Real-World Evaluation of Patient Characteristics and Treatment Patterns Among Patients With CML-CP Treated With Asciminib

Real-World Evaluation of Patient Characteristics and Treatment Patterns Among Patients With CML-CP Treated With Asciminib (ABL-2022-02)

A retrospective, non-interventional cohort study design using data obtained from the Flatiron Health oncology electronic health record (EHR)-derived de-identified database, was used to address the study objectives.

The overall asciminib cohort included adult patients with Philadelphia positive (Ph+) chronic myeloid leukemia (CML) in chronic phase (CML-CP), with or without the T3151 mutation, who initiated asciminib in any line of therapy. The third-line or later (3L+) asciminib cohort included adult patients with Ph+ CML-CP who did not have T315I mutation and initiated asciminib after prior use of at least 2 different tyrosine kinase inhibitors (TKIs) or omacetaxine. The 3L asciminib cohort included the subgroup of the 3L+ asciminib cohort who initiated asciminib after prior use of 2 different TKIs or omacetaxine. The fourth-line or later (4L+) asciminib cohort included the subgroup of the 3L+ asciminib cohort who initiated asciminib after prior use of at least 3 different TKIs or omacetaxine.

Study Overview

• Status: Completed
• Conditions: Chronic Myeloid Leukemia, Chronic Phase

• Study Type: Observational
• Enrollment (Actual): 111

Contacts and Locations

Study Locations

• United States
  • New Jersey
    • East Hanover, New Jersey, United States, 07936
      • Novartis Pharmaceuticals

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

This was a retrospective, noninterventional cohort study.

Description

Inclusion criteria:

• Patients who initiated asciminib as identified by Flatiron through the Flatiron Health-Novartis Scemblix CML Spotlight database study.
• Patients had at least one diagnosis for CML (International Classification of Diseases, 9th Edition, Clinical Modification [ICD-9-CM]: 205.1x; International Classification of Diseases, 10th Edition, Clinical Modification [ICD-10-CM]: C92.1x).
• Diagnosed with CP as identified by Flatiron through chart abstraction.
• Had 2 or more documented clinical visits, on different days in the Flatiron data on or after 1 January 2011.
• Had evidence of treatment with asciminib on or after Food and Drug Administration (FDA) approval date (29 October 2021), with data granularity availability at least at the month level for dates of asciminib use.
• Had CML initial diagnosis date on or after 1 January 2011.
• Had 1 or more clinical activity within 6 months prior to asciminib initiation (e.g., medical visit, medication order, lab test, etc.).

Exclusion criteria:

• Patients who did not meet inclusion criteria listed above.
• Had stem-cell transplant (SCT) on or prior to asciminib initiation or unknown SCT date.
• Diagnosed with CML in accelerated phase (AP) or blast crisis (BC) before asciminib initiation, as identified by Flatiron through chart abstraction, or had blast greater than 15% or unknown blast result within 60 days prior to asciminib initiation.
• Patients had received a clinical study drug between 15 November 2017 and 4 December 2019 (randomization dates for the ASCEMBL trial).

