

Efficace F.,Italian Group for Adult Hematologic Diseases GIMEMA | Gaidano G.,University of Piemonte Orientale | Breccia M.,University of Rome La Sapienza | Voso M.T.,University Of Rome Cattolica ore | And 17 more authors.
The Lancet Oncology | Year: 2015

Background: The clinical presentation of myelodysplastic syndromes is highly variable and so accurate prediction of outcomes in these patients is crucial. We aimed to assess whether self-reported fatigue severity predicts overall survival beyond gold-standard prognostic indices in patients with higher-risk myelodysplastic syndromes. Methods: We did a multicentre, prospective, observational, cohort study of patients from 37 centres in Europe, USA, and east Asia. Adults (≥18 years) with myelodysplastic syndromes were consecutively enrolled within 6 months of diagnosis with an intermediate-2-risk or high-risk score according to the International Prognostic Scoring System (IPSS). Patients were enrolled irrespective of older age, comorbidities, performance status, and progression from a lower IPSS risk score category. All patients had to complete a quality of life assessment at baseline. With use of univariate and then multivariate Cox proportional hazards regression analysis, we constructed a multivariate model of how prognostic variables, including IPSS and fatigue score from the European Organisation for Research and Treatment of Cancer quality-of-life questionnaire-core 30, predicted overall survival. The primary endpoint was overall survival by baseline self-reported fatigue scale ratings. This study was registered with, number NCT00809575. Findings: Between Nov 10, 2008, and Aug 13, 2012, we enrolled 280 patients with a median age of 71 years (IQR 64-77). The median follow-up was 15 months (IQR 8-27), and the last patient was assessed Feb 16, 2015. The median overall survival from diagnosis was 17 months (95% CI 15-19). In univariate analysis, the baseline factors that were significantly associated with reduced overall survival were increasing age, transfusion dependency (defined as having received at least one red blood cell transfusion every 8 weeks over a period of 4 months), Eastern Cooperative Oncology Group (ECOG) performance status of two or more, increased white blood cell count, high-risk IPSS score, and higher self-reported fatigue severity. In multivariate analysis, baseline factors independently associated with reduced overall survival were high-risk IPSS score (hazard ratio [HR] 2·525, 95% CI 1·357-4·697; p=0·0035) and a higher score for fatigue (1·110, 1·040-1·170, for every ten points of fatigue deterioration; p=0·0007). In further multivariate models for survival, including either the WHO-based prognostic scoring system or the revised version of the IPSS classification, fatigue remained a statistically significant independent prognostic factor with a HR of 1·120 (1·050-1·180, p=0.0003) and a HR of 1·130 (1·060-1·190, p=0·0002), respectively. Interpretation: In patients with newly diagnosed higher-risk myelodysplastic syndromes, self-reported fatigue severity provides prognostic information for survival independent from gold-standard risk classifications. Our findings suggest that fatigue assessment should be included in routine diagnostic investigation for these patients and considered as a standard baseline stratification factor in future randomised controlled trials. Funding: Associazione Italiana contro le Leucemie, Linfomi e Mieloma (AIL). © 2015 Elsevier Ltd.

Efficace F.,Italian Group for Adult Hematologic Diseases GIMEMA | Gaidano G.,University of Piemonte Orientale | Sprangers M.,University of Amsterdam | Cottone F.,Italian Group for Adult Hematologic Diseases GIMEMA | And 10 more authors.
Annals of Oncology | Year: 2014

Background: The main objective of this study was to assess preferences for involvement in treatment decisions and requests for prognostic information in newly diagnosed higher-risk myelodysplastic syndrome (MDS) patients. Patient and methods: This was a prospective cohort observational study that consecutively enrolled MDS patients with an international prognostic scoring system (IPSS) risk category of intermediate-2 or high risk (summarized as 'higher risk'). The control preference scale was used to assess patient preferences for involvement in treatment decisions, and whether a request by patients for prognostic information during consultation was made, was also recorded. All of the patients were surveyed at the time of diagnosis before receiving treatment. Univariate and multivariate analyses were carried out to assess how sociodemographic, clinical and laboratory data related to decision-making preferences and requests for prognostic information. Relationship with the health-related quality of life (HRQOL) profile was also examined. Results: A total of 280 patients were enrolled, 74% with intermediate-2 and 26% with high-risk IPSS. The mean age of patients was 70-year old (range: 32-89 years). One hundred thirty-two patients (47%) favored a passive role in treatment decision-making, whereas only 14% favored an active role. The remaining 39% of patients favored a shared decisionmaking approach. Patients with lower hemoglobin levels were more likely to prefer a passive role (P = 0.037). HRQOL was generally better in patients preferring an active role versus those preferring a passive one. Overall, 61% (N = 171) of patients requested prognostic information on survival during consultation. The likelihood of not requesting prognostic information was higher for older patients (P = 0.003) and for those with lower education (P = 0.010). Conclusion: Decision-making preferences vary among patients with newly diagnosed higher-risk MDS. Current findings suggest that patients with worse underlying health conditions are more likely to prefer less involvement in treatment decisions. © The Author 2014. Published by Oxford University Press on behalf of the European Society for Medical Oncology. All rights reserved.

