Real-World Disease Management and Outcomes in Chronic Myeloid Leukaemia

NCT05893836 | Completed

• 1 other identifier: CABL001AGB01
• Study Type: observational
• Target: 555
• Locations: 1 country
• Sites: 1

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.

Conditions

Chronic Myeloid Leukaemia

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (8)

• Number of patients with response to TKIs

  Up to 12 months

• Time to response to TKIs

  Up to 12 months

• Reason for switching TKI

  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

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

This was a retrospective, noninterventional cohort study

You may qualify if:

• 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.

You may not qualify if:

• None specified.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (1)

Haematological Malignancy Research Network

York, United Kingdom

Location

MeSH Terms

Conditions

Leukemia, Myelogenous, Chronic, BCR-ABL Positive

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Myeloproliferative Disorders; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 17, 2023
• First Posted: June 8, 2023
• Study Start: April 27, 2021
• Primary Completion: June 30, 2021
• Study Completion: June 30, 2021
• Last Updated: June 8, 2023

Record last verified: 2023-06

Locations

GB (1)