A Study of Real-world Treatment and Burden of Chronic Myeloid Leukemia Patients in Japan

Completed

• Conditions: Myeloid Leukemia, Chronic

• Registration Number: NCT07150793
• Lead Sponsor: Novartis Pharmaceuticals

Brief Summary

The main goal of this study was to describe treatment patterns of patients with chronic myeloid leukemia (CML) who were treated with tyrosine kinase inhibitors (TKIs) in Japan.

This was a retrospective observational study with secondary use of data from the Medical Data Vision (MDV) hospital-based database. The index date was the date of the first prescription of any TKI of interest registered within the selection period (01 January 2011 to 31 December 2019). The follow-up period was from the index date until the end of the study period, end of continuous follow-up in the MDV database, or death, whichever came first.

Detailed Description

Not available

Study Timeline

• Study Start: 2022-07-05
• Primary Completion: 2023-02-08
• Study Completion: 2023-02-08
• Status Verified: 2025-08
• Study First Submitted: 2025-08-27
• Study First Submitted QC: 2025-08-27
• Last Update Submitted: 2025-08-27
• Study First Posted: 2025-09-02
• Last Update Posted: 2025-09-02

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 754

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 by Line of Treatment (LoT) and TKI Received	Up to approximately 9 years	TKIs included imatinib, bosutinib, dasatinib, nilotinib, and ponatinib.
Number of Patients who Switched TKI Treatment by LoT and TKI	Up to approximately 9 years	TKIs included imatinib, bosutinib, dasatinib, nilotinib, and ponatinib.
Number of Patients who Discontinued TKI Treatment by Type of TKI	Up to approximately 9 years	TKIs included imatinib, bosutinib, dasatinib, nilotinib, and ponatinib.
Number of Patients who Discontinued TKI Treatment for at Least One Year by Type of TKI	Up to approximately 9 years	TKIs included imatinib, bosutinib, dasatinib, nilotinib, and ponatinib.
Number of Patients who Restarted any TKI Treatment After Discontinuing the TKI of Interest by Type of TKI	Up to approximately 9 years	TKIs included imatinib, bosutinib, dasatinib, nilotinib, and ponatinib.
TKI Dose by LoT	Baseline, up to approximately 9 years	TKIs included imatinib, bosutinib, dasatinib, nilotinib, and ponatinib.
TKI Persistence Rate by LoT	Months 6, 12, 18, and Year 5
Duration of TKI Treatment by LoT	Up to approximately 9 years

Secondary Outcome Measures

Name	Time	Method
Annual Incidence of Inpatient and Outpatient Visits by LoT	Up to approximately 9 years
Annual Mean Cost of All-cause Healthcare Resource Use by LoT	Up to approximately 9 years	All-cause care included inpatient and outpatient visits. The following costs were calculated in Japanese yen (JPN): * Total costs; * Inpatient costs; * Outpatient costs; * Pharmacy costs
Annual Incidence of CML-related Healthcare Resource Use by LoT	Up to approximately 9 years	CML-related care included: * Major BCR-ABL1 procedures (diagnostic and response to treatment testing); * Complete blood count testing - peripheral blood general testing; * Hematological examination diagnosis fee; * TKI prescriptions
Annual Mean Cost of CML-related Healthcare Resource Use by LoT	Up to approximately 9 years	CML-related care costs were calculated in Japanese Yen (JPY) and included the following: * Total CML-related costs; * Major BCR-ABL1 procedure costs; * Complete blood count testing - peripheral blood general testing costs; * Hematological examination costs; * TKI prescription costs
Number of Patients With Adverse Events by TKI	Up to approximately 9 years	TKIs included imatinib, bosutinib, dasatinib, nilotinib, and ponatinib.

Trial Locations

Locations (1)

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