Real-World Disease Management and Outcomes in Chronic Myeloid Leukaemia

Chronic Myeloid Leukaemia Clinical Trial

Official title:

Real-World Disease Management and Outcomes in Chronic Myeloid Leukaemia

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05893836
• Other study ID #: CABL001AGB01
• Status: Completed
• Start date: April 27, 2021
• Est. completion date: June 30, 2021

Study information

• Verified date: June 2023
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 555
• Est. completion date: June 30, 2021
• Est. primary completion date: June 30, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Chronic Myeloid Leukaemia
• Leukemia
• Leukemia, Myelogenous, Chronic, BCR-ABL Positive
• Leukemia, Myeloid

Locations

Country	Name	City	State
United Kingdom	Haematological Malignancy Research Network	York

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of patients who received tyrosine kinase inhibitors (TKIs), by treatment llne		Up to 18 years
Primary	Year treatment started, by TKI		Up to 18 years
Secondary	Number of patients with response to TKIs	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).	Up to 12 months
Secondary	Time to response to TKIs		Up to 12 months
Secondary	Reason for switching TKI		Up to 12 months
Secondary	Number of patients tested for T315I mutation by treatment line		Up to 12 months
Secondary	Overall survival (OS)	OS was defined as the time (in years) from initiation of treatment (i.e., the index date) to death (any cause).	Up to 10 years
Secondary	Progression-free survival	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.	Up to 10 years
Secondary	Time to treatment discontinuation		Up to 10 years
Secondary	Relative survival by treatment line	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.	Up to 10 years