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
Overall Asciminib Cohort; Adult patients diagnosed with Ph+ CML-CP, with or without the T3151 mutation, who initiated asciminib in any line of therapy.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Age at Index Date	Index date was defined as the date of initiation for asciminib.	Up to 137 months
Gender at Index Date	Index date was defined as the date of initiation for asciminib.	Up to 137 months
Race at Index Date	Index date was defined as the date of initiation for asciminib.	Up to 137 months
Type of Practice at Index Date	Types of practices included: community and academic practices. Index date was defined as the date of initiation for asciminib.	Up to 137 months
Eastern Cooperative Oncology Group Performance Score (ECOG-PS) at Index Date	ECOG-PS describes a patient's level of functioning in terms of their ability to care for themself, daily activity, and physical ability (walking, working, etc.). Scores ranged from a lower value of 0 (fully active, able to carry on all pre-disease performance without restriction) up to 4 (completely disabled; cannot carry on any selfcare; totally confined to bed or chair). Index date was defined as the date of initiation for asciminib.	Up to 137 months
Number of Patients Categorized by the Five Most Common Baseline Comorbidities	Baseline was defined as the time between the date of initial CML diagnosis up to (and excluding) the date of asciminib initiation (index date).	Baseline
Number of Patients Categorized by Last Molecular Response (MR) Achieved Within 3-months Prior to Index Date	MR categories included: MR0 (BCR::ABL1 greater than 10%); MR1 (BCR::ABL1 less than or equal to 10%); MR2 (BCR::ABL1 less than or equal to 1%); MR3/Major MR (BCR::ABL1 less than or equal to 0.1%); MR4 or better (BCR::ABL1 less than or equal to 0.01%) Index date was defined as the date of initiation for asciminib.	Up to 3 months pre-index date
Median Duration of Observation from Index Date to the end of the Study Period	The study period was defined as the time between the index date (inclusive) until the earliest of stem-cell transplant (SCT), death, loss-to-follow-up (i.e., last activity in the electronic medical record data), or end of data availability (i.e., 30 November 2022). The index date was the date of initiation for asciminib.	Up to 13 months
Number of Patients by First Observed Line of Therapy with Asciminib at Index Date	Index date was defined as the date of initiation for asciminib.	Up to 137 months
Number of Patients Categorized by Dosage of Asciminib at Index Date	Index date was defined as the date of initiation for asciminib.	Up to 137 months
Number of Patients by Status at End of First Observed Line of Therapy with Asciminib	Status: Continued asciminib treatment (included dose change/held).; Discontinued asciminib treatment.	From date of asciminib initiation to end of study period, up to 13 months
Number of Patients by Type of First Tyrosine Kinase Inhibitor (TKI) Received Before Asciminib Initiation		Up to 137 months
Number of Patients by Type of TKI Received Immediately Before Asciminib Initiation		Up to 137 months
Number of Patients by Type of TKI Received Anytime Before Asciminib Initiation		Up to 137 months
Number of Patients by Most Common Treatment Sequence		From initial CML diagnosis until the end of study period, up to 143 months
Asciminib Treatment Persistence Rate Post-Index Date	Index date was defined as the date of initiation for asciminib.	Week 12, week 24 post-index date

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Patients Categorized by Best Molecular Response (MR) Achieved Within 12-months Post-index Date	MR categories included: MR0 (BCR::ABL1 greater than 10%); MR1 (BCR::ABL1 less than or equal to 10%); MR2 (BCR::ABL1 less than or equal to 1%); MR3/Major MR (BCR::ABL1 less than or equal to 0.1%); MR4 or better (BCR::ABL1 less than or equal to 0.01%) Index date was defined as the date of initiation for asciminib.	12 months post-index date
Number of Patients With one or More MR Test Post-index Date who Achieved or Maintained MR3/Major MR (MMR)	MR3/MMR was defined as BCR::ABL1 less than or equal to 0.1%. Index date was defined as the date of initiation for asciminib.	Week 12, week 24 post-index date
Number of Patients With one or More MR Test Post-index Date who Achieved or Maintained MR2	MR2 was defined as BCR::ABL1 less than or equal to 1%. Index date was defined as the date of initiation for asciminib.	Week 12, week 24 post-index date
Number of Patients Without MR3/MMR Prior to Asciminib Initiation who Achieved MR3/MMR Post-index Date	MR3/MMR was defined as BCR::ABL1 less than or equal to 0.1%. Index date was defined as the date of initiation for asciminib.	Week 12, week 24 post-index date
Median Time to MR3/MMR Post-index Date Among Patients Without MR3/MMR Prior to Asciminib Initiation	MR3/MMR was defined as BCR::ABL1 less than or equal to 0.1%. Index date was defined as the date of initiation for asciminib.	From index date until the end of study period, up to 13 months
Number of Patients Without MR2 Prior to Asciminib Initiation who Achieved MR2 Post-index Date	MR2 was defined as BCR::ABL1 less than or equal to 1%. Index date was defined as the date of initiation for asciminib.	Week 12, week 24 post-index date
Median Time to MR2 Post-index Date Among Patients Without MR2 Prior to Asciminib Initiation	MR3/MMR was defined as BCR::ABL1 less than or equal to 1%. Index date was defined as the date of initiation for asciminib.	From index date until the end of study period, up to 13 months

Collaborators and Investigators

• Sponsor: Novartis
• Investigators: Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): April 21, 2023
• Primary Completion (Actual): October 2, 2023
• Study Completion (Actual): October 2, 2023

Study Registration Dates

• First Submitted: July 7, 2024
• First Submitted That Met QC Criteria: July 17, 2024
• First Posted (Actual): July 23, 2024

Study Record Updates

• Last Update Posted (Actual): July 23, 2024
• Last Update Submitted That Met QC Criteria: July 17, 2024
• Last Verified: July 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms by Histologic Type; Neoplasms; Disease Attributes; Bone Marrow Diseases; Hematologic Diseases; Myeloproliferative Disorders; Leukemia; Leukemia, Myeloid; Chronic Disease; Leukemia, Myelogenous, Chronic, BCR-ABL Positive
• Other Study ID Numbers: CABL001A0US03