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The aim of this study was to collect existing information from the medical charts of patients enrolled in the ongoing asciminib Managed Access Program (MAP) to better understand the effectiveness and safety of asciminib when used to treat adult patients with Ph+ ALL who are refractory, resistant or intolerant to available treatments.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Asciminib Cohort | Ph+ ALL patients who received at least one dose of asciminib through the asciminib MAP. |
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| Measure | Description | Time Frame |
|---|---|---|
| Proportion of Patients Achieving Hematological Complete Remission (CR) or Hematological Complete Remission With Incomplete Count Recovery (CRi) in the First 3 Months of Treatment | Hematological CR was defined as no circulating lymphoblasts, absolute neutrophil count (ANC) ≥1,000/μL, platelets ≥100K/μL. Hematological CRi was defined as no circulating lymphoblasts, ANC ˂1,000/μL, platelets ˂100K/μL. | 3 months |
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of Patients With Minimal Residual Disease (MRD) Evaluated in Peripheral Blood at Best Response | MRD, defined as the percentage of leukemia cells remaining after treatment with asciminib with the use of flow cytometry, polymerase chain reaction (PCR), and/or next generation sequencing (NGS) techniques (flow or molecular based MRD is defined as non-detectable if less than 0.01%. NGS MRD is defined as non-detected if less than 1 in 1 x 10^6 cells). Best response: CR or CRi achievement. CR was defined as no circulating lymphoblasts, ANC ≥1,000/μL, platelets ≥100K/μL. CRi was defined as no circulating lymphoblasts, ANC ˂1,000/μL, platelets ˂100K/μL. |
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Inclusion criteria
Adult patients enrolled in the asciminib MAP.
Diagnosis of Ph+ ALL.
Patients received at least one dose of asciminib through the asciminib MAP.
Appropriate approval obtained for the use of patient data including:
Exclusion criteria
• Age < 18 years at the time of initiating asciminib treatment.
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Patients with Ph+ ALL who were enrolled in the asciminib MAP and received at least one dose of asciminib.
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis Investigative Site | Boston | Massachusetts | 02115 | United States | ||
| Novartis Investigative Site |
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| From Day 1 to 120, and at Baseline, Day 28, 60, 90, 180, 270, 360 |
| Proportion of Patients Who Showed MRD Positivity and MRD Negativity | MRD, defined as the percentage of leukemia cells remaining after treatment with asciminib with the use of flow cytometry, PCR, and/or NGS techniques (flow or molecular based MRD is defined as non-detectable if less than 0.01%. NGS MRD is defined as non-detected if less than 1 in 1 x 10^6 cells). | Baseline, Day 28, 60, 90, 180, 270, 360 |
| Duration of Response (DoR) | DoR measured as the time from achievement of CR or CRi, whichever occurs first, to relapse or death due to acute lymphoblastic leukemia (ALL) at the time of data cut-off. CR was defined as no circulating lymphoblasts, ANC ≥1,000/μL, platelets ≥100K/μL. CRi was defined as no circulating lymphoblasts, ANC ˂1,000/μL, platelets ˂100K/μL. | Up to 5 years and 4 months |
| Proportion of Patients in Hematological CR or CRi Within 13 Months From Start of Treatment | Hematological CR was defined as no circulating lymphoblasts, ANC ≥1,000/μL, platelets ≥100K/μL. Hematological CRi was defined as no circulating lymphoblasts, ANC ˂1,000/μL, platelets ˂100K/μL. | By Day 28 ± 7 (Day 1 to Day 35), at Day 90 ± 30, by Day 90 ± 30 (Day 1 to Day 120), at Day 180 ± 30, by Day 180 ± 30 (Day 1 to Day 210), at Day 270 ± 30, by Day 270 ± 30 (Day 1 to Day 300), at Day 360 ± 30, by Day 360 ± 30 (Day 1 to Day 390) |
| Proportion of Patients in Complete Remission | Proportion of patients in complete remission (as per peripheral blood and bone marrow, when both available). Complete Remission was defined as:
| Day 28 ± 7 (Day 1 to Day 35), at Day 90 ± 30, by Day 90 ± 30 (Day 1 to Day 120), at Day 180 ± 30, by Day 180 ± 30 (Day 1 to Day 210), at Day 270 ± 30, by Day 270 ± 30 (Day 1 to Day 300), at Day 360 ± 30, by Day 360 ± 30 (Day 1 to Day 390) |
| Proportion of Patients in Complete Remission With Incomplete Recovery | Proportion of patients in complete remission with incomplete recovery (as per peripheral blood and bone marrow, when both available). Complete remission with incomplete recovery was defined as meeting all criteria for complete remission except without recovery of platelet count or without recovery of ANC:
