International development of an EORTC questionnaire for assessing health-related quality of life in chronic myeloid leukemia patients: the EORTC QLQ-CML24

The treatment policy of chronic myeloid leukemia (CML), particularly with tyrosine kinase inhibitors, has been influenced by several recent studies that were well designed and rapidly performed, but their interpretation is of some concern because different end points and methodologies were used. To understand and compare the results of the previous and future studies and to translate their conclusion into clinical practice, there is a need for common definitions and methods for analyses of CML studies. A panel of experts was appointed by the European LeukemiaNet with the aim of developing a set of definitions and recommendations to be used in design, analyses, and reporting of phase 3 clinical trials in this disease. IntroductionApplied statistics are important tools in medical evaluations. The relevance of statistical designs and statistical results in trials is based on concise definitions regarding diagnosis, management, and treatment strategies. The choice of adequate statistical tests depends on the parameters to be analyzed and on specific end points.After the initial descriptions of chronic myeloid leukemia (CML) more than 160 years ago, little progress was made in its treatment for more than a century. 1 Survival prolongation was first achieved with drugs, such as hydroxyurea. 1 Then, major improvements were obtained with allogeneic hematopoietic stem cell transplantation. First priority at that time was to analyze survival.The understanding of the pathogenesis of CML began with the discovery of the Philadelphia (Ph) chromosome followed later on by the recognition of its molecular counterpart, the BCR-ABL fusion gene. In CML, clinical trials performed during the past 2 to 3 decades have profoundly improved the outcome of patients with CML. 2 In Europe, the coordinators of national CML Study Groups (European Investigators on CML) joined forces to perform European trials and long-term observations, 3 to conduct common meta-analyses, 4,5 and to elaborate new prognostic scores. 6,7 With the advents of IFN-␣ and of tyrosine kinase inhibitors (TKIs), analyses of treatment response moved more into focus.Thus, the relationship between responses and survival became of particular interest. The cytogenetic outcome is usually considered as a reliable surrogate marker for survival in CML, 7,8 whereas the relationship between molecular response and survival is still under investigation. 9 Molecular response is probably more relevant to the issue of treatment discontinuation without relapse and cure. 10 On the other hand, because of the recent improvement of survival offered by the use of TKIs, other diseases and deaths from causes other than CML progression have become more frequent in CML patients. Analysis of survival may include these new parameters. Quality of life and compliance have also to be considered in the context of this chronic disease with an expected long duration of treatment.Consequently, statistical methods in addition to Kaplan-Meier (KM) 11 analyses and initial Cox regression models 12 should be a...

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“…A crosscultural initial development of an EORTC health-related quality of life questionnaire for patients with CML is ongoing. 40 …”

Section: Quality Of Lifementioning

confidence: 99%

Definitions, methodological and statistical issues for phase 3 clinical trials in chronic myeloid leukemia: a proposal by the European LeukemiaNet

Guilhot

Baccarani

Clark

et al. 2012

Blood

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“…Early-phase development of 2 other patient-reported scales to measure symptoms in CML have been reported in abstract form: the Comprehensive Symptom Profile for CML 24 and a European Organization for Research and Treatment of Cancer (EORTC) CML questionnaire. 25 Because few studies have followed health-related QOL longitudinally in patients with CML, 9,11,26 and no study has longitudinally compared symptoms related to various TKIs, our secondary goal was to explore the occurrence of symptoms reported by patients with CML over a 1-year period.…”

Section: Introductionmentioning

confidence: 99%

Measuring the symptom burden associated with the treatment of chronic myeloid leukemia

