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NCT Number: NCT07374120

Treatment Patterns and Key Endpoints Among Patients With Chronic-Phase Chronic Myeloid Leukemia in a Community Oncology Setting

This was a retrospective observational study to examine treatment patterns, molecular testing patterns, treatment response, and clinical outcomes among patients initiating first-line (1L) tyrosine kinase inhibitor (TKI) treatment for CML-CP in The United States (US) Oncology Network practices.

Patients who initiated 1L therapy between 01 January 2016 and 31 December 2022 were eligible for inclusion in the study. Study-eligible patients were followed longitudinally post-index until death (if the patient had documentation of death during the study observation period) or last available patient record that occurred on or before the end of the study observation period. The study observation period was from 01 January 2016 to 30 November 2023. The index date was defined as the start date of 1L therapy for CML-CP.

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Key information

Conditions

Leukemia, Myeloid, Chronic-Phase 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 whose data were accessible for research purposes during the study observation period.
  • Patients diagnosed with CML-CP at first recorded diagnosis of CML.
  • Patients ≥ 18 years of age at first recorded diagnosis of CML.
  • Patients who initiated qualifying 1L therapy for CML-CP during the study identification period. Qualifying 1L therapy treatments consisted of imatinib, dasatinib, bosutinib, or nilotinib as monotherapy.
  • Patients with ≥1 visit within The US Oncology Network practices following initiation of 1L therapy and through the end of the study observation period.

Exclusion criteria

  • Patients enrolled in interventional clinical trials during the study observation period.
  • Patients who received any systemic treatment indicated for another primary cancer during the study observation period.

Treatment and study plan

Primary outcomes

  1. Time From Initial CML Diagnosis to Initiation of 1L TKI Treatment

    Time frame: Baseline

  2. Number of Patients who Received a Stem Cell Transplant (SCT)

    Time frame: Up to approximately 7 years

  3. Number of Patients by Type of 1L and Second-line (2L) TKI Treatment

    Time frame: Up to approximately 7 years

  4. Number of Patients by Initial Dose of Each TKI in 1L and 2L Treatment

    Time frame: Up to approximately 7 years

  5. Number of Patients by Treatment Sequence From 1L TKI to 2L TKI Treatment

    Time frame: Up to approximately 7 years

  6. Number of Patients by Dose Modification of 1L and 2L TKI Treatment

    Time frame: Up to approximately 7 years

    Dose modifications included dose escalation, reduction, or hold.

  7. Number of Patients by Reason for Dose Modification of 1L and 2L TKI Treatment

    Time frame: Up to approximately 7 years

    Dose modifications included dose escalation, reduction, or hold.

  8. Number of Patients who Discontinued 1L and 2L TKI Treatment

    Time frame: Up to approximately 7 years

  9. Number of Patients by Reason for Discontinuing 1L and 2L TKI Treatment

    Time frame: Up to approximately 7 years

  10. Number of Patients by Clinical Events of Interest During 1L and 2L TKI Treatment

    Time frame: Up to approximately 7 years

    Clinical events of interest included:

    • Alopecia
    • Anemia
    • Arthralgia/Myalgia
    • Diarrhea
    • Elevated liver enzyme
    • Elevated serum creatinine
    • Fatigue
    • Febrile neutropenia
    • Infection secondary to neutropenia
    • Interstitial lung disease-like events
    • Leukopenia
    • Nausea
    • Neutropenia
    • Pain
    • Prolongation of QT interval
    • Thrombocytopenia
    • Venous embolism (including pulmonary embolism and/or deep vein thrombosis)
    • Vomiting

  11. Number of Patients by Best Overall Molecular Response (MR) Achieved During 1L and 2L TKI Treatment

    Time frame: Up to approximately 7 years

    MR was based on BCR::ABL (International Scale [IS]) qPCR testing results. Molecular response was categorized as:

    • MR 2: if BCR::ABL (IS) >0.1% - ≤ 1%
    • Major MR (MMR)/MR 3: if BCR::ABL(IS) >0.01% - ≤0.1%
    • MR 4: if BCR::ABL1 (IS) >0.0032% -≤0.01%
    • MR 4.5: if BCR::ABL (IS) ≤0.0032%
    • Not documented

  12. Number of Patients by Best Overall MR Achieved Within 12 Months of Initiating 1L and 2L TKI Treatment

    Time frame: From Baseline up to 12 months

    MR was based on BCR::ABL (IS) qPCR testing results. Molecular response was categorized as:

