Low rates of chronic graft-versus-host disease with ruxolitinib maintenance following allogeneic HCT

Z Zachariah Defilipp (7Massachusetts General Hospital, Hematopoietic Cell Transplant and Cellular Therapy Program, 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) L Laura W. Knight (3Cancer Center Protocol Office, Massachusetts General Hospital, Boston, MA) S Suzanne M. O’Connor (3Cancer Center Protocol Office, Massachusetts General Hospital, Boston, MA) S Shilton E. Dhaver (3Cancer Center Protocol Office, Massachusetts General Hospital, Boston, MA) M Meghan White (3Cancer Center Protocol Office, Massachusetts General Hospital, Boston, MA) B Bhagirathbhai Dholaria M Mark A. Schroeder (5Bone Marrow Transplantation and Leukemia Section, Division of Oncology, Washington University School of Medicine in Saint Louis, Saint Louis, MO) S Sumithira Vasu (29Department of Internal Medicine, The Ohio State University, Columbus, OH) S Sameem Abedin (1Medical College of Wisconsin, Medicine - Hematology/Oncology, Milwaukee, United States) J Jooho Chung (1Hematopoietic Cell Transplant and Cellular Therapy Program, Massachusetts General Hospital, Boston, MA) A Areej El-Jawahri (1Cellular Immunotherapy Program, Massachusetts General Hospital Cancer Center, Harvard Medical School, Boston, MA) M Matthew J. Frigault (3Massachusetts General Hospital, Boston, MA) S Steven McAfee (1Hematopoietic Cell Transplant and Cellular Therapy Program, Massachusetts General Hospital, Boston, MA) R Richard A. Newcomb (1Hematopoietic Cell Transplant and Cellular Therapy Program, Massachusetts General Hospital, Boston, MA) P Paul V. O’Donnell (1Hematopoietic Cell Transplant and Cellular Therapy Program, Massachusetts General Hospital, Boston, MA) T Thomas R. Spitzer (Department of Medicine, Harvard Medical School, Boston) Y Yi-Bin Chen (1Cellular Immunotherapy Program, Massachusetts General Hospital Cancer Center, Harvard Medical School, Boston, MA) G Gabriela S. Hobbs (10Department of Hematology-Oncology, Massachusetts General Hospital Cancer Center and Harvard Medical School, Boston, MA)

Abstract

Abstract Despite recent advances in graft-versus-host disease (GVHD) prophylaxis, novel approaches to effective prevention of chronic GVHD (cGVHD) remain of high importance. In this prospective, multicenter, phase 2 trial, ruxolitinib, an oral inhibitor of Janus kinase (JAK) 1 and 2, was administered as maintenance therapy after reduced-intensity allogeneic hematopoietic cell transplantation (HCT). GVHD prophylaxis consisted of tacrolimus and methotrexate. Ruxolitinib began between day +30 to 100 and was administered continuously in 28-day cycles for up to 24 cycles. Seventy-eight participants were enrolled before HCT; 63 participants received the intervention. The median start date of ruxolitinib after HCT was day +45. The most common grade ≥3 adverse events were neutropenia, thrombocytopenia, and anemia. Seven participants experienced grade ≥3 infectious events. GVHD-free, relapse-free survival at 1 year after HCT, the primary end point, was 70%. Grade 3 to 4 acute GVHD at 6 months was 4.8%, and moderate-severe cGVHD at 2 years was 16%. cGVHD requiring systemic therapy was 9.5% at 1 year and 13% at 2 years. Overall survival and progression-free survival at 2 years were 76% and 68%, respectively. Prolonged administration of ruxolitinib following HCT is associated with low rates of clinically significant cGVHD. The incorporation of JAK inhibition into GVHD prevention approaches warrants further investigation. This trial was registered at www.clinicaltrials.gov as #NCT03286530.

Article Details

Journal Blood
Volume / Issue Vol. 145, Issue 20
Published May 15, 2025
Pages 2312-2316
ISSN 0006-4971
Publisher Elsevier BV

Journal Info

Blood

Elsevier BV

ISSN: 0006-4971 Health Sciences

Authors (19)

Z

Zachariah Defilipp

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

L

Laura W. Knight

3Cancer Center Protocol Office, Massachusetts General Hospital, Boston, MA

S

Suzanne M. O’Connor

3Cancer Center Protocol Office, Massachusetts General Hospital, Boston, MA

S

Shilton E. Dhaver

3Cancer Center Protocol Office, Massachusetts General Hospital, Boston, MA

M

Meghan White

3Cancer Center Protocol Office, Massachusetts General Hospital, Boston, MA

B

Bhagirathbhai Dholaria

M

Mark A. Schroeder

5Bone Marrow Transplantation and Leukemia Section, Division of Oncology, Washington University School of Medicine in Saint Louis, Saint Louis, MO

S

Sumithira Vasu

29Department of Internal Medicine, The Ohio State University, Columbus, OH

S

Sameem Abedin

1Medical College of Wisconsin, Medicine - Hematology/Oncology, Milwaukee, United States

J

Jooho Chung

1Hematopoietic Cell Transplant and Cellular Therapy Program, Massachusetts General Hospital, Boston, MA

A

Areej El-Jawahri

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

M

Matthew J. Frigault

3Massachusetts General Hospital, Boston, MA

S

Steven McAfee

1Hematopoietic Cell Transplant and Cellular Therapy Program, Massachusetts General Hospital, Boston, MA

R

Richard A. Newcomb

1Hematopoietic Cell Transplant and Cellular Therapy Program, Massachusetts General Hospital, Boston, MA

P

Paul V. O’Donnell

1Hematopoietic Cell Transplant and Cellular Therapy Program, Massachusetts General Hospital, Boston, MA

T

Thomas R. Spitzer

Department of Medicine, Harvard Medical School, Boston

Y

Yi-Bin Chen

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

G

Gabriela S. Hobbs

10Department of Hematology-Oncology, Massachusetts General Hospital Cancer Center and Harvard Medical School, Boston, MA