Studying First Line Treatment of Chronic Myeloid Leukemia (CML) in a Real-world Setting

Study Overview
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Brief Summary
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The purpose of this study is to better understand the use of tyrosine kinase inhibitors (TKI) in patients newly diagnosed with CML and their quality of life in a real-world setting.

Detailed Description
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Time Perspective : Most patients are expected to be a mix of retrospective and prospective data collection. Patients can be enrolled after their study index date (retrospective component) and have to be followed until 5 years from study index date are complete (time between enrollment and 5 year follow-up is the prospective component)

Official Title
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Studying Interventions for Managing Patients With Chronic Myeloid Leukemia (CML) in Chronic Phase: The 5-Year Prospective Cohort Study (SIMPLICITY)

Conditions
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Chronic Myeloid Leukemia

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Newly-diagnosed chronic phase chronic myeloid leukemia (CP-CML) patients who started their first-line Tyrosine Kinase Inhibitor (TKI) treatment on imatinib, dasatinib or nilotinib in accordance with the timelines below 18 years or older at time of of CP-CML diagnosis a) Imatinib Cohorts Patients who started their first-line Imatinib treatment between January 2, 2008 and September 30, 2010.Patients fitting this criterion are defined as retrospective Imatinib patients Patients who started their first-line Imatinib treatment on or after October 1, 2010 b) Dasatinib Cohort Patients who started their first-line Dasatinib treatment after the drug was approved in this indication c) Nilotinib Cohort Patients who started their first-line Nilotinib treatment after the drug was approved in this indication Patients are also eligible when they have already switched to a subsequent therapy (TKI or other) at the time of enrollment, as long as their first-line and subsequent CML treatment information is available at site for data entry into the study Electronic Case Report Form (eCRF) Receiving treatment at medical practice (eg. community-based, office-based, hospital-based, academic setting, oncology center) Exclusion Criteria: Patients who are participating in an interventional trial which may influence the management of their CML disease will be excluded Discontinuation Criteria: Enrolled patients who join an interventional trial which may influence the management of their CML disease will be excluded at the time of entry into the interventional trial

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 |
| --- | --- |
| First line TKI treatment: Imatinib<br>Diagnosed CML patients who receive first line TKI treatment: Imatinib | |
| First line TKI treatment: Nilotinib<br>Diagnosed CML patients who receive first line TKI treatment: Nilotinib | |
| First line TKI treatment: Dasatinib<br>Diagnosed CML patients who receive first line TKI treatment: Dasatinib | |
| Imatinib treated patients<br>Imatinib treated patients if their study index date is between January 2, 2008 and September 30, 2010 | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The rate of Complete Cytogenetic Response |  | 12 months | 
| The duration of initial TKI treatment | Initiation of first-line TKI, (whether Dasatinib, Imatinib, Nilotinib) | 5-years from study index date | 
| The rate of discontinuation and treatment changes after initial TKI treatment | Dates of switches in therapy from initial TKI treatment, Reasons for treatment discontinuation (i.e. side effects, mutations, etc.), Subsequent lines of CML treatments (start and stop dates) | Every 6 months for a follow-up period of 5-years from study index date | 
| The rate of best response to therapy (i.e. hematologic, cytogenetic, molecular response) | Results and dates of: all bone marrow aspirates, blood tests, cytogenetics, Polymerase Chain Reaction (PCR), Fluorescent In-Situ Hybridization (FISH), and Physical exam | Every 6 months for a follow-up period of 5-years from study index date | 
| The adherence to treatment | Morisky Medication Adherence Scale - 8 Items is a validated self-reported measure of medication adherence. | Every 6 months for a follow-up period of 5-years from study index date | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Impact of first-line treatment options on quality of life | Questionnaires used for assessment: Functional Assessment of Cancer Therapy - General (FACT-G), Cancer Therapy Satisfaction Questionnaire (CTSQ), MD Anderson Symptom Inventory - CML (MDASI-CML). | Every 6 months | 
| Non-hematologic side effects from treatment affecting patient quality of life and outcomes | Treatment discontinuations and changes | Every 6 months | 
| Patient satisfaction with CML treatment | Cancer Therapy Satisfaction Questionnaire (CTSQ) | Every 6 months | 
| Patterns of disease monitoring as observed in a real-world setting | MD Anderson Symptom Inventory - CML (MDASI-CML) Questionnaire - disease-specific module of the MDASI7 which is a brief measure of severity and impact of cancer-related symptoms on daily function | Every 6 months | 
| Resource utilization associated with CML management | To evaluate healthcare resource utilization, descriptive statistics will describe real-world disease monitoring patterns, frequency of testing, and resources used for disease management for each treatment cohort. | Every 6 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Chronic Phase - Chronic Myeloid Leukemia