A Study of Asciminib Safety and Efficacy in Young Adults With Chronic Myeloid Leukemia in the Gulf Region

Primary outcomes

• 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 outcomes

• 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)

Sex: All, ages: 18 Years to 99 Years

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

• Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals