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.
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 Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
6
A dosage of 5mg once daily will be applied as initiation. After one week use, dose escalation or de-escalation would depend on clinical response.
Chang Gung Memorial Hospital
Taoyuan, Taiwan
GI symptoms secondary to GVHD
Diarrhea event exceeds a frequency of 5 times a day
Time frame: 3 years
Skin symptoms secondary to GVHD
Skin rash extends more than 25% of body surface area
Time frame: 3 years
Overall survival
From diagnosis of SR-GVHD to mortality due to any cause
Time frame: 3 years
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