    • MR 2: if BCR::ABL (IS) >0.1% - ≤ 1%
    • Major MR (MMR)/MR 3: if BCR::ABL(IS) >0.01% - ≤0.1%
    • MR 4: if BCR::ABL1 (IS) >0.0032% -≤0.01%
    • MR 4.5: if BCR::ABL (IS) ≤0.0032%
    • Not documented

  13. Number of Patients by MR Achieved After Initiating 1L and 2L TKI Treatment

    Time frame: 6, 12, 18, and 24 months

    MR was based on BCR::ABL (IS) qPCR testing results. Molecular response was categorized as:

    • MR 2: if BCR::ABL (IS) >0.1% - ≤ 1%
    • Major MR (MMR)/MR 3: if BCR::ABL(IS) >0.01% - ≤0.1%
    • MR 4: if BCR::ABL1 (IS) >0.0032% -≤0.01%
    • MR 4.5: if BCR::ABL (IS) ≤0.0032%
    • Not documented

  14. Number of Patients who Achieved a BCR::ABL (IS) Result ≤10% Within 6 Months of Initiating 1L and 2L TKI Treatment

    Time frame: From Baseline up to 6 months

  15. Number of Patients who Achieved or Sustained a BCR::ABL (IS) Result <1% Between 6 and 12 Months After Initiating 1L and 2L TKI Treatment

    Time frame: From Month 6 to Month 12

  16. Number of Patients who Achieved or Sustained a BCR::ABL (IS) Result ≤0.1% Between 13 and 24 Months After Initiating 1L and 2L TKI Treatment

    Time frame: From Month 13 to Month 24

  17. Number of Patients who Achieved or Sustained a BCR::ABL (IS) Result ≤0.1% After 24 Months Following Initiating 1L and 2L TKI Treatment

    Time frame: From Month 24 to end of study, up to approximately 6 years

  18. Number of Patients who Achieved Complete Hematologic Response (CHR) During 1L and 2L TKI Treatment

    Time frame: Up to approximately 7 years

    CHR was observed when white blood cells, hemoglobin, and platelet counts were all within each respective normal lab range.

  19. Number of Patients who Achieved CHR Within 12 Months of Initiating 1L and 2L TKI Treatment

    Time frame: From Baseline up to 12 months

    CHR was observed when white blood cells, hemoglobin, and platelet counts were all within each respective normal lab range.

  20. Number of Patients who Achieved Complete Cytogenetic Response (CCyR) During 1L and 2L TKI Treatment

    Time frame: Up to approximately 7 years

    Cytogenetic response was based on the Philadelphia chromosome results. CCyR was defined as having no Philadelphia chromosome-positive metaphases.

  21. Number of Patients who Achieved CCyR Within 12 Months of Initiating 1L and 2L TKI Treatment

    Time frame: From Baseline up to 12 months

    Cytogenetic response was based on the Philadelphia chromosome results. CCyR was defined as having no Philadelphia chromosome-positive metaphases.

  22. Duration of Therapy (DOT) for 1L TKI Treatment

    Time frame: Up to approximately 7 years

    DOT was defined as the interval between the start date of the 1L therapy (index date) and the end date of 1L therapy, including any treatment interruptions or other breaks.

  23. DOT for 2L TKI Treatment

    Time frame: Up to approximately 7 years

    DOT was defined as the interval between the start date of the 2L therapy and the end date of 2L therapy, including any treatment interruptions or other breaks.

  24. Progression-free Survival (PFS) for 1L TKI Treatment

    Time frame: Up to approximately 7 years

    PFS was defined as physician-documented progression, loss of MR2, BCR::ABL (IS) ≤1%, or an increase in BCR::ABL1 transcript to >1%, or a 1-log increase in BCR::ABL1 transcript levels with loss of MMR.

  25. PFS for 2L TKI Treatment

    Time frame: Up to approximately 7 years

    PFS was defined as physician-documented progression, loss of MR2, BCR::ABL (IS) ≤1%, or an increase in BCR::ABL1 transcript to >1%, or a 1-log increase in BCR::ABL1 transcript levels with loss of MMR.

  26. Overall Survival (OS) for 1L TKI Treatment

    Time frame: Up to approximately 7 years

    OS was defined as the interval between the start date of 1L therapy (index date) and the date of death (any cause).

  27. Overall Survival (OS) for 2L TKI Treatment

    Time frame: Up to approximately 7 years

    OS was defined as the interval between the start date of 2L therapy and the date of death (any cause).

