Randomized, Placebo-Controlled Trial of B-Cell Depletion for Prevention of Corticosteroid-Requiring Chronic Graft-Versus-Host Disease

C Corey Cutler (1Dana Farber Cancer Institute, Boston, United States) H Haesook T. Kim (2Department of Data Science, Dana-Farber Cancer Institute and Harvard T.H. Chan School of Public Health, Boston, MA) H Hassan El Banna (Division of Transplantation and Cellular Therapy, Dana-Farber Cancer Institute, Boston, MA) E Elizabeth Halloran (Division of Transplantation and Cellular Therapy, Dana-Farber Cancer Institute, Boston, MA) E Emily Matozel (Division of Transplantation and Cellular Therapy, Dana-Farber Cancer Institute, Boston, MA) V Vincent T. Ho (Dana-Farber Cancer Institute, Boston, MA) J John Koreth (1Dana Farber Cancer Institute, Boston, United States) M Mahasweta Gooptu (1Dana Farber Cancer Institute, Boston, United States) R Roman Shapiro (1Dana Farber Cancer Institute, Boston, United States) A Amar Kelkar (1Dana Farber Cancer Institute, Boston, United States) C Christopher Gibson (Dana-Farber Cancer Institute, Boston, Massachusetts, United States) S Sarah Nikiforow (2Department of Medical Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA) P Prashant Nageshwar (1Dana Farber Cancer Institute, Boston, United States) C Carol Reynolds (1Dana Farber Cancer Institute, Boston, United States) M Michela Ansuinelli (1Sapienza University, Rome, Italy) R Rakuyo Tamada (Division of Transplantation and Cellular Therapy, Dana-Farber Cancer Institute, Boston, MA) C Chloe Au (Division of Transplantation and Cellular Therapy, Dana-Farber Cancer Institute, Boston, MA) K Kevin Panaro (Division of Transplantation and Cellular Therapy, Dana-Farber Cancer Institute, Boston, MA) C Casey Gervais (Division of Transplantation and Cellular Therapy, Dana-Farber Cancer Institute, Boston, MA) Z Zachariah Defilipp (7Massachusetts General Hospital, Hematopoietic Cell Transplant and Cellular Therapy Program, Boston, United States) A Areej El-Jawahri (1Cellular Immunotherapy Program, Massachusetts General Hospital Cancer Center, Harvard Medical School, Boston, MA) Y Yi-Bin Chen (1Cellular Immunotherapy Program, Massachusetts General Hospital Cancer Center, Harvard Medical School, Boston, MA) N Najla El Jurdi (3CIBMTR® (Center for International Blood and Marrow Transplant Research), Medical College of Wisconsin, Milwaukee, United States) D Daniel Weisdorf (1University of Minnesota, Division of Hematology, Oncology and Transplantation, Minneapolis, United States) D Daniel Couriel (1Huntsman Cancer Institute, SALT LAKE CITY, United States) C Catherine Lee S Sally Arai B Bita Sahaf J Juliana Bacigalupi (BMT and Cell Therapy Program, Stanford University, Palo Alto, CA) K Kathleen Ji (BMT and Cell Therapy Program, Stanford University, Palo Alto, CA) R Robert Soiffer (1Dana Farber Cancer Institute, Boston, United States) D David Miklos J Joseph H. Antin (1Dana-Farber Cancer Institute) J Jerome Ritz (Dana–Farber Cancer Institute, Boston)

Abstract

PURPOSE Chronic graft-versus-host disease (cGVHD) is a multisystem alloimmune disorder associated with abnormal B-cell biology and aberrant antibody responses. As B-cell–directed therapy can effectively treat established cGVHD, we tested whether prophylactic B-cell depletion could prevent the development of corticosteroid-requiring cGVHD following allogeneic transplantation. METHODS We performed a randomized, placebo-controlled, and blinded trial comparing four doses of the B-cell–depleting antibody obinutuzumab (1,000 mg once on days 90, 180, 270, and 365 after transplantation) with placebo in transplant recipients receiving tacrolimus-based GVHD prevention at higher risk of cGVHD. The primary end point was the 1-year incidence of corticosteroid-requiring cGVHD. We measured antibody responses against Y chromosome–encoded minor histocompatibility (H-Y) antigens and correlated their occurrence with corticosteroid-requiring cGVHD incidence. RESULTS One hundred seventy-eight participants were analyzed. The prophylactic administration of obinutuzumab resulted in profound B-cell depletion, a significant reduction in the incidence of steroid-requiring cGVHD at 1 year (13.3% v 35.2%; P = .0005), and an improvement in immunosuppression-free, relapse-free survival (48% v 34% at 2 years; P = .02). Neutropenia was more common in the obinutuzumab arm, but nonrelapse mortality was not different. In participants without preformed H-Y antibodies at the time of study intervention, obinutuzumab resulted in the most significant reduction in steroid-requiring cGVHD at 12 months (8.6%) compared with obinutuzumab participants with H-Y antibodies (40%) or placebo participants regardless of antibody status (41% with antibodies, 57% without antibodies). CONCLUSION In allogeneic transplant recipients at higher risk of cGVHD, early B-cell depletion results in a significant reduction in the incidence of corticosteroid-requiring cGVHD.

