A plasma-based DNA test for quantification of disease burden in acute myeloid leukemia patients undergoing bone marrow transplantation

Y Yuxuan Wang J Jiajun Xie (Department of Oncology, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine) S Sergiu Pasca (Division of Hematologic Malignancies, Johns Hopkins University School of Medicine) M Maria Popoli (Department of Oncology, the Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine) J Janine Ptak (Department of Oncology, the Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine) L Lisa Dobbyn (Department of Oncology, the Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine) N Natalie Silliman (Department of Oncology, the Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine) S Suman Paul (Department of Oncology, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine) R Richard J. Jones (Department of Oncology, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine) M Mark J. Levis (Department of Oncology, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine) S Samuel D. Curtis (Department of Pharmacology and Molecular Sciences, Johns Hopkins University School of Medicine) C Christopher Douville (Department of Oncology, the Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine) C Cynthia Shams (Department of Oncology, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine) M Matthew Z. Guo (Department of Oncology, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine) S Shirley Mo (Division of Hematologic Malignancies, Johns Hopkins University School of Medicine) C Christopher D. Gocke (Department of Oncology, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine) S Sami N. Malek (Department of Internal Medicine, Division of Hematology and Oncology, University of Michigan) C Catherine M. Bollard (Department of Oncology, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine) C Chetan Bettegowda K Kenneth W. Kinzler (Department of Oncology, the Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine) B Bert Vogelstein (Department of Oncology, the Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine) N Nickolas Papadopoulos (Department of Oncology, the Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine) L Lukasz P. Gondek (Department of Oncology, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine)

Abstract

Allogeneic hematopoietic cell transplantation is the only curative option for many patients with acute myeloid leukemia (AML). In the current study, we designed and implemented a personalized assay, called v96, incorporating up to 96 mutations in 30 AML patients undergoing transplantation. The assay was performed on DNA derived from cells isolated from the bone marrow as well as in cell-free plasma. All 30 (100%) of patients harbored molecular evidence of residual leukemia during remission that was detectable by the v96 assay, while only 6 (20%) had evidence of disease as assessed by conventional clinical assays. Furthermore, cell-free DNA from plasma proved to be more sensitive than DNA from cells of the bone marrow for identifying residual leukemia. The median number of mutants was 352-fold higher in plasma taken prior to transplantation for patients who relapsed compared to those who did not relapse. At 2 mo posttransplantation, 27 of the 30 patients still harbored detectable leukemia as assessed by the v96 assay. Twenty-two of these patients had a subsequent decrease in leukemic burden assessed by the v96 assay. In the majority of them (20 of 22 patients), the decrease occurred only after immunosuppression was discontinued, supporting a graft-versus-leukemia effect. These results document the feasibility of using a relatively large panel of carefully chosen mutations and a highly specific assay as noninvasive markers of therapeutic response in AML patients, minimizing the need for multiple bone marrow biopsies.

Article Details

Volume / Issue Vol. 123, Issue 16
Published April 21, 2026
ISSN 0027-8424
Publisher National Academy of Sciences

Authors (23)

Y

Yuxuan Wang

J

Jiajun Xie

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

S

Sergiu Pasca

Division of Hematologic Malignancies, Johns Hopkins University School of Medicine

M

Maria Popoli

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

J

Janine Ptak

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

L

Lisa Dobbyn

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

N

Natalie Silliman

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

S

Suman Paul

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

R

Richard J. Jones

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

M

Mark J. Levis

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

S

Samuel D. Curtis

Department of Pharmacology and Molecular Sciences, Johns Hopkins University School of Medicine

C

Christopher Douville

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

C

Cynthia Shams

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

M

Matthew Z. Guo

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

S

Shirley Mo

Division of Hematologic Malignancies, Johns Hopkins University School of Medicine

C

Christopher D. Gocke

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

S

Sami N. Malek

Department of Internal Medicine, Division of Hematology and Oncology, University of Michigan

C

Catherine M. Bollard

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

C

Chetan Bettegowda

K

Kenneth W. Kinzler

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

B

Bert Vogelstein

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

N

Nickolas Papadopoulos

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

L

Lukasz P. Gondek

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