Study Conducted Among Patients With CML

Real-World Treatment Patterns, Healthcare Resource Utilization and Associated Costs Among Patients With Chronic Myeloid Leukemia in Later Lines of Therapy

Retrospective, non-interventional observational cohort study conducted among patients with CML.

Study Overview

• Status: Completed
• Conditions: Chronic Myeloid Leukemia
• Intervention / Treatment: Other: Tyrosine Kinase Inhibitors (TKIs)

Detailed Description

A retrospective, non-interventional cohort study was used to address the study objectives. A cohort of adult patients with CML who were treated with TKIs were identified using the IBM® MarketScan® Commercial and Medicare Supplemental databases (commercial claims; the MarketScan database) to have a better understanding of real-world treatment patterns, HRU and healthcare costs among patients with CML treated with later lines of therapy (i.e., third line or later).

For Phase I, the IBM® MarketScan® Commercial Claims and Encounters and Medicare Supplemental Databases were used (commercial claims). The commercial claims covered the period from 01/01/2001 to 06/30/2019.

The study consisted of the following periods:

• The baseline period was defined as the 6-month period before the first line therapy initiation for CML.
• The observation period was defined as the period of at least 12 months from the first CML diagnosis to the end of data availability or end of health plan coverage, whichever occurs first; the observation period varied by patient.

• Study Type: Observational
• Enrollment (Actual): 3234

Contacts and Locations

Study Locations

• United States
  • New Jersey
    • East Hanover, New Jersey, United States, 07936-1080
      • Novartis Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 85 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

The study was conducted among patients with CML.

Description

Inclusion Criteria:

Patients were selected for the analysis of later lines of therapy in commercial claims (i.e., were previously treated with TKIs, who are relapsed/refractory to/intolerant of TKIs) if they met the following criteria:

• Had at least one diagnosis for CML, with first CML diagnosis observed in claims on or after May 10, 2001, the date of FDA approval for imatinib
• Were at least 18 years of age as of the first CML diagnosis
• Started a first line therapy for CML with imatinib, dasatinib, nilotinib, or bosutinib (conditional on FDA-approval dates)
• Initiated first line therapy within a maximum of 1 month prior to the first diagnosis for CML or a maximum 3 months following the first diagnosis for CML
• Had continuous health plan enrollment (pharmacy and medical benefits) from the washout period to at least 12 months following the first CML diagnosis

Exclusion Criteria:

• Patients had a diagnosis for CML remission or relapse anytime prior to first line therapy
• Patients had a medical claim associated with a clinical trial during the washout period up to the end of the observation period
• Patients had an HSCT during the washout period up to the first line therapy initiation
• Patients had chemotherapy treatment (except hydroxyurea) during the washout period up to the first line therapy initiation

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Retrospective

Number of groups / cohorts

4

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
First-line Therapy; A cohort of adult patients with CML who were treated with TKIs were identified using the IBM® MarketScan® Commercial and Medicare Supplemental databases (commercial claims; the MarketScan database)	Other: Tyrosine Kinase Inhibitors (TKIs); The study included patients with CML who were previously treated with TKIs, who are relapsed/refractory to/intolerant of TKIs on third or later lines of therapy in the US.
Second-line Therapy; A cohort of adult patients with CML who were treated with TKIs were identified using the IBM® MarketScan® Commercial and Medicare Supplemental databases (commercial claims; the MarketScan database)	Other: Tyrosine Kinase Inhibitors (TKIs); The study included patients with CML who were previously treated with TKIs, who are relapsed/refractory to/intolerant of TKIs on third or later lines of therapy in the US.
Third-line Therapy; A cohort of adult patients with CML who were treated with TKIs were identified using the IBM® MarketScan® Commercial and Medicare Supplemental databases (commercial claims; the MarketScan database)	Other: Tyrosine Kinase Inhibitors (TKIs); The study included patients with CML who were previously treated with TKIs, who are relapsed/refractory to/intolerant of TKIs on third or later lines of therapy in the US.
Fourth-line Therapy; A cohort of adult patients with CML who were treated with TKIs were identified using the IBM® MarketScan® Commercial and Medicare Supplemental databases (commercial claims; the MarketScan database)	Other: Tyrosine Kinase Inhibitors (TKIs); The study included patients with CML who were previously treated with TKIs, who are relapsed/refractory to/intolerant of TKIs on third or later lines of therapy in the US.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of patients across all lines of therapy	Number of patients across all lines of therapy were reported to evaluate treatment sequences among the patients.	throughout the study, approximately 20 years
Number of patients with the use of pre-treatment hydroxyurea	Number of patients with the use of pre-treatment hydroxyurea were reported to evaluate treatment patterns in patients.	throughout the study, approximately 20 years
Number of patients Treatment received and initial dose at each line	Number of patients Treatment received and initial dose at each line were reported to evaluate treatment patterns in patients.	throughout the study, approximately 20 years
Number of patients as per the calendar year of line of therapy initiation	Number of patients as per the calendar year of line of therapy initiation were reported to evaluate treatment patterns in patients.	throughout the study, approximately 20 years
Duration of the line of therapy	Duration of the line of therapy was reported to evaluate treatment patterns in patients.	throughout the study, approximately 20 years
Treatment-free period among those with an observed subsequent line of therapy	Treatment-free period among those with an observed subsequent line of therapy was reported to evaluate treatment patterns in patients.	throughout the study, approximately 20 years
Number of patients who discontinued treatment	Number of patients who discontinued treatment were reported to evaluate treatment patterns in patients.	throughout the study, approximately 20 years
Number of patients: All cause Health Resource Utilization among patients with three lines of therapy or more	Number of patients: All cause Health Resource Utilization were reported to estimate HRU among patients with three lines of therapy or more.	throughout the study, approximately 20 years
Healthcare costs among patients with three lines of therapy or more	Healthcare costs among patients with three lines of therapy or more were reported.	throughout the study, approximately 20 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Prevalence of second and third or later lines of therapy	Prevalence was defined as the number of cases alive per year of patients with CML who were currently or previously treated on later lines of therapy (third line or later), by calendar year from 2006 to 2018	Calendar year 2006 to 2018, approximately 12 years
Healthcare costs among patients with earlier lines of therapy	Healthcare costs among patients with earlier lines of therapy were reported.	throughout the study, approximately 20 years
Number of patients: All cause Health Resource Utilization among patients with earlier lines of therapy	Number of patients: All cause Health Resource Utilization among patients with earlier lines of therapy were reported.	throughout the study, approximately 20 years

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals

Publications and helpful links

Helpful Links

• Results for CABL001AUS07 from the Novartis Clinical Trials Website

Study record dates

Study Major Dates

• Study Start (Actual): March 8, 2021
• Primary Completion (Actual): October 15, 2021
• Study Completion (Actual): October 15, 2021

Study Registration Dates

• First Submitted: July 25, 2022
• First Submitted That Met QC Criteria: July 25, 2022
• First Posted (Actual): July 27, 2022

Study Record Updates

• Last Update Posted (Actual): November 8, 2022
• Last Update Submitted That Met QC Criteria: November 7, 2022
• Last Verified: November 1, 2022

More Information

Terms related to this study

• Keywords: costs; Chronic myeloid leukemia,; tyrosine kinase inhibitors,; treatment patterns,; HRU,
• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Bone Marrow Diseases; Hematologic Diseases; Myeloproliferative Disorders; Leukemia; Leukemia, Myeloid; Leukemia, Myelogenous, Chronic, BCR-ABL Positive
• Other Study ID Numbers: CABL001AUS07

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No