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

N/ACompletedNCT07151820

Novartis Pharmaceuticals2,546 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

2,546

Conditions

Leukemia, Chronic Myeloid

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion criteria: Patients who met all of the following criteria were considered for inclusion: * 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 Exclusion criteria: 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

Outcomes

Primary Outcomes

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.

Time frame: 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.

Time frame: Up to approximately 7 years and 3 months

Secondary Outcomes

Number of Patients With Treatment Adjustments During the Observation Period

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

Time frame: Up to approximately 7 years and 3 months

Duration of First Treatment Interruption

Time frame: Up to approximately 7 years and 3 months

Time From TKI Initiation to Treatment Adjustment

Treatment adjustments included: * Treatment discontinuation * Treatment interruption * Switching treatment * Dose reduction

Time frame: Up to approximately 7 years and 3 months

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

Treatment adjustments included: * Treatment discontinuation * Treatment interruption * Dose reduction

Time frame: 6 months

Number of AEs of Interest Within 30 Days Before Treatment Adjustment

Treatment adjustments included: * Treatment discontinuation * Treatment interruption * Dose reduction

Time frame: 6 months

Number of Patients by AE of Interest Experienced Within 30 Days Before Treatment Adjustment

Treatment adjustments included: * Treatment discontinuation * Treatment interruption * Dose reduction

Time frame: 6 months

Time Between the Last AE of Interest and Treatment Adjustment

Treatment adjustments included: * Treatment discontinuation * Treatment interruption * Dose reduction

Time frame: 6 months

Number of Patients by Last AE of Interest Experienced Before Treatment Adjustment

Treatment adjustments included: * Treatment discontinuation * Treatment interruption * Dose reduction

Time frame: 6 months

Number of All-cause Healthcare Visits Per-Patient-Per-Year (PPPY)

Healthcare visits included: * Inpatient admissions * Emergency department visits * Outpatient visits * Other visits

Time frame: 6 months

Number of Patients With One or More All-cause Healthcare Visits

Healthcare visits included: * Inpatient admissions * Emergency department visits * Outpatient admissions * Other visits

Time frame: 6 months

Number of All-cause Inpatient Days

Time frame: 6 months

Number of All-cause Inpatient Days Among Hospitalized Patients

Time frame: 6 months

All-cause Healthcare Costs PPPY

Healthcare costs included: * Total costs * Medical costs * Inpatient costs * Emergency department costs * Outpatient costs * Other costs * Pharmacy costs

Time frame: 6 months

Annual Mean Number of Healthcare Visits Associated With AEs of Interest

Healthcare visits included: * Inpatient admissions * Emergency department visits * Outpatient admissions * Other visits

Time frame: 6 months

Annual Mean Costs of Healthcare Visits Associated With AEs of Interest

Healthcare costs included: * Total costs * Medical costs * Inpatient costs * Emergency department costs * Outpatient costs * Other costs * Pharmacy costs

Time frame: 6 months

NCT07151820 - A Real-world Study About the Impact of Adverse Events (AEs) on Treatment Adjustments, Healthcare Resource Use, and Costs Among Chronic Myeloid Leukemia Patients | Crick | Crick