A retrospective multi-center cohort study design was used to address the study objectives, using medical records obtained from three clinical centers in France.
The index date for patients in the 3L cohort was defined as the date of initiation of 3L therapy. The index date for the T315I cohort was defined as the date of treatment initiation with TKI or allogeneic stem cell transplantation (allo-SCT) after identification of T315I mutation status. The baseline (i.e., pre-index) period was defined as the 6 months prior to the index date, and the post-index period was defined as the time from the index date to the date of last patient contact or patient death. Patients who were alive at the end of the follow-up period were censored at the date of last contact.
Study Type
OBSERVATIONAL
Enrollment
200
Treatments received in 3L were dasatinib, nilotinib, imatinib, ponatinib, bosutinib, and allo-SCT
Patients with chronic myeloid leukemia with T315I mutation
Novartis Investigative Site
Lyon, France
Number of patients: Year patient initiated third-line treatment
Following categories included: Before 2010 2010-2014 2015-2019 2020-2021
Time frame: throughout the study (study used data from 2000 to 2021)
Duration of third-line treatment
Duration of third-line treatment was reported
Time frame: throughout the study (study used data from 2000 to 2021)
Number of patients: Type of therapy received in third-line treatment
* TKI agent * Allo-SCT
Time frame: throughout the study (study used data from 2000 to 2021)
Time from 2L discontinuation to third-line treatment initiation
Time from 2L discontinuation to third-line treatment initiation was reported
Time frame: throughout the study (study used data from 2000 to 2021)
Number of patients still on 3L therapy as of data collection date
Number of patients still on 3L therapy as of data collection date was reported
Time frame: throughout the study (study used data from 2000 to 2021)
Number of patients discontinued 3L
Number of patients who discontinued 3L treatment were reported
Time frame: throughout the study (study used data from 2000 to 2021)
Number of patients: Reasons for 3L discontinuation
* Treatment switch due to AEs or intolerance * Treatment switch due to resistance * Treatment switch due to signs of ineffectiveness * Treatment switch due to other reasons
Time frame: throughout the study (study used data from 2000 to 2021)
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Number of lines of therapy for patients with chronic myeloid leukemia in third-line treatment
Number of lines of therapy for patients with chronic myeloid leukemia in third-line treatment were reported
Time frame: throughout the study (study used data from 2000 to 2021)
Number of most frequent treatment sequences for patients with chronic myeloid leukemia in third-line treatment
Number of most frequent treatment sequences for patients with chronic myeloid leukemia in third-line treatment were reported
Time frame: throughout the study (study used data from 2000 to 2021)
Number of last line of therapy for patients with chronic myeloid leukemia in third-line treatment
Number of last line of therapy for patients with chronic myeloid leukemia in third-line treatment were reported
Time frame: throughout the study (study used data from 2000 to 2021)
Number of patients: Year patient initiated on the line identified as T315I line of interest
Following categories included: Before 2010 2010-2014 2015-2019
Time frame: throughout the study (study used data from 2000 to 2021)
Duration of the line identified as T315I line of interest
Duration of the line identified as T315I line of interest was reported
Time frame: throughout the study (study used data from 2000 to 2021)
Number of patients: Type of therapy received in the line identified as T315I line of interest
* TKI agent * Allo-SCT
Time frame: throughout the study (study used data from 2000 to 2021)
Number of patients still on line of therapy identified as T315I line of interest as of data collection date
Number of patients still on line of therapy identified as T315I line of interest as of data collection date were reported
Time frame: throughout the study (study used data from 2000 to 2021)
Number of patients: Reasons for discontinuation on the line identified as T315I line of interest
* Adverse events or intolerance * Resistance * Signs of ineffectiveness Suboptimal response Progression to accelerated phase or blast phase * Other reasons Acquired mutations
