Acute Lymphoblastic Leukemia
Conditions
Keywords
Asciminib, Ph+ ALL, Real world study
Brief summary
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.
Interventions
None listed
Sponsors
Novartis Pharmaceuticals
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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: * Signed informed consent form (ICF), or * ICF waiver granted by an Institutional review board/Independent Ethics Committee (IRB/IEC).
Exclusion criteria
• Age \< 18 years at the time of initiating asciminib treatment.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of Patients Achieving Hematological Complete Remission (CR) or Hematological Complete Remission With Incomplete Count Recovery (CRi) in the First 3 Months of Treatment | 3 months | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of Patients With Minimal Residual Disease (MRD) Evaluated in Peripheral Blood at Best Response | From Day 1 to 120, and at Baseline, Day 28, 60, 90, 180, 270, 360 | 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. |
| Proportion of Patients Who Showed MRD Positivity and MRD Negativity | Baseline, Day 28, 60, 90, 180, 270, 360 | 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). |
| Duration of Response (DoR) | Up to 5 years and 4 months | 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. |
| Proportion of Patients in Hematological CR or CRi Within 13 Months From Start of Treatment | 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) | 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. |
| Proportion of Patients in Complete Remission | 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 (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. |
| Proportion of Patients in Complete Remission With Incomplete Recovery | 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 (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 |
| Proportion of Patients who Proceed to Stem Cell Transplantation (SCT) | Up to 5 years and 4 months | Proportion of patients who proceed to SCT by the cutoff date. |
| Proportion of Patients who Continued Treatment With Asciminib After Last SCT | Up to 5 years and 4 months | Proportion of patients who proceed to SCT and continue treatment with asciminib by the cutoff date. |
| 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 | Up to 5 years and 4 months | 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. |
| Overall Survival (OS) | Up to 5 years and 4 months | OS was defined as the time from start of treatment with asciminib to death due to any cause. |
| Relapse-free Survival (RFS) | Up to 5 years and 4 months | 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. |
| 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 | During the first 3 months of treatment and at any time point until Day 360 ± 30 | 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. |
| Among Patients Intolerant to Prior Therapy With MRD Negativity at Baseline, Duration of MRD | Up to 5 years and 4 months | 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) |
| 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 | Up to 5 years and 4 months | Patient characteristics included age group, gender, race and ethnicity, medical history, prior treatments, relapse/remission status, and treatment phase. |
| Proportion of Patients by DoR and Patient Characteristics | Up to 5 years and 4 months | 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. |
| Proportion of Patients With Adverse Events | Up to 5 years and 4 months | — |
Countries
Australia, Canada, China, France, Israel, Italy, Netherlands, Pakistan, Poland, Spain, United Kingdom, United States
Contacts
STUDY_DIRECTORNovartis Pharmaceuticals
Novartis Pharmaceuticals
Outcome results
None listed