Ruxolitinib Based GVHD Prophylaxis Regimen Before, During, and After Hematopoietic Cell Transplantation in Older Adult Patients With Acquired Aplastic Anemia

Summary

This phase II trial tests how well a ruxolitinib-based graft versus host disease (GVHD) prevention (prophylaxis) regimen works before, during, and after bone marrow/stem cell transplantation (hematopoietic cell transplantation \[HCT\]) in patients with acquired aplastic anemia. Acquired aplastic anemia (AA) is a condition in which the bone marrow is unable to produce blood cells. Affected patients typically present with infections due to abnormally low number of neutrophils, bleeding due to low platelet count, and/or fatigue due to a lower-than-normal number of red blood cells (anemia). Its incidence varies with age, occurring most frequently in patients aged 2-5 years, 20-25 years, and 55 years and older. Treatment of AA includes either immunosuppressive therapy (IST) or bone marrow/stem cell transplantation (HCT) with first-line therapy in younger adults often being HCT, while adults over 40 still frequently trial IST first due to the morbidity and mortality concerns with HCT. GVHD is a common complication after donor stem cell transplantation, resulting from donor immune cells recognizing recipients' cells and attacking them. Ruxolitinib, a drug in a class of oral medications called JAK inhibitors has been approved for the treatment of acute and chronic GVHD. It has also been shown to decrease GVHD when used in the prevention setting in patients with myelofibrosis. The current study aims to assess whether adding ruxolitinib to a standard GVHD prevention regimen may reduce the risk of Grade II-IV acute and chronic GVHD after bone marrow/stem cell transplantation in older patients with acquired aplastic anemia.

Conditions

• Aplastic Anemia

Interventions

PROCEDURE

Biospecimen Collection

Undergo blood sample collection

PROCEDURE

Bone Marrow Aspiration

Undergo bone marrow biopsy and aspiration

PROCEDURE

Bone Marrow Biopsy

Undergo bone marrow biopsy and aspiration

PROCEDURE

Bone Marrow Transplantation

Undergo bone marrow transplant

PROCEDURE

Computed Tomography

Undergo CT

DRUG

Cyclosporine

Given PO

DRUG

Fludarabine

Given IV

DRUG

Granulocyte Colony-Stimulating Factor

Given SC

DRUG

Mycophenolate Mofetil

Given PO

PROCEDURE

Peripheral Blood Stem Cell Transplantation

Undergo PBSC transplantation

DRUG

Ruxolitinib

Given PO

DRUG

Sirolimus

Given PO

RADIATION

Total-Body Irradiation

Undergo TBI

PROCEDURE

Multigated Acquisition Scan

Undergo MUGA

PROCEDURE

Echocardiography

Undergo ECHO

Sponsors & Collaborators

• Incyte Corporation

  collaborator INDUSTRY

• Fred Hutchinson Cancer Center

  lead OTHER

Principal Investigators

• Rachel B. Salit, MD · Fred Hutch/University of Washington Cancer Consortium

Study Design

Allocation

NA

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Min Age

18 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2025-09-25

Primary Completion

2026-01-23

Completion

2026-10-15

FDA Drug

Yes

Countries

• United States

Study Locations