This was a retrospective, non-interventional, observational cohort study using Optum's de-identified Clinformatics® Data Mart Database. Adult patients newly diagnosed with chronic myeloid leukemia (CML) treated with tyrosine kinase inhibitors (TKIs) were identified using the Optum database and classified into the following cohorts: * First treatment cohort: Patients newly diagnosed with CML who received first treatment with a TKI. * Second treatment cohort: Patients from first treatment cohort with a subsequent line of therapy (i.e., second treatment) with a TKI. The observation period spanned from the start of data availability (i.e., 01 January 2007) to the earliest of end of data (i.e., 30 June 2022), end of continuous health plan enrollment, or death (if available). The index date was defined as the first treatment initiation for the first treatment TKI cohort and as the second treatment initiation for the second treatment TKI cohort. The baseline period consisted of the 6 months prior to the index date. The follow-up period started on the index date and ended at the earliest of end of observation period or hematopoietic stem cell transplantation (HSCT).
Study Type
OBSERVATIONAL
Enrollment
2,043
Novartis
East Hanover, New Jersey, United States
Treatment Patterns
Tyrosine kinase inhibitor (TKI) treatment management patterns in CML patients on first line or second line TKI were assessed.
Time frame: Up to approximately 10 years
Proportion of Days Covered (PDC)
PDC was defined as the number of days of medication covered divided by the number of calendar days during the study period. The study period spanned from index date to the next line of therapy initiation (switch), hematopoietic stem cell transplantation (HSCT), initiation of a CML-related chemotherapy for accelerated phase (AP)/blast crisis (BC) (day prior to HSCT/CML chemotherapy) or last supply day if treatment gap ≥ 90 days, whichever occurred first. The index date was defined as the first treatment initiation for the first treatment cohort and as the second treatment initiation for the second treatment cohort.
Time frame: Up to approximately 10 years
Number of Patients per PDC Category
PDC was defined as the number of days of medication covered divided by the number of calendar days during the study period. The study period spanned from index date to the next line of therapy initiation (switch), HSCT, initiation of a CML-related chemotherapy for AP/BC (day prior to HSCT/CML chemotherapy) or last supply day if treatment gap ≥ 90 days, whichever occurred first. The index date was defined as the first treatment initiation for the first treatment cohort and as the second treatment initiation for the second treatment cohort. PDC categories included: * PDC (%) ≤ 50% * PDC (%) ≤ 70% * PDC (%) ≥ 80% * PDC (%) \> 90%
Time frame: Up to approximately 10 years
Time to Treatment Discontinuation
Time frame: Up to approximately 10 years
Time to Treatment Switch
Time frame: Up to approximately 10 years
Time to First Treatment Interruption
Time frame: Up to approximately 10 years
Time to First Dose Reduction
Time frame: Up to approximately 10 years
Rate of Treatment Discontinuation
Time frame: Months 1, 3, 6, 9, 12, 18, and years 2, 3, 4
Rate of Treatment Switching
Time frame: Months 1, 3, 6, 9, 12, 18, and years 2, 3, 4
Rate of Treatment Interruption
Time frame: Months 1, 3, 6, 9, 12, 18, and years 2, 3, 4
Rate of Dose Reduction
Time frame: Months 1, 3, 6, 9, 12, 18, and years 2, 3, 4
Number of Patients With Non-optimal Treatment (NOPT)
Three categories of NOPT were defined: 1. Base Case: NOPT was defined as 1) treatment switch ≤ 6 months post-index, 2) temporary treatment interruption/dose reduction ≤6 months post-index followed by treatment switch ≤12 months post-index, or 3) PDC ≤50%. 2. Sensitivity 1: NOPT was defined as 1) treatment switch ≤ 6 months post-index, 2) temporary treatment interruption/dose reduction ≤6 months post-index followed by treatment switch ≤12 months post-index, or 3) PDC ≤70%. 3. Sensitivity 2: NOPT was defined as treatment switch ≤ 6 months post-index. Index date was defined as the first treatment initiation for the first treatment cohort and as the second treatment initiation for the second treatment cohort.
