Measurable residual disease and posttransplantation gilteritinib maintenance for patients with <i>FLT3-ITD</i>–mutated AML

M Mark J. Levis (Department of Oncology, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine) M Mehdi Hamadani (12Blood and Marrow Transplant and Cellular Therapy Program, Medical College of Wisconsin, Milwaukee, WI) B Brent R. Logan (2Division of Hematology and Oncology, Department of Medicine, Center for International Blood and Marrow Transplant Research/Medical College of Wisconsin, Milwaukee, WI) R Richard J. Jones (Department of Oncology, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine) A Anurag K. Singh (3Division of Hematologic Malignancies and Cellular Therapeutics, University of Kansas, Kansas City, KS) M Mark R. Litzow (Mayo Clinic) J John R. Wingard (5Department of Medicine, University of Florida, Gainesville, FL) E Esperanza B. Papadopoulos (6Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY) A Alexander E. Perl (Abramson Cancer Center, University of Pennsylvania, Philadelphia, Pennsylvania, United States) R Robert J. Soiffer (Dana-Farber Cancer Institute) C Celalettin Ustun (9Rush University, Chicago, United States) M Masumi Ueda Oshima (10Fred Hutchinson Cancer Center, Seattle, United States) G Geoffrey L. Uy (Division of Oncology, Washington University School of Medicine, Saint Louis, Missouri, United States) E Edmund K. Waller S Sumithira Vasu (29Department of Internal Medicine, The Ohio State University, Columbus, OH) M Melhem Solh (14Bone marrow Transplant Group of Georgia, Atlanta, United States) A Asmita Mishra (H. Lee Moffitt Cancer Center and Research Institute, TAMPA, Florida, United States) L Lori S. Muffly (Stanford University School of Medicine) H Hee-Je Kim (17Catholic Hematology Hospital, Seoul St. Mary’s Hospital, College of Medicine, The Catholic University of Korea, Seoul, Republic of Korea) M Matthias Stelljes (16Department of Medicine A, University Hospital Münster, Münster, Germany) Y Yuho Najima M Masahiro Onozawa (20Hokkaido University Hospital, Sapporo, Japan) K Kirsty Thomson (21University College Hospital, London, United Kingdom) A Arnon Nagler (3Division of Hematology and Bone Marrow Transplantation, Chaim Sheba Medical Center, Tel HaShomer, Ramat Gan, Israel) A Andrew H. Wei G Guido Marcucci C Caroline Chen (Department of Chemistry, University of California 1 , Berkeley, California 94720,) N Nahla Hasabou (22Astellas Pharma Global Development, Inc, Northbrook, United States) M Matt Rosales (Oncology Global Development, Astellas Pharma, Northbrook, IL) J Jason Hill (Dept. Bioproducts and Biosystems Engineering, University of Minnesota) S Stanley C. Gill (Astellas Pharma, Northbrook, IL) R Rishita Nuthethi (22Astellas Pharma Global Development, Inc, Northbrook, United States) D Denise King (23The Emmes Company, Rockville, United States) A Adam Mendizabal (23The Emmes Company, Rockville, United States) S Steven M. Devine (27National Marrow Donor Program, Minneapolis, MN) M Mary M. Horowitz (2Division of Hematology and Oncology, Department of Medicine, Center for International Blood and Marrow Transplant Research/Medical College of Wisconsin, Milwaukee, WI) Y Yi-Bin Chen (1Cellular Immunotherapy Program, Massachusetts General Hospital Cancer Center, Harvard Medical School, Boston, MA)

Abstract

Abstract BMT CTN (Blood and Marrow Transplant Clinical Trials Network) 1506 (“MORPHO”) was a randomized study of gilteritinib compared with placebo as maintenance therapy after hematopoietic cell transplantation (HCT) for patients with FLT3-ITD–mutated acute myeloid leukemia (AML). A key secondary end point was to determine the impact on survival of before and/or after HCT measurable residual disease (MRD), as determined using a highly sensitive assay for FLT3-ITD mutations. Generally, gilteritinib maintenance therapy was associated with improved relapse-free survival (RFS) for participants with detectable peri-HCT MRD, whereas no benefit was evident for those lacking detectable MRD. We conducted a post hoc analysis of the data and found that the level of MRD detected with this approach correlated remarkably with RFS and relapse risk, and that MRD detectable at any level negatively affected RFS. In the placebo arm, 42.2% of participants with detectable FLT3-ITD MRD relapsed compared with 13.4% of those without detectable MRD. We found that 14.8% of participants had multiple FLT3-ITD clones detected as MRD and had worse survival irrespective of treatment arm. Finally, we examined the kinetics of FLT3-ITD clonal relapse or eradication and found that participants on the placebo arm with detectable MRD relapsed rapidly after HCT, often within a few weeks. MRD-positive participants on the gilteritinib arm relapsed either with FLT3 wild-type clones (assessed by capillary electrophoresis), after cessation of gilteritinib with persistent MRD, or on progression of multiclonal disease. These data demonstrate the potential of FLT3-ITD MRD to guide therapy with gilteritinib for this subtype of AML. This trial was registered at www.clinicaltrials.gov as #NCT02997202.

