The aim of this study is to assess demographics, clinical features, treatment patterns, and the comorbidity burden and its impact on CML patients in the real-world clinical setting in Kuwait. Adult patients with Philadelphia positive-chromosome (Ph+ve) CML who have received at least one line of tyrosine kinase inhibitor (TKI) treatment, such as but not limited to imatinib, dasatinib, nilotinib, bosutinib, ponatinib, and asciminib will be included. The study will use data from the hospital records of CML patients between January 2014 and January 2024.
Study Type
OBSERVATIONAL
Enrollment
400
Number of Patients by Demographic Category
Demographics include gender and ethnicity.
Time frame: Baseline
Age at Diagnosis
Time frame: Baseline
Number of Patients by Disease Characteristics at Diagnosis
Disease characteristics include: * Disease phase * BCR-ABL1 status * Level of BCR-ABL1 transcription * Mutations * Risk score (Sokal or European Treatment And Outcome Study score (EUTOS) or according to local hospital utilization) * Baseline laboratory parameters (complete blood count, organ function tests, symptom presence, spleen size)
Time frame: Baseline
Number and Percentage of Patients by TKI and Line of Therapy
Time frame: Up to approximately 10 years
Time-to-Treatment
Time-to-treatment is defined as the number of days between CML diagnosis and treatment initiation.
Time frame: Up to approximately 10 years
Duration of Each Line of TKI Treatment
Time frame: Up to approximately 10 years
Initial and Maximum TKI Daily Dose
Time frame: Up to approximately 10 years
Number and Percentage of Patients With a Dose Escalation
Time frame: Up to approximately 10 years
Number and Percentage of Patients who Switch TKI Treatment Across All Treatment Lines
Time frame: Up to approximately 10 years
Number of Treatment Modifications by Type of Modification
Treatment modifications include dose reduction, dose escalation, interruption, and switching.
Time frame: Up to approximately 10 years
Number of Treatment Modifications by Reason for Modification
Treatment modifications include dose reduction, dose escalation, interruption, and switching.
Time frame: Up to approximately 10 years
Proportion of Patients Achieving Predefined BCR-ABL1 Quantitative Polymerase Chain Reaction (Q-PCR) Transcript Levels
Predefined BCR-ABL1 Q-PCR Transcript Levels include: * ≤10% BCR::ABL1 International Scale (IS) * ≤1% BCR::ABL1 IS * ≤0.1% BCR::ABL1 IS
Time frame: 3, 6, and 12 months, and annually thereafter up to approximately 10 years
Percentage of Patients Achieving Complete Hematological Response (CHR)
CHR is defined as a white blood cell count of less than 10×10\^9/L, no immature cells (myelocytes, promyelocytes, or blasts), platelets \<450×10\^9/L, and a non-palpable spleen.
Time frame: 3, 6, and 12 months
CHR Rate for Each Line of Treatment
CHR is defined as a white blood cell count of less than 10×10\^9/L, no immature cells (myelocytes, promyelocytes, or blasts), platelets \<450×10\^9/L, and a non-palpable spleen.
Time frame: 3, 6, and 12 months
Percentage of Patients Achieving Complete Cytogenetic Response (CcyR)
CcyR is defined as the absence of Ph+ve metaphases.
Time frame: 3, 6, and 12 months
Overall Survival (OS)
OS is defined as the period from the initiation of treatment until death from any cause at any time.
Time frame: Up to approximately 10 years
Event-Free Survival (EFS)
Event-free-survival will be calculated from the initiation of treatment to loss of CHR, loss of major cytogenetic response, transformation to accelerated phase or blast phase, or death from any cause during study treatment.
Time frame: Up to approximately 10 years
Transformation-Free Survival
Transformation-free survival will be calculated from the initiation of treatment to transformation to accelerated phase or blast phase or death during study treatment.
Time frame: Up to approximately 10 years
Proportion of Patients Achieving Treatment-Free Remission (TFR) ≥12 months
Time frame: Up to approximately 10 years
Percentage of Patients who Die While on TKI Treatment
Time frame: Up to approximately 10 years
Time From Diagnosis to Death
Time frame: Up to approximately 10 years
Time From Initiation of Each Line of TKI Treatment to Death
Time frame: Up to approximately 10 years
Number of Patients by Charlson Comorbidity Index (CCI) Score
The CCI score is used to predict the 10-year survival in patients with several comorbid diseases. Comorbidity is assessed using the CCI, categorized as low (0-1) and high (≥2).
Time frame: Baseline
Number of Patients by Comorbidity at the Start of Each Line of Treatment
Time frame: Up to approximately 10 years
Number of Patients by Comorbidity During Each Line of TKI Treatment
Time frame: Up to approximately 10 years
Association Between Comorbidities and Treatment Selection
Multivariate regression analysis will be performed to assess the association between comorbidities and treatment selection.
Time frame: Up to approximately 10 years
Association Between Comorbidities and Treatment Adjustments
Multivariate regression analysis will be performed to assess the association between comorbidities and treatment adjustments.
Time frame: Up to approximately 10 years
Association Between the Presence of Comorbidities at Diagnosis and the Achievement of Major Molecular Response (MMR)
MMR is defined as ≤0.1% BCR::ABL1 IS. Multivariate regression analysis will be performed to assess the association between the presence of comorbidities at diagnosis and achieving MMR.
Time frame: 12 months
Correlation Between Comorbidity Development During Treatment and the Achievement of Complete/Deep Molecular Response (DMR)
DMR is defined as ≤0.01% BCR::ABL1 \[IS\], MR4.0; ≤0.0032% BCR::ABL1 \[IS\], MR4.5. Multivariate regression analysis will be performed to assess the correlation between the comorbidity development and achieving DMR.
Time frame: Up to approximately 10 years
Novartis Pharmaceuticals
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