Real-World Evaluation of Patient Characteristics and Treatment Patterns Among Patients With CML-CP Treated With Asciminib
NCT Number: NCT06516029
Status: COMPLETED
Study Type: OBSERVATIONAL
Conditions: Chronic Myeloid Leukemia, Chronic Phase
Sponsor: Novartis
Sex: ALL
Age: ADULT, OLDER_ADULT
Enrollment: 111
Study Design: Observational Model: |Time Perspective: p
Start Date: 2023-04-21
Completion Date: 2023-10-02
Locations: Novartis Pharmaceuticals, East Hanover, New Jersey, 07936, United States

Summary:
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.

Primary Outcome Measures: 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: 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