Stem Cell Transplant for Severe Scleroderma Improves Survival, Quality of Life

New clinical trial findings show that a therapeutic regimen involving transplantation of a person’s own blood-forming stem cells can improve survival and quality of life for people with severe scleroderma, a life-threatening autoimmune disease. 

The regimen, known as myeloablative autologous hematopoietic stem cell transplant (HSCT), includes chemotherapy and total body radiation to destroy the bone marrow followed by transplantation of the person’s own blood-forming stem cells to reconstitute the marrow and immune system. 

The study, funded by NIH, found myeloablative HSCT to be superior to treatment with the immune-suppressing drug cyclophosphamide.

The findings appeared in the Jan. 4 issue of the New England Journal of Medicine.

Scleroderma is characterized by hardening of the skin and connective tissues. Diffuse systemic sclerosis is a severe, often fatal form of the disease that also involves the internal organs. Treatment options are limited. People with the disease may take anti-rheumatic drugs and immune-suppressing drugs such as cyclophosphamide to help manage symptoms, but none of these medications has been proven to provide long-term benefit.

The clinical trial, called Scleroderma: Cyclophosphamide or Transplantation (SCOT), compared the safety and potential benefits of the 2 treatment regimens among 75 people with diffuse systemic sclerosis who had lung or kidney involvement.

Compared with cyclophosphamide, transplantation offered significantly greater long-term benefits, but also carried known short-term risks such as infections and low blood cell counts.

“We need effective therapies for scleroderma and other severe autoimmune diseases, which can be not only debilitating to the patient but also difficult to treat,” said NIAID director Dr. Anthony Fauci, whose institute sponsored the study. “These results add to the growing evidence that stem cell transplants should be considered as a potential treatment option for people with poor-prognosis scleroderma.”