C92.1
Conditions
Interventions
Group 1: Asciminib cohort: Approximately 300 patients, including both newly diagnosed and pre-treated individuals. At least 50% of patients are expected to be treated in first-line. In case of substan
n ˜ 50). These patients will be included for cross-sectional baseline comparison only and will not be followed longitudinally. These patients are included in the cross-sectional baseline comparison (i
Sponsors
Novartis Pharma GmbH
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Inclusion criteria
Inclusion criteria: 1. Written informed consent must be obtained before participation in the study/prior to any study-related documentation. 2. Adult patients (=18 years of age) with a confirmed diagnosis of Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP). 3. Patients who are either newly diagnosed or have received treatment with exactly one prior tyrosine kinase inhibitor (TKI). Prior TKI treatment is only permitted for patients in the asciminib cohort. Patients in the comparator cohorts (imatinib, dasatinib, bosutinib, nilotinib) must be newly diagnosed and must not have received any prior TKI treatment. 4. Patients for whom the treating physician has made a clinical decision to initiate treatment with asciminib or another TKI (imatinib, dasatinib, bosutinib, nilotinib) as part of routine care. The clinical decision for treatment must have been made prior to enrollment. To ensure accurate baseline assessments, treatment must not have started more than 14 days before study inclusion, and treatment may also begin after baseline assessment. 5. Patients who are expected to participate in routine follow-up visits and complete patient-reported outcome questionnaires over the course of the study.
Exclusion criteria
Exclusion criteria: Study participants meeting any of the following criteria are not eligible for inclusion in this study: 1. Patients with contraindications to their respective CML treatment as per the applicable Summary of Product Characteristics (SmPC) and relevant national treatment guidelines (e.g. Onkopedia CML), including the following asciminib-specific considerations: - In first- or second-line treatment: presence of BCR::ABL1 fusion transcripts lacking exon a2 (e.g. e13a3, e14a3). - In second-line treatment: known BCR::ABL1 mutations associated with partial or complete resistance to asciminib (e.g. M244V, F359I/V/C;T315I) 2. Patients receiving or planned to receive asciminib or other TKIs outside the approved label (off-label use), including use in unapproved dosing regimens or frequency not covered by the respective SmPC. 3. Patients currently participating in an interventional clinical trial. 4. Patients unable or unwilling to provide written informed consent. 5. Patients who are unable to reliably complete patient-reported outcome questionnaires due to cognitive or language limitations relevant to the study assessments. 6. Patients for whom long-term follow-up is not feasible due to expected relocation or other logistical constraints. 7. The restriction to a maximum of one prior ATP-competitive TKI applies exclusively to patients treated with asciminib. Patients in the comparator cohorts (imatinib, dasatinib, bosutinib, nilotinib) must be TKI-naïve and are included at the start of their first-line TKI therapy and are not eligible if they have received any prior TKI treatment.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To assess the real-world effectiveness of asciminib by estimating the proportion of adult patients with early chronic phase Ph+ CML-CP who achieve Major Molecular Response (MMR) at visit 4 (~ 12 months after baseline) in routine clinical practice in Germany. | — |
Secondary
| Measure | Time frame |
|---|---|
| Description of available baseline characteristics (e.g. detection of the Philadelphia chromosome or BCR::ABL1 transcript, if applicable, prior pharmacological treatment of the primary disease (in second line of therapy), ECOG performance status) by treatment cohort Reason for treatment decision documented by the treating physician Proportion of patients with dose reduction and underlying reason Proportion of patients with treatment interruption and underlying reason Proportion of patients who discontinued treatment and underlying reason Time to treatment discontinuation, interruption or dose reduction Time to treatment discontinuation due to adverse events (TTDAE) Molecular response (EMR, MR2, MMR, MR4.0, MR4.5) Patient questionnaire MARS-5, EORTC QLQ-C30 and EORTC QLQ-CML24 Patient-reported ecosocial impact assessed using the WPAIGH questionnaire | — |
Countries
Germany
Contacts
Public ContactAndreas Hochhaus
Universitätsklinikum Jena Abteilung für Hämatologie und Internistische Onkologie der KIM II
Outcome results
None listed