Article Details

Journal Blood
Volume / Issue Vol. 145, Issue 19
Published May 08, 2025
Pages 2138-2148
ISSN 0006-4971
Publisher Elsevier BV

Journal Info

Blood

Elsevier BV

ISSN: 0006-4971 Health Sciences

Authors (37)

M

Mark J. Levis

Department of Oncology, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine

M

Mehdi Hamadani

12Blood and Marrow Transplant and Cellular Therapy Program, Medical College of Wisconsin, Milwaukee, WI

B

Brent R. Logan

2Division of Hematology and Oncology, Department of Medicine, Center for International Blood and Marrow Transplant Research/Medical College of Wisconsin, Milwaukee, WI

R

Richard J. Jones

Department of Oncology, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine

A

Anurag K. Singh

3Division of Hematologic Malignancies and Cellular Therapeutics, University of Kansas, Kansas City, KS

M

Mark R. Litzow

Mayo Clinic

J

John R. Wingard

5Department of Medicine, University of Florida, Gainesville, FL

E

Esperanza B. Papadopoulos

6Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY

A

Alexander E. Perl

Abramson Cancer Center, University of Pennsylvania, Philadelphia, Pennsylvania, United States

R

Robert J. Soiffer

Dana-Farber Cancer Institute

C

Celalettin Ustun

9Rush University, Chicago, United States

M

Masumi Ueda Oshima

10Fred Hutchinson Cancer Center, Seattle, United States

G

Geoffrey L. Uy

Division of Oncology, Washington University School of Medicine, Saint Louis, Missouri, United States

E

Edmund K. Waller

S

Sumithira Vasu

29Department of Internal Medicine, The Ohio State University, Columbus, OH

M

Melhem Solh

14Bone marrow Transplant Group of Georgia, Atlanta, United States

A

Asmita Mishra

H. Lee Moffitt Cancer Center and Research Institute, TAMPA, Florida, United States

L

Lori S. Muffly

Stanford University School of Medicine

H

Hee-Je Kim

17Catholic Hematology Hospital, Seoul St. Mary’s Hospital, College of Medicine, The Catholic University of Korea, Seoul, Republic of Korea

M

Matthias Stelljes

16Department of Medicine A, University Hospital Münster, Münster, Germany

Y

Yuho Najima

M

Masahiro Onozawa

20Hokkaido University Hospital, Sapporo, Japan

K

Kirsty Thomson

21University College Hospital, London, United Kingdom

A

Arnon Nagler

3Division of Hematology and Bone Marrow Transplantation, Chaim Sheba Medical Center, Tel HaShomer, Ramat Gan, Israel

A

Andrew H. Wei

G

Guido Marcucci

C

Caroline Chen

Department of Chemistry, University of California 1 , Berkeley, California 94720,

N

Nahla Hasabou

22Astellas Pharma Global Development, Inc, Northbrook, United States

M

Matt Rosales

Oncology Global Development, Astellas Pharma, Northbrook, IL

J

Jason Hill

Dept. Bioproducts and Biosystems Engineering, University of Minnesota

S

Stanley C. Gill

Astellas Pharma, Northbrook, IL

R

Rishita Nuthethi

22Astellas Pharma Global Development, Inc, Northbrook, United States

D

Denise King

23The Emmes Company, Rockville, United States

A

Adam Mendizabal

23The Emmes Company, Rockville, United States

S

Steven M. Devine

27National Marrow Donor Program, Minneapolis, MN

M

Mary M. Horowitz

2Division of Hematology and Oncology, Department of Medicine, Center for International Blood and Marrow Transplant Research/Medical College of Wisconsin, Milwaukee, WI

Y

Yi-Bin Chen

1Cellular Immunotherapy Program, Massachusetts General Hospital Cancer Center, Harvard Medical School, Boston, MA