Time frame: throughout the study (study used data from 2000 to 2021)
Number of lines of therapy for patients with chronic myeloid leukemia with T315I mutation
Number of lines of therapy for patients with chronic myeloid leukemia with T315I mutation were reported
Time frame: throughout the study (study used data from 2000 to 2021)
Number of line identified as T315I line of interest
Number of line identified as T315I line of interest were reported
Time frame: throughout the study (study used data from 2000 to 2021)
Number of treatment sequence for patients with chronic myeloid leukemia with T315I mutation
Number of treatment sequence for patients with chronic myeloid leukemia with T315I mutation were reported
Time frame: throughout the study (study used data from 2000 to 2021)
Number of last line of therapy for patients with chronic myeloid leukemia with T315I mutation
Number of last line of therapy for patients with chronic myeloid leukemia with T315I mutation were reported
Time frame: throughout the study (study used data from 2000 to 2021)
Cytogenetic response (CyR) for 3L patients
CCyR, defined as an absence of Philadelphia chromosome-positive (Ph+) metaphases in bone marrow cytogenetics
Time frame: 12 and 24 months
Proportion of patients achieving response among patients with chronic myeloid leukemia in third-line treatment in 12 months
Proportion of patients achieving response among patients with chronic myeloid leukemia in third-line treatment in 12 months were reported
Time frame: 12 months post treatment
Proportion of patients achieving response among patients with chronic myeloid leukemia in third-line treatment in 24 months
Proportion of patients achieving response among patients with chronic myeloid leukemia in third-line treatment in 24 months were reported.
Time frame: 24 months post treatment
Proportion of patients achieving sustained molecular responses among patients with chronic myeloid leukemia in third-line treatment in 12 months
Proportion of patients achieving sustained molecular responses among patients with chronic myeloid leukemia in third-line treatment in 12 months were reported.
Time frame: 12 months post treatment
Proportion of patients achieving sustained molecular responses among patients with chronic myeloid leukemia in third-line treatment in 24 months
Proportion of patients achieving sustained molecular responses among patients with chronic myeloid leukemia in third-line treatment in 24 months were reported.
Time frame: 24 months post treatment
Time to major molecular response (MMR) among patients with chronic myeloid leukemia in third-line treatment
Time to MMR was defined as the time from third-line (3L) treatment initiation to the time patients achieved MMR (i.e., 0.01% IS \< BCR::ABL1 transcript level ≤ 0.1% IS). Patients were censored at the earliest of treatment discontinuation or switch, loss to follow-up, end of data availability, or death.
Time frame: 12, 24, 36, 48, 60, 72 and 84 months post treatment
Time to deep molecular response (MR4.0) among patients with chronic myeloid leukemia in third-line treatment
Time to MR4.0 was defined as the time from third-line (3L) treatment initiation to the time patients achieved MR4.0 (i.e., 0.0032% IS \< BCR::ABL1 transcript level ≤ 0.01% IS). Patients were censored at the earliest of treatment discontinuation or switch, loss to follow-up, end of data availability, or death.
Time frame: 12, 24, 36, 48, 60, 72 and 84 months post treatment
Time to sustained deep molecular response (MR4.0) among patients with chronic myeloid leukemia in third-line treatment
Time to sustained MR4.0 was defined as the time from third-line (3L) treatment initiation to the time patients achieved sustained MR4.0 or better (i.e., BCR::ABL1 ≤ 0.01%) in all consecutive assessments performed for at least 12 months (i.e., 365.25 days). Patients were censored at the earliest of treatment discontinuation or switch, loss to follow-up, end of data availability, or death.
Time frame: 12, 24, 36, 48, 60, 72 and 84 months post treatment
Time to deep molecular response (MR4.5) among patients with chronic myeloid leukemia in third-line treatment
Time to MR4.5 was defined as the time from third-line (3L) treatment initiation to the time patients achieved MR4.5 (i.e., 0.001% IS ≤ BCR::ABL1 transcript level ≤ 0.0032% IS). Patients were censored at the earliest of treatment discontinuation or switch, loss to follow-up, end of data availability, or death.
