Itacitinib for prevention of graft-versus-host disease and cytokine release syndrome in haploidentical transplantation

R Ramzi Abboud (4Washington University St Louis, St Louis, United States) M Mark A. Schroeder (5Bone Marrow Transplantation and Leukemia Section, Division of Oncology, Washington University School of Medicine in Saint Louis, Saint Louis, MO) M Michael P. Rettig (1Division of Oncology, Department of Medicine, Washington University School of Medicine, St. Louis, MO) R Reyka G. Jayasinghe (1Division of Oncology, Department of Medicine, Washington University School of Medicine, St. Louis, MO) F Feng Gao J Jeremy Eisele (1Division of Oncology, Department of Medicine, Washington University School of Medicine, St. Louis, MO) L Leah Gehrs (1University of Alabama at Birmingham, Pathology, Birmingham, United States) J Julie Ritchey (1Washington University in St. Louis, St. Louis, United States) J Jaebok Choi (1Division of Oncology, Department of Medicine, Washington University School of Medicine, St. Louis, MO) C Camille N. Abboud (1Division of Oncology, Department of Medicine, Washington University School of Medicine, St. Louis, MO) I Iskra Pusic (6Washington University in St. Louis School of Medicine, St. Louis, United States) M Meagan Jacoby (1Washington University School of Medicine in St Louis, Hematology-Oncology, St Louis, United States) P Peter Westervelt (11Massachusetts General Hospital, Boston, United States) M Matthew Christopher (1Division of Oncology, Department of Medicine, Washington University School of Medicine, St. Louis, MO) A Amanda Cashen (1Washington University School of Medicine, Division of Oncology, St Louis, United States) A Armin Ghobadi (5Washington University School of Medicine, St. Louis, United States) K Keith Stockerl-Goldstein (1Washington University School of Medicine, Division of Oncology, Department of Medicine, Saint Louis, United States) G Geoffrey L. Uy (Division of Oncology, Washington University School of Medicine, Saint Louis, Missouri, United States) J John F. DiPersio (Department of Medicine, Washington University School of Medicine, St. Louis)

Abstract

Abstract Haploidentical hematopoietic cell transplantation (haplo-HCT) is an increasingly used treatment for hematologic malignancies. Although posttransplant cyclophosphamide (PtCy) has improved graft-versus-host disease (GVHD) prophylaxis in haplo-HCT, patients continue to experience life-threatening complications. Interferon gamma and interleukin-6 are central in the pathophysiology of GVHD and cytokine release syndrome (CRS), and both cytokines signal through Janus kinase 1 (JAK-1). We tested the effect of adding the JAK-1 selective inhibitor, itacitinib, to PtCy–haplo-HCT to mitigate these complications and improve overall survival (OS). This open-label, single-arm study evaluated the safety and efficacy of itacitinib combined with standard GVHD prophylaxis after haplo-HCT. A total of 42 patients were treated with itacitinib 200 mg daily from day −3 through +100 or +180, followed by a taper. Itacitinib resulted in low CRS grades, all patients had grade 0 (22%) or grade 1 (78%) CRS and there were no cases of grade 2 to 5 CRS. There were no cases of primary graft failure. No patients developed grade 3 to 4 acute GVHD (aGVHD) through day +180. The cumulative incidence of grade 2 aGVHD at day +100 was 21.9%. The 1-year cumulative incidence of moderate or severe chronic GVHD was 5%. The cumulative incidence of relapse at 2 years was 14%. OS at 1 year was 80%. The cumulative incidence of nonrelapse mortality (NRM) at day 180 was 8%. Itacitinib, when added to standard GVHD prophylaxis, was well tolerated and resulted in low rates of CRS, acute and chronic GVHD, and NRM, and encouraging rates of GVHD-free relapse-free survival and OS after haplo-HCT. This trial was registered at www.ClinicalTrials.gov as #NCT03755414.

Article Details

Journal Blood
Volume / Issue Vol. 145, Issue 13
Published March 27, 2025
Pages 1382-1394
ISSN 0006-4971
Publisher Elsevier BV

Journal Info

Blood

Elsevier BV

ISSN: 0006-4971 Health Sciences

Authors (19)

R

Ramzi Abboud

4Washington University St Louis, St Louis, United States

M

Mark A. Schroeder

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

M

Michael P. Rettig

1Division of Oncology, Department of Medicine, Washington University School of Medicine, St. Louis, MO

R

Reyka G. Jayasinghe

1Division of Oncology, Department of Medicine, Washington University School of Medicine, St. Louis, MO

F

Feng Gao

J

Jeremy Eisele

1Division of Oncology, Department of Medicine, Washington University School of Medicine, St. Louis, MO

L

Leah Gehrs

1University of Alabama at Birmingham, Pathology, Birmingham, United States

J

Julie Ritchey

1Washington University in St. Louis, St. Louis, United States

J

Jaebok Choi

1Division of Oncology, Department of Medicine, Washington University School of Medicine, St. Louis, MO

C

Camille N. Abboud

1Division of Oncology, Department of Medicine, Washington University School of Medicine, St. Louis, MO

I

Iskra Pusic

6Washington University in St. Louis School of Medicine, St. Louis, United States

M

Meagan Jacoby

1Washington University School of Medicine in St Louis, Hematology-Oncology, St Louis, United States

P

Peter Westervelt

11Massachusetts General Hospital, Boston, United States

M

Matthew Christopher

1Division of Oncology, Department of Medicine, Washington University School of Medicine, St. Louis, MO

A

Amanda Cashen

1Washington University School of Medicine, Division of Oncology, St Louis, United States

A

Armin Ghobadi

5Washington University School of Medicine, St. Louis, United States

K

Keith Stockerl-Goldstein

1Washington University School of Medicine, Division of Oncology, Department of Medicine, Saint Louis, United States

G

Geoffrey L. Uy

Division of Oncology, Washington University School of Medicine, Saint Louis, Missouri, United States

J

John F. DiPersio

Department of Medicine, Washington University School of Medicine, St. Louis