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The study was a retrospective, non-interventional patient chart review and used a panel of oncologists/hematologists from the US to collect real-world clinical outcomes of patients with CML-CP in 3L+ and those with the T315I mutation.
This study included two distinct cohorts of CML-CP patients; that is, patients with CML-CP who initiated 3L for CML-CP (i.e., 3L cohort) and patients with CML-CP with T315I mutation (i.e., T315I cohort).
Study design for the analyses of the 3L cohort:
Study design for the analyses of the T315I cohort:
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Third-line (3L) Cohort | patients with Chronic Myeloid Leukemia - Chronic Phase (CML-CP) who initiated 3L for CML-CP | ||
| T315I Cohort | patients with CML-CP with T315I mutation |
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| Measure | Description | Time Frame |
|---|---|---|
| Number of lines of therapy | To evaluate treatment patterns in patients with CML-CP who were previously treated with TKI or other CML treatments and were relapsed/refractory to/were intolerant/had other reasons for switching of CML therapy | throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018) |
| Number of patients with total number of lines | To evaluate treatment patterns in patients with CML-CP who were previously treated with TKI or other CML treatments and were relapsed/refractory to/were intolerant/had other reasons for switching of CML therapy | throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018) |
| Number of patients: Treatment received | To evaluate treatment patterns in patients with CML-CP who were previously treated with TKI or other CML treatments and were relapsed/refractory to/were intolerant/had other reasons for switching of CML therapy | throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018) |
| Number of patients: Calendar year of line of therapy initiation | To evaluate treatment patterns in patients with CML-CP who were previously treated with TKI or other CML treatments and were relapsed/refractory to/were intolerant/had other reasons for switching of CML therapy | throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018) |
| Duration of line of therapy | To evaluate treatment patterns in patients with CML-CP who were previously treated with TKI or other CML treatments and were relapsed/refractory to/were intolerant/had other reasons for switching of CML therapy |
| Measure | Description | Time Frame |
|---|---|---|
| Number of patients who achieved molecular response during third-line therapy | To evaluate the effectiveness of TKIs and other CML treatments used in 3L+ settings in patients with CML-CP | throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018) |
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Inclusion Criteria:
Physician selection
Physicians were eligible to participate in the study if they fulfilled all of the following criteria:
Patient selection Participating physicians were directed to provide information on patients who were included into the following separate cohorts. Each participating physician contributed up to 5 patient medical charts from each cohort.
For the 3L cohort:
For the T315I cohort:
For both cohorts:
Exclusion Criteria:
- Physicians and patients who did not meet study inclusion criteria detailed above were excluded.
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Patients with Chronic Myeloid Leukemia and those with T315I Mutation in the United States
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis Investigative Site | East Hanover | New Jersey | 07936 | United States |
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| Label | URL |
|---|---|
| Results for CABL001AUS09 from the Novartis Clinical Trials Website | View source |
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| ID | Term |
|---|---|
| D015464 | Leukemia, Myelogenous, Chronic, BCR-ABL Positive |
| ID | Term |
|---|---|
| D007951 | Leukemia, Myeloid |
| D007938 | Leukemia |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
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| throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018) |
| Number of patients with most frequent treatment sequences from first- to third-line of therapy | To evaluate treatment patterns in patients with CML-CP who were previously treated with TKI or other CML treatments and were relapsed/refractory to/were intolerant/had other reasons for switching of CML therapy | throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018) |
| Number of patients who died after initiation of third-line therapy | To evaluate treatment patterns in patients with CML-CP who were previously treated with TKI or other CML treatments and were relapsed/refractory to/were intolerant/had other reasons for switching of CML therapy | throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018) |
| Number of patients who progressed to AP/BC after initiation of third-line therapy | To evaluate treatment patterns in patients with CML-CP who were previously treated with TKI or other CML treatments and were relapsed/refractory to/were intolerant/had other reasons for switching of CML therapy | throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018) |
| Number of patients who underwent HSCT after initiation of third-line therapy | To evaluate treatment patterns in patients with CML-CP who were previously treated with TKI or other CML treatments and were relapsed/refractory to/were intolerant/had other reasons for switching of CML therapy | throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018) |
