New findings show that autologous bone marrow transplantation, that is transplantation of the patient's own hematopoietic stem cells, can improve survival and quality of life in severe systemic sclerosis.
Systemic scleroderma is no more. This disease is characterized by hardening of the skin and connective tissue associated with diffuse fibrosis. Systemic diffuse scleroderma is a serious and often fatal form of the disease that also affects internal organs. The study appeared in the New England Journal of Medicine.
A new treatment
Treatment has progressed for many complications, but treatment options are limited for cutaneous and pulmonary fibrosis. Until a study funded by the National Institutes of Health, is interested in the subject. This treatment, known as autologous myoablative hematopoietic stem cell transplantation, involves the initial collection of the patient's stem cells, followed by high-dose chemotherapy and total body radiotherapy to destroy the bone marrow. Then comes the transplantation of these hematopoietic stem cells from the person to reconstitute the marrow and the immune system.
Autograft vs reference treatment
The randomized study compared bone marrow autograft (n = 36) with baseline treatment with cyclophosphamide (a monthly course of treatment for one year, n = 33) in 75 patients with severe diffuse systemic sclerosis. pulmonary and renal involvement During the year of the study, 7 patients died in the autograft group and 14 in the cyclophosphamide group, including 3 in each group who did not follow the protocol.
In the end, those who received the autograft had less progression of their disease: only 2 patients died because of the progression of their disease, against 11 in the treatment group with cyclophosphamide.
These results add to the growing evidence that stem cell transplants may be considered a potential treatment option for people with diffuse systemic sclerosis and poor prognosis.
A heavy process for severe forms
Participants in the study experienced side effects, such as infections. The most serious adverse events in transplant recipients occurred within the first 26 months after transplantation. Overall infection rates in both arms of study were similar
"Our results indicate that autologous stem cell transplantation for severe scleroderma poses more risk in the short term, but offers greater long-term gains than cyclophosphamide therapy," said the study's author. Keith M. Sullivan.
"We hope this work will help set a new standard of care for this serious and life-threatening autoimmune disease."