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

Completed

• Conditions: Leukemia, Chronic Myeloid

• Registration Number: NCT07151820
• Lead Sponsor: Novartis Pharmaceuticals

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2024-04-08
• Primary Completion: 2024-10-22
• Study Completion: 2024-10-22
• Status Verified: 2025-08
• Study First Submitted: 2025-08-25
• Study First Submitted QC: 2025-08-25
• Last Update Submitted: 2025-08-25
• Study First Posted: 2025-09-03
• Last Update Posted: 2025-09-03

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 2546

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Number of Patients With AEs of Interest During the Study Period Among Those Without the Specific Event During the Baseline Period	Up to approximately 7 years and 3 months	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.
Number of Patients With AEs of Interest Among All Patients During the Study Period	Up to approximately 7 years and 3 months	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.

Secondary Outcome Measures

Name	Time	Method
Number of Patients With Treatment Adjustments During the Observation Period	Up to approximately 7 years and 3 months	The Observation Period spanned from treatment initiation to the end of data availability, end of health plan coverage, or death, whichever occurred first.; Treatment adjustments included: * Treatment discontinuation; * Treatment interruption; * Switching treatment; * Dose reduction
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	Treatment adjustments included: * Treatment discontinuation; * Treatment interruption; * Switching treatment; * Dose reduction
Number of Patients With AEs of Interest Within 30 Days Before Treatment Adjustment	6 months	Treatment adjustments included: * Treatment discontinuation; * Treatment interruption; * Dose reduction
Number of AEs of Interest Within 30 Days Before Treatment Adjustment	6 months	Treatment adjustments included: * Treatment discontinuation; * Treatment interruption; * Dose reduction
Number of Patients by AE of Interest Experienced Within 30 Days Before Treatment Adjustment	6 months	Treatment adjustments included: * Treatment discontinuation; * Treatment interruption; * Dose reduction
Time Between the Last AE of Interest and Treatment Adjustment	6 months	Treatment adjustments included: * Treatment discontinuation; * Treatment interruption; * Dose reduction
Number of Patients by Last AE of Interest Experienced Before Treatment Adjustment	6 months	Treatment adjustments included: * Treatment discontinuation; * Treatment interruption; * Dose reduction
Number of All-cause Healthcare Visits Per-Patient-Per-Year (PPPY)	6 months	Healthcare visits included: * Inpatient admissions; * Emergency department visits; * Outpatient visits; * Other visits
Number of Patients With One or More All-cause Healthcare Visits	6 months	Healthcare visits included: * Inpatient admissions; * Emergency department visits; * Outpatient admissions; * Other visits
Number of All-cause Inpatient Days	6 months
Number of All-cause Inpatient Days Among Hospitalized Patients	6 months
All-cause Healthcare Costs PPPY	6 months	Healthcare costs included: * Total costs; * Medical costs; * Inpatient costs; * Emergency department costs; * Outpatient costs; * Other costs; * Pharmacy costs
Annual Mean Number of Healthcare Visits Associated With AEs of Interest	6 months	Healthcare visits included: * Inpatient admissions; * Emergency department visits; * Outpatient admissions; * Other visits
Annual Mean Costs of Healthcare Visits Associated With AEs of Interest	6 months	Healthcare costs included: * Total costs; * Medical costs; * Inpatient costs; * Emergency department costs; * Outpatient costs; * Other costs; * Pharmacy costs

Trial Locations

Locations (1)

Novartis; 🇺🇸 East Hanover, New Jersey, United States