Patient Reported Outcomes in Chronic Myeloid Leukemia

Study Overview
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Brief Summary
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Although the impact of disease and treatment related burden on health-related quality of life (HRQOL) in patients with solid tumours has been well studied, with several clinical trials that included HRQOL as an endpoint, the general understanding in patients with Chronic Myeloid Leukaemia (CML) is lacking in comparison. The literature shows that patients' perspective is unique and should always be measured with methodologically sound instruments that are devised for this purpose. The main scope of this project is develop to an international validated questionnaire for the purpose of HRQOL assessment; such a tool will then be used to provide important data, from the patients' perspective, to make more informed treatment decisions.

Detailed Description
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The development process will follow the international guidelines published by the EORTC Quality of Life Group (QLG). This process will follow three main phases. Phase 1 of will deal with the review of the literature, identification of an item list and interviews with experts in the treatment of CML and CML patients (undergoing all possible treatments). In Phase II the identified issues from Phase I will be worded and translated into possible items for the provisional questionnaire using existing EORTC questionnaires and the EORTC QLG Item Bank. In the Phase III the provisional CML questionnaire will be pre-tested by to a larger sample of CML patients (not the same patients involved in previous phases) and individual structured interviews will be conducted as well. These latter will identify questions that might be irrelevant or whether there will be additional issues not previously included. Overall up to 480 patients will be enrolled in this study (up to 180 in phase I and up to 300 in phase III).

Official Title
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Patient Reported Outcomes in Chronic Myeloid Leukemia. GIMEMA QoL - CML0310.

Conditions
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Chronic Myeloid Leukemia

Intervention / Treatment
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* Other: HRQOL questionnaire


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Adult patients (18 years of age or older) Confirmed diagnosis of Philadelphia chromosome positive CML Informed consent provided Patients enrolled in investigational treatment trials are eligible Ability to speak and read language of the Questionnaire Exclusion Criteria: Freedom from overt cognitive impairment or major psychiatric conditions that may confound HRQOL evaluation

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Adults with CML<br>Adult patients (18 years of age or older) with confirmed diagnosis of CML | Other: HRQOL questionnaire<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Development of an EORTC questionnaire to assess HRQOL of patients with CML. |  | By the end of the study. | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Comparison between physicians' perception of relevance of HRQOL issues with that of patients. |  | By the end of the study. | 
| Development of an EORTC CML symptom checklist. |  | By the end of the study. | 
| HRQOL in CML patients undergoing 2nd line treatment with TKIs. |  | By the end of the study. | 
| Decision making process for choosing between different 2nd line treatments with TKIs. |  | By the end of the study. | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Chronic Myeloid Leukemia, Quality of Life, Symptoms, Patient-Reported outcomes, Questionnaire