Long-term evidence for a treatment used in severe disease
Autologous hematopoietic cell transplantation showed sustained disease control in a long-term international study of people with early severe systemic sclerosis. Researchers reported an estimated progression-free survival rate of 60% at 10 years after transplantation and an overall survival rate of 71%.
The results come from the NISSC1-LTE study, conducted by the Autoimmune Diseases Working Party of the European Society for Blood and Marrow Transplantation. Published in The Lancet Rheumatology, the prospective study followed 80 patients for a median of 9.2 years after their transplants.
Systemic sclerosis is a rare, chronic, and heterogeneous autoimmune disease marked by progressive fibrosis of the skin and internal organs. Severe forms can carry high morbidity and mortality. Autologous hematopoietic cell transplantation, also called HCT, is already an established option for carefully selected people with early diffuse severe disease and has grade A evidence from randomized clinical trials. What has been less available is evidence tracking outcomes close to a decade after treatment.
What the study measured
NISSC1-LTE extends the earlier prospective EBMT NISSC1 study. It included patients who received their first autologous HCT for severe progressive systemic sclerosis between 2012 and 2016. Follow-up data came from 13 participating centers in France, Germany, the Netherlands, Italy, Spain, Switzerland, and Brazil.
The 10-year progression-free survival estimate was 60%, with a 95% confidence interval of 47% to 71%. Overall survival at 10 years was estimated at 71%, with a 95% confidence interval of 56% to 82%. Those figures describe the study cohort’s longer-term outcomes after a treatment designed for carefully selected patients with aggressive disease.
At the last follow-up, 34 of the 80 participants, or 43%, were alive and had not needed additional immunosuppressive treatment. The finding is significant because it indicates that a substantial group remained alive without a subsequent need for that category of therapy at their most recent assessment. It does not mean every patient achieved that outcome, nor does it eliminate the risks that accompany transplantation.
Changes in skin and lung measures
The study also reported clinical measures relevant to systemic sclerosis. The median modified Rodnan skin score, a measure of skin involvement, fell from 24 at baseline to 6 at five years and 5 at 10 years. This substantial decline was observed across the long-term follow-up period in the reported cohort.
Lung measures were also tracked. Median forced vital capacity was 72% of predicted at baseline and 71% at 10 years. The near-similar values show that this measure was maintained at the cohort level over the reported period. The source also reports that the study assessed diffusing capacity of the lungs for carbon monoxide, another pulmonary measure, as part of its long-term outcomes.
These data matter because systemic sclerosis can affect internal organs as well as the skin. The study’s value lies not only in its survival estimates but in its extended observation of disease control and organ-related measures after transplantation.
Benefits must be considered alongside risk
The investigators present the work as evidence of sustained positive clinical outcomes and long-term safety, while emphasizing the treatment’s long-term risks. Autologous HCT is not a general treatment for every person with systemic sclerosis. The source describes it as an established option for carefully selected patients with early diffuse severe disease.
That distinction is important when interpreting the results. The study followed people treated at specialist centers after their first transplant for severe progressive disease. Its findings add prospective, international follow-up data to the existing evidence base, but they should not be read as a recommendation that transplantation is appropriate for all forms or stages of systemic sclerosis.
Why the follow-up matters
- It followed 80 patients for a median of 9.2 years after autologous HCT.
- Estimated progression-free survival was 60% at 10 years.
- Estimated overall survival was 71% at 10 years.
- Forty-three percent of participants were alive without further immunosuppressive treatment at last follow-up.
- Skin-score measures improved markedly from baseline through 10 years.
For a rare disease in which severe forms can progress and damage internal organs, decade-scale follow-up helps clarify how a high-intensity treatment performs beyond the time horizon of many clinical reports. NISSC1-LTE does not remove uncertainty for individual patients, but it provides a longer view of benefits and risks for a treatment already used in selected severe cases.
This article is based on reporting by Medical Xpress. Read the original article.
Originally published on medicalxpress.com
