Trastuzumab Duocarmazine in Pretreated Human Epidermal Growth Factor Receptor 2–Positive Advanced or Metastatic Breast Cancer: An Open-Label, Randomized, Phase III Trial (TULIP)

C Cristina Saura (Vall d’Hebron University Hospital, Barcelona) E Evelyn van den Tweel (Byondis B.V., Nijmegen, the Netherlands) M Mayke Oesterholt (Byondis B.V., Nijmegen, the Netherlands) N Norbert Koper (Byondis B.V., Nijmegen, the Netherlands) J Joline Si Jing Lim (National University Hospital, Singapore, Singapore) N Nathalie Quenel-Tueux (Department of Medical Oncology, Institut Bergonié, Comprehensive Cancer Centre, Bordeaux, France) T Tira J. Tan (Division of Medical Oncology, National Cancer Centre, Singapore, Singapore) S Santiago Escrivá-de-Romaní (Vall d’Hebron Institut of Oncology, Hospital Vall d’Hebron, Barcelona) E Evelien Kuip E Elisabeth G.E. de Vries W Willemien Menke-van der Houven van Oordt P Philippe Aftimos (Institut Jules Bordet, Hôpital Universitaire de Bruxelles, Brussels) K Konstantinos Papadimitriou H Hannelore Denys (Ghent University Hospital, Department of Medical Oncology, Ghent, Belgium) K Kevin Punie (Medical Oncology, Oncology Center Antwerp, Ziekenhuis aan de Stroom, Antwerp, Belgium) G Guy Jerusalem M Marleen Borms F Francois Duhoux N Nicholas Turner I Iain Macpherson A Anne Armstrong (Medical Oncology, The Christie NHS Foundation Trust and the University of Manchester, Manchester, United Kingdom) N Nicola Levitt C Carlo Palmieri A Annabel Borley T Timothy Crook (Cromwell, London, United Kingdom) E Estela Vega Alonso S Serafin Morales S Santiago Escriva de Romani Muñoz A Agostina Stradella (Department of Medical Oncology, Institut Catala d'Oncologia – IDIBELL (ICO L'Hospitalet), Barcelona, Spain) Y Yolanda Jerez Gilarranz (Hospital General Universitario Gregorio Marañón, Instituto de Investigación Sanitaria Gregorio Marañón, CIBERONC. GEICAM Spanish Breast Cancer Group, Madrid, Spain) A Antonio Antón (Hospital Universitario Miguel Servet, Zaragoza, Spain) J Jose Juan Ponce B Barbara Adamo (Institute of Cancer and Blood Diseases, Hospital Clinic of Barcelona / Translational Genomics and Targeted Therapies in Solid Tumors group, August Pi i Sunyer Biomedical Research Institute IDIBAPS, Barcelona, Spain) J Javier Cortes Castan M Maria Martinez (3Universidad de los Andes, Facultad de Medicina, Bogotá, Colombia) T Thierry Petit E Emilie Kaczmarek S Sylvain Ladoire (Centre Georges Francois Leclerc, Dijon, France) N Nathalie Quenel Tueux L Luis Teixeira J Jean Christophe Thery (Université de Rouen Normandie, Inserm U1245, FHU G4 Genomics, CHU Rouen, Département de Génétique, Rouen, France) S Sophie Abadie-Lacourtoisie A Alain Lortholary (Groupe d'Investigateurs National des Etudes des Cancers Ovariens et du sein (GINECO) and Hôpital Privé du Confluent, Nantes, France) E Elisabeth Luporsi H Hubert Orfeuvre N Nathalie Bonnin G Giampaolo Bianchini (IRCCS Ospedale San Raffaele, Milan) F Federico Piacentini F Francesco Cognetti P Paolo Marchetti (IDI-IRCCS, Roma, Italy) E Evaristo Maiello M Marina Cazzaniga (Phase 1 Research Unit, Fondazione IRCCS San Gerardo, Monza, Italy) V Valentina Guarneri (Veneto Institute of Oncology, Istituto di Ricovero e Cura a Carattere Scientifico, Padua, Italy) M Marco Colleoni L Laura Doni R Roberto Bordonaro C Claudio Zamagni (IRCCS Azienda Ospedaliero Universitaria di Bologna, Bologna, Italy) G Giulia Bianchi (Medical Oncology Department, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy) L Laura Biganzoli M Michael Thirlwell X Xinni Song (The Ottawa Hospital Cancer Centre, Ottawa, ON, Canada) A Anil Joy S Sara Taylor T Teresa Helsten M Madhu Chaudhry H Haythem Ali S Shaker Dakhil (11Cancer Center of Kansas, Wichita, United States) R Rex Mowat A Adam Brufsky (Hillman Cancer Center, Magee-Womens Hospital, University of Pittsburgh Medical Center, Pittsburgh) M Melody Cobleigh M Manuel Modiano M Michelina Cairo M Michael Meshad M Michael A. Danso (Department of Medical Oncology, Brock Cancer Center, Virginia Oncology Associates, Norfolk) J Jay Andersen A Allyson Harroff R Rami Owera A Anne Favret (Virginia Cancer Specialists, PC, Fairfax, VA) J Joyce O'Shaughnessy (Baylor University Medical Center, Texas Oncology, Sarah Cannon Research Institute, Dallas, TX) T Timothy Pluard P Paula Rosenblatt (University of Maryland Greenebaum Comprehensive Cancer Center, Baltimore, MD) S Sharad Jain J Jeanette Dupont Jensen N Nina Jeppesen K Kim Wedervang P Per Edlund H Henrik Lindman R Renske Altena (Department of Oncology-Pathology, Karolinska Institutet and Karolinska Comprehensive Cancer Center, Karolinska University Hospital, Stockholm, Sweden) L Leif Klint T Tira Tan Jing Ying J Joline Lim Si Jing

