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      Health-Related Quality of Life, Treatment Satisfaction, Adherence and Persistence in β -Thalassemia and Myelodysplastic Syndrome Patients with Iron Overload Receiving Deferasirox: Results from the EPIC Clinical Trial

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          Abstract

          Treatment of iron overload using deferoxamine (DFO) is associated with significant deficits in patients' health-related quality of life (HRQOL) and low treatment satisfaction. The current article presents patient-reported HRQOL, satisfaction, adherence, and persistence data from β -thalassemia ( n = 274) and myelodysplastic syndrome (MDS) patients ( n = 168) patients participating in the Evaluation of Patients' Iron Chelation with Exjade (EPIC) study (NCT00171821); a large-scale 1-year, phase IIIb study investigating the efficacy and safety of the once-daily oral iron chelator, deferasirox. HRQOL and satisfaction, adherence, and persistence to iron chelation therapy (ICT) data were collected at baseline and end of study using the Medical Outcomes Short-Form 36-item Health Survey (SF-36v2) and the Satisfaction with ICT Questionnaire (SICT). Compared to age-matched norms, β -thalassemia and MDS patients reported lower SF-36 domain scores at baseline. Low levels of treatment satisfaction, adherence, and persistence were also observed. HRQOL improved following treatment with deferasirox, particularly among β -thalassemia patients. Furthermore, patients reported high levels of satisfaction with deferasirox at end of study and greater ICT adherence, and persistence. Findings suggest deferasirox improves HRQOL, treatment satisfaction, adherence, and persistence with ICT in β -thalassemia and MDS patients. Improving such outcomes is an important long-term goal for patients with iron overload.

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          Further evidence supporting an SEM-based criterion for identifying meaningful intra-individual changes in health-related quality of life.

          This study used the standard error of measurement (SEM) to evaluate intra-individual change on both the Chronic Respiratory Disease Questionnaire (CRQ) and the SF-36. After analyzing the reliability and validity of both instruments at baseline among 471 COPD outpatients, the SEM was compared to established minimal clinically important difference (MCID) standards for three CRQ dimensions. A value of one SEM closely approximated the MCID standards for all CRQ dimensions. This SEM-based criterion was then validated by cross-classifying the change status (improved, stable, or declined) of 393 follow-up outpatients using the one-SEM criterion and the MCID standard. Excellent agreement was achieved for all three CRQ dimensions. Although MCID standards have not been established for the SF-36, the one-SEM criterion was explored in these change scores. Among SF-36 scales demonstrating acceptable reliability and reasonable variance, the percent of individuals within each change category was consistent with those seen in the CRQ dimensions. These results replicate previous findings where a value of one SEM also closely approximated MCIDs for all dimensions of the Chronic Heart Disease Questionnaire among cardiovascular outpatients. The one-SEM criterion should be explored in other health-related quality of life instruments with established MCIDs.
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            Time-dependent prognostic scoring system for predicting survival and leukemic evolution in myelodysplastic syndromes.

            The aims of this study were to identify the most significant prognostic factors in myelodysplastic syndromes (MDS) taking into account both their values at clinical onset and their changes in time and to develop a dynamic model for predicting survival and leukemic evolution that can be applied at any time during the course of the disease. We studied a learning cohort of 426 MDS patients diagnosed at the Department of Hematology, San Matteo Hospital, Pavia, Italy, between 1992 and 2004, and a validation cohort of 739 patients diagnosed at the Heinrich-Heine-University Hospital, Düsseldorf, Germany, between 1982 and 2003. All patients were reclassified according to WHO criteria. Univariable and multivariable analyses were performed using Cox models with time-dependent covariates. The most important variables for the prognostic model were WHO subgroups, karyotype, and transfusion requirement. We defined a WHO classification-based prognostic scoring system (WPSS) that was able to classify patients into five risk groups showing different survivals (median survival from 12 to 103 months) and probabilities of leukemic evolution (P < .001). WPSS was shown to predict survival and leukemia progression at any time during follow-up (P < .001), and its prognostic value was confirmed in the validation cohort. WPSS is a dynamic prognostic scoring system that provides an accurate prediction of survival and risk of leukemic evolution in MDS patients at any time during the course of their disease. This time-dependent system seems particularly useful in lower risk patients and may be used for implementing risk-adapted treatment strategies.
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              Prognostic factors and life expectancy in myelodysplastic syndromes classified according to WHO criteria: a basis for clinical decision making.

