Steroid Refractory GVHD
Conditions
Keywords
acute lymphoblastic leukemia, hematopoietic stem cell transplantation
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.
Interventions
DRUGRuxolitinib
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.
Sponsors
Yi-Lun Wang
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Cases with steroid-refractory GVHD would be given an add-on therapy of age/weight adjusted dosage of Ruxolitinib
Eligibility
Sex/Gender
ALL
Age
3 Months to 30 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| Overall survival | 3 years | From diagnosis of SR-GVHD to mortality due to any cause |
Countries
Taiwan
Outcome results
None listed