Genomics Define Malignant Transformation in Myeloma Precursor Conditions

F Francesco Maura (Memorial Sloan Kettering Cancer Center, New York) P P. Leif Bergsagel (Mayo Clinic Arizona, Scottsdale) B Bachisio Ziccheddu (Memorial Sloan Kettering Cancer Center, United States) S Shaji Kumar K Kylee Maclachlan (2Myeloma Service, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY) A Andriy Derkach J Juan-Jose Garces (Memorial Sloan Kettering Cancer Center) R Ross Firestone (1Memorial Sloan Kettering Cancer Center, Myeloma Service, Department of Medicine, New York, United States) E Esteban Braggio (2Department of Medicine, Mayo Clinic, Phoenix, AZ) Y Yan Asmann M Michael Durante (1Myeloma Division, Sylvester Comprehensive Cancer Center, University of Miami Health System, Miami, FL) B Benjamin T. Diamond (Myeloma Institute, Sylvester Comprehensive Cancer Center, University of Miami, Miami, FL) M Marios Papadimitriou (1Myeloma Institute, Sylvester Comprehensive Cancer Center, University of Miami, Miami, United States) M Malin Hultcrantz (1Memorial Sloan Kettering Cancer Center, Myeloma Service, Department of Medicine, New York, United States) A Alessio Marella (7University of Milan, Milano, Italy) G Giancarlo Castellano (5Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), Barcelona, Spain) A Akihiro Maeda (6Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milano, Italy) M Marta Lionetti (7University of Milan, Milano, Italy) A Antonio Matera (7University of Milan, Milano, Italy) S Stefania Pioggia (6Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milano, Italy) M Matteo Claudio Da Vià (2Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Hematology Unit, Milan, Italy) C Claudio de Magistris (6Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milano, Italy) D Daniel Leongamornlert D Danny DeAvila (H. Lee Moffitt Cancer Center and Research Institute, Tampa, FL) P Praneeth Reddy Sudalagunta (1H. Lee Moffitt Cancer Center & Research Institute, Tampa, United States) R Rafael Renatino Canevarolo (1H. Lee Moffitt Cancer Center & Research Institute, Tampa, United States) E Erin M. Siegel P Phaedra Agius J Jamie Teer (9Moffitt Cancer Center, Tampa, United States) A Andrew McPherson Y Yusuke Yamashita (National Museum of Japanese History) A Ariosto S. Silva P Patrick Blaney (1Multiple Myeloma Research Program, Perlmutter Cancer Center, NYU Langone Health, New York, NY) R Rachid Baz (1Department of Malignant Hematology, H. Lee Moffitt Cancer Center and Research Institute, Tampa, FL) K Krina K. Patel (The University of Texas, MD Anderson Cancer Center, Houston, Texas, United States) P Peter Campbell G Gareth Morgan R Rafael Fonseca (IDOMED Vista Carioca, RIO DE JANEIRO, Brazil) O Ola Landgren R Robert Z. Orlowski (The University of Texas MD Anderson Cancer Center, Houston, Texas, United States) K Kenneth H. Shain N Niccolò Bolli (1Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico) S Saad Usmani (8Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY) S S. Vincent Rajkumar

Abstract

Multiple myeloma (MM) is consistently preceded by monoclonal gammopathy of undetermined significance (MGUS) and smoldering multiple myeloma (SMM). While these precursor conditions are asymptomatic, they are not entirely benign and carry a lifelong risk of progression to MM. Unlike other cancers defined by pathology, malignant transformation from MGUS or SMM to MM has so far relied on demonstration of clinical end-organ damage as morphology and cytogenetics cannot reliably distinguish them. In this study, using genomic data from 374 patients with MGUS or SMM (277 training, 97 validation), to our knowledge, we demonstrate for the first time the ability to identify malignant transformation in MGUS and SMM. We introduce the concept of genomic MM and genomic MGUS to differentiate the subsets of MGUS and SMM that are biologically malignant with genomic features indistinguishable from MM from the subset that is premalignant and unlikely to progress to malignancy. Importantly, we find that most SMM has biological features of malignant transformation indistinguishable from MM. As expected, this subset that we consider having genomic MM is associated with a high risk of progression to MM although some patients remained progression-free beyond 5 years. Conversely, 60% of MGUS and 10% of SMM have no evidence of malignant transformation (genomic MGUS), with no progression during follow-up. Integration of genomic features with the 2/20/20 International Myeloma Working Group model significantly improved the prediction of progression among genomic MM. These findings support the use of genomic criteria to refine the classification and the risk stratification in myeloma precursor conditions.