  28. Treatment-free Interval (TFI)

    Time frame: Up to approximately 7 years

    TFI was defined as the interval between the discontinuation date of 1L therapy and the start date of 2L therapy or date of death.

Secondary outcomes

  1. Number of Patients With BCR::ABL Testing (per National Comprehensive Cancer Network (NCCN) Guidelines) at Diagnosis

    Time frame: Baseline

  2. Number of Patients With BCR::ABL Testing (per NCCN Guidelines) in 3-Month Intervals After Initiation of 1L and 2L TKI Treatment Until BCR::ABL1 ≤1% was Achieved

    Time frame: Up to approximately 7 years

  3. After Initiation of 1L and 2L TKI Treatment, Number of Patients With BCR::ABL Testing (per NCCN Guidelines) in 3-Month Intervals Within 2 Years After BCR::ABL1 ≤1% was Achieved

    Time frame: 2 years

  4. After Initiation of 1L and 2L TKI Treatment, Number of Patients With BCR::ABL Testing (per NCCN Guidelines) Between 1 and 3 Months Following 2 Years After BCR::ABL1 ≤1% was Achieved

    Time frame: Up to approximately 5 years

  5. Number of BCR::ABL Tests (per NCCN Guidelines) After Initiation of 1L and 2L TKI Treatment Until BCR::ABL1 ≤1% was Achieved

    Time frame: Up to approximately 7 years

  6. Following Initiation of 1L and 2L TKI Treatment, Number of BCR::ABL Tests (per NCCN Guidelines) Within 2 Years After Achieving BCR::ABL1 ≤1%

    Time frame: 2 years

  7. Following Initiation of 1L and 2L TKI Treatment, Number of BCR::ABL Tests (per NCCN Guidelines) Performed After 2 Years of Achieving BCR::ABL1 ≤1%

    Time frame: Up to approximately 5 years

  8. Number of Patients With BCR::ABL Testing (per European LeukemiaNet (ELN) Guidelines) at Diagnosis

    Time frame: Baseline

  9. Number of Patients With BCR::ABL Testing (per ELN Guidelines) in 3-Month Intervals After Initiation of 1L and 2L TKI Treatment Until BCR::ABL1 ≤0.1% was Achieved

    Time frame: Up to approximately 7 years

  10. Number of Patients With BCR::ABL Testing (per ELN Guidelines) Tested in 3-Month Intervals After Initiation of 1L Until BCR::ABL1 ≤0.1% was Achieved and Tested Between 2 to 6 Months Until CCyR was Achieved

    Time frame: 5 months

  11. Number of Patients With BCR::ABL Testing (per ELN Guidelines) Tested in 3-Month Intervals After Initiation of 2L Until BCR::ABL1 ≤0.1% was Achieved and Tested Between 2 to 6 Months Until CCyR was Achieved

    Time frame: 5 months

  12. Number of Patients With BCR::ABL Testing (per ELN Guidelines) Tested in 3-Month Intervals After Initiation of 1L Until BCR::ABL1 ≤0.1% was Achieved and Tested Every 12 Months Until CCyR was Achieved

    Time frame: Up to approximately 7 years

  13. Number of Patients With BCR::ABL Testing (per ELN Guidelines) Tested in 3-Month Intervals After Initiation of 2L Until BCR::ABL1 ≤0.1% was Achieved and Tested Every 12 Months Until CCyR was Achieved

    Time frame: Up to approximately 7 years

  14. Number of BCR::ABL Tests (per ELN Guidelines) After Initiation of 1L and 2L TKI Treatment Until BCR::ABL1 ≤0.1% was Achieved

    Time frame: Up to approximately 7 years

  15. Number of BCR::ABL Tests (per ELN Guidelines) After Initiation of 1L and 2L TKI Treatment Until CCyR was Achieved

    Time frame: Up to approximately 7 years

  16. Number of Molecular Testing by Year Following Initiation of 1L TKI Treatment During Which the T315I Mutation was Identified

    Time frame: Up to approximately 7 years

  17. Number of Patients With T3151 Mutation Testing During 1L and 2L TKI Treatment

    Time frame: Up to approximately 7 years

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study title

Treatment Patterns and Key Endpoints Among Patients With Chronic-Phase Chronic Myeloid Leukemia (CML-CP) in a Community Oncology Setting

Important dates

Study start
2024
Primary completion
2025
Study completion
2025
First posted
Jan 28, 2026
Registry last updated
Feb 2, 2026

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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