Time frame: 12, 24, 36, 48, 60, 72 and 84 months post treatment
Time to sustained deep molecular response (MR4.5) among patients with chronic myeloid leukemia in third-line treatment
Time to sustained MR4.5 was defined as the time from third-line (3L) treatment initiation to the time patients achieved sustained MR4.5 or better (i.e., BCR::ABL1 ≤ 0.0032%) in all consecutive assessments performed for at least 2 years (i.e., 730.5 days). Patients were censored at the earliest of treatment discontinuation or switch, loss to follow-up, end of data availability, or death.
Time frame: 12, 24, 36, 48, 60, 72 and 84 months post treatment
Time to Progression-free survival (PFS) for 3L patients
Time to PFS was defined as the time from third-line treatment initiation to earliest occurrence of documented disease progression to accelerated phase or blast crisis, or the date of death from any cause. Patients were censored at the earliest of treatment discontinuation or switch, loss to follow-up, or end of data availability.
Time frame: 12, 24, 36, 48, 60, 72 and 84 months post treatment
Time to Overall Survival (OS) for 3L patients
Time to OS was defined as the time from third-line treatment initiation to death from any cause. Patients were censored at the earliest of treatment discontinuation or switch, date of last follow-up, or end of data availability.
Time frame: 12, 24, 36, 48, 60, 72 and 84 months post treatment
Time to treatment discontinuation (TTD) for 3L patients
TTD was defined as the time from third-line treatment initiation to treatment discontinuation or switch. Patients were censored at the earliest of loss to follow-up, or end of data availability, or death
Time frame: 12, 24, 36, 48, 60, 72 and 84 months post treatment
Proportion of patients with Adverse Events (AEs) in third-line treatment
Proportion of patients with Adverse Events (AEs) were reported to evaluate the safety profile of third-line treatments for chronic myeloid leukemia
Time frame: throughout the study (study used data from 2000 to 2021)
Age of patients with chronic myeloid leukemia in third-line treatment
Age information was reported.
Time frame: Index date defined as the date of initiation of 3L therapy (data from 2000 to 2021)
Number of patients: Year of chronic myeloid leukemia in chronic phase (CML-CP) diagnosis of patients with chronic myeloid leukemia in third-line treatment
Year of CML-CP diagnosis of patients with chronic myeloid leukemia in third-line treatment was reported.
Time frame: throughout the study (study used data from 2000 to 2021)
Sex of patients with chronic myeloid leukemia in third-line treatment
Sex information was reported.
Time frame: Index date defined as the date of initiation of 3L therapy (data from 2000 to 2021)
Number of patients: Medical center of patients with chronic myeloid leukemia in third-line treatment
The following centers were included: Centre Léon Bérard, Lyon Hématologie Institut Bergonié, Bordeaux Institut Universitaire du Cancer Toulouse, Toulouse
Time frame: Index date defined as the date of initiation of 3L therapy (data from 2000 to 2021)
Length of follow-up (month) characteristics of patients with chronic myeloid leukemia in third-line treatment
Length of follow-up was defined as time from index date to the date of last known contact with patient or patient death.
Time frame: throughout the study (study used data from 2000 to 2021)
Number of patients: Smoking status at index date of patients with chronic myeloid leukemia in third-line treatment
Smoking status at index date of patients with chronic myeloid leukemia in third-line treatment was reported.
Time frame: Index date defined as the date of initiation of 3L therapy (data from 2000 to 2021)
Time from CML-CP diagnosis to index date (month) of patients with chronic myeloid leukemia in third-line treatment
Time from CML-CP diagnosis to index date (month) of patients with chronic myeloid leukemia in third-line treatment was reported.
