Leukemia, Chronic Myeloid
Conditions
Keywords
Tyrosine kinase inhibitor, Adverse events, Treatment adjustment, Healthcare resource use, Healthcare costs
Brief summary
The aim of this study was to assess the frequency of adverse events (AEs) experienced by chronic myeloid leukemia (CML) patients treated with tyrosine kinase inhibitors (TKIs) and evaluate their impact on treatment adjustments as well as the burden of AEs on healthcare resource use (HRU) and costs. This study used real-world administrative claims data from the PharMetrics Plus database. AEs of interest were selected based on information obtained from the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines, Chronic Myeloid Leukemia Version 1. 2024 and clinical inputs. Given the nature of claims data, only AEs of interest that required medical attention (i.e., resulted in a claim record) were captured in this study.
Interventions
None listed
Sponsors
Novartis Pharmaceuticals
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Patients who met all of the following criteria were considered for inclusion: * Had at least two diagnoses for CML- International Classification of Disease, 10th revision, Clinical Modification (ICD-10-CM) code: C92.1x * Aged 18 years or older at the time of first CML diagnosis * Received a 1G or 2G TKI after the first CML diagnosis * Had continuous health plan enrollment (pharmacy and medical benefits) for at least 6 months before the index date (baseline) and 6 months after the index date
Exclusion criteria
Patients who met any of the following criteria were excluded: * Patients had 2 or more diagnoses of gastrointestinal stromal tumor (\[GIST\], ICD-10: C49.Ax) or chronic myelomonocytic leukemia (\[CMML\], ICD-10: C93.1x) at any time * Patients had a hematopoietic stem cell transplantation (HSCT) during the baseline period * Patients had CML-related chemotherapy treatment for accelerated phase (AP)/blast crisis (BC) during the baseline period * Patients had medical claims associated with a clinical trial during the baseline period up to the end of the observation period
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Patients With AEs of Interest During the Study Period Among Those Without the Specific Event During the Baseline Period | Up to approximately 7 years and 3 months | The Study Period spanned from treatment initiation until treatment discontinuation, one day before treatment switch to another CML treatment, end of data availability, end of health plan coverage, or death, whichever came first. |
| Number of Patients With AEs of Interest Among All Patients During the Study Period | Up to approximately 7 years and 3 months | The Study Period spanned from treatment initiation until treatment discontinuation, one day before treatment switch to another CML treatment, end of data availability, end of health plan coverage, or death, whichever came first. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Patients With Treatment Adjustments During the Observation Period | Up to approximately 7 years and 3 months | The Observation Period spanned from treatment initiation to the end of data availability, end of health plan coverage, or death, whichever occurred first. Treatment adjustments included: * Treatment discontinuation * Treatment interruption * Switching treatment * Dose reduction |
| Duration of First Treatment Interruption | Up to approximately 7 years and 3 months | — |
| Time From TKI Initiation to Treatment Adjustment | Up to approximately 7 years and 3 months | Treatment adjustments included: * Treatment discontinuation * Treatment interruption * Switching treatment * Dose reduction |
| Number of Patients With AEs of Interest Within 30 Days Before Treatment Adjustment | 6 months | Treatment adjustments included: * Treatment discontinuation * Treatment interruption * Dose reduction |
| Number of AEs of Interest Within 30 Days Before Treatment Adjustment | 6 months | Treatment adjustments included: * Treatment discontinuation * Treatment interruption * Dose reduction |
| Number of Patients by Last AE of Interest Experienced Before Treatment Adjustment | 6 months | Treatment adjustments included: * Treatment discontinuation * Treatment interruption * Dose reduction |
| Number of Patients by AE of Interest Experienced Within 30 Days Before Treatment Adjustment | 6 months | Treatment adjustments included: * Treatment discontinuation * Treatment interruption * Dose reduction |
| Number of Patients With One or More All-cause Healthcare Visits | 6 months | Healthcare visits included: * Inpatient admissions * Emergency department visits * Outpatient admissions * Other visits |
| Number of All-cause Inpatient Days | 6 months | — |
| Number of All-cause Inpatient Days Among Hospitalized Patients | 6 months | — |
| All-cause Healthcare Costs PPPY | 6 months | Healthcare costs included: * Total costs * Medical costs * Inpatient costs * Emergency department costs * Outpatient costs * Other costs * Pharmacy costs |
| Annual Mean Number of Healthcare Visits Associated With AEs of Interest | 6 months | Healthcare visits included: * Inpatient admissions * Emergency department visits * Outpatient admissions * Other visits |
| Annual Mean Costs of Healthcare Visits Associated With AEs of Interest | 6 months | Healthcare costs included: * Total costs * Medical costs * Inpatient costs * Emergency department costs * Outpatient costs * Other costs * Pharmacy costs |
| Number of All-cause Healthcare Visits Per-Patient-Per-Year (PPPY) | 6 months | Healthcare visits included: * Inpatient admissions * Emergency department visits * Outpatient visits * Other visits |
| Time Between the Last AE of Interest and Treatment Adjustment | 6 months | Treatment adjustments included: * Treatment discontinuation * Treatment interruption * Dose reduction |
Countries
United States
Outcome results
None listed