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

NCT07374120 | Completed

• 1 other identifier: CABL001A0US12
• Study Type: observational
• Target: 1,480
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Leukemia, Myeloid, Chronic-Phase

Keywords

CML; Tyrosine kinase inhibitors; Treatment patterns; Real-world

Outcome Measures

Primary Outcomes (28)

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

  Baseline

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

  Up to approximately 7 years

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

  Up to approximately 7 years

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

  Up to approximately 7 years

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

  Up to approximately 7 years

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

  Dose modifications included dose escalation, reduction, or hold.

  Up to approximately 7 years

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

  Dose modifications included dose escalation, reduction, or hold.

  Up to approximately 7 years

• Number of Patients who Discontinued 1L and 2L TKI Treatment

  Up to approximately 7 years

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

  Up to approximately 7 years

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

  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

  Up to approximately 7 years

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

  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

  Up to approximately 7 years

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

  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

  From Baseline up to 12 months

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

  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

  6, 12, 18, and 24 months

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

  From Baseline up to 6 months

• 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

  From Month 6 to Month 12

• 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

  From Month 13 to Month 24

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

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

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

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

  Up to approximately 7 years

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

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

  From Baseline up to 12 months

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

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

  Up to approximately 7 years

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

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

  From Baseline up to 12 months

• Duration of Therapy (DOT) for 1L TKI Treatment

  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.

  Up to approximately 7 years

• DOT for 2L TKI Treatment

  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.

  Up to approximately 7 years

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

  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.

  Up to approximately 7 years

• PFS for 2L TKI Treatment

  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.

  Up to approximately 7 years

• Overall Survival (OS) for 1L TKI Treatment

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

  Up to approximately 7 years

• Overall Survival (OS) for 2L TKI Treatment

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

  Up to approximately 7 years

• Treatment-free Interval (TFI)

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

  Up to approximately 7 years

Secondary Outcomes (17)

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

  Baseline

• 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

  Up to approximately 7 years

• 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

  2 years

• 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

  Up to approximately 5 years

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

  Up to approximately 7 years

Study Arms (1)

CML-CP Group

Patients with a diagnosis of CML-CP who initiated 1L TKI treatment in The US Oncology Network practices between 01 January 2016 and 31 December 2022.

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

This was a retrospective, noninterventional cohort study.

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (1)

Novartis

East Hanover, New Jersey, 07936, United States

Location

Related Links

• Link to study results

MeSH Terms

Conditions

Leukemia, Myeloid, Chronic-Phase

Condition Hierarchy (Ancestors)

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

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 20, 2026
• First Posted: January 28, 2026
• Study Start: February 1, 2024
• Primary Completion: January 21, 2025
• Study Completion: January 21, 2025
• Last Updated: February 2, 2026

Record last verified: 2026-01

Locations

US (1)