Aberrant single-cell phenotype and clinical implications of genotypically defined polyclonal plasma cells in myeloma

M Matteo Claudio Da Vià (2Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Hematology Unit, Milan, Italy) F Francesca Lazzaroni (2Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy) A Antonio Matera (7University of Milan, Milano, Italy) A Alessio Marella (7University of Milan, Milano, Italy) A Akihiro Maeda (6Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milano, Italy) C Claudio de Magistris (6Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milano, Italy) L Loredana Pettine (16Hematology Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy) A Antonio Giovanni Solimando (3Guido Baccelli Unit of Internal Medicine Department of Precision and Regenerative Medicine and Ionian Area, University of Bari “Aldo Moro” Medical School, Bari, Italy) V Vanessa Desantis (4Section of Pharmacology, Department of Precision and Regenerative Medicine and Ionian Area, University of Bari Aldo Moro Medical School, Bari, Italy) G Giuseppe M. Peretti (5University Team of Regenerative and Reconstructive Orthopedics, IRCCS Istituto Ortopedico Galeazzi, Milan, Italy) L Laura Mangiavini (5University Team of Regenerative and Reconstructive Orthopedics, IRCCS Istituto Ortopedico Galeazzi, Milan, Italy) R Riccardo Giorgino (5University Team of Regenerative and Reconstructive Orthopedics, IRCCS Istituto Ortopedico Galeazzi, Milan, Italy) S Sonia Fabris (2Hematology Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy) S Stefania Pioggia (6Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milano, Italy) A Alfredo Marchetti (1Department of Oncology and Hematology-Oncology, University of Milan, Milan, Italy) M Marzia Barbieri (2Hematology Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy) S Silvia Lonati (1Department of Oncology and Hematology-Oncology, University of Milan, Milan, Italy) A Alessandra Cattaneo (3Flow Cytometry Laboratory, Clinical Pathology Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy) M Marta Tornese (3Flow Cytometry Laboratory, Clinical Pathology Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy) M Margherita Scopetti (1Department of Oncology and Hematology-Oncology, University of Milan, Milan, Italy) E Emanuele Calvi (2Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy) N Nayyer Latifinavid (2Department of Oncology and Hemato-Oncology, University of Milan, Milan, Italy) G Giancarlo Castellano (5Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), Barcelona, Spain) F Federica Torricelli (1Azienda USL-IRCCS di Reggio Emilia, Reggio Emilia, Italy) A Antonino Neri (Azienda USL-IRCCS di Reggio Emilia, Reggio Emilia, Italy) C Cathelijne Fokkema (1Department of Hematology, Erasmus MC Cancer Institute, Rotterdam, The Netherlands) T Tom Cupedo (Erasmus MC Cancer Institute) M Marta Lionetti (7University of Milan, Milano, Italy) F Francesco Passamonti (University of Milan, Milan) N Niccolò Bolli (1Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico)

Abstract

Abstract Multiple myeloma (MM) is driven by clonal plasma cell (cPC)–intrinsic factors and changes in the tumor microenvironment (TME). To investigate whether residual polyclonal PCs (pPCs) are disrupted, single-cell (sc) RNA sequencing (scRNA-seq) and sc B-cell receptor analysis were applied in a cohort of 46 samples with PC dyscrasias and 21 healthy donors (HDs). Of 234 789 PCs, 64 432 were genotypically identified as pPCs with frequencies decreasing over different disease stages, from 23.66% in monoclonal gammopathy of undetermined significance to 3.23% in MMs (P = .00012). Both cPCs and pPCs had a comparable expression of typical lineage markers (ie, CD38 and CD138), whereas others were more variable (CD27 and ITGB7). Only cPCs overexpressed oncogenes (eg, CCND1/2 and NSD2), but CCND3 was often expressed in pPCs. B-cell maturation antigen was expressed on both pPCs and cPCs, whereas GPRC5D was mostly upregulated in cPCs with implications for on-target, off-tumor activity of targeted immunotherapies. In comparison with HDs, pPCs from patients showed upregulated autophagy and disrupted interaction with TME. Importantly, interferon-related pathways were significantly enriched in pPCs from patients vs HDs (adjusted P < .05) showing an inflamed phenotype affecting genotypically normal PCs. The function of pPCs was consequently affected and correlated with immunoparesis, driven by disrupted cellular interactions with TME. Leveraging our scRNA-seq data, we derived a “healthy PC signature” that could be applied to bulk transcriptomics from the CoMMpass data set and predicted significantly better progression-free survival and overall survival (log-rank P < .05 for both). Our findings show that genotypic sc identification of pPCs in PC dyscrasias has relevant pathogenic and clinical implications.

Article Details

Journal Blood
Volume / Issue Vol. 145, Issue 26
Published June 26, 2025
Pages 3124-3138
ISSN 0006-4971
Publisher Elsevier BV

Journal Info

Blood

Elsevier BV

ISSN: 0006-4971 Health Sciences

Authors (30)

M

Matteo Claudio Da Vià

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

F

Francesca Lazzaroni

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

A

Antonio Matera

7University of Milan, Milano, Italy

A

Alessio Marella

7University of Milan, Milano, Italy

A

Akihiro Maeda

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

C

Claudio de Magistris

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

L

Loredana Pettine

16Hematology Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy

A

Antonio Giovanni Solimando

3Guido Baccelli Unit of Internal Medicine Department of Precision and Regenerative Medicine and Ionian Area, University of Bari “Aldo Moro” Medical School, Bari, Italy

V

Vanessa Desantis

4Section of Pharmacology, Department of Precision and Regenerative Medicine and Ionian Area, University of Bari Aldo Moro Medical School, Bari, Italy

G

Giuseppe M. Peretti

5University Team of Regenerative and Reconstructive Orthopedics, IRCCS Istituto Ortopedico Galeazzi, Milan, Italy

L

Laura Mangiavini

5University Team of Regenerative and Reconstructive Orthopedics, IRCCS Istituto Ortopedico Galeazzi, Milan, Italy

R

Riccardo Giorgino

5University Team of Regenerative and Reconstructive Orthopedics, IRCCS Istituto Ortopedico Galeazzi, Milan, Italy

S

Sonia Fabris

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

S

Stefania Pioggia

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

A

Alfredo Marchetti

1Department of Oncology and Hematology-Oncology, University of Milan, Milan, Italy

M

Marzia Barbieri

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

S

Silvia Lonati

1Department of Oncology and Hematology-Oncology, University of Milan, Milan, Italy

A

Alessandra Cattaneo

3Flow Cytometry Laboratory, Clinical Pathology Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy

M

Marta Tornese

3Flow Cytometry Laboratory, Clinical Pathology Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy

M

Margherita Scopetti

1Department of Oncology and Hematology-Oncology, University of Milan, Milan, Italy

E

Emanuele Calvi

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

N

Nayyer Latifinavid

2Department of Oncology and Hemato-Oncology, University of Milan, Milan, Italy

G

Giancarlo Castellano

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

F

Federica Torricelli

1Azienda USL-IRCCS di Reggio Emilia, Reggio Emilia, Italy

A

Antonino Neri

Azienda USL-IRCCS di Reggio Emilia, Reggio Emilia, Italy

C

Cathelijne Fokkema

1Department of Hematology, Erasmus MC Cancer Institute, Rotterdam, The Netherlands

T

Tom Cupedo

Erasmus MC Cancer Institute

M

Marta Lionetti

7University of Milan, Milano, Italy

F

Francesco Passamonti

University of Milan, Milan

N

Niccolò Bolli

1Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico