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Table 1 Key studies of hypomethylating agents as maintenance therapy in AML patients after allo-HSCT

From: Maintenance therapy in acute myeloid leukemia after allogeneic hematopoietic stem cell transplantation

References Study design Patients, N Maintenance therapy regimen Relapse Survival
Jabbour [14] Retrospective, single arm 8 acute leukemia (7 AML, 1 ALL) low-dose AZA daily × 5 days every 28 days for a median of 8 cycles 3/8 (37.5%) 5 alive with CR, 2 alive with leukemia
de Lima [15] Phase I 45 high-risk AML/ MDS (37 AML, 8 MDS) low-dose AZA daily × 5 days every 30 days for a maximum of 4 cycles 24/45 (53.3%) 1-year OS: 77%; 1-year LFS: 58%
Maples [18] Retrospective, 2 arms 25 (18 AML, 7 MDS) AZA 32 mg/m2/day × 5 days every 28 days for 4–6 cycles 16% (AZA) versus 14% (Control) 1-year OS: 60% (AZA) versus 64% (Control)
Oshikawa [19] Retrospective, 2 arms 10 high-risk AML AZA (30 mg/m2/day on days 1–7) and GO (3 mg/m2 on day 8) every 4 weeks for up to 4 cycles AZA-GO: 4/10 (40.0%) AZA-GO versus Control
1-year OS: 70.0% versus 59.8%; 1-year LFS: 60.0% versus 42.8%
Pusic [16] Phase I 22 (17 AML, 5 MDS) DAC × 5 days every 6 weeks for a maximum of 8 cycles 2-year CIR: 28% 2-year OS: 56%; 2-year LFS: 48%
Ma [17] Retrospective, 2 arms 21 high-risk AML DAC 20 mg/m2/day × 5 days every 3 months for up to 4–6 cycles 3-year CIR: 5.9% (DAC) versus 45.3% (Control) 3-year OS: 92.9% (DAC) versus 51.8% (Control)
Platzbecker [20] Phase II 24 high-risk AML or MDS with MRD-positive post-transplantation preemptive therapy with AZA 75 mg/m2/day on days 1–7 of a 29-day cycle for a minimum of 6 cycles 8/24 (33.3%) 2-year OS: 62%; 2-year LFS: 54%,
de Lima [21] Phase I/II 30 (26 AML, 4 MDS) CC-486 daily × 7 days every 28 days for up to 12 cycles 1-year CIR: 21% 1-year OS in the 7-day and 14-day dosing cohorts of 86% and 81%
Gao [22] Phase II RCT 202 high-risk MRD- negative AML (G-DAC: 100; Non-G-DAC: 102) G-DAC: 5 mg/m2 of DAC on days 1–5 and 100 mg/m2 of rhG-CSF on days 0–5 every 6–8 weeks for up to 6 cycles; Non-G-DAC: no intervention 2-year CIR: 15.0% (G-DAC) versus 38.3% (Non-G-DAC) G-DAC versus Non-G-DAC
2-year OS: 85.8% versus 69.7%; 2-year LFS: 81.9% versus 60.7%
  1. Ref reference, N number, AML acute myeloid leukemia, ALL acute lymphoblastic leukemia, AZA azacitidine, CR complete remission, MDS myelodysplastic syndrome, OS overall survival, LFS leukemia-free survival, GO gemtuzumab ozogamicin, DAC decitabine, CIR cumulative incidence of relapse, MRD measurable residual disease, RCT randomized controlled trial, rhG-CSF recombinant human granulocyte colony-stimulating factor