In a rare and relentless autoimmune illness, where the skin and internal organs slowly harden, a group of eighty patients with early, severe disease chose to stake their bodies on a stem cell transplant between 2012 and 2016. Ten years later, the record they leave behind is neither triumph nor failure but a careful accounting: most who survived kept their skin softer and their lungs holding steady, while a meaningful minority paid for that gain with their lives. Reported in The Lancet Rheumatology by researchers across seven countries, the study offers the longest international look yet at thi
Stem Cell Transplant Shows Durable 10-Year Control of Severe Systemic Sclerosis, With Ongoing Risks
Eighteen of 80 patients died during follow-up, including five early deaths attributed to the transplant procedure and nine to disease progression.
Thirty-four of 80 patients were alive and needed no further immunosuppression
Mark
Sixty percent progression-free at ten years sounds strong. Is that the main takeaway?
Mimi
It's the headline, but the skin score may say more to patients. The median fell from 24 to 5, and lung measures stayed essentially flat, 72% to 71% for forced vital capacity. In a disease defined by progressive fibrosis, holding steady matters.
Luke
Holding steady compared to what, though? There's no control group. The study can't say transplant beats other treatments.
Mark
So what can it say?
Luke
That in 80 selected patients at experienced centres, these were the outcomes over a median of 9.2 years. That's durability data, not a comparison.
Mimi
And the risks are laid out plainly. Five deaths, 6% of the cohort, were attributed to the transplant itself, and estimated non-relapse mortality reached 13% at ten years.
Mark
Nine deaths came from disease progression, too.
Mimi
Yes, so the disease still wins for some patients even after the procedure. Eighteen of 80 died in total.
Luke
Also keep in mind that 12 patients were lost to follow-up and some longitudinal data were incomplete. The confidence intervals are wide: 47 to 71 for progression-free survival.
Mark
Why does a ten-year reference matter now?
Mimi
The authors say it gives a benchmark for newer cellular therapies. Anything new will be measured against these numbers.
Luke
Which is useful only if the patients are comparable. The source doesn't say how newer therapies will be tested against this cohort, so that's still open.
El Pulso
- Eighty patients entered a decade-long wait, and eighteen did not return from the journey, leaving families and clinicians to hold grief beside hope.
- Five people died from the transplant itself, a reminder that the cure can carry its own danger into the body.
- Skin scores fell from a median of 24 to 5, and lung measures held steady, suggesting the disease was checked where it matters most.
- Careful patient selection and specialist monitoring are now being pressed as the path forward, with the data set as a benchmark for newer cellular therapies.
In a rare and relentless autoimmune illness, where the skin and internal organs slowly harden, a group of eighty patients with early, severe disease chose to stake their bodies on a stem cell transplant between 2012 and 2016. Ten years later, the record they leave behind is neither triumph nor failure but a careful accounting: most who survived kept their skin softer and their lungs holding steady, while a meaningful minority paid for that gain with their lives. Reported in The Lancet Rheumatology by researchers across seven countries, the study offers the longest international look yet at this treatment, though without a control group it can only chart the road, not prove the destination. It asks clinicians to weigh durable hope against real risk, and to choose their patients with the same care the procedure demands.
Eighty people with early, severe systemic sclerosis, a rare autoimmune disease that slowly stiffens the skin and organs, underwent a stem cell transplant between 2012 and 2016. Using their own cells, the procedure aims to reset a immune system that has turned against the body. Now, a decade later, a long-term study published in The Lancet Rheumatology has followed them closely.
The picture is mixed. At ten years, about 60% of patients were free of progression, and 71% were still alive. Skin scores fell sharply, from a median of 24 to 5, and lung function held steady. Those are the gains that matter to the people living with this disease.
The cost is also written into the record. Eighteen patients died during follow-up. Five died from complications of the transplant itself, and nine from the disease continuing to advance. Non-relapse mortality reached 13% at ten years, a figure the authors say demands careful selection of patients and experienced, structured follow-up.
The study cannot say whether transplantation is better than other treatments, since it had no control group and some data were incomplete. Its authors, led by Joerg Henes and Dominique Farge, present it as a long-term reference point: a measure of durable benefit, and of the risks that come with it.
Eighty patients with early, severe systemic sclerosis underwent a stem cell transplant between 2012 and 2016. A decade on, 34 of them (43%) were alive and had needed no further immunosuppressive treatment as of their last follow-up. That is one of the headline findings of a new long-term study published in The Lancet Rheumatology.
Systemic sclerosis is a rare, chronic autoimmune disease marked by progressive fibrosis of the skin and internal organs. Its severe forms carry high rates of illness and death. Autologous haematopoietic cell transplantation, in which a patient's own cells are used, is already an established option for carefully selected patients with early, diffuse, severe disease. It holds a grade A level of evidence, backed by randomised trials. What has been thin is evidence stretching toward a decade.
The study, called NISSC1-LTE, extends the earlier prospective EBMT NISSC1 study. It was run by the Autoimmune Diseases Working Party of the European Society for Blood and Marrow Transplantation (EBMT) and draws on 13 centres in France, Germany, the Netherlands, Italy, Spain, Switzerland and Brazil. The patients were followed for a median of 9.2 years after their first transplant. The authors describe it as the first international prospective study to report long-term results of this treatment in early severe systemic sclerosis.
At 10 years, estimated progression-free survival was 60% (95% CI 47–71) and overall survival was 71% (95% CI 56–82). The measures of disease also held steady or improved. The median modified Rodnan skin score, which gauges skin involvement, fell from 24 at baseline to 6 at five years and 5 at ten years. Median forced vital capacity was 72% of predicted at baseline and 71% at ten years. Median diffusing capacity of the lungs for carbon monoxide was 59% at baseline and 61% at ten years.
The cost is also recorded. Eighteen patients died during follow-up. Five early deaths, 6% of the cohort, were attributed to the transplant itself. Nine deaths were due to disease progression, two to malignancies after transplant, and two to late serious infections. Estimated non-relapse mortality at ten years was 13% (95% CI 6–24). The authors say this underlines the need for careful patient selection, specialist assessment before transplant, and structured long-term monitoring in experienced centres.
The study has limits. It is observational and has no control group, so it does not directly compare transplantation with other treatments. Some longitudinal data were incomplete, and 12 patients were lost to follow-up.
The lead authors are Joerg Henes of University Hospital Tübingen in Germany and Dominique Farge of St-Louis Hospital in Paris. They said the findings help clinicians understand how durable disease control is after the procedure while highlighting risks that need continued monitoring. They also credited years of collaboration among haematologists, rheumatologists and data specialists. Patient representatives helped shape the research question and the conduct of the study.
The results were first presented at the 2026 EBMT Annual Meeting. They support considering autologous transplantation in carefully selected patients, in line with existing EBMT, EULAR and CIBMTR recommendations. The authors also position the data as a long-term reference point for judging emerging cellular therapies.
Citas Notables
The findings help clinicians understand how durable disease control is after autologous HCT, while highlighting risks that require continued monitoring.— Joerg Henes and Dominique Farge, lead authors (paraphrased)
Quieres la nota completa?
Lee el original en News-Medical ↗
Health & Medicine