In systemic sclerosis, 60% without progression over 10 years — News-Medical

The international NISSC1-LTE study showed that estimated progression-free survival 10 years after autologous hematopoietic cell transplantation was 60% among patients with early severe systemic sclerosis. Overall survival over this period reached 71%, News-Medical reports.

Follow-up lasted more than nine years

Researchers analyzed data from 80 patients who underwent autologous hematopoietic cell transplantation for the first time in 2012–2016. The median follow-up duration after the procedure was 9.2 years. The project involved 13 centers in France, Germany, the Netherlands, Italy, Spain, Switzerland, and Brazil.

At the time of the last follow-up, 34 of the 80 participants, or 43%, were alive and did not require further immunosuppressive therapy. The median skin involvement score on the modified Rodnan scale decreased from 24 points at the start of the study to six after five years and to five after 10 years.

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The risks of the procedure remain significant

Lung function indicators generally remained stable: median forced vital capacity was 72% of the predicted value before transplantation and 71% after 10 years. Lung diffusing capacity for carbon monoxide changed from 59% to 61% of the predicted value.

During follow-up, 18 patients died. Researchers attributed five early deaths, or 6% of the entire cohort, to transplantation; nine to disease progression, two to post-procedure malignancies, and another two to severe late infections. Estimated relapse-free mortality at 10 years was 13%.

The authors noted that the study was observational and did not include a control group for direct comparison with other treatment methods. Data for some participants were incomplete, and 12 patients were lost to follow-up. The results were published in The Lancet Rheumatology.