Leukemia, Myeloid, Chronic-Phase
Conditions
Keywords
CML, Chronic Myeloid Leukemia
Brief summary
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.
Interventions
None listed
Sponsors
Novartis Pharmaceuticals
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
21 Years to 90 Years
Healthy volunteers
No
Inclusion criteria
* Diagnosed with Ph+ve CML based on the European LeukemiaNet (ELN) and National Comprehensive Cancer Network (NCCN) diagnostic criteria. * Received at least one line of TKI therapy. * Having a documented pre-index period (equal to either 6 months prior to the index date or less in case of newly diagnosed patients).
Exclusion criteria
• Patients not fulfilling any of the above-mentioned inclusion criteria.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Patients by Disease Characteristics at Diagnosis | Baseline | 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) |
| Number of Patients by Demographic Category | Baseline | Demographics include gender and ethnicity. |
| Age at Diagnosis | Baseline | — |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| CHR Rate for Each Line of Treatment | 3, 6, and 12 months | 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. |
| Percentage of Patients Achieving Complete Cytogenetic Response (CcyR) | 3, 6, and 12 months | CcyR is defined as the absence of Ph+ve metaphases. |
| Overall Survival (OS) | Up to approximately 10 years | OS is defined as the period from the initiation of treatment until death from any cause at any time. |
| Event-Free Survival (EFS) | Up to approximately 10 years | 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. |
| Transformation-Free Survival | Up to approximately 10 years | Transformation-free survival will be calculated from the initiation of treatment to transformation to accelerated phase or blast phase or death during study treatment. |
| Proportion of Patients Achieving Treatment-Free Remission (TFR) ≥12 months | Up to approximately 10 years | — |
| Percentage of Patients who Die While on TKI Treatment | Up to approximately 10 years | — |
| Time From Diagnosis to Death | Up to approximately 10 years | — |
| Time From Initiation of Each Line of TKI Treatment to Death | Up to approximately 10 years | — |
| Number of Patients by Charlson Comorbidity Index (CCI) Score | Baseline | 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). |
| Number of Patients by Comorbidity at the Start of Each Line of Treatment | Up to approximately 10 years | — |
| Number of Patients by Comorbidity During Each Line of TKI Treatment | Up to approximately 10 years | — |
| Association Between Comorbidities and Treatment Selection | Up to approximately 10 years | Multivariate regression analysis will be performed to assess the association between comorbidities and treatment selection. |
| Association Between Comorbidities and Treatment Adjustments | Up to approximately 10 years | Multivariate regression analysis will be performed to assess the association between comorbidities and treatment adjustments. |
| Association Between the Presence of Comorbidities at Diagnosis and the Achievement of Major Molecular Response (MMR) | 12 months | 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. |
| Correlation Between Comorbidity Development During Treatment and the Achievement of Complete/Deep Molecular Response (DMR) | Up to approximately 10 years | 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. |
| Initial and Maximum TKI Daily Dose | Up to approximately 10 years | — |
| Number and Percentage of Patients With a Dose Escalation | Up to approximately 10 years | — |
| Number and Percentage of Patients who Switch TKI Treatment Across All Treatment Lines | Up to approximately 10 years | — |
| Number of Treatment Modifications by Type of Modification | Up to approximately 10 years | Treatment modifications include dose reduction, dose escalation, interruption, and switching. |
| Duration of Each Line of TKI Treatment | Up to approximately 10 years | — |
| Number and Percentage of Patients by TKI and Line of Therapy | Up to approximately 10 years | — |
| Number of Treatment Modifications by Reason for Modification | Up to approximately 10 years | Treatment modifications include dose reduction, dose escalation, interruption, and switching. |
| Time-to-Treatment | Up to approximately 10 years | Time-to-treatment is defined as the number of days between CML diagnosis and treatment initiation. |
| Proportion of Patients Achieving Predefined BCR-ABL1 Quantitative Polymerase Chain Reaction (Q-PCR) Transcript Levels | 3, 6, and 12 months, and annually thereafter up to approximately 10 years | Predefined BCR-ABL1 Q-PCR Transcript Levels include: * ≤10% BCR::ABL1 International Scale (IS) * ≤1% BCR::ABL1 IS * ≤0.1% BCR::ABL1 IS |
| Percentage of Patients Achieving Complete Hematological Response (CHR) | 3, 6, and 12 months | 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. |
Contacts
CONTACTNovartis Pharmaceuticals
STUDY_DIRECTORNovartis Pharmaceuticals
Novartis Pharmaceuticals
Outcome results
None listed