Weis J.,Albert Ludwigs University of Freiburg | Arraras J.I.,Hospital Of Navarra | Conroy T.,Center Alexis Vautrin | Efficace F.,Italian Group for Adult Hematologic Diseases GIMEMA | And 10 more authors.
Psycho-Oncology | Year: 2013

Background European Organisation for Research and Treatment of Cancer (EORTC) has developed a new multidimensional instrument measuring cancer-related fatigue that can be used in conjunction with the quality of life core questionnaire, EORTC QLQ-C30. The paper focuses on the development of the phase III module, collaborating with seven European countries, including a patient sample of 318 patients. Methods The methodology followed the EORTC guidelines for developing phase III modules. Patients were assessed by questionnaires (EORTC QLQ-C30 with the EORTC Fatigue Module FA15) followed by an interview, asking for their opinions on the difficulty in understanding, on annoyance and on intrusiveness Results The phase II FA15 was revised on the basis of qualitative analyses (comments of the patients), quantitative results (descriptive statistics) as well as the multi-item response theory analyses. The three dimensions (physical, emotional and cognitive) of the scale could be confirmed Conclusions As a result, EORTC QLQ-FA13 is now available as a valid phase III module measuring cancer-related fatigue in clinical trials and will be psychometrically improved in the upcoming phase IV. Copyright © 2012 John Wiley & Sons, Ltd.

PubMed | University of Leeds, Sharp Corporation, Medical Products Agency, Italian Group for Adult Hematologic Diseases GIMEMA and 5 more.
Type: Journal Article | Journal: Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation | Year: 2016

In 2014, the European Medicines Agency (EMA) released for comment a draft reflection paper on the use of patient-reported outcome (PRO) measures in oncology studies. A twelve-member International Society for Quality of Life Research (ISOQOL) taskforce was convened to coordinate the ISOQOL response. Twenty-one ISOQOL members provided detailed comments and suggestions on the paper: 81% from academia and 19% from industry. Taskforce members consolidated and further refined these comments and shared the recommendations with the wider ISOQOL membership. A final response was submitted to the EMA in November 2014. The impending publication of the EMA reflection paper presents a valuable opportunity for ISOQOL to comment on the current direction of EMA PRO guidance and strategy. The EMA paper, although focused on cancer, could serve as a model for using PROs in other conditions, as it provides a useful update surrounding some of the design issues common to all trial research including PRO endpoints. However, we believe there are a number of additional areas in need of greater consideration. The purpose of this commentary is therefore to highlight the strengths of this timely and potentially useful document, but also to outline areas that may warrant further discussion.

Efficace F.,Italian Group for Adult Hematologic Diseases GIMEMA | Baccarani M.,University of Bologna | Rosti G.,University of Bologna | Cottone F.,Italian Group for Adult Hematologic Diseases GIMEMA | And 11 more authors.
British Journal of Cancer | Year: 2012

Background: Optimal adherence to imatinib therapy is of paramount importance to maximise treatment effectiveness in patients with chronic myeloid leukaemia (CML). The main objective of this study was to investigate patient-reported personal factors associated with adherence behaviour. Methods :Analysis was conducted on 413 CML patients receiving long-term therapy with imatinib. Adherence behaviour was measured with the Morisky Medication Adherence Scale and personal factors investigated included: quality of life, perceived social support, fatigue, symptom burden, psychological wellbeing and desire for additional information. Key socio-demographic and treatment-related factors were also taken into account. Univariate and multivariate logistic regression analyses were used to investigate factors associated with optimal adherence to therapy.Results:In all, 53% of patients reported an optimal adherence behaviour. The final multivariate model retained the following variables as independent predictors of optimal adherence to therapy: desire for more information (ref. no), odds ratio (OR)0.43 (95% confidence interval (CI), 0.29-0.66; P<0.001), social support (higher score representing greater support), OR1.29 (95% CI, 1.11-1.49; P<0.001) and concomitant drug burden (ref. no), OR1.82 (95% CI, 1.18-2.80; P0.006). Conclusion :This study suggests that a higher level of social support, satisfaction with information received and concomitant drug burden are the main factors associated with greater adherence to long-term imatinib therapy. © 2012 Cancer Research UK All rights reserved.

