The purpose of this study is to determine whether the response to treatment for extensive chronic Graft versus Host Disease (cGvHD)is improved with the addition of myfortic alongside cyclosporine A and prednisone, compared to the reference treatment of cyclosporine A and prednisone alone.
This clinical trial is a European, multi-center, randomized, double blinded placebo-controlled trial comparing CsA+PDN+MPA versus the reference treatment of CsA+PDN alone + placebo, in patients with extensive chronic GvHD. Randomization will be stratified according to: * Platelet number (low versus high risk) * Source of transplantable cells (marrow versus PBSC versus cord blood) Patients not in progression at 6 weeks post randomization (progression defined as primary failure) will be evaluated for remission (complete or partial) at 3, 6, 9, \& 12 months post randomization
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
34
1440mg twice daily
Prednisone and Cyclosporine given according to protocol. The drugs are tapered according to patient response
Hopital St. Louis
Paris, France
University Regensburg
Regensburg, Germany
Ospedale San Martino
Genova, Italy
University Hospital
Maastricht, Netherlands
To test whether the addition of Myfortic improves the efficacy of prednisone plus cyclosporine for treatment of newly diagnosed chronic GvHD, as defined by the proportion of patients with efficacy success at 1 year after enrollment.
Time frame: 1 year
The hazard rates of efficacy success between the two arms. Loss of donor chimerism or recurrent malignancy before secondary systemic therapy and before discontinuation of all immunosuppressive meds will be treated as competing risks.
Time frame: 1 year
efficacy failure, and treatment failure defined as efficacy failure or premature discontinuation of study-drug administration due to toxicity
Time frame: 1 year
survival without recurrent malignancy
Time frame: 1 year
Overall survival
Time frame: 1 year
cumulative incidence of secondary systemic treatment for cGvHD before recurrent malignancy
Time frame: 1 year
the cumulative incidence of death without recurrent or malignancy
Time frame: 1 year
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Hospital Clínico Universitario
Valencia, Spain
Karolinska University Hospital
Huddinge, Sweden
University Hospital
Basel, Switzerland
University Faculty of Medicine
Ankara, Turkey (Türkiye)