| Day 28 ± 7 (Day 1 to Day 35), at Day 90 ± 30, by Day 90 ± 30 (Day 1 to Day 120), at Day 180 ± 30, by Day 180 ± 30 (Day 1 to Day 210), at Day 270 ± 30, by Day 270 ± 30 (Day 1 to Day 300), at Day 360 ± 30, by Day 360 ± 30 (Day 1 to Day 390) |
| Proportion of Patients who Proceed to Stem Cell Transplantation (SCT) | Proportion of patients who proceed to SCT by the cutoff date. | Up to 5 years and 4 months |
| Proportion of Patients who Continued Treatment With Asciminib After Last SCT | Proportion of patients who proceed to SCT and continue treatment with asciminib by the cutoff date. | Up to 5 years and 4 months |
| Proportion of Patients With BCR::ABL1 T315I Mutation at Baseline and Correlation With Hematological CR/CRi With Asciminib Monotherapy or as a Combination by the Cutoff Date | Hematological CR was defined as no circulating lymphoblasts, ANC ≥1,000/μL, platelets ≥100K/μL. Hematological CRi was defined as no circulating lymphoblasts, ANC ˂1,000/μL, platelets ˂100K/μL. | Up to 5 years and 4 months |
| Overall Survival (OS) | OS was defined as the time from start of treatment with asciminib to death due to any cause. | Up to 5 years and 4 months |
| Relapse-free Survival (RFS) | RFS was defined as the time from achievement of hematological CR or CRi, whichever occurs first, to relapse or death due to any cause. Hematological CR was defined as no circulating lymphoblasts, ANC ≥1,000/μL, platelets ≥100K/μL. Hematological CRi was defined as no circulating lymphoblasts, ANC ˂1,000/μL, platelets ˂100K/μL. | Up to 5 years and 4 months |
| Proportion of Patients Intolerant to Prior Therapy and not in Hematological CR or CRi at Baseline who Achieved CR or CRi as the Best Response | Hematological CR was defined as no circulating lymphoblasts, ANC ≥1,000/μL, platelets ≥100K/μL. Hematological CRi was defined as no circulating lymphoblasts, ANC ˂1,000/μL, platelets ˂100K/μL. | During the first 3 months of treatment and at any time point until Day 360 ± 30 |
| Among Patients Intolerant to Prior Therapy With MRD Negativity at Baseline, Duration of MRD | MRD, defined as the percentage of leukemia cells remaining after treatment with asciminib with the use of flow cytometry, PCR, and/or NGS techniques (flow or molecular based MRD is defined as non-detectable if less than 0.01%. NGS MRD is defined as non-detected if less than 1 in 1 x 10^6 cells) | Up to 5 years and 4 months |
| Proportion of Patients by Reasons for Starting Asciminib | Up to 5 years and 4 months |
| Proportion of Patients Who Achieved CR or CRi by Patient Characteristics | Patient characteristics included age group, gender, race and ethnicity, medical history, prior treatments, relapse/remission status, and treatment phase. | Up to 5 years and 4 months |
| Proportion of Patients by DoR and Patient Characteristics | DoR measured as the time from achievement of CR or CRi, whichever occurs first, to relapse or death due to ALL at the time of data cut-off. CR was defined as no circulating lymphoblasts, ANC ≥1,000/μL, platelets ≥100K/μL. CRi was defined as no circulating lymphoblasts, ANC ˂1,000/μL, platelets ˂100K/μL. Patient characteristics included age group, gender, race and ethnicity, medical history, prior treatments, relapse/remission status, and treatment phase. | Up to 5 years and 4 months |
| Proportion of Patients With Adverse Events | Up to 5 years and 4 months |
| Sydney |
| New South Wales |
| 2145 |
| Australia |
| Novartis Investigative Site | Montreal | H1T 2M4 | Canada |
| Novartis Investigative Site | Hong Kong | China |
| Novartis Investigative Site | Le Chesnay | 78150 | France |
| Novartis Investigative Site | Ramat Gan | 52621 | Israel |
| Novartis Investigative Site | Ascoli Piceno | 63100 | Italy |
| Novartis Investigative Site | Bari | 70124 | Italy |
| Novartis Investigative Site | Catania | 95123 | Italy |
| Novartis Investigative Site | Cuneo | 12100 | Italy |
| Novartis Investigative Site | Pescara | 65124 | Italy |
| Novartis Investigative Site | Torino | 10126 | Italy |
| Novartis Investigative Site | Rotterdam | 3015 | Netherlands |
| Novartis Investigative Site | Islamabad | 44000 | Pakistan |
| Novartis Investigative Site | Rawalpindi | 46000 | Pakistan |
| Novartis Investigative Site | Bialystok | 15-276 | Poland |
| Novartis Investigative Site | Madrid | 28006 | Spain |
| Novartis Investigative Site | London | SE5 9RS | United Kingdom |
| ID | Term |
|---|---|
| D054198 | Precursor Cell Lymphoblastic Leukemia-Lymphoma |
| ID | Term |
|---|---|
| D007945 | Leukemia, Lymphoid |
| D007938 | Leukemia |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D008232 | Lymphoproliferative Disorders |
| D008206 | Lymphatic Diseases |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
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