Ruxolitinib Versus Best Available Therapy in Patients With Steroid-Refractory Acute Graft-Versus-Host Disease: Final Analysis From the Randomized Phase III REACH2 Trial

M Mohamad Mohty G Gerard Socie (Hematology Transplantation, Assistance Publique–Hôpitaux de Paris, Hospital Saint Louis, Paris, France) J Jeff Szer (5Peter MacCallum Cancer Centre, The Royal Melbourne Hospital, Melbourne, Australia) D Dietger Niederwieser (2University of Pennsylvania & Penn Medicine, Pennsylvania, United States) J Jason Butler (1University of Florida, Medicine, Gainesville, United States) E Eva Wagner-Drouet (Department of Hematology and Oncology, University Medical Center Mainz, Mainz, Germany) R Reuven Or (Cancer Immunotherapy Research Center, Hadassah University Medical Center, Jerusalem, Israel) T Tsila Rovenvald-Zuckerman (Hematology Institute and Bone Marrow Transplantation, Clinical Research Institute at Rambam, Rambam Health Care Campus, Rappaport Faculty of Medicine, Technion, Haifa, Israel) S Sinem Civriz Bozdag (Ankara University Medical Faculty, Ankara, Turkey) E Edouard Forcade G Giovanni Grillo (17Dipartimento di Ematologia e Trapianto di midollo, ASST Grande Ospedale Metropolitano Niguarda, Milan, Italy) N Nicolaus Kröger (From the Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.) F Friedrich Stölzel (8Department of Internal Medicine II, Hematology and Oncology, University Medical Center Schleswig-Holstein, Campus Kiel, Kiel, Germany) D Domenico Russo (Institute of Endotypes in Oncology, Metabolism and Immunology “G. Salvatore,” National Research Council of Italy) J Jaime Sanz (2Hospital Universitari i Politècnic La Fe, Valencia Departament de Medicina Universitat de Valencia, CIBERONC, Instituto Carlos III, Madrid, Spain, Hematology Department, Valencia, Spain) R Rajendra Sarkar (Novartis Healthcare Pvt Ltd, Hyderabad, India) T Tommaso Stefanelli (Novartis Pharma, Basel, Switzerland) C Celine Wilke (Novartis Pharma, Basel, Switzerland) R Robert Zeiser N Nikolas von Bubnoff

Abstract

Approximately 30%-50% of patients develop acute graft-versus-host disease (aGVHD) after allogeneic hematopoietic cell transplantation (Allo-HCT), representing a major limitation of this treatment. Although corticosteroids remain the standard first-line therapy for aGVHD, up to 50% of patients become steroid-refractory (SR). REACH2 is a phase III study of ruxolitinib versus best available therapy (BAT) in patients age 12 years and older with SR-aGVHD after Allo-HCT. We present the final efficacy and safety outcomes from REACH2 after 24 months of treatment. Cumulative median (range) duration of response was 167 (22-677) days with ruxolitinib and 106 (10-526) days with BAT. Median overall survival and event-free survival were 10.7 and 8.3 months for ruxolitinib, compared with 5.8 and 4.2 months, respectively, with BAT. Median failure-free survival was significantly longer with ruxolitinib than with BAT (4.86 v 1.02 months, P < .001). Similar numbers of nonrelapse mortality events were observed with ruxolitinib and BAT (72 v 71), and malignancy relapse/progression events remained low across both groups. Numerically higher chronic GVHD rates were noted with ruxolitinib than with BAT from 12 months; however, 95% confidence intervals overlapped. Safety observations were consistent with the primary analysis results. Ruxolitinib provided efficacy advantages over BAT in patients with SR-aGVHD over 24 months.

Article Details

Volume / Issue Vol. 43, Issue 34
Published December 01, 2025
Pages 3639-3645
ISSN 0732-183X
Publisher Lippincott Williams & Wilkins

Journal Info

Journal of Clinical Oncology

Lippincott Williams & Wilkins

ISSN: 0732-183X Health Sciences

Authors (20)

M

Mohamad Mohty

G

Gerard Socie

Hematology Transplantation, Assistance Publique–Hôpitaux de Paris, Hospital Saint Louis, Paris, France

J

Jeff Szer

5Peter MacCallum Cancer Centre, The Royal Melbourne Hospital, Melbourne, Australia

D

Dietger Niederwieser

2University of Pennsylvania & Penn Medicine, Pennsylvania, United States

J

Jason Butler

1University of Florida, Medicine, Gainesville, United States

E

Eva Wagner-Drouet

Department of Hematology and Oncology, University Medical Center Mainz, Mainz, Germany

R

Reuven Or

Cancer Immunotherapy Research Center, Hadassah University Medical Center, Jerusalem, Israel

T

Tsila Rovenvald-Zuckerman

Hematology Institute and Bone Marrow Transplantation, Clinical Research Institute at Rambam, Rambam Health Care Campus, Rappaport Faculty of Medicine, Technion, Haifa, Israel

S

Sinem Civriz Bozdag

Ankara University Medical Faculty, Ankara, Turkey

E

Edouard Forcade

G

Giovanni Grillo

17Dipartimento di Ematologia e Trapianto di midollo, ASST Grande Ospedale Metropolitano Niguarda, Milan, Italy

N

Nicolaus Kröger

From the Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.

F

Friedrich Stölzel

8Department of Internal Medicine II, Hematology and Oncology, University Medical Center Schleswig-Holstein, Campus Kiel, Kiel, Germany

D

Domenico Russo

Institute of Endotypes in Oncology, Metabolism and Immunology “G. Salvatore,” National Research Council of Italy

J

Jaime Sanz

2Hospital Universitari i Politècnic La Fe, Valencia Departament de Medicina Universitat de Valencia, CIBERONC, Instituto Carlos III, Madrid, Spain, Hematology Department, Valencia, Spain

R

Rajendra Sarkar

Novartis Healthcare Pvt Ltd, Hyderabad, India

T

Tommaso Stefanelli

Novartis Pharma, Basel, Switzerland

C

Celine Wilke

Novartis Pharma, Basel, Switzerland

R

Robert Zeiser

N

Nikolas von Bubnoff