Blood stem cells against systemic sclerosis: A study conducted between the United States and Canada gave encouraging results on a new treatment for the most severe forms of this disease, the transplantation of stem cells (i.e. hematopoietic, also known as ‘marrow’) from the patient himself (i.e. ”autologous”) to restore immune function. Among those who have been subjected to transplantation, the prognosis and survival rates have improved in the context of short-term side effects. However, research findings suggest that stem cell transplantation should also be considered as a potentially effective treatment for scleroderma, a topic that the scientific community will have to investigate further. We talk about it with Professor Carlo Selmi, Head of Rheumatology and Clinical Immunology at Humanitas and lecturer at the University of Milan.
Systemic sclerosis
Systemic sclerosis is a chronic autoimmune inflammatory disease of connective tissue that often affects the skin and is then referred to scleroderma. In the context of changes in the immune system, the skin and also the tissues of the internal organs (often the lungs), are affected by the formation of fibrosis. The pathology is very debilitating and has few effective treatments. Patients are given rheumatic and immunosuppressive drugs such as cyclophosphamide to relieve symptoms.
For this reason, international research is committed to identifying new treatments that can guarantee a better quality of life and a more favorable prognosis for patients affected by this disease.
Researchers at Duke University (United States) have tested the potential efficacy of new treatment characterized by stem cell transplantation associated with chemotherapy and radiotherapy. Previous studies had already experimented with this new approach. The prognosis improved but patients often experienced a resurgence of the disease. Therefore, conventional immunosuppressive drug therapy remained the standard form for its treatment.
The research
The authors of the study published in the New England Journal of Medicine involved seventy-five patients with systemic sclerosis who also had their kidneys and lungs affected. Thirty-six have been assigned to receive transplants, while thirty-nine have received a one-year treatment based on monthly injections of cyclophosphamide.
Hematopoietic stem cells, i.e. those that lead to the formation of blood cells, have been taken from patients in the first group. The patients were then subjected to chemotherapy and radiotherapy with total body irradiation in order to destroy the bone marrow. After that the stem cells, which were treated in a laboratory to eliminate the lymphocytes responsible for attacks on the internal organs, were re-infused in order to reconstruct the marrow and thus the immune system.
Four and a half years after treatment, survival in the first group improved. Parameters such as organ function, quality of life and fibrosis levels were better among patients who underwent a transplant. Among these, the overall survival rate was 86% and 51% among those who had received drug therapy. In addition, only 9% of transplants had to use rheumatic drugs to counteract the progression of systemic sclerosis against 44% of the control group. However, subjects in the first group had important side effects in the short term, such as the onset of infections, including two deaths associated with treatment. No association between mortality and treatment was found in the control group.
In conclusion, this treatment is promising but should be reserved for patients with very aggressive or advanced forms of the disease.
