Sclerotic chronic graft-versus-host disease (scl-cGVHD) is a severe subtype of chronic GVHD after allogeneic hematopoietic stem cell transplantation (allo-HSCT), characterized by skin sclerosis, fascial involvement, and restricted joint mobility, leading to substantial functional impairment and reduced quality of life. Current standard therapies, including corticosteroids and other immunosuppressive agents, have limited efficacy in patients with refractory or steroid-dependent disease. Madecassoside tablets, a standardized extract of Centella asiatica, have demonstrated anti-inflammatory and anti-fibrotic properties in previous studies and may provide therapeutic benefit in fibrotic diseases. This study is a prospective, multicenter, single-arm phase 2 clinical trial designed to evaluate the efficacy and safety of madecassoside tablets combined with standard therapy in patients with scl-cGVHD after allo-HSCT. Eligible participants will receive oral madecassoside tablets (0.2 g three times daily) in addition to standard treatment for 6 months. Clinical assessments will be performed at baseline and at weeks 4, 8, 12, and 24. The primary endpoint is the improvement rate of skin and/or joint/fascia NIH cGVHD scores at 6 months according to the 2014 NIH consensus criteria. Secondary endpoints include overall cGVHD response rate, failure-free survival, non-relapse mortality, corticosteroid dose reduction, patient-reported outcomes, and safety assessments. Peripheral blood samples will also be collected to explore changes in inflammatory cytokines and lymphocyte subsets during treatment.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
30
Madecassoside tablets will be administered orally at a dose of 0.2 g (2 tablets) three times daily for 6 months in combination with standard therapy.
Standard therapy may include corticosteroids, calcineurin inhibitors, ruxolitinib, belumosudil, topical medications, and supportive care according to institutional guidelines and physician discretion.
Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
Wuhan, Hubei, China
RECRUITINGChange in Skin and/or Joint/Fascia NIH cGVHD Scores
The proportion of participants achieving complete response (CR), partial response (PR), stable disease (SD), or progressive disease (PD) in skin and/or joint/fascia manifestations according to the 2014 NIH chronic graft-versus-host disease response criteria after 6 months of treatment.
Time frame: 6 months
Overall cGVHD Response Rate
Overall response rate of chronic graft-versus-host disease based on the 2014 NIH response criteria.
Time frame: 6 months
Failure-Free Survival
Failure-free survival defined as survival without relapse, non-relapse mortality, or addition of new systemic therapy.
Time frame: 2 years
Non-Relapse Mortality
Incidence of death without relapse of the underlying disease during follow-up.
Time frame: 2 years
Corticosteroid Dose Reduction
Proportion of participants achieving at least 50% reduction or discontinuation of corticosteroid therapy.
Time frame: 6 months
Patient-Reported Outcomes
Changes in patient-reported outcomes including Lee Symptom Scale (LSS), Scleroderma Health Assessment Questionnaire (SHAQ), and Photographic Range of Motion (P-ROM) scores.
Time frame: Baseline to 6 months
Traditional Chinese Medicine Symptom Score
Changes in Traditional Chinese Medicine symptom scores during treatment.
Time frame: Baseline to 6 months
Safety and Adverse Events
Incidence and severity of adverse events assessed according to CTCAE version 5.0.
Time frame: Up to 2 years
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