Methods
After IRB approval, a retrospective chart review identified patients
diagnosed with acute leukemia or MDS at age 30 years or younger who
received venetoclax combination therapy at UCSF Benioff Children’s
Hospitals. Eligible diagnoses included AML, MDS, and ALL. Patients who
were treated on a clinical trial were excluded from this study.
Complete remission (CR) was defined as disappearance of all clinical
and/or radiologic evidence of disease, plus absolute neutrophil count
(ANC) ≥1.0 × 103/L, platelet count ≥100 ×
103/L, and bone marrow differential with
<5% blasts by morphology or flow cytometry of bone marrow.
Partial response (PR) was defined as no peripheral blasts or peripheral
blood absolute blast count decreased by ≥ 50% from baseline, bone
marrow with 5 – 25% blasts and at least a 50% decrease in bone marrow
blast percent from baseline, and no evidence of extramedullary disease.
Progressive disease (PD) was defined as > 50% increase in
absolute peripheral or bone marrow blasts by morphology or flow
cytometry. Stable disease (SD) was defined as the conditions under which
criteria for CR, PR, or PD were not met.
Minimal residual disease (MRD) was defined as multiparameter flow
cytometry of bone marrow with less than 0.01% blasts. Venetoclax
toxicities were graded per the Common Terminology Criteria for Adverse
Events version 5.0. Overall survival (OS) defined as the time in months
from the start of venetoclax therapy to death, and progression-free
survival (PFS) was defined as the time from the start of venetoclax
administration until disease progression or relapse. Patients alive
without relapse or progression were censored at their date of last
follow-up. Kaplan–Meier curves of OS and PFS were generated.