Clinical trial NCT05893836: Real-World Disease Management and Outcomes in Chronic Myeloid Leukaemia
Official title: Real-World Disease Management and Outcomes in Chronic Myeloid Leukaemia
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Chronic Myeloid Leukaemia
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2023-06-08
Enrollment: 555
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: This was a retrospective, noninterventional cohort study

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Number of patients who received tyrosine kinase inhibitors (TKIs), by treatment llne
timeFrame: Up to 18 years
measure: Year treatment started, by TKI
timeFrame: Up to 18 years
Secondary outcomes:
measure: Number of patients with response to TKIs
timeFrame: Up to 12 months
measure: Time to response to TKIs
timeFrame: Up to 12 months
measure: Reason for switching TKI
timeFrame: Up to 12 months
measure: Number of patients tested for T315I mutation by treatment line
timeFrame: Up to 12 months
measure: Overall survival (OS)
timeFrame: Up to 10 years
measure: Progression-free survival
timeFrame: Up to 10 years
measure: Time to treatment discontinuation
timeFrame: Up to 10 years
measure: Relative survival by treatment line
timeFrame: Up to 10 years
Other outcomes:
Locations:
city: York
country: United Kingdom
facility: Haematological Malignancy Research Network
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: