A Trial of the FMS-like Tyrosine Kinase 3 (FLT3) Inhibitor Gilteritinib Administered as Maintenance Therapy Following Allogeneic Transplant for Patients With FLT3/Internal Tandem Duplication (ITD) Acute Myeloid Leukemia (AML)

Summary

The purpose of this study was to compare relapse-free survival between participants with FLT3/ITD AML in first morphologic complete remission (CR1) who underwent hematopoietic stem cell transplant (HCT) and were randomized to receive gilteritinib or placebo beginning after the time of engraftment for a two year period.

Timeline

Start

2017-08-16

Primary completion

2023-01-07

Completion

2023-05-09

Outcome

Missed primary endpoint

paper Although RFS was higher in the gilteritinib arm, the difference was not statistically significant (hazard ratio [HR], 0.679 [95% CI, 0.459 to 1.005]; two-sided P = .0518). PMID 38471061 ↗

Publications

• Levis MJ, Hamadani M, Logan BR, Jones RJ, Singh AK, Litzow MR, Wingard JR, Papadopoulos EB, Perl AE, Soiffer RJ, Ustun C, Oshima MU, Uy GL, Waller EK, Vasu S, Solh M, Mishra A, Muffly LS, Kim HJ, Stelljes M, Najima Y, Onozawa M, Thomson K, Chen C, Hasabou N, Rosales M, Hill JE, Gill SC, Nuthethi R, King D, Mendizabal A, Devine SM, Horowitz MM, Chen YB. Impact of transplant conditioning, NPM1 mutations, and measurable residual disease in FLT3-ITD acute myeloid leukemia. Blood Adv. 2025 Oct 28;9(20):5123-5133. doi: 10.1182/bloodadvances.2025016306.
• Levis MJ, Hamadani M, Logan BR, Jones RJ, Singh AK, Litzow MR, Wingard JR, Papadopoulos EB, Perl AE, Soiffer RJ, Ustun C, Oshima MU, Uy GL, Waller EK, Vasu S, Solh M, Mishra A, Muffly LS, Kim HJ, Stelljes M, Najima Y, Onozawa M, Thomson K, Nagler A, Wei AH, Marcucci G, Chen C, Hasabou N, Rosales M, Hill J, Gill SC, Nuthethi R, King D, Mendizabal A, Devine SM, Horowitz MM, Chen YB. Measurable residual disease and posttransplantation gilteritinib maintenance for patients with FLT3-ITD-mutated AML. Blood. 2025 May 8;145(19):2138-2148. doi: 10.1182/blood.2024025154.
• Hamilton BK, Pandya BJ, Ivanescu C, Elsouda D, Hamadani M, Chen YB, Levis MJ, Ueda Oshima M, Litzow MR, Soiffer RJ, Ustun C, Perl AE, Singh AK, Geller N, Hasabou N, Rosales M, Cella D, Corredoira L, Pestana C, Horowitz MM, Logan B. Health-related quality of life with gilteritinib vs placebo posttransplant for FLT3-ITD+ acute myeloid leukemia. Blood Adv. 2024 Oct 8;8(19):5091-5099. doi: 10.1182/bloodadvances.2024013746.
• Pandya BJ, Burns LJ, Wang T, Xie B, Touya M, Spalding J, Block A, Kuperman G, Young C. Clinical Outcomes and Treatment Patterns in Adults With FLT3-ITDmut+ Acute Myeloid Leukemia Undergoing Allogeneic Hemopoietic Cell Transplantation in the United States and Canada. Transplant Cell Ther. 2024 Jul;30(7):683.e1-683.e13. doi: 10.1016/j.jtct.2024.04.016. Epub 2024 Apr 23.
• Levis MJ, Hamadani M, Logan B, Jones RJ, Singh AK, Litzow M, Wingard JR, Papadopoulos EB, Perl AE, Soiffer RJ, Ustun C, Ueda Oshima M, Uy GL, Waller EK, Vasu S, Solh M, Mishra A, Muffly L, Kim HJ, Mikesch JH, Najima Y, Onozawa M, Thomson K, Nagler A, Wei AH, Marcucci G, Geller NL, Hasabou N, Delgado D, Rosales M, Hill J, Gill SC, Nuthethi R, King D, Wittsack H, Mendizabal A, Devine SM, Horowitz MM, Chen YB; BMT-CTN 1506/MORPHO Study Investigators. Gilteritinib as Post-Transplant Maintenance for AML With Internal Tandem Duplication Mutation of FLT3. J Clin Oncol. 2024 May 20;42(15):1766-1775. doi: 10.1200/JCO.23.02474. Epub 2024 Mar 12.

Drugs

Indications