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

NCT ID: NCT07151820

Last Updated: 2025-09-03

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 2546 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2024-04-08
• Study Completion Date: 2024-10-22

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

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: RETROSPECTIVE

Study Groups

First Generation (1G) TKI Cohort

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

No interventions assigned to this group

Second Generation (2G) TKI Cohort

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

No interventions assigned to this group

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Novartis Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

Novartis

East Hanover, New Jersey, United States

Countries

United States

Other Identifiers

CABL001A0US08

Identifier Type: -

Identifier Source: org_study_id