Around 25 years ago, we began to see studies of mouse models and case reports of successful autologous stem cell transplantation (aSCT) treatment of patients with autoimmune diseases with hematological malignancies as a comorbidity. As a result, aSCT was introduced into the treatment armamentarium of autoimmune diseases, and has been increasingly used since. As our understanding of autoimmune diseases grew, many alternative treatment options became available and aSCT was shown to be less effective in several inflammatory rheumatic diseases like rheumatoid arthritis and juvenile idiopathic arthritis. However, aSCT still constitutes a cornerstone in the treatment of progressive systemic sclerosis and other related connective tissue diseases due to the dismal prognosis and lack of efficacious alternative therapies. In systemic sclerosis, three randomized controlled trials have shown improved efficacy on skin and lung involvement, quality of life and survival.
Due to frequent cardiopulmonary disease manifestations and intensive immunoablative conditioning, treatment toxicity and risk of infections exceeded what was regarded as common in aSCT for hematological indications. Although successful efforts were made to reduce treatment related mortality – e.g. to roughly 6% in patients with systemic sclerosis - it still constitutes a relevant risk.
There are several questions remaining, requiring thorough research. These efforts are hampered by the rarity of appropriate disease subgroups, and the sheer effort and complexity of randomized aSCT studies in AID:
This Research Topic aims at answering these questions at least partly, to improve a potentially life-saving treatment modality, which still lacks proper alternatives in some disease subgroups. We welcome articles providing solutions related, but not limited to, the following sub-topics:
• How to optimize patient selection and screening? Evidence for CMR, PET-CT and fluid challenge to detect and monitor subclinical cardiopulmonary involvement? Should such patients be excluded?
• Should HSCT be used upfront in high-risk patients or rather as salvage therapy in those not responding to iv CYC and/or MMF.
• What have we learned from biomarker and immune reconstitution studies?
• Long-term outcomes after HSCT in SSc (versus conventional treatment)
• How can the success of mobilization regimes be increased?
• How can toxicity of immunoablative condition regimens be reduced without losing efficacy?
• Is CD34 selection or ATG application necessary at all?
• How can the considerable risk of infections be reduced?
• How do we treat relapses after aSCT, or do we need maintenance therapy?
• Do we really achieve an immunological reset, and how long does it last?
• What are the mechanisms of the development of secondary autoimmune diseases after aSCT?
Around 25 years ago, we began to see studies of mouse models and case reports of successful autologous stem cell transplantation (aSCT) treatment of patients with autoimmune diseases with hematological malignancies as a comorbidity. As a result, aSCT was introduced into the treatment armamentarium of autoimmune diseases, and has been increasingly used since. As our understanding of autoimmune diseases grew, many alternative treatment options became available and aSCT was shown to be less effective in several inflammatory rheumatic diseases like rheumatoid arthritis and juvenile idiopathic arthritis. However, aSCT still constitutes a cornerstone in the treatment of progressive systemic sclerosis and other related connective tissue diseases due to the dismal prognosis and lack of efficacious alternative therapies. In systemic sclerosis, three randomized controlled trials have shown improved efficacy on skin and lung involvement, quality of life and survival.
Due to frequent cardiopulmonary disease manifestations and intensive immunoablative conditioning, treatment toxicity and risk of infections exceeded what was regarded as common in aSCT for hematological indications. Although successful efforts were made to reduce treatment related mortality – e.g. to roughly 6% in patients with systemic sclerosis - it still constitutes a relevant risk.
There are several questions remaining, requiring thorough research. These efforts are hampered by the rarity of appropriate disease subgroups, and the sheer effort and complexity of randomized aSCT studies in AID:
This Research Topic aims at answering these questions at least partly, to improve a potentially life-saving treatment modality, which still lacks proper alternatives in some disease subgroups. We welcome articles providing solutions related, but not limited to, the following sub-topics:
• How to optimize patient selection and screening? Evidence for CMR, PET-CT and fluid challenge to detect and monitor subclinical cardiopulmonary involvement? Should such patients be excluded?
• Should HSCT be used upfront in high-risk patients or rather as salvage therapy in those not responding to iv CYC and/or MMF.
• What have we learned from biomarker and immune reconstitution studies?
• Long-term outcomes after HSCT in SSc (versus conventional treatment)
• How can the success of mobilization regimes be increased?
• How can toxicity of immunoablative condition regimens be reduced without losing efficacy?
• Is CD34 selection or ATG application necessary at all?
• How can the considerable risk of infections be reduced?
• How do we treat relapses after aSCT, or do we need maintenance therapy?
• Do we really achieve an immunological reset, and how long does it last?
• What are the mechanisms of the development of secondary autoimmune diseases after aSCT?