Long-term follow-up of patients receiving hematopoietic stem cell transplantation (HSCT) for severe scleroderma has confirmed a durable survival benefit compared to conventional treatment with cyclophosphamide.
In the SCOT trial, 75 patients with severe scleroderma and internal organ involvement were randomised to either myeloablative CD34+ selected autologous HSCT or 12-monthly infusions of cyclophosphamide.
It found event-free survival (74% v 47%) and overall survival (86% v 51%) at 72 months favoured transplantation.
Now 6-11 years after randomisation, no new deaths have been identified among patients treated with stem cells compared to four new deaths among those treated with cyclophosphamide.
Overall survival was 80% for stem cell transplant recipients versus 52% for patients on the immunosuppressant.
Physical functioning, performance status and weight gain were also improved following transplantation. Organ failure developed in two transplant patients and six cyclophosphamide patients.
Additionally, 92% of stem cell transplant recipients versus 61% of patients treated with cyclophosphamide remained DMARDs-free.
Research lead Professor Keith Sullivan, from Duke University School of Medicine, told the meeting there was an urgent, unmet need to reduce the morbidity and mortality associated with severe scleroderma.
“This study now extends follow-up of the study to six to 11 years after randomisation. To show that the scleroderma has improved after transplant and results are durable for a decade off immunosuppressive drugs is a truly exciting development and a new approach to definitive treatment of autoimmune disease,” he said.
“With this clear demonstration of effectiveness and durability, our current research focus is to understand why it works.”
“Fundamental discoveries include demonstration that after hematopoietic stem cell transplantation, but not after conventional cyclophosphamide treatment, the genomic signatures of scleroderma resolve, suggesting establishment of a genomic new normal after autologous stem cell transplant.”