Genomic landscape of IgM-MGUS and patients with stable or progressive asymptomatic Waldenström macroglobulinemia

T Tina Bagratuni (1Department of Clinical Therapeutics, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece) O Ourania Theologi (1Department of Clinical Therapeutics, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece) C Christos Vlachos (1Department of Clinical Therapeutics, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece) I Ioannis Kollias (1Department of Clinical Therapeutics, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece) K Kylee Maclachlan (2Myeloma Service, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY) F Foteini Aktypi (1Department of Clinical Therapeutics, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece) N Nefeli Mavrianou-Koutsoukou (1Department of Clinical Therapeutics, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece) C Christine Liacos (1Department of Clinical Therapeutics, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece) K Konstantina Taouxi (1Department of Clinical Therapeutics, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece) A Alexandra Papadimou (1Department of Clinical Therapeutics, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece) K Katerina Chrisostomidou (1Department of Clinical Therapeutics, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece) M Maria Sakkou (1Department of Clinical Therapeutics, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece) I Irene Solia (12Department of Clinical Therapeutics, National and Kapodistrian University of Athens, Athens, Greece) F Foteini Theodorakakou (Department of Clinical Therapeutics, National and Kapodistrian University of Athens, School of Medicine, Athens, Greece) G Gianmarco Favrin (University of Pavia, Pavia, Italy) M Maria Gavriatopoulou (National and Kapodistrian University of Athens) E Evangelos Terpos M Marzia Varettoni (4Division of Hematology, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico, Policlinico San Matteo, Pavia, Italy) Z Zachary R. Hunter S Steven Treon (5Bing Center for Waldenström’s Macroglobulinemia, Department of Medical Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA) F Francesco Maura (Memorial Sloan Kettering Cancer Center, New York) M Meletios A. Dimopoulos E Efstathios Kastritis

Abstract

Abstract Immunoglobulin M (IgM) monoclonal gammopathy of undetermined significance (IgM-MGUS) and asymptomatic Waldenström macroglobulinemia (WM; aWM) are precursor conditions of symptomatic WM with an annual 1.5% to 12% risk of progression. Although clinical prognostic models exist for risk stratification, it remains challenging to distinguish asymptomatic patients who will eventually progress from those who will not. Hence, the characterization of genomic features that shape disease progressors could potentially improve risk stratification. We performed whole-exome sequencing on 229 samples from 139 patients, including 9 patients with sequential samples. We observed an increasing mutation burden through the stages of disease evolution. Genes such as CD79B, ARID1A, and CREBBP were more often mutated in the aWM progressed (aWMpr) compared with the stable aWM (aWMst) group, whereas MYD88L265 variant allele frequency was significantly higher in patients with aWMpr than those with aWMst. In addition, patients with IgM-MGUS with MYD88WT genotype showed a distinct genomic profile compared with the MYD88MUT patients. Furthermore, the presence of more aneuploidies showed a significant association with a higher risk of progression to the symptomatic disease. Overall, our study shows that genomic profiling of patients’ tumor at the time of aWM diagnosis might represent an improved strategy for identifying patients at high risk to progression who could benefit from earlier intervention.

Article Details

Journal Blood
Volume / Issue Vol. 146, Issue 25
Published December 18, 2025
Pages 3086-3097
ISSN 0006-4971
Publisher Elsevier BV

Journal Info

Blood

Elsevier BV

ISSN: 0006-4971 Health Sciences

Authors (23)

T

Tina Bagratuni

1Department of Clinical Therapeutics, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece

O

Ourania Theologi

1Department of Clinical Therapeutics, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece

C

Christos Vlachos

1Department of Clinical Therapeutics, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece

I

Ioannis Kollias

1Department of Clinical Therapeutics, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece

K

Kylee Maclachlan

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

F

Foteini Aktypi

1Department of Clinical Therapeutics, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece

N

Nefeli Mavrianou-Koutsoukou

1Department of Clinical Therapeutics, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece

C

Christine Liacos

1Department of Clinical Therapeutics, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece

K

Konstantina Taouxi

1Department of Clinical Therapeutics, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece

A

Alexandra Papadimou

1Department of Clinical Therapeutics, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece

K

Katerina Chrisostomidou

1Department of Clinical Therapeutics, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece

M

Maria Sakkou

1Department of Clinical Therapeutics, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece

I

Irene Solia

12Department of Clinical Therapeutics, National and Kapodistrian University of Athens, Athens, Greece

F

Foteini Theodorakakou

Department of Clinical Therapeutics, National and Kapodistrian University of Athens, School of Medicine, Athens, Greece

G

Gianmarco Favrin

University of Pavia, Pavia, Italy

M

Maria Gavriatopoulou

National and Kapodistrian University of Athens

E

Evangelos Terpos

M

Marzia Varettoni

4Division of Hematology, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico, Policlinico San Matteo, Pavia, Italy

Z

Zachary R. Hunter

S

Steven Treon

5Bing Center for Waldenström’s Macroglobulinemia, Department of Medical Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA

F

Francesco Maura

Memorial Sloan Kettering Cancer Center, New York

M

Meletios A. Dimopoulos

E

Efstathios Kastritis