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cyclophosphamide + anti-thymocyte globulin + autologous hematopoietic stem cell transplantation + mycophenolate mofetil
- Development stage
- Unknown
- Lead developer
- Fred Hutchinson Cancer Center
- Modality
- Stem Cell Therapies → Cell Therapies, Antibody-Based Therapeutics, Small Molecules
- Administration
- Intravenous, Oral
01
Overview
This therapeutic regimen, specifically evaluated by the Fred Hutchinson Cancer Center in the STAT trial, is an intensive treatment for severe systemic sclerosis (SSc). The protocol utilizes high-dose immunoablation therapy (HDIT) consisting of high-dose cyclophosphamide and anti-thymocyte globulin (ATG) to deplete the patient's autoreactive immune system. This is followed by autologous hematopoietic stem cell transplantation (HCT) to restore hematopoiesis and promote the reconstitution of a more tolerant immune system. To maintain remission and prevent disease reactivation, patients receive mycophenolate mofetil (MMF) as post-transplant maintenance therapy. The goal of this multi-component approach is to halt the progression of skin and internal organ fibrosis in patients with diffuse cutaneous systemic sclerosis.
- Other names
- STAT trial regimenFred Hutch SSc transplant protocolHigh-dose immunoablation and autologous hematopoietic cell transplantation
02
Targets
IMPDH (Inosine-5'-monophosphate dehydrogenase 1)DNA
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