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Completed

NCT Number: NCT07188428

Treatment Patterns and Outcomes Among Patients With Chronic Myeloid Leukemia (CML) in All Lines of Treatment

The aim of this study was to evaluate the real-world treatment patterns, healthcare resource use and costs among patients newly diagnosed with CML who were treated with tyrosine kinase inhibitors (TKIs) in first- and second-line therapy.

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Key information

About this study

The study used claims from Medicare FFS data from 1 January 2016 to 31 December 2022 (observation period). The patient identification period spanned from 1 January 2017 to 30 June 2022. The index date was the date of treatment line initiation. Patients were followed from index date until death, end of enrollment, or study end (31 December 2022), whichever came first. Each patient's follow-up was variable in length.

Variables were assessed during the follow-up period in first-line (1L) and second-line (2L) treatment cohorts and were stratified by generations of TKIs and treatment profiles. Patients were categorized into non-optimal treatment (NOPT) profiles and reference groups, and treatment patterns, healthcare resource utilization (HCRU), and payer healthcare costs were summarized descriptively for each group.

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • L Cohort:
  • Patients had at least one diagnosis for CML (International Classification of Diseases, 10th Revision, Clinical Modification [ICD-10-CM]: C92.1x) during the observation period.
  • Patients ≥18 years of age as of the first CML diagnosis.
  • Patients received imatinib, dasatinib, nilotinib, or bosutinib in 1L.
  • Patients initiated 1L within a maximum of 3 months following the first observed CML diagnosis.
  • Patients had at least 12 months of continuous enrollment prior to the index date.
  • Patients had at least 6 months of continuous enrollment post index date (including index date).
  • L Cohort:
  • Patients in the 1L cohort.
  • Patients received imatinib, dasatinib, nilotinib, bosutinib, ponatinib or asciminib in 2L.
  • Patients with at least 12 months of continuous enrollment prior to the index date.
  • Patients with at least 6 months of continuous enrollment post index date (including index date).

Exclusion criteria

  • L Cohort:
  • Patients with medical services with a procedure code for imatinib any time during the observation period.
  • Patients with a diagnosis for CML remission (identified by ICD-10-CM code: C92.11) or CML relapse (ICD-10-CM code: C92.12) any time in the baseline period before the 1L initiation.
  • Patients with a medical claim associated with a clinical trial drug during the baseline period before the 1L initiation up to the end of the observation period.
  • Patients who received chemotherapy, or a TKI of interest or had a hematopoietic stem cell transplantation (HSCT) during the baseline period before 1L initiation.
  • L Cohort:
  • Patients with a diagnosis for CML remission (identified by ICD-10-CM code: C92.11) or CML relapse (ICD-10-CM code: C92.12) any time before the 2L initiation.

Treatment and study plan

Primary outcomes

  1. Number of Patients Who Switched to Another CML-related Treatment by TKI Generation and Treatment Profile

    Time frame: Up to approximately 6 years

  2. Number of Patients Who Switched to Another CML-related Treatment Within 6 Months of Starting Treatment by TKI Generation and Treatment Profile

    Time frame: 6 months

  3. Time to Treatment Switch by TKI Generation and Treatment Profile

    Time frame: Up to approximately 6 years

Secondary outcomes

  1. Proportion of Days Covered (PDC) by TKI Generation and Treatment Profile

    Time frame: Up to approximately 6 years

    Adherence was assessed using PDC. The PDC was defined as the number of days of medication covered during the treatment line, divided by the number of calendar days between the start and end dates of the line of treatment.

  2. Number of Patients With PDC >80% by TKI Generation and Treatment Profile

    Time frame: Up to approximately 6 years

    Adherence was assessed using PDC. The PDC was defined as the number of days of medication covered during the treatment line, divided by the number of calendar days between the start and end dates of the line of treatment.

  3. Number of Patients who Discontinued Treatment by TKI Generation and Treatment Profile

    Time frame: Up to approximately 6 years

  4. Time to Treatment Discontinuation by TKI Generation and Treatment Profile

    Time frame: Up to approximately 6 years

  5. Number of Patients With Treatment Interruption by TKI Generation and Treatment Profile

    Time frame: Up to approximately 6 years

    Treatment interruption was defined as a gap of ≥14 days in CML treatment with resumption of that treatment after the gap.

  6. Time to First Treatment Interruption by TKI Generation and Treatment Profile

    Time frame: Up to approximately 6 years

  7. Number of Patients With a Dose Reduction by TKI Generation and Treatment Profile

    Time frame: Up to approximately 6 years

  8. Time to Dose Reduction by TKI Generation and Treatment Profile

    Time frame: Up to approximately 6 years

  9. Mean Number of All-cause Healthcare Visits per Person per Year (PPPY) by Treatment Profile

    Time frame: Up to approximately 6 years

    Healthcare visits included inpatient visits, outpatient visits, emergency department visits, and pharmacy visits.

  10. Mean Number of CML-related Healthcare Visits per Person per Year (PPPY) by Treatment Profile

    Time frame: Up to approximately 6 years

    Healthcare visits included inpatient visits, outpatient visits, emergency department visits, and pharmacy visits.

  11. Mean Length of Stay (LoS) of Inpatient Visits PPPY by Treatment Profile

    Time frame: Up to approximately 6 years

  12. Mean All-cause Healthcare Cost PPPY by Treatment Profile

    Time frame: Up to approximately 6 years

    Healthcare costs included:

    • Total costs
    • Inpatient costs
    • Emergency department costs
    • Outpatient costs
    • Other costs
    • Pharmacy costs
  13. Mean CML-related Healthcare Cost PPPY by Treatment Profile

    Time frame: Up to approximately 6 years

    Healthcare costs included:

    • Total 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

Treatment Patterns and Outcomes Among Patients With Chronic Myeloid Leukemia (CML) in All Lines of Therapy: An Analysis of Medicare Fee-for-Service (FFS) Data

Important dates

Study start
2024
Primary completion
2024
Study completion
2024
First posted
Sep 23, 2025
Registry last updated
Sep 23, 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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