Autologous stem cell therapy has been shown to be effective in patients with systemic sclerosis. Nevertheless treatment is associated with treatment related mortality and patients die during follow up despite successful transplantation. Intention of this trial is to improve overall survival by modifying the existing protocol used for the ASTIS trial. To reduce treatment toxicity we reduce the dose of Cyclophosphamide (CYC) for mobilisation to 2x1g. Especially in patients with cardiac manifestations we also modify the conditioning regimen by adding thiotepa and reducing CYC; as CYC has known cardiotoxic side effects.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
44
If no active alveolitis: mobilisation with 2x1g Cyclophosphamide If active alveolitis: mobilisation with 2x1.5g Cyclophosphamid If cardiac manifestation: Conditioning with CYC 2 x 50mg + thiotepa 2x5mg + ATG If no cardiac manifestation: Conditioning with 4 x 50mg CYC + ATG
University Hospital Tuebingen; Department of oncology, hematology, rheumatology, immunology and pulmology
Tübingen, Germany
Efficay - Overall survival
Number of patients that are alive after 3 years
Time frame: 3 years
Safety - Treatment related mortality
Treatment related mortality: number of patients who die during the first 100 days after transplantation
Time frame: 100 days
Time to engraftment
Time in days from day 0 to platelet count \> 20.000 and granulocytes \>500/µl
Time frame: 2 months
Progression free survival
Time after transplantation without symptoms of disease activity
Time frame: 3 years
Efficacy - Lung function test and Skin
Number of patients that achieve either improvement of \>25% in mRSS or \> 10% in FVC or DLCO
Time frame: 3 years
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