Clinical trial NCT05619978: French Study to Highlight the Unmet Treatment Needs of 3L+ CP-CML and With T315I-mutated CML Patients
Official title: A Multi-Center Retrospective Chart Review Study Examining the Patient Characteristics, Treatment Patterns, Clinical Outcomes, and Burden of Illness Among Patients With Chronic Myeloid Leukemia in Third-line Treatment or With T315I Mutation in France (CML 3L+ & T315I)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Chronic Myeloid Leukemia
Keywords: BCR::ABL,
Chronic myeloid leukemia,
Tyrosine kinase inhibitor,
T315I mutation
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2022-11-17
Enrollment: 200
Sex: ALL
Minimum age (years): 18
Maximum age (years): 99
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The study included CML-CP patients (i.e., 3L patients and patients with T315I mutation) who met the eligibility criteria

Brief summary: A retrospective multi-center cohort study design was used to address the study objectives, using medical records obtained from three clinical centers in France.

Detailed description: 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.

Eligibility criteria: Inclusion Criteria:

* Diagnosed with CML-CP
* Age ≥18 years at the time of CML-CP diagnosis
* For 3L patients: initiated one of the following 3L therapies in CML-CP after failing on ≥2 TKIs (i.e., bosutinib, dasatinib, imatinib, nilotinib, or ponatinib) or allo-SCT
* For T315I mutation patients: evidence of T315I mutation and treatment with TKI or allo-SCT

Exclusion Criteria:

* History of other active malignancies within the 3 years prior to the time of CML-CP diagnosis
* Documentation of anti-cancer therapies for any other malignancies prior to the time of 3L therapy initiation or at the time of treatment initiation after identification of T315I mutation
* Enrollment in a clinical trial at the time of 3L therapy initiation or at the time of treatment initiation after identification of T315I mutation

