Ten years after a stem cell transplant, an estimated 60% of patients in an international study were still alive without their systemic sclerosis getting worse. The result offers a rare long view of a treatment used for carefully selected people with an early, severe form of the disease—and a reminder that lasting benefits must be weighed against serious risks.
Systemic sclerosis is a chronic autoimmune disease that can cause scarring, or fibrosis, in the skin and internal organs. Severe disease can lead to major illness and early death. In an autologous hematopoietic cell transplant, doctors use a patient’s own blood-forming stem cells as part of treatment. The transplant is an established option for some patients, but evidence about outcomes many years later has been limited.
The prospective study, called NISSC1-LTE, followed 80 people who received their first transplant for severe, progressive systemic sclerosis between 2012 and 2016. Patients were treated at 13 centers in France, Germany, the Netherlands, Italy, Spain, Switzerland and Brazil. The median follow-up was 9.2 years. The study was conducted by the Autoimmune Diseases Working Party of EBMT, a European network for blood and marrow transplantation, and its results were published in The Lancet Rheumatology.
At 10 years, estimated progression-free survival was 60%. This measure counts patients who were alive without disease progression. Estimated overall survival was 71%. At their last follow-up, 34 patients—43% of the group—were alive and had not needed further treatment to suppress the immune system.
Several measures of health also changed little, or improved, over time. The median modified Rodnan skin score, which tracks skin thickening, fell from 24 before treatment to 6 at five years and 5 at 10 years. Median lung capacity, measured as forced vital capacity, was 72% of predicted before transplantation and 71% at 10 years. Another lung measure, the ability to transfer gas into the blood, was 59% of predicted at the start and 61% at 10 years.
The long follow-up also recorded substantial harms. Eighteen patients died: five early deaths, or 6% of the cohort, were attributed to the transplant; nine to disease progression; two to cancers after transplantation; and two to serious infections that occurred later. The estimated risk of death not caused by disease returning was 13% at 10 years. The authors said the findings point to the need for careful patient selection, specialist checks before treatment and ongoing monitoring at experienced centers.
Because the study had no comparison group, it cannot show how transplantation compares directly with other treatments. Some long-term records were incomplete, and 12 people could not be followed. Still, the results give patients and clinicians a clearer picture of what may lie years after treatment. The researchers say the evidence supports considering transplantation for selected patients, while longer follow-up remains important as new cell-based treatments are assessed.