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Completed

NCT Number: NCT07151820

A Real-world Study About the Impact of Adverse Events (AEs) on Treatment Adjustments, Healthcare Resource Use, and Costs Among Chronic Myeloid Leukemia Patients

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.

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Key information

Conditions

Leukemia, Chronic Myeloid 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

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

Treatment and study plan

Primary outcomes

  1. Number of Patients With AEs of Interest During the Study Period Among Those Without the Specific Event During the Baseline Period

    Time frame: 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.

  2. Number of Patients With AEs of Interest Among All Patients During the Study Period

    Time frame: 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 outcomes

  1. Number of Patients With Treatment Adjustments During the Observation Period

    Time frame: 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

  2. Duration of First Treatment Interruption

    Time frame: Up to approximately 7 years and 3 months

  3. Time From TKI Initiation to Treatment Adjustment

    Time frame: Up to approximately 7 years and 3 months

    Treatment adjustments included:

    • Treatment discontinuation
    • Treatment interruption
    • Switching treatment
    • Dose reduction

  4. Number of Patients With AEs of Interest Within 30 Days Before Treatment Adjustment

    Time frame: 6 months

    Treatment adjustments included:

    • Treatment discontinuation
    • Treatment interruption
    • Dose reduction

  5. Number of AEs of Interest Within 30 Days Before Treatment Adjustment

    Time frame: 6 months

    Treatment adjustments included:

    • Treatment discontinuation
    • Treatment interruption
    • Dose reduction

  6. Number of Patients by AE of Interest Experienced Within 30 Days Before Treatment Adjustment

    Time frame: 6 months

    Treatment adjustments included:

    • Treatment discontinuation
    • Treatment interruption
    • Dose reduction

  7. Time Between the Last AE of Interest and Treatment Adjustment

    Time frame: 6 months

    Treatment adjustments included:

    • Treatment discontinuation
    • Treatment interruption
    • Dose reduction

  8. Number of Patients by Last AE of Interest Experienced Before Treatment Adjustment

    Time frame: 6 months

    Treatment adjustments included:

    • Treatment discontinuation
    • Treatment interruption
    • Dose reduction

  9. Number of All-cause Healthcare Visits Per-Patient-Per-Year (PPPY)

    Time frame: 6 months

    Healthcare visits included:

    • Inpatient admissions
    • Emergency department visits
    • Outpatient visits
    • Other visits

  10. Number of Patients With One or More All-cause Healthcare Visits

    Time frame: 6 months

    Healthcare visits included:

    • Inpatient admissions
    • Emergency department visits
    • Outpatient admissions
    • Other visits

  11. Number of All-cause Inpatient Days

    Time frame: 6 months

  12. Number of All-cause Inpatient Days Among Hospitalized Patients

    Time frame: 6 months

  13. All-cause Healthcare Costs PPPY

    Time frame: 6 months

    Healthcare costs included:

    • Total costs
    • Medical costs
    • Inpatient costs
    • Emergency department costs
    • Outpatient costs
    • Other costs
    • Pharmacy costs

  14. Annual Mean Number of Healthcare Visits Associated With AEs of Interest

    Time frame: 6 months

    Healthcare visits included:

    • Inpatient admissions
    • Emergency department visits
    • Outpatient admissions
    • Other visits

  15. Annual Mean Costs of Healthcare Visits Associated With AEs of Interest

    Time frame: 6 months

    Healthcare costs included:

    • Total costs
    • Medical costs
    • Inpatient costs
    • Emergency department costs
    • Outpatient costs
    • Other costs
    • Pharmacy costs

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study title

Association of Adverse Events in the Real World With Treatment Adjustments, and Burden of AEs in Patients With Chronic Myeloid Leukemia

Important dates

Study start
2024
Primary completion
2024
Study completion
2024
First posted
Sep 3, 2025
Registry last updated
Sep 3, 2025

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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