Time frame: Up to approximately 10 years
Number of Patients With NOPT by First Generation TKI
Three categories of NOPT were defined: 1. Base Case: NOPT was defined as 1) treatment switch ≤ 6 months post-index, 2) temporary treatment interruption/dose reduction ≤6 months post-index followed by treatment switch ≤12 months post-index, or 3) PDC ≤50%. 2. Sensitivity 1: NOPT was defined as 1) treatment switch ≤ 6 months post-index, 2) temporary treatment interruption/dose reduction ≤6 months post-index followed by treatment switch ≤12 months post-index, or 3) PDC ≤70%. 3. Sensitivity 2: NOPT was defined as treatment switch ≤ 6 months post-index. Index date was defined as the first treatment initiation for the first treatment cohort and as the second treatment initiation for the second treatment cohort.
Time frame: Up to approximately 10 years
Number of Patients With NOPT by Second Generation TKI
Three categories of NOPT were defined: 1. Base Case: NOPT was defined as 1) treatment switch ≤ 6 months post-index, 2) temporary treatment interruption/dose reduction ≤6 months post-index followed by treatment switch ≤12 months post-index, or 3) PDC ≤50%. 2. Sensitivity 1: NOPT was defined as 1) treatment switch ≤ 6 months post-index, 2) temporary treatment interruption/dose reduction ≤6 months post-index followed by treatment switch ≤12 months post-index, or 3) PDC ≤70%. 3. Sensitivity 2: NOPT was defined as treatment switch ≤ 6 months post-index. Index date was defined as the first treatment initiation for the first treatment cohort and as the second treatment initiation for the second treatment cohort.
Time frame: Up to approximately 10 years
All-cause Healthcare Resource Utilization (HRU) per Patient per Year in NOPT Patients Compared to Reference Subgroup
The reference subgroup was defined based on the following treatment adherence and persistence criteria denoting potential indicators of TKI treatment success based on observations from the clinical practice: (1) high treatment adherence (defined as PDC \>90%) with no observed treatment discontinuation anytime post-index; or (2) high treatment adherence (defined as PDC \>90%) with treatment discontinuation occurring \>12 months post-index. All-cause HRU included: * Inpatient admission * Inpatient days * Emergency department visits * Outpatient visits
Time frame: 2 years
CML-related HRU per Patient per Year in NOPT Patients Compared to Reference Subgroup
The reference subgroup was defined based on the following treatment adherence and persistence criteria denoting potential indicators of TKI treatment success based on observations from the clinical practice: (1) high treatment adherence (defined as PDC \>90%) with no observed treatment discontinuation anytime post-index; or (2) high treatment adherence (defined as PDC \>90%) with treatment discontinuation occurring \>12 months post-index. All-cause HRU included: * Inpatient admission * Inpatient days * Emergency department visits * Outpatient visits
Time frame: 2 years
All-cause Direct Healthcare Costs per Patient per Year in NOPT Patients Compared to Reference Subgroup
The reference subgroup was defined based on the following treatment adherence and persistence criteria denoting potential indicators of TKI treatment success based on observations from the clinical practice: (1) high treatment adherence (defined as PDC \>90%) with no observed treatment discontinuation anytime post-index; or (2) high treatment adherence (defined as PDC \>90%) with treatment discontinuation occurring \>12 months post-index. All-cause direct healthcare costs included: * Total medical costs * Inpatient costs * Outpatient costs * Emergency department costs
Time frame: 2 years
CML-related Direct Healthcare Costs per Patient per Year in NOPT Patients Compared to Reference Subgroup
The reference subgroup was defined based on the following treatment adherence and persistence criteria denoting potential indicators of TKI treatment success based on observations from the clinical practice: (1) high treatment adherence (defined as PDC \>90%) with no observed treatment discontinuation anytime post-index; or (2) high treatment adherence (defined as PDC \>90%) with treatment discontinuation occurring \>12 months post-index. All-cause direct healthcare costs included: * Total medical costs * Inpatient costs * Outpatient costs * Emergency department costs
Time frame: 2 years
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