2.1 Patients
Clinical data of pediatric AML patients enrolled in Children’s Oncology
Group (COG) trials AAML0531 and AAML03P1 were extracted from the TARGET
dataset
(https://portal.gdc.cancer.gov).
Cases diagnosed with acute promyelocytic leukemia, significant data
missing, and age ≥21 years were excluded. A total of 713 cases were
included in the present study after data filtering. The treatment
protocols of the three clinical trials have been reported
previously17,18. AAML0531 and AAML03P1 shared similar
conventional chemotherapy dosages and durations. However, in the
AAML03P1 trial, patients routinely underwent GO therapy during two
cycles of chemotherapy, whereas in the AAML0531 trial, patients were
subject to random allocation, determining their eligibility for the GO
regimen. Lumbar puncture was carried out as a component of the
diagnostic assessment. CNS disease was defined as the presence with any
leukemic blasts in cerebrospinal fluid (CSF) without blood
contamination, clinical manifestations of CNS leukemia, or radiographic
evidence of intradural myeloid sarcoma. Myeloid sarcoma was confirmed by
pathology and/or imaging.
In the AAML0531 and AAML03P1 trials, CNS prophylaxis involved
administering intrathecal (IT) cytarabine on the first day of induction
I/II and intensification I/II or biweekly, with a maximum of six doses.
Patients with CNS involvement received IT cytarabine biweekly until the
CSF was transparent and two additional IT cytarabine treatments.
Patients were excluded from the trials when CNS leukemia still persisted
after receiving six doses of IT cytarabine.