Time frame: throughout the study (study used data from 2000 to 2021)
Number of patients with Hasford score at CML-CP diagnosis (month) of patients with chronic myeloid leukemia in third-line treatment
Low risk (≤780) Intermediate risk (\>780 to ≤1480) High risk (\>1480) Not assessed Unknown / not sure
Time frame: throughout the study (study used data from 2000 to 2021)
Number of patients with Sokal score at CML-CP diagnosis (month) of patients with chronic myeloid leukemia in third-line treatment
Low risk (\<0.8) Intermediate risk (≥0.8 to ≤1.2) High risk (\>1.2) Not assessed Unknown / not sure
Time frame: throughout the study (data source from 2000 to 2021)
Number of patients with ELTS risk score at CML-CP diagnosis (month) of patients with chronic myeloid leukemia in third-line treatment
ELTS: EUTOS long-term survival Low risk (≤1.5680) Intermediate risk (\>1.5680 to ≤2.2185) High risk (\>2.2185) Not assessed Unknown / not sure
Time frame: throughout the study (data source from 2000 to 2021)
Number of patients with BCR::ABL1 rearrangement at CML-CP diagnosis of patients with chronic myeloid leukemia in third-line treatment
Major Minor Other
Time frame: throughout the study (data source from 2000 to 2021)
Number of patients with Additional chromosomal abnormalities at CML-CP diagnosis of patients with chronic myeloid leukemia in third-line treatment
Yes No
Time frame: throughout the study (study used data from 2000 to 2021)
Number of comorbid conditions prior to the index date in patients with chronic myeloid leukemia in third-line treatment
Number of comorbid conditions prior to the index date in patients with chronic myeloid leukemia in third-line treatment were reported.
Time frame: Baseline (6 months prior to index date, defined as the date of initiation of 3L therapy)
Number of patients with comorbid conditions prior to the index date in patients with chronic myeloid leukemia in third-line treatment
Cardiovascular disease Pulmonary disease/pulmonary arterial hypertension Gastrointestinal issues Renal disease Diabetes Liver disease Other No comorbidities Unknown / not sure
Time frame: Baseline (6 months prior to index date, defined as the date of initiation of 3L therapy)
Number of patients with any ischemic condition prior to the index date in patients with chronic myeloid leukemia in third-line treatment
Cardiovascular disease Cerebrovascular disease Peripheral arterial
Time frame: Baseline (6 months prior to index date, defined as the date of initiation of 3L therapy)
Number of patients with Cardiovascular risk factors prior to the index date in patients with chronic myeloid leukemia in third-line treatment
Hypertension Hyperlipidemia/dyslipidemia Diabetes Obesity
Time frame: Baseline (6 months prior to index date, defined as the date of initiation of 3L therapy)
Laboratory and clinical results: Random blood glucose (mg/dl)
Laboratory and clinical results were reported.
Time frame: throughout the study (study used data from 2000 to 2021)
Laboratory and clinical results: Fasting blood glucose (mg/dl)
Laboratory and clinical results were reported.
Time frame: throughout the study (study used data from 2000 to 2021)
Laboratory and clinical results: LDL cholesterol (mg/dl)
Laboratory and clinical results were reported.
Time frame: throughout the study (study used data from 2000 to 2021)
Laboratory and clinical results: Total cholesterol (mg/dl)
Laboratory and clinical results were reported.
Time frame: throughout the study (study used data from 2000 to 2021)
Laboratory and clinical results: Blood pressure
Laboratory and clinical results were reported.
Time frame: throughout the study (study used data from 2000 to 2021)
Laboratory and clinical results: Spleen size below costal margin
Laboratory and clinical results were reported.
Time frame: throughout the study (study used data from 2000 to 2021)
Laboratory and clinical results: Blood cell count
Laboratory and clinical results were reported.
Time frame: throughout the study (study used data from 2000 to 2021)
Laboratory and clinical results: Bone marrow differential cell count
Laboratory and clinical results were reported.
Time frame: throughout the study (study used data from 2000 to 2021)
Number of patients with BCR::ABL1 mutation status by line of therapy for patients with chronic myeloid leukemia in third-line treatment
Laboratory and clinical results were reported.
Time frame: throughout the study (study used data from 2000 to 2021)
Proportion of patients achieving response among patients with chronic myeloid leukemia with T315I mutation
Laboratory and clinical results were reported.
Time frame: throughout the study (study used data from 2000 to 2021)