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

Treatment Patterns and Key Endpoints Among Patients With Chronic-Phase Chronic Myeloid Leukemia (CML-CP) 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.

Study Overview

• Status: Completed
• Conditions: Leukemia, Myeloid, Chronic-Phase

• Study Type: Observational
• Enrollment (Actual): 1480

Contacts and Locations

Study Locations

• United States
  • New Jersey
    • East Hanover, New Jersey, United States, 07936
      • Novartis

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

This was a retrospective, noninterventional cohort study.

Description

Inclusion criteria:

1. Patients whose data were accessible for research purposes during the study observation period.
2. Patients diagnosed with CML-CP at first recorded diagnosis of CML.
3. Patients ≥ 18 years of age at first recorded diagnosis of CML.
4. 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.
5. 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:

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

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Time Frame
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
Following Initiation of 1L and 2L TKI Treatment, Number of BCR::ABL Tests (per NCCN Guidelines) Within 2 Years After Achieving BCR::ABL1 ≤1%	2 years
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%	Up to approximately 5 years
Number of Patients With BCR::ABL Testing (per European LeukemiaNet (ELN) Guidelines) at Diagnosis	Baseline
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	Up to approximately 7 years
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	5 months
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	5 months
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	Up to approximately 7 years
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	Up to approximately 7 years
Number of BCR::ABL Tests (per ELN Guidelines) After Initiation of 1L and 2L TKI Treatment Until BCR::ABL1 ≤0.1% was Achieved	Up to approximately 7 years
Number of BCR::ABL Tests (per ELN Guidelines) After Initiation of 1L and 2L TKI Treatment Until CCyR was Achieved	Up to approximately 7 years
Number of Molecular Testing by Year Following Initiation of 1L TKI Treatment During Which the T315I Mutation was Identified	Up to approximately 7 years
Number of Patients With T3151 Mutation Testing During 1L and 2L TKI Treatment	Up to approximately 7 years

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals
• Investigators: Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Publications and helpful links

Helpful Links

• Link to study results

Study record dates

Study Major Dates

• Study Start (Actual): February 1, 2024
• Primary Completion (Actual): January 21, 2025
• Study Completion (Actual): January 21, 2025

Study Registration Dates

• First Submitted: January 20, 2026
• First Submitted That Met QC Criteria: January 20, 2026
• First Posted (Actual): January 28, 2026

Study Record Updates

• Last Update Posted (Actual): February 2, 2026
• Last Update Submitted That Met QC Criteria: January 29, 2026
• Last Verified: January 1, 2026

More Information

Terms related to this study

• Keywords: CML; Tyrosine kinase inhibitors; Real-world; Treatment patterns
• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms; Chronic Disease; Disease Attributes; Neoplasms by Histologic Type; Hematologic Diseases; Leukemia, Myeloid; Bone Marrow Diseases; Leukemia; Myeloproliferative Disorders; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Pathological Conditions, Signs and Symptoms; Hemic and Lymphatic Diseases; Leukemia, Myeloid, Chronic-Phase
• Other Study ID Numbers: CABL001A0US12