Ruxolitinib as add-on Therapy in Steroid-refractory Graft-vs-host Disease

Phase 3

Completed

• Conditions: Steroid Refractory GVHD
• Interventions: Drug: Ruxolitinib

• Registration Number: NCT05663827
• Lead Sponsor: Yi-Lun Wang

Brief Summary

Steroid-refractory graft-vs-host disease (SR-GVHD) is a major cause of mortality after allogeneic hematopoietic stem cell transplantation (HSCT). We sought to evaluate the effect and safety of ruxolitinib (RUX) add-on in the treatment of patients with SR-GVHD.

Detailed Description

Nowadays, second-line treatment for SR-GVHD differs between centers and depends on the physician's experience. The agents commonly used as second and further lines of therapy include anti-thymocyte globulin, mycophenolate mofetil, calcineurin inhibitor, or extracorporeal photopheresis. However, the response varied. This study highlighted that corticosteroid (CS) is not a panacea for treating GVHD, and some patients with remission still experience further relapse. Additionally, the identification of multiple infections adds complexity.

Hope was supposed to come in 2019 after the Food and Drug Administration (FDA) approved RUX for the therapy of SR-GVHD in adult and pediatric patients \> 12 years. RUX add-on should be withheld as the risks outweigh the benefits, especially for patients who developed scleroderma and bronchiolitis obliterans. RUX can be added on as needed for the treatment of GVHD, especially in patients receiving DLI as relapse therapy with the conversion from mixed chimerism to complete chimerism. Despite some studies showing the efficacy of RUX across affected organs, some other data bring the opposite results. Thus we conducted this study to reinforce the efficacy of RUX as an add-on therapy for SR-GVHD.

Study Timeline

• Study Start: 2019-01-01
• Primary Completion: 2020-01-01
• Study First Submitted: 2022-11-23
• Study Completion: 2022-11-30
• Study First Submitted QC: 2022-12-15
• Study First Posted: 2022-12-23
• Status Verified: 2024-03
• Last Update Submitted: 2024-03-02
• Last Update Posted: 2024-03-05

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 6

Inclusion Criteria

• Patients diagnosed with hematologic disorder

Exclusion Criteria

• Those who are not applicable of receiving Ruxolitinib as immunodeficiency-modulation therapy
• Those who are actively encountering acute infectious illness

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Ruxolitinib add-on group	Ruxolitinib	Once diagnosed with steroid-refractory GVHD, after discussion with family, as per their willing, Ruxolitinib will be administered as add-on therapy. Its dose depends on participants' age and body weight. Usually a dosage of 5mg once per day will be applied as initiation and titrated in accordance with clinical response.

Primary Outcome Measures

Name	Time	Method
GI symptoms secondary to GVHD	3 years	Diarrhea event exceeds a frequency of 5 times a day
Skin symptoms secondary to GVHD	3 years	Skin rash extends more than 25% of body surface area

Secondary Outcome Measures

Name	Time	Method
Overall survival	3 years	From diagnosis of SR-GVHD to mortality due to any cause

Trial Locations

Locations (1)

Chang Gung Memorial Hospital; 🇨🇳 Taoyuan, Taiwan