Article Details

Volume / Issue Vol. 44, Issue 3
Published January 20, 2026
Pages 188-199
ISSN 0732-183X
Publisher Lippincott Williams & Wilkins

Journal Info

Journal of Clinical Oncology

Lippincott Williams & Wilkins

ISSN: 0732-183X Health Sciences

Authors (44)

F

Francesco Maura

Memorial Sloan Kettering Cancer Center, New York

P

P. Leif Bergsagel

Mayo Clinic Arizona, Scottsdale

B

Bachisio Ziccheddu

Memorial Sloan Kettering Cancer Center, United States

S

Shaji Kumar

K

Kylee Maclachlan

2Myeloma Service, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY

A

Andriy Derkach

J

Juan-Jose Garces

Memorial Sloan Kettering Cancer Center

R

Ross Firestone

1Memorial Sloan Kettering Cancer Center, Myeloma Service, Department of Medicine, New York, United States

E

Esteban Braggio

2Department of Medicine, Mayo Clinic, Phoenix, AZ

Y

Yan Asmann

M

Michael Durante

1Myeloma Division, Sylvester Comprehensive Cancer Center, University of Miami Health System, Miami, FL

B

Benjamin T. Diamond

Myeloma Institute, Sylvester Comprehensive Cancer Center, University of Miami, Miami, FL

M

Marios Papadimitriou

1Myeloma Institute, Sylvester Comprehensive Cancer Center, University of Miami, Miami, United States

M

Malin Hultcrantz

1Memorial Sloan Kettering Cancer Center, Myeloma Service, Department of Medicine, New York, United States

A

Alessio Marella

7University of Milan, Milano, Italy

G

Giancarlo Castellano

5Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), Barcelona, Spain

A

Akihiro Maeda

6Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milano, Italy

M

Marta Lionetti

7University of Milan, Milano, Italy

A

Antonio Matera

7University of Milan, Milano, Italy

S

Stefania Pioggia

6Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milano, Italy

M

Matteo Claudio Da Vià

2Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Hematology Unit, Milan, Italy

C

Claudio de Magistris

6Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milano, Italy

D

Daniel Leongamornlert

D

Danny DeAvila

H. Lee Moffitt Cancer Center and Research Institute, Tampa, FL

P

Praneeth Reddy Sudalagunta

1H. Lee Moffitt Cancer Center & Research Institute, Tampa, United States

R

Rafael Renatino Canevarolo

1H. Lee Moffitt Cancer Center & Research Institute, Tampa, United States

E

Erin M. Siegel

P

Phaedra Agius

J

Jamie Teer

9Moffitt Cancer Center, Tampa, United States

A

Andrew McPherson

Y

Yusuke Yamashita

National Museum of Japanese History

A

Ariosto S. Silva

P

Patrick Blaney

1Multiple Myeloma Research Program, Perlmutter Cancer Center, NYU Langone Health, New York, NY

R

Rachid Baz

1Department of Malignant Hematology, H. Lee Moffitt Cancer Center and Research Institute, Tampa, FL

K

Krina K. Patel

The University of Texas, MD Anderson Cancer Center, Houston, Texas, United States

P

Peter Campbell

G

Gareth Morgan

R

Rafael Fonseca

IDOMED Vista Carioca, RIO DE JANEIRO, Brazil

O

Ola Landgren

R

Robert Z. Orlowski

The University of Texas MD Anderson Cancer Center, Houston, Texas, United States

K

Kenneth H. Shain

N

Niccolò Bolli

1Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico

S

Saad Usmani

8Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY

S

S. Vincent Rajkumar