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Completed

NCT Number: NCT06692803

Real-World Treatment Patterns and Outcomes Among Patients With Chronic Myeloid Leukemia in Earlier Lines of Therapy

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).

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Key information

Conditions

Chronic Myeloid Leukemia Bone Marrow Diseases Chronic Disease Disease Attributes Hematologic Diseases Hemic and Lymphatic Diseases Leukemia Leukemia, Myelogenous, Chronic, BCR-ABL Positive Leukemia, Myeloid Myeloproliferative Disorders Neoplasms Neoplasms by Histologic Type Pathologic Processes Pathological Conditions, Signs and Symptoms

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Novartis

East Hanover, New Jersey, 07936, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Adult patients who initiated their first treatment with imatinib, dasatinib, nilotinib, or bosutinib (conditional on Food and Drug Administration (FDA) approval dates) with 6 months continuous health plan enrollment prior to the first prescription fill date.
  • Patients with 2 or more diagnoses for CML (adult as of the first diagnosis for CML).
  • Patients had index date on or after first CML diagnosis.
  • Patients had no diagnoses for CML remission/relapse prior to index date.
  • Patients had no gastrointestinal stomach tumor (GIST) or chronic myelomonocytic leukemia (CMML) at any time.
  • Patients had no medical claims associated with a clinical trial during the baseline period.
  • Patients had no hematopoietic stem cell transplantation (HSCT) during the baseline period.
  • Patients had no CML-related chemotherapy treatments for accelerated phase (AP)/blast crisis (BC) during the baseline period.
  • First treatment cohort:
  • Patients started first treatment for CML with imatinib, dasatinib, nilotinib, or bosutinib in 2012 or later.
  • Patients had no HSCT during the first treatment baseline period.
  • Patients had no CML-related chemotherapy treatment for AP/BC during the first treatment baseline period.
  • Second treatment cohort:
  • Patients started second treatment for CML with imatinib, dasatinib, nilotinib, or bosutinib in 2012 or later.
  • Patients had no HSCT from the first treatment baseline period up to second treatment initiation.
  • Patients had no CML-related chemotherapy treatment for AP/BC from the first treatment baseline period up to second treatment initiation.

Treatment and study plan

Primary outcomes

  1. Treatment Patterns

    Time frame: Up to approximately 10 years

    Tyrosine kinase inhibitor (TKI) treatment management patterns in CML patients on first line or second line TKI were assessed.

Secondary outcomes

  1. Proportion of Days Covered (PDC)

    Time frame: Up to approximately 10 years

    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.

  2. Number of Patients per PDC Category

    Time frame: Up to approximately 10 years

    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%

  3. Time to Treatment Discontinuation

    Time frame: Up to approximately 10 years

  4. Time to Treatment Switch

    Time frame: Up to approximately 10 years

  5. Time to First Treatment Interruption

    Time frame: Up to approximately 10 years

  6. Time to First Dose Reduction

    Time frame: Up to approximately 10 years

  7. Rate of Treatment Discontinuation

    Time frame: Months 1, 3, 6, 9, 12, 18, and years 2, 3, 4

  8. Rate of Treatment Switching

    Time frame: Months 1, 3, 6, 9, 12, 18, and years 2, 3, 4

  9. Rate of Treatment Interruption

    Time frame: Months 1, 3, 6, 9, 12, 18, and years 2, 3, 4

  10. Rate of Dose Reduction

    Time frame: Months 1, 3, 6, 9, 12, 18, and years 2, 3, 4

  11. Number of Patients With Non-optimal Treatment (NOPT)

    Time frame: Up to approximately 10 years

    Three categories of NOPT were defined:

    • 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%.
    • 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%.
    • 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.

  12. Number of Patients With NOPT by First Generation TKI

    Time frame: Up to approximately 10 years

    Three categories of NOPT were defined:

    • 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%.
    • 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%.
    • 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.

  13. Number of Patients With NOPT by Second Generation TKI

    Time frame: Up to approximately 10 years

    Three categories of NOPT were defined:

    • 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%.
    • 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%.
    • 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.

  14. All-cause Healthcare Resource Utilization (HRU) per Patient per Year in NOPT Patients Compared to Reference Subgroup

    Time frame: 2 years

    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

  15. CML-related HRU per Patient per Year in NOPT Patients Compared to Reference Subgroup

    Time frame: 2 years

    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

  16. All-cause Direct Healthcare Costs per Patient per Year in NOPT Patients Compared to Reference Subgroup

    Time frame: 2 years

    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

  17. CML-related Direct Healthcare Costs per Patient per Year in NOPT Patients Compared to Reference Subgroup

    Time frame: 2 years

    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

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study title

Real-World Treatment Patterns and Outcomes Among Patients With Chronic Myeloid Leukemia in Earlier Lines of Therapy (ABL-2022-03)

Acronym: ABL-2022-03

Important dates

Study start
2023
Primary completion
2023
Study completion
2023
First posted
Nov 18, 2024
Registry last updated
Nov 18, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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