Primary outcomes:
measure: Number of patients: Year patient initiated third-line treatment
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Duration of third-line treatment
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Number of patients: Type of therapy received in third-line treatment
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Time from 2L discontinuation to third-line treatment initiation
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Number of patients still on 3L therapy as of data collection date
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Number of patients discontinued 3L
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Number of patients: Reasons for 3L discontinuation
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Number of lines of therapy for patients with chronic myeloid leukemia in third-line treatment
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Number of most frequent treatment sequences for patients with chronic myeloid leukemia in third-line treatment
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Number of last line of therapy for patients with chronic myeloid leukemia in third-line treatment
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Number of patients: Year patient initiated on the line identified as T315I line of interest
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Duration of the line identified as T315I line of interest
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Number of patients: Type of therapy received in the line identified as T315I line of interest
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Number of patients still on line of therapy identified as T315I line of interest as of data collection date
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Number of patients: Reasons for discontinuation on the line identified as T315I line of interest
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Number of lines of therapy for patients with chronic myeloid leukemia with T315I mutation
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Number of line identified as T315I line of interest
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Number of treatment sequence for patients with chronic myeloid leukemia with T315I mutation
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Number of last line of therapy for patients with chronic myeloid leukemia with T315I mutation
timeFrame: throughout the study (study used data from 2000 to 2021)
Secondary outcomes:
measure: Cytogenetic response (CyR) for 3L patients
timeFrame: 12 and 24 months
measure: Proportion of patients achieving response among patients with chronic myeloid leukemia in third-line treatment in 12 months
timeFrame: 12 months post treatment
measure: Proportion of patients achieving response among patients with chronic myeloid leukemia in third-line treatment in 24 months
timeFrame: 24 months post treatment
measure: Proportion of patients achieving sustained molecular responses among patients with chronic myeloid leukemia in third-line treatment in 12 months
timeFrame: 12 months post treatment
measure: Proportion of patients achieving sustained molecular responses among patients with chronic myeloid leukemia in third-line treatment in 24 months
timeFrame: 24 months post treatment
measure: Time to major molecular response (MMR) among patients with chronic myeloid leukemia in third-line treatment
timeFrame: 12, 24, 36, 48, 60, 72 and 84 months post treatment
measure: Time to deep molecular response (MR4.0) among patients with chronic myeloid leukemia in third-line treatment
timeFrame: 12, 24, 36, 48, 60, 72 and 84 months post treatment
measure: Time to sustained deep molecular response (MR4.0) among patients with chronic myeloid leukemia in third-line treatment
timeFrame: 12, 24, 36, 48, 60, 72 and 84 months post treatment
measure: Time to deep molecular response (MR4.5) among patients with chronic myeloid leukemia in third-line treatment
timeFrame: 12, 24, 36, 48, 60, 72 and 84 months post treatment
measure: Time to sustained deep molecular response (MR4.5) among patients with chronic myeloid leukemia in third-line treatment
timeFrame: 12, 24, 36, 48, 60, 72 and 84 months post treatment
measure: Time to Progression-free survival (PFS) for 3L patients
timeFrame: 12, 24, 36, 48, 60, 72 and 84 months post treatment
measure: Time to Overall Survival (OS) for 3L patients
timeFrame: 12, 24, 36, 48, 60, 72 and 84 months post treatment
measure: Time to treatment discontinuation (TTD) for 3L patients
timeFrame: 12, 24, 36, 48, 60, 72 and 84 months post treatment
measure: Proportion of patients with Adverse Events (AEs) in third-line treatment
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Age of patients with chronic myeloid leukemia in third-line treatment
timeFrame: Index date defined as the date of initiation of 3L therapy (data from 2000 to 2021)
measure: Number of patients: Year of chronic myeloid leukemia in chronic phase (CML-CP) diagnosis of patients with chronic myeloid leukemia in third-line treatment
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Sex of patients with chronic myeloid leukemia in third-line treatment
timeFrame: Index date defined as the date of initiation of 3L therapy (data from 2000 to 2021)
measure: Number of patients: Medical center of patients with chronic myeloid leukemia in third-line treatment
timeFrame: Index date defined as the date of initiation of 3L therapy (data from 2000 to 2021)
measure: Length of follow-up (month) characteristics of patients with chronic myeloid leukemia in third-line treatment
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Number of patients: Smoking status at index date of patients with chronic myeloid leukemia in third-line treatment
timeFrame: Index date defined as the date of initiation of 3L therapy (data from 2000 to 2021)
measure: Time from CML-CP diagnosis to index date (month) of patients with chronic myeloid leukemia in third-line treatment
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Number of patients with Hasford score at CML-CP diagnosis (month) of patients with chronic myeloid leukemia in third-line treatment
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Number of patients with Sokal score at CML-CP diagnosis (month) of patients with chronic myeloid leukemia in third-line treatment
timeFrame: throughout the study (data source from 2000 to 2021)
measure: Number of patients with ELTS risk score at CML-CP diagnosis (month) of patients with chronic myeloid leukemia in third-line treatment
timeFrame: throughout the study (data source from 2000 to 2021)
measure: Number of patients with BCR::ABL1 rearrangement at CML-CP diagnosis of patients with chronic myeloid leukemia in third-line treatment
timeFrame: throughout the study (data source from 2000 to 2021)
measure: Number of patients with Additional chromosomal abnormalities at CML-CP diagnosis of patients with chronic myeloid leukemia in third-line treatment
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Number of comorbid conditions prior to the index date in patients with chronic myeloid leukemia in third-line treatment
timeFrame: Baseline (6 months prior to index date, defined as the date of initiation of 3L therapy)
measure: Number of patients with comorbid conditions prior to the index date in patients with chronic myeloid leukemia in third-line treatment
timeFrame: Baseline (6 months prior to index date, defined as the date of initiation of 3L therapy)
measure: Number of patients with any ischemic condition prior to the index date in patients with chronic myeloid leukemia in third-line treatment
timeFrame: Baseline (6 months prior to index date, defined as the date of initiation of 3L therapy)
measure: Number of patients with Cardiovascular risk factors prior to the index date in patients with chronic myeloid leukemia in third-line treatment
timeFrame: Baseline (6 months prior to index date, defined as the date of initiation of 3L therapy)
measure: Laboratory and clinical results: Random blood glucose (mg/dl)
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Laboratory and clinical results: Fasting blood glucose (mg/dl)
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Laboratory and clinical results: LDL cholesterol (mg/dl)
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Laboratory and clinical results: Total cholesterol (mg/dl)
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Laboratory and clinical results: Blood pressure
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Laboratory and clinical results: Spleen size below costal margin
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Laboratory and clinical results: Blood cell count
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Laboratory and clinical results: Bone marrow differential cell count
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Number of patients with BCR::ABL1 mutation status by line of therapy for patients with chronic myeloid leukemia in third-line treatment
timeFrame: throughout the study (study used data from 2000 to 2021)
measure: Proportion of patients achieving response among patients with chronic myeloid leukemia with T315I mutation
timeFrame: throughout the study (study used data from 2000 to 2021)
Other outcomes:
Locations:
city: Lyon
country: France
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: