Deep molecular profiling of synovial biopsies in the STRAP trial identifies signatures predictive of treatment response to biologic therapies in rheumatoid arthritis

M Myles J. Lewis (Centre for Experimental Medicine & Rheumatology, William Harvey Research Institute and Barts and The London School of Medicine and Dentistry, Queen Mary University of London) C Cankut Cubuk (Centre for Experimental Medicine & Rheumatology, William Harvey Research Institute and Barts and The London School of Medicine and Dentistry, Queen Mary University of London) A Anna E. A. Surace E Elisabetta Sciacca R Rachel Lau K Katriona Goldmann (Centre for Experimental Medicine & Rheumatology, William Harvey Research Institute and Barts and The London School of Medicine and Dentistry, Queen Mary University of London) G Giovanni Giorli L Liliane Fossati-Jimack A Alessandra Nerviani F Felice Rivellese C Costantino Pitzalis L Louise Warren E Edyta Jaworska M Michele Bombardieri F Frances Humby A Arthur G. Pratt A Andrew Filer N Nagui Gendi A Alberto Cauli E Ernest Choy I Iain McInnes P Patrick Durez C Christopher J. Edwards M Maya H. Buch E Elisa Gremese P Peter C. Taylor N Nora Ng J Juan D. Cañete S Sabrina Raizada N Neil D. McKay D Deepak Jadon P Pier Paolo Sainaghi R Richard Stratton (Royal Free Hospital, London) M Michael R. Ehrenstein P Pauline Ho J Joaquim P. Pereira B Bhaskar Dasgupta (Medical Technology Research Centre, Anglia Ruskin University, Chelmsford, United Kingdom) C Claire Gorman A Ahmed Zayat A Ana Rita Machado A Andrea Cuervo A Arti Mahto C Charlotte Rawlings C Chijioke Mosanya C Christopher D. Buckley C Chris Holroyd D Deborah Maskall F Francesco Carlucci G Georgina Thorburn G Gina Tan G Gloria Lliso-Ribera H Hasan Rizvi J Joanna Peel J João Eurico Fonseca J John D. Isaacs J Julio Ramírez L Laurent Meric de Bellefon M Mary Githinji M Mattia Congia N Neal Millar N Nirupam Purkayastha R Rakhi Seth R Raquel Celis R Rebecca Hands-Greenwood R Robert Landewé S Simone Perniola S Stefano Alivernini (Friedrich Alexander Universität Erlangen–Nürnberg, Erlangen, Germany) S Stefano Marcia S Stefano Marini S Stephen Kelly V Vasco Romão J James Galloway H Hector Chinoy D Désirée van der Heijde P Peter Sasieni A Anne Barton

Abstract

Abstract Approximately 40% of patients with rheumatoid arthritis do not respond to individual biologic therapies, while biomarkers predictive of treatment response are lacking. Here we analyse RNA-sequencing (RNA-Seq) of pre-treatment synovial tissue from the biopsy-based, precision-medicine STRAP trial (n = 208), to identify gene response signatures to the randomised therapies: etanercept (TNF-inhibitor), tocilizumab (interleukin-6 receptor inhibitor) and rituximab (anti-CD20 B-cell depleting antibody). Machine learning models applied to RNA-Seq predict clinical response to etanercept, tocilizumab and rituximab at the 16-week primary endpoint with area under receiver operating characteristic curve (AUC) values of 0.763, 0.748 and 0.754 respectively (n = 67-72) as determined by repeated nested cross-validation. Prediction models for tocilizumab and rituximab are validated in an independent cohort (R4RA): AUC 0.713 and 0.786 respectively (n = 65-68). Predictive signatures are converted for use with a custom synovium-specific 524-gene nCounter panel and retested on synovial biopsy RNA from STRAP patients, demonstrating accurate prediction of treatment response (AUC 0.82-0.87). The converted models are combined into a unified clinical decision algorithm that has the potential to transform future clinical practice by assisting the selection of biologic therapies.

Article Details

Volume / Issue Vol. 16, Issue 1
Published July 02, 2025
ISSN 2041-1723
Publisher Nature Portfolio

Journal Info

Nature Communications

Nature Portfolio

ISSN: 2041-1723 Open Access Life Sciences

Authors (76)

M

Myles J. Lewis

Centre for Experimental Medicine & Rheumatology, William Harvey Research Institute and Barts and The London School of Medicine and Dentistry, Queen Mary University of London

C

Cankut Cubuk

Centre for Experimental Medicine & Rheumatology, William Harvey Research Institute and Barts and The London School of Medicine and Dentistry, Queen Mary University of London

A

Anna E. A. Surace

E

Elisabetta Sciacca

R

Rachel Lau

K

Katriona Goldmann

Centre for Experimental Medicine & Rheumatology, William Harvey Research Institute and Barts and The London School of Medicine and Dentistry, Queen Mary University of London

G

Giovanni Giorli

L

Liliane Fossati-Jimack

A

Alessandra Nerviani

F

Felice Rivellese

C

Costantino Pitzalis

L

Louise Warren

E

Edyta Jaworska

M

Michele Bombardieri

F

Frances Humby

A

Arthur G. Pratt

A

Andrew Filer

N

Nagui Gendi

A

Alberto Cauli

E

Ernest Choy

I

Iain McInnes

P

Patrick Durez

C

Christopher J. Edwards

M

Maya H. Buch

E

Elisa Gremese

P

Peter C. Taylor

N

Nora Ng

J

Juan D. Cañete

S

Sabrina Raizada

N

Neil D. McKay

D

Deepak Jadon

P

Pier Paolo Sainaghi

R

Richard Stratton

Royal Free Hospital, London

M

Michael R. Ehrenstein

P

Pauline Ho

J

Joaquim P. Pereira

B

Bhaskar Dasgupta

Medical Technology Research Centre, Anglia Ruskin University, Chelmsford, United Kingdom

C

Claire Gorman

A

Ahmed Zayat

A

Ana Rita Machado

A

Andrea Cuervo

A

Arti Mahto

C

Charlotte Rawlings

C

Chijioke Mosanya

C

Christopher D. Buckley

C

Chris Holroyd

D

Deborah Maskall

F

Francesco Carlucci

G

Georgina Thorburn

G

Gina Tan

G

Gloria Lliso-Ribera

H

Hasan Rizvi

J

Joanna Peel

J

João Eurico Fonseca

J

John D. Isaacs

J

Julio Ramírez

L

Laurent Meric de Bellefon

M

Mary Githinji

M

Mattia Congia

N

Neal Millar

N

Nirupam Purkayastha

R

Rakhi Seth

R

Raquel Celis

R

Rebecca Hands-Greenwood

R

Robert Landewé

S

Simone Perniola

S

Stefano Alivernini

Friedrich Alexander Universität Erlangen–Nürnberg, Erlangen, Germany

S

Stefano Marcia

S

Stefano Marini

S

Stephen Kelly

V

Vasco Romão

J

James Galloway

H

Hector Chinoy

D

Désirée van der Heijde

P

Peter Sasieni

A

Anne Barton