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.
Study Type
OBSERVATIONAL
Enrollment
37
Novartis Investigative Site
Boston, Massachusetts, United States
Novartis Investigative Site
Sydney, New South Wales, Australia
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.
Time frame: 3 months
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.
Time frame: 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).
Time frame: 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.
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Novartis Investigative Site
Montreal, Canada
Novartis Investigative Site
Hong Kong, China
Novartis Investigative Site
Le Chesnay, France
Novartis Investigative Site
Ramat Gan, Israel
Novartis Investigative Site
Ascoli Piceno, Italy
Novartis Investigative Site
Bari, Italy
Novartis Investigative Site
Catania, Italy
Novartis Investigative Site
Cuneo, Italy
...and 8 more locations
Time frame: 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.
Time frame: 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: * No circulating lymphoblasts or extramedullary disease * No lymphadenopathy, splenomegaly, skin/gum infiltration, testicular mass, central nervous system (CNS) involvement. * Trilineage hematopoiesis (TLH) and \<5% blasts. * ANC ≥1000/μL. * Platelets ≥100,000/μL.
Time frame: 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: * Platelets \<100,000/μL and ANC ≥1000/μL or * Platelets ≥100,000/μL and ANC \<1000/μL
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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)
Time frame: Up to 5 years and 4 months
Proportion of Patients by Reasons for Starting Asciminib
Time frame: 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.
Time frame: 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.
Time frame: Up to 5 years and 4 months
Proportion of Patients With Adverse Events
Time frame: Up to 5 years and 4 months