Williams¹,

Gonzalez²,

Ault

et al. 2013

Blood

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• The MD Anderson SymptomInventory for CML can be used to collect patientreported symptoms for research and clinical practice.• Thirty percent of patients in chronic-phase CML and on TKIs experience moderate symptoms that interfere with daily functioning.We developed a module of the MD Anderson Symptom Inventory (MDASI) for patients with chronic myeloid leukemia (CML). To develop the MDASI-CML, we identified CMLspecific symptoms from qualitative interviews with 35 patients. A list of candidate symptoms was reduced by a panel of patients, caregivers, and clinicians to the 13 core MDASI symptom items and 6 CML-specific items; these items were subsequently administered to 30 patients. Cognitive debriefing confirmed that the items were clear, relevant, and easy to use. One additional CML-specific symptom item was added, for a total of 7. The refined MDASI-CML was administered to 152 patients once every 2 weeks for 1 year. The content, concurrent, known-group, and construct validity of the MDASI-CML were evaluated. The internal consistency and test-retest reliabilities of the module were adequate. Longitudinal analysis showed relatively stable symptom severity scores over time. The most severe symptoms were fatigue, drowsiness, disturbed sleep, muscle soreness and cramping, and trouble remembering things. Approximately one-third of the patients who completed the MDASI-CML reported persistent moderate-to-severe symptoms. The MDASI-CML is a valid and reliable symptom assessment instrument that can be used in clinical studies of symptom status in patients with CML. This trial was registered at www.clinicaltrials.gov as #NCT01046305. (Blood. 2013;122(5):641-647)

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“…Recently, 2 CML-specific questionnaires, the EORTC QLQ-CML24 (European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Chronic Myeloid Leukemia 24) 30 and the MDASI-CML (MD Anderson Symptom Inventory Chronic Myeloid Leukemia Module), 31 have been published. The EORTC QLQ-CML24 has been tested in 655 CML patients in treatment with various TKIs from 10 different countries (Europe, the United States, and Asia) and has shown satisfactory validity and applicability across multiple languages and cultures.…”

Section: How Should We Measure Pros In CML Clinical Research?mentioning

confidence: 99%

The value of quality of life assessment in chronic myeloid leukemia patients receiving tyrosine kinase inhibitors

Efficace

Cannella

2016

Hematology

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The development of the oral tyrosine kinase inhibitors (TKIs) to treat chronic myeloid leukemia (CML) is one of the great triumphs of cancer research. Although the efficacy of TKIs has dramatically improved the disease-specific overall survival rate, the prevalence of CML is increasing worldwide. Currently, CML patients receive prolonged (even lifelong) treatment, and over the last decade, clinical decision making has become challenging. Therefore, consideration of the effects of TKI therapies on patients' quality of life (QoL) and symptom burden (ie, patient-reported outcomes [PROs]) is now critical to more robustly inform patient care and improve health care quality. Over the last 5 years, a number of studies have generated valuable PRO data, for example, on long-term QoL effects of imatinib therapy or symptom burden of patients switching from imatinib to second-generation TKIs. PRO findings are important, as they provide a unique patient perspective on the burden of the disease and treatments effects. We will review main evidence-based data on the use of PROs in clinical research and highlight the importance of methodological rigor of PRO assessment. Also, we will describe the potential value of using PRO assessment in routine clinical practice, for example, to facilitate timely management of side effects. Areas for future research will also be discussed. Learning Objectives• List main evidence-based data on the impact of tyrosine kinase inhibitors on patients' quality of life and symptom burden • Describe the value of quality of life assessment in CML clinical research • Illustrate the importance of integrating quality of life assessment into routine practice and the relationship between quality of life assessment and adherence to therapy

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International development of an EORTC questionnaire for assessing health-related quality of life in chronic myeloid leukemia patients: the EORTC QLQ-CML24

Abstract: The EORTC QLQ-CML24 is an internationally developed HRQOL questionnaire for CML patients, and its implementation in clinical research and practice can provide important information to facilitate clinical decision-making.

Cited by 70 publications

References 22 publications

Definitions, methodological and statistical issues for phase 3 clinical trials in chronic myeloid leukemia: a proposal by the European LeukemiaNet

Definitions, methodological and statistical issues for phase 3 clinical trials in chronic myeloid leukemia: a proposal by the European LeukemiaNet

Measuring the symptom burden associated with the treatment of chronic myeloid leukemia

The value of quality of life assessment in chronic myeloid leukemia patients receiving tyrosine kinase inhibitors

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