Article Details

Volume / Issue Vol. 44, Issue 16
Published June 01, 2026
Pages 1529-1539
ISSN 0732-183X
Publisher Lippincott Williams & Wilkins

Journal Info

Journal of Clinical Oncology

Lippincott Williams & Wilkins

ISSN: 0732-183X Health Sciences

Authors (34)

C

Corey Cutler

1Dana Farber Cancer Institute, Boston, United States

H

Haesook T. Kim

2Department of Data Science, Dana-Farber Cancer Institute and Harvard T.H. Chan School of Public Health, Boston, MA

H

Hassan El Banna

Division of Transplantation and Cellular Therapy, Dana-Farber Cancer Institute, Boston, MA

E

Elizabeth Halloran

Division of Transplantation and Cellular Therapy, Dana-Farber Cancer Institute, Boston, MA

E

Emily Matozel

Division of Transplantation and Cellular Therapy, Dana-Farber Cancer Institute, Boston, MA

V

Vincent T. Ho

Dana-Farber Cancer Institute, Boston, MA

J

John Koreth

1Dana Farber Cancer Institute, Boston, United States

M

Mahasweta Gooptu

1Dana Farber Cancer Institute, Boston, United States

R

Roman Shapiro

1Dana Farber Cancer Institute, Boston, United States

A

Amar Kelkar

1Dana Farber Cancer Institute, Boston, United States

C

Christopher Gibson

Dana-Farber Cancer Institute, Boston, Massachusetts, United States

S

Sarah Nikiforow

2Department of Medical Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA

P

Prashant Nageshwar

1Dana Farber Cancer Institute, Boston, United States

C

Carol Reynolds

1Dana Farber Cancer Institute, Boston, United States

M

Michela Ansuinelli

1Sapienza University, Rome, Italy

R

Rakuyo Tamada

Division of Transplantation and Cellular Therapy, Dana-Farber Cancer Institute, Boston, MA

C

Chloe Au

Division of Transplantation and Cellular Therapy, Dana-Farber Cancer Institute, Boston, MA

K

Kevin Panaro

Division of Transplantation and Cellular Therapy, Dana-Farber Cancer Institute, Boston, MA

C

Casey Gervais

Division of Transplantation and Cellular Therapy, Dana-Farber Cancer Institute, Boston, MA

Z

Zachariah Defilipp

7Massachusetts General Hospital, Hematopoietic Cell Transplant and Cellular Therapy Program, Boston, United States

A

Areej El-Jawahri

1Cellular Immunotherapy Program, Massachusetts General Hospital Cancer Center, Harvard Medical School, Boston, MA

Y

Yi-Bin Chen

1Cellular Immunotherapy Program, Massachusetts General Hospital Cancer Center, Harvard Medical School, Boston, MA

N

Najla El Jurdi

3CIBMTR® (Center for International Blood and Marrow Transplant Research), Medical College of Wisconsin, Milwaukee, United States

D

Daniel Weisdorf

1University of Minnesota, Division of Hematology, Oncology and Transplantation, Minneapolis, United States

D

Daniel Couriel

1Huntsman Cancer Institute, SALT LAKE CITY, United States

C

Catherine Lee

S

Sally Arai

B

Bita Sahaf

J

Juliana Bacigalupi

BMT and Cell Therapy Program, Stanford University, Palo Alto, CA

K

Kathleen Ji

BMT and Cell Therapy Program, Stanford University, Palo Alto, CA

R

Robert Soiffer

1Dana Farber Cancer Institute, Boston, United States

D

David Miklos

J

Joseph H. Antin

1Dana-Farber Cancer Institute

J

Jerome Ritz

Dana–Farber Cancer Institute, Boston