PubMed | Ospedali Riuniti, Clinica Ematologica, Azienda Ospedaliero Universitaria di Udine, University of Turin and 10 more.
Type: Journal Article | Journal: Annals of hematology | Year: 2016

The primary objective of this study was to investigate whether the presence of comorbidities was associated with a lower health-related quality of life (HRQOL) in elderly patients with chronic myeloid leukemia (CML). A sample of 174 CML patients aged 60years or above was analyzed. HRQOL was assessed with the Medical Outcomes Study 36-Item Short-Form Health Survey (SF-36). A number of pre-selected sociodemographic and disease-related factors were considered as potential confounding factors for the association between comorbidity and HRQOL. Mean age of the 174 patients analyzed was 70years (range 60-87years) and 55% were male. Overall, 111 patients (64%) reported at least one comorbidity. Analysis stratified by age group category showed a greater proportion of patients with comorbidities in the older sub-group population (70years) compared to younger patients (60 to 69years). Differences in HRQOL outcomes between patients with no comorbidity at all and those with two or more comorbid conditions were at least twice the magnitude of a clinically meaningful difference in all the physical and mental health scales of the SF-36. In multivariate analysis, after adjusting for key confounding factors, the following scales were significantly lower in those with comorbidity: general health (p<0.001), bodily pain (p<0.001), physical functioning (p=0.002), and vitality (p=0.002). Assessing comorbidity in elderly patients with CML is important to facilitate identification of those most in need of HRQOL improvements.

Efficace F.,Italian Group for Adult Hematologic Diseases GIMEMA | Cocks K.,University of Leeds | Breccia M.,University of Rome La Sapienza | Sprangers M.,University of Amsterdam | And 4 more authors.
Critical Reviews in Oncology/Hematology | Year: 2012

Health-related quality of life (HRQOL) and other patient-reported outcomes (PROs) might be crucial in comparing effectiveness of treatments as they could provide invaluable information to better inform clinical decision-making. This is particularly true in the era of targeted therapies (TT). A systematic review was undertaken on all studies with CML patients published from 1980 to 2010 and including a PRO evaluation. Out of 619 articles scrutinized, 15 met eligibility criteria and no study was published before 1995. Six dealt mainly with interferon-based therapies, 7 with bone marrow transplantation and only 2 evaluated PROs in the context of TT. No disease-specific, validated PRO instrument for these patients was found. The main evidence being that Imatinib provides clear advantage in terms of HRQOL over interferon-based treatments. There is lack of data concerning PROs in patients treated with current TT. Documenting HRQOL and side effects of CML treatments, from the patients' perspective is needed to evaluate overall treatment effectiveness and net clinical benefit of newer therapeutic strategies. © 2011 Elsevier Ireland Ltd.

Efficace F.,Italian Group for Adult Hematologic Diseases GIMEMA | Rosti G.,University of Bologna | Aaronson N.,Netherlands Cancer Institute | Cottone F.,Italian Group for Adult Hematologic Diseases GIMEMA | And 5 more authors.
Haematologica | Year: 2014

The main objective of this study was to compare the reporting of health status and symptom severity, for a set of core symptoms related to imatinib therapy, between chronic myeloid leukemia patients and their treating physicians. Patients were asked to complete a questionnaire including questions on symptom severity and health status. The symptoms assessed were: abdominal discomfort, diarrhea, edema, fatigue, headache, muscle cramps, musculoskeletal pain, nausea and skin problems. The physicians were asked to complete a questionnaire for each of their patients entering the study. Four hundred twenty-two patients were included in the study. All respective paired physicians (n=29) completed the questionnaire, and thus the analyses are based on 422 patient-physician dyads. Agreement on symptom ratings ranged from 34% (for muscle cramps) to 66% (for nausea). For all symptoms, patients reported higher severity more often than their physicians. The three symptoms whose severity was most frequently underestimated by physicians were fatigue (51%), muscle cramps (49%) and musculoskeletal pain (42%). Health status was overestimated by physicians in 67% of the cases. Physicians and their patients with chronic myeloid leukemia often disagree in their ratings of the patients' symptom severity. Most typically, physicians tend to underestimate symptom severity and overestimate the overall health status of their patients. Current findings support the use of patient-reported outcome measures as a possible means to enhance the management of patients with chronic myeloid leukemia. © Ferrata Storti Foundation.

Efficace F.,Italian Group for Adult Hematologic Diseases GIMEMA | Cardoni A.,Italian Group for Adult Hematologic Diseases GIMEMA | Cottone F.,Italian Group for Adult Hematologic Diseases GIMEMA | Vignetti M.,Italian Group for Adult Hematologic Diseases GIMEMA | Mandelli F.,Italian Group for Adult Hematologic Diseases GIMEMA
Leukemia Research | Year: 2013

The main objective of this systematic review is to quantify and to summarize all studies that have included health-related quality of life (HRQOL) or, any other type of patient-reported outcomes (PROs), in patients with chronic myeloid leukemia (CML) treated with tyrosine kinase inhibitors (TKIs). Nine papers were found and none of these were published before 2003. Overall, 3290 CML patients were enrolled in the studies reviewed. Four studies reported HRQOL data on patients treated with imatinib only. The most solid data in this area indicate that CML patients receiving TKIs have a worse HRQOL profile when compared to their peers, without cancer, in the general population and interventions to improve HRQOL outcomes are thus needed. Our review revealed the paucity of evidence-based data in this area. However, HRQOL assessment in these studies emphasize the unique information provided by the patient's perspective. Urgent efforts are needed to provide solid PROs data to complement current knowledge on clinical efficacy of TKIs. © 2012 Elsevier Ltd.

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