Skip to main content

Table 2 Key studies of FLT3 inhibitors 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 Flt3 inhibitors, maintenance duration Relapse Survival
Chen [29] Phase I 22 FLT3-ITD AML Sorafenib, 12 months 3/22 (13.6%) 1-year OS: 95%; 1-year LFS: 85%
Brunner [30] Retrospective, 2 arms 81 FLT3-ITD AML (Sorafenib: 26; Control: 55) Sorafenib, median duration of 336.5 (19–1556) days 2-year CIR: 8.2% (Sorafenib) versus 37.7% (Control) Sorafenib versus Control
2-year OS: 81% versus 62%; 2-year LFS: 82% versus 53%
Battipaglia [31] Retrospective, single arm 27 FLT3-mutated AML (25 FLT3- ITD, 2 FLT3-TKD) Sorafenib, median duration of 8.4 (0.2–46) months 3/27 (11.1%) 2-year OS: 80% ± 8%
2-year LFS: 73% ± 9%
Xuan [25] Retrospective, 2 arms 144 FLT3-ITD AML (Sorafenib: 58; Control: 86) Sorafenib, median duration of 146 (51–240) days 3-year CIR: 17.3% (Sorafenib) versus 34.2% (Control) Sorafenib versus Control
3-year OS: 81.3% versus 62.9%; 3-year LFS: 79.3% versus 52.1%
Bazarbachi [33] Retrospective, EBMT registry-based analysis 462 FLT3-mutated AML (Sorafenib: 28; Control: 434) Sorafenib, median duration of ≥ 12 months 2-year CIR of total 462 patients: 34% Matched-pair analysis 26 sorafenib patients versus 26 controls: 2-year OS: 83% versus 62%; 2-year LFS: 79% versus 54%
Xuan [35] Phase III RCT 202 FLT3-ITD AML (Sorafenib: 100; Control: 102) Sorafenib was administered at 30–60-day post-transplantation and continued until day 180 Sorafenib versus Control
1-year CIR: 7.0% versus 24.5%; 2-year CIR: 11.9% versus 31.6%
Sorafenib versus Control
2-year OS: 82.1% versus 68.0%; 2-year LFS: 78.9% versus 56.6%
Burchert [36] Phase II RCT (SORMAIN, terminated early due to slow accrual) 83 FLT3-ITD AML (Sorafenib: 43; Placebo: 40) Sorafenib, 24 months Sorafenib (8/43, 18.6%) versus Placebo (17/40, 42.5%) Sorafenib versus Placebo
2-year OS: 90.5% versus 66.2%; 2-year LFS: 85.0% versus53.3%
Sandmaier [40] Phase I 13 AML Quizartinib (AC220), a maximum of 24 months 1/13 (7.7%) NA
Maziarz [42] Phase II RCT (the preliminary result) 60 FLT3-ITD AML (Midostaurin + SOC: 30; SOC: 30) Midostaurin, 12 months 18-month estimated relapse rate: 11% (Midostaurin + SOC) versus 24% (SOC) 18-month LFS: 89% (Midostaurin + SOC) versus 76% (SOC)
Levis [43] Phase III RCT (Ongoing, NCT02997202) 346 FLT3-ITD AML (target number, Gilteritinib: 173; Placebo: 173) Gilteritinib, 2 years NA NA
  1. Ref reference, N number, FLT3-ITD FLT3 internal tandem duplication, OS overall survival, LFS leukemia-free survival, CIR cumulative incidence of relapse, FLT3-TKD FLT3 tyrosine kinase domain, EBMT European Society for Blood and Marrow Transplantation, RCT randomized controlled trial, NA not available, SOC standard of care