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

NCT07151820 | Completed

• 1 other identifier: CABL001A0US08
• Study Type: observational
• Target: 2,546
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Leukemia, Chronic Myeloid

Keywords

Tyrosine kinase inhibitor; Adverse events; Treatment adjustment; Healthcare resource use; Healthcare costs

Outcome Measures

Primary Outcomes (2)

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

  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.

  Up to approximately 7 years and 3 months

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

  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.

  Up to approximately 7 years and 3 months

Secondary Outcomes (15)

• Number of Patients With Treatment Adjustments During the Observation Period

  Up to approximately 7 years and 3 months

• Duration of First Treatment Interruption

  Up to approximately 7 years and 3 months

• Time From TKI Initiation to Treatment Adjustment

  Up to approximately 7 years and 3 months

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

  6 months

• Number of AEs of Interest Within 30 Days Before Treatment Adjustment

  6 months

Study Arms (2)

First Generation (1G) TKI Cohort

Adult CML patients who received imatinib, a 1G TKI.

Second Generation (2G) TKI Cohort

Adult CML patients who received the following 2G TKIs: dasatinib, nilotinib, or bosutinib.

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:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (1)

Novartis

East Hanover, New Jersey, 07936, United States

Location

MeSH Terms

Conditions

Leukemia, Myelogenous, Chronic, BCR-ABL Positive

Condition Hierarchy (Ancestors)

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: August 25, 2025
• First Posted: September 3, 2025
• Study Start: April 8, 2024
• Primary Completion: October 22, 2024
• Study Completion: October 22, 2024
• Last Updated: September 3, 2025

Record last verified: 2025-08

Locations

US (1)