| Number of patients developed graft versus host disease after undergoing HSCT3 | To evaluate treatment patterns in patients with CML-CP who were previously treated with TKI or other CML treatments and were relapsed/refractory to/were intolerant/had other reasons for switching of CML therapy | throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018) |
| Number of patients who were still on the third-line therapy as of the data collection date | To evaluate treatment patterns in patients with CML-CP who were previously treated with TKI or other CML treatments and were relapsed/refractory to/were intolerant/had other reasons for switching of CML therapy | throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018) |
| Number of patients who achieved cytogenic response during third-line therapy |
To evaluate the effectiveness of TKIs and other CML treatments used in 3L+ settings in patients with CML-CP |
| throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018) |
| Number of patients who achieved complete hematologic response during third-line therapy | To evaluate the effectiveness of TKIs and other CML treatments used in 3L+ settings in patients with CML-CP | throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018) |
| Number of patients who achieved molecular response during the line of therapy identified as the T315I line of interest | To evaluate the effectiveness of TKIs and other treatments used in a line of therapy identified as the T315I line of interest in patients with CML-CP | throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018) |
| Number of patients who achieved cytogenic response during the line of therapy identified as the T315I line of interest | To evaluate the effectiveness of TKIs and other treatments used in a line of therapy identified as the T315I line of interest in patients with CML-CP | throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018) |
| Number of patients who achieved complete hematologic response during the line of therapy identified as the T315I line of interest | To evaluate the effectiveness of TKIs and other treatments used in a line of therapy identified as the T315I line of interest in patients with CML-CP | throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018) |
| Number of lines of therapy in T315I cohort | To evaluate treatment patterns in patients with CML-CP with T315I mutation | throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018) |
| Number of patients: Total number of lines in T315I cohort | To evaluate treatment patterns in patients with CML-CP with T315I mutation | throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018) |
| Number pf patients: Line identified as the T315I line of interest | To evaluate treatment patterns in patients with CML-CP with T315I mutation | throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018) |
| Number of patients: Treatment received in T315I Cohort | To evaluate treatment patterns in patients with CML-CP with T315I mutation | throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018) |
| Number of patients: Calendar year of line of therapy initiation in T315I Cohort | To evaluate treatment patterns in patients with CML-CP with T315I mutation | throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018) |
| Duration of line of therapy in T315I Cohort | To evaluate treatment patterns in patients with CML-CP with T315I mutation | throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018) |
| Most frequent treatment sequences in T315I Cohort | To evaluate treatment patterns in patients with CML-CP with T315I mutation | throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018) |
| Number of patients who died after initiation of the line identified as the T315I line of interest | To evaluate treatment patterns in patients with CML-CP with T315I mutation | throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018) |
| Number of patients who progressed to AP/BC after initiation of the line identified as the T315I line of interest | To evaluate treatment patterns in patients with CML-CP with T315I mutation | throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018) |
| Number of patients who underwent HSCT after initiation of the line identified as the T315I line of interest | To evaluate treatment patterns in patients with CML-CP with T315I mutation | throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018) |
| Number of patients developed graft versus host disease after undergoing HSCT | To evaluate treatment patterns in patients with CML-CP with T315I mutation | throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018) |
| Number of patients who were still on the line identified as the T315I line of interest as of the data collection date | To evaluate treatment patterns in patients with CML-CP with T315I mutation | throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018) |
| Number of patients with BCR-ABL testing in 3L cohort | To evaluate real-world BCR-ABL testing frequency per the latest National Comprehensive Cancer Network (NCCN) guidelines in 3L settings in patients with CML-CP | throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018) |
| Number of patients with BCR-ABL testing in T315I cohort | To evaluate real-world BCR-ABL testing frequency per the latest NCCN guidelines in patients with CML-CP with T315I mutation | throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018) |
| D009196 |
| Myeloproliferative Disorders |
| D001855 | Bone Marrow Diseases |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D002908 | Chronic Disease |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |