Clinical trial NCT06692803: Real-World Treatment Patterns and Outcomes Among Patients With Chronic Myeloid Leukemia in Earlier Lines of Therapy
Official title: Real-World Treatment Patterns and Outcomes Among Patients With Chronic Myeloid Leukemia in Earlier Lines of Therapy (ABL-2022-03)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Chronic Myeloid Leukemia
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2024-11-18
Enrollment: 2043
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: This was a retrospective, noninterventional cohort study.

Brief summary: This was a retrospective, non-interventional, observational cohort study using Optum's de-identified Clinformatics® Data Mart Database. Adult patients newly diagnosed with chronic myeloid leukemia (CML) treated with tyrosine kinase inhibitors (TKIs) were identified using the Optum database and classified into the following cohorts:

* First treatment cohort: Patients newly diagnosed with CML who received first treatment with a TKI.
* Second treatment cohort: Patients from first treatment cohort with a subsequent line of therapy (i.e., second treatment) with a TKI.

The observation period spanned from the start of data availability (i.e., 01 January 2007) to the earliest of end of data (i.e., 30 June 2022), end of continuous health plan enrollment, or death (if available). The index date was defined as the first treatment initiation for the first treatment TKI cohort and as the second treatment initiation for the second treatment TKI cohort. The baseline period consisted of the 6 months prior to the index date. The follow-up period started on the index date and ended at the earliest of end of observation period or hematopoietic stem cell transplantation (HSCT).


Eligibility criteria: Inclusion Criteria:

* Adult patients who initiated their first treatment with imatinib, dasatinib, nilotinib, or bosutinib (conditional on Food and Drug Administration (FDA) approval dates) with 6 months continuous health plan enrollment prior to the first prescription fill date.
* Patients with 2 or more diagnoses for CML (adult as of the first diagnosis for CML).
* Patients had index date on or after first CML diagnosis.
* Patients had no diagnoses for CML remission/relapse prior to index date.
* Patients had no gastrointestinal stomach tumor (GIST) or chronic myelomonocytic leukemia (CMML) at any time.
* Patients had no medical claims associated with a clinical trial during the baseline period.
* Patients had no hematopoietic stem cell transplantation (HSCT) during the baseline period.
* Patients had no CML-related chemotherapy treatments for accelerated phase (AP)/blast crisis (BC) during the baseline period.
* First treatment cohort:

  * Patients started first treatment for CML with imatinib, dasatinib, nilotinib, or bosutinib in 2012 or later.
  * Patients had no HSCT during the first treatment baseline period.
  * Patients had no CML-related chemotherapy treatment for AP/BC during the first treatment baseline period.
* Second treatment cohort:

  * Patients started second treatment for CML with imatinib, dasatinib, nilotinib, or bosutinib in 2012 or later.
  * Patients had no HSCT from the first treatment baseline period up to second treatment initiation.
  * Patients had no CML-related chemotherapy treatment for AP/BC from the first treatment baseline period up to second treatment initiation.

Primary outcomes:
measure: Treatment Patterns
timeFrame: Up to approximately 10 years
Secondary outcomes:
measure: Proportion of Days Covered (PDC)
timeFrame: Up to approximately 10 years
measure: Number of Patients per PDC Category
timeFrame: Up to approximately 10 years
measure: Time to Treatment Discontinuation
timeFrame: Up to approximately 10 years
measure: Time to Treatment Switch
timeFrame: Up to approximately 10 years
measure: Time to First Treatment Interruption
timeFrame: Up to approximately 10 years
measure: Time to First Dose Reduction
timeFrame: Up to approximately 10 years
measure: Rate of Treatment Discontinuation
timeFrame: Months 1, 3, 6, 9, 12, 18, and years 2, 3, 4
measure: Rate of Treatment Switching
timeFrame: Months 1, 3, 6, 9, 12, 18, and years 2, 3, 4
measure: Rate of Treatment Interruption
timeFrame: Months 1, 3, 6, 9, 12, 18, and years 2, 3, 4
measure: Rate of Dose Reduction
timeFrame: Months 1, 3, 6, 9, 12, 18, and years 2, 3, 4
measure: Number of Patients With Non-optimal Treatment (NOPT)
timeFrame: Up to approximately 10 years
measure: Number of Patients With NOPT by First Generation TKI
timeFrame: Up to approximately 10 years
measure: Number of Patients With NOPT by Second Generation TKI
timeFrame: Up to approximately 10 years
measure: All-cause Healthcare Resource Utilization (HRU) per Patient per Year in NOPT Patients Compared to Reference Subgroup
timeFrame: 2 years
measure: CML-related HRU per Patient per Year in NOPT Patients Compared to Reference Subgroup
timeFrame: 2 years
measure: All-cause Direct Healthcare Costs per Patient per Year in NOPT Patients Compared to Reference Subgroup
timeFrame: 2 years
measure: CML-related Direct Healthcare Costs per Patient per Year in NOPT Patients Compared to Reference Subgroup
timeFrame: 2 years
Other outcomes:
Locations:
city: East Hanover
country: United States
facility: Novartis
state: New Jersey
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: