Increasing aclarubicin dose in low-dose cytarabine and aclarubicin in combination with granulocyte colony-stimulating factor (CAG regimen) is efficacious as salvage chemotherapy for relapsed/refractory mixed-phenotype acute leukemia

• Efficacy of HD-CAG for relapsed/refractory MPAL exceeded DOAP regimen.
• Efficacy of HD-CAG group for relapsed/refractory B + My MPAL was lower than T + My MPAL.
• Efficacy of HD-CAG for relapsed/refractory MPAL, AML, and ALL were similar.
• Side effects were similar between HD-CAG and DOAP regimen.

We treated 60 relapsed/refractory mixed-phenotype acute leukemia patients (MPAL-1) with increasing the aclarubicin dose in CAG regimen (HD-CAG, cytarabine (10 mg/m2/12 h, days 1–14), aclarubicin (5–7 mg/m2/day, days 1–14), granulocyte colony-stimulating factor (200 μg/m2/day, days 1–14). This was compared to 64 relapsed/refractory MPAL patients (MPAL-2) treated with DOAP regimen (daunorubicin, vincristine/vindesine, cytarabine and prednisone), 113 relapsed/refractory acute myeloid leukemia (AML) patients and 78 acute lymphocytic leukemia (ALL) patients treated with HD-CAG regimen. After one course, complete remission (CR) and overall response [OR, CR + partial remission (PR)] rates for MPAL-1 exceeded MPAL-2 (CR, 61.02% vs. 28.13%, P = 0.000; OR, 72.88% vs. 34.38%, P = 0.000), but these data were similar to AML and ALL (P > 0.05). In MPAL-1 group, CR and OR rates of T-lymphoid + myeloid immunophenotype were higher than B-lymphoid + myeloid immunophenotype (CR, 81.82% vs. 44.12%, P = 0.005; OR, 90.91% vs. 58.82%, P = 0.009). The overall survival at 3 years in MPAL-1, MPAL-2, AML and ALL groups were 14.2% ± 6.8%, 14.1% ± 6.4%, 17.3% ± 5.0% and 15.0% ± 5.3% (P > 0.05). Side effects were similar between HD-CAG and DOAP (P > 0.05). HD-CAG regimen is efficacious for relapsed/refractory MPAL, especially for T + My patients.