Chronic Myeloid Leukemia
Conditions
Keywords
Asciminib, Chronic Myeloid Leukemia, Young Adults, Real-world Evidence, Efficacy and Safety, Major Molecular Response, Unmet Medical Need, Gulf Region
Brief summary
The aim of this study is to evaluate the real-world effectiveness and safety of asciminib among young adults with chronic myeloid leukemia (CML) across the Gulf region. The data source for this study will consist of routinely collected clinical information documented within the electronic health records (EHR) of participating centers.
Interventions
None listed
Sponsors
Novartis Pharmaceuticals
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to 99 Years
Healthy volunteers
No
Inclusion criteria
* Confirmed diagnosis of Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP) * Adult patients aged ≥ 18 years at the index date (or adult as defined by applicable national regulations) * Documented exposure to asciminib during the identification period (May 2023 to May 2026) * Provision of signed informed consent for secondary use of data, or an ethics committee-approved waiver of consent, where applicable (including for deceased patients or where data were previously collected within the EHR system)
Exclusion criteria
* Patients who do not meet the eligibility criteria specified above * Patients with insufficient medical record documentation to determine asciminib exposure or key study outcomes
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Major Molecular Response (MMR) Rate in Young Adults | Approximately 12 Months | MMR is defined as a BCR::ABL1 level on the International Scale (BCR::ABL1 IS) ≤ 0.1%. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of Adverse Events (AEs) | Approximately 3, 6, and 12 months | — |
| Number of Patients by Type and Severity of AEs | Approximately 3, 6, and 12 months | Number of patients by type and severity of AEs including serious AEs and grade ≥ 3 AEs according to the Common Terminology Criteria for Adverse Events (CTCAE) v6.0. |
| Incidence of AEs Leading to Dose Modifications | Approximately 3, 6, and 12 months | Dose modifications include treatment interruptions and dose reductions. |
| Number of Treatment Interruptions Due to AEs | Approximately 3, 6, and 12 months | — |
| Duration of Treatment Interruptions Due to AEs | Approximately 3, 6, and 12 months | — |
| Time to Treatment Discontinuation due to Adverse Events (TTDAE) | Approximately 3, 6, and 12 months | — |
| Proportion of Patients who Achieve an Early Molecular Response Milestone of BCR::ABL1 IS ≤ 10% After 3 Months of Treatment | 3 months | — |
| Proportion of Patients who Achieve an Early Molecular Response Milestone of BCR::ABL1 IS ≤ 1% After 6 Months of Treatment | 6 months | — |
| MMR in Patients Aged > 40 years | Approximately 12 months | MMR is defined as BCR::ABL1 IS ≤ 0.1%. |
| Rate of Deep Molecular Response | Approximately 12 months | Deep molecular response includes: * Molecular response (MR) 4.0 (BCR::ABL1 IS ≤ 0.01%), and * MR4.5 (BCR::ABL1 IS ≤ 0.0032%), where available. |
| Number of Patients With Prior Tyrosine Kinase Inhibitor (TKI) Treatment | Up to 12 months prior to Baseline | — |
| Duration of CML at Time of Asciminib Treatment Initiation | Baseline | — |
| Asciminib Starting Dose | Baseline | — |
| Number of Patients With Dose Modifications | Approximately 3, 6, and 12 months | Number of patients with dose modifications including up titration or dose reduction. |
| Number of Treatment Interruptions | Approximately 3, 6, and 12 months | — |
| Duration of Treatment Interruptions | Approximately 3, 6, and 12 months | — |
| Number of Patients by Reasons for Treatment Interruptions and Subsequent Treatment Switching | Approximately 3, 6, and 12 months | — |
| Number of Patients by Reason for Treatment Switch | Approximately 3, 6, and 12 months | — |
| Number of Patients by Clinicopathological Characteristics | Up to 12 months pre-Baseline, Baseline, and approximately 3, 6 , and 12 months post-Baseline | Clinicopathological characteristics include: * Demographics (gender, ethnicity, nationality, smoking status, and insurance status) * Clinical presentation (signs and symptoms of disease) * Laboratory parameters (lipid panel, complete blood count, clinical chemistry, kidney, liver and pancreas function tests) * Molecular findings (BCR::ABL1 transcript types, additional cytogenetic abnormalities, and other reported mutations) |
Contacts
CONTACTNovartis Pharmaceuticals
STUDY_DIRECTORNovartis Pharmaceuticals
Novartis Pharmaceuticals
Outcome results
None listed