              The aim of this study was to evaluate the prognostic value of the WHO proposal, to assess the role of the main prognostic factors in myelodysplastic syndromes (MDSs) classified into WHO subgroups, and to estimate mortality (standardized mortality ratio [SMR]) and life expectancy in these groups as a basis for clinical decision making. Four hundred sixty-seven patients who were diagnosed as having de novo MDS at the Division of Hematology, University of Pavia (Pavia, Italy), between 1992 and 2002, were evaluated retrospectively for clinical and hematologic features at diagnosis, overall survival (OS), and progression to leukemia (leukemia-free survival). Significant differences in survival were noted between patients with refractory anemia (RA), refractory cytopenia with multilineage dysplasia, RA with excess blasts, type 1 (RAEB-1), and RAEB-2. The effect of demographic factors on OS was observed in MDS patients without excess blasts (age, P = .001; sex, P = .006), as in the general population. The mortality of RA patients 70 years or older did not differ significantly from that of the general population (SMR, 1.62; P = .06). Cytogenetics was the only International Prognostic Scoring System variable showing a prognostic value in MDS classified into WHO subgroups. Transfusion-dependent patients had a significantly shorter survival than patients who did not require transfusions (P < .001). Developing a secondary iron overload significantly affected the survival of transfusion-dependent patients (P = .003). These data show that the WHO classification of MDSs has a relevant prognostic value. This classification, along with cytogenetics, might be useful in decisions regarding transplantation. MDS with isolated erythroid lineage dysplasia identifies a subset of truly low-risk patients, for whom a conservative approach is advisable.
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                Author and article information

                Journal
                Anemia
                Anemia
                ANE
                Anemia
                Hindawi Publishing Corporation
                2090-1267
                2090-1275
                2012
                12 August 2012
                : 2012
                : 297641
                Affiliations
                1Department of Haematology, UCL Cancer Institute, University College London, Paul O'Gorman Building, 72 Huntley Street, London WC1E 6BT, UK
                2Monash Medical Centre, Melbourne, VIC 3168, Australia
                3Thalassemia Clinical Care Services Unit, Hippokration General Hospital Thessaloniki, Egnatia Street 106, 54622 Thessaloniki, Greece
                4Hematology, Hopital Edouard Herriot, 6 Rue Antoine Lumiere, 69008 Lyon, France
                5Medizinische Hochschule Hannover (MHH), Department of Hematology, Hemostasis, Oncology and Stem Cell Transplantation, Carl-Neuberg Strasse 1, 30625 Hannover, Germany
                6Novartis Pharmaceutical Corporation, 180 Park Avenue, 105-3E065, Florham Park, NJ 07932-1080, USA
                7Novartis Pharma AG Postfach, 4002 Basel, Switzerland
                8Adelphi Values, Adelphi Mill, Grimshaw Lane, Bollington, Cheshire SK10 5JB, UK
                9Universita di Milano, Can Granda Foundation IRCCS, Via F. Sforza 35, 20122 Milan, Italy
                Author notes

                Academic Editor: Sezaneh Haghpanah

                Article
                10.1155/2012/297641
                3424665
                22924125
                1579e9d8-18ee-4511-afe7-df916750323e
                Copyright © 2012 John Porter et al.

                This is an open access article distributed under the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

                History
                : 20 January 2012
                : 1 May 2012
                : 19 May 2012
                Categories
                Research Article

                Hematology
                Hematology

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