Real-World Disease Management and Outcomes in Chronic Myeloid Leukaemia

Completed

• Conditions: Chronic Myeloid Leukaemia

• Registration Number: NCT05893836
• Lead Sponsor: Novartis Pharmaceuticals

Brief Summary

Chronic myeloid leukaemia (CML) diagnosis is based on the demonstration of a BCR-ABL fusion transcript expressed by the Philadelphia (Ph) chromosome by RQ-PCR and/or the demonstration of t(9;22)(q34;q11) by conventional karyotyping or interphase FISH. As per standard practice, response to therapy is monitored using either molecular or cytogenetic tests or both; specifically, patients are monitored by quantitative PCR on peripheral blood, supplemented by bone marrow karyotyping if it was clinically indicated. ABL kinase mutational analysis is carried out when the transcript ratio has increased over two sequential samples or on clinical demand. Testing for T315I mutation is also performed for patients who fail to respond to first line TKI and all patients who acquire TKI resistance over the course of their treatment.

Data collection is initiated six months after date of diagnosis; research nurses working to agreed operating procedures and data standards visit each of the 14 hospitals in the region and abstract a core clinical dataset from the patients' medical records. The information collected includes demographic details, baseline blood count data and first line treatment. All details are abstracted onto structured forms and entered onto the web-based system, which integrates Haematological Malignancy Research Network (HMRN) and Haematological Malignancy Diagnostic Service (HMDS) data. An important feature of data acquisition is the emphasis on primary source information; data from radiology reports, blood tests, clinical examination, and clinician summaries are recorded, enabling embedded algorithms in the database system to automatically generate stage and prognostic scores. Further data abstraction from the medical records has been undertaken to capture information on subsequent treatment lines. Information on date and cause of death were obtained from the National Health Service (NHS) Central Register.

Detailed Description

Not available

Study Timeline

• Study Start: 2021-04-27
• Primary Completion: 2021-06-30
• Study Completion: 2021-06-30
• Study First Submitted: 2023-05-17
• Status Verified: 2023-06
• Study First Submitted QC: 2023-06-07
• Last Update Submitted: 2023-06-07
• Study First Posted: 2023-06-08
• Last Update Posted: 2023-06-08

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 555

Inclusion Criteria

• Adult (18+ years) patients newly diagnosed with CML in chronic phase (ICD-O-3: 9875/3) by HMDS between 1st September, 2004 to 31st August, 2019 whilst resident in the HMRN region and treated within the Network.

Exclusion Criteria

None specified.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Number of patients who received tyrosine kinase inhibitors (TKIs), by treatment llne	Up to 18 years
Year treatment started, by TKI	Up to 18 years

Secondary Outcome Measures

Name	Time	Method
Reason for switching TKI	Up to 12 months
Time to treatment discontinuation	Up to 10 years
Progression-free survival	Up to 10 years	PFS was defined from the initiation of treatment (i.e., the index date) to the earliest documentation of disease progression to accelerated phase/blast crisis (AP/BC) or date of death from any cause.
Time to response to TKIs	Up to 12 months
Number of patients tested for T315I mutation by treatment line	Up to 12 months
Overall survival (OS)	Up to 10 years	OS was defined as the time (in years) from initiation of treatment (i.e., the index date) to death (any cause).
Number of patients with response to TKIs	Up to 12 months	Disease response was defined as either a major molecular response (MMR, ≤ 0.1% BCR-ABL1) or as an MR2, which is a molecular response (MR, ≤ 1.0% BCR-ABL1), or complete cytogenetic remission (CCyR).
Relative survival by treatment line	Up to 10 years	Relative survival (RS) was estimated to examine the CML-specific mortality rate. The Stata program strel (v1.2.7) was used to estimate RS and corresponding 95% Confidence Intervals (95%CI); with age and sex-specific background mortality rates being obtained from national life tables.

Trial Locations

Locations (1)

Haematological Malignancy Research Network; 🇬🇧 York, United Kingdom