Abstract

PURPOSE Human epidermal growth factor receptor 2 (HER2)–targeted therapy is standard of care for HER2-positive (HER2+) breast cancer, but most patients develop progressive disease with persistent HER2 expression. No definitive treatment guidance currently exists beyond second line. Trastuzumab duocarmazine (T-Duo) is a third-generation, HER2-targeted antibody-drug conjugate that demonstrated efficacy and acceptable safety in phase I studies of heavily pretreated patients with HER2+/HER2-low breast cancer. METHODS In this open-label, randomized, phase III trial, T-Duo was compared with physician's choice (PC) in patients with unresectable locally advanced/metastatic HER2+ breast cancer with progression during/after ≥2 HER2-targeted therapies or after trastuzumab emtansine (T-DM1). The primary endpoint was progression-free survival (PFS) by blinded independent central review. RESULTS In total, 437 patients were randomly assigned 2:1 to T-Duo (n = 291) or PC (n = 146). The median age was 56.0 years (range, 24-86); most patients (93.6%) had metastatic disease. The median time from diagnosis of metastatic disease to trial entry was 3.5 years; the median number of prior HER2-targeted therapies in metastatic setting was three. The median PFS was 7.0 months (95% CI, 5.4 to 7.2) with T-Duo versus 4.9 months (95% CI, 4.0 to 5.5; hazard ratio [HR], 0.64 [95% CI, 0.49 to 0.84]; P = .002) with PC. PFS benefit was maintained across most predefined subgroups. The median overall survival (first analysis) was 20.4 (T-Duo) versus 16.3 months (PC; HR, 0.83 [95% CI, 0.62 to 1.09]; P = .153). Objective response rate was 27.8% (T-Duo) versus 29.5% (PC); other efficacy end points—clinical benefit rate, duration of response, and reduction in target lesion measurement—tended to favor T-Duo. Grade ≥3 treatment-emergent adverse events occurred in 52.8% (T-Duo) versus 48.2% (PC). CONCLUSION Treatment with T-Duo was manageable, but tolerability was affected by prevalent ocular toxicity, leading to a higher discontinuation rate in the T-Duo arm. T-Duo significantly reduced the risk of progression in patients with advanced HER2+ breast cancer who have progressed during/after ≥2 HER2-targeted therapies or after T-DM1.

Article Details

Volume / Issue Vol. 43, Issue 5
Published February 10, 2025
Pages 513-523
ISSN 0732-183X
Publisher Lippincott Williams & Wilkins

Journal Info

Journal of Clinical Oncology

Lippincott Williams & Wilkins

ISSN: 0732-183X Health Sciences

Authors (91)

C

Cristina Saura

Vall d’Hebron University Hospital, Barcelona

E

Evelyn van den Tweel

Byondis B.V., Nijmegen, the Netherlands

M

Mayke Oesterholt

Byondis B.V., Nijmegen, the Netherlands

N

Norbert Koper

Byondis B.V., Nijmegen, the Netherlands

J

Joline Si Jing Lim

National University Hospital, Singapore, Singapore

N

Nathalie Quenel-Tueux

Department of Medical Oncology, Institut Bergonié, Comprehensive Cancer Centre, Bordeaux, France

T

Tira J. Tan

Division of Medical Oncology, National Cancer Centre, Singapore, Singapore

S

Santiago Escrivá-de-Romaní

Vall d’Hebron Institut of Oncology, Hospital Vall d’Hebron, Barcelona

E

Evelien Kuip

E

Elisabeth G.E. de Vries

W

Willemien Menke-van der Houven van Oordt

P

Philippe Aftimos

Institut Jules Bordet, Hôpital Universitaire de Bruxelles, Brussels

K

Konstantinos Papadimitriou

H

Hannelore Denys

Ghent University Hospital, Department of Medical Oncology, Ghent, Belgium

K

Kevin Punie

Medical Oncology, Oncology Center Antwerp, Ziekenhuis aan de Stroom, Antwerp, Belgium

G

Guy Jerusalem

M

Marleen Borms

F

Francois Duhoux

N

Nicholas Turner

I

Iain Macpherson

A

Anne Armstrong

Medical Oncology, The Christie NHS Foundation Trust and the University of Manchester, Manchester, United Kingdom

N

Nicola Levitt

C

Carlo Palmieri

A

Annabel Borley

T

Timothy Crook

Cromwell, London, United Kingdom

E

Estela Vega Alonso

S

Serafin Morales

S

Santiago Escriva de Romani Muñoz

A

Agostina Stradella

Department of Medical Oncology, Institut Catala d'Oncologia – IDIBELL (ICO L'Hospitalet), Barcelona, Spain

Y

Yolanda Jerez Gilarranz

Hospital General Universitario Gregorio Marañón, Instituto de Investigación Sanitaria Gregorio Marañón, CIBERONC. GEICAM Spanish Breast Cancer Group, Madrid, Spain

A

Antonio Antón

Hospital Universitario Miguel Servet, Zaragoza, Spain

J

Jose Juan Ponce

B

Barbara Adamo

Institute of Cancer and Blood Diseases, Hospital Clinic of Barcelona / Translational Genomics and Targeted Therapies in Solid Tumors group, August Pi i Sunyer Biomedical Research Institute IDIBAPS, Barcelona, Spain

J

Javier Cortes Castan

M

Maria Martinez

3Universidad de los Andes, Facultad de Medicina, Bogotá, Colombia

T

Thierry Petit

E

Emilie Kaczmarek

S

Sylvain Ladoire

Centre Georges Francois Leclerc, Dijon, France

N

Nathalie Quenel Tueux

L

Luis Teixeira

J

Jean Christophe Thery

Université de Rouen Normandie, Inserm U1245, FHU G4 Genomics, CHU Rouen, Département de Génétique, Rouen, France

S

Sophie Abadie-Lacourtoisie

A

Alain Lortholary

Groupe d'Investigateurs National des Etudes des Cancers Ovariens et du sein (GINECO) and Hôpital Privé du Confluent, Nantes, France

E

Elisabeth Luporsi

H

Hubert Orfeuvre

N

Nathalie Bonnin

G

Giampaolo Bianchini

IRCCS Ospedale San Raffaele, Milan

F

Federico Piacentini

F

Francesco Cognetti

P

Paolo Marchetti

IDI-IRCCS, Roma, Italy

E

Evaristo Maiello

M

Marina Cazzaniga

Phase 1 Research Unit, Fondazione IRCCS San Gerardo, Monza, Italy

V

Valentina Guarneri

Veneto Institute of Oncology, Istituto di Ricovero e Cura a Carattere Scientifico, Padua, Italy

M

Marco Colleoni

L

Laura Doni

R

Roberto Bordonaro

C

Claudio Zamagni

IRCCS Azienda Ospedaliero Universitaria di Bologna, Bologna, Italy

G

Giulia Bianchi

Medical Oncology Department, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy

L

Laura Biganzoli

M

Michael Thirlwell

X

Xinni Song

The Ottawa Hospital Cancer Centre, Ottawa, ON, Canada

A

Anil Joy

S

Sara Taylor

T

Teresa Helsten

M

Madhu Chaudhry

H

Haythem Ali

S

Shaker Dakhil

11Cancer Center of Kansas, Wichita, United States

R

Rex Mowat

A

Adam Brufsky

Hillman Cancer Center, Magee-Womens Hospital, University of Pittsburgh Medical Center, Pittsburgh

M

Melody Cobleigh

M

Manuel Modiano

M

Michelina Cairo

M

Michael Meshad

M

Michael A. Danso

Department of Medical Oncology, Brock Cancer Center, Virginia Oncology Associates, Norfolk

J

Jay Andersen

A

Allyson Harroff

R

Rami Owera

A

Anne Favret

Virginia Cancer Specialists, PC, Fairfax, VA

J

Joyce O'Shaughnessy

Baylor University Medical Center, Texas Oncology, Sarah Cannon Research Institute, Dallas, TX

T

Timothy Pluard

P

Paula Rosenblatt

University of Maryland Greenebaum Comprehensive Cancer Center, Baltimore, MD

S

Sharad Jain

J

Jeanette Dupont Jensen

N

Nina Jeppesen

K

Kim Wedervang

P

Per Edlund

H

Henrik Lindman

R

Renske Altena

Department of Oncology-Pathology, Karolinska Institutet and Karolinska Comprehensive Cancer Center, Karolinska University Hospital, Stockholm, Sweden

L

Leif Klint

T

Tira Tan Jing